Pazopanib in patients with von Hippel-Lindau disease: a single-arm, single-centre, phase 2 trial.

ABSTRACT:

Background

No approved systemic therapy exists for von Hippel-Lindau disease, an autosomal dominant disorder with pleiotropic organ manifestations that include clear cell renal cell carcinomas; retinal, cerebellar, and spinal haemangioblastomas; pheochromocytomas; pancreatic serous cystadenomas; and pancreatic neuroendocrine tumours. We aimed to assess the activity and safety of pazopanib in patients with von Hippel-Lindau disease.

Methods

In this non-randomised, single-centre, open-label, phase 2 trial, adult patients with clinical manifestations of von Hippel-Lindau disease were recruited from the University of Texas MD Anderson Cancer Center (Houston, TX, USA) and were treated with pazopanib (800 mg orally daily) for 24 weeks, with an option to continue treatment if desir

SUBMITTER: Jonasch E 

PROVIDER: S-EPMC6338228 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature