Anlotinib for Metastatic Progressed Pheochromocytoma and Paraganglioma: A Retrospective Study of Real-World Data

• DOI: 10.1210/jendso/bvae061
• Publication Date: 2024-04-01T14:06:04Z
• Authors: Rui Tian; Xiaochen Yao; Jieping Song; Jun Wang; Jingjing Fu; Liang Shi; Fei Yu; Pengjun Zhang; Chuan Zhang; Yudan Ni; Feng Wang
• Keywords: Real world data

Abstract Introduction Pheochromocytomas (PCC) and paragangliomas (PGL) (collectively PPGL) are a type of rare hypervascular neuroendocrine tumors that very challenging to treat. This study aimed determine the efficacy safety multi-tyrosine kinase inhibitor anlotinib for treatment locally advanced or metastatic (LA/M) PPGL. Methods A total 37 eligible patients with unresectable progressive LA/M PPGL were enrolled. Of them, 27 received alone (n = 19) in combination 8) radionuclide therapies, including peptide receptor therapy (PRRT) iodine 131 meta-iodobenzylguanidine (131I-MIBG). The primary endpoints included objective response rate (ORR), defined as partial (PR) complete (CR), disease-control rate, PR, CR, stable disease (SD). secondary progression-free survival (PFS), duration response, drug safety. Results In evaluation all patients, ORR was 44.44% (95% CI: 24.4%-64.5%) 96.29% 88.7%-100%). Twelve cases (44.44%) achieved 14 (51.85%) SD. median PFS 25.2 months 17.2 not reached). shorter monotherapy group than receiving (P .2). There no serious treatment-related AEs. Conclusion Anlotinib therapies shows promising PCC PGL. Multi-tyrosine inhibitors might represent novel therapeutic strategy PPGL; however, large-scale prospective randomized, blinded, controlled clinical research studies required.