Haplo-identical or mismatched unrelated donor hematopoietic cell transplantation for Fanconi anemia: results from the Severe Aplastic Anemia Working Party of the EBMT.

Allogeneic hematopoietic cell transplantation (HCT) is the only curative option for bone marrow failure or hematopoietic malignant diseases for Fanconi anemia (FA) patients. Although results have improved over the last decades, reaching more than 90% survival when a human leukocyte antigen (HLA)-identical donor is available, alternative HCT donors are still less reported. We compared HCT outcomes using HLA-mismatched unrelated donors (MMUD; n=123) or haplo-identical donors (HDs), either using only in vivo T cell depletion (n=33) or T cells depleted in-vivo with some type of graft manipulation ex-vivo (n=59) performed for FA between 2000 and 2018. Overall survival (OS) by 24 months was 62% (53-71%) for MMUD, versus 80% (66-95%) for HDs with only in vivo T cell depletion and 60% (47-73%) for HDs with in vivo and ex vivo T cell depletion (p 0.22). Event free survival (EFS) was better for HD-transplanted FA patients with only in vivo T cell depletion 86% (73-99%) than for those transplanted from a MMUD 58% (48-68%) or those with graft manipulation 56% (42-69%) (p= 0.046). Grade II-IV acute graft-versus-host disease (GVHD) was 41% (MMUD) versus 40% (HDs with no graft manipulation) versus 17% (HDs with T cell depleted graft), (p=0.005). No differences were found for the other transplant related outcomes. These data suggest that HDs might be considered as an alternative option for FA patients with better EFS using unmanipulated grafts. This article is protected by copyright. All rights reserved.

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Authors: Josune Zubicaray, Daria Pagliara, Julian Sevilla, Dirk-Jan Eikema, Paul Bosman, Mouhab Ayas, Marco Zecca, Akif Yesilipek, Savas Kansoy, Cécile Renard, Jean H Dalle, Antonio Campos, Maura Faraci, Alphan Kupesiz, Frans J W Smiers, Andrea Velardi, Manuel Abecasis, Paola Corti, Franca Fagioli, Soledad González Muñiz, Gergely Kriván, Carlo Dufour, Antonio Risitano, Selim Corbacioglu, Régis Peffault de Latour