{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,4]],"date-time":"2026-09-04T04:13:41Z","timestamp":1788495221394,"version":"build-2803163510"},"reference-count":24,"publisher":"S. Karger AG","issue":"6","license":[{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/karger.com\/pages\/terms-and-conditions"},{"start":{"date-parts":[[2023,1,1]],"date-time":"2023-01-01T00:00:00Z","timestamp":1672531200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/karger.com\/pages\/terms-and-conditions"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Dermatology"],"published-print":{"date-parts":[[2023]]},"abstract":"<jats:p>&lt;b&gt;&lt;i&gt;Background:&lt;\/i&gt;&lt;\/b&gt; 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. &lt;b&gt;&lt;i&gt;Objectives:&lt;\/i&gt;&lt;\/b&gt; 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. &lt;b&gt;&lt;i&gt;Methods:&lt;\/i&gt;&lt;\/b&gt; A multicenter observational study was performed by twelve Italian centers in the period January 1, 2016 \u2013 October 15, 2022. Patients aged \u226518 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. &lt;b&gt;&lt;i&gt;Results:&lt;\/i&gt;&lt;\/b&gt; 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 (&lt;i&gt;n =&lt;\/i&gt; 84, sonidegib; &lt;i&gt;n =&lt;\/i&gt; 45, vismodegib) and 3 patients of mBCC (&lt;i&gt;n =&lt;\/i&gt; 2, vismodegib; &lt;i&gt;n =&lt;\/i&gt; 1, sonidegib, off-label). Objective response rate was 76.7% (95% confidence interval [CI]: 82.3\u201368.7) and 33.3% (95% CI: 88.2\u20131.7) 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 &amp;gt;2 therapy-related AEs were significantly associated with nonresponse to HHI therapy ([OR: 2.61; 95% CI: 1.09\u20136.05; &lt;i&gt;p&lt;\/i&gt;: 0.03] and [OR: 2.74; 95% CI: 1.03\u20137.9; &lt;i&gt;p&lt;\/i&gt;: 0.04]), respectively. Majority of our cohort (54.5%) developed at least 1 therapy-related AE, most of which were mild-moderate in severity. &lt;b&gt;&lt;i&gt;Conclusions:&lt;\/i&gt;&lt;\/b&gt; Our results demonstrate the effectiveness and safety profile of HHI and confirm the reproducibility of pivotal trial results in real-life clinical setting. <\/jats:p>","DOI":"10.1159\/000531280","type":"journal-article","created":{"date-parts":[[2023,6,13]],"date-time":"2023-06-13T14:16:01Z","timestamp":1686665761000},"page":"868-876","source":"Crossref","is-referenced-by-count":14,"title":["Clinical Characteristics of an Italian Patient Population with Advanced BCC and Real-Life Evaluation of Hedgehog Pathway Inhibitor Safety and Effectiveness"],"prefix":"10.1159","volume":"239","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7246-7951","authenticated-orcid":false,"given":"Maria","family":"Mannino","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alfredo","family":"Piccerillo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0064-1874","authenticated-orcid":false,"given":"Gabriella","family":"Fabbrocini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pietro","family":"Quaglino","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giuseppe","family":"Argenziano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3186-2861","authenticated-orcid":false,"given":"Emi","family":"Dika","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paolo 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