Lenvatinib as a Therapeutic Option in Unresectable Metastatic Pheochromocytoma and Paragangliomas

Lauren Hassan Nelson, Harry Fuentes-Bayne, Jun Yin, Erik Asmus, Mabel Ryder, John C. Morris, Crystal R. Hilger, Keith C. Bible, Ashish V. Chintakuntlawar, Sarika N. Rao

Research output: Contribution to journalArticlepeer-review


Context: Metastatic pheochromocytomas and paragangliomas (mPPGL) are rare vascular neuroendocrine tumors that highly express vascular growth factors. Systemic treatment options in cases of unresectable multisite disease are limited. Multikinase inhibitors that inhibit angiogenesis, such as lenvatinib, have proven effective in several other malignancies, and may be a viable option for mPPGL. Objective: We aimed to evaluate the efficacy of lenvatinib as salvage therapy in mPPGLs. Methods: This was a retrospective analysis of mPPGL patients ≥ 18 years of age who received lenvatinib from 2015 to 2020 at a tertiary referral center. Patients were started on lenvatinib 20 mg daily and dose was adjusted according to tolerance or disease progression. Results: Eleven patients were included. Median treatment duration was 14.7 months (95% CI, 2.3-NE). Treatment was discontinued due to disease progression, adverse events, or death. Overall survival at 12 months was 80.8% (95% CI, 42.3-94.9%) but its median was not reached. Median progression-free survival was 14.7 months (95% CI, 1.7-NE). Among the 8 patients with measurable disease, overall response rate was 63%, as 5/8 experienced a partial response and 3/8 had stable disease. Worsening hypertension and anemia were the most common adverse events. Conclusion: Lenvatinib may be a viable treatment option for mPPGL, although at the potential risk of worsening hypertension. Larger, multicenter studies are needed to better characterize treatment efficacy.

Original languageEnglish (US)
Article numberbvac044
JournalJournal of the Endocrine Society
Issue number5
StatePublished - May 1 2022


  • cancer
  • multikinase inhibitor
  • paraganglioma
  • pheochromocytoma

ASJC Scopus subject areas

  • Endocrinology, Diabetes and Metabolism


Dive into the research topics of 'Lenvatinib as a Therapeutic Option in Unresectable Metastatic Pheochromocytoma and Paragangliomas'. Together they form a unique fingerprint.

Cite this