Proposals for an EU MDS Registry

1
Proposals for an EU MDS Registry

• Prof Carlo Bernasconi
• Dr David Bowen

2
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

3
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.

4
Goals national registry datasets

• Voluntary
• identification of new variants
• Governmental
• If quality controlled, will add to
  population-based datasets

5
Set up months 1-12
Clinical Trials Registry
European MDS Registry
National/Referral Centre Registry
Population-based Registry
Set up months 12-24
Set up months 12-24
WP 3, 17
Interaction months 12-60
6
Questionnaire results

• Questionnaire formatted by WP 2
• Dr Nicola Gökbuget
• 33 questionnaires emailed
• 22 responses
• 21 evaluable (1 Paediatric centre)

7
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
8
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

9
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

10
Registries

• Issues raised
• Definition of MDS
• Consistent morphological diagnosis
• Quality of follow-up
• Legal / ownership issues for transferred data

11
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

12
Other thoughts

• Interface with International MDS Registry
• later
• Involvement of new EU countries