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Cabozantinib Shows Promising Activity Among Patients With Progressive, Unresectable Metastatic Phaeochromocytomas and Paragangliomas

Stephanie Holland 

Results from the phase 2 Natalie trial found that cabozantinib, an antiangiogenic multi-tyrosine kinase inhibitor, showed promising clinical activity among patients with progressive, unresectable, metastatic phaeochromocytomas and paragangliomas (MPPGs). 

According to Camilo Jimenez, MD, MD Anderson Cancer Center, Houston, Texas, and coauthors, “Many non-familial MPPGs exhibit a phenotype that is characterized by abnormal angiogenesis.”

In this ongoing single-arm trial, 17 patients with histologically confirmed, progressive, unresectable MPPGs with a performance status of 0 to 2 received 60 mg of oral cabozantinib daily. The primary end point was investigator-assessed objective response rate (ORR). A key secondary end point was safety. 

At a median follow-up of 25 months, the ORR was 25%. Grade 3 adverse events were reported by 6 patients and included hand-and-foot syndrome, hypertension, rectal fistula, QT prolongation, asymptomatic hypomagnesaemia, and asymptomatic elevations of amylase and lipase. No grade ≥4 adverse events were reported. 

“Cabozantinib shows promising activity in patients with MPPGs,” concluded Dr Jimenez et al. 


Source: 

Jimenez C, Habra MA, Campbell MT, et al. Cabozantinib in patients with unresectable and progressive metastatic phaeochromocytoma or paraganglioma (the Natalie trial): a single-arm, phase 2 trial. Lancet Oncol. Published online: April 9, 2024. doi:10.1016/S1470-2045(24)00133-5