Vismodegib Safety Profile in Long-Term ‘Real-World’ Use in Advanced Basal Cell Carcinoma


Key Points

  • Median duration of vismodegib exposure was 36.4 weeks; treatment was discontinued due to adverse events 36% of patients.
  • Response rates were 67% in locally advanced disease, including many complete responses, and 38% in metastatic disease.

In a preplanned interim analysis of the international open-label STEVIE trial reported in The Lancet Oncology, Basset-Seguin et al have provided safety data and efficacy outcomes with the use of vismodegib (Erivedge) for 1 year in patients with advanced basal cell carcinoma. STEVIE was designed to assess the safety of vismodegib in situations similar to routine practice with long-term follow-up.

Study Details

In this trial, adult patients with locally advanced basal cell carcinoma deemed ineligible for surgery or metastatic basal cell carcinoma are treated with vismodegib at 150 mg per day on a continuous basis in 28-day cycles. Safety is assessed on day 1 of each cycle. An interim analysis was planned after 500 patients achieved 1 year of follow-up. Between June 2011 and November 2014, 1,227 patients were enrolled.

At data cutoff in November 2013, 499 patients, including 468 with locally advanced disease and 31 with metastatic disease, had received study drug and had the potential to be followed up for ≥ 12 months.

Safety Data

Median duration of vismodegib exposure was 36.4 weeks (interquartile range = 17.7–62.0 weeks). Overall, treatment was discontinued in 400 (80%) patients, due to adverse events in 180 (36%), progressive disease in 70 (14%), and patient request in 51 (10%). The most common adverse events leading to discontinuation were muscle spasms (9%), dysgeusia (6%), and weight loss (5%).

The most common adverse events of any grade were muscle spasms (64%), alopecia (62%), dysgeusia (54%), weight loss (33%), asthenia (28%), decreased appetite (25%), ageusia (22%), diarrhea (17%), nausea (16%), and fatigue (16%). Grade 3, 4, and 5 adverse events occurred in 34%, 5%, and 4% of patients, respectively. The most common grade 3 events were muscle spasms (8%) and decreased weight (3%). Grade 4 events consisted of one case each of asthenia, decreased weight, ageusia, and fatigue. Median times to onset of common adverse events of any grade were 2.9 months for muscle spasms, 5.5 months for alopecia, and 6.5 months for dysgeusia.

Serious adverse events were reported in 22%, with pneumonia (2%) being the most common. Of 31 patients who died, 21 died from adverse events, with 2 deaths (due to cardiorespiratory arrest and myocardial infarction) considered possibly related to study treatment.

Response Rates

As assessed by investigators, response occurred in 302 (66.7%) of 453 evaluable patients with locally advanced disease (including 153 complete responses) and in 11 (37.9%) of 29 patients with metastatic disease (including 2 complete responses).

The investigators concluded: “This study assessed the use of vismodegib in a setting representative of routine clinical practice for patients with advanced basal cell carcinoma. Our results show that treatment with vismodegib adds a novel therapeutic modality from which patients with advanced basal cell carcinoma can benefit substantially.”

Johan Hansson, MD, of the Karolinska Institutet, is the corresponding author for the Lancet Oncology article.

The study was funded by F. Hoffmann-La Roche. For full disclosures of the study authors, visit

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