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单倍型相合移植治疗获得性重型再生障碍性贫血的多中心前瞻性研究

Haplo-identical transplantation for acquired severe aplastic anaemia in a multicentre prospective study.

作者信息

Xu Lan-Ping, Wang Shun-Qing, Wu De-Pei, Wang Jian-Min, Gao Su-Jun, Jiang Ming, Wang Cun-Bang, Zhang Xi, Liu Qi-Fa, Xia Ling-Hui, Wang Xin, Huang Xiao-Jun

机构信息

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

Guangzhou First People's Hospital, Guangzhou, China.

出版信息

Br J Haematol. 2016 Oct;175(2):265-274. doi: 10.1111/bjh.14225. Epub 2016 Jun 28.

DOI:10.1111/bjh.14225
PMID:27352174
Abstract

We conducted a prospective, multicentre study to confirm the feasibility of haplo-identical transplantation in treatment of severe aplastic anaemia (SAA) as salvage therapy, by analysing the outcomes of 101 patients who received haplo-identical transplantation between June 2012 and October 2015. All cases surviving for more than 28 d achieved donor myeloid engraftment. The median time for myeloid engraftment was 12 (range, 9-25) days and 15 (range, 7-101) days for platelets, with a cumulative platelet engraftment incidence of 94·1 ± 0·1%. With a median follow-up of 18·3 (3·0-43·6) months, recipients from haplo-identical transplantation had more cumulative incidence of grade II-IV acute graft-versus-host disease (aGVHD, 33·7% vs. 4·2%, P < 0·001), more chronic GVHD (22·4% vs. 6·6%, P = 0·014) at 1 year, but similar grade III-IV aGVHD (7·9% vs. 2·1%, P = 0·157), 3-year estimated overall survival (OS, 89·0% vs. 91·0%, P = 0·555) and failure-free survival (FFS, 86·8% vs. 80·3%, P = 0·659) when compared with 48 patients who received contemporaneous transplantation from matched related donors. Multivariate analysis showed no significant difference in engraftment and survival between the two cohorts. Both OS and FFS for the entire population correlated significantly with grades III-IV aGVHD. In conclusion, haplo-identical transplantation is a feasible choice for SAA with favourable outcomes.

摘要

我们进行了一项前瞻性多中心研究,通过分析2012年6月至2015年10月期间接受单倍体相合移植的101例患者的结局,以确认单倍体相合移植作为挽救疗法治疗重型再生障碍性贫血(SAA)的可行性。所有存活超过28天的病例均实现了供体髓系植入。髓系植入的中位时间为12天(范围9 - 25天),血小板植入的中位时间为15天(范围7 - 101天),累积血小板植入发生率为94.1±0.1%。中位随访18.3个月(3.0 - 43.6个月),单倍体相合移植受者II - IV级急性移植物抗宿主病(aGVHD)的累积发生率更高(33.7%对4.2%,P < 0.001),1年时慢性GVHD更多(22.4%对6.6%,P = 0.014),但III - IV级aGVHD相似(7.9%对2.1%,P = 0.157),与48例接受同期匹配相关供体移植的患者相比,3年估计总生存率(OS,89.0%对91.0%,P = 0.555)和无失败生存率(FFS,86.8%对80.3%,P = 0.659)相似。多变量分析显示两组之间在植入和生存方面无显著差异。整个人群的OS和FFS均与III - IV级aGVHD显著相关。总之,单倍体相合移植是SAA的一种可行选择,结局良好。

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