Hematopoietic cell transplantation for aplastic anemia

Area of Science:

• Hematology
• Immunology
• Oncology

Background:

• Severe aplastic anemia (SAA) management has evolved significantly.
• Allogeneic hematopoietic cell transplantation (HCT) is increasingly considered an earlier treatment option.
• Advances in donor selection, conditioning, and graft-versus-host disease (GVHD) prophylaxis enhance HCT safety and efficacy.

Purpose of the Study:

• To review recent advancements in allogeneic HCT for SAA.
• To discuss the integration of HCT earlier in the SAA treatment algorithm.
• To address challenges and adaptations for HCT in developing countries.

Main Methods:

• Review of current literature on SAA treatment and HCT.
• Analysis of recent findings on clonality, mutations, and telomere disorders in SAA.
• Evaluation of eltrombopag combined with immunosuppressive therapy (IST).

Main Results:

• Eltrombopag plus IST shows superior results compared to historical IST.
• Matched unrelated-donor (MUD) and haploidentical HCT (haplo-HCT) offer survival rates comparable to sibling transplants in younger patients.
• Guidelines need adaptation for cost-effectiveness in resource-limited countries.
• Bone marrow is the preferred graft source; peripheral blood stem cells are an alternative in specific contexts.

Conclusions:

• Novel findings and therapeutic trials require validation across diverse populations and settings.
• Personalized care strategies are crucial for optimizing SAA patient outcomes.
• HCT can be effectively implemented earlier in SAA management, with considerations for global accessibility.