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Vismodegib in patients with advanced basal cell carcinoma: Primary analysis of STEVIE, an international, open-label
N Basset-Séguin1, A Hauschild2, R Kunstfeld3
1Department of Dermatology, Hôpital Saint Louis, 1 Avenue Claude Vellefaux, 75475, Paris, France.
Background:
The SafeTy Events in VIsmodEgib study (STEVIE, ClinicalTrials.gov, NCT01367665), assessed safety and efficacy of vismodegib-a first-in-class Hedgehog pathway inhibitor demonstrating clinical benefit in advanced basal cell carcinoma (BCC)-in a patient population representative of clinical practice. Primary analysis data are presented.
Patients And Methods:
Patients with locally advanced or metastatic BCC received oral vismodegib 150 mg/d until progressive disease, unacceptable toxicity, or withdrawal. Primary objective was safety. Efficacy variables were assessed as secondary end-points.
Results:
Evaluable adult patients (N = 1215, 1119 locally advanced; 96 metastatic BCC) from 36 countries were treated; 147 patients (12%) remained on study at time of reporting. Median (range) treatment duration was 8.6 (0-44) months. Most patients (98%) had ≥1 treatment-emergent adverse event (TEAE). The incidence of the most common TEAEs was consistent with reports in previous analyses. No association between creatine phosphokinase (CPK) abnormalities and muscle spasm was observed. Serious TEAEs occurred in 289 patients (23.8%). Exposure ≥12 months did not lead to increased incidence or severity of new TEAEs. The majority of the most common TEAEs ongoing at time of treatment discontinuation resolved by 12 months afterwards, regardless of Gorlin syndrome status. Response rates (investigator-assessed) in patients with histologically confirmed measurable baseline disease were 68.5% (95% confidence interval (CI) 65.7-71.3) in patients with locally advanced BCC and 36.9% (95% CI 26.6-48.1) in patients with metastatic BCC.
Conclusions:
The primary analysis of STEVIE demonstrates that vismodegib is tolerable in typical patients in clinical practice; safety profile is consistent with that in previous reports. Long-term exposure was not associated with worsening severity/frequency of TEAEs. Investigator-assessed response rates showed high rate of tumour control. CLINICALTRIALS.GOV: NCT01367665.
Insights
Vismodegib is a tolerable treatment for advanced basal cell carcinoma (BCC) in clinical practice, with a consistent safety profile and high tumor control rates. Long-term use did not worsen side effects.
Area of Science:
- Oncology
- Dermatology
- Pharmacology
Background:
- The SafeTy Events in VIsmodEgib study (STEVIE) evaluated vismodegib, a Hedgehog pathway inhibitor, in advanced basal cell carcinoma (BCC).
- Vismodegib has demonstrated clinical benefit in BCC patients.
- The STEVIE study aimed to assess vismodegib's safety and efficacy in a real-world clinical setting.
Purpose of the Study:
- To assess the safety and efficacy of vismodegib in a broad patient population representative of clinical practice.
- To present primary analysis data from the STEVIE study.
- To evaluate the tolerability and treatment outcomes of vismodegib in advanced BCC.
Main Methods:
- Patients with locally advanced or metastatic BCC received oral vismodegib 150 mg/day.
- Treatment continued until disease progression, unacceptable toxicity, or withdrawal.
- Safety was the primary objective, with efficacy variables assessed as secondary endpoints.
Main Results:
- 1215 patients were treated; 147 remained on study at reporting.
- 98% experienced treatment-emergent adverse events (TEAEs), consistent with prior reports; serious TEAEs occurred in 23.8%.
- Investigator-assessed response rates were 68.5% for locally advanced BCC and 36.9% for metastatic BCC.
Conclusions:
- Vismodegib is tolerable in typical clinical practice, with a safety profile consistent with previous reports.
- Long-term exposure did not increase the incidence or severity of TEAEs.
- Investigator-assessed response rates indicated high tumor control in advanced BCC.
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