Immunotherapy vs Best Supportive Care for Patients With Hepatocellular Cancer With Child-Pugh B Dysfunction
Authors: Fulgenzi Claudia Angela Maria , Scheiner Bernhard , D’Alessio Antonio , Mehan Aman , Manfredi Giulia F. , Celsa Ciro , Nishida Naoshi , Ang Celina , Marron Thomas U. , Wu Linda , Saeed Anwaar , Wietharn Brooke , Cammarota Antonella , Pressiani Tiziana , Pinter Matthias , Sharma Rohini , Cheon Jaekyung , Huang Yi-Hsiang , Lee Pei-Chang , Phen Samuel , Gampa Anuhya , Pillai Anjana , Napolitano Andrea , Vivaldi Caterina , Salani Francesca , Masi Gianluca , Silletta Marianna , Lo Prinzi Federica , Di Giacomo Emanuela , Vincenzi Bruno , Bettinger Dominik , Thimme Robert , Vogel Arndt , Schönlein Martin , von Felden Johann , Schulze Kornelius , Wege Henning , Galle Peter R. , Pirisi Mario , Park Joong-Won , Kudo Masatoshi , Rimassa Lorenza , Singal Amit G. , El Tomb Paul , Ulahannan Susanna , Parisi Alessandro , Chon Hong Jae , Hsu Wei-Fan , Ghittoni Giorgia , Cammà Calogero , Stefanini Benedetta , Trevisani Franco , Giannini Edoardo G. , Cortellini Alessio , Pinato David James 
Published: July 2024

ImportanceWhether 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.ObjectiveTo 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 ParticipantsThis 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.ExposuresPatients 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 MeasuresOS 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.ResultsThe 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 &amp;lt; .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 &amp;lt; .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 RelevanceThe 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.