Proposals for an EU MDS Registry

Prof Carlo BernasconiDr David Bowen

Goals population-based datasets

• Demographic epidemiological study• gender differences • temporal changes • identification of rare, previously unrecognised disease

entities.

• Molecular epidemiological / pathological study

• easy identification of samples from selected subtypes of MDS available in Biobanks

• Derivation / validation of new prognostic scoring systems

• based on unselected patient cohorts with high quality follow up data

Goals clinical trial dataset

• Improved definition of response predictors

• by combining data from several studies of the same drugs (including meta-analyses)

• Comparison of patient selection within clinical trials to the “real world” of population-based demographics

• interaction with Pharma.

• Voluntary – identification of new variants

• Governmental– If quality controlled, will add to

population-based datasets

Goals national registry datasets

European MDS Registry

National/Referral Centre RegistryPopulation-based Registry

Clinical Trials Registry

Set up: months 1-12

Set up: months 12-24 Set up: months 12-24

WP 3, 17

Interaction months 12-60

Questionnaire results

• Questionnaire formatted by WP 2– Dr Nicola Gökbuget

• 33 questionnaire’s emailed– 22 responses

• 21 evaluable (1 Paediatric centre)

Clinical Trials entry

Local HR

Local LR

National HR

National LR

International HR

International LR

No. of centres recruiting 1 pt

9 17 13 13 14 9

No of patients

343 603 406 241 246 151

Median 50 34 18 6 18 5

Range 1-100 1-150 2-100 1-100 1-63 1-100

Registries

• Local or regional– 15/21 centres– 8/15 sought consent– IT platform

• Access (5), SQL (3), SPSS (2), Excel (2), Web (1)

• National– 6 countries, all voluntary

Registries

• Centres consenting for EBMT– 14/20

• Will seeking consent for an MDS registry be a problem– 15/17 = no.

• Prepared to register patients via Web– 21/21

• Prepared to contribute data to an MDS registry– 21/21

Registries

Issues raised• Definition of MDS

– Consistent morphological diagnosis

• Quality of follow-up

• Legal / ownership issues for transferred data

Action

• Central registry set-up• University of Dundee Health Informatics Centre• Funding required

• Clinical trials• Form subgroup

• Population-based registries– Further data to be sought from the 14 centres

• Quality of follow up• Comprehensive diagnosis / coverage of area• Morphology review

• National registries• later

Other thoughts

• Interface with International MDS Registry

• later

• Involvement of “new” EU countries