Choosing an appropriate statistical test -...

28
Choosing Appropriate Descriptive Statistics, Graphs and Statistical Tests Brian Yuen 15 January 2013

Transcript of Choosing an appropriate statistical test -...

Choosing Appropriate

Descriptive Statistics

Graphs and Statistical Tests

Brian Yuen 15 January 2013

Slide - 2

2

Using appropriate statistics and

graphs

bull Report statistics and graphs depends on the types of variables of

interest

ndash For continuous (Normally distributed) variables

bull N mean standard deviation minimum maximum

bull histograms dot plots box plots scatter plots

ndash For continuous (skewed) variables

bull N median lower quartile upper quartile minimum maximum

geometric mean

bull histograms dot plots box plots scatter plots

ndash For categorical variables

bull frequency counts percentages

bull one-way tables two-way tables

bull bar charts

Bar chart

Clustered bar charts (two categorical variables)

Bar charts with error bars

Histogram (can be plotted against a

categorical variable)

Box amp Whisker plot (can be plotted against

a categorical variable)

Dot plot (can be plotted against a

categorical variable)

Scatter plot (two continuous variables)

Mean

Median

Standard deviation

Range (Min Max)

Inter-quartile range (LQ UQ)

Flow chart of commonly used

descriptive statistics and

graphical illustrations

Frequency

Percentage (Row Column or Total)

Exploring data

Descriptive statistics

Graphical illustrations

Categorical data

Continuous data Measure of location

Continuous data Measure of variation

Categorical data

Continuous data

Slide - 4

Choosing appropriate statistical test

bull Having a well-defined hypothesis helps to distinguish the

outcome variable and the exposure variable

bull Answer the following questions to decide which statistical test is

appropriate to analysis your data

ndash What is the variable type for the outcome variable

bull Continuous (Normal Skew) Binary Time dependent

bull If more than one outcomes are they paired or related

ndash What is the variable type for the main exposure variable

bull Categorical (1 group 2 groups gt2 groups) Continuous

bull For 2 or gt2 groups Independent (Unrelated) Paired

(Related)

ndash Any other covariates confounding factors

4

5

Continuous

Categorical Outcome

variable

Normal Skew

Survival

1 group

2 groups

gt2 groups

Paired

Sign test Signed rank test

Mann-Whitney U test

Wilcoxon signed rank test

Kruskal Wallis test

1 group

2 groups

gt2 groups

Paired

Chi-square test Exact test

Chi-square test Fisherrsquos exact test Logistic regression

McNemarrsquos test Kappa statistic

Chi-square test Fisherrsquos exact test Logistic regression

2 groups

gt2 groups

KM plot with Log-rank test

KM plot with Log-rank test

Continuous

Continuous

Continuous

Spearman Corr Linear Reg

Logistic regression Sensitivity amp specificity ROC

Cox regression

Two-sample t test

Paired t test

One-way ANOVA test

Pearson Corr Linear Reg

One-sample t test

Exposure

variable

Flow chart of

commonly used

statistical tests

Case Studies

Slide - 7

7

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 8

8

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 9

9

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes

bull Outcomes amp type

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 10

10

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 11

11

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes

bull Outcomes amp type

bull Choice of test

ndash

ndash

bull Note ndash

Note ndash

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 2

2

Using appropriate statistics and

graphs

bull Report statistics and graphs depends on the types of variables of

interest

ndash For continuous (Normally distributed) variables

bull N mean standard deviation minimum maximum

bull histograms dot plots box plots scatter plots

ndash For continuous (skewed) variables

bull N median lower quartile upper quartile minimum maximum

geometric mean

bull histograms dot plots box plots scatter plots

ndash For categorical variables

bull frequency counts percentages

bull one-way tables two-way tables

bull bar charts

Bar chart

Clustered bar charts (two categorical variables)

Bar charts with error bars

Histogram (can be plotted against a

categorical variable)

Box amp Whisker plot (can be plotted against

a categorical variable)

Dot plot (can be plotted against a

categorical variable)

Scatter plot (two continuous variables)

Mean

Median

Standard deviation

Range (Min Max)

Inter-quartile range (LQ UQ)

Flow chart of commonly used

descriptive statistics and

graphical illustrations

Frequency

Percentage (Row Column or Total)

Exploring data

Descriptive statistics

Graphical illustrations

Categorical data

Continuous data Measure of location

Continuous data Measure of variation

Categorical data

Continuous data

Slide - 4

Choosing appropriate statistical test

bull Having a well-defined hypothesis helps to distinguish the

outcome variable and the exposure variable

bull Answer the following questions to decide which statistical test is

appropriate to analysis your data

ndash What is the variable type for the outcome variable

bull Continuous (Normal Skew) Binary Time dependent

bull If more than one outcomes are they paired or related

ndash What is the variable type for the main exposure variable

bull Categorical (1 group 2 groups gt2 groups) Continuous

bull For 2 or gt2 groups Independent (Unrelated) Paired

(Related)

ndash Any other covariates confounding factors

4

5

Continuous

Categorical Outcome

variable

Normal Skew

Survival

1 group

2 groups

gt2 groups

Paired

Sign test Signed rank test

Mann-Whitney U test

Wilcoxon signed rank test

Kruskal Wallis test

1 group

2 groups

gt2 groups

Paired

Chi-square test Exact test

Chi-square test Fisherrsquos exact test Logistic regression

McNemarrsquos test Kappa statistic

Chi-square test Fisherrsquos exact test Logistic regression

2 groups

gt2 groups

KM plot with Log-rank test

KM plot with Log-rank test

Continuous

Continuous

Continuous

Spearman Corr Linear Reg

Logistic regression Sensitivity amp specificity ROC

Cox regression

Two-sample t test

Paired t test

One-way ANOVA test

Pearson Corr Linear Reg

One-sample t test

Exposure

variable

Flow chart of

commonly used

statistical tests

Case Studies

Slide - 7

7

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 8

8

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 9

9

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes

bull Outcomes amp type

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 10

10

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 11

11

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes

bull Outcomes amp type

bull Choice of test

ndash

ndash

bull Note ndash

Note ndash

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Bar chart

Clustered bar charts (two categorical variables)

Bar charts with error bars

Histogram (can be plotted against a

categorical variable)

Box amp Whisker plot (can be plotted against

a categorical variable)

Dot plot (can be plotted against a

categorical variable)

Scatter plot (two continuous variables)

Mean

Median

Standard deviation

Range (Min Max)

Inter-quartile range (LQ UQ)

Flow chart of commonly used

descriptive statistics and

graphical illustrations

Frequency

Percentage (Row Column or Total)

Exploring data

Descriptive statistics

Graphical illustrations

Categorical data

Continuous data Measure of location

Continuous data Measure of variation

Categorical data

Continuous data

Slide - 4

Choosing appropriate statistical test

bull Having a well-defined hypothesis helps to distinguish the

outcome variable and the exposure variable

bull Answer the following questions to decide which statistical test is

appropriate to analysis your data

ndash What is the variable type for the outcome variable

bull Continuous (Normal Skew) Binary Time dependent

bull If more than one outcomes are they paired or related

ndash What is the variable type for the main exposure variable

bull Categorical (1 group 2 groups gt2 groups) Continuous

bull For 2 or gt2 groups Independent (Unrelated) Paired

(Related)

ndash Any other covariates confounding factors

4

5

Continuous

Categorical Outcome

variable

Normal Skew

Survival

1 group

2 groups

gt2 groups

Paired

Sign test Signed rank test

Mann-Whitney U test

Wilcoxon signed rank test

Kruskal Wallis test

1 group

2 groups

gt2 groups

Paired

Chi-square test Exact test

Chi-square test Fisherrsquos exact test Logistic regression

McNemarrsquos test Kappa statistic

Chi-square test Fisherrsquos exact test Logistic regression

2 groups

gt2 groups

KM plot with Log-rank test

KM plot with Log-rank test

Continuous

Continuous

Continuous

Spearman Corr Linear Reg

Logistic regression Sensitivity amp specificity ROC

Cox regression

Two-sample t test

Paired t test

One-way ANOVA test

Pearson Corr Linear Reg

One-sample t test

Exposure

variable

Flow chart of

commonly used

statistical tests

Case Studies

Slide - 7

7

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 8

8

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 9

9

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes

bull Outcomes amp type

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 10

10

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 11

11

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes

bull Outcomes amp type

bull Choice of test

ndash

ndash

bull Note ndash

Note ndash

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 4

Choosing appropriate statistical test

bull Having a well-defined hypothesis helps to distinguish the

outcome variable and the exposure variable

bull Answer the following questions to decide which statistical test is

appropriate to analysis your data

ndash What is the variable type for the outcome variable

bull Continuous (Normal Skew) Binary Time dependent

bull If more than one outcomes are they paired or related

ndash What is the variable type for the main exposure variable

bull Categorical (1 group 2 groups gt2 groups) Continuous

bull For 2 or gt2 groups Independent (Unrelated) Paired

(Related)

ndash Any other covariates confounding factors

4

5

Continuous

Categorical Outcome

variable

Normal Skew

Survival

1 group

2 groups

gt2 groups

Paired

Sign test Signed rank test

Mann-Whitney U test

Wilcoxon signed rank test

Kruskal Wallis test

1 group

2 groups

gt2 groups

Paired

Chi-square test Exact test

Chi-square test Fisherrsquos exact test Logistic regression

McNemarrsquos test Kappa statistic

Chi-square test Fisherrsquos exact test Logistic regression

2 groups

gt2 groups

KM plot with Log-rank test

KM plot with Log-rank test

Continuous

Continuous

Continuous

Spearman Corr Linear Reg

Logistic regression Sensitivity amp specificity ROC

Cox regression

Two-sample t test

Paired t test

One-way ANOVA test

Pearson Corr Linear Reg

One-sample t test

Exposure

variable

Flow chart of

commonly used

statistical tests

Case Studies

Slide - 7

7

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 8

8

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 9

9

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes

bull Outcomes amp type

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 10

10

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 11

11

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes

bull Outcomes amp type

bull Choice of test

ndash

ndash

bull Note ndash

Note ndash

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

5

Continuous

Categorical Outcome

variable

Normal Skew

Survival

1 group

2 groups

gt2 groups

Paired

Sign test Signed rank test

Mann-Whitney U test

Wilcoxon signed rank test

Kruskal Wallis test

1 group

2 groups

gt2 groups

Paired

Chi-square test Exact test

Chi-square test Fisherrsquos exact test Logistic regression

McNemarrsquos test Kappa statistic

Chi-square test Fisherrsquos exact test Logistic regression

2 groups

gt2 groups

KM plot with Log-rank test

KM plot with Log-rank test

Continuous

Continuous

Continuous

Spearman Corr Linear Reg

Logistic regression Sensitivity amp specificity ROC

Cox regression

Two-sample t test

Paired t test

One-way ANOVA test

Pearson Corr Linear Reg

One-sample t test

Exposure

variable

Flow chart of

commonly used

statistical tests

Case Studies

Slide - 7

7

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 8

8

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 9

9

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes

bull Outcomes amp type

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 10

10

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 11

11

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes

bull Outcomes amp type

bull Choice of test

ndash

ndash

bull Note ndash

Note ndash

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Case Studies

Slide - 7

7

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 8

8

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 9

9

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes

bull Outcomes amp type

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 10

10

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 11

11

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes

bull Outcomes amp type

bull Choice of test

ndash

ndash

bull Note ndash

Note ndash

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 7

7

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 8

8

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 9

9

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes

bull Outcomes amp type

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 10

10

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 11

11

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes

bull Outcomes amp type

bull Choice of test

ndash

ndash

bull Note ndash

Note ndash

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 8

8

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 9

9

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes

bull Outcomes amp type

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 10

10

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 11

11

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes

bull Outcomes amp type

bull Choice of test

ndash

ndash

bull Note ndash

Note ndash

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 9

9

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes

bull Outcomes amp type

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 10

10

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 11

11

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes

bull Outcomes amp type

bull Choice of test

ndash

ndash

bull Note ndash

Note ndash

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 10

10

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type

bull Exposure amp type

bull If the continuous outcome is

ndash Normally distributed

ndash Not Normally distributed

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 11

11

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes

bull Outcomes amp type

bull Choice of test

ndash

ndash

bull Note ndash

Note ndash

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 11

11

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes

bull Outcomes amp type

bull Choice of test

ndash

ndash

bull Note ndash

Note ndash

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 12

12

When the continuous outcome is not

normally distributed

bull If outcome normally distributed use t-tests ANOVA

ndash easy to obtain confidence interval for differences

bull So far wersquove recommended using non-parametric tests when data not normal

ndash often less powerful

ndash non-parametric confidence intervals problematic

bull Recall another possibility ndash take logs (natural log) of the outcome

ndash check to see if outcome looks normal after logging

ndash can then use t-tests ANOVA

ndash estimate of the difference and its confidence interval on log scale easily

available

ndash back transform to get estimate of percent change between groups

ndash back transform confidence interval

ndash better to analyse on log scale if data become normally distributed than to

use non-parametric test

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 13

13

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

BINARY DATA

Case Study 7

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 14

14

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

ndash

bull Note ndash

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 15

15

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash If any of the variables is Normally distributed

ndash If both variables are not Normally distributed

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 16

16

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type

bull Exposure amp type

bull Choice of test

ndash

bull Note ndash

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 17

17

References

bull Altman DG Practical Statistics for Medical Research Chapman and Hall 1991

bull Kirkwood BR amp Sterne JAC Essential Medical Statistics 2nd Edition Oxford Blackwell

Science Ltd 2003

bull Bland M An Introduction to Medical Statistics 3rd Edition Oxford Oxford Medical

Publications 2000

bull Altman DG Machin D Bryant TN amp Gardner MJ Statistics with Confidence 2nd Edition

BMJ Books 2000

bull Campbell MJ amp Machin D Medical Statistics A Commonsense Approach 3rd Edition 1999

bull Field A Discovering Statistics Using SPSS for Windows 2nd edition London Sage

Publications 2005

bull Bland JM Altman DG (1986) Statistical methods for assessing agreement between two

methods of clinical measurement Lancet i 307-310

bull Mathews JNS Altman DG Campbell MJ Royston P (1990) Analysis of serial measurements in

medical research British Medical Journal 300 230-235

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 18

18

Other web and software resources

bull UCLA ndash What statistical analysis should I use

ndash httpwwwatsuclaedustatmult_pkgwhatstatdefaulthtm

bull DISCUS

ndash Discovering Important Statistical Concepts Using Spreadsheets

ndash Interactive spreadsheets designed for teaching statistics

ndash Web-sites for download and information -

httpwwwcoventryacukecresearchdiscusdiscus_homehtml

bull Choosing the correct statistical test

ndash httpbamauaedu~jleeper627choosestathtml

bull SPSS for Windows

ndash Help

ndash Statistics Coach

bull Statistics for the Terrified

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Solutions to

Case Studies

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 20

20

bull A simple study investigating

ndash the fitness level of our locally selected group of healthy volunteers

ndash with the published average value on fitness level which was done previously

on the national level

ndash fitness level was measured by the length of time walking on a treadmill

before stopping through tiredness

bull Objective any difference between the group average and the published value

bull Outcome amp type fitness level (length of time) ndash continuous

bull Exposure amp type one group only

bull If the continuous outcome is

ndash Normally distributed One-sample t test

ndash Not Normally distributed Sign test Signed rank test

vs

CONTINUOUS amp ORDINAL DATA

Case Study 1

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 21

21

bull A clinical trial investigating

ndash the effect of two physiotherapy treatments (standard and enhanced

exercise) for patients with a broken leg

ndash on their fitness level (length of time walking on a treadmill before stopping

through tiredness)

bull Objective any difference between the 2 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash binary independent

(or unrelated)

bull If the continuous outcome is

ndash Normally distributed Two-sample t test

ndash Not Normally distributed Mann-Whitney U test

CONTINUOUS amp ORDINAL DATA

Case Study 2

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 22

22

bull Now each patient performs the walking test before and after

enhanced physiotherapy treatment

ndash data might be presented as two variables one as before data and

the other as after data but the values for individual patients are

paired

bull Objective any difference between the before and the after averages

bull Number of outcomes 2 (before and after)

bull Outcomes amp type fitness level ndash continuous

paired (or related)

bull If the difference in outcomes (eg after - before) is

ndash Normally distributed Paired t test

ndash Not Normally distributed Wilcoxon signed rank test

CONTINUOUS amp ORDINAL DATA

Case Study 3

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 23

23

bull Based on Case Study 2 (standard vs enhanced exercises) but now

with a control group

ndash ie patients without a broken leg

bull Objective any difference among the 3 group averages

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type treatment group ndash categorical (more

than two levels) independent (or unrelated)

bull If the continuous outcome is

ndash Normally distributed One-way ANOVA test

ndash Not Normally distributed Kruskal-Wallis test

CONTINUOUS amp ORDINAL DATA

Case Study 4

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 24

24

bull Before the participants started their fitness test their blood pressure (BP) was recorded

by two different machines

ndash machine 1 was the lsquogold standardrsquo

ndash machine 2 was newly made and claimed to be more accurate

ndash aim to validate the measurements recorded from machine 2 by assessing the level of

agreement with that obtained from machine 1

bull Objective any agreement between measuring tools

bull Number of outcomes 2 (machines)

bull Outcomes amp type blood pressure ndash continuous paired

(or related)

bull Choice of test

ndash Bland-Altman method (amp Paired t-test)

bull Note ndash the Bland-Altman method is not a statistical test

Note ndash see the Bland and Altman paper for details

CONTINUOUS amp ORDINAL DATA

Case Study 6

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 25

25

bull Fitness is now assessed only as Unfit Fit

ndash could be as a result of dichotomising the previous continuous outcome (0-5

minutes = Unfit gt5 minutes = Fit)

ndash investigate whether the proportions of Unfit and Fit are equal (ie 50 each)

after the standard treatment

ndash or compare the proportions to specific values (eg 10 Fit 90 Unfit)

bull Objective any difference in proportion within the group

(or any difference from the specific proportions)

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type one group only

bull Choice of test

ndash Chi-square test (large sample size)

ndash Exact test (small sample size)

Unfit Fit

Standard

BINARY DATA

Case Study 7

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 26

26

Unfit Fit

Standard

Enhanced

BINARY DATA

Case Study 8

bull Similar setting as Case Study 2 but with the binary outcome defined from

Case Study 7 (Unfit Fit)

ndash to find out if the enhanced treatment is better than the standard treatment

ie more patients into the Fit category

bull Objective any difference in proportion between the groups

bull Outcome amp type fitness level category ndash binary

bull Exposure amp type treatment groups ndash binary independent (or unrelated)

bull Choice of test

ndash Chi-square test (large sample size)

ndash Fisherrsquos exact test (small sample size)

bull Note ndash same tests for more than 2 groups

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 27

27

CONTINUOUS amp ORDINAL DATA

Case Study 12

bull Now in the physiotherapy trial we wanted to investigate

ndash if there was any relationship between the participantsrsquo fitness level and their age at

assessment

ndash we suspected that age at assessment affected their fitness level regardless of the

treatment group they were in

ndash quantify the relationship by the direction strength and magnitude

bull Objective assess and quantify the relationship between two variables

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash If any of the variables is Normally distributed

Pearson correlation

ndash If both variables are not Normally distributed

Spearmanrsquos rank correlation

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates

Slide - 28

28

CONTINUOUS amp ORDINAL DATA

Case Study 13

bull We now found in Case Study 12 that age at assignment had some linear

relationship with participantsrsquo fitness level

ndash needed to quantify this relationship ie what is the average fitness level at

different age at assignment

ndash also wanted to predict fitness level for future patients given their age at

assignment

bull Objective set up a statistical model to quantify the effect of exposure variable

on the outcome variable

bull Outcome amp type fitness level ndash continuous

bull Exposure amp type age at assessment ndash continuous

bull Choice of test

ndash (Simple) Linear regression

bull Note ndash Linear regression is also appropriate when the exposure variable is

categorical eg exercise treatment group (standard amp enhanced) as well as

controlling for other covariates