An open-labeled, randomized, multicenter phase IIa study of gambogic acid injection for advanced malignant tumors
An open-labeled, randomized, multicenter phase IIa study of gambogic acid injection for advanced malignant tumors
复制标题
DOI：
10.3760/cma.j.issn.0366-6999.20122582
复制
发表时间：
2013-05-05
影响因子：
6.1
通讯作者：
Wang Jin-wan
中科院分区：
文献类型：
--
作者：
Chi Yihebali;Zhan Xiao-kai;Wang Jin-wan
背景藤黄酸是从中药藤黄(Garcinia Morella Desv.)中分离得到的一种纯活性化合物。基于I期研究的初步结果，这项IIa期研究比较了不同剂量方案对晚期恶性肿瘤患者的疗效和安全性。方法将未接受任何常规有效常规治疗或对现有常规治疗无效的晚期或转移性癌症患者随机分为两组，A组接受45 mg/m(2)静脉注射，2周为1~5天，B组接受45 mg/m(2)隔日静脉注射，共5次，共2周。结果A组患者21例，B组患者26例，均进入最终分析。A组和B组的ORR分别为14.3%和0%。由于其中一个值为零，所以无法分析两者之间的差异。疾病控制率A组为76.2%，B组为61.5%(P=0.0456)。观察到的不良反应大多为I级和II级，大多数患者在服用试验药物后发生。两组不良反应发生率无显著差异。结论本次IIa期探索性研究的初步结果表明，当剂量为45 mg/m(2)时，藤黄酸具有良好的安全性。在2周周期的第1-5天接受藤黄酸治疗的患者的DCR较大，但不良反应的发生率相似，与给药方案无关。
Background Gambogic acid is a pure active compound isolated from the traditional Chinese medicinal plant gamboge (Garcinia morella Desv.). Based on the preliminary results of a phase I study, this phase IIa study compared the efficacy and safety of different dosage schedules of gambogic acid in patients with advanced malignant tumors.Methods Patients with advanced or metastases cancer who had not received any effective routine conventional treatment or who had failed to respond to the existing conventional treatment were randomly assigned to receive either 45 mg/m(2) gambogic acid intravenously from Days 1 to 5 of a 2-week cycle (Group A), or 45 mg/m(2) every other day for a total of five times during a 2-week cycle (Group B). The primary endpoint was objective response rate (ORR).Results Twenty-one patients assigned to Group A and 26 to Group B were included in the final analysis. The ORRs were 14.3% in Group A and 0% in Group B. It was not possible to analyze the significant difference because one of the values was zero. The disease control rates (DCRs) were 76.2% in Group A and 61.5% in Group B (P=0.0456). The observed adverse reactions were mostly Grades I and II, and occurred in most patients after administration of the trial drug. There was no significant difference in the incidence of adverse reactions between the two arms.Conclusions The preliminary results of this phase IIa exploratory study suggest that gambogic acid has a favorable safety profile when administered at 45 mg/m(2). The DCR was greater in patients receiving gambogic acid on Days 1-5 of a 2-week cycle, but the incidence of adverse reactions was similar irrespective of the administration schedule.