Safety and efficacy of PD-1 inhibitors plus tyrosine kinase inhibitors combination therapy in patients with advanced hepatocellular carcinoma combined with hyperbilirubinemia: a retrospective cohort study

BackgroundProgrammed death-1 (PD-1) inhibitors plus tyrosine kinase inhibitors (TKIs) combination therapy are considered as a first-line treatment recommendation for advanced hepatocellular carcinoma (HCC). However, patients with hyperbilirubinemia are excluded from this therapeutic option due to li...

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Main Authors: Shida Pan, Jianing Wang, Jiahe Tian, Yilin Wang, Siyu Wang, Yingying Yu, Fengyi Li, Yan-Mei Jiao, Yingjuan Shen, Luo Yang, Xiaomeng Liu, Qin Qiu, Junqing Luan, Fu-Sheng Wang, Fanping Meng
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Language:English
Published: Frontiers Media S.A. 2025-03-01
Series:Frontiers in Immunology
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Online Access:https://www.frontiersin.org/articles/10.3389/fimmu.2025.1530477/full
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author Shida Pan
Shida Pan
Jianing Wang
Jianing Wang
Jiahe Tian
Jiahe Tian
Yilin Wang
Yilin Wang
Siyu Wang
Yingying Yu
Yingying Yu
Fengyi Li
Yan-Mei Jiao
Yingjuan Shen
Luo Yang
Xiaomeng Liu
Qin Qiu
Junqing Luan
Fu-Sheng Wang
Fu-Sheng Wang
Fu-Sheng Wang
Fu-Sheng Wang
Fanping Meng
Fanping Meng
Fanping Meng
author_facet Shida Pan
Shida Pan
Jianing Wang
Jianing Wang
Jiahe Tian
Jiahe Tian
Yilin Wang
Yilin Wang
Siyu Wang
Yingying Yu
Yingying Yu
Fengyi Li
Yan-Mei Jiao
Yingjuan Shen
Luo Yang
Xiaomeng Liu
Qin Qiu
Junqing Luan
Fu-Sheng Wang
Fu-Sheng Wang
Fu-Sheng Wang
Fu-Sheng Wang
Fanping Meng
Fanping Meng
Fanping Meng
author_sort Shida Pan
collection DOAJ
description BackgroundProgrammed death-1 (PD-1) inhibitors plus tyrosine kinase inhibitors (TKIs) combination therapy are considered as a first-line treatment recommendation for advanced hepatocellular carcinoma (HCC). However, patients with hyperbilirubinemia are excluded from this therapeutic option due to limitations in indications. There is a notable absence of published studies evaluating the safety and efficacy of the PD-1 inhibitors plus TKIs combination therapy in patients with HCC combined with hyperbilirubinemia.MethodsPatients with HCC complicated with hyperbilirubinemia who received combination therapy with PD-1 inhibitors and TKIs were retrospectively analyzed. Adverse events, tumor response, and laboratory parameters were recorded to assess the safety and efficacy of the treatment, as well as to identify potential risk factors influencing survival.ResultsA total of 108 participants were included in the study, with 56 patients (51.9%) reporting at least one adverse event, the majority of which were mild. The objective response rate (ORR) for the enrolled participants was 11.9%, and the disease control rate(DCR) reached 61.2%. The median overall survival (OS) for the entire cohort was 5.03 months, while the median progression-free survival (PFS) was 3.63 months. Multifactorial analysis showed that MELD score >18 and increased total bilirubin (TBIL) levels within one week were significant risk factors for OS. Patients with a decrease in TBIL levels within one week had significantly prolonged median OS (not reached vs 3.3months, P =0.013) and median PFS (7.03 months vs 2.77 months, P =0.010).ConclusionCombination therapy demonstrated favorable safety and tolerability among patients with HCC combined with hyperbilirubinemia. Patients who experienced a rapid decline in TBIL levels during the early phase of treatment with PD-1 inhibitors and TKIs were observed to derive clinical benefits. Early initiation of aggressive interventions aimed at reducing TBIL levels is recommended to optimize treatment outcomes.
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spelling doaj-art-a60d8a74b3a549b7952153c4702be0742025-08-20T02:57:53ZengFrontiers Media S.A.Frontiers in Immunology1664-32242025-03-011610.3389/fimmu.2025.15304771530477Safety and efficacy of PD-1 inhibitors plus tyrosine kinase inhibitors combination therapy in patients with advanced hepatocellular carcinoma combined with hyperbilirubinemia: a retrospective cohort studyShida Pan0Shida Pan1Jianing Wang2Jianing Wang3Jiahe Tian4Jiahe Tian5Yilin Wang6Yilin Wang7Siyu Wang8Yingying Yu9Yingying Yu10Fengyi Li11Yan-Mei Jiao12Yingjuan Shen13Luo Yang14Xiaomeng Liu15Qin Qiu16Junqing Luan17Fu-Sheng Wang18Fu-Sheng Wang19Fu-Sheng Wang20Fu-Sheng Wang21Fanping Meng22Fanping Meng23Fanping Meng24Beijing Ditan Hospital, Capital Medical University, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaPeking University 302 Clinical Medical School, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaPeking University 302 Clinical Medical School, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaChinese People's Liberation Army (PLA) Medical School, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaThe First Affiliated Hospital of University of Science and Technology of China (USTC), Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaBeijing Ditan Hospital, Capital Medical University, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaPeking University 302 Clinical Medical School, Beijing, ChinaChinese People's Liberation Army (PLA) Medical School, Beijing, ChinaSenior Department of Infectious Diseases, The Fifth Medical Center of Chinese People's Liberation Army (PLA) General Hospital, Beijing, ChinaPeking University 302 Clinical Medical School, Beijing, ChinaChinese People's Liberation Army (PLA) Medical School, Beijing, ChinaBackgroundProgrammed death-1 (PD-1) inhibitors plus tyrosine kinase inhibitors (TKIs) combination therapy are considered as a first-line treatment recommendation for advanced hepatocellular carcinoma (HCC). However, patients with hyperbilirubinemia are excluded from this therapeutic option due to limitations in indications. There is a notable absence of published studies evaluating the safety and efficacy of the PD-1 inhibitors plus TKIs combination therapy in patients with HCC combined with hyperbilirubinemia.MethodsPatients with HCC complicated with hyperbilirubinemia who received combination therapy with PD-1 inhibitors and TKIs were retrospectively analyzed. Adverse events, tumor response, and laboratory parameters were recorded to assess the safety and efficacy of the treatment, as well as to identify potential risk factors influencing survival.ResultsA total of 108 participants were included in the study, with 56 patients (51.9%) reporting at least one adverse event, the majority of which were mild. The objective response rate (ORR) for the enrolled participants was 11.9%, and the disease control rate(DCR) reached 61.2%. The median overall survival (OS) for the entire cohort was 5.03 months, while the median progression-free survival (PFS) was 3.63 months. Multifactorial analysis showed that MELD score >18 and increased total bilirubin (TBIL) levels within one week were significant risk factors for OS. Patients with a decrease in TBIL levels within one week had significantly prolonged median OS (not reached vs 3.3months, P =0.013) and median PFS (7.03 months vs 2.77 months, P =0.010).ConclusionCombination therapy demonstrated favorable safety and tolerability among patients with HCC combined with hyperbilirubinemia. Patients who experienced a rapid decline in TBIL levels during the early phase of treatment with PD-1 inhibitors and TKIs were observed to derive clinical benefits. Early initiation of aggressive interventions aimed at reducing TBIL levels is recommended to optimize treatment outcomes.https://www.frontiersin.org/articles/10.3389/fimmu.2025.1530477/fullhepatocellular carcinomahyperbilirubinemiaprogrammed death 1immunotherapysurvival
spellingShingle Shida Pan
Shida Pan
Jianing Wang
Jianing Wang
Jiahe Tian
Jiahe Tian
Yilin Wang
Yilin Wang
Siyu Wang
Yingying Yu
Yingying Yu
Fengyi Li
Yan-Mei Jiao
Yingjuan Shen
Luo Yang
Xiaomeng Liu
Qin Qiu
Junqing Luan
Fu-Sheng Wang
Fu-Sheng Wang
Fu-Sheng Wang
Fu-Sheng Wang
Fanping Meng
Fanping Meng
Fanping Meng
Safety and efficacy of PD-1 inhibitors plus tyrosine kinase inhibitors combination therapy in patients with advanced hepatocellular carcinoma combined with hyperbilirubinemia: a retrospective cohort study
Frontiers in Immunology
hepatocellular carcinoma
hyperbilirubinemia
programmed death 1
immunotherapy
survival
title Safety and efficacy of PD-1 inhibitors plus tyrosine kinase inhibitors combination therapy in patients with advanced hepatocellular carcinoma combined with hyperbilirubinemia: a retrospective cohort study
title_full Safety and efficacy of PD-1 inhibitors plus tyrosine kinase inhibitors combination therapy in patients with advanced hepatocellular carcinoma combined with hyperbilirubinemia: a retrospective cohort study
title_fullStr Safety and efficacy of PD-1 inhibitors plus tyrosine kinase inhibitors combination therapy in patients with advanced hepatocellular carcinoma combined with hyperbilirubinemia: a retrospective cohort study
title_full_unstemmed Safety and efficacy of PD-1 inhibitors plus tyrosine kinase inhibitors combination therapy in patients with advanced hepatocellular carcinoma combined with hyperbilirubinemia: a retrospective cohort study
title_short Safety and efficacy of PD-1 inhibitors plus tyrosine kinase inhibitors combination therapy in patients with advanced hepatocellular carcinoma combined with hyperbilirubinemia: a retrospective cohort study
title_sort safety and efficacy of pd 1 inhibitors plus tyrosine kinase inhibitors combination therapy in patients with advanced hepatocellular carcinoma combined with hyperbilirubinemia a retrospective cohort study
topic hepatocellular carcinoma
hyperbilirubinemia
programmed death 1
immunotherapy
survival
url https://www.frontiersin.org/articles/10.3389/fimmu.2025.1530477/full
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