Vanderbilt University School of Medicine. Nashville, TN.
Department of Gastroenterology, Hospital Privado Universitario de Cordoba, Instituto Universitario de Ciencias Biomedicas de Cordoba, Cordoba.
J Clin Gastroenterol. 2019 Jul;53(6):464-469. doi: 10.1097/MCG.0000000000001085.
We aim to describe the efficacy, safety profile, and variables associated with survival in patients with hepatocellular carcinoma (HCC) treated with sorafenib in South America.
Sorafenib has been shown to improve survival in patients with advanced HCC. There are few data on sorafenib use for HCC in South America.
We performed a retrospective analysis of HCC cases treated with sorafenib from 8 medical centers in 5 South American countries, between January 2010 and June 2017. The primary endpoint was overall survival (OS), which was defined as time from sorafenib initiation to death or last follow-up. Risk factors for decreased OS were assessed using Cox proportional hazard regression and log-rank tests.
Of 1336 evaluated patients, 127 were treated with sorafenib and were included in the study. The median age of individuals was 65 years (interquartile range, 55 to 71) and 70% were male individuals. Median OS in all patients was 8 months (interquartile range, 2 to 17). Variables associated with survival on multivariate analysis were platelets >/<250,000 mm (2 vs. 8 mo, P=0.01) and Barcelona Clinic Liver Cancer (BCLC) stage (A/B, 13 vs. C/D, 6 mo; P=0.04). In a subanalysis of patients with BCLC stage C, platelets >/<250,000 mm were also independently associated with survival (2 vs. 5.5 mo, P=0.03). Patients lived longer if they experienced any side effects from sorafenib use (11 vs. 2 mo, P=0.009). Patients who stopped sorafenib because of side effects had shorter survival compared with patients who were able to tolerate side effects and continue treatment (7.5 vs. 13 mo, P=0.01).
Pretreatment elevation of platelets and advanced BCLC stage were independently associated with poor survival on sorafenib in a South American cohort.
我们旨在描述在南美洲接受索拉非尼治疗的肝细胞癌（HCC）患者的疗效、安全性概况以及与生存相关的变量。
索拉非尼已被证明可改善晚期 HCC 患者的生存。在南美洲，有关索拉非尼治疗 HCC 的数据很少。
我们对 2010 年 1 月至 2017 年 6 月期间来自南美洲 5 个国家的 8 家医疗中心的 1336 例 HCC 病例进行了回顾性分析，这些患者接受了索拉非尼治疗。主要终点是总生存期（OS），定义为从索拉非尼开始治疗到死亡或最后一次随访的时间。使用 Cox 比例风险回归和对数秩检验评估 OS 降低的危险因素。
在评估的 1336 名患者中，有 127 名患者接受了索拉非尼治疗并被纳入研究。个体的中位年龄为 65 岁（四分位间距为 55 至 71），70%为男性。所有患者的中位 OS 为 8 个月（四分位间距为 2 至 17）。多变量分析中与生存相关的变量是血小板>250,000/mm 和血小板<250,000/mm（2 个月 vs. 8 个月，P=0.01）和巴塞罗那临床肝癌（BCLC）分期（A/B，13 个月 vs. C/D，6 个月；P=0.04）。在 BCLC 分期 C 的患者亚分析中，血小板>250,000/mm 也与生存独立相关（2 个月 vs. 5.5 个月，P=0.03）。与因副作用而停止索拉非尼治疗的患者相比，经历任何索拉非尼副作用的患者生存时间更长（11 个月 vs. 2 个月，P=0.009）。因副作用而停止索拉非尼治疗的患者与能够耐受副作用并继续治疗的患者相比，生存时间更短（7.5 个月 vs. 13 个月，P=0.01）。
在南美洲队列中，血小板升高和晚期 BCLC 分期是与索拉非尼治疗不良预后相关的独立因素。
