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Outcomes of haploidentical haematopoietic stem cell transplantation for paroxysmal nocturnal haemoglobinuria

Bone Marrow Transplantation volume 55, pages 1635–1637 (2020)Cite this article

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Allogeneic haematopoietic stem cell transplantation (allo-HSCT) might be used in paroxysmal nocturnal haemoglobinuria (PNH) patients outside open clinical trials assessing the complement inhibitors and the presence of a PNH clone with evidence of clonal evolution (e.g. myelodysplastic syndrome and leukaemia) [1]. Haploidentical HSCT (haplo-HSCT) is now increasingly applied as a curative therapy for patients with haematologic diseases. This study aimed to evaluate the outcomes of HSCT treatment for 28 patients with PNH who underwent haplo-HSCT at our centres.

All 28 patients with PNH who underwent haplo-HSCT at our centre between October 2010 and December 2018 were enroled in this study. Details of conditioning regimen, graft collection and infusion, graft versus host disease (GVHD) prophylaxis and treatment strategy, supportive care and post-transplantation surveillance, and other supportive care are consistent with our previous experience [2].

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Acknowledgements

This work was partly supported by grants from the National Key R&D Program of China (2016YFC0902800, 2017YFA0104502, and 2017ZX09304021), Innovation Capability Development Project of Jiangsu Province (BM2015004), Jiangsu Provincial Key Medical Center (YXZXA2016002), Jiangsu Medical Outstanding Talents Project (JCRCA2016002), and Priority Academic Program Development of Jiangsu Higher Education Institutions.

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  1. These authors contributed equally: Limin Liu, Yanming Zhang, Shan Liu

Authors and Affiliations

  1. The First Affiliated Hospital of Soochow University, Jiangsu Institute of Hematology, Key Laboratory of Thrombosis and Hemostasis of Ministry of Health, Collaborative Innovation Center of Hematology, 188 Shizi Street, Suzhou, China

    Limin Liu, Huifen Zhou, Qingyuan Wang, Hong Tian, Feng Chen, Huiying Qiu, Xiaowen Tang, Yue Han, Chengcheng Fu, Zhengming Jin, Suning Chen, Aining Sun, Miao Miao & Depei Wu

  2. Department of hematology, The Affiliated Huai’an Hospital of Xuzhou Medical University and The Second People’s Hospital of Huai’an, No 62, Huaihai Road (S.), Huai’an, China

    Yanming Zhang

  3. Department of Laboratory Medicine, Affiliated Hospital of University of Electronic Science and Technology of China, Sichuan Academy of Medical Sciences and Sichuan Provincial People’s Hospital, Chengdu, China

    Shan Liu

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  1. Limin Liu
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Correspondence to Miao Miao or Depei Wu.

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Liu, L., Zhang, Y., Liu, S. et al. Outcomes of haploidentical haematopoietic stem cell transplantation for paroxysmal nocturnal haemoglobinuria. Bone Marrow Transplant 55, 1635–1637 (2020). https://doi.org/10.1038/s41409-019-0751-6

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