Phase 2 trial shows 95.5% disease control in metastatic pheochromocytoma patients treated with anlotinib, indicating potential benefits but highlighting adverse events.
Background Metastatic Pheochromocytoma and Paraganglioma (MPP) currently lack definitive curative therapies. The treatment of MPP presents a formidable challenge. Anlotinib hydrochloride, characterized as a multi-targeted tyrosine kinase receptor inhibitor, has demonstrated potential in this domain. The principal aim of our study is to scrutinize the efficacy and safety profiles of anlotinib hydrochloride in patients suffering from MPP. Methods A phase 2 trial (NCT04860700) evaluated anlotinib in progressive MPP patients. The primary objective was to determine the disease control rate (DCR) and objective response rate (ORR) as determined by the RECIST 1.1 criteria. Secondary objectives were to evaluate the biochemical response, progression-free survival (PFS) and safety. Results Twenty-nine patients with MPP were enrolled. 24.1% (7/29) of patients discontinued the treatment after 1-2 cycles because of a significant increase in hormone levels or adverse events. Among 22 patients who could be evaluated by the RECIST 1.1 criteria. The overall DCR was 95.5% (21/22) and the ORR was 31.8% (7/22). The PFS was calculated to be 22.6 ± 3.2 months, There is no significant difference in ORR or PFS between patients with and without germline mutations. The most common adverse events included arterial hypertension (24/29, 82.8%), subclinical primary hypothyroidism (11/29, 37.9%), hyperlipidemia (10/29, 34.5%), rash (8/29, 27.6%) and fatigue (8/29, 27.6%). Conclusions Among the evaluable patients,Anlotinib showed promising efficacy with high DCR. Close monitoring remains essential due to treatment-related adverse events.
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Cui et al. (2025) studied this question.
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