A Phase II Trial of Sunitinib in Patients With Renal Cell Cancer and Untreated Brain Metastases

BackgroundThe expanded access program and anecdotal cases suggested sunitinib is safe in RCC patients with BM and might have worthwhile activity.Patients and MethodsIn a phase II trial, patients with untreated BM received the standard regimen of sunitinib. The primary end point was objective response (OR) rate in BM after 2 cycles. An OR rate of 35% was prospectively defined as the minimum needed to warrant further investigation. According to Simon's optimal 2-stage design, at least 3 of the initial 15 patients had to have an OR for accrual to continue.ResultsAmong 16 evaluable patients, 1 had a complete response outside the central nervous system (CNS). CNS disease was stabilized in 5 (31%). However, no BM showed an OR. Therefore, no further accrual took place. Median time to progression was 2.3 months and overall survival was 6.3 months. There was 1 toxic death, from peritonitis with gastric perforation. Three patients experienced at least 1 treatment-related grade 3 or greater toxicity but no neurological adverse events were attributable to sunitinib.ConclusionAlthough tolerability was acceptable in RCC patients with previously untreated BM, sunitinib has limited efficacy in this setting.