NEJM:清髓性自体造血干细胞移植治疗重度硬皮病

2018-01-04 zhangfan MedSci原创

研究认为,清髓性自体造血干细胞移植可使硬皮病患者获得长期受益,表现为无事件生存以及总生存期延长

弥漫性皮肤系统性硬化症(硬皮病)的治疗效果不佳。近日研究人员比较了清髓CD 34选择性自体造血干细胞移植与环磷酰胺免疫抑制对硬皮病患者的影响。

研究招募了成年重度硬皮病患者,其中36人接受清髓自体干细胞移植,39人接受环磷酰胺。主要终点为54个月后疾病特征层次结构评估:包括死亡、无事件生存(无呼吸、肾功能或心脏衰竭生存)、肺活量、残疾指数评分、健康评估问卷、改良罗德南皮肤评分。

54个月后,移植组综合评估分数显示出极大的优越性(67%的指标移植组显示优越性,33%的指标化疗组显示出优越性)。接受移植或完成9轮化疗的患者中,54个月,移植组无事件生存率为79%,化疗组为50%(P=0.02)。72个月后,Kaplan-Meier无事件生存率(74% vs. 47%)以及总生存率(86% vs. 51%)移植组占优。54个月后,移植组9%的参与者进行了抗风湿治疗,而化疗组为44%。治疗相关的患者死亡率,54个月和72个月,移植组分别为3%和6%,化疗组未发生治疗相关的死亡事件。

研究认为,清髓性自体造血干细胞移植可使硬皮病患者获得长期受益,表现为无事件生存以及总生存期延长。

原始出处:

Keith M. Sullivan et al. Myeloablative Autologous Stem-Cell Transplantation for Severe Scleroderma. N Engl J Med. January 4, 2018.

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    2018-01-06 rgjl
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    2018-01-06 俅侠
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