Vismodegib Demonstrates Efficacy in Basal Cell Carcinoma

Basal cell carcinoma
Basal cell carcinoma
Vismodegib treatment in local advanced and metastatic basal cell carcinoma is assessed.

Treatment with hedgehog pathway inhibitor vismodegib was well tolerated and associated with high rates of response in a real-world cohort of patients with locally advanced and metastatic basal cell carcinoma (BCC), according to study findings published in Dermatologic Therapy.

In the study, a team of researchers from Turkey retrospectively evaluated the tolerability and efficacy of vismodegib treatment in 29 patients (median age at diagnosis, 73 years) with advanced and metastatic BCC.

Patients in the real-world study received 150 mg oral vismodegib until disease progression. The investigators evaluated treatment response in patients with measurable or non-measurable disease, and response time was also examined. Additional assessments included toxicity, progression-free survival (PFS), and overall survival (OS).

The location of BCC was predominantly in the head and neck (86.2%), and 17.2%of   patients presented with metastatic disease. Gorlin’s syndrome was reported in 10.3% patients. The most common pathological subtypes included nodular (44.9%), mixed (27.6%), and infiltrative (20.7%) types. Before vismodegib initiation, more than half of all patients had previously received radiotherapy (51.7%), and 62.1% of patients had undergone surgery.

The complete response rate to vismodegib at the median follow-up of 17 months was 27.6%. In addition, the rates of partial response and stable response were 55.2% and 10.3%, respectively. Overall, the objective response rate was 82.8%. According to the investigators, treatment responses were most often observed within the first 2 months of starting therapy.

Across all patients, the median OS was 43.3 months (95% CI, 25.6–61.1). In addition, the median PFS was 15.7 months (95% CI, 12.2–19.3) in patients with locally advanced BCC and 12.1 months (95% CI, 2.9–21.2) for patients with metastatic BCC. More than half of patients (53.8%) received subsequent chemotherapy or underwent surgical treatment following disease progression.

The most frequently reported adverse events during vismodegib treatment included fatigue (58.6%), muscle spasms (48.3%), alopecia (13.8%), and weight loss (13.8%). More than a quarter of patients (27.6%) died during the study.

According to a univariable analysis for OS, only subsequent chemotherapy was significantly better than no treatment (hazard ratio [HR], 10.6; 1.03–108.6; P =.04) or surgery (HR, 8.72; 95% CI, 1.01–75.19; P =.04).

Limitations of the study included the small sample size as well as its retrospective design, which the researchers suggested contributed to the heterogenous nature of the patient groups.

The investigators concluded that their “study with real life data shows that vismodegib treatment in locally advanced and metastatic BCC was well tolerated and highly effective.”


Gürbüz M, Doğan İ, Akkuş E, et al. Efficacy and tolerability of vismodegib treatment in locally advanced and metastatic basal cell carcinoma: Retrospective real-life data. Dermatol Ther. Published online September 3, 2021. doi:10.1111/dth.15122