Vismodegib: therapeutic application in advanced basal cell carcinoma and special groups. Experience at the Prof. A. Posadas Hospital

Abstract

Introduction: basal cell carcinoma (BCC) accounts for 75% of non-melanoma skin cancers. Vismodegib is a Hedgehog signaling pathway inhibitor approved by the FDA (Food and Drug Administration) in 2012 for the treatment of locally advanced BCC (LA-BCC). Regarding the response to treatment, a partial or complete decrease in tumor mass is evident. Adverse effects are generally mild. The most frequent are: cramps, alopecia, dysgeusia, diarrhea, fatigue, asthenia, among others.

Objectives: demonstrate the response to treatment with vismodegib in patients with BCC-LA, metastatic and special groups, evaluated from 2/1/2018 to 29/12/202, at Posadas Hospital. Describe the variables: sex, age, number of lesions, location, size, histological subtype, time of evolution, primary/recurrence, treatment duration, response to treatment, adverse effects, medical coverage, discontinuation due to toxicity and discontinuation due to lack of access to medication.

Materials and methods: descriptive and retrospective study. A search was conducted of photographic archives and medical records of patients with BCC-LA who received vismodegib.

Results: 31 patients with BCC-LA, including 2 metastatic, 1 Gorlin syndrome and 1 chronic endemic hydroarsenicism (CEHA). Complete response was observed in 11 (35.5%) patients and partial response in 13 (41.9%). Regarding toxicity, 18 (58%) patients presented adverse effects and 7 (22.6%) suspended treatment due to intolerance.

Conclusions: Hedgehog pathway inhibitors are novel treatments for LA-CBC. They represent a great therapeutic alternative, thus avoiding mutilating surgeries. In our population, response rates like those described in the literature were obtained. Although adverse effects are common, the drug has a mild toxicity profile.