Efficacy and safety of vismodegib in periocular and orbital basal cell carcinoma: a systematic review and meta-analysis - Report - MDSpire

Efficacy and safety of vismodegib in periocular and orbital basal cell carcinoma: a systematic review and meta-analysis

  • By

  • Dalal Alessa

  • May 11, 2026

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Clinical Report: Effectiveness and Safety of Vismodegib for BCC

Overview

Revise to specify the patient population and context of the complete response rate.

Background

Periocular and orbital BCC poses significant treatment challenges due to its location near critical structures and the risk of disfigurement. Vismodegib, a Hedgehog pathway inhibitor, offers a non-surgical treatment option, but its effectiveness and tolerability in this specific patient population require thorough evaluation. Understanding the outcomes associated with vismodegib is crucial for optimizing management strategies in patients with advanced BCC.

Data Highlights

OutcomePooled Rate (95% CI)
Complete Response Rate38.4% (36.6–57.4)
Partial Response Rate39.2% (32.4–47.5)
Recurrence Rate17.4% (7.0–30.3)
Exenteration Rate28.1% (12.3–34.5)
Adverse Events90.3% (81.0–94.8)
Severe Adverse Events (Grade ≥3)25.5%

Key Findings

  • The pooled complete response rate for vismodegib in periocular and orbital BCC was 38.4%.
  • Partial response was observed in 39.2% of patients.
  • Recurrence after treatment discontinuation occurred in 17.4% of cases.
  • Exenteration was necessary in 28.1% of patients despite vismodegib therapy.
  • Adverse events were reported in 90.3% of patients, with common events including alopecia and muscle spasms.
  • 31.2% of patients discontinued treatment due to toxicity.

Clinical Implications

Clinicians should consider vismodegib as a viable treatment option for patients with periocular and orbital BCC, particularly when surgical options are limited. However, the high rates of adverse events and recurrence necessitate careful patient selection and ongoing monitoring to manage potential complications effectively.

Conclusion

Vismodegib demonstrates significant tumor regression in periocular and orbital BCC, but the associated adverse events and recurrence rates highlight the importance of judicious use and patient management.

Related Resources & Content

  1. The ASCO Post, 2012 -- Vismodegib: Novel Agent for Treating Advanced Basal Cell Carcinoma
  2. BMC Cancer, 2017 -- Long-term safety and efficacy of vismodegib in patients with advanced basal cell carcinoma: final update of the pivotal ERIVANCE BCC study
  3. Oregon.gov, 2025 -- NCCN Clinical Practice Guidelines for Basal Cell Skin Cancer
  4. the ophthalmologist — VIS-101 Shows Activity in nAMD Study
  5. The ASCO Post — Gathering Data Point to Potential Advantages of Vismodegib in Basal Cell Carcinoma and Other Advanced Cancers
  6. Retinal Physician — RPS: From the Podium to the Practice
  7. Gathering Data Point to Potential Advantages of Vismodegib in Basal Cell Carcinoma and Other Advanced Cancers
  8. VIS-101 Shows Activity in nAMD Study
  9. https://www.oregon.gov/oha/HPA/DSI-HERC/MembersOnly/7.9d%20NCCN%202.2025%20BCC.pdf
  10. Long-term safety and efficacy of vismodegib in patients with advanced basal cell carcinoma: final update of the pivotal ERIVANCE BCC study | BMC Cancer | Springer Nature Link
  11. Oral Hedgehog Inhibitor, Vismodegib, for Locally Advanced Periorbital and Orbital Basal Cell Carcinoma

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