Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance...

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Literature Appraisal Literature Appraisal Effectiveness of Therapy Effectiveness of Therapy

Transcript of Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance...

Page 1: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

Literature AppraisalLiterature Appraisal

Effectiveness of TherapyEffectiveness of Therapy

Page 2: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

Measures of treatment effect

• Statistical significance• Odds ratio• Relative risk• Absolute risk reduction• Number needed to treat

Page 3: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

Measures of treatment effect

Outcome (death)

Yes No

Control a b

Experiment c d

Page 4: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200

Total

Page 5: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

Total in each group

Page 6: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25

Die

10

Total in each group

After 1 year

Page 7: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive)Total in each group

+

+

After 1 year

Page 8: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

Risk & Relative Risk

Page 9: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk? (a proportion)

What is the Total in each group

+

+

After 1 year

Page 10: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) RiskTotal in each group

+

+

After 1 year

Page 11: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) RiskTotal in each group

+

+

After 1 year

Page 12: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) RiskTotal in each group

+

+

After 1 year

Page 13: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.2525%

+

+

After 1 year

Page 14: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.25 25%

+

+

After 1 year

Page 15: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.25 25%

0.110%+

+

Page 16: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.25 25%

0.1 10%+

+

Page 17: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

Risk ratio or Relative risk (RR) =

+

+ 0.25 25%

0.1 10%

Page 18: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

Risk ratio or Relative risk (RR) =

+

+ 0.25 25%

0.1 10%

Page 19: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

Risk ratio or Relative risk (RR) =

2.5

+

+ 0.25 25%

0.1 10%

Page 20: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

Odds & Odds Ratio

Page 21: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

Odds

• The ratio between the amounts staked by parties in a bet, based on the expected probability either way.

• The balance of advantage or superiority.

Page 22: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.25

0.1

Odds?What about

+

+

Page 23: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

(90)

(Survive) Risk

0.25

0.1

Odds

10 +

+

Page 24: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

(90)

(Survive) Risk

0.25

0.1

Odds

10

1to3

+

+

Page 25: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.25

0.1

Odds

1to3

+

+

Page 26: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.25

0.1

Odds

1to3

1to9+

+

Page 27: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.25

0.1

Odds

1to3

1to9+

+

Page 28: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.25

0.1

Odds

1to3

1to9

Odds Ratio (O.R.) =

+

+

Page 29: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.25

0.1

Odds

1to3

1to9

Odds Ratio (O.R.) =

+

+

Page 30: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.25

0.1

Odds

1to3

1to9

Odds Ratio (O.R.) =

+

+

3

Page 31: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25 (75)

Die

10 (90)

(Survive) Risk

0.25

0.1

Odds

1to3

1to9

3

Odds Ratio (O.R.) =

+

+

Risk ratio or Relative risk (RR) =

2.5

Page 32: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

Measures of treatment effect influence clinicians decisions

• Clinicians:– more inclined to treat if the results are

presented as relative risk

– less inclined to treat if the results are presented as absolute risk reduction

Forrow et al. Am J Med 1992;92:121

Page 33: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

• Control group event rate (CER) = Deaths / Controls

• Experiment group event rate (EER)

= Deaths / Treated

Absolute risk reduction (difference)

ARR = CER - EER

Absolute Risk Reduction

Page 34: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25

Die

10

Risk

0.25 25%

0.110%

Absolute Risk Reduction

(ARR) =

CER

EER

Page 35: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25

Die

10

Risk

0.2525%

0.110%

Absolute Risk Reduction

(ARR) =0.15 15%

Page 36: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

Number Needed to Treat

NNT

Page 37: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

Clinical value of measures of treatment effect

Number Needed To Treat• The odds ratio etc. not easy to understand,

especially for patients.

• The number needed to treat (NNT) to prevent an adverse event is a more clinically relevant measure of the consequences of treatment

Sackett DL. EBM 1996; 1: 164-6

Sinclair JC. J Clin Epidemiol 1994; 47: 881-9

Page 38: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

Number Need to Treat (NNT)

Out of 100 patients treated 10 died compared to 25 in the placebo group

and 15 extra survived.

Therefore:

To get 1 more patient to survive, 6.7 (100/15) have to be treated.

Page 39: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

100/15

1/ 0.15

NNT = 1/ ARR

Page 40: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

200100

100

25

Die

10

Risk

0.2525%

0.110%

Absolute Risk Reduction (ARR) =

0.1515%

NNT=1/ARR=1/0.15=

6.7

Page 41: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

Q.E.D.

Page 42: Literature Appraisal Effectiveness of Therapy. Measures of treatment effect Statistical significance Odds ratio Relative risk Absolute risk reduction.

MAGPIEOf the patients treated (5015) 40 fitted compared to 96 in the placebo group (5055)

In % Mg 0.8% vs Placebo 1.9%

Therefore: ARR 1.8 – 0.8 = 1.1% (11 per 1000)

To get 1 more patient to survive, 91 (100/1.1) have to be treated. = NNT