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

Summary 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.