Eligibility criteria for the study included patients with (i) clinical features or genetic confirmation of VHL disease and (ii) measurable lesions. Patients were treated with 800 mg pazopanib PO daily for two 12-week cycles of therapy. The primary outcome of decreased lesion size was evaluated using the RECIST criteria after the two cycles of pazopanib. There were 32 patients enrolled and 31 patients treated between 2012 and 2016. The median age was 37 (range: 19-67) of which 23 patients had genetically confirmed VHL, four patients had a family and personal history of the disease but did not undergo genetic testing, and five patients had clinical features of VHL with negative testing. Forty-two percent of patients showed a response to treatment, 58% had stable disease, and no patients had disease progression. Among responders, responses were seen in renal (2 complete responses, 29 partial responses/59 total), pancreatic (9 partial responses/17 total), and CNS (2 partial responses/49 total) target lesions. There were 12 patients that had dose reduction to 600 mg and six patients to 400 mg pazopanib daily. Eight patients discontinued therapy secondary to adverse events. One possible limitation of the trial is a lack of data regarding lesion size at study enrollment.
In summary, this is an important trial for patients with VHL. Pazopanib demonstrated efficacy for shrinking and stabilizing disease with an acceptable safety profile. Ideally, pazopanib may be a possible alternative to surgical therapy for these young, high-risk patients.
Clinical trial: NCT01436227
Presented By: Eric Jonasch, The University of Texas MD Anderson Cancer Center, Houston, TX, USA
Co-Authors: Dan S. Gombos, Steven G. Waguespack, Valerie Marcott, Diane D Liu, Justin Alexander Weldon, Shelly Bird, Christine Robichaux, Nizar M. Tannir, Ashley Henriksen Woodson, Greg Fuller, Ian E. McCutcheon, Surena F. Matin
Written By: Zachary Klaassen, MD, Urologic Oncology Fellow, University of Toronto, Princess Margaret Cancer Centre
at the 2017 ASCO Annual Meeting - June 2 - 6, 2017 – Chicago, Illinois, USA