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Experience with sonidegib in patients with advanced basal cell carcinoma: case reports
Susana Puig1,2, Carlos Serra-Guillén3, Gemma Pérez-Pastor4
1Dermatology Department, Hospital Clínic Barcelona, University of Barcelona, Barcelona, Spain.
Abstract:
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.
Insights
Sonidegib effectively treats advanced basal cell carcinoma (BCC), including challenging recurrent cases. This Hedgehog pathway inhibitor showed positive results with manageable side effects in patients with locally advanced BCC and Gorlin syndrome.
Area of Science:
- Oncology
- Dermatology
- Pharmacology
Background:
- Advanced basal cell carcinoma (BCC) presents challenges, especially when surgery or radiotherapy are not options.
- Sonidegib is a Hedgehog signalling pathway inhibitor approved for advanced BCC.
- Gorlin syndrome is a rare genetic disorder associated with multiple BCCs.
Purpose of the Study:
- To describe clinical experience with sonidegib in patients with advanced and recurrent basal cell carcinoma.
- To evaluate the efficacy and safety of sonidegib in challenging BCC cases, including those with Gorlin syndrome.
Main Methods:
- Clinical case series involving three patients with advanced basal cell carcinoma.
- One patient had locally advanced BCC near the eye, another near the ear, and a third had Gorlin syndrome.
- Patients received sonidegib 200 mg/day.
Main Results:
- Complete remission was observed in both patients with locally advanced BCC within 7 months.
- The patient with Gorlin syndrome experienced a reduction in lesion size and number after 4 months.
- Adverse effects (cramps, alopecia, ageusia, weight loss) were mild and consistent with sonidegib's known profile.
Conclusions:
- Sonidegib demonstrates significant efficacy in treating advanced and intractable basal cell carcinoma.
- The drug offers a valuable treatment option for complex BCC cases, including those in patients with Gorlin syndrome.
- Improved management protocols, including earlier intervention and multidisciplinary care, are needed for advanced BCC.
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