文章：

艾曲泊帕联合或不联合他克莫司治疗复发/难治性获得性再生障碍性贫血：一项前瞻性随机试验

Eltrombopag with or without Tacrolimus for relapsed/refractory acquired aplastic anaemia: a prospective randomized trial

原文发布日期：2023-09-19

DOI: 10.1038/s41408-023-00921-8

类型: Article

开放获取: 是

英文摘要：

This trial compared eltrombopag (EPAG)+tacrolimus and EPAG monotherapy in patients with refractory/relapsed acquired aplastic anaemia (AA). Patients with refractory/relapsed AA were randomly assigned to receive either EPAG+tacrolimus or EPAG monotherapy at a ratio of 2:1. Patient response, safety, clonal evolution and survival were compared. In total, 114 patients were included in the analysis, with 76 patients receiving EPAG+tacrolimus and 38 receiving EPAG only. With a median follow-up of 18 (6–24) months, the overall response rate (ORR) for patients treated with EPAG+tacrolimus and EPAG alone was 38.2% vs. 31.6% (P = 0.490) at the 3rd month, 61.8% vs. 39.5% (P = 0.024) at the 6th month, 64.5% vs. 47.1% (P = 0.097) at the 12th month, and 60.5% vs. 34.2% (P = 0.008) at the last follow-up. The rate of each adverse event, overall survival curves (P = 0.635) and clonal evolution rate (P = 1.000) were comparable between the groups. A post hoc subgroup analysis showed that EPAG+tacrolimus could have advantage over EPAG monotherapy in terms of the ORR at the 6th month (P = 0.030)/last follow-up (P = 0.013) and the cumulative relapse-free survival (RFS) curves (P = 0.048) in patients <60 years old.
 

摘要翻译： 

本试验比较了艾曲波帕联合他克莫司与单用艾曲波帕在难治/复发获得性再生障碍性贫血患者中的疗效。研究将难治/复发患者按2:1比例随机分配至艾曲波帕联合他克莫司组或单用艾曲波帕组，并对患者反应率、安全性、克隆演变及生存情况进行比较。共114例患者纳入分析，其中76例接受联合治疗，38例接受单药治疗。中位随访18（6-24）个月时，联合治疗组与单药治疗组的总反应率分别为：第3个月38.2%对31.6%（P=0.490），第6个月61.8%对39.5%（P=0.024），第12个月64.5%对47.1%（P=0.097），末次随访时60.5%对34.2%（P=0.008）。两组间各不良事件发生率、总生存曲线（P=0.635）及克隆演变率（P=1.000）均无显著差异。事后亚组分析显示，在60岁以下患者中，联合治疗组在第6个月（P=0.030）/末次随访（P=0.013）的总反应率及累计无复发生存曲线（P=0.048）方面可能优于单药治疗组。

原文链接：

Eltrombopag with or without Tacrolimus for relapsed/refractory acquired aplastic anaemia: a prospective randomized trial