Long‐term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial

Le résumé fourni par la source

To the Editor: HLA-matched sibling donor transplantation accounts for the majority of transplants performed for sickle cell disease (SCD). However, only about 18% of patients in the United States with SCD will have unaffected human leukocyte antigen (HLA)-matched siblings or one with sickle trait, limiting applicability. A Blood and Marrow Transplant Clinical Trials Network phase II trial of HLA-matched unrelated donor bone marrow transplantation (URD BMT) for severe SCD was conducted between 2008 and 2014 and enrolled patients aged 3–19 years (BMT CTN 0601, NCT00745420).1 A reduced intensity immunosuppressive conditioning regimen (RIC) of alemtuzumab (45 mg; days −22 to −19), fludarabine (150 mg/m2; day −8 to −4) and melphalan (140 mg/m2; day −3) was employed using alemtuzumab early to provide recipient immune suppression to overcome a higher risk of graft rejection (GR) with URD BMT in SCD patients while also limiting toxicities associated with myeloablative agents.1 The trial met a pre-specified primary endpoint of 75% 1-year event-free survival (EFS). However, as previously reported, the incidence of 1-year acute and extensive chronic graft-versus-host disease (GVHD) were unacceptably high at 17% and 38% respectively. This report details long-term outcomes in this previously reported cohort as an important consideration for therapeutic trials as follow-up is usually limited to early time-points.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.