【摘要】 目的探索新药螺旋藻肽聚糖复合物胶囊(K-001)在晚期肝细胞癌(HCC)患者中的安全性和有效性。方法本研究为前瞻性、随机、双盲、阳性对照、全国多中心的Ⅱ期临床研究。自2012年9月至2016年3月,共入组100例晚期HCC患者,随机分为K-001组(试验组)和肝复乐胶囊组(对照组)。采用RECIST 1.1版标准评价客观疗效; NCI CTC 4.0版标准评价不良事件。结果治疗后,试验组和对照组的中位疾病进展时间(TTP)分别为1.48个月和1.41个月(P=0.946),中位总生存时间(OS)分别为5.17个月和4.57个月(P=0.374),差异均无统计学意义。无高危因素亚组分析显示,K-001有延长生存的趋势(7.0个月vs.4.67个月,P=0.447; 9.10个月vs.4.67个月,P=0.675)。K-001治疗后血红蛋白、白蛋白、淋巴细胞和嗜酸性粒细胞较对照组显著升高,且以上指标与死亡风险呈负相关;碱性磷酸酶和血小板/淋巴细胞比值(PLR)较对照组显著降低,且与死亡风险呈正相关。"无门脉侵犯"亚组中,K-001组治疗后疼痛轻于对照组(P=0.002)。K-001安全性良好,无与药物肯定或可能相关的严重不良事件发生。结论K-001治疗国人晚期HCC的疗效与肝复乐胶囊相似,分层分析显示,在部分亚组有生存获益的趋势和减轻癌痛的作用;同时,安全性良好,值得在特定人群扩大样本量进一步临床探索。更多还原

【Abstract】 Objective To investigate the safety and efficacy of Spirulina peptidoglycan complex( K-001) in advanced hepatocellular carcinoma( HCC) patients.Methods The multicencer,randomized,phase II,double blinded,positive controlled clinical study involved 100 patients with untreated local advanced or metastatic HCC in China.They were randomly assigned at a ratio of1: 1 to receive either K-001( n = 50) or Ganfule capsule( n = 50) treatment.Objective efficacy was evaluated by RECIST 1.1 and adverse events were evaluated by NCI CTC AE 4.0.Results The median time to tumor progress( TTP) in K-001 group and control group were 1.48 months and 1.41 months( P = 0.946),median overall survival( OS) were 5.17 months and 4.57 months( P =0.374),respectively.There was no significant difference in the two groups.Patients with no high risk trended to have longer OS( 7.0 months vs.4.67 months,P = 0.447; 9.10 months vs.4.67 months,P = 0.675),but no significant difference was observed.The hemoglobin,lymphocyte count,eosinophilic granulocyte count and albumin were significantly higher after K-001 treatment,rather than control group,which were negatively related to death.While the PLR and AKP were significantly lower after K-001 treatment,which were positively related to death.In the non-portal invasion subgroup,the pain of patients in K-001 group was less than that of control group.K-001 had excellent safety,and no definite drug related SAE was observed.Conclusion The effect of K-001 in Chinese advanced HCC patients is similar to Ganfule capsule.K-001 trended to have survival benefit and reducing pain in some subgroups.K-001 shows good safety in this clinical trial,and it is worth further clinical exploration in some specific population.更多还原