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- Title
Clinical Characteristics of an Italian Patient Population with Advanced BCC and Real-Life Evaluation of Hedgehog Pathway Inhibitor Safety and Effectiveness.
- Authors
Mannino, Maria; Piccerillo, Alfredo; Fabbrocini, Gabriella; Quaglino, Pietro; Argenziano, Giuseppe; Dika, Emi; Ascierto, Paolo Antonio; Pellacani, Giovanni; Longo, Caterina; Fargnoli, Maria Concetta; Bianchi, Luca; Calzavara-Pinton, Piergiacomo; Zalaudek, Iris; Fava, Paolo; Scalvenzi, Massimiliano; Bocchino, Enrico; Di Stefani, Alessandro; Peris, Ketty
- Abstract
Background: Advanced basal cell carcinoma (aBCC) represents a complex and clinically heterogeneous group of lesions for which curative surgery and/or radiotherapy is unlikely. Systemic therapy with hedgehog pathway inhibitors (HHIs) changed the treatment landscape for this complex patient population. Objectives: The aims of the present study are to describe the clinical characteristics of a real-life Italian cohort diagnosed with aBCC and to investigate effectiveness and safety of HHI. Methods: A multicenter observational study was performed by twelve Italian centers in the period January 1, 2016 – October 15, 2022. Patients aged ≥18 years and diagnosed with aBCC (locally advanced [laBCC] and metastatic BCC [mBCC]) were eligible for the study. Methods for investigating tumor response to HHI included clinical and dermatoscopic evaluation, radiological imaging, and histopathology. For HHI safety assessment, therapy-related adverse events (AEs) were reported and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Results: We enrolled 178 patients under treatment with HHI: 126 (70.8%) and 52 patients (29.2%) received sonidegib and vismodegib, respectively. Comprehensive data on HHI effectiveness and disease outcome were available for 132 (74.1%) of 178 patients: 129 patients had a diagnosis of laBCC (n = 84, sonidegib; n = 45, vismodegib) and 3 patients of mBCC (n = 2, vismodegib; n = 1, sonidegib, off-label). Objective response rate was 76.7% (95% confidence interval [CI]: 82.3–68.7) and 33.3% (95% CI: 88.2–1.7) for laBCC (complete response [CR]: 43/129; PR: 56/129) and mBCC (CR: 0/3; PR: 1/3), respectively. High-risk aBCC histopathological subtypes and occurrence of >2 therapy-related AEs were significantly associated with nonresponse to HHI therapy ([OR: 2.61; 95% CI: 1.09–6.05; p: 0.03] and [OR: 2.74; 95% CI: 1.03–7.9; p: 0.04]), respectively. Majority of our cohort (54.5%) developed at least 1 therapy-related AE, most of which were mild-moderate in severity. Conclusions: Our results demonstrate the effectiveness and safety profile of HHI and confirm the reproducibility of pivotal trial results in real-life clinical setting.
- Subjects
HEDGEHOG signaling proteins; BASAL cell carcinoma; OFF-label use (Drugs); LANDSCAPE changes; VISMODEGIB
- Publication
Dermatology (10188665), 2023, Vol 239, Issue 6, p868
- ISSN
1018-8665
- Publication type
Article
- DOI
10.1159/000531280