Purpose of review
Survival outcomes from haematopoietic stem cell transplantation (HSCT) in severe aplastic anaemia (SAA) have improved steadily over the past decades, largely reflecting progress in supportive care and conditioning regimens. Here we review recently published data that highlight the improvements and current issues.
Human leukocyte antigen (HLA)-matched sibling donor (MSD) HSCT remains the gold standard for SAA patients younger than 40–50 years, with HLA-matched unrelated donor (MUD) HSCT for second line after failure to respond to immunosuppressive therapy (IST). The use of alternative donor sources for aplastic anaemia patients remains limited and problematic, but novel conditioning regimens, particularly in the haploidentical setting, justify further evaluation. In recent studies when comparing alemtuzumab-based conditioning with standard antithymocyte globulin conditioning regimens, lower rates of acute and chronic graft-versus-host disease and better tolerance in older patients are seen.
Improving outcomes may lead to an expanded frontline HSCT role in the future. In children lacking a MSD, increasingly MUD HSCT is being considered as first-line treatment and is also being considered more for young adults. Further research is needed to advance our understanding of the role HSCT has to play in SAA with particular emphasis on alternative donor sources and identifying optimal conditioning regimens.