In 2007 an International Working Group (IWG) of ITP experts came together to propose a standardized terminology on ITP, with the aim of establishing a common language that would have benefit research, knowledge sharing and ultimately patients. This effort resulted in the publication in 2009 of the paper “Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group”, which for the first time proposed the adoption of the term “Immune Thrombocytopenia” (ITP) and defined relevant outcomes and important aspects of the disease and its management.

The introduction of innovative drugs, in particular of TPO receptor agonists, and a better knowledge of ITP pathophysiology profoundly modified the clinical practice in ITP over the last 15 years and has made necessary an update of some of the previous definitions and terms, as well as the establishment of consensus on novel concepts.

The goal of this project is to produce a new standardization document, updated to the current knowledge and practice standards, using a modified Delphi method.

A Steering Committee (SC), composed by all ERCI founding members plus 5 major international ITP experts, has been formed and has started working on updated and novel definitions. The SC will soon produce a first draft of definitions and highlight areas of agreement and disagreement on different concepts.

Subsequently, a large international group of hematologists with expertise in ITP from North and South America, Europe, Asia, Africa and Australia, as well as patients advocates and representatives of EMA and FDA will be involved. The definitions drafted by the SC will finally be submitted to this large group of people in the form of a Delphi questionnaire to be further reviewed and improved with the aim of reaching a full consensus.

The project is coordinated by ERCI founding members and Co-chairs Prof. Francesco Rodeghiero (Hematology Project Foundation, Vicenza, Italy) and Prof. Cindy Neunert (Columbia University Medical Center, New York, US).

This is a multicenter, retrospective, European study to evaluate the standards of use of thrombopoietin receptor agonists (TPO-RA) in adult patients with primary immune thrombocytopenia (ITP). Data will be collected through a retrospective chart review of patients with ITP who started TPO-RA treatment between January 2014 and December 2018.

Each of the 18 designated sites in the UK, Spain, Italy, Norway, France and Switzerland will collect data from patients with ITP who meet the inclusion criteria and agree to participate. The aim of the study is to provide a realistic and broad view of the use of TPO-RA in Europe at a time when so many new molecules are being tested in this disease, without being biased by the clinical practice of any particular center. The information to be analyzed will include patient or disease characteristics in the selection of TPO-RA, long-term efficacy and safety data of these agents, and treatment-free responses, among others.

This project was initiated in 2022 and will be completed in the middle of next year, when data from approximately 350 patients will have been collected. Currently, centers from the 6 European countries are actively recruiting and results of the study will be published by the end of 2024.

The project is coordinated by ERCI founding member Maria Luisa Lozano, from the Hospital Universitario Morales Meseguer, Universidad de Murcia, Spain, and is conducted in collaboration with the Hematology Project Foundation, Vicenza, Italy.

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 2^nd (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.