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• W4382011714 abstract "Objective: The purpose of this study is to determine the efficacy of haploidentical donor hematopoietic stem cell transplantation in the treatment of severe aplastic anemia. Methods: The clinical data of 76 patients with severe aplastic anemia (SAA) patients who underwent haplo-HSCT from December 2014 to October 2020 were selectively analyzed. There were 50 males and 26 females with a median age of 16 (3-52) years old. There were 49 SAA-Ⅰ patients, 18 SAA-Ⅱ patients, and 9 patients with hepatitis-associated aplastic anemia. There were 15 cases of bone marrow put together with peripheral blood stem cell transplantation and 61 cases of peripheral blood stem-cell transplantation. Conditioning regimens were Cyclophosphamide (CY) + Fludarabine (Flu) + ATG for 46 patients and Busulfan (Bu) + CY+Flu+ATG for 30 patients. Results: Three patients died during the myelosuppressive phase following transplantation, and 73 patients had a median time of neutrophil engraftment of 12 (9-21) days; in addition to 3 patients who died early, 8 patients did not obtain platelet reconstruction after transplantation, and 65 patients had platelet engraftment with a medium time of 14 (9-90) d. The incidence of primary graft failure was 10.9% and the incidence of secondary graft failure was 5.5%. The incidence of Ⅱ-Ⅳ acute graft-versus-host disease (aGVHD) was 38.4%, the incidence of Ⅲ-Ⅳ aGVHD was 16.4%, the incidence of chronic graft anti-host disease (cGVHD) was 35.8%, and the incidence of extensive cGVHD was 22.4%. The medium follow-up time was 19.5 (1-75) months, the prospective overall survival (OS) for 2 years was (78.6±5.0) %, the failure-free survival (FFS) was (75.9±5.1) %, and the transplant-related mortality was (20.2±4.9) %. Multi-factor analysis revealed that the patient older than 35 years old, Ⅲ/Ⅳ aGVHD, HCT-CI≥3, the pre-transplant ferritin ≥1 500 μg/L, the number of neutrophils >1×10(9)/L at the time of onset were risk factors affecting OS (P=0.008, 0.008, 0.014, 0.004, 0.027) . Patients with graft failure had lower OS and FFS than other patients (P<0.001) . Conclusion: Haplo-HSCT is an effective method for treating SAA in children, adolescents, and young patients, and the occurrence of severe aGVHD and severe infection, as well as graft failure, are the main causes of survival rate. The prevention and treatment of severe aGVHD and infection are essential to improve efficacy.目的： 评价单倍体造血干细胞移植（haplo-HSCT）治疗重型再生障碍性贫血（SAA）的疗效。 方法： 对2014年12月至2020年10月在中国医学科学院血液病医院接受haplo-HSCT的76例SAA患者进行回顾性分析。 结果： ①76例SAA患者中男50例，女26例，中位年龄为16（3~52）岁；SAA-Ⅰ型49例，SAA-Ⅱ型18例，肝炎相关再生障碍性贫血（HAAA）9例；外周血干细胞移植61例，骨髓移植+外周血干细胞移植15例。预处理：环磷酰胺+氟达拉滨+抗人胸腺细胞球蛋白方案46例，白消安+环磷酰胺+氟达拉滨+抗人胸腺细胞球蛋白方案30例。②3例患者移植后早期死亡而未获得粒细胞植入，8例患者未获得血小板植入。73例患者获得粒细胞植入，中位植入时间为12（9~21）d；65例患者获得血小板植入，中位植入时间为14（9~90）d。原发性植入失败发生率为10.9%，继发性植入失败的发生率为5.5%。③Ⅱ~Ⅳ度急性移植物抗宿主病（aGVHD）发生率为38.4%，Ⅲ/Ⅳ度aGVHD的发生率为16.4%，慢性移植物抗宿主病（cGVHD）的发生率为35.3%，中重度cGVHD的发生率为22.1%。④中位随访时间19.5（1~75）个月，移植后2年总生存（OS）率为（78.6±5.0）%，无失败生存（FFS）率为（75.9±5.1）%，移植相关死亡率（TRM）为（20.2±4.9）%。⑤多因素分析显示：影响haplo-HSCT后OS的危险因素包括：患者年龄>35岁（P=0.008）、发生Ⅲ/Ⅳ度aGVHD（P=0.008）、造血干细胞移植合并症指数（HCT-CI）评分≥3分（P=0.014）、移植前铁蛋白>1500 μg/L（P=0.004）、初诊中性粒细胞计数>1×10(9)/L（P=0.027）。植入失败为影响患者OS及FFS的危险因素（P<0.001）。 结论： haplo-HSCT是治疗SAA的有效方法，在儿童青少年以及年轻患者中疗效较好，发生重度aGVHD、严重感染、植入失败是影响生存率的主要因素。." @default.
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• W4382011714 date "2023-03-14" @default.
• W4382011714 modified "2023-09-27" @default.
• W4382011714 title "[Clinical analysis of 76 patients with severe aplastic anemia treated with haploid hematopoietic stem cell transplantation]." @default.
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• W4382011714 doi "https://doi.org/10.3760/cma.j.issn.0253-2727.2023.03.005" @default.
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