Valenti Mario, Di Giulio Sara, Carugno Andrea, Frascione Pasquale, Marzano Angelo Valerio, Mercuri Santo Raffaele, Nazzaro Gianluca, Spallone Giulia, Paolino Giovanni, Ardigò Marco
Dermatology Unit, IRCCS Humanitas Research Hospital, Rozzano (MI); Department of Biomedical Sciences, Humanitas University, Pieve Emanuele (MI).
Dermatology Unit, Department of Medicine and Surgery, University of Insubria, Varese.
Dermatol Reports. 2025 Aug 22;17(3). doi: 10.4081/dr.2025.10196. Epub 2025 Feb 18.
Vismodegib and sonidegib are smoothened (SMO) inhibitors approved for the treatment of advanced basal cell carcinoma (aBCC). This study investigates the real-world experiences and outcomes associated with these therapies across multiple Italian centers. A retrospective, observational, multicenter study was conducted. Medical records of patients with local advanced basal cell carcinoma (laBCC) treated with SMO inhibitors outside of clinical trials from July 2019 to April 2024 were analyzed. A total of 57 patients were included in the study, with 19 (33.3%) receiving vismodegib and 38 (66.7%) receiving sonidegib. Vismodegib demonstrated a complete response (CR) in 31.6% of cases and a partial response (PR) in 26.3%. Sonidegib achieved a CR of 47.4% and a PR of 36.8%. The median treatment duration was 8 months for vismodegib and 12 months for sonidegib. Adverse events were more frequently reported with vismodegib. Treatment interruption due to adverse events occurred in 47.4% of vismodegib recipients and in 13.2% of those receiving sonidegib. The progression of the disease under treatment occurred in 7.9% of cases of patients under sonidegib and in 26.3% of cases under vismodegib. ************************************************************************* *Appendix Authors list Valentina Benzecry Mancin,1 Veronica Benzi,2 Matteo Bianco,2,3 Alessandra Bulotta,4 Antonio Costanzo,2,3 Giuseppe Damiano,5 Nerina Denaro,6 Laura Eibenschutz,7 Barbara Merelli,8 Giulia Murgia,1 Alessandra Narcisi,3 Emanuela Passoni,1 Francesco Piscazzi,2,3 Vincenzo Russo5 1Dermatology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; 2Department of Biomedical Sciences, Humanitas University, Pieve Emanuele (MI); 3Dermatology Unit, IRCCS Humanitas Research Hospital, Rozzano (MI); 4Department of Oncology, IRCCS San Raffaele, Milan; 5Unit of Immuno-Biotherapy of Melanoma and Solid Tumors, IRCCS Ospedale San Raffaele, Milan; 6Oncology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan; 7Oncologic and Preventive Dermatology Unit, IFO-San Gallicano Dermatological Institute IRCCS, Rome; 8Oncology Unit, ASST Papa Giovanni XXIII, Bergamo, Italy.
维莫德吉和索尼德吉是已获批用于治疗晚期基底细胞癌（aBCC）的 smoothened（SMO）抑制剂。本研究调查了在意大利多个中心使用这些疗法的真实世界经验和结果。开展了一项回顾性、观察性、多中心研究。分析了2019年7月至2024年4月在临床试验之外接受SMO抑制剂治疗的局部晚期基底细胞癌（laBCC）患者的病历。共有57名患者纳入研究，其中19名（33.3%）接受维莫德吉治疗，38名（66.7%）接受索尼德吉治疗。维莫德吉在31.6%的病例中显示出完全缓解（CR），在26.3%的病例中显示出部分缓解（PR）。索尼德吉的CR为47.4%，PR为36.8%。维莫德吉的中位治疗持续时间为8个月，索尼德吉为12个月。维莫德吉更频繁地报告不良事件。因不良事件导致治疗中断的情况在接受维莫德吉治疗的患者中占47.4%，在接受索尼德吉治疗的患者中占13.2%。接受索尼德吉治疗的患者中有7.9%的病例在治疗期间疾病进展，接受维莫德吉治疗的患者中有26.3%的病例出现疾病进展。
附录
作者名单
瓦伦蒂娜·本泽克里·曼钦，1 维罗妮卡·本齐，2 马泰奥·比安科，2,3 亚历山德拉·布洛塔，4 安东尼奥·科斯坦佐，2,3 朱塞佩·达米亚诺，5 内里娜·德纳罗，6 劳拉·艾本舒茨，7 芭芭拉·梅雷利，8 朱利亚·穆尔贾，1 亚历山德拉·纳西西，3 埃玛努埃拉·帕索尼，1 弗朗切斯科·皮斯卡齐，2,3 文森佐·鲁索5
1米兰IRCCS圣心医院皮肤科，Ca' Granda 基金会；2皮耶韦埃马努埃莱（米兰）人文大学基础生物医学科学系；3罗扎诺（米兰）IRCCS 人文研究医院皮肤科；4米兰IRCCS圣拉斐尔肿瘤学系；5米兰IRCCS圣拉斐尔医院黑色素瘤和实体瘤免疫生物治疗科；6米兰IRCCS圣心医院肿瘤学病房，Ca' Granda 基金会；7罗马IFO-圣加里卡诺皮肤病研究所IRCCS肿瘤与预防性皮肤病科；8意大利贝加莫ASST 教皇约翰二十三世肿瘤学病房。
