Outcomes of haploidentical bone marrow transplantation in patients with severe aplastic anemia-II that progressed from non-severe acquired aplastic anemia
Outcomes of haploidentical bone marrow transplantation in patients with severe aplastic anemia-II that progressed from non-severe acquired aplastic anemia
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由非严重获得性再生障碍性贫血进展而来的严重再生障碍性贫血-II 患者的单倍相合骨髓移植的结果
DOI：
10.1007/s11684-020-0807-4
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发表时间：
2021-06-25
影响因子：
8.1
通讯作者：
Yan, Jinsong
中科院分区：
文献类型：
--
作者：
Liu, Hongchen;Zheng, Xiaoli;Yan, Jinsong
重型再生障碍性贫血II（SAA-II）是从非重型再生障碍性贫血（NSAA）发展而来的。由于缺乏有效的治疗方法，需要对缺乏人类白细胞抗原（HLA）匹配供体的患者进行单倍相合骨髓移植（haplo-BMT）。本研究旨在探讨单倍骨髓移植对SAA-II患者的疗效。纳入并随访22例患者，采用FLU/BU/CY/ATG作为预处理方案。在这些患者中，21例移植成功，其中19例在单倍骨髓移植后存活。4名患者经历了II-IV级aGvHD，包括2名III-IV级aGvHD。6例患者发生慢性GvHD，其中4例为轻度，2例为中度。12例患者在BMT期间发生感染。一名被诊断为移植后淋巴组织增生性疾病，一名被诊断为可能的EBV疾病，两人都在利妥昔单抗输注后康复。Haplo-BMT在中位随访42个月后实现了3年总生存率和86.4% ± 0.73%的无病生存率，表明其作为挽救治疗的有效性。这些有希望的结果可能支持haplo-BMT作为缺乏HLA匹配供体的SAA-II患者的替代治疗策略。
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.