[Allogeneic hematopoietic stem cell transplantation for aplastic anemia]

Area of Science:

• Hematology
• Immunology
• Transplantation

Background:

• Aplastic anemia treatment has improved with eltrombopag and immunosuppressive therapy (IST).
• However, a significant portion of patients remain refractory or relapse, necessitating hematopoietic stem cell transplantation (HSCT).
• Current HSCT indications vary based on age, donor availability, and disease severity (e.g., fulminant aplastic anemia).

Purpose of the Study:

• To review current transplant indications for aplastic anemia.
• To discuss donor selection strategies, including alternatives when HLA-matched donors are unavailable.
• To evaluate the role of haploidentical transplantation with post-transplantation cyclophosphamide (PTCY) in managing aplastic anemia.

Main Methods:

• Review of existing literature on aplastic anemia treatment and HSCT.
• Analysis of transplant outcomes based on donor type and conditioning regimens.
• Discussion of recent advancements in haploidentical transplantation protocols.

Main Results:

• Eltrombopag combined with IST improves initial response rates in aplastic anemia.
• Allogeneic HSCT is a primary option for young patients with matched sibling donors.
• Fulminant aplastic anemia warrants immediate transplantation irrespective of donor type.
• Haploidentical transplantation with PTCY demonstrates favorable outcomes and engraftment rates.

Conclusions:

• Optimizing HSCT strategies is crucial for aplastic anemia patients resistant to initial therapies.
• Haploidentical HSCT with PTCY represents a viable alternative for patients without HLA-matched donors.
• Careful consideration of donor type and conditioning regimens is essential for successful aplastic anemia transplantation.