20.500.12556/DiRROS-15459 Vismodegib in locally advanced basal cell carcinoma in Slovenia Background: Vismodegib is a first-in-class inhibitor of the hedgehog pathway for treatment of locally advanced basal cell carcinoma (laBCC) and metastatic BCC. Objectives: The purpose of this study is to report outcomes of patients with laBCC, with basal cell carcinoma nevus syndrome (Gorlin Goltz syndrome [G-G Syn]) treated with vismodegib in routine clinical practice in Slovenia in 8.3-year period. Methods: In this retrospective cohort study, we analyzed baseline characteristics, outcomes, and treatment-related adverse events from locally advanced BCC. The patients were divided into two cohorts: 39 laBCC or multiple BCC patients and 7 patients with G-G Syn who were treated with vismodegib from November 2012 till January 2021. Results: During 100-month period, 46 patients were diagnosed with laBCC (26), multiple BCC (13), and G-G Syn (7), all inappropriate for surgery or radiotherapy. Baseline characteristics: median age was 72.8 years in laBCC + multiple BCC cohort and 47.4 years in G-G Syn cohort. The objective response rate was 80% in laBCC + multiple BCC and 86% in G-G Syn cohort. Disease control rate (DCR) was 95% in laBCC + multiple BCC and 100% in G-G Syn cohort. Median duration of treatment was 9.9 months (range: 1.5-43.1) in laBCC and multiple BCC cohort and 19.5 months (range: 3.6-94.1) in G-G Syn cohort. Majority of treatment-emergent adverse events (TEAEs) in laBCC or multiple BCC cohort were grade 1 or 2 (96%), only 4% of AEs were grade 3. Majority of TEAEs in G-G Syn cohort were also grade 1 or 2 (87%), 13% of AEs were grade 3. No grade 4 or 5 vismodegib-related AEs were reported. Conclusion: Vismodegib has shown meaningful efficacy with DCR from 95% to 100% in patients with laBCC, multiple BCC, and G-G Syn in Slovenia. TEAEs were successfully alleviated with multidisciplinary approach and early supportive care. basal cell carcinoma early supportive care Gorlin Goltz syndrome multidisciplinary approach bazalnocelični rak sindrom Gorlin Goltz večdisciplinarni pristop true false true S. Karger Angleški jezik Slovenski jezik by Authors Neznano 2022-09-07 03:37:47 2022-09-07 07:59:21 2022-09-08 03:36:07 0000-00-00 00:00:00 2022 0 0 str. 1-7 no. Vol. 2022 0000-00-00 Zaloznikova Objavljeno apc 0000-00-00 0000-00-00 2022-01-01 616.5 1018-8665 10.1159/000525612 117583107 RAZ_Mesti_Tanja_2022.pdf RAZ_Mesti_Tanja_2022.pdf 1 66E91885F3EF84346935C832F834BC41 3ac1f259f96da5b97833baebfead376f40154e729fe69a9175506a446b17d70d bb67d918-2e61-11ed-bea5-001a4af901a5 https://dirros.openscience.si/Dokument.php?lang=slv&id=20446 https://www.karger.com/Article/Pdf/525612 0 a50a3577-2e61-11ed-bea5-001a4af901a5 https://dirros.openscience.si/Dokument.php?lang=slv&id=20445 Onkološki inštitut Ljubljana 0 0 0