Leng Jiahua, Wu Jing, Han Rong, Wei Huan, Chen Ken
Key Laboratory of Carcinogenesis and Translational Research (Ministry of Education/Beijing), Gastrointestinal Center, Unit III, Peking University Cancer Hospital and Institute, Beijing, China.
Department of Medical Insurance, Peking University Cancer Hospital and Institute, Key Laboratory of Carcinogenesis and Translational Research (Ministry of Education), Beijing, China.
J Gastrointest Oncol. 2025 Aug 30;16(4):1597-1609. doi: 10.21037/jgo-2024-1015. Epub 2025 Aug 27.
In recent years, the emerging new drugs in the Chinese market diversified the choice of systemic treatment, especially second-line (2L), for unresectable or advanced hepatic carcinoma (HC) patients, and their inclusion into the National Reimbursement Drug List (NRDL) further changed the clinical practice. The objective of this study was to describe the 2L treatment pattern among Chinese unresectable or advanced HC patients in a real-world setting.
All adult unresectable or advanced HC patients receiving 2L treatment were retrospectively identified from Tianjin Healthcare and Medical Big Data Platform, a regional electronic health record database in Tianjin, China. Patients were observed between 2018 and 2021. A descriptive analysis was conducted for study outcomes for the overall cohort and by subgroups before and after NRDL 2020 version implementation.
A total of 123 patients were eligible for study inclusion. Targeted therapy (n=93, 75.6%) was the most commonly used 2L treatment category and regorafenib monotherapy (n=71, 57.7%) was the most common treatment type. Among patients receiving regorafenib as 2L treatment, sorafenib was the most commonly used first-line treatment. The percentage of patients using 2L treatments recommended by 2020 China Society of Clinical Oncology (CSCO) guideline changed from 87.2% to 59.2% after NRDL 2020 version implementation. The average daily doses of targeted therapies were generally lower than the labeling doses, while those of immune therapies were close to labeling doses. There was a trend towards increased treatment duration and reduced direct medical cost after NRDL 2020 version implementation.
Targeted therapy, especially regorafenib, was the most common 2L treatment for unresectable or advanced HC in China. Future study is necessary to evaluate their effectiveness and safety as 2L treatment for unresectable or advanced HC patients in China.
近年来，中国市场上新兴的新药使不可切除或晚期肝癌（HC）患者全身治疗的选择更加多样化，尤其是二线（2L）治疗，并且这些药物被纳入国家医保药品目录（NRDL）进一步改变了临床实践。本研究的目的是描述中国不可切除或晚期HC患者在真实世界中的二线治疗模式。
从中国天津的一个区域电子健康记录数据库——天津医疗大数据平台中，回顾性识别所有接受二线治疗的成年不可切除或晚期HC患者。观察时间为2018年至2021年。对整个队列以及2020版NRDL实施前后的亚组研究结果进行描述性分析。
共有123例患者符合纳入研究的条件。靶向治疗（n = 93，75.6%）是最常用的二线治疗类别，瑞戈非尼单药治疗（n = 71，57.7%）是最常见的治疗类型。在接受瑞戈非尼作为二线治疗的患者中，索拉非尼是最常用的一线治疗药物。2020版NRDL实施后，使用2020年中国临床肿瘤学会（CSCO）指南推荐的二线治疗的患者比例从87.2%降至59.2%。靶向治疗的平均每日剂量通常低于标注剂量，而免疫治疗的平均每日剂量接近标注剂量。2020版NRDL实施后，治疗持续时间有延长趋势，直接医疗费用有降低趋势。
靶向治疗，尤其是瑞戈非尼，是中国不可切除或晚期HC最常见的二线治疗药物。未来有必要开展研究，评估其作为中国不可切除或晚期HC患者二线治疗的有效性和安全性。
