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Haploidentical stem cell transplantation for aplastic anemia: the current advances and future challenges

Bone Marrow Transplantation volume 56, pages 779–785 (2021)Cite this article

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Abstract

Haematopoietic stem cell transplantation (HSCT) is a curative option for severe aplastic anemia (SAA). Finding a suitable matched donor in a timely manner is a challenge. The availability of haploidentical donors and their successful use in transplantation have expanded valid choices for SAA. In recent decades, haploidentical HSCT (haplo-HSCT) for the treatment of SAA has been continuously attempted, and great strides have been made. Nowadays, haplo-HSCT using different regimens has overcome the difficulty of graft failure and severe graft-versus-host disease (GvHD), and achieved inspiring survival outcomes in SAA. The regimens consist mainly of granulocyte colony-stimulating factor (G-CSF) plus antithymocyte globulin (ATG), posttransplantation cyclophosphamide (PT-Cy), and ex vivo graft T-cell depletion (TCD). In particular, the G-CSF and ATG-based regimen includes the largest sample size and the successful wide use of the G-CSF and ATG-based regimen has promoted haplo-HSCT a higher priority in SAA patients without matched related or unrelated donors in China. Recent studies have also indicated that haplo-HSCT using PT-Cy or TCD regimen is a practicable alternative, but the sample size is relatively small. Here, we offer an overview of clinical results obtained through the use of haploidentical transplantation in SAA, mainly focusing on current advances and future challenges.

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Acknowledgements

This work was partly supported by the Foundation for Innovative Research Groups of the National Natural Science Foundation of China (Grant No. 81621001), the Key Program of National Natural Science Foundation of China (Grant No. 81530046), the National Key Research and Development Program of China (No. 2017YFA0104500), and Peking University Clinical Scientist Program (No. BMU2019LCKXJ003).

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  1. Peking University People’s Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China

    Zheng-Li Xu & Xiao-Jun Huang

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X-JH designed the review, and both authors wrote the manuscript and gave final approval for the manuscript.

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Correspondence to Xiao-Jun Huang.

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Xu, ZL., Huang, XJ. Haploidentical stem cell transplantation for aplastic anemia: the current advances and future challenges. Bone Marrow Transplant 56, 779–785 (2021). https://doi.org/10.1038/s41409-020-01169-7

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