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

Abstract
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.
AuthorsZheng-Li Xu, Lan-Ping Xu, De-Pei Wu, Shun-Qing Wang, Xi Zhang, Rui Xi, Su-Jun Gao, Ling-Hui Xia, Jian-Min Yang, Ming Jiang, Xin Wang, Qi-Fa Liu, Jia Chen, Ming Zhou, Xiao-Jun Huang
JournalHaematologica (Haematologica) Vol. 107 Issue 12 Pg. 2918-2927 (12 01 2022) ISSN: 1592-8721 [Electronic] Italy
PMID35615930 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
Topics
  • Adult
  • Child
  • Humans
  • Anemia, Aplastic (therapy)
  • Siblings
  • Quality of Life
  • Graft vs Host Disease (etiology)
  • Hematopoietic Stem Cell Transplantation
  • Unrelated Donors
  • Registries
  • Transplantation Conditioning

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