Comparable long-term outcomes between upfront haploidentical and identical sibling donor transplant in aplastic anemia: a national registry-based study

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains a curative option for severe aplastic anemia (SAA), and transplantation from identical sibling donors (ISD) has been recommended as a first-line treatment. Haploidentical donor (HID) transplantation for SAA has made great advances; thus, an increased role of HID-SCT in SAA should be considered. We performed a national registry-based analysis comparing long-term outcomes in the upfront HID or upfront ISD SCT setting. A total of 342 SAA patients were enrolled, with 183 patients receiving HID SCT and 159 receiving ISD SCT. The estimated 9-year overall survival and failure-free survival were 87.1 +/- 2.5% and 89.3 +/- 3.7% (P=0.173) and 86.5 +/- 2.6% versus 88.1 +/- 3.8% (P=0.257) for patients in the HID and ISD SCT groups, respectively. Transplantation from HID or ISD SCT has greatly improved quality of life (QoL) levels post-HSCT compared to pre-HSCT. The occurrence of chronic graft-versus-host disease was the only identified adverse factor affecting each subscale of QoL. Physical and mental component summaries in adults as well as physical, mental, social, and role well-being in children were all similar between HID and ISD SCT at 5-year time points. At the last follow-up, the proportion of returning to society was comparable between the HID and ISD groups, showing 78.0% versus 84.6% among children and 74.6% versus 81.2% among adults. These data suggest that haploidentical transplant can be considered a potential therapeutic option in the upfront setting for SAA patients in the absence of an HLA-identical related or unrelated donor.

Automated summary

Allogeneic hematopoietic stem cell transplantation is an effective method for treating severe aplastic anemia, and haploidentical donor transplantation has made significant progress in this field. Based on a national registry analysis, the long-term outcomes of upfront haploidentical or identical sibling donor stem cell transplantation were compared, and it was found that both approaches led to similar overall survival and failure-free survival rates. Quality of life improved significantly after transplantation, and the occurrence of chronic graft-versus-host disease was identified as the only adverse factor affecting quality of life. The physical and mental well-being of patients were similar between the two transplantation methods.