Proposals for an EU MDS Registry Prof Carlo Bernasconi Dr David Bowen.
Transcript of Proposals for an EU MDS Registry Prof Carlo Bernasconi Dr David Bowen.
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Proposals for an EU MDS Registry
Prof Carlo BernasconiDr David Bowen
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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
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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.
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• Voluntary – identification of new variants
• Governmental– If quality controlled, will add to
population-based datasets
Goals national registry datasets
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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
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Questionnaire results
• Questionnaire formatted by WP 2– Dr Nicola Gökbuget
• 33 questionnaire’s emailed– 22 responses
• 21 evaluable (1 Paediatric centre)
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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
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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
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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
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Registries
Issues raised• Definition of MDS
– Consistent morphological diagnosis
• Quality of follow-up
• Legal / ownership issues for transferred data
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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
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Other thoughts
• Interface with International MDS Registry
• later
• Involvement of “new” EU countries