학술논문
T-cell depleted allogeneic hematopoietic stem cell transplant for the treatment of Fanconi anemia and MDS/AML
Document Type
Original Paper
Author
Satty, Alexandra M.; Klein, Elizabeth; Mauguen, Audrey; Kunvarjee, Binni; Boelens, Jaap Jan; Cancio, Maria; Curran, Kevin J.; Kernan, Nancy A.; Prockop, Susan E.; Scaradavou, Andromachi; Spitzer, Barbara; Tamari, Roni; Ruggiero, Julianne; Torok-Castanza, Joanne; Mehta, Parinda A.; O’Reilly, Richard J.; Boulad, Farid
Source
Bone Marrow Transplantation: Official journal of the European Society for Blood and Marrow Transplantation. 59(1):23-33
Subject
Language
English
ISSN
0268-3369
1476-5365
1476-5365
Abstract
The only curative approach for myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) arising in patients with Fanconi anemia (FA) is allogeneic hematopoietic stem cell transplantation (HCT); however, HCT approaches are inconsistent and limited data on outcomes exist. We retrospectively evaluated outcomes of thirty patients with FA and MDS/AML who underwent first allogeneic HCT with a T-cell depleted (TCD) graft at our institution. Patients were transplanted on successive protocols with stepwise changes in cytoreduction and GVHD prophylaxis. All but two patients (93%) experienced durable hematopoietic engraftment. With median follow-up of 8.7 years, 5-year OS was 66.8% and DFS 53.8%. No significant differences in survival were found in patients with high-risk prognostic features (age ≥20 years, AML diagnosis, alternative donor graft) or when stratified by conditioning regimen. The 5-year cumulative incidences of relapse and NRM were 24.3% and 21.9%, respectively. NRM was higher in patients ≥20 years at HCT but did not otherwise differ. We herein demonstrate promising outcomes following allogeneic HCT for patients with FA and MDS/AML using TCD grafts, particularly in a cohort of high-risk patients with 50% ≥20 years and a majority receiving mismatched grafts. Future prospective studies are needed to compare this approach with other HCT platforms.