Safety and efficacy of Cabozantinib for metastatic renal cell carcinoma (mRCC): real world data from an Italian Expanded Access Program (EAP)

Abstract

Background: Final results from the randomised phase III METEOR trial confirmed a survival benefit of cabozantinib over everolimus in patients (pts) with advanced clear‐ cell renal cell carcinoma who progressed after at least one previous antiangiogenic inhibitor. The EAP provided the opportunity to treat pts in real world clinical practice. Methods: Data were collected from 91 pts treated with cabozantinib across 23 Italian hospitals. Cabozantinib was available, upon physician request, from September to December 2016. Pts were aged 18 years and older, with mRCC and measurable disease, with Perfomance Status (ECOG) 0 to 2, who had relapsed after one or more prior systemic treatment. 73 pts had clear‐cell RCC, while the other 18 had non‐clear‐cell histologies (type II papillary and chromophobe). The most frequent sites of disease were: lung 53 (58%), lymph nodes 41 (45%), bone 28 (31%), liver 15 (16%) and brain 5 (5%); 42 (46%) pts had two or more sites of disease. Cabozantinib was administered orally at 60 mg once a day in 28 days‐cycles. Dose reductions to 40 or 20 mg were allowed if toxicity was encountered. Pts were monitored for adverse events (AEs) using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v.4.0. The aim of this analysis was to evaluate the safety and activity of cabozantinib in a large unselected population. Results: Cabozantinib was administered as second line therapy in 28 (30%) pts, as III line in 18 (19%) pts and as further lines in the remaining 45 (51%) pts. At the time of our analysis, grade 3 and 4 AEs were observed in 21% of pts. Among 91 pts, only 5 (5%) discontinued treatment due to AEs. The best overall response was partial in 28 cases (31%), whereas 23 (25%) pts had stable disease and 23 (25%) had progressive disease; 17 pts (18%) have not reached the first response assessment. With a median follow‐up of 4 months, the median progression‐free survival observed was 3.5 months irrespective of the line of treatment. Conclusions: Our data suggest that cabozantinib is safe and active in a large unselected population treated according to everyday clinical practice.