Updated results of a prospective noninterventional study of everolimus after failure of the first VEGF-targeted therapy.

Abstract

361 Background: Everolimus (EVE, Afinitor) is approved for the treatment of metastatic renal cell cancer (mRCC) after failure of VEGF-targeted therapy. The option to treat mRCC with six approved targeted agents has sparked debate on proper sequencing of these agents. However, data beyond clinical trials is limited. Here, we report prospective non-interventional data on EVE in routine use after failure of the first VEGF-targeted therapy. Methods: A prospective, single arm, open label, multi center non-interventional study for patients with mRCC was initiated in Germany in 08/2009 to determine effectiveness defined as time between first EVE intake until disease progression due to any cause (TTP) and treatment duration. Targeted enrollment is 400 patients. A first interim analysis was conducted 3 months after 100 patients had been enrolled and was presented previously (Bergmann et al., J Clin Oncol 29: 2011 [suppl; abstr 4552]). Here we present a second interim analysis to corroborate the results of the first interim analysis. Patients were analyzed 14 months after the first 100 patients had been enrolled. Results: 59 sites included 113 patients between August 2009 and July 2010. At the cut-off date of the first interim analysis (12 November 2011), these patients had been followed for a median of 116 days. The safety population consisted of 99 patients with documented EVE treatment. The median treatment duration had not yet been reached at the time of the first interim analysis. Median time to progression (TTP) was 9.7 months (95% CI: 6 months; n.d.). A total of 230 AEs were reported in 61% of patients including 18 SAEs in 12 patients (12%). These preliminary results are substantiated in this interim analysis with a total observation period of at least 14 months (cut-off date 30 September 2011). Updated data on effectiveness and safety of EVE will be presented. Conclusions: This non-interventional study on EVE in treatment of mRCC after prior failure of VEGF-targeted therapy provides first evidence on routine use of EVE. Further analysis of this ongoing study affords insight into effectiveness and safety of EVE in routine use after failure of the first VEGF-targeted therapy.