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

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.