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DOI: 10.1055/s-0045-1808034
Real life safety and efficacy of immunotherapy for unresectable hepatocellular carcinoma in clinical practice
Background: Phase III trials have established the superiority of immunotherapy-based combinations over sorafenib or lenvatinib in patients with unresectable hepatocellular carcinoma (HCC). The adoption of immunotherapy in routine practice is challenged by the risk of liver decompensation and treatment-related adverse events. Therefore, data on safety and effectiveness outside clinical trials are needed.
Aim: We aim to describe the clinical characteristics and outcomes of patients with HCC treated with immunotherapy in a real-world setting.
Methods: A database of patients with HCC treated across Brazilian centers between 2020 and 2024 was analyzed and patients who received immunotherapy-based regimens were included. Clinicopathologic characteristics were assessed for their prognostic influence on overall survival (OS). Overall response rate (ORR) and treatment-related adverse events (TRAEs) were reported.
Results: A total of 120 patients were included: median age was 72 yo, 83.6% had Child-Pugh A and 84.2% had performance status (PS) 0-1. Viral hepatitis was the predominant etiology (35%) and 18.3% had metabolic-associated liver disease. The majority were classified as BCLC-C stage (77.6%), 39.5% had portal vein invasion and 29.2% had extrahepatic metastasis. The most used regimen was atezolizumab+bevacizumab (n = 93) followed by durvalumab±tremelimumab (n = 11), with a median treatment duration of 3.8 months. Baseline endoscopy was performed in 82 (68.3%) patients and 38 of them presented esoophageal varices. After a median follow-up of 13.8 months (95% confidence interval (CI): 11.7 - 18.0), the median OS was 14.7 months (95% CI: 11.1 - 25.7). In the response-evaluable patients (n = 84), the ORR was 23.8% and the disease control rate was 50%. Immune-TRAEs were observed in 31 patients (4 with grade≥3) and 11 patients had bleeding events (4 gastroesophageal varices bleeding). ALBI-score 1 (p = 0.03) and partial response (p = 0.007) were significantly associated with better OS.
Conclusion: This observational study included subgroups that are not routinely enrolled in clinical trials, such as Child-Pugh B and PS>1. Nevertheless, the results in real life reproduce the safety and efficacy of immunotherapy observed in clinical trials. Baseline ALBI-score and partial response during treatment were favorable prognostic factors.
Corresponding author: Cecília Souza Freire (e-mail: ceciliasouzafreire@gmail.com).
No conflict of interest has been declared by the author(s).
Publication History
Article published online:
06 May 2025
© 2025. The Author(s). This is an open access article published by Thieme under the terms of the Creative Commons Attribution 4.0 International License, permitting copying and reproduction so long as the original work is given appropriate credit (https://creativecommons.org/licenses/by/4.0/)
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Cecília Souza Freire, Leonardo Gomes da Fonseca, Rodrigo Antonio Vieira Guedes, Marcos Castro Lyra, Rodrigo Tancredi, Mariana Bruno Siqueira, Maria de Lourdes Lopes de Oliveira, Duilio Reis da Rocha Filho, Lucila Soares da Silva Rocha, Guilherme Luiz Stelko Pereire, Paulo Marcelo Gehm Hoff. Real life safety and efficacy of immunotherapy for unresectable hepatocellular carcinoma in clinical practice. Brazilian Journal of Oncology 2025; 21.
DOI: 10.1055/s-0045-1808034