Clinical Characteristics of an Italian Patient Population with Advanced BCC and Real-Life Evaluation of Hedgehog Pathway Inhibitor Safety and Effectiveness.
Dermatology
; 239(6): 868-876, 2023.
Article
em En
| MEDLINE
| ID: mdl-37311439
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.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Neoplasias Cutâneas
/
Carcinoma Basocelular
/
Antineoplásicos
Tipo de estudo:
Clinical_trials
/
Observational_studies
Limite:
Adolescent
/
Adult
/
Humans
Idioma:
En
Revista:
Dermatology
Assunto da revista:
DERMATOLOGIA
Ano de publicação:
2023
Tipo de documento:
Article