安罗替尼单药及联合方案治疗晚期嗜铬细胞瘤/副神经节瘤的疗效与安全性研究

Efficacy and safety of anlotinib monotherapy and combinated therapy in the treatment of advanced pheo⁃chromocytoma/paraganglioma

目的探讨晚期嗜铬细胞瘤/副神经节瘤(pheochromocytoma and paraganglioma,PPGL)患者接受安罗替尼单药及联合方案治疗的疗效和安全性。方法纳入2018年1月至2023年8月于中山大学肿瘤防治中心泌尿外科就诊的晚期嗜铬细胞瘤/副神经节瘤患者9例;根据患者接受的不同治疗方案,分为4组:安罗替尼单药组3例,安罗替尼联合PD-1单抗免疫治疗组3例,安罗替尼联合免疫及化疗组2例,安罗替尼联合化疗组1例;分析安罗替尼不同治疗方案的有效性及安全性。结果接受安罗替尼单药及联合治疗的客观反应率(objective response rate,ORR)(44%),部分缓解(partial response,PR)(44%),疾病稳定(stable disease,SD)(44%),疾病进展(progressive disease,PD)(11%),疾病控制率(disease control rate,DCR)(89%),其中SDH基因突变患者ORR 100%,分别为SDHB和SDHD;中位总生存时间(overall survival,OS)为16.3月(IQR:11.3~21.8个月);中位无进展生存时间(progression free survival,PFS)为16.3月(IQR:9.8~20.8个月);药物不良反应事件≥3/4级者有2例,均为高血压。结论安罗替尼单药及联合方案治疗晚期嗜铬细胞瘤/副神经节瘤具有初步的疗效和可控的安全性。

Objective To evaluate the efficacy and safety of anlotinib monotherapy and combined therapy in patients with advanced pheochromocytoma/paraganglioma.Methods Nine patients with advanced pheochromo-cytoma/paraganglioma(PPGL)who were admitted to the Department of Urology,Sun Yat-sen University Cancer Center from January 2018 to June 2023 were collected.Patients were divided into four groups according to different treatments:anlotinib monotherapy group(3 patients),anlotinib combined with PD-1 monoclonal antibody immunotherapy group(3 patients),anlotinib combined with immunotherapy and chemotherapy group(2 patients),and anlotinib combined with chemotherapy group(1 patients).The effectiveness and safety of different treatment regiments of anlotinib were analyzed.Results Objective response rate(ORR):(44%),Partial response(PR):(44%),Stable disease(SD):(44%),Progressive disease(PD):(11%),Disease control rate(DCR):(89%).The ORR of 2 patients with SDH gene mutation,SDHB and SDHD respectively,was 100%.Median overall survival time(OS)was 16.3 months(IQR:11.3~21.8 months).Median progression-free survival(PFS)was 16.3 months(IQR:9.8~20.8 months).There were 2 patients with adverse events grade≥3/4,all of which were hypertension.Conclusions Anlotinib monotherapy and combined therapy have preliminary efficacy and manageable safety in the treatment of advanced pheochromocytoma/paraganglioma.