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

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

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.

MeSH terms

  • Anilides / adverse effects
  • Antineoplastic Agents* / adverse effects
  • Carcinoma, Basal Cell* / pathology
  • Hedgehog Proteins / therapeutic use
  • Humans
  • Skin Neoplasms* / pathology
  • Treatment Outcome

Substances

  • HhAntag691
  • Hedgehog Proteins
  • Anilides
  • Antineoplastic Agents