Full Dose Cyclophosphamide with the Addition of Fludarabine for Matched Sibling Transplants in Severe Aplastic Anemia

The recommended therapy for severe aplastic anemia (SAA) in younger patients with a matched sibling donor (MSD) is allogeneic hematopoietic cell transplantation (allo-HCT). A number of conditioning regimens and protocols have been used for these patients. Here we report a homogeneous cohort of SAA patients receiving a uniform transplantation protocol. This study is a retrospective analysis of 82 consecutive patients with SAA who underwent MSD allo-HCT at a single center. The median duration of follow-up for survivors was 100 months, the 10year overall survival (OS) was 87.5%, and the 10-year event-free survival was 75.3%. The OS was 97.4% for mobilized bone marrow (BM) graft recipients and 78.9% for nonmobilized BM graft recipients (P =.01. The cumulative incidence of acute graft-versus-host disease (GVHD) was 25.6%, that of chronic GVHD was 27.16%, and that of graft failure was 16.2%. Recipient age >= 30 years and transplantation at >6 months after SAA diagnosis were associated with a increased risk of events. In the presence of a fully matched sibling donor, allo-HCT with a mobilized BM graft and fludarabine-cyclophosphamide conditioning is an efficacious and safe approach. Early transplantation is associated with a better outcome, emphasizing the importance of not delaying transplantation in these patients. Prospective trials are needed to determine the optimal regimen. (C) 2021 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.

Automated summary

This study is a retrospective analysis of 82 consecutive patients with severe aplastic anemia who underwent allogeneic hematopoietic cell transplantation with a matched sibling donor. The results show that using mobilized bone marrow grafts and fludarabine-cyclophosphamide conditioning is an effective and safe approach for treating these patients.