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A case series identified 10 patients treated with sonidegib (n=6) or vismodegib (n=4) from March 2012 to March 2022, evaluating response activity, toxicity, and durability to oral hedgehog inhibitors. All patients achieved complete remission, with treatment markedly reducing new tumor counts. Median follow-up was 30.7–48.4 months; median time to new basal cell cancer was 47.3 months.",{"@graph":69,"@context":126},[70,84,105],{"@type":71,"itemListElement":72},"BreadcrumbList",[73,77,79,82],{"item":74,"name":75,"@type":76,"position":8},"https://docshare.wps.com","Home","ListItem",{"item":78,"name":9,"@type":76,"position":14},"https://docshare.wps.com/document/",{"item":80,"name":35,"@type":76,"position":81},"https://docshare.wps.com/document/healthcare/",3,{"item":83,"name":65,"@type":76,"position":19},"https://docshare.wps.com/document/sustained-suppression-of-gorlin-syndrome-associated-basal-cell-carcinomas-with-vismodegib-or-sonideg-a-case-series/384155/",{"url":83,"name":65,"@type":85,"image":86,"author":91,"headline":65,"publisher":94,"fileFormat":97,"inLanguage":63,"description":67,"dateModified":98,"datePublished":99,"encodingFormat":97,"isAccessibleForFree":100,"interactionStatistic":101},"DigitalDocument",{"url":87,"@type":88,"width":89,"height":90},"https://docshare.wps.com/thumbnails/sustained-suppression-of-gorlin-syndrome-associated-basal-cell-carcinomas-with-vismodegib-or-sonideg-a-case-series/384155.png","ImageObject",300,407,{"name":92,"@type":93},"Grenda","Person",{"url":74,"name":95,"@type":96},"DocShare","Organization","application/pdf","2026-10-28","2026-09-24",true,{"@type":102,"interactionType":103,"userInteractionCount":52},"InteractionCounter",{"@type":104},"ViewAction",{"@type":106,"mainEntity":107},"FAQPage",[108,114,118,122],{"name":109,"@type":110,"acceptedAnswer":111},"What is the purpose of this case series?","Question",{"text":112,"@type":113},"To evaluate how sustained oral hedgehog inhibitors affect the activity, toxicity, and durability of responses in patients with Gorlin syndrome-associated basal cell carcinomas.","Answer",{"name":115,"@type":110,"acceptedAnswer":116},"Which drugs and how many patients were included?",{"text":117,"@type":113},"Ten Gorlin patients were treated with sonidegib (n=6) or vismodegib (n=4) between March 2012 and March 2022.",{"name":119,"@type":110,"acceptedAnswer":120},"What outcomes were observed during hedgehog inhibitor treatment?",{"text":121,"@type":113},"All patients achieved complete remission, and the number of new basal cell cancers was significantly reduced during treatment compared with before treatment.",{"name":123,"@type":110,"acceptedAnswer":124},"Did patients require changes due to side effects?",{"text":125,"@type":113},"Yes. Six patients required treatment modifications for toxicity, and dose/schedule adjustments improved tolerability without altering efficacy.","https://schema.org",{"og:url":83,"og:type":128,"og:title":65,"og:site_name":95,"og:description":67},"article",{"robots":130,"canonical":83},"index,follow",{"doc_id":132,"site_id":62},384155,1790471909,{"code":4,"msg":5,"data":135},{"doc_id":132,"user_id":136,"nickname":92,"user_avatar":137,"doc_module":4,"category_id":34,"category_name":35,"doc_title":65,"doc_description":67,"doc_content":138,"file_id":139,"file_url":140,"file_type":141,"file_size":142,"view_count":52,"is_deleted":4,"is_public":8,"is_downloadable":8,"audit_status":8,"page_count":143,"language":144,"language_code":63,"site_id":62,"html_lang":63,"table_of_contents":145,"faqs":146,"seo_title":147,"seo_description":67,"update_tm":148,"read_time":41},7971474921005,"https://ap-avatar.wpscdn.com/davatar_29158cc5080c5b710cf443261637dec0","Article  \nSustained Suppression of Gorlin Syndrome-Associated Basal Cell Carcinomas with Vismodegib or Sonidegib: A Case Series  \nRaquel Wescott 1 and Wolfram Samlowski 1,2,3, *  \nCitation: Wescott, R.; Samlowski, W. Sustained Suppression of Gorlin Syndrome-Associated Basal Cell Carcinomas with Vismodegib or Sonidegib: A Case Series. Curr. Oncol.  \n2023, 30, 9156–9167. [https://doi.org/](https://doi.org/)[ ](https://doi.org/)[10.3390/curroncol30100661](10.3390/curroncol30100661)  \nReceived: 3 September 2023  \nRevised: 7 October 2023  \nAccepted: 10 October 2023  \nPublished: 16 October 2023  \nCopyright: © 2023 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license ([https://](https://)[ ](https://)[creativecommons.org/licenses/by/](creativecommons.org/licenses/by/)[ ](creativecommons.org/licenses/by/)[4.0/](4.0/)) .  \n1 School of Medicine, University of Nevada, Reno, NV 89557, USA; [rwescott@med.unr.edu](rwescott@med.unr.edu)  \n2 Comprehensive Cancer Centers of Nevada (Medical Oncology), Las Vegas, NV 89148, USA  \n3 Department of Medicine, Kirk Kirkorian School of Medicine, University of Nevada Las Vegas (UNLV), Las Vegas, NV 89106, USA  \n* [Correspondence: wsamlowski1@gmail.com](Correspondence: wsamlowski1@gmail.com); Tel.: +1-702-321-3930  \nAbstract: Nevoid basal-cell carcinoma syndrome (Gorlin syndrome) is characterized by numerous cutaneous basal cell carcinomas mediated by mutations in the hedgehog pathway. Vismodegib orsonidegib represent promising treatment options. We identified 10 Gorlin patients who were treated with sonidegib (n = 6) or vismodegib (n = 4) between March 2012 and March 2022 . We analyzed the activity, toxicity, and duration of the response to oral hedgehog inhibitors. The number of new tumors that developed prior to treatment or after treatment as well as the time of response and durability of responses were assessed. All patients achieved a complete remission. With a 30.7 􀀆 48.4-month median follow-up, the drug treatment significantly reduced the number of new basal cell cancers from a mean of 28.3 􀀆 24.6 prior to treatment to a mean of 1.4 􀀆 2.0 during treatment (p = 0.0048) . The median time to develop a new basal cell cancer was 47.3 months. Three patients eventually developed localized recurrences. After resection, ongoing treatment suppressed the development of additional lesions. One patient developed numerous new drug-resistant basal cell cancers and died of acute leukemia. Six patients required treatment modifications for toxicity. Sustained hedgehog inhibitor treatment can suppress the progression of both new and existing basal cell carcinomas for an extended period. Drug administration schedule adjustments improved tolerance without altering efficacy, potentially contributing to a prolonged response duration.  \nKeywords: nevoid basal cell carcinoma syndrome; sonidegib; vismodegib; hedgehog inhibitors; Gorlin syndrome; PTCH mutation  \n1. Introduction  \nGorlin syndrome (nevoid basal-cell carcinoma syndrome) is a rare autosomal dominant genetic condition that causes affected individuals to develop dozens to hundreds of basal cell carcinomas (BCCs) over their lifetime [1] . Few studies on Gorlin syndrome prevalence exist (reviewed by Evans et al. [1]) . The most quoted prevalence figure, 1:57,000, comes from a study of a UK population of four million people in northwest England. The potential range is between 1:30,827 and 1:164,000 . There is a possible predilection for Caucasians (although this may be due to ascertainment bias related to BCC development, which is less frequent in other races) . As expected, there is a similar rate of Gorlin syndrome in both sexes due to autosomal dominant inheritance. Approximately 70–80% of individuals with Gorlin syndrome have a pertinent family history, while about 20–30% result from a de novo pathogenetic variant. The genetic penetranc","cbCaiqRTvkOXR7Cy","https://ap.wps.com/l/cbCaiqRTvkOXR7Cy","pdf",2022832,12,"English","# Introduction\n## Diagnosis of Gorlin syndrome\n# Abstract\n## Treatment response and durability","[{\"question\":\"What is the purpose of this case series?\",\"answer\":\"To evaluate how sustained oral hedgehog inhibitors affect the activity, toxicity, and durability of responses in patients with Gorlin syndrome-associated basal cell carcinomas.\"},{\"question\":\"Which drugs and how many patients were included?\",\"answer\":\"Ten Gorlin patients were treated with sonidegib (n=6) or vismodegib (n=4) between March 2012 and March 2022.\"},{\"question\":\"What outcomes were observed during hedgehog inhibitor treatment?\",\"answer\":\"All patients achieved complete remission, and the number of new basal cell cancers was significantly reduced during treatment compared with before treatment.\"},{\"question\":\"Did patients require changes due to side effects?\",\"answer\":\"Yes. Six patients required treatment modifications for toxicity, and dose/schedule adjustments improved tolerability without altering efficacy.\"}]","Sustained Suppression of Gorlin Syndrome-Associated Basal Cell Carcinomas with Vismodegib or Sonideg - A Case Series | PDF",1790261418]