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Multicenter Retrospective Case Series on the Real-World Experience With Sonidegib for the Management of Gorlin
Gemma Pérez-Pastor1, Dario Perosanz Lobo2, Cristina Ciudad Blanco3
1Department of Dermatology, Consorcio Hospital General Universitario de Valencia, Valencia, Spain.
Background:
Gorlin syndrome (GS) is a hereditary genodermatosis characterised by the development of multiple basal cell carcinomas (BCC). The hedgehog pathway inhibitors (HPI) like sonidegib have been shown to be effective, but the frequency of adverse events (AE) limits their long-term use. Dose modifications and off-therapy periods are frequently necessary to improve tolerability.
Objectives:
To describe the dosing regimens employed during treatment with sonidegib for GS in clinical practice and evaluate the efficacy and tolerability of its long-term use.
Methods:
Multicenter retrospective study involving 10 referral centres in Spain including patients with GS who received treatment with sonidegib for at least 12 weeks.
Results:
Twenty-four patients were included: 11 (45.8%) women and 13 (54.2%) men, with a mean age of 50.7 ± 12.1 years. The mean duration of treatment with sonidegib was 10.4 ± 5.1 months. Sonidegib was started at 200 mg daily in 22 (91.7%) patients. Thirteen (59.1%) of them switched to alternate-day dosing motivated most frequently by the development of AEs (76.9%), and less frequently by dosing convenience (23.1%). Two patients followed on-off regimens. Four patients received an off-label regimen of 2-3 tablets per week. Nine (37.5%) of the 24 patients achieved a complete response, 13 (54.2%) a partial response and 2 (8.3%) presented stable disease. Twenty (83.3%) patients presented at least one AE, the most frequent being alopecia (45.8%), followed by muscle cramps (37.5%), dysgeusia (29.2%), asthenia (29.2%), weight-loss (20.8%), diarrhoea (12.5%) and nausea (8.3%).
Conclusions:
Our data on the use of sonidegib for GS in clinical practice show similar response rates to those reported in previous studies and improved tolerability compared to pivotal studies. Regimen modifications are frequently used in clinical practice to improve AEs. Although the alternate-day dose is the most common modification, dermatologists also resort to off-label regimens for this purpose.
Insights
Hedgehog pathway inhibitors like sonidegib effectively treat Gorlin syndrome (GS) by reducing basal cell carcinomas (BCC). Dose adjustments and modified regimens improve tolerability and enable long-term use of sonidegib for GS patients.
Area of Science:
- Oncology
- Dermatology
- Genetics
Background:
- Gorlin syndrome (GS) is a hereditary disorder leading to multiple basal cell carcinomas (BCC).
- Hedgehog pathway inhibitors (HPIs) show efficacy but adverse events (AEs) limit long-term sonidegib use.
- Dose modifications and treatment interruptions are often required to manage AEs.
Purpose of the Study:
- To document sonidegib dosing strategies in clinical practice for Gorlin syndrome.
- To assess the efficacy and tolerability of long-term sonidegib treatment in GS patients.
Main Methods:
- A multicenter retrospective study was conducted across 10 Spanish referral centers.
- Included patients with Gorlin syndrome treated with sonidegib for a minimum of 12 weeks.
- Data on dosing regimens, efficacy, and adverse events were collected and analyzed.
Main Results:
- Twenty-four patients with Gorlin syndrome were analyzed, with a mean treatment duration of 10.4 months.
- Most patients (91.7%) started sonidegib at 200 mg daily; 59.1% switched to alternate-day dosing due to AEs.
- Response rates included 37.5% complete response and 54.2% partial response; 83.3% experienced AEs, most commonly alopecia and muscle cramps.
Conclusions:
- Sonidegib demonstrates comparable efficacy in clinical practice for Gorlin syndrome to previous studies.
- Regimen modifications, including alternate-day dosing and off-label schedules, improve sonidegib tolerability.
- These adjustments facilitate long-term management of Gorlin syndrome with HPIs.
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