Rodrigo Perez Pereira, Dienifer Hermann Sirena, Sérgio Jobim de Azevedo, M S da Silva, Mariani Magnus da Luz Andrade, Charles Francisco Ferreira, Tiago Elias Heinen, Renato Marchiori Bakos
ABSTRACT Recently, new treatment strategies have been developed for advanced or metastatic basal cell carcinoma (BCC), including Hedgehog pathway inhibitors. Itraconazole has demonstrated clinical activity in such cases by blocking the smoothened (SMO) receptor. Such a strategy could be used in early disease. The objective of this Phase II study was to evaluate the clinical and molecular efficacy of Itraconazole in low‐risk BCC patients who were primarily candidates for surgical excision. Lesions were assessed according to RECIST 1.1 criteria, with target lesions being at least 10 mm in size after confirmatory biopsy. Patients received itraconazole tablets 200 mg twice daily for 60 days before resection. The median tumour diameter before treatment was 14 mm (IQR 11–16 mm), and after treatment, 13 mm (IQR 11–15 mm), and this reduction in tumour size was statistically significant ( p < 0.0001). Coupled with the decrease in tumour size, a decrease in the expression of CD105, an endothelial marker ( p < 0.0001), was observed. In conclusion, neoadjuvant itraconazole for 2 months was able to reduce the diameter of low‐risk BCC, and the effect was associated with a decrease in tumour angiogenesis and a favourable safety profile.