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The results from a phase II clinical trial, which demonstrates the potential for first-line treatment with pazopanib as an alternative to surgery in patients with von Hippel-Lindau disease, were presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago this week.
Thirty-two patients with von Hippel-Lindau disease (VHL) confirmed by genetic testing, or with a family/personal history of VHL, or with clinical features of VHL but negative genetic testing, were assessed in this study to evaluate the effects of the tyrosine kinase inhibitor (TKI), pazopanib, on tumour shrinkage. Of 31 evaluable patients, 18 had stable disease, 13 had a response, and none had progressive disease during the study. Side effects included diarrhoea and abnormal liver function tests (the latter resulted in discontinuation of treatment in 4 patients). Twelve patients required dose reductions, and 8 patients discontinued treatment due to adverse events.
These results from the largest prospective VHL-specific study to date demonstrate the potential for pazopanib as an alternative to surgery.