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Exploring vismodegib: A non-surgical breakthrough in the management of advanced periocular basal cell carcinoma
Georgios Lavasidis1, Argyrios Tzamalis2, Ioannis Tsinopoulos2
1MSc Ocular Surgery, School of Medicine, Aristotle University of Thessaloniki, University Campus, 54124 Thessaloniki, Greece; Evidence-based Medicine Unit, Department of Hygiene and Epidemiology, School of Medicine, University of Ioannina, University Campus, 45110 Ioannina, Greece; Department of Ophthalmology, Elpis General Hospital of Athens, Dimitsanas 7, 11522 Athens, Greece.
Abstract:
The management of periocular basal cell carcinoma (BCC) is challenging due to its proximity to the eyeball. Vismodegib, a Hedgehog pathway inhibitor, has emerged as a therapeutic option for locally advanced and metastatic BCC. To critically appraise the relevant evidence, we conducted a systematic review of observational and experimental studies assessing the efficacy and safety of vismodegib for periocular BCC. Thirty-seven trials, including 435 patients, were eligible. No randomized trials were retrieved. Complete and overall clinical response rates were 20-88 % and 68-100 %, respectively. Disease progression was observed at a maximum rate of 14 %. Recurrence rates varied between 0 % and 31 %. The most common side effects were muscle cramps, dysgeusia, weight loss and alopecia. Treatment with vismodegib improved health-related quality of life. In conclusion, vismodegib represents an important novel treatment for advanced periocular BCC, with good response rates and acceptable tolerability profile. Nevertheless, its full potential needs clarification through randomized controlled trials.
Insights
Vismodegib effectively treats advanced periocular basal cell carcinoma (BCC), showing good response rates and improving quality of life. Further randomized trials are needed to confirm its full potential in managing this challenging eye-area cancer.
Area of Science:
- Oncology
- Dermatology
- Ophthalmology
Background:
- Periocular basal cell carcinoma (BCC) presents management challenges due to its anatomical location near the eye.
- Vismodegib, a Hedgehog pathway inhibitor, is a therapeutic option for advanced and metastatic BCC.
Purpose of the Study:
- To systematically review the efficacy and safety of vismodegib for periocular BCC.
- To critically appraise existing evidence on vismodegib treatment for this specific BCC subtype.
Main Methods:
- Systematic review of observational and experimental studies.
- Inclusion of 37 trials involving 435 patients.
- Exclusion of randomized controlled trials.
Main Results:
- Complete and overall clinical response rates ranged from 20-88% and 68-100%, respectively.
- Disease progression observed in up to 14% of cases, with recurrence rates varying from 0% to 31%.
- Common side effects included muscle cramps, dysgeusia, weight loss, and alopecia; health-related quality of life improved.
Conclusions:
- Vismodegib demonstrates significant efficacy and an acceptable safety profile for advanced periocular BCC.
- It represents a valuable therapeutic option, improving patient quality of life.
- Further randomized controlled trials are essential to fully elucidate its long-term potential and optimal use.
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