维迪西妥单抗治疗HER2阳性伴肝转移乳腺癌：RC48-C006 2/3期试验的3期结果。
完整摘要 · 中文译文
HER2阳性伴肝转移的晚期乳腺癌（BCLM）患者预后较差。RC48-C006研究是一项前瞻性、开放标签、多中心、随机2/3期试验（ClinicalTrials.gov注册号，NCT03500380）。基于2期结果，启动了3期阶段，以评估维迪西妥单抗（DV）对比拉帕替尼联合卡培他滨（L + C）在既往接受过曲妥珠单抗和紫杉类治疗的HER2阳性BCLM患者中的疗效。本文报告3期阶段的结果。共104例患者按1:1随机分组，接受DV（53例）或L + C（51例）治疗。盲态独立评审委员会（BIRC）评估的无进展生存期（PFS）显示，DV组较L + C组显著改善（中位数：9.9个月 vs 4.9个月；分层风险比[HR]：0.56 [95.48% CI，0.35-0.91]；双侧P = 0.01），达到了预设的主要终点。研究者评估的PFS与BIRC评估结果一致。预设的PFS亚组分析一致倾向于DV。DV组与L + C组≥3级治疗相关不良事件的发生率相似（37.7% vs 36.0%）。本研究结果支持DV作为HER2阳性BCLM患者在曲妥珠单抗和紫杉类治疗后的治疗选择。
完整摘要 · 英文原文
Patients with HER2-positive advanced breast cancer with liver metastases (BCLM) have poor prognosis. Study RC48-C006 is a prospective, open-label, multi-center, randomized phase 2/3 trial (ClinicalTrials.gov identifier, NCT03500380). Based on the phase 2 results, the phase 3 stage was initiated to evaluate disitamab vedotin (DV) versus lapatinib plus capecitabine (L + C) in patients with HER2-positive BCLM who were previously treated with trastuzumab and taxanes. The results from the phase 3 stage are reported in this article. A total of 104 patients were randomized at 1:1 to receive DV (53 patients) or L + C (51 patients). Progression-free survival (PFS) assessed by the blinded Independent Review Committee (BIRC) was significantly improved with DV versus with L + C (median: 9.9 vs 4.9 months; stratified hazard ratio [HR]: 0.56 [95.48% CI, 0.35-0.91]; 2-sided P = 0.01), meeting the pre-specified primary endpoint. The investigator-assessed PFS was consistent with the BIRC assessment. Prespecified subgroup analysis of PFS consistently favored DV. Similar incidences of grade ≥3 treatment-related adverse events were reported with DV versus L + C (37.7% vs 36.0%). The present findings support DV as a treatment option for HER2-positive BCLM following trastuzumab and taxane therapy.