期刊
HAEMATOLOGICA
卷 107, 期 12, 页码 2918-2927出版社
FERRATA STORTI FOUNDATION
DOI: 10.3324/haematol.2022.280758
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资金
- National Key Research and Development Program [2021YFA1100904, 2019YFC0840606]
- National Natural Science Foundation of China [82100227, 81930004]
- Foundation for Innovative Research Groups of the National Natural Science Foundation of China [81621001]
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.
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.
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