Outcomes and prognosis of haploidentical haematopoietic stem cell transplantation in children with FLT3-ITD mutated acute myeloid leukaemia

Author:

Shang Qianwen1,Bai Lu,Cheng Yifei,Suo Pan2,Hu Guanhua,Yan Chenhua,Wang Yu,Zhang Xiaohui,Xu Lanping,Liu Kaiyan3,Huang XiaoJun4

Affiliation:

1. Peking University People’s Hospital

2. Peking University People’s Hospital, Peking University Institute of Hematology, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking University

3. Peking University Institute of Hematology, People's hospital

4. Peking University People's Hospital

Abstract

Abstract The presence of internal tandem duplication mutations in the FMS-like tyrosine kinase 3 receptor (FLT3-ITD) is a poor prognostic predictor in paediatric patients with acute myeloid leukaemia (AML). We evaluated the treatment outcomes and prognostic factors of 45 paediatric patients with FLT3-ITD AML who achieved complete remission before haploidentical haematopoietic stem cell transplantation (haplo-HSCT) at our institution from 2012 to 2021. Among the 45 patients, the overall survival (OS), event‑free survival (EFS), and cumulative incidence of relapse (CIR) rates were 74.9%±6.6%, 64.1%±7.2%, and 31.4%±7.1%, respectively, with 48.8 months of median follow-up. Univariate and multivariate analyses associated positive minimal residual disease (MRD) at pre-HSCT , MRD by flow cytometry (FCM)≥0.1% after two cycles induction and time from diagnosis to HSCT more than 24 months with inferior long-term survival. The 4-year CIR of grade II–IV acute graft-versus-host (GVHD) and chronic GVHD after transplantation were 53.3% ± 7.6% and 35.7% ± 9.8%, respectively. In conclusion, haplo-HSCT may be a feasible strategy for paediatric patients with FLT3-ITD AML. MRD status at pre-HSCT, MRD by FCM after two cycles induction and the time from diagnosis to HSCT affect patient outcomes.

Publisher

Research Square Platform LLC

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