Division of Hepatology, Department of Internal Medicine, College of Medicine, The Catholic University of Korea, #222 Banpo-daero, Seocho-gu, Seoul, 06591, Republic of Korea.
The Catholic University Liver Research Center, Seoul, Republic of Korea.
Hepatol Int. 2021 Feb;15(1):137-145. doi: 10.1007/s12072-020-10131-0. Epub 2021 Jan 26.
Although sorafenib has been used to treat advanced hepatocellular carcinoma (HCC), the efficacy of sorafenib in patients with recurrent HCCs after liver transplantation (LT) has not been compared with that in patients without LT (non-LT).
Between 2008 and 2019, a total of 832 consecutive HCC patients treated with sorafenib (790 in the non-LT group and 42 in the LT group) were enrolled. The primary outcome was overall survival (OS). Secondary outcomes were time-to-progression (TTP), objective response rate (ORR) and disease control rate (DCR). Treatment outcomes were assessed by multiple subgroup analyses and propensity-score matching (PSM).
The median follow-up duration was 152.5 days. The LT group was younger and had smaller intrahepatic HCC than the non-LT group. The LT group showed significantly better OS (16.8 vs. 7.1 months, p < 0.001), TTP, ORR and DCR than the non-LT group. The superior efficacy of sorafenib in the LT group was corroborated in multiple subgroup analyses stratified by metastasis, effective sorafenib maintenance dose, or Child-Turcotte-Pugh class A. LT was identified as an independent factor for favorable OS. Intrahepatic HCC was the strongest tumor-related factor for both OS and TTP and was significantly associated with tumor response and hepatic function. Finally, subanalyses including only patients with small intrahepatic HCC or PSM modeling showed no difference in sorafenib efficacy between the LT and the non-LT groups.
Sorafenib provides better outcomes in the LT setting than the non-LT setting. This benefit may be associated with the smaller intrahepatic HCC coupled with preserved hepatic function in LT recipients.
虽然索拉非尼已被用于治疗晚期肝细胞癌（HCC），但它在肝移植（LT）后 HCC 复发患者中的疗效尚未与未接受 LT（非 LT）的患者进行比较。
在 2008 年至 2019 年间，共纳入 832 例接受索拉非尼治疗的 HCC 患者（非 LT 组 790 例，LT 组 42 例）。主要结局是总生存期（OS）。次要结局是无进展生存期（TTP）、客观缓解率（ORR）和疾病控制率（DCR）。通过多项亚组分析和倾向评分匹配（PSM）评估治疗结局。
中位随访时间为 152.5 天。LT 组比非 LT 组更年轻，肝内 HCC 更小。LT 组的 OS（16.8 个月比 7.1 个月，p<0.001）、TTP、ORR 和 DCR 明显优于非 LT 组。在分层为转移、有效索拉非尼维持剂量或 Child-Turcotte-Pugh 分级 A 的多项亚组分析中，LT 组的索拉非尼疗效均较好。LT 被确定为 OS 有利的独立因素。肝内 HCC 是 OS 和 TTP 的最强肿瘤相关因素，与肿瘤反应和肝功能显著相关。最后，仅包括肝内 HCC 较小的患者或 PSM 模型的亚组分析显示，LT 组和非 LT 组的索拉非尼疗效无差异。
与非 LT 组相比，LT 组中索拉非尼的疗效更好。这种益处可能与 LT 受者较小的肝内 HCC 加上保留的肝功能有关。
