A meta-analysis of 764 patients with periocular or orbital basal cell carcinoma looked at how well vismodegib worked. The study found that about 38% of patients had their tumors completely disappear, and another 39% saw partial shrinkage. These results show the drug can be effective for these specific eye-area cancers.
However, the treatment came with a high burden of side effects. Over 90% of patients experienced some adverse events, with common issues like hair loss, muscle spasms, and weight loss. About 25% had severe side effects, and 31% stopped the drug because of toxicity.
After stopping the medication, the cancer came back in about 17% of patients. A notable 28% needed exenteration, which is a major surgery to remove the eye and surrounding tissue. These numbers highlight the challenges of managing this disease.
The study's limitations include high recurrence rates after stopping treatment and the significant side effect burden, which limits long-term use. Careful patient selection and close monitoring are essential for optimizing outcomes.