Hematopoietic stem cell transplantation for acquired aplastic anemia

Area of Science:

• Hematology
• Transplantation Medicine
• Immunology

Background:

• Severe aplastic anemia (SAA) treatment has seen significant outcome improvements.
• Advances in conditioning regimens, hematopoietic cell sources, and supportive care have driven progress.

Purpose of the Study:

• To review recent data on improvements in SAA treatment outcomes.
• To highlight current challenges and advancements in allogeneic bone marrow transplantation (BMT) for SAA.

Main Methods:

• Review of recently published data on SAA treatment.
• Analysis of outcomes and current issues in BMT for SAA.

Main Results:

• Approximately one-third of SAA patients on immune suppressive therapy (IST) develop myeloid cancer gene mutations.
• Human leukocyte antigen (HLA)-matched sibling donor BMT is the preferred first-line treatment for SAA.
• HLA-matched unrelated donor (URD) BMT is a suitable first-line option for younger patients and a second-line option after IST failure.
• HLA-haploidentical BMT with posttransplant cyclophosphamide is a reasonable second-line treatment.

Conclusions:

• BMT is increasingly utilized as a first-line therapy for SAA due to improved outcomes.
• Bone marrow remains the optimal cell source from an HLA-matched donor.
• Further research is required to establish optimal conditioning regimens for HLA-haploidentical donors.