Peking Union Medical College Hospital (PUMCH), Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing, China.
Blood Cancer J. 2023 Sep 19;13(1):146. doi: 10.1038/s41408-023-00921-8.
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 3 month, 61.8% vs. 39.5% (P = 0.024) at the 6 month, 64.5% vs. 47.1% (P = 0.097) at the 12 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 6 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.
这项试验比较了艾曲波帕（EPAG）+他克莫司与 EPAG 单药治疗难治/复发获得性再生障碍性贫血（AA）患者的效果。难治/复发 AA 患者以 2:1 的比例随机接受 EPAG+他克莫司或 EPAG 单药治疗。比较了患者的应答率、安全性、克隆演变和生存情况。共有 114 例患者纳入分析，76 例患者接受 EPAG+他克莫司治疗，38 例患者仅接受 EPAG 治疗。中位随访 18（6-24）个月，接受 EPAG+他克莫司和 EPAG 单药治疗的患者的总体反应率（ORR）在 3 个月时分别为 38.2%和 31.6%（P=0.490），在 6 个月时分别为 61.8%和 39.5%（P=0.024），在 12 个月时分别为 64.5%和 47.1%（P=0.097），在末次随访时分别为 60.5%和 34.2%（P=0.008）。两组间的每种不良事件发生率、总生存曲线（P=0.635）和克隆演变率（P=1.000）相当。事后亚组分析显示，在 6 个月（P=0.030）/末次随访（P=0.013）和累积无复发生存（RFS）曲线（P=0.048）时，EPAG+他克莫司相对于 EPAG 单药治疗在<60 岁的患者中可能具有优势。
