Division of Solid Tumor Oncology, Department of Medicine, Thoracic Oncology Service Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, New York, New York.
Division of Cancer Medicine, Department of Thoracic/Head and Neck Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, Texas.
Clin Cancer Res. 2017 Dec 15;23(24):7483-7489. doi: 10.1158/1078-0432.CCR-17-2169. Epub 2017 Sep 28.
Determine the 1-year progression-free survival (PFS) rate among patients with malignant pleural mesothelioma (MPM) receiving the WT1 peptide vaccine galinpepimut-S after multimodality therapy versus those receiving control adjuvants. This double-blind, controlled, two center phase II trial randomized MPM patients after surgery and another treatment modality to galinpepimut-S with GM-CSF and Montanide or GM-CSF and Montanide alone. An improvement in 1-year PFS from 50% to 70% was the predefined efficacy threshold, and 78 patients total were planned. The study was not powered for comparison between the two arms. Forty-one patients were randomized. Treatment-related adverse events were mild, self-limited, and not clinically significant. On the basis of a stringent prespecified futility analysis (futility = ≥10 of 20 patients on one arm experiencing progression < 1 year), the control arm closed early. The treatment arm was subsequently closed because of the resultant unblinding. The PFS rate at 1 year from beginning study treatment was 33% and 45% in the control and vaccine arms, respectively. Median PFS was 7.4 months versus 10.1 months and median OS was 18.3 months versus 22.8 months in the control and vaccine arms, respectively. The favorable safety profile was confirmed. PFS and OS were greater in those who received vaccine, but the trial was neither designed nor powered for comparison between the arms. On the basis of these promising results, the investigators are planning a larger randomized trial with greater statistical power to define the optimal use and benefit of galinpepimut-S in the treatment of MPM. .
评估恶性胸膜间皮瘤（MPM）患者在多模式治疗后接受 WT1 肽疫苗 galinpepimut-S 与接受对照佐剂相比的 1 年无进展生存率（PFS）。这是一项双盲、对照、两中心的 II 期临床试验，将手术后和另一种治疗方式的 MPM 患者随机分为 galinpepimut-S 联合 GM-CSF 和 Montanide 或 GM-CSF 和 Montanide 单药组。1 年 PFS 从 50%提高到 70%是预先设定的疗效阈值，共计划招募 78 名患者。该研究没有对两组之间进行比较的效力。41 名患者被随机分配。治疗相关的不良事件是轻微的、自限性的，且无临床意义。根据严格的预先规定的无效性分析（无效性=在一个臂上有 20 名患者中有≥10 名在 1 年内进展<1 年），对照组提前关闭。由于由此导致的失盲，治疗组随后关闭。从开始研究治疗起，1 年的 PFS 率分别为对照组和疫苗组的 33%和 45%。对照组和疫苗组的中位 PFS 分别为 7.4 个月和 10.1 个月，中位 OS 分别为 18.3 个月和 22.8 个月。良好的安全性得到了确认。接受疫苗的患者 PFS 和 OS 更高，但该试验既不是为比较两组之间的差异而设计，也没有为此提供足够的效力。基于这些有希望的结果，研究人员正在计划进行一项更大规模的随机试验，以更大的统计效力来确定 galinpepimut-S 在治疗 MPM 中的最佳应用和获益。
