Systematic review and meta-analysis of vismodegib for periocular and orbital basal cell carcinoma
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Key Takeaway
Consider vismodegib for periocular BCC but monitor closely due to high adverse event rates and recurrence after discontinuation.
This systematic review and meta-analysis evaluated the efficacy and safety of vismodegib in patients with periocular and orbital basal cell carcinoma, including 764 patients from multiple studies. The pooled complete response rate was 38.4% (95% CI: 36.6–57.4) and partial response rate was 39.2% (95% CI: 32.4–47.5). Recurrence following discontinuation occurred in 17.4% (95% CI: 7.0–30.3), and exenteration was performed in 28.1% (95% CI: 12.3–34.5).
Adverse events were almost universal, occurring in 90.3% (95% CI: 81.0–94.8). Common toxicities included muscle spasms (65.8%), alopecia (57.1%), weight loss (38.2%), and decreased appetite/anorexia (28.2%). Severe adverse events (grade ≥3) occurred in 25.5%, and 31.2% of patients discontinued treatment due to toxicity.
The authors note limitations including high recurrence rates after discontinuation and a substantial burden of adverse events that may limit long-term tolerability. The analysis did not report comparator data or follow-up duration, and causality cannot be inferred beyond the reported associations.
For clinical practice, these results underscore that while vismodegib can achieve tumor response in many patients, the high rate of adverse events and recurrence after stopping require careful patient selection and close monitoring to optimize outcomes.
View Original Abstract ↓
BackgroundPeriocular and orbital basal cell carcinoma (BCC) represents a therapeutic challenge due to its proximity to critical structures, the risk of disfigurement, and the potential need for exenteration in advanced disease. Vismodegib, a Hedgehog pathway inhibitor, offers a non-surgical alternative, but its overall efficacy and tolerability in this subset remain unclear.AimThis systematic review and meta-analysis aimed to evaluate the efficacy, safety, and recurrence outcomes of vismodegib in periocular and orbital BCC.MethodsA systematic search of PubMed, Scopus, and Web of Science was conducted. Eligible studies included prospective or retrospective cohorts and case series with ≥7 patients treated with vismodegib. Primary outcomes were complete and partial response rates; secondary outcomes included recurrence, exenteration, and adverse events. Pooled incidence rates with 95% confidence intervals (CI) were calculated using a random-effects model.ResultsEighteen studies, with 764 patients, were included. The pooled complete response rate was 38.4% (95% CI: 36.6–57.4), and partial response occurred in 39.2% (95% CI: 32.4–47.5). Recurrence following discontinuation was reported in 17.4% (95% CI: 7.0–30.3). Despite vismodegib therapy, exenteration was performed in 28.1% (95% CI: 12.3–34.5). Adverse events were almost universal (90.3, 95% CI: 81.0–94.8), with alopecia (57.1%), muscle spasms (65.8%), decreased appetite/anorexia (28.2%), and weight loss (38.2%) being the most frequent. Severe adverse events (grade ≥3) occurred in 25.5% of patients, and treatment discontinuation due to toxicity was noted in 31.2%.ConclusionVismodegib provides meaningful tumor regression in periocular and orbital BCC, enabling globe preservation in a subset of patients and reducing the need for exenteration. However, high recurrence rates after discontinuation and the substantial burden of adverse events limit long-term tolerability. Careful patient selection and close monitoring are essential for optimizing outcomes.
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