Registries are cornerstone sources of data in rare diseases like immune thrombocytopenia. The European Research Consortium on ITP (ERCI) defined some priority unmet needs as regards knowledge about epidemiology and pharmacoepidemiology of ITP in order to improve patient care. This project provides an overview of existing registries in Europe (both clinical registries and population-based cohorts) and is meant to propose a harmonization of existing registries and to response the need of a European registry on ITP to analyze the discrepancies in treatment availability, increase the number of informative patients for specific investigations like the assessment of risks of rare events and of real world use, effectiveness and safety of newly marketed ITP drugs, and to facilitate the selection of patients in clinical trials.
The project is divided in three steps:
1) Record of available registries and description of their structure (clinical registries versus cohorts built in health insurance databases) and content (inclusion criteria, date of start, categories of variables recorded, time of data collection during the follow-up, availability of biological collections and of quality of life/patient reported outcomes). This step has been achieved in 2022 (see publication at: https://pubmed.ncbi.nlm.nih.gov/35303315/). Given the existence of major sources of high-quality data, the results of this first step reinforced the project to harmonize existing registries instead of building a new, concurrent European registry, which would be much more time-consuming and expensive.
2) Organization of meetings with registry coordinators to achieve an in-depth review of recorded variables in each registry (locations in the database structure, tables, labels, codes).
3) Redaction of common extraction models to extract similar data from each registry and transform the variables using the same coding system.
The ERCI group is currently searching funds to achieve steps 2-3.
The project is coordinated by ERCI founding member Guillaume Moulis, from the Department of Internal Medicine, Toulouse University Hospital, France.
Update in November 2023
After having described the registry sources (https://pubmed.ncbi.nlm.nih.gov/35303315/), ERCI launched a first feasibility study assessing the percentage of adult patients with primary ITP necessitating a 2nd (at least) line of treatment, and describing these lines. All European prospective registries gathering these data are participating: France, Germany, Italy, Norway, Switzerland and United Kingdom. The analyses will be performed in parallel in each country using the same protocol and statistical analysis plan. Aggregated results will be share for a common publication.
The next step will be to work on individual data linkage, with both regulatory and technical (data harmonization) tasks.