title: Comparison of outcomes after umbilical cord blood and unmanipulated haploidentical hematopoietic stem cell transplantation in children with high-risk acute lymphoblastic leukemia.
abstract: Allogeneic hematopoietic stem cell transplantation (HSCT) is an effective therapy for children with high-risk acute lymphoblastic leukemia (ALL). Human leukocyte antigen (HLA)-haploidentical HSCT (haplo-HSCT) or umbilical cord blood transplantation (UCBT) are both important alternative sources of stem cells for those without an HLA-identical sibling donor or unrelated matched donor. We aimed to compare the therapeutic effects of single UCBT and unmanipulated haplo-HSCT in high-risk ALL children (n = 129). Hematopoietic recovery was significantly faster in haplo-HSCT recipients than in UCBT recipients. The 2-year cumulative incidences of relapse in the haplo-HSCT and UCBT groups were 16.1% and 24.1%, respectively (p = 0.169). The 2-year cumulative incidences of non-relapse mortality in the haplo-HSCT and UCBT groups were 12.8% and 18.8%, respectively (p = 0.277). The 2-year probabilities of overall survival in the haplo-HSCT and UCBT groups were 82.0% and 69.6%, respectively (p = 0.071), and the 2-year probability of disease-free survival in the haplo-HSCT group was higher than in the UCBT group (71.0% vs. 57.2%, p = 0.040). However, several variables (such as leukocyte count and cytogenetics at diagnosis) were different between the groups, and a possible center effect should also be considered. In addition, only mild and moderate chronic graft-versus-host disease (GVHD) was associated with significantly improved survival compared to those without chronic GVHD in multivariate analysis. Thus, our results show that both unmanipulated haplo-HSCT and UCBT are valid for high-risk ALL children lacking a HLA matched donor, and both strategies expand the donor pool for children in need.
authors.affiliation: Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.|Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.
authors.forename: Xiao-Dong
authors.initials: XD
authors.lastname: Mo
authors.affiliation: Department of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei, China.
authors.forename: Bao-Lin
authors.initials: BL
authors.lastname: Tang
authors.affiliation: Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.|Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.
authors.forename: Xiao-Hui
authors.initials: XH
authors.lastname: Zhang
authors.affiliation: Department of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei, China.
authors.forename: Chang-Cheng
authors.initials: CC
authors.lastname: Zheng
authors.affiliation: Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.|Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.
authors.forename: Lan-Ping
authors.initials: LP
authors.lastname: Xu
authors.affiliation: Department of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei, China.
authors.forename: Xiao-Yu
authors.initials: XY
authors.lastname: Zhu
authors.affiliation: Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.|Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.
authors.forename: Yu
authors.initials: Y
authors.lastname: Wang
authors.affiliation: Department of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei, China.
authors.forename: Hui-Lan
authors.initials: HL
authors.lastname: Liu
authors.affiliation: Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.|Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.
authors.forename: Chen-Hua
authors.initials: CH
authors.lastname: Yan
authors.affiliation: Department of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei, China.
authors.forename: Xian-Deng
authors.initials: XD
authors.lastname: Chu
authors.affiliation: Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.|Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.
authors.forename: Huan
authors.initials: H
authors.lastname: Chen
authors.affiliation: Department of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei, China.
authors.forename: Liang-Quan
authors.initials: LQ
authors.lastname: Geng
authors.affiliation: Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.|Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.
authors.forename: Kai-Yan
authors.initials: KY
authors.lastname: Liu
authors.affiliation: Department of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei, China.
authors.forename: Zi-Min
authors.initials: ZM
authors.lastname: Sun
authors.affiliation: Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China.|Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.|Peking-Tsinghua Center for Life Sciences, Beijing, China.
authors.forename: Xiao-Jun
authors.initials: XJ
authors.lastname: Huang
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods*; D005260:Female; D006086:Graft vs Host Disease / Q000453:epidemiology*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000453:epidemiology*; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000401:mortality / Q000628:therapy*; D012008:Recurrence; D016019:Survival Analysis; D016896:Treatment Outcome
keywords: acute lymphoblastic leukemia; children; haploidentical; stem cell transplantation; umbilical cord blood transplantation