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Immunosuppressive therapy versus haploidentical transplantation in adults with acquired severe aplastic anemia

Bone Marrow Transplantation volume 54, pages 1319–1326 (2019)Cite this article

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Abstract

Our study aimed to compare treatment outcomes between hematopoietic stem cell transplantation (HSCT) from haploidentical donors (HID) and immunosuppressive therapy (IST) in adults with acquired severe aplastic anemia (SAA). The medical records of 113 SAA adults who received IST, including rabbit ATG and cyclosporin (N = 37), or HID HSCT (N = 76) within 6 months of diagnosis at two institutions were retrospectively reviewed. Estimated 8-year overall survival (OS) was comparable between the IST and HID HSCT groups (75.6 vs. 83.7%, respectively, P = 0.328), but failure free survival (FFS) was significantly lower in IST group than HID HSCT group (38.5 vs. 83.7%, respectively, P = 0.001). Furthermore, a significant improvement in FFS was observed with HSCT over IST in patients under 40 years old. At the last follow-up, patients in HSCT group achieved better Karnofsky Performance Status (KPS) than those in IST group (100 [20–100] vs. 90 [20–100], P = 0.002). In terms of blood count, 83.1% (54/65) of patients in HSCT group showed complete recovery compared to only 38.2% (13/34) in IST group (P < 0.001). These data suggest that HID HSCT could be an effective alternative treatment option for SAA adults, and additional prospective studies are necessary.

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Acknowledgements

This work was partly supported by the National Natural Science Foundation of China (Grant No. 81670167 and 81670166), and Innovative Research Groups of the National Natural Science Foundation of China (No. 81621001). We thank all colleagues for participating in this research.

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  1. These authors contributed equally: Zheng-Li Xu, Ming Zhou, Shun-Qing Wang, Lan-Ping Xu

Authors and Affiliations

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

    Zheng-Li Xu, Jin-Song Jia, Xiao-Hui Zhang, Yu Wang, Xiao-Jun Huang & Lan-Ping Xu

  2. Guangzhou First People’s Hospital, Guangzhou, China

    Ming Zhou, Wen-Jian Mo, Yu-Ping Zhang, Yu-Miao Li & Shun-Qing Wang

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

    Xiao-Jun Huang

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

    Xiao-Jun Huang

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  1. Zheng-Li Xu
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Correspondence to Lan-Ping Xu.

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Xu, ZL., Zhou, M., Jia, JS. et al. Immunosuppressive therapy versus haploidentical transplantation in adults with acquired severe aplastic anemia. Bone Marrow Transplant 54, 1319–1326 (2019). https://doi.org/10.1038/s41409-018-0410-3

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