Assessments
The primary endpoint was ASIS questionnaire responses at week 12 and change from baseline (CFB) from patients ≥12 years old and with the assistance of caregivers for patients aged 9-11 years. ASIS is a vali-dated 17-item questionnaire that contains a signs domain (items A1-A9) and an impact domain (emotional impact [items A10-A15] and social impact [items A16-A17]) (Figure 1).29,30 All items in the questionnaire are scored on a 5-point adjectival response scale (score 0-4). Higher scores indicate severe symptoms or a negative impact of AV on appearance, emotions, or social activities, and a score of 0 indicates lack of negative impact from AV or positive impact on psychosocial well-being and QoL. Data were scored according to developer guidelines, reporting domain
(signs and impact), and subdomain (emotional impact and social im-pact) scores.29,30 The secondary endpoint was the IGA of AV severity at week 12. This measure uses a 5-point adjectival response scale (score 0 [clear] – 4 [severe]) and IGA success was defined as a 2-point decrease in IGA score and a score of 0 (clear) or 1 (almost clear) at week 12. Ad-ditional outcomes included clinician satisfaction with AV treatment for individual patients as well as safety.
Data Analysis
All patients who received ≥1 dose of sarecycline during the study comprised the safety population and were included in the safety data analyses. All patients in the safety population who had ≥1 question an-swered pertaining to the study’s primary endpoint at week 12 comprised the analytic population, which was included in analyses of all non-safety endpoints.
Continuous variables are presented as mean, median, standard devia-tion (SD), and number of patients. Categorical variables are presented as counts and percentages. Discrete variables were analyzed using Chi-square tests. Statistical differences in continuous measures were assessed using paired-sample t-tests. Missing values for IGA at weeks 4 and 8 were imputed using the last observation carried forward (LOCF). All statistical analyses were conducted using SAS statistical software, and P ≤.05 was considered significant.
Continuous variables are presented as mean, median, standard devia-tion (SD), and number of patients. Categorical variables are presented as counts and percentages. Discrete variables were analyzed using Chi-square tests. Statistical differences in continuous measures were assessed using paired-sample t-tests. Missing values for IGA at weeks 4 and 8 were imputed using the last observation carried forward (LOCF). All statistical analyses were conducted using SAS statistical software, and P ≤.05 was considered significant.
RESULTS
Patient and Caregiver Characteristics
The disposition of patients is summarized in Figure 2. A diverse population of 300 patients (N=253 in the analytic population at week 12) with AV were recruited, comprising 61.3% adults (N=184; mean age ± SD: 26.5 ± 7.6 years; 77.2% female) and 38.7% pediatric patients (N=116; 14.8 ± 1.7 years; 50.9% female). The majority of adult (61.4%) and pediatric (79.3%) patients were White/Caucasian (Table 1). The demographics of the analytic population were comparable to the safety population with 86.6% having moderate and 13.4% having severe facial AV. Many patients reported previous use of medications for AV, including topical retinoids (47.8%), benzoyl peroxide (39.5%), topical antibiotics (36.4%), and tetracycline or macrolide oral antibiotics (24.5%). Sarecycline monotherapy was used by 49.8% of patients. The most frequently used concomitant medications were topical retinoids (24.5%), topical antibiotics (13.4%), and adapalene/benzoyl peroxide combination (11.1%).
