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. 2023 Feb;37(3):531-536.
doi: 10.1038/s41433-022-01982-y. Epub 2022 Feb 24.

The initial rate of tumour response to vismodegib treatment, can predict a complete response outcome for periocular LA-BCC

Alon Tiosano  1   2 , Meydan Ben-Ishai  3   4 , Eyal Fenig  5   4 , Guy J Ben Simon  6   4 , Iftach Yassur  3   4
Affiliations

The initial rate of tumour response to vismodegib treatment, can predict a complete response outcome for periocular LA-BCC

Alon Tiosano et al. Eye (Lond). 2023 Feb.

Abstract

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 (ErivedgeTM), 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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Conflict of interest statement

Dr. Fenig reported serving as an investigator in the Safety Events in Vismodegib (STEVIE) study. No other disclosures were reported.

Figures

Fig. 1
Fig. 1. Change in tumour size during the study.
Change in tumour size over time. Trends of the different tumour sizes according to LOESS regression.
Fig. 2
Fig. 2. ROC curve for reduction in size at 3, and 6 months.
ROC curves by size reduction at 3 and 6 months, CR Complete response, AUC Area under the curve.
Fig. 3
Fig. 3. Decision algorithm during treatment.
Decision algorithm based on treatment response at 3 and 6 months.
See this image and copyright information in PMC

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