Background Axitinib is a new oral cytostatic VEGFR-1,-2 and -3 inhibitor, used in the treatment of renal cell carcinoma (RCC), available through an expanded access programme.

Purpose To analyse the effectiveness and safety of axitinib treatment in patients with RCC in a tertiary hospital.

Materials and methods A retrospective descriptive study of patients taking axitinib from November 2012 to April 2013. The following information was collected: demographics (gender and age), diagnosis, basal situation (ECOG performance status (PS) and staging), dose of axitinib, pre-treatments, effectiveness (response rate and overall survival after four months) and adverse reactions. The information source was the electronic health record.

Results 7 patients were recruited. 3 (42.8%) were women. The mean age was 57.8 (32–71). 6 patients were diagnosed with clear cell carcinoma and the other one with papillary carcinoma. The PSs were: 0 (n = 2), 1 (n = 4) and 2 (n = 1). All patients had metastatic disease (stage IV). All patients received axitinib 5 mg/12 h. A total of 5 (71.4%) patients had been treated once already, and 2 (28.6%) patients had been treated with at least two prior regimens. Pre-treatments: the majority of patients 4 (57.1%) received sunitinib before starting axitinib treatment, 2 (28.6%) received pazopanib and everolimus and 1 (14.3%) received only pazopanib. Effectiveness: the response rates were stable disease (n = 3; 48.8%), partial response (n = 2; 28.6%) and no response (n = 2, 28.6%). The global survival rate after 4 months was 57.1%. Safety: the most frequent adverse reactions were: mucositis (n = 5; 71%), diarrhoea (n = 3; 43%), asthenia (n = 3; 43%), hypertension (n = 2; 29%) and rash (n = 2; 29%). One patient had a reduction to 5 mg/24 h.

Conclusions The number of patients included in the expanded access, and therefore in this study, was very low, so that the effectiveness of the treatment cannot be demonstrated. Nevertheless, it is important to highlight that 2 out of 7 patients had a partial response and 3 out of 7 have stable disease. Gastrointestinal problems were the most frequent adverse reactions.

No conflict of interest.