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Pembrolizumab Versus Placebo as Second-Line Therapy in Patients From Asia With Advanced Hepatocellular Carcinoma: A Randomized, Double-Blind, Phase III Trial

Qin S 1 , Chen Z 2 , Fang W 3 , Ren Z 4 , Xu R 5 , Ryoo BY 6 Show all authors , Meng Z 7 , Bai Y 8 , Chen X 9 , Liu X 1 , Xiao J 10 , Ho GF 11 , Mao Y 12 , Wang X 13 , Ying J 14 , Li J 15 , Zhong W 16 , Zhou Y 16 , Siegel AB 17 , Hao C 18

Affiliations 

  • 1 Jinling Hospital, Nanjing University of Chinese Medicine, Nanjing, China
  • 2 The Second Affiliated Hospital of Anhui Medical University, Hefei, China
  • 3 The First Affiliated Hospital Zhejiang University School of Medicine, Hangzhou, China
  • 4 Zhongshan Hospital, Fudan University, Shanghai, China
  • 5 Hunan Cancer Hospital, Changsha, China
  • 6 Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea
  • 7 Fudan University Shanghai Cancer Center, Shanghai, China
  • 8 Harbin Medical University Cancer Hospital, Harbin, China
  • 9 Guangdong Provincial People's Hospital, Guangzhou, China
  • 10 The First Affiliated Hospital of Xi'An Jiaotong University, Xi'an, China
  • 11 University Malaya Medical Centre, Kuala Lumpur, Malaysia
  • 12 Shanghai Jiaotong University School of Medicine, Renji Hospital, Shanghai, China
  • 13 West China Hospital of Sichuan University, Chengdu, China
  • 14 Cancer Hospital of the University of Chinese Academy of Sciences (Zhejiang Cancer Hospital), Hangzhou, China
  • 15 MSD China, Beijing, China
  • 16 MSD China, Shanghai, China
  • 17 Merck & Co, Inc, Rahway, NJ
  • 18 Peking University Cancer Hospital, Beijing, China
J Clin Oncol, 2023 Mar 01;41(7):1434-1443.
PMID: 36455168 DOI: 10.1200/JCO.22.00620

Abstract

PURPOSE: We evaluated the efficacy and safety of pembrolizumab in patients from Asia with previously treated advanced hepatocellular carcinoma (HCC).

METHODS: In a double-blind, phase III trial, 453 patients with advanced HCC and progression during or after treatment with or intolerance to sorafenib or oxaliplatin-based chemotherapy were randomly assigned in a 2:1 ratio to receive pembrolizumab (200 mg) or placebo once every 3 weeks for ≤ 35 cycles plus best supportive care. The primary end point was overall survival (one-sided significance threshold, P = .0193 [final analysis]). Secondary end points included progression-free survival (PFS) and objective response rate (ORR; one-sided significance threshold, P = .0134 and .0091, respectively [second interim analysis]; RECIST version 1.1, by blinded independent central review).

RESULTS: Median overall survival was longer in the pembrolizumab group than in the placebo group (14.6 v 13.0 months; hazard ratio for death, 0.79; 95% CI, 0.63 to 0.99; P = .0180). Median PFS was also longer in the pembrolizumab group than in the placebo group (2.6 v 2.3 months; hazard ratio for progression or death, 0.74; 95% CI, 0.60 to 0.92; P = .0032). ORR was greater in the pembrolizumab group (12.7% [95% CI, 9.1 to 17.0]) than in the placebo group (1.3% [95% CI, 0.2 to 4.6]; P < .0001). Treatment-related adverse events occurred in 66.9% of patients (grade 3, 12.0%; grade 4, 1.3%; grade 5, 1.0%) in the pembrolizumab group and 49.7% of patients (grade 3, 5.9%; grade 4, 0%; grade 5, 0%) in the placebo group.

CONCLUSION: In patients from Asia with previously treated advanced HCC, pembrolizumab significantly prolonged overall survival and PFS, and ORR was greater versus placebo.

* Title and MeSH Headings from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.

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