Purpose: To establish a model to predict treatment outcome of periocular locally advanced basal cell carcinoma (POLA BCC) based on initial response to treatment with vismodegib (Erivedge), a sonic hedgehog inhibitor.

Design: Subgroup analysis of data from the STEVIE study database.

Methods: Analysis of medical history, treatment protocol, and treatment outcome of POLA BCC tumours in a STEVIE study population of 244 POLA BCC patients treated with ≥1 dose of vismodegib.

Results: A predictive model for complete response (CR) was established based on the initial treatment response. A cutoff value of 20% reduction in tumour size at 3 months of treatment identified the patients with a high probability (82.76%) to achieve CR. A second cutoff value of 67.7% reduction in tumour size at 6 months of treatment improved the prediction to a 95.42% probability of a CR outcome.

Conclusions: A treatment model was constructed based on the prediction of a CR outcome and the initial response to vismodegib treatment at 3 and 6 months. The study result provide significant new insights can facilitate decision-making on treatment management according to tumour response in patients with POLA BCC.

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http://www.ncbi.nlm.nih.gov/pmc/articles/PMC9905569PMC
http://dx.doi.org/10.1038/s41433-022-01982-yDOI Listing

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Review of Targeted Therapy, Vismodegib, for the Treatment of Periocular Basal Cell Carcinoma.

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January 2024

Department of Ophthalmology and Visual Sciences, W.K, Kellogg Eye Center, University of Michigan, Ann Arbor, Michigan, U.S.A.

Purpose: Periocular locally advanced basal cell carcinoma (POLA-BCC) is characterized by orbital involvement and/or extensive invasion of periocular structures. Hedgehog pathway inhibitors have been used for POLA-BCC with promising outcomes.

Methods: The authors reviewed 11 articles published in English literature from January 2012 to July 2022 and reported the outcomes of patients with POLA-BCC who were treated with vismodegib.

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Purpose: To establish a model to predict treatment outcome of periocular locally advanced basal cell carcinoma (POLA BCC) based on initial response to treatment with vismodegib (Erivedge), a sonic hedgehog inhibitor.

Design: Subgroup analysis of data from the STEVIE study database.

Methods: Analysis of medical history, treatment protocol, and treatment outcome of POLA BCC tumours in a STEVIE study population of 244 POLA BCC patients treated with ≥1 dose of vismodegib.

View Article and Find Full Text PDF

Objectives: To evaluate the effect of locally advanced periocular basal cell carcinoma (POLA-BCC) on health-related quality of life (HRQoL) and the benefit of vismodegib treatment among participants in the Safety Events in Vismodegib (STEVIE) trial between 2011 and 2017.

Methods: The STEVIE trial was conducted in patients with BCC (all anatomic locations) who were treated with vismodegib in 28-day cycles. Patients completed the Skindex-16, a validated questionnaire for the analysis symptoms, emotions, and functioning, at baseline, on day 1 of cycle 2, on day 1 of cycle 7, and at the end-of-study visit.

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Importance: The outcomes of vismodegib treatment in a relatively large cohort of study participants with periocular locally advanced basal cell carcinoma (POLA-BCC) may guide physicians when considering this treatment.

Objective: To report the outcomes of vismodegib treatment in patients with POLA-BCC in the Safety Events in Vismodegib (STEVIE) study.

Design, Setting, And Participants: This post hoc subgroup analysis from the STEVIE single-arm, multicenter, open-label cohort study screened all 1215 participants for ocular or periocular involvement and identified 244 participants with POLA-BCC or metastatic BCC.

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