The following is a summary of “Anlotinib for Metastatic Progressed Pheochromocytoma and Paraganglioma: A Retrospective Study of Real-World Data,” published in the June 2024 issue of Endocrinology by Tian, et al.

Pheochromocytomas (PCC) and paragangliomas (PGL) (collectively known as PPGL) are rare hypervascular neuroendocrine tumors that pose significant treatment challenges. For a study, researchers sought to assess the efficacy and safety of the multi-tyrosine kinase inhibitor anlotinib in treating locally advanced or metastatic (LA/M) PPGL.

A total of 37 eligible patients with unresectable or progressive LA/M PPGL were enrolled, with 27 patients receiving anlotinib alone (n = 19) or in combination (n = 8) with radionuclide therapies such as peptide receptor radionuclide therapy (PRRT) and iodine 131 meta-iodobenzylguanidine (131I-MIBG). The primary endpoints included objective response rate (ORR) and disease-control rate, while secondary endpoints comprised progression-free survival (PFS), duration of response, and drug safety.

Among the 27 patients evaluated for efficacy, the ORR was 44.44% (95% CI: 24.4%-64.5%), and the disease-control rate was 96.29% (95% CI: 88.7%-100%). Notably, 12 cases (44.44%) achieved partial response (PR), while 14 (51.85%) exhibited stable disease (SD). The median PFS was 25.2 months (95% CI: 17.2 months to not reached), with a trend of shorter PFS observed in the anlotinib monotherapy group compared to the combination therapy group (P = .2). No serious treatment-related adverse events (AEs) were reported.

Anlotinib, either as monotherapy or in combination with radionuclide therapies, demonstrates promising efficacy and safety for treating LA/M PCC and PGL. Multi-tyrosine kinase inhibitors hold potential as a novel therapeutic strategy for patients with PPGL. However, large-scale prospective randomized, blinded, controlled clinical trials were warranted to confirm these findings.

Reference: academic.oup.com/jes/article/8/6/bvae061/7639138