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

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Allogeneic stem cell transplants offer a cure for bone marrow failure syndromes like aplastic anemia. Advances in 2026 now expand this treatment to more patients, including those without matched sibling donors.

Keywords:
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Area of Science:

  • Hematology
  • Transplantation Immunology
  • Oncology

Background:

  • Bone marrow failure syndromes, including acquired aplastic anemia (AA), lead to severe cytopenia, increasing morbidity and mortality.
  • Allogeneic hematopoietic stem cell transplantation (alloHSCT) is a potentially curative option for AA, enabling rapid hematopoietic reconstitution.

Purpose of the Study:

  • To review contemporary evidence on the expanded role of alloHSCT in bone marrow failure syndromes.
  • To provide a framework for decision-making regarding alternative donor transplantation.

Main Methods:

  • Review of current scientific literature and clinical evidence.
  • Analysis of advancements in reduced-toxicity conditioning and graft-versus-host disease prevention.

Main Results:

  • Reduced-toxicity conditioning and post-transplant cyclophosphamide (PTCy)-based platforms have made alternative donor transplantation mainstream.
  • Transplantation is increasingly utilized beyond younger patients with matched sibling donors.

Conclusions:

  • Contemporary alloHSCT strategies have broadened the applicability of this curative therapy for bone marrow failure syndromes.
  • A pragmatic, context-adapted approach is essential for optimal decision-making in expanding transplant indications.