Mohamed Yehia I, Lee Sunyoung, Xiao Lianchun, Hassan Manal M, Qayyum Aliya, Hiatia Rikita, Pestana Roberto Carmagnani, Haque Abedul, George Bhawana, Rashid Asif, Duda Dan G, Elghazaly Hesham, Wolff Robert A, Morris Jeffrey S, Yao James, Amin Hesham M, Kaseb Ahmed O
Department of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
Oncotarget. 2021 Apr 13;12(8):756-766. doi: 10.18632/oncotarget.27924.
Sorafenib was the first systemic therapy approved for the treatment of Child-Turcotte-Pugh (CTP) class A patients with advanced hepatocellular carcinoma (HCC). However, there are no biomarkers to predict survival and treatment outcomes and guide HCC systemic therapy. Type 1 insulin-like growth factor (IGF-1)/CTP composite score has emerged as a potential hepatic reserve assessment tool. Our study investigated the association of the IGF/CTP score with overall survival (OS) and progression-free survival (PFS) of HCC patients treated with sorafenib.
In this prospective study, patients with HCC were treated with sorafenib and followed up until progression/death. We calculated the IGF/CTP score and used the Kaplan-Meier method and log-rank test to estimate and compare the time-to-event outcomes between patient subgroups.
171 patients were included, 116 of whom were CTP class A. Median PFS for IGF/CTP score AA and AB patients were 6.88 and 4.28 months, respectively ( = 0.1359). Median OS for IGF/CTP score AA and AB patients were 14.54 and 7.60 months, respectively ( = 0.1378). The PFS and OS was superior in AA patients, but the difference was not significant, likely due to the sample size. However, there was a significant difference in early OS and PFS curves between AA and AB ( = 0.0383 and = 0.0099), respectively.
In CTP class A patients, IGF/CTP score B was associated with shorter PFS and OS, however, study was underpowered to reach statistical significance. If validated in larger cohorts, IGF/CTP score may serve as stratification tool in clinical trials, a hepatic reserve assessment tool for HCC outcomes prediction and to assist in therapy decisions.
索拉非尼是首个被批准用于治疗Child-Turcotte-Pugh(CTP)A级晚期肝细胞癌(HCC)患者的全身治疗药物。然而,目前尚无生物标志物可用于预测生存和治疗结果以及指导HCC的全身治疗。1型胰岛素样生长因子(IGF-1)/CTP综合评分已成为一种潜在的肝储备评估工具。我们的研究调查了IGF/CTP评分与接受索拉非尼治疗的HCC患者的总生存期(OS)和无进展生存期(PFS)之间的关联。
在这项前瞻性研究中,HCC患者接受索拉非尼治疗并随访至病情进展/死亡。我们计算了IGF/CTP评分,并使用Kaplan-Meier方法和对数秩检验来估计和比较患者亚组之间的事件发生时间结局。
共纳入171例患者,其中116例为CTP A级。IGF/CTP评分AA和AB患者的中位PFS分别为6.88个月和4.28个月( = 0.1359)。IGF/CTP评分AA和AB患者的中位OS分别为14.54个月和7.60个月( = 0.1378)。AA患者的PFS和OS更佳,但差异不显著,可能是由于样本量的原因。然而,AA和AB患者的早期OS和PFS曲线存在显著差异(分别为 = 0.0383和 = 0.0099)。
在CTP A级患者中,IGF/CTP评分B与较短的PFS和OS相关,然而,该研究的效能不足以达到统计学显著性。如果在更大的队列中得到验证,IGF/CTP评分可作为临床试验中的分层工具、用于预测HCC结局的肝储备评估工具以及辅助治疗决策。