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Design of the DREPAGREFFE trial: A prospective controlled multicenter study evaluating the benefit of genoidentical hematopoietic stem cell transplantation over chronic transfusion in sickle cell anemia children detected to be at risk of stroke by transcranial Doppler (NCT 01340404)

Sylvie Chevret 1, 2 Suzanne Verlhac 3, 4, 2 Elisabeth Ducros-Miralles 3, 5 Jean-Hugues Dalle 4, 2 Regis Peffault de Latour 1, 2 Mariane de Montalembert 6, 7 Malika Benkerrou 4, 7 Corinne Pondarré 8, 3, 5 Isabelle Thuret 9, 10 Corinne Guitton 11, 12 Emmanuelle Lesprit 13, 2 Maryse Etienne-Julan 14, 15 Gisèle Elana 16, 14 Jean-Pierre Vannier 17, 18 Patrick Lutz 19, 20 Bénédicte Neven 6, 7 Claire Galambrun 9, 10 Catherine Paillard 19, 20 Camille Runel 21, 22 Charlotte Jubert 21, 22 Cécile Arnaud 3, 5 Annie Kamdem 3, 5 Valentine Brousse 6, 7 Florence Missud 4, 2 Marie Petras 14, 15 Lydia Doumdo-Divialle 14, 15 Claire Berger 23, 24 Françoise Fréard 3, 5 Olivier Taieb 25, 26 Elise Drain 25, 26 Monique Elmaleh 4, 2 Manuela Vasile 4, 2 Yacine Khelif 27 Myriam Bernaudin 27 Philippe Chadebech 28, 5, 29 France Pirenne 28, 5, 29 Gérard Socié 1, 2 Françoise Bernaudin 3, 5
Abstract : Background: Children with sickle cell anemia (SCA) have an 11% risk of stroke by the age of 18. Chronic transfusion applied in patients detected to be at risk by transcranial Doppler allows a significant reduction of stroke risk. However, chronic transfusion exposes to several adverse events, including alloimmunization and iron overload, and is not curative. Hematopoietic stem cell transplantation allows termination of the transfusion program, but its benefit has not been demonstrated. Design: DREPAGREFFE (NCT01340404) is a multicenter, prospective trial enrolling SCA children younger than 15years receiving chronic transfusion due to a history of abnormal transcranial Doppler (velocities ≥200cm/s). Only those with at least one non-SCA sibling and parents accepting HLA-typing and transplantation with a genoidentical donor were eligible. Chronic transfusion was pursued in patients with no available donor, whereas others were transplanted. Comparison between the 2 arms (transfusion vs transplantation) was analyzed using both genetic randomization and propensity-score matching as a sensitivity analysis. The primary end-point was the velocity measure at 1year. Secondary endpoints were the incidence of stroke, silent cerebral infarcts and stenoses, cognitive performance in comparison with siblings, allo-immunization, iron-overload, phosphatidyl-serine, angiogenesis/hypoxia, brain injury-related factor expression, quality of life and cost. Objectives: To show that genoidentical transplantation decreases velocities significantly more than chronic transfusion in SCA children at risk of stroke. Discussion: DREPAGREFFE is the first prospective study to evaluate transplantation in SCA children. It compares the outcome of cerebral vasculopathy following genoidentical transplantation versus chronic transfusion using genetic randomization and causal inference methods.
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https://www.hal.inserm.fr/inserm-03238783
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Submitted on : Thursday, May 27, 2021 - 11:26:06 AM
Last modification on : Friday, June 4, 2021 - 11:24:21 AM

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Sylvie Chevret, Suzanne Verlhac, Elisabeth Ducros-Miralles, Jean-Hugues Dalle, Regis Peffault de Latour, et al.. Design of the DREPAGREFFE trial: A prospective controlled multicenter study evaluating the benefit of genoidentical hematopoietic stem cell transplantation over chronic transfusion in sickle cell anemia children detected to be at risk of stroke by transcranial Doppler (NCT 01340404). Contemporary Clinical Trials, Elsevier, 2017, 62, pp.91-104. ⟨10.1016/j.cct.2017.08.008⟩. ⟨inserm-03238783⟩

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