Outcomes of haploidentical bone marrow transplantation in patients with severe aplastic anemia-II that progressed from non-severe acquired aplastic anemia

Abstract

Severe aplastic anemia II (SAA-II) progresses from non-severe aplastic anemia (NSAA). The unavailability of efficacious treatment has prompted the need for haploidentical bone marrow transplantation (haplo-BMT) in patients lacking a human leukocyte antigen (HLA)-matched donor. This study aimed to investigate the efficacy of haplo-BMT for patients with SAA-II. Twenty-two patients were included and followed up, and FLU/BU/CY/ATG was used as conditioning regimen. Among these patients, 21 were successfully engrafted, 19 of whom survived after haplo-BMT. Four patients experienced grade II–IV aGvHD, including two with grade III–IV aGvHD. Six patients experienced chronic GvHD, among whom four were mild and two were moderate. Twelve patients experienced infections during BMT. One was diagnosed with post-transplant lymphoproliferative disorder and one with probable EBV disease, and both recovered after rituximab infusion. Haplo-BMT achieved 3-year overall survival and disease-free survival rate of 86.4%±0.73% after a median follow-up of 42 months, indicating its effectiveness as a salvage therapy. These promising outcomes may support haplo-BMT as an alternative treatment strategy for patients with SAA-II lacking HLA-matched donors.

Keywords

severe aplastic anemia / non-severe acquired aplastic anemia / haploidentical bone marrow transplantation / outcomes

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Acknowledgements

The authors thank the physicians, laboratory coworkers, and nurses who were dedicated to the patients. This work was supported by grants from the National Natural Science Foundation of China (No. 81570124 to Jinsong Yan, No. 81702683 to Jiajun Xie, No. 81773389 to Jing Shao), Science and Technology Innovation Leading Talent Program of Liaoning Province (No. XLYC1902036 to Jinsong Yan), Basic Research on the Application of Dalian Innovation Fund (No. 2019J12SN56 to Jinsong Yan), Key R&D projects in Liaoning Province (No. 2019JH8/10300027 to Jinsong Yan), Key Project of the Educational Department of Liaoning Province (No. LZ2020003 to Jinsong Yan), the Capital Health Research and Development of Special Project (No. 2014-2-5122 to Hengxiang Wang), the Beijing Municipal Science and Technology Project (No. Z151100004015016 to Hengxiang Wang), and the Dalian Medical Scientific Research Project (No. 1712040 to Yan Yang).

Compliance with ethics guidelines

Hongchen Liu, Xiaoli Zheng, Chengtao Zhang, Jiajun Xie, Beibei Gao, Jing Shao, Yan Yang, Hengxiang Wang, and Jinsong Yan have no conflicts of interest to declare. All procedures used in the present study adhered to the ethical standards of the institutional review boards of the participating hospitals, and with the Helsinki Declaration of 1975, as revised in 2000(5). The patients, bone marrow donors, and their guardians provided written informed consents for all procedures in this study.

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