Abstract:

Objective To evaluate the efficacy and safety of anlotinib monotherapy and combined therapy in patients with advanced pheochromocytoma/paraganglioma. Methods Nine patients with advanced pheochromocytoma/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.

Key words: anlotinib, pheochromocytoma, paraganglioma, efficacy, safety