Sonidegib is a Hedgehog signalling pathway inhibitor approved for use in patients with advanced basal cell carcinoma (BCC) not eligible for surgery or radiotherapy. This report describes clinical experience with sonidegib in two patients with locally advanced BCC (one with a tumour adjacent to the right eye and the other with a tumour associated with the left ear) and in one patient with Gorlin syndrome. Two of the patients had recurrent and intractable tumours. Treatment with sonidegib 200 mg/day led to remission in both patients with locally advanced BCC within 7 months and to a reduction in the size and number of lesions after 4 months in the patient with Gorlin syndrome. Adverse effects reported in these patients were cramps, alopecia, ageusia and weight loss, all of which were mild and consistent with the known toxicity profile for sonidegib. Sonidegib has an important role to play in the effective treatment of challenging cases of advanced BCC. In parallel, a need remains to improve management protocols for patients with advanced BCC, particularly through earlier intervention and a multidisciplinary team approach.

The characteristics of the patients were similar to those published in datasets from continental Europe, although the pigmentary features were darker in those originating from Macaronesia. The prognostic parameters described in the 7th edition of the American Joint Committee on Cancer (AJCC) independently predict MSSV in our patients.

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