Retrospective Real-Life Data, Efficacy and Safety of Vismodegib Treatment in Patients with Advanced and Multiple Basal Cell Carcinoma: 3-Year Experience from a Spanish Center

• Published in: International journal of environmental research and public health Vol. 20; no. 10; p. 5824
• Main Authors: Ayén-Rodríguez, Angela, Linares-González, Laura, Llamas-Segura, Carlos, Almazán-Fernández, Francisco Manuel, Ruiz-Villaverde, Ricardo
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI AG 15.05.2023; MDPI
• Subjects: Aged; Antineoplastic Agents - adverse effects; Basal cell carcinoma; Biopsy; Brief Report; Cancer therapies; Carcinoma; Carcinoma, Basal Cell - drug therapy; Cardiovascular disease; Care and treatment; Complications and side effects; Dermatology; Drug approval; Drug dosages; FDA approval; Female; Health services; Hedgehog Proteins; Humans; Male; Medical research; Medicine, Experimental; Metastasis; Patient compliance; Patients; Photodynamic therapy; Radiation therapy; Retrospective Studies; Skin cancer; Skin Neoplasms - chemically induced; Skin Neoplasms - drug therapy; Spasms; Surgery; Tumors; Vismodegib; hedgehog inhibitor; nonmelanoma skin cancer; vismodegib; locally advanced basal cell carcinoma
• ISSN: 1660-4601; 1661-7827; 1660-4601
• DOI: 10.3390/ijerph20105824

Basal cell carcinoma (BCC) is the most common type of skin cancer and can represent a therapeutic challenge in patients with locally advanced disease. Vismodegib is a hedgehog pathway inhibitor approved by the FDA for use in this type of tumor. We present a case series to describe our experience with the use of vismodegib. A retrospective study that included patients treated with vismodegib at our dermatology unit was conducted. Monthly follow-up was performed, and we registered the clinical evolution and adverse reactions. A total of six patients with locally advanced BCCs were included (50% males and 50% females), with a mean age of 78.5 years old. The treatment was administered over a mean of 5 months. A complete response was observed in four cases and partial response in two cases. No recurrence was detected, with a median follow-up duration after discontinuation of 18 months. Most patients (83%) had at least one adverse event, and two needed dose adjustment temporarily or permanently to continue. The main adverse effect was muscle spasms (66.7%). The main limitation of our study was the small sample, which was not representative of the general population. Vismodegib is a safe and effective treatment for locally advanced BCC, and its role in unresectable BCC seems to be an important option in these challenging cases.