background：compared with single-drug tace, our previous phase iii study demonstrated that triple-drug transarterial chemoembolization (tace) prolonged overall survival (os) in patients with unresectable hepatocellular carcinoma (hcc). the aim of this study was to find which patients can benefit from the triple drugs tace compared with single-drug tace.
methods：patients in the triple-drug tace arm received sponge embolization and emulsions composed of 50 mg epirubicin, 50 mg lobaplatin, 6 mg mitomycin c, and lipiodol, while patients in the single-drug tace arm received sponge embolization and emulsions composed of 50 mg epirubicin and lipiodol. from july 2007 to november 2009, 244 patients (224 men and 20 women; age ranged from 21 to 75 years) from our phase iii study formed the initial cohort. from january 2010 to june 2015, external validation cohort was composed of 449 patients (411 men and 38 women; age ranged from 18 to 75 years) from another institution. the validation cohort after propensity score matching (psm) (n = 374) was analyzed. cox proportional hazard model was used to evaluate the interaction term between treatments for each subgroup. this retrospective study was approved by the institutional review board at each center.
方法：三药tace组患者分别给予50 mg表柔比星、50 mg洛铂、6 mg丝裂霉素c及碘化油组成的乳剂进行海绵栓塞，单药tace组患者给予50 mg表柔比星和碘油醇组成的乳剂进行海绵栓塞。2007年7月至2009年11月，我们第三阶段研究中的244例患者(男性224例，女性20例；年龄21-75岁)形成了最初的研究队列。2010年1月至2015年6月，外部验证队列由来自另一个机构的449例患者组成(男性411例，女性38例；年龄从18-75岁)。分析倾向性评分匹配(psm)后的验证队列(n = 374)。采用cox比例危险模型评估各亚组间治疗的相互作用项。这项回顾性研究由各中心的机构审查委员会进行批准。
results：no difference was observed in the baseline characteristic of three cohorts. this exploratory analysis showed that triple-drug tace brought a survival benefit in the initial cohort, validation cohort (before psm), and validation cohort (after psm) compared with single-drug tace. the outcomes of three cohorts all showed that a significantly greater os triple-drug chemotherapy benefit versus single-drug chemotherapy was seen in patients with large tumors (larger than 10 cm) while no survival difference was seen in patients with small tumors (10 cm or smaller).
结果：三个队列患者的基线特征无显著性差异。本探索性分析表明，与单药tace相比，三药tace在初始队列、验证队列(psm前)和验证队列(psm后)中均有生存获益。三个队列的结果均显示，三药tace治疗大肿瘤（直径＞10 cm）患者的os显著高于单药tace，而在小肿瘤(直径≤10 cm)患者的生存获益上，两组无差异。
conclusions：triple-drug tace seems to benefit patients with hcc larger than 10 cm in particular compared with single-drug tace.