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Aplastic Anemia

Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia

Bone Marrow Transplantation volume 52, pages 381–387 (2017)Cite this article

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Abstract

Techniques for haploidentical hematopoietic stem cell transplantation (haplo-HSCT) to treat severe aplastic anemia (SAA) have recently improved, but no protocol has been evaluated in a large number of pediatric patients. Fifty-two children with SAA received haplo-HSCT in our center. The treatment protocol used G-CSF-primed bone marrow with G-CSF-mobilized PBSCs without in vitro T-cell depletion. The conditioning regimen included busulfan/cyclophosphamide and antithymocyte globulin. Fifty-one patients achieved primary engraftment; one child died of regimen-related toxicity on the day +1. Secondary graft failure occurred in three patients. The cumulative incidences of aGVHD grade II–IV and grade III–IV were 39.2±0.5 and 13.7±0.2%, respectively. The cumulative incidence of cGVHD was 34.2±0.5%. The 3-year overall and failure-free survival rates were 84.5±5.0 and 82.7±5.2%, respectively, with a median follow-up time of 744.5 days (100–3294) for surviving patients. The Eastern Cooperative Oncology Group score was the only predictor of overall and failure-free survival rates. Clinical outcomes were similar between the upfront and salvage group. This result suggests that both newly diagnosed and refractory pediatric SAA patients benefit from haplo-HSCT, especially when patients are in good general condition. Therefore, haplo-HSCT might be an alternative therapy for pediatric SAA patients without HLA-matched sibling donors.

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Acknowledgements

This work was partly supported by the Key Program of National Natural Science Foundation of China (Grant No 81230013 and 81370666) and Beijing Municipal Science & Technology Commission (No Z121107002612035). We thank all colleagues for participating in the research.

Author contributions

XJ Huang designed the research; LP Xu and XJ Huang analyzed the data and wrote the manuscript; and all authors, provided patient data and gave final approval for the manuscript.

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Authors and Affiliations

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

    L P Xu, X H Zhang, F R Wang, X D Mo, T T Han, W Han, Y H Chen, Y Y Zhang, J Z Wang, C H Yan, Y Q Sun, S N Zuo & X J Huang

  2. Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China

    X J Huang

  3. Peking-Tsinghua Center for Life Sciences, Beijing, China

    X J Huang

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Correspondence to X J Huang.

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Xu, L., Zhang, X., Wang, F. et al. Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia. Bone Marrow Transplant 52, 381–387 (2017). https://doi.org/10.1038/bmt.2016.281

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