Patients were randomly assigned to receive resminostat or placebo (200 mg orally per day; days 1-5 and 8-12) and S-1 group (80-120 mg orally per day by body surface area; days 1-14) over a 21-day cycle. The primary endpoint was progression-free survival (PFS). Secondary endpoints comprised overall survival (OS), response rate (RR), disease control rate (DCR), and safety.
Among 101 patients enrolled, 50 received resminostat+S-1 and 51 received placebo+S-1. Median PFS was 2.9 months for resminostat+S-1 vs. 3.0 months for placebo+S-1 (HR: 1.154, 95% CI: 0.759-1.757, p = 0.502); median OS was 7.8 months vs. 7.5 months, respectively (HR: 1.049, 95% CI: 0.653-1.684, p = 0.834); the RR and DCR were 6.0% vs. 9.8% and 70.0% vs. 78.4%, respectively. Treatment-related adverse events (TrAEs) of grade ≥ 3 occurring more frequently (≥10% difference) in the resminostat+S-1 than in the placebo+S-1 comprised platelet count decreased (18.0% vs. 2.0%) and decreased appetite (16.0% vs. 2.0%).
Resminostat plus S-1 therapy improved neither PFS nor OS for patients with pre-treated BTCs. Addition of resminostat to S-1 was associated with higher incidence of TrAEs, but these were manageable (JapicCTI-183883).
在21天的周期内,患者被随机分配接受瑞舒坦或安慰剂(每天200mg,第1-5天和第8-12天)和S-1组(每天80-120mg,按体表面积计算,每天80-120mg,第1-14天)。主要终点是无进展生存期(PFS)。次要终点包括总生存期(OS),反应率(RR),疾病控制率(DCR),和安全。
在101名患者中,50人接受瑞舒坦+S-1,51人接受安慰剂+S-1。resminostat+S-1的平均PFS为2.9个月,与安慰剂+S-1为3.0个月(HR:1.154,95%CI:0.759-1.757,p=0.502);中位OS为7.8个月。7.5个月,(HR:1.049,95%CI:0.653-1.684,p=0.834);RR和DCR分别为6.0%和9.8%和70.0%vs.78.4%,分别。与治疗相关的不良事件(TrAE)≥3级(≥10%差异)在resminostat+S-1中发生的频率高于安慰剂+S-1,包括血小板计数降低(18.0%vs.2.0%)和食欲下降(16.0%vs.2.0%)。
Resminostat联合S-1治疗既不能改善BTC患者的PFS也不能改善OS。在S-1中添加瑞司诺司他与更高的TrAE发生率相关,但这些都是可控的(JapicCTI-183883)。