title: A novel conditioning regimen improves outcomes in β-thalassemia major patients using unrelated donor peripheral blood stem cell transplantation.
abstract: We used a novel NF-08-TM transplant protocol based on intravenous busulfan, cyclophosphamide, fludarabine, and thiotepa in 82 consecutive patients with β-thalassemia major (TM), including 52 with allogeneic peripheral blood stem cell transplantation (PBSCT) from unrelated donors (UDs) with well-matched human leukocyte antigens and 30 with hematopoietic stem cell transplantation (HSCT) from matched sibling donors (MSDs). The median age at transplantation was 6.0 years (range, 0.6-15.0 years), and the ratio of male-to-female patients was 56:26. The median follow-up time was 24 months (range, 12-39 months). The estimated 3-year overall survival and TM-free survival were 92.3% and 90.4% in the UD-PBSCT group and 90.0% and 83.3% in the MSD-HSCT group. The cumulative incidences of graft rejection and grades III-IV acute graft-versus-host disease were 1.9% and 9.6%, respectively, in the UD-PBSCT group and 6.9% and 3.6%, respectively, in the MSD-HSCT group. The cumulative incidence of transplant-related mortality was 7.7% in the UD-PBSCT group and 10.0% in the MSD-HSCT group. In conclusion, UD-PBSCTs using the well-tolerated NF-08-TM protocol show similar results to MSD-HSCTs and can be used to treat β-thalassemia patients in the absence of MSDs.
authors.affiliation: Departments of Pediatrics, Nanfang Hospital, Southern Medical University, Guangzhou, China.
authors.forename: Chunfu
authors.initials: C
authors.lastname: Li
authors.forename: Xuedong
authors.initials: X
authors.lastname: Wu
authors.forename: Xiaoqing
authors.initials: X
authors.lastname: Feng
authors.forename: Yuelin
authors.initials: Y
authors.lastname: He
authors.forename: Huaying
authors.initials: H
authors.lastname: Liu
authors.forename: Fuyu
authors.initials: F
authors.lastname: Pei
authors.forename: Jianyu
authors.initials: J
authors.lastname: Liao
authors.forename: Lan
authors.initials: L
authors.lastname: He
authors.forename: Lei
authors.initials: L
authors.lastname: Shi
authors.forename: Na
authors.initials: N
authors.lastname: Li
authors.forename: Qiujun
authors.initials: Q
authors.lastname: Liu
authors.forename: Shiting
authors.initials: S
authors.lastname: Liu
authors.forename: Geyu
authors.initials: G
authors.lastname: Chen
authors.forename: Qingxia
authors.initials: Q
authors.lastname: Su
authors.forename: Yuqiong
authors.initials: Y
authors.lastname: Ren
authors.forename: Yanhua
authors.initials: Y
authors.lastname: Wang
authors.forename: Wanxia
authors.initials: W
authors.lastname: Tan
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000188:drug therapy / Q000209:etiology; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D036102:Peripheral Blood Stem Cell Transplantation / Q000009:adverse effects; D019172:Transplantation Conditioning; D016896:Treatment Outcome; D061349:Unrelated Donors; D055815:Young Adult; D017086:beta-Thalassemia / Q000150:complications / Q000401:mortality / Q000628:therapy*