Immunotherapy vs Best Supportive Care for Patients With Hepatocellular Cancer With Child-Pugh B Dysfunction (2024)

• ¹Department of Surgery and Cancer, Imperial College London, Hammersmith Hospital, London, England

• ²Operative Research Unit of Oncology, Fondazione Policlinico Universitario Campus Bio-Medico, Rome, Italy

• ³Division of Gastroenterology and Hepatology, Department of Internal Medicine III, Medical University of Vienna, Vienna, Austria

• ⁴Department of Translational Medicine, University of Piemonte Orientale, Novara, Italy

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• ⁵Gastroenterology and Hepatology Unit, Department of Health Promotion, Mother and Child Care, Internal Medicine & Medical Specialties, University of Palermo, Palermo, Italy

• ⁶Department of Gastroenterology and Hepatology, Kindai University Faculty of Medicine, Osaka, Japan

• ⁷Department of Medicine, Division of Hematology/Oncology, Tisch Cancer Institute, Mount Sinai Hospital, New York, New York

• ⁸Department of Medicine, Division of Medical Oncology, Kansas University Cancer Center, Kansas City

• ⁹Department of Biomedical Sciences, Humanitas University, Milan, Italy

• ¹⁰Medical Oncology and Hematology Unit, Humanitas Cancer Center, IRCCS Humanitas Research Hospital, Rozzano, Milan, Italy

• ¹¹Medical Oncology, Department of Internal Medicine, CHA Bundang Medical Center, CHA University, Seongnam, Korea

• ¹²Healthcare and Services Center, Taipei Veterans General Hospital, Taipei, Taiwan

• ¹³Division of Gastroenterology and Hepatology, Taipei Veterans General Hospital, Taipei, Taiwan

• ¹⁴Institute of Clinical Medicine, Faculty of Medicine, National Yang Ming Chiao Tung University, Taipei, Taiwan

• ¹⁵Division of Gastroenterology and Hepatology, Taipei Veterans General Hospital, Taipei, Taiwan

• ¹⁶Faculty of Medicine, National Yang Ming Chiao Tung University, Taipei, Taiwan

• ¹⁷Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas

• ¹⁸Medical Oncology/TSET Phase 1 Program, Stephenson Cancer Center, Section of Gastroenterology, University of Oklahoma, Oklahoma City

• ¹⁹Hepatology & Nutrition, the University of Chicago Medicine, Chicago, Illinois

• ²⁰The Royal Marsden National Health Service Foundation Trust, London, England

• ²¹Unit of Medical Oncology 2, Azienda Ospedaliero- Universitaria Pisana, Pisa, Italy

• ²²Scuola Superiore Sant’Anna Pisa, Interdisciplinary Research Center, Pisa, Italy

• ²³Department of Gastroenterology, Hepatology, Endocrinology and Infectious Diseases, Freiburg University Medical Center, University of Freiburg, Freiburg, Germany

• ²⁴Hannover Medical School, Hannover, Germany

• ²⁵Department of Oncology, Hematology and Bone Marrow Transplantation with Section of Pneumology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany

• ²⁶Department of Medicine, University Medical Center Hamburg-Eppendorf, Hamburg, Germany

• ²⁷Department of Internal Medicine I, University Medical Center Mainz, Mainz, Germany

• ²⁸National Cancer Centre Hospital, Goyang, South Korea

• ²⁹Stephenson Cancer Center, Oklahoma City, Oklahoma

• ³⁰Department of Oncology, Università Politecnica delle Marche, Azienda Ospedaliero-Universitaria delle Marche, Ancona, Italy

• ³¹Center for Digestive Medicine, Department of Internal Medicine, China Medical University Hospital, Taichung, Taiwan

• ³²Gastroenterology Unit, Belcolle hospital, Viterbo, Italy

• ³³Semeiotics and Liver and Alcohol-Related Disease Unit, Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy

• ³⁴Gastroenterology Unit, Department of Internal Medicine, University of Genoa, Genoa, Italy

• ³⁵IRCCS Ospedale Policlinico San Martino, Genoa, Italy

Key Points

Question Do patients with unresectable hepatocellular carcinoma (HCC) and suboptimal liver function benefit from immunotherapy vs best supportive care?

Findings In this case series of 343 patients with Child-Pugh class B liver dysfunction, immunotherapy-based systemic therapy was associated with significant reduction in the risk of death compared with best supportive care.

Meaning The results of this study suggest that immunotherapy may be a safe and effective option for patients with unresectable HCC and suboptimal liver function.

Importance Whether patients with Child-Pugh class B (CP-B) cancer with unresectable hepatocellular carcinoma (uHCC) benefit from active anticancer treatment vs best supportive care (BSC) is debated.

Objective To evaluate the association of immune checkpoint inhibitor (ICI)–based therapies vs BSC with overall survival (OS) of patients with uHCC and CP-B liver dysfunction.

Design, Setting, and Participants This retrospective, multicenter, international clinical case series examined data of patients with CP-B with uHCC who were receiving first-line ICI-based regimens from September 2017 to December 2022 whose data were extracted from an international consortium and compared with a cohort of patients with CP-B receiving BSC. Patients were treated in tertiary care centers across Europe, US, and Asia in routine clinical practice. After applying the inclusion criteria, 187 and 156 patients were left in the ICI and BSC groups, respectively. The propensity score was calculated for the following variables: age, alpha-fetoprotein levels, Child-Pugh score, extrahepatic spread, portal vein tumor thrombosis, cirrhosis, ascites, and baseline Eastern Cooperative Oncology Group performance status.

Exposures Patients in the ICI group received first-line systemic therapy with either atezolizumab plus bevacizumab (A+B) (n = 141) or nivolumab (n = 46).

Main Outcomes and Measures OS in the inverse probability of treatment weighting (IPTW) populations was the main outcome, and it was estimated with Kaplan-Meier method; univariable Cox regression test was used to make comparisons between the 2 groups.

Results The median age was 66 (IQR, 61-72) and 73 (IQR, 66-81) years in the ICI (33 women [18%]) and BSC groups (41 women [26%]), respectively. In the IPTW populations, median OS was significantly longer in the ICI group (7.50 months; 95% CI, 5.62-11.15) compared with BSC (4.04 months; 95% CI, 3.03-5.03; hazard ratio, 0.59; 95% CI, 0.43-0.80; P < .001). Multivariable analysis confirmed that ICI exposure was associated with a reduction of approximately 50% in the risk of death (hazard ratio, 0.55; 95% CI, 0.35-0.86; P < .001), and the presence of portal vein tumor thrombosis, an Eastern Cooperative Oncology Group performance score of greater than 1, and alpha-fetoprotein levels of 400 ng/mL or greater were associated with increased risk of death.

Conclusions and Relevance The results of this case series provide comparative evidence of improved survival in association with ICI treatment compared with BSC in patients with uHCC with CP-B liver dysfunction.

Hepatobiliary Disease Gastroenterology and Hepatology Gastroenterology Gastrointestinal Cancer Hepatobiliary Cancer Oncology

Citation

Fulgenzi CAM, Scheiner B, D’Alessio A, et al. Immunotherapy vs Best Supportive Care for Patients With Hepatocellular Cancer With Child-Pugh B Dysfunction. JAMA Oncol. Published online July 18, 2024. doi:10.1001/jamaoncol.2024.2166

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