{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,3]],"date-time":"2026-02-03T14:52:17Z","timestamp":1770130337480,"version":"3.49.0"},"reference-count":22,"publisher":"Wiley","issue":"8","license":[{"start":{"date-parts":[[2025,10,23]],"date-time":"2025-10-23T00:00:00Z","timestamp":1761177600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2025,10,23]],"date-time":"2025-10-23T00:00:00Z","timestamp":1761177600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/501100013671","name":"Sun Pharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100013671","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Aust J Dermatology"],"published-print":{"date-parts":[[2025,12]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>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\u2010term use. Dose modifications and off\u2010therapy periods are frequently necessary to improve tolerability.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives<\/jats:title>\n                    <jats:p>To describe the dosing regimens employed during treatment with sonidegib for GS in clinical practice and evaluate the efficacy and tolerability of its long\u2010term use.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Multicenter retrospective study involving 10 referral centres in Spain including patients with GS who received treatment with sonidegib for at least 12\u2009weeks.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Twenty\u2010four patients were included: 11 (45.8%) women and 13 (54.2%) men, with a mean age of 50.7\u2009\u00b1\u200912.1\u2009years. The mean duration of treatment with sonidegib was 10.4\u2009\u00b1\u20095.1\u2009months. Sonidegib was started at 200\u2009mg daily in 22 (91.7%) patients. Thirteen (59.1%) of them switched to alternate\u2010day dosing motivated most frequently by the development of AEs (76.9%), and less frequently by dosing convenience (23.1%). Two patients followed on\u2013off regimens. Four patients received an off\u2010label regimen of 2\u20133 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\u2010loss (20.8%), diarrhoea (12.5%) and nausea (8.3%).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>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\u2010day dose is the most common modification, dermatologists also resort to off\u2010label regimens for this purpose.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/ajd.14614","type":"journal-article","created":{"date-parts":[[2025,10,23]],"date-time":"2025-10-23T07:43:02Z","timestamp":1761205382000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Multicenter Retrospective Case Series on the Real\u2010World Experience With Sonidegib for the Management of Gorlin Syndrome"],"prefix":"10.1111","volume":"66","author":[{"given":"Gemma","family":"P\u00e9rez\u2010Pastor","sequence":"first","affiliation":[{"name":"Department of Dermatology Consorcio Hospital General Universitario de Valencia  Valencia Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dario","family":"Perosanz Lobo","sequence":"additional","affiliation":[{"name":"Department of Dermatology Hospital 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Bellvitge, L'Hospitalet de Llobregat  Barcelona Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0330-7599","authenticated-orcid":false,"given":"V\u00edctor","family":"Gonzalez\u2010Delgado","sequence":"additional","affiliation":[{"name":"Department of Dermatology Hospital Cl\u00ednico de Valencia  Valencia Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mireia","family":"Y\u00e9benes Marsal","sequence":"additional","affiliation":[{"name":"Department of Dermatology Consorci Corporaci\u00f3 Sanit\u00e0ria Parc Taul\u00ed  Sabadell Barcelona Spain"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elia","family":"Samaniego Gonz\u00e1lez","sequence":"additional","affiliation":[{"name":"Department of Dermatology Hospital de Le\u00f3n  Le\u00f3n 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