Unrelated cord blood transplantation in patients with acquired refractory aplastic anemia: a nationwide phase II study.
Fiche publication
Date publication
mai 2018
Journal
Blood
Auteurs
Membres identifiés du Cancéropôle Est :
Pr RUBIO Marie Thérèse
Tous les auteurs :
Peffault de Latour R, Chevret S, Jubert C, Sirvent A, Galambrun C, Ruggeri A, Gandemer V, Cornillon J, Rialland F, Dalle JH, Forcade E, Bruno B, Paillard C, Rorlich PS, Salmon A, Fürst S, Sicre de Fontbrune F, Rubio MT, Bay JO, Mohty M, Larghero J, Gluckman E, Socié G
Lien Pubmed
Résumé
Outcomes remain poor for patients with refractory severe aplastic anemia (SAA). Alternative donor transplantation may be considered, but results from previous studies have not been encouraging. We conducted a prospective nationwide phase II study to assess the efficacy and safety of unrelated cord blood transplantation (CBT) in patients with refractory SAA (APCORD protocol, NCT 01343953). To demonstrate a significant difference in one-year survival from 20% (null hypothesis) to 50% (alternative hypothesis), we needed to include 25 transplanted patients. Twenty-six patients (median age: 16 years) were therefore included. Eligibility criteria required one or two unrelated CB units, containing separately or together more than 4x10 frozen nucleated cells/kg recipient body weight. The conditioning regimen comprised fludarabine, cyclophosphamide, thymoglobulin and 2-Gy total body irradiation. With a median follow-up of 38.8 months, engraftment occurred in 23 patients (88%); cumulative incidences of grade II-IV acute and chronic GvHD were 45.8% and 36%, respectively. Twenty-three patients were alive at one year, with an overall survival rate of 88.5%, differing significantly from the expected 20% (p<0.0001) (84% overall survival at 2 years). CBT with units containing at least 4x10 frozen nucleated cells/kg is therefore a valuable curative option for young adults with refractory SAA and no available matched unrelated donors.
Référence
Blood. 2018 May 14;: