Information about Project 17: Registries

Objectives

• Establishing a close cooperation with all major leukemia study groups in Europe in order to create a platform, which allows for performing efficiently the tasks of this WP.
• To create core data sets for the different leukemia disease entities CML, AML, ALL, CLL and MDS and CMPD. Such lists and definitions of variables, which should be collected in a uniform and standardized manner within all European therapeutic studies, are essential for trans-European research.
• To provide the biometric expertise for the proper collection and analysis of the data of the European leukemia registries as proposed in the various WPs, e.g. CML.
• To pool individual patient data from the various European leukemia study groups in order to develop and validate prognostic models (scores) for the different leukemia disease entities and to perform meta-analyses (overviews).

Combating cancer is a major priority of the 6th Framework Programme. This workpackage will contribute essentially to combat leukemia more efficiently by facilitating transeuropean research and thus speeding up the process of validating and disseminating therapeutic advancements. Especially the core data sets and the development and validation of prognostic factors and meta-analyses will contribute essentially to the development of evidence-based guidelines and accelerate the translation of existing results into applications. The work package will support clinical research, particularly registries and clinical trials, aimed at validating new and improved interventions. Thus, there is no doubt that this workpackage is highly relevant to the objectives of the LifeSciHealth Priorities.

Description of work

The European biometric platform will have a high integrating potential. A starting point of the integration process is the development of core data sets. A core data set is a list of variables, endpoints and definitions, which are agreed upon and will be used in all therapeutic research in a uniform manner. Core data sets are essential for a methodologically sound valid comparative interpretation of research results and meta-analyses, which form an important ground for evidence-based medicine and guidelines. To achieve this, consensus meetings involving experts from almost all work packages and representatives from all major therapeutic research groups in Europe have to be organized.

Similarly, the development of prognostic models needs a very close integration of this WP with the leukemia disease entities WPs. This objective cannot be achieved by biometricians working isolated on their own. Once a prognostic model has been properly validated, close integration with 'WP guidelines' and the 'central services' is asked for to ensure and enforce implementation. The spectre of integration activities include personal meetings, expert workshops and seminars and all types of telecommunications. Major interim results and achievements will be made accessible in the Intranet of the NOE to allow all members of the NOE to comment on.

The first jointly executed research activity focuses on the development of core data sets for the different leukemia disease entities.

A core data set is a list of variables, endpoints and definitions, which are agreed upon and will be used in all therapeutic research in a uniform manner. Core data sets specify the minimum of data, which should be collected from each patient. Of course, each study group has the right to collect more data, e.g. with regard to their particular research priorities, but the uniformly defined and collected (time points etc.) data will subsequently allow for a methodologically sound, valid comparative interpretation of study results and overviews, which form an important basis for evidence-based medicine and guidelines.

The final core data set will specify the variables, which should be collected at baseline and during follow up, the time points for collection and if appropriate the diagnostics used, and the endpoints including definitions. A consensus process has to be initiated and the core data sets will be updated integrating new biomedical evidence. An important issue for getting the core data sets accepted by the scientific community is that all major research groups are involved in this cooperative effort. Thus consensus meetings involving experts from all major study groups and from almost all WPs of this NOE will be organized.

The core data sets are essential for all those WPs, e.g. CML, which plan to establish a trans-European registry, as it provides the content and structure of the data bank. Registries require a careful definition of its objectives, of the eligibility criteria, the geographic representation and the like. In addition, logistics and quality control procedures have to be developed and implemented. In close collaboration with all relevant WPs, WP17 will ensure that the registries will be run in agreement with the current methodological state of the art.

In the beginning, a detailed research plan has to be written, which specifies in detail crucial points like: the objectives, the inception cohort, the eligibility criteria for studies and patients, the definition of outcome variables, the selection of learning and validation samples, quality control, and the statistical analyses. This research plan has to be agreed upon by all those involved. Then the collection of the individual patient data starts, followed by quality control procedures and finally the statistical analyses.

The development of prognostic models needs a very close integration of this WP17 with the leukemia disease entities WPs, especially clinical and diagnostic experts. This will be achieved by organizing workshops and a highly intensive use of internet-based communication facilities. Once a prognostic model has been properly validated, close integration with the PW Guidelines and the Central Services will ensure implementation. Basically the same procedures will be followed for meta-analyses.

The first step is the establishment of a close cooperation with all leukemia study groups in Europe in order to create a platform which allows for performing efficiently the tasks of this WP. Identification of responsible contact persons and establishing efficient means of communication are essential steps at this stage. The second step, whose results will be particularly relevant for other WPs too is the development of uniform core data sets, i.e. a list of variables, endpoints and definitions which will be used in all registries and other therapeutic research. To achieve these core data sets consensus meetings involving experts from almost all WPs and representatives from all major leukemia study groups in Europe have to be organized. While this is performed there will be a close communication with all those WPs, which plan for running registries and planning clinical trials so that the core data sets will be immediately used once they have been approved and accepted as standard. It is evident that the objectives 1, 2, and 3 deserve some priority compared to objective 4, although the activities to collect individual patient data from the various European leukemia study groups can be started almost without delay. Then, these data have to be quality controlled. Having done that the data will be pooled in order to develop and validate prognostic models for the various leukemia disease entities and to perform meta-analyses.