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Vismodegib in Locally Advanced Basal Cell Carcinoma in Slovenia
Tanja Mesti1,2, Maša Sever1, Janja Ocvirk1,2
1Department for Solid Tumors, Institute of Oncology Ljubljana, Ljubljana, Slovenia.
Background:
Vismodegib is a first-in-class inhibitor of the hedgehog pathway for treatment of locally advanced basal cell carcinoma (laBCC) and metastatic BCC.
Objectives:
The purpose of this study is to report outcomes of patients with laBCC, with basal cell carcinoma nevus syndrome (Gorlin Goltz syndrome [G-G Syn]) treated with vismodegib in routine clinical practice in Slovenia in 8.3-year period.
Methods:
In this retrospective cohort study, we analyzed baseline characteristics, outcomes, and treatment-related adverse events from locally advanced BCC. The patients were divided into two cohorts: 39 laBCC or multiple BCC patients and 7 patients with G-G Syn who were treated with vismodegib from November 2012 till January 2021.
Results:
During 100-month period, 46 patients were diagnosed with laBCC (26), multiple BCC (13), and G-G Syn (7), all inappropriate for surgery or radiotherapy. Baseline characteristics: median age was 72.8 years in laBCC + multiple BCC cohort and 47.4 years in G-G Syn cohort. The objective response rate was 80% in laBCC + multiple BCC and 86% in G-G Syn cohort. Disease control rate (DCR) was 95% in laBCC + multiple BCC and 100% in G-G Syn cohort. Median duration of treatment was 9.9 months (range: 1.5-43.1) in laBCC and multiple BCC cohort and 19.5 months (range: 3.6-94.1) in G-G Syn cohort. Majority of treatment-emergent adverse events (TEAEs) in laBCC or multiple BCC cohort were grade 1 or 2 (96%), only 4% of AEs were grade 3. Majority of TEAEs in G-G Syn cohort were also grade 1 or 2 (87%), 13% of AEs were grade 3. No grade 4 or 5 vismodegib-related AEs were reported.
Conclusion:
Vismodegib has shown meaningful efficacy with DCR from 95% to 100% in patients with laBCC, multiple BCC, and G-G Syn in Slovenia. TEAEs were successfully alleviated with multidisciplinary approach and early supportive care.
Insights
Vismodegib effectively treated advanced basal cell carcinoma (BCC) and Gorlin Goltz syndrome, achieving high disease control rates. Adverse events were manageable with supportive care, demonstrating vismodegib
Area of Science:
- Oncology
- Dermatology
- Pharmacology
Background:
- Vismodegib is a hedgehog pathway inhibitor used for advanced basal cell carcinoma (BCC).
- It targets locally advanced BCC (laBCC) and metastatic BCC.
Purpose of the Study:
- To evaluate vismodegib's effectiveness in routine clinical practice for laBCC and Gorlin Goltz syndrome (G-G Syn) in Slovenia.
- To report outcomes over an 8.3-year period.
Main Methods:
- Retrospective cohort study of 46 patients treated with vismodegib from November 2012 to January 2021.
- Patients were divided into laBCC/multiple BCC (n=39) and G-G Syn (n=7) cohorts.
- Analysis included baseline characteristics, outcomes, and treatment-related adverse events.
Main Results:
- Objective response rates were 80% (laBCC/multiple BCC) and 86% (G-G Syn).
- Disease control rates (DCR) were 95% and 100%, respectively.
- Most treatment-emergent adverse events (TEAEs) were grade 1 or 2, with no grade 4 or 5 events reported.
Conclusions:
- Vismodegib demonstrated significant efficacy with high DCRs (95-100%) in patients with laBCC, multiple BCC, and G-G Syn.
- TEAEs were manageable through multidisciplinary approaches and supportive care.
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