目的:评估尼拉帕尼治疗严重预处理卵巢癌的日本妇女的长期疗效和安全性。
方法:这是第二阶段多中心的后续分析,开放标签,同源重组缺陷型日本女性的单臂研究,铂敏感,复发,高级别浆液性上皮性卵巢,输卵管,或完成3-4行化疗并且是聚(ADP-核糖)聚合酶抑制剂未治疗的原发性腹膜癌。参与者接受尼拉帕尼(起始剂量,300mg),连续28天,每天一次,直至客观疾病进展,不可接受的毒性,或同意撤回。主要终点是确认客观缓解率(ORR),使用实体瘤1.1版的反应评估标准进行评估。安全性评价包括治疗引起的不良事件(TEAE)。
结果:20名患者纳入研究,并纳入疗效和安全性分析。总研究持续时间的中位数为759.5天。中位剂量强度为201.3mg/天。确认ORR为60.0%(90%置信区间[CI]=39.4-78.3);2例患者完全缓解,10例患者部分缓解。中位缓解时间为9.9个月(95%CI=3.9-26.9),疾病控制率为90.0%(95%CI=68.3-98.8)。最常见的TEAE是贫血(n=15),恶心(n=12),血小板计数减少(n=11)。TEAE导致研究药物剂量减少,中断,或停药报告为16例(80.0%),15(75.0%),和2名患者(10.0%),分别。
结论:尼拉帕尼的长期疗效和安全性与非日本患者同等人群的先前发现一致。没有发现新的安全信号。
背景:ClinicalTrials.gov标识符:NCT03759600。
OBJECTIVE: To evaluate the long-term efficacy and safety of niraparib in Japanese women with heavily pretreated ovarian cancer.
METHODS: This was the follow-up analysis of a phase 2, multicenter, open-label, single-arm study in Japanese women with homologous recombination-deficient, platinum-sensitive, relapsed, high-grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who had completed 3-4 lines of chemotherapy and were poly(ADP-ribose) polymerase inhibitor naïve. Participants received niraparib (starting dose, 300 mg) once daily in continuous 28-day cycles until objective disease progression, unacceptable toxicity, or consent withdrawal. The primary endpoint was confirmed objective response rate (ORR), as assessed using Response Evaluation Criteria in Solid Tumors version 1.1. Safety evaluations included treatment-emergent adverse events (TEAEs).
RESULTS: 20 patients were enrolled in the study and included in both efficacy and safety analyses. Median total study duration was 759.5 days. Median dose intensity was 201.3 mg/day. Confirmed ORR was 60.0% (90% confidence interval [CI]=39.4-78.3); 2 patients had complete response and 10 patients had partial response. Median duration of response was 9.9 months (95% CI=3.9-26.9) and the disease control rate was 90.0% (95% CI=68.3-98.8). The most common TEAEs were anemia (n=15), nausea (n=12), and decreased platelet count (n=11). TEAEs leading to study drug dose reduction, interruption, or discontinuation were reported in 16 (80.0%), 15 (75.0%), and 2 patients (10.0%), respectively.
CONCLUSIONS: The long-term efficacy and safety profile of niraparib was consistent with previous findings in the equivalent population in non-Japanese patients. No new safety signals were identified.
BACKGROUND: ClinicalTrials.gov Identifier: NCT03759600.