The clinical implications of trial endpoints in immunotherapy for hepatocellular carcinoma.

IF 3.8 3区医学 Q2 GASTROENTEROLOGY & HEPATOLOGY

Expert Review of Gastroenterology & Hepatology Pub Date : 2025-05-12 DOI:10.1080/17474124.2025.2500369

Xun Zhao, Talia Dufault, Gonzalo Sapisochin, Anna Saborowski, Arndt Vogel

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Abstract

Introduction: Investigative work in the treatment of hepatocellular carcinoma is rapidly growing with the advent of immunotherapy. Nonetheless, trial endpoints and, more importantly, clinically meaningful endpoints need to be accurately chosen depending on the phase of trial and the patient population studied. We provide a scoping review focusing on trial endpoints on the use of immunotherapy in hepatocellular carcinoma.

Areas covered: We searched PubMed and Google Scholar for prospective phase II and III trials using immunotherapy, whether in the neoadjuvant, adjuvant, bridging, downstaging, or palliative settings, while discussing the clinical implications of trial endpoints.

Expert opinion: The field of immune oncology is rapidly progressing and has become the standard of care in advanced hepatocellular carcinoma. However, the role of immunotherapy in the treatment of early and intermediate stage hepatocellular carcinoma is yet to be defined. Prospective trials for all stages of disease must strive for endpoints that are not only statistically significant but also clinically consequential. Whereas overall response rate may be a reasonable trial endpoint in phase II trials, phase III trials should rather aim for the improvement of overall survival or quality of life to have clinically meaningful impacts.

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肝细胞癌免疫治疗试验终点的临床意义

导论：随着免疫疗法的出现，肝细胞癌治疗的调查工作正在迅速发展。然而，试验终点，更重要的是，临床有意义的终点需要根据试验的阶段和研究的患者群体来准确选择。我们对肝细胞癌免疫治疗的试验终点进行了范围综述。涵盖领域：我们检索了PubMed和谷歌Scholar中使用免疫治疗的前瞻性II期和III期试验，无论是在新辅助、辅助、桥化、降期还是姑息治疗的情况下，同时讨论了试验终点的临床意义。专家意见：免疫肿瘤学领域正在迅速发展，并已成为晚期肝细胞癌的治疗标准。然而，免疫疗法在早期和中期肝细胞癌治疗中的作用尚未明确。所有疾病阶段的前瞻性试验必须争取终点不仅具有统计学意义，而且具有临床意义。在II期试验中，总缓解率可能是一个合理的试验终点，而III期试验的目标应该是改善总体生存期或生活质量，从而产生有临床意义的影响。

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来源期刊

Expert Review of Gastroenterology & Hepatology GASTROENTEROLOGY & HEPATOLOGY-

CiteScore

6.80

自引率

2.60%

发文量

审稿时长

6-12 weeks

期刊介绍： The enormous health and economic burden of gastrointestinal disease worldwide warrants a sharp focus on the etiology, epidemiology, prevention, diagnosis, treatment and development of new therapies. By the end of the last century we had seen enormous advances, both in technologies to visualize disease and in curative therapies in areas such as gastric ulcer, with the advent first of the H2-antagonists and then the proton pump inhibitors - clear examples of how advances in medicine can massively benefit the patient. Nevertheless, specialists face ongoing challenges from a wide array of diseases of diverse etiology.