Vismodegib in the treatment of basal cell carcinoma — Polish clinical experience in the frame of therapeutic program

Introduction. Vismodegib is a small-molecule inhibitor of the sonic hedgehog pathway, registered for the treat- ment of patients with metastatic or locally advanced basal cell carcinoma, who were disqualified from surgical excision or radiotherapy. The full treatment refund from the National Health Fund has been available in Poland since 1st January 2018. The aim of the study was to analyse the frequency of occurrence of adverse events based on CTCAE and the treatment results based on the RECIST 1.1 criteria, in a group of patients treated for six or 12 months with vismodegib. Material and methods. The patient database was gathered from three sites and consisted of 42 patients, who represented 53.8% of the patients treated with vismodegib in Poland. The duration of the treatment ranged between three weeks and 68 months. The median of the treatment period was 8.25 months (0.75–68); the median of the observation of patients treated for less than 12 months was eight months (6–11), and for those treated for more than 12 months it was 14 months (12–68). Results. The summary of the treatment results after six and 12 months was performed on 29/42 and 17/42 patients accordingly. Complete response was achieved in 3/29 (10.3%) and 3/16 (17.6%) patients after six and 12 months of treatment, respectively, partial response in 13/29 (44.8%) and 5/16 (29.4%) patients, respectively, and stable disease in 13/29 (44.8%) and 8/16 (50.0%) patients, respectively. Progression of the disease was experienced by 7/42 (16.6%) patients within the period of 3–28 months of treatment. One patient with brain metastases died due to the progression of the disease. Adverse events were reported in 31/42 (73.8%) patients, more than one adverse event in a single patient was reported in 22/42 (52.3%) patients. No serious adverse events were observed.