STEVIE: A Study of Vismodegib in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma

Dose intensity was defined as the percentage of actual number of doses received versus planned.

Time frame: Baseline to the data cut-off of 14 June 2017 (up to 6 years)

Duration on treatment was the number of days between first and last dose of study treatment.

Time frame: Baseline to the data cut-off of 14 June 2017 (up to 6 years)

Reasons for other included unknown, natural causes, cardiac decompensation, general state alteration, deterioration of general state, clinical deterioration taking into consideration patient's age, old age, and disease progression of mediastinal squamous cell carcinoma (SCC).

Time frame: Baseline to the data cut-off of 14 June 2017 (up to 6 years)

Adverse events were graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.03. Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activity of daily living with inability to perform bathing, dressing and undressing, feeding self, using the toilet, taking medications but not bedridden. Grade 4: An immediate threat to life. Urgent medical intervention is required in order to maintain survival.

Time frame: Baseline to the data cut-off of 14 June 2017 (up to 6 years)

Time frame: Baseline to the data cut-off of 14 June 2017 (up to 6 years)

BORR was defined as the percentage of participants achieving either a complete response (CR) or a partial response (PR) as assessed by the Investigator according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. CR was defined as the disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) required a reduction in short axis to \<10 mm. PR was defined as at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.

Time frame: Baseline to the data cut-off of 14 June 2017 (up to 6 years)

Population: Only included participants that had histologically confirmed measurable disease at baseline.

The Skindex-16 questionnaire includes three domains for the assessment of the effects of skin disease on participants' quality of life: symptoms, emotions and function. For each domain, responses from the questionnaire were transformed to a linear scale of 100 varying from 0 (never bothered, i.e., best) to 100 (always bothers, i.e., worst).

Time frame: Baseline to the data cut-off date of 14 June 2017 (up to 6 years).

Population: Only included participants that had histologically confirmed disease at baseline. For each time point, Number Analyzed refers to patients with non-missing values.

Duration of response was defined as the time interval between the date of the earliest qualifying response (CR or PR) and the date of disease progression or death for any cause. Median duration of response was estimated using Kaplan-Meier estimates.

Time frame: Baseline to the data cut-off of 14 June 2017 (up to 6 years)

Population: Only included participants that had histologically confirmed disease, measureable disease status at baseline (measureable or non-measureable) and reported a response of CR or PR.

OS was defined as the time from the date of first treatment to the date of death, regardless of the cause of death.

Time frame: Baseline to the data cut-off of 14 June 2017 (up to 6 years)

Population: Only included participants that had histologically confirmed disease and measureable disease status at baseline (measureable or non-measureable).

M.D. Anderson Symptom Inventory (MDASI) scale. The MDASI core instrument is a 19-item patient self-report questionnaire whose items comprise two scales, symptom severity and symptom interference. For 13 items (i.e., pain, fatigue, nausea, disturbed sleep, distress, shortness of breath, difficulty remembering things, lack of appetite, drowsiness, dry mouth, sadness, vomiting, and numbness or tingling), participants were asked to rate how severe the symptoms were when at their worst in the last 24 hours. For the remaining 6 items, participants were asked to rate how much the symptoms have interfered with 6 areas of functioning (i.e., general activity, walking, work, mood, relations with other people, and enjoyment of life) in the last 24 hours.

Time frame: 08-May-2013 (Protocol Version ≥ 4) to the data cut-off date of 14 June 2017 (approximately 4 years and 1 month).

Population: Included only participants who were enrolled after Protocol Version 4 was implemented, had non-missing data and average baseline score ≥ 4. Per protocol, the MDASI measurements were measured in Metastatic patients only.

M.D. Anderson Symptom Inventory (MDASI) scale. The MDASI core instrument is a 19-item patient self-report questionnaire whose items comprise two scales, symptom severity and symptom interference. For 13 items (i.e., pain, fatigue, nausea, disturbed sleep, distress, shortness of breath, difficulty remembering things, lack of appetite, drowsiness, dry mouth, sadness, vomiting, and numbness or tingling), participants were asked to rate how severe the symptoms were when at their worst in the last 24 hours. For the remaining 6 items, participants were asked to rate how much the symptoms have interfered with 6 areas of functioning (i.e., general activity, walking, work, mood, relations with other people, and enjoyment of life) in the last 24 hours.

Time frame: 08-May-2013 (Protocol Version ≥ 4) to the data cut-off date of 14 June 2017 (approximately 4 years and 1 month).

Population: Included only participants who were enrolled after Protocol Version 4 was implemented, had non-missing data and baseline score ≥4. Per protocol, the MDASI measurements were measured in Metastatic patients only.

PFS was defined as the time interval between the date of the first therapy and the date of progression or death for any causes, whichever occurs first. Disease progression was assessed by the investigator.

Time frame: Baseline to the data cut-off of 14 June 2017 (up to 6 years)

Population: Only included participants that had histologically confirmed disease and measureable disease status at baseline (measureable or non-measureable).

Time to response was defined as the interval between the date of first treatment and the date of first documentation of confirmed CR or PR (whichever occur first).

Time frame: Baseline to the data cut-off of 14 June 2017 (up to 6 years)

Population: Only included participants that had histologically confirmed measurable disease at baseline.