Data from Pembrolizumab Monotherapy in Sorafenib-Treated and Treatment-Naïve Advanced Hepatocellular Carcinoma: Long-Term Follow-up of the Open-Label, Phase II KEYNOTE-224 Study

作者：Richard S. Finn, Masatoshi Kudo, Ivan Borbath, Julien Edeline, Stéphane Cattan, Hans Van Vlierberghe, Chris Verslype, D. Palmer, Per Stål, Sadahisa Ogasawara, Arndt Vogel, Stephen L. Chan, Jennifer J. Knox, Bruno Daniele, Amos Odeleye-Ajakaye, Ken Hatogai, Abby B. Siegel, Ann‐Lii Cheng, Jean‐Luc Van Laethem · 年份：2026 · DOI：10.1158/1078-0432.c.8542591 · 研究领域：Hepatocellular Carcinoma Treatment and Prognosis、Cancer Immunotherapy and Biomarkers、Cholangiocarcinoma and Gallbladder Cancer Studies

<div>AbstractPurpose:In the phase II KEYNOTE-224 study, pembrolizumab showed durable antitumor activity and manageable safety in participants with sorafenib-treated (cohort 1) or treatment-naïve (cohort 2) advanced hepatocellular carcinoma (HCC). We present data after a median follow-up of ∼7 years for cohort 1 and ∼5 years for cohort 2.Patients and Methods:Adults with advanced HCC received pembrolizumab 200 mg intravenously every 3 weeks until disease progression or unacceptable toxicity for ≤35 cycles. The primary endpoint was objective response rate per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) by blinded independent central review. Secondary endpoints included overall survival (OS), progression-free survival (PFS), and safety.Results:Overall, 155 participants received ≥1 dose of pembrolizumab (cohort 1, n = 104; cohort 2, n = 51). The median follow-up was 83 months (range, 79.3–87.3) for cohort 1 and 58.8 months (range, 55.3–60.8) for cohort 2. The median OS was 13.2 months [95% confidence interval (CI), 9.7–15.3] and 16.9 months (95% CI, 8.3–23.1), respectively; 24- and 48-month OS rates were 31%/17% and 34%/20%, respectively. The median PFS was 4.9 months (95% CI, 3.5–7) and 4.3 months (95% CI, 2.1–7.8), respectively. Treatment-related adverse events occurred in 76 participants [73.1%; grade 3–5, 27 (26%)] in cohort 1 and 28 participants [54.9%; grade ...