PDF(Pubmed)
Abstract：
Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary end point, may be published when key planned co-primary or secondary analyses are not yet available. Clinical Trial Updates provide an opportunity to disseminate additional results from studies, published in JCO or elsewhere, for which the primary end point has already been reported.nab-Sirolimus is approved in the United States for the treatment of metastatic or locally advanced malignant perivascular epithelioid cell tumor (PEComa) on the basis of the primary analysis results of the phase II Advanced Malignant Perivascular Epithelioid Cell Tumors (AMPECT) trial (ClinicalTrials.gov identifier: NCT02494570). Results from the primary analysis were previously published; however, the median duration of response (mDOR) had not been reached at that time. Here, 3 years after the primary analysis, we report final efficacy and safety data (data cutoff: April 29, 2022). At study completion, the confirmed overall response rate (by independent radiologist review using RECIST v1.1) was 38.7% (95% CI, 21.8 to 57.8), with an additional converted confirmed complete response (n = 2). Median progression-free survival remained the same at 10.6 months (95% CI, 5.5 to 41.2). The mDOR was reached at 39.7 months (95% CI, 6.5 to not reached [NR]), and the median overall survival at completion was 53.1 months (95% CI, 22.2 to NR). The most common treatment-related adverse events (TRAEs) were stomatitis (82.4%) and fatigue and rash (each 61.8%). No new or unexpected adverse events occurred, and no grade ≥4 TRAEs were reported. These results highlight the long-term clinical benefit of nab-sirolimus in patients with advanced malignant PEComa, with a DOR of >3 years.
摘要：
临床试验通常包括在不同时间成熟的多个终点。初次报告,通常基于主要终点，当尚未获得关键计划的共同主要或次要分析时，可能会发布。临床试验更新提供了传播其他研究结果的机会，发表在JCO或其他地方，已经报告了主要终点。根据II期晚期恶性血管周围上皮样细胞肿瘤（AMPECT）试验的主要分析结果，nab-西罗莫司在美国被批准用于治疗转移性或局部晚期恶性血管周围上皮样细胞肿瘤（PEComa）（ClinicalTrials.gov标识符：NCT02494570）。初步分析的结果以前发表过；然而，当时尚未达到中位缓解持续时间(mDOR).这里,初步分析3年后，我们报告最终疗效和安全性数据（数据截止日期：2022年4月29日）。研究完成时，确认的总体反应率（通过使用RECISTv1.1的独立放射科医师审查）为38.7％（95％CI，21.8至57.8），另一个转化的确认完全反应（n=2）。中位无进展生存期在10.6个月时保持不变（95％CI，5.5至41.2）。在39.7个月时达到mDOR(95%CI，6.5到未达到[NR])，完成时的中位总生存期为53.1个月(95%CI,22.2至NR).最常见的治疗相关不良事件（TRAEs）是口腔炎（82.4%）、疲劳和皮疹（各61.8%）。没有新的或意外的不良事件发生，无≥4级TRAE报告。这些结果突出了nab-西罗莫司对晚期恶性PEComa患者的长期临床益处，DOR>3年。
