Vita-Salute San Rafaele University, Milan, Italy.
Department of Medical Oncology, San Rafaele Scientifc Institute IRCCS, Milan, Italy.
Liver Int. 2021 Jun;41(6):1389-1397. doi: 10.1111/liv.14817. Epub 2021 Feb 20.
Data from common clinical practice were used to generate balanced cohorts of patients receiving either sorafenib or lenvatinib, for unresectable hepatocellular carcinoma, with the final aim to investigate their declared equivalence.
Clinical features of lenvatinib and sorafenib patients were balanced through inverse probability of treatment weighting (IPTW) methodology, which weights patients' characteristics and measured outcomes of each patient in both treatment arms. Overall survival was the primary endpoint and occurrence of adverse events was the secondary.
The analysis included 385 patients who received lenvatinib, and 555 patients who received sorafenib. In the unadjusted cohort, lenvatinib did not show a survival advantage over sorafenib (HR: 0.85, 95% CI 0.70-1.02). After IPTW adjustment, lenvatinib still not returned a survival advantage over sorafenib (HR: 0.82, 95% CI: 0.62-1.07) even in presence of balanced baseline characteristics. Lenvatinib provided longer survival than sorafenib in patients previously submitted to TACE (HR: 0.69), with PS of 0 (HR: 0.73) or without extrahepatic disease (HR: 0.69).
Present results confirmed randomized controlled trial in the real-life setting, but also suggests that in earlier stages some benefit can be expected.
利用来自普通临床实践的数据，为不可切除的肝细胞癌患者生成接受索拉非尼或仑伐替尼治疗的平衡队列，最终目的是研究它们所宣称的等效性。
通过逆概率治疗加权（IPTW）方法平衡仑伐替尼和索拉非尼患者的临床特征，该方法对每个治疗组中患者的特征和测量结果进行加权。总生存期是主要终点，不良事件的发生是次要终点。
该分析纳入了 385 名接受仑伐替尼治疗的患者和 555 名接受索拉非尼治疗的患者。在未调整的队列中，仑伐替尼与索拉非尼相比并未显示生存优势（HR：0.85，95%CI 0.70-1.02）。在 IPTW 调整后，即使基线特征平衡，仑伐替尼与索拉非尼相比仍未显示出生存优势（HR：0.82，95%CI：0.62-1.07）。仑伐替尼在先前接受 TACE（HR：0.69）、PS 为 0（HR：0.73）或无肝外疾病（HR：0.69）的患者中提供了比索拉非尼更长的生存时间。
目前的结果证实了真实环境下的随机对照试验，但也表明在早期阶段可能会有一些获益。
