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Treatment With Pazopanib in von Hippel-Lindau Disease

By: Meg Barbor, MPH
Posted: Friday, July 28, 2017

In patients with von Hippel-Lindau (VHL) disease, treatment with pazopanib resulted in significant and sustained disease control, with an acceptable safety profile, according to data from a phase II study presented by Eric Jonasch, MD, of The University of Texas MD Anderson Cancer Center, at the 2017 American Society of Clinical Oncology (ASCO) Annual Meeting (Abstract 4516). This is reportedly the largest prospective VHL-specific study to date.

VHL is an inherited disorder that causes tumors in multiple sites, including the eyes, brain, pancreas, adrenal glands, and kidneys. Current standards of care include surveillance imaging and surgical intervention.

A total of 32 patients with either genomically confirmed VHL (n=23), clinical features of VHL but negative genetic testing (n=5), or a family/personal history of VHL (n=4) were treated with pazopanib at 800 mg/day for two 12-week cycles. Of 31 evaluable patients, 13 had a response, 18 had stable disease, and no patients exhibited progressive disease.

The most common side effect was grade 1/2 diarrhea in 14 patients. Twelve patients required dose reductions, and eight patients discontinued treatment due to adverse events.