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Superior survival of unmanipulated haploidentical haematopoietic stem cell transplantation compared with intensive chemotherapy as post-remission treatment for children with very high-risk philadelphia chromosome negative B-cell acute lymphoblastic leukaemia in first complete remission.

Abstract
We explored the prognostic factors for children with very high-risk (VHR) Philadelphia chromosome (Ph) negative B-cell acute lymphoblastic leukaemia (B-ALL) and compared the therapeutic effects of intensive chemotherapy and unmanipulated haploidentical haematopoietic stem cell transplantation (haplo-HSCT) as post-remission treatment in these patients undergoing first complete remission (CR1). A total of 104 paediatric patients with VHR B-ALL in CR1 were retrospectively enrolled in this study, including 42 receiving unmanipulated haplo-HSCT (Group A) and 62 receiving ongoing chemotherapy (Group B). Estimated 3-year overall survival (OS), disease-free survival (DFS) and cumulative incidence of relapse (CIR) at 36·2 months median follow-up were 69·5 ± 4·7%, 63·5 ± 4·8% and 32·4 ± 4·7%, respectively. Maintenance of persistent positive or conversion from negative to positive of measurable residual disease (MRD) and chemotherapy were independent risk factors associated with inferior long-term survival and higher CIR. OS, DFS, and CIR differed significantly between the groups in patients with persistent positive or negative-to-positive MRD. Haplo-HSCT may be an option for children with VHR Ph-negative B-ALL in CR1, especially for patients with persistent positive or negative-to-positive MRD, and could achieve better survival than intensive chemotherapy as post-remission treatment.
AuthorsYu-Juan Xue, Pan Suo, Xiao-Jun Huang, Ai-Dong Lu, Yu Wang, Ying-Xi Zuo, Chen-Hua Yan, Jun Wu, Jun Kong, Xiao-Hui Zhang, Yu-Hong Chen, Yue-Ping Jia, Kai-Yan Liu, Wei Han, Lan-Ping Xu, Le-Ping Zhang, Yi-Fei Cheng
JournalBritish journal of haematology (Br J Haematol) Vol. 188 Issue 5 Pg. 757-767 (03 2020) ISSN: 1365-2141 [Electronic] England
PMID31725190 (Publication Type: Clinical Trial, Comparative Study, Journal Article, Research Support, Non-U.S. Gov't)
Copyright© 2019 British Society for Haematology and John Wiley & Sons Ltd.
Topics
  • Adolescent
  • Child
  • Child, Preschool
  • Disease-Free Survival
  • Female
  • Follow-Up Studies
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Infant
  • Male
  • Neoplasm, Residual
  • Philadelphia Chromosome
  • Precursor B-Cell Lymphoblastic Leukemia-Lymphoma (mortality, therapy)
  • Remission Induction
  • Risk Factors
  • Survival Rate
  • Transplantation, Homologous

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