Department of Clinical Epidemiology and Biostatistics, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand.
Oncology Clinic, Sriphat Medical Center, Faculty of Medicine, Chiang Mai University, Chiang Mai, Thailand.
Cancer Med. 2023 Feb;12(3):3046-3053. doi: 10.1002/cam4.5224. Epub 2022 Sep 9.
Multikinase inhibitors (MKIs) represent the main treatment options for advanced hepatocellular carcinoma (aHCC). However, accessibility in developing countries is limited. A chemotherapy, Fluorouracil and Oxaliplatin (FOLFOX), offers a less expensive treatment. Therefore, this study sought to compare the clinical effectiveness of FOLFOX with Sorafenib as a first-line treatment for aHCC in real-life practice.
A retrospective aHCC cohort from four Thai hospitals was investigated for patients who received FOLFOX or Sorafenib between 2013-2019. Multiple imputation by chained equations addressed missing covariate data in a treatment effect model using Weight-adjusted-censoring inverse-probability-weighted regression adjustment; overall survival (OS) and progression-free survival (PFS) were estimated.
A total of 504 patients were included, (Sorafenib [n = 382] and FOLFOX [n = 122]). The treatment effect model estimated a median OS for Sorafenib and FOLFOX of 11.38 and 8.22 months, representing a significantly shorter OS (95% confidence interval) of -3.16 (-6.21, -0.11) months for FOLFOX, p = 0.042. A significant shorter median PFS of FOLFOX to Sorafenib of -2.13 (-3.03, -1.24) months, p < 0.001, was reported.
Despite significantly shorter median OS and PFS than Sorafenib, FOLFOX still extended OS by 8.22 months. This evidence may offer clinical utility to physicians considering treatment options for aHCC in low resource settings.
多激酶抑制剂(MKIs)是治疗晚期肝细胞癌(aHCC)的主要选择。然而,发展中国家的可及性有限。化疗药物氟尿嘧啶和奥沙利铂(FOLFOX)提供了一种更便宜的治疗方法。因此,本研究旨在比较 FOLFOX 与索拉非尼作为一线治疗 aHCC 的临床效果,在真实实践中。
从四家泰国医院的 aHCC 回顾性队列中,调查了 2013-2019 年期间接受 FOLFOX 或索拉非尼治疗的患者。使用加权校正的删失逆概率加权回归调整的连锁方程多重插补处理治疗效果模型中的缺失协变量数据;总体生存(OS)和无进展生存(PFS)进行了估计。
共纳入 504 例患者,(索拉非尼 [n=382] 和 FOLFOX [n=122])。治疗效果模型估计索拉非尼和 FOLFOX 的中位 OS 分别为 11.38 和 8.22 个月,代表 FOLFOX 的 OS 明显更短(95%置信区间)-3.16(-6.21,-0.11)个月,p=0.042。FOLFOX 至索拉非尼的中位 PFS 明显更短-2.13(-3.03,-1.24)个月,p<0.001。
尽管 FOLFOX 的中位 OS 和 PFS 明显短于索拉非尼,但 FOLFOX 仍将 OS 延长了 8.22 个月。这一证据可能为在资源有限的环境中考虑 aHCC 治疗方案的医生提供临床实用性。