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非调控性单倍体相合移植预处理方案:采用马利兰、环磷酰胺和抗胸腺球蛋白治疗成人重型再生障碍性贫血。

Unmanipulated haploidentical transplantation conditioning with busulfan, cyclophosphamide and anti-thymoglobulin for adult severe aplastic anaemia.

机构信息

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

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

出版信息

Bone Marrow Transplant. 2018 Feb;53(2):188-192. doi: 10.1038/bmt.2017.237. Epub 2018 Jan 15.

Abstract

We conducted a retrospective analysis to evaluate outcomes of haploidentical transplantation in adult severe aplastic anaemia (SAA) patients. Fifty-one adults received haploidentical transplantation between May 2011 and December 2016. Patients were administered busulfan (Bu), cyclophosphamide (Cy) and anti-thymoglobulin (ATG) as conditioning regimens, followed by bone marrow and peripheral blood transplantation. The patients' median age was 25 years. Forty-nine patients survived for more than 28 days and all achieved donor myeloid engraftment. The median time for myeloid engraftment and platelet recovery was 13 days (range, 10-21) and 17.5 (range, 7-101) days. The cumulative incidence (CI) of grade II-IV and III-IV acute GvHD) was 20.00±0.33% and 6.00±0.12%, respectively. The incidence of chronic GvHD was 14.00±0.36% and 25.90±0.71%, and that of moderate-severe chronic GvHD was 2.51±0.06% and 6.92±0.25% at 1 and 3 years, respectively. The 3-year estimated overall survival and failure-free survival were both 83.5±5.4% with a median follow-up of 21.1 months. Multivariate analysis showed hematopoietic cell transplantation-specific comorbidity index (HCT-CI) score of ⩾3 was significantly associated with worse outcome. Haploidentical transplantation conditioning including Bu/Cy/ATG was a safe and effective strategy for adult SAA patients, and HCT-CI might be an outcome predictor in these patients.

摘要

我们进行了一项回顾性分析,以评估单倍体相合移植在成人重型再生障碍性贫血(SAA)患者中的疗效。51 例成人于 2011 年 5 月至 2016 年 12 月接受了单倍体相合移植。患者采用白消安(Bu)、环磷酰胺(Cy)和抗胸腺球蛋白(ATG)作为预处理方案,随后进行骨髓和外周血移植。患者的中位年龄为 25 岁。49 例患者存活超过 28 天,所有患者均实现了供者骨髓造血重建。骨髓造血重建和血小板恢复的中位时间分别为 13 天(范围:10-21 天)和 17.5 天(范围:7-101 天)。Ⅱ-Ⅳ度和Ⅲ-Ⅳ度急性移植物抗宿主病(GvHD)的累积发生率分别为 20.00±0.33%和 6.00±0.12%。慢性 GvHD 的发生率为 14.00±0.36%和 25.90±0.71%,1 年和 3 年时中重度慢性 GvHD 的发生率分别为 2.51±0.06%和 6.92±0.25%。3 年的估计总生存率和无失败生存率均为 83.5±5.4%,中位随访时间为 21.1 个月。多因素分析显示,造血细胞移植特异性合并症指数(HCT-CI)评分 ⩾3 与不良预后显著相关。含 Bu/Cy/ATG 的单倍体相合移植预处理方案是治疗成人 SAA 患者的一种安全有效的策略,HCT-CI 可能是该类患者的预后预测因子。

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