Risk of squamous cell carcinoma in patients treated for basal cell carcinoma with Vismodegib or other therapies

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  VISMODEGIB
• Medical condition to be studied:
  Squamous cell carcinoma

Population studied

• Short description of the study population:
  Patients treated with vismodegib as part of 2 phase II (STEVIE and ERIVANCE) and 2 phase I clinical trials.
• Age groups:
  • Adults (18 to < 46 years)
  • Adults (46 to < 65 years)
  • Adults (65 to < 75 years)
  • Adults (75 to < 85 years)
  • Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Squamous cell carcinoma patients
• Estimated number of subjects:
  1700

Study design details

• Main study objective:
  To determine whether patients with basal cell carcinoma treated with vismodegib have an increased risk of SCC at one year compared to patients treated with other therapies.
• Outcomes:
  1) Incidence rates of SCC at one year post initiation of treatment, stratified by key potential risk factors for development of SCC (eg. age, gender, prior NMSC). 2) Standardized incidence rate ratio of SCC at one year post initiation of treatment.3) Relative hazard of SCC in vismodegib treated vs non-exposed patients.
• Data analysis plan:
  Baseline demographic characteristics were assessed for both cohorts at the time of treatment of BCC and were analyzed using descriptive statistics. Means and standard deviations were used for continuous variables, and percentages were used for categorical variables.Standardized incidence ratios were calculated for diagnosis of SCC at 1 year after treatment start (vismodegib treatment and surgery, respectively) using vismodegib treated patients as the exposed group. Relative risks were also calculated for clinical variables of age,gender, ethnicity, and basal cell nevus syndrome. Cox proportional hazards models were used to estimate hazard ratios (HR) and their 95% confidence intervals (CI) to assess the association between treatment with vismodegib and the development of SCC.