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Pediatric Transplants

Long-term outcomes of unmanipulated haploidentical HSCT for paediatric patients with acute leukaemia

Abstract

Allogeneic hematopoietic SCT is indicated for children whose disease demonstrates dismal prognosis with chemotherapy. This study aims to analyse the most recent outcomes of unmanipulated haploidentical (HID) HSCT for paediatric patients with acute leukaemia. Those from matched sibling donors (MSD) HSCT provided a parallel cohort to illustrate the benefits of HID. Conditioning regimen was modified BuCy2. Anti-thymoglobulin was used for HID. Mobilised marrow and blood stem cells were used as the grafts. All patients in HID achieved neutrophil recovery and 96.7% platelet recovery. In HID, the incidences of acute GVHD 3–4 and extensive chronic GVHD were 14.3 and 26.6%. Play-performance score 90–100% was recorded in 79.7% of all survivors. The 5-year leukaemia-free survival (LFS) in CR1, CR2, beyond CR2 or non-remission were 68.9%, 56.6%, 22.2% and 82.5%, 59.4%, 42.9% for ALL and AML, respectively. In MSD group, LFS for ALL and AML in CR1 were 62.5 and 71.7%. Outcomes of the HID HSCT for paediatric patients with acute leukaemia showed benefits that were similar to those of the parallel cohort of MSD HSCT.

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Acknowledgements

We thank the laboratory teams for their supporting to clinical work. This work was supported in part by grants from National Natural Science Foundation of China (grant No. 30971292), National Clinical Priority Specialty of Ministry of Health of China and National Science & Technology Pillar Program (Grant no. 2008BAI61B01).

Author Countributions

X-J Huang designed research and revised the paper. D-H Liu performed research, analysed data and wrote the paper. L-P Xu, K-Y Liu, Y Wang, H Chen, W Han, X-H Zhang, C-H Yan, Y-Y Zhang, J-Z Wang, Y-H Chen and F-R Wang participated in the research.

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Correspondence to X-J Huang.

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Liu, DH., Xu, LP., Liu, KY. et al. Long-term outcomes of unmanipulated haploidentical HSCT for paediatric patients with acute leukaemia. Bone Marrow Transplant 48, 1519–1524 (2013). https://doi.org/10.1038/bmt.2013.99

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