Journal: American journal of hematology
This review article focuses on contemporary use of allogeneic hematopoietic stem cell transplantation (alloHSCT) for acquired aplastic anemia (AA), emphasizing how evolving transplant strategies have broadened eligibility beyond the traditional setting of younger patients with matched sibling donors.
Key points:
- Clinical problem: In AA, morbidity and mortality are largely driven by persistent severe cytopenias. While immunosuppressive therapy is effective for many, it is not curative for all, and relapses or clonal evolution can occur.
- Rationale for alloHSCT: AlloHSCT remains the only reliably curative option and provides the most rapid route to hematopoietic recovery, addressing the immediate risks from profound cytopenias.
- Modern transplant advances (as of 2026):
- Reduced-toxicity conditioning regimens have lowered treatment-related morbidity and mortality, making transplant more feasible for older and/or more comorbid patients.
- Supportive care improvements (infection prophylaxis, transfusion support, monitoring) have improved overall transplant outcomes.
- Post-transplant cyclophosphamide (PTCy)-based graft-versus-host disease (GVHD) prophylaxis has enabled safer use of alternative donors, particularly haploidentical and mismatched unrelated donors.
- Expanded donor options: With PTCy and better supportive care, alternative donor alloHSCT has moved into the mainstream for AA, mitigating the historical reliance on matched sibling donors and allowing transplant consideration for a broader patient population.
- Decision-making framework: The review proposes a pragmatic, context-adapted approach to selecting alloHSCT versus non-transplant therapy, integrating:
- Patient factors (age, performance status, comorbidities)
- Disease factors (severity and duration of cytopenias, response to prior therapy)
- Donor availability and type
- Institutional expertise and resource setting
Overall, the article synthesizes current evidence to support more individualized, risk-adapted use of alloHSCT in AA, with particular attention to how reduced-toxicity regimens and PTCy-based strategies have reshaped the role of alternative donor transplantation.