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

Bone Marrow Transplantation (2024)Cite this article

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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 and non-remission (NR) after introduce 1 with inferior long-term survival. The 100-day cumulative incidence of grade II–IV acute graft-versus-host disease (aGVHD) was 35.6% ± 5.2%, and that of grade III–IV aGVHD was 15.6% ± 3.0% The overall 4-year cumulative incidence of chronic graft-versus-host disease after transplantation was 35.7% ± 9.8%, respectively. In conclusion, haplo-HSCT may be a feasible strategy for paediatric patients with FLT3-ITD AML, and pre‐HSCT MRD status and NR after introduce 1 significantly affected the outcomes.

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Fig. 1: Flow Chart of acute myeloid protocol in our institution.
Fig. 2: Results of 45 paediatric patients with FLT3-ITD AML undergoing haplo-HSCT.
Fig. 3: Results of 45 paediatric patients with FLT3-ITD AML undergoing haplo-HSCT.

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Data availability

The datasets used and analysed during the current study are available from the corresponding authors on reasonable request. Our Figshare DOI is https://doi.org/10.6084/m9.figshare.24417739 [39].

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Acknowledgements

We thank Editage (www.editage.cn) for the linguistic assistance and scientific consultation during the preparation of this manuscript.

Funding

Beijing Clinical Key Specialist Project of Beijing Municipal Health Commission (2018) (No. 2199000726).

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  1. These authors contributed equally: Qianwen Shang, Lu Bai.

Authors and Affiliations

  1. Department of Paediatrics, Peking University People’s Hospital, Peking University, Beijing, China

    Qianwen Shang

  2. Department of Haematology, Peking University People’s Hospital, Peking University Institute of Haematology, Beijing Key Laboratory of Haematopoietic Stem Cell Transplantation Research Unit of Key Technique for Diagnosis and Treatments of Haematologic Malignancies, Chinese Academy of Medical Sciences, Peking University, 2019RU029, Beijing, China

    Lu Bai, Yifei Cheng, Pan Suo, Guanhua Hu, Chenhua Yan, Yu Wang, Xiaohui Zhang, Lanping Xu, Kaiyan Liu & Xiaojun Huang

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Contributions

QWS and LB performed the investigations, analysed data, and wrote the manuscript; YFC, PS, GHH, CHY, YW, XHZ, LPX and KYL performed the investigations; XJH designed the study. All the authors have read and approved the final manuscript.

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Correspondence to Xiaojun Huang.

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Shang, Q., Bai, L., Cheng, Y. et al. Outcomes and prognosis of haploidentical haematopoietic stem cell transplantation in children with FLT3-ITD mutated acute myeloid leukaemia. Bone Marrow Transplant (2024). https://doi.org/10.1038/s41409-024-02214-5

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