Open AccessEffectiveness, Safety and Utilization of Vismodegib for Locally Advanced Basal Cell Carcinoma Under Real-world Conditions: Non-interventional Cohort Study JONAS
Author(s) -
Martin Kaatz,
Peter Möhr,
Elisabeth Livingstone,
Michael Weichenthal,
Alexander Kreuter,
Claudia Pföhler,
Ulrike Leiter,
Jens Ulrich,
Jochen Utikal,
Ralf Gutzmer,
Rudolf Herbst,
Dirk Schadendorf
Publication year - 2022
Publication title -
acta dermato-venereologica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.982
H-Index - 83
eISSN - 1651-2057
pISSN - 0001-5555
DOI - 10.2340/actadv.v102.293
Subject(s) - vismodegib , medicine , basal cell carcinoma , adverse effect , oncology , radiation therapy , clinical endpoint , hedgehog signaling pathway , basal cell , urology , surgery , clinical trial , signal transduction , biochemistry , chemistry
Most patients with advanced basal cell carcinomas (BCCs) may not benefit sufficiently from standard treatment comprising surgery and radiation. Vismodegib, an oral selective hedgehog pathway inhibitor, is approved for treatment of patients with locally advanced BCC inappropriate for surgery or radiotherapy, or for patients with symptomatic metastatic BCC. In order to enhance understanding of the effectiveness, safety and utilization of vismodegib in clinical practice in Germany, a non-interventional study, JONAS, was conducted. A total of 53 patients with locally advanced BCC who initiated treatment with vismodegib between 2016 and 2018 were included in the study, which was embedded in the German ADOReg skin cancer registry. Duration of response, the primary endpoint, was 12.4 months, progression-free survival 32.2 months and overall response rate 77.4%. Most adverse events were mild to moderate. Overall, results confirmed previous findings, demonstrating favourable responses and manageable safety of vismodegib in patients with locally advanced BCC in clinical practice.