1. Celebrating the registration of 9.000 patients treated with CAR T cells in the EBMT registry: Collection of real-world data in the context of hematopoietic cellular therapies.
- Author
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Chabannon C, Ruggeri A, Montoto S, van Biezen A, van der Werf S, Markslag A, Sanchez-Ortega I, Camara R, Ljungman P, Mohty M, Kröger N, Sureda A, McGrath E, Bonini C, and Kuball J
- Subjects
- Humans, Europe, Hematopoietic Stem Cell Transplantation, Receptors, Chimeric Antigen therapeutic use, Registries, Immunotherapy, Adoptive methods
- Abstract
The European society for Blood and Marrow Transplantation (EBMT) has a long-standing interest in the evaluation of hematopoietic cell transplantation. More than three decades ago, its members established a continental registry. Today, more than 700,000 patients have been registered, and information has been gathered on more than 800,000 transplants. This huge amount of information has allowed conducting multiple retrospective studies, evaluating changes in practices over time and for different categories of diseases, benchmarking outcome across EBMT affiliated centers, and increasingly serves to build synthetic comparators to evaluate the introduction of therapeutic innovations in the field of hematology. CAR-T cells therapies draw on human and technical resources that are also used to deliver HCT; they elicit side effects that require the implementation of risk mitigation plans; they are living drugs that persist in the body of the recipient and thus deserve prolonged follow-up; the introduction of CAR-T cells in the pharmacopeia is likely to significantly impact on the practice of BMT; for all these reasons and even before the first approvals of CAR-T Cells in Europe, EBMT engaged in a project aiming at complementing the EBMT Registry with a Cellular Therapy Form, with the objective to register CAR-T cells treated patients and collect information on their short-, middle- and long-term outcome. The goal is to provide EBMT investigators with a tool for primary analyses of the collected information and to support secondary use of data transferred at the individual level to Marketing Authorization Holders and other interested parties, to fulfill their obligations to health authorities and further evaluate the actual medical values of CAR-T Cells in different contexts and indications. The EBMT Registry received a positive opinion from the European Medicines agency in 2019, and five years later contains information on more than 9.000 treated patients. This article describes the journey to start this new activity, lessons to be drawn in view of improving the collection of real-world data, and what existing information tells us in terms of patient access., Competing Interests: Declaration of competing interest AvB, SvdW, AM, ISO, EMG are or have been permanent and paid employees with EBMT. CC, AR, SM, RdlC, PL, MM, NK, AS, CB & JK volunteer part of their professional time to contribute to EBMT missions and tasks, in different and temporary positions. In their different capacities, all co-authors have interacted with Pharma companies (including Novartis, Kite/Gilead, BMS, Janssen) that develop and commercialize CAR-T Cells and other advanced therapies to establish the European CAR-T Cells Registry described in this manuscript, and launch PASS and PAES. Interactions with Pharma companies as well as with other public or private, not-for profit or for-profit organizations is ongoing in the context of the GoCART Coalition. JK received funding from Novartis, Miltenyi Biotech, is a cofounder of Gadeta as well as shareholder of floow-up companies of Gadeta such as Century Therapeutics and Adeta., (Copyright © 2024 The Authors. Published by Elsevier Ltd.. All rights reserved.)
- Published
- 2024
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