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Lenvatinib as a Therapeutic Option in Unresectable Metastatic Pheochromocytoma and Paragangliomas
Lauren Hassan Nelson1, Harry Fuentes-Bayne2, Jun Yin3
1Division of Endocrinology, Mayo Clinic, Jacksonville, FL 32224, USA.
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.
Insights
Lenvatinib shows promise as a salvage therapy for metastatic pheochromocytomas and paragangliomas (mPPGL), offering a response in most patients. However, potential risks like worsening hypertension require careful consideration.
Area of Science:
- Oncology
- Vascular Biology
- Neuroendocrinology
Background:
- Metastatic pheochromocytomas and paragangliomas (mPPGL) are rare neuroendocrine tumors with limited systemic treatment options for unresectable disease.
- These tumors highly express vascular growth factors, suggesting anti-angiogenic therapies may be effective.
- Multikinase inhibitors, including lenvatinib, are used in other cancers and may be a viable option for mPPGL.
Purpose of the Study:
- To evaluate the efficacy of lenvatinib as a salvage therapy in patients with metastatic pheochromocytomas and paragangliomas (mPPGL).
Main Methods:
- Retrospective analysis of 11 mPPGL patients aged ≥18 years treated with lenvatinib (20 mg daily) between 2015-2020.
- Dose adjustments were made based on tolerance or disease progression.
- Outcomes assessed included overall survival, progression-free survival, objective response rate, and adverse events.
Main Results:
- Median treatment duration was 14.7 months.
- Median progression-free survival was 14.7 months, with 80.8% overall survival at 12 months.
- An overall response rate of 63% was observed in patients with measurable disease (5 partial responses, 3 stable disease). Worsening hypertension and anemia were common adverse events.
Conclusions:
- Lenvatinib demonstrates potential as a viable salvage treatment for mPPGL.
- Careful monitoring for adverse events, particularly hypertension, is crucial.
- Larger, multicenter studies are necessary to confirm efficacy and safety in a broader mPPGL population.
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