Background: The expanded access program and anecdotal cases suggested sunitinib is safe in RCC patients with BM and might have worthwhile activity.
Patients and methods: In 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.
Results: Among 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.
Conclusion: Although tolerability was acceptable in RCC patients with previously untreated BM, sunitinib has limited efficacy in this setting.
Keywords: Brain metastases; Clinical trial; Renal cell carcinoma; Sunitinib.
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