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Cancer drug may benefit patients with metastatic pheochromocytoma, paraganglioma
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BOSTON — Use of the antiangiogenic tyrosine kinase inhibitor cabozantinib, approved for treatment of medullary thyroid cancer and renal cell carcinoma, appears to be a safe and effective therapy for treating rare unresectable metastatic pheochromocytomas and paragangliomas, according to preliminary data from a phase 2 study presented here.
“These tumors are frequently associated with an increased secretion of catecholamines, predisposing patients to cardiovascular and gastrointestinal disease,” Camilo Jimenez, MD, associate professor in the department of endocrine neoplasia and hormonal disorders at The University of Texas MD Anderson Cancer Center, told Endocrine Today. “Patients exhibit a decreased overall survival because of complications related to the hormonal excess and their frequently massive tumor burden. Currently, there are no FDA-approved therapies for the treatment of these patients.”
Jimenez and colleagues assigned 14 patients with histologically confirmed metastatic pheochromocytoma (n = 7) or paraganglioma (n = 7) with disease progression over the past year (median age, 53 years; 10 men; five with SDHB gene mutations; eight with predominantly bone metastases) to 60 mg cabozantinib (Cabometyx, Exelixis) daily with dose reduced to 40 mg or 20 mg depending on tolerability in 11 participants.
Progression-free survival before treatment was a mean of 5 months, which increased to 12.1 months after treatment. Researchers observed tumor shrinkage with six participants exhibiting a greater than 30% reduction in disease progression, and three with a moderate 15% to 30% reduction; five participants showed disease stabilization relative to bone metastases. Improvements in blood pressure were observed in six participants.
Adverse events were mostly grade 1 to 2 dysgeusia, hand and foot syndrome and fatigue.
“Cabozantinib is the tyrosine kinase inhibitor associated with the most impressive clinical responses reported to date in patients with metastatic pheochromocytomas and paragangliomas,” Jimenez said.
“The most surprising aspects related to this clinical trial are the impressive response rate to the drug, the fact that no severe cardiovascular toxicity has been noticed, demonstration of acceptable toxicity with manageable side effects with supportive measures and/or dose reduction, [and] positive clinical responses in SDHB carriers and in patients with apparently sporadic tumors,” he said – by Jill Rollet
Reference:
Jimenez C, et al. Abstract 142. Presented at: AACE Annual Scientific and Clinical Congress; May 16-20, 2018; Boston.
Disclosure: Jimenez reports no relevant financial disclosures.