Allogeneic HSCT in Aplastic Anemia: Current Evidence, Controversies, and Practical Decision-Making

Am J Hematol. 2026 Jul;101(7):1561-1574. doi: 10.1002/ajh.70319. Epub 2026 Apr 13.

Abstract

Morbidity and mortality in bone marrow failure syndromes such as acquired aplastic anemia (AA) are driven by severe and prolonged cytopenia. Allogeneic hematopoietic stem cell transplantation (alloHSCT) is potentially curative and offers a rapid route to hematopoietic reconstitution. In 2026, reduced-toxicity conditioning, improved supportive care, and novel graft versus host disease prevention through post-transplant cyclophosphamide (PTCy)-based platforms have brought alternative donor transplantation into the mainstream. This review examines contemporary evidence, particularly looking at the expanded role of transplant outside the widely accepted setting of younger patients with matched sibling donors and offers a pragmatic framework for context-adapted decision-making.

Keywords: alternative donors; aplastic anemia; haploidentical donors; stem cell transplantation.

Publication types

  • Review

MeSH terms

  • Anemia, Aplastic* / therapy
  • Clinical Decision-Making
  • Cyclophosphamide / therapeutic use
  • Graft vs Host Disease / prevention & control
  • Hematopoietic Stem Cell Transplantation* / adverse effects
  • Hematopoietic Stem Cell Transplantation* / methods
  • Humans
  • Transplantation Conditioning / methods
  • Transplantation, Homologous

Substances

  • Cyclophosphamide