Development and Validation of the Patient-Reported Submental Fat Rating Scale and the Patient-Reported Submental Fat Impact Scale.

BACKGROUND: No validated assessment tools are available to evaluate patient-reported outcomes specifically related to submental fat (SMF). OBJECTIVE: To develop and validate scales measuring the severity (Patient-Reported SMF Rating Scale [PR-SMFRS]) and psychological impact (Patient-Reported SMF Impact Scale [PR-SMFIS]) of SMF. MATERIALS AND METHODS: A literature review, content validation interviews (concept elicitation [n = 29] and cognitive debriefing [n = 15]) in adults with SMF, and expert interviews (n = 3) were conducted to develop the PR-SMFRS and PR-SMFIS. Psychometric validity (acceptability, reliability, and validity) for the PR-SMFRS and PR-SMFIS was assessed using data from 1 phase 2 and 5 phase 3 ATX-101 studies in patients with excess SMF. RESULTS: The PR-SMFRS was constructed as a single-item, 5-point rating of the SMF amount/size. The PR-SMFIS was constructed as a 6-item scale, with an 11-point numeric rating for each item. Both scales demonstrated acceptable psychometric properties (test-retest reliability and internal consistency). The anchor-based minimally important difference analysis suggests a 1-point improvement on the PR-SMFRS (scale of 0‒4) and a 3-point improvement on the PR-SMFIS (scale of 0‒10) represent clinically meaningful change. CONCLUSION: The PR-SMFRS and PR-SMFIS are reliable, valid instruments for assessing the severity and psychosocial impact, respectively, of SMF and detecting clinically meaningful change with intervention.

Measuring outcomes for temple hollowing treatment: Content validity of new and existing FACE-Q scales.

BACKGROUND: The FACE-Q, a validated, modular patient-reported outcome measure with global uptake, currently does not have a scale to measure the appearance of the temples. Objectives of our study were to develop a new FACE-Q scale for appearance of temples and assess content validity of two existing FACE-Q scales in the context of temple hollowing: Satisfaction with Facial Appearance and Psychological Function. METHODS: A heterogeneous sample of adults who were seeking or had received treatments for temple hollowing was recruited from three outpatient clinics in the United States. Semi-structured interviews using an interpretive description approach were completed to elicit concepts and generate an item pool and assess content validity of the two existing FACE-Q scales. The item pool data were used to develop preliminary Temple scale, which was refined based on patient and expert feedback. RESULTS: Participants (N = 15, 55 ± 9 years) described a range of esthetic concerns related to temple hollowing and its treatment. The data were used to draft the FACE-Q Satisfaction with Temples scale, which was refined through input from patients (N = 12) and clinicians (N = 5), resulting in a 16-item FACE-Q Satisfaction with Temples scale. The scale covers concepts of fullness, harmony, scenarios (eg, mirror, bright lights), age, and shape. Content validity of the two existing FACE-Q scales was substantiated. CONCLUSION: The FACE-Q Satisfaction with Temples scale fills an important gap in patient-reported outcome measurement in facial esthetics. The scale will be field-tested to finalize content and develop the scoring algorithm prior to implementation in clinical practice and research.

Patient-reported Outcomes in Chinese Subjects Treated with OnabotulinumtoxinA for Crow's Feet Lines.

BACKGROUND: Crow's feet lines (CFLs) can impact the emotional state, self-perception, and consciousness regarding appearance of patients. OBJECTIVE: This study sought to assess patient-reported outcomes after onabotulinumtoxinA treatment for CFLs among Chinese subjects. METHODS: A five-month, double-blind, randomized, parallel-group, placebo-controlled Phase III clinical study was conducted including Chinese adults with moderate-to-severe CFLs at maximum smile. Subjects were randomized 3:1 to 24 U of onabotulinumtoxinA or placebo and completed the 11-item Facial Line Outcomes (FLO-11) questionnaire and Facial Line Satisfaction Questionnaire (FLSQ) at baseline; on Days 8, 15, and 30; and monthly thereafter until Day 150. Item-level and/or domain analyses for the FLO-11 and FLSQ were conducted. RESULTS: Of 417 treated subjects, 316 received onabotulinumtoxinA and 101 received placebo. For all 10 validated stand-alone FLO-11 items, there was a significantly greater proportion of responders in the onabotulinumtoxinA group versus placebo (P<0.001) at Day 30 that was maintained through Day 150. Significant improvements at Day 30 were reported for all FLSQ items and the FLSQ Follow-up Impact Domain (P≤0.01). CONCLUSION: FLO-11 and FLSQ data indicated high satisfaction and significant improvements in appearance-related and emotional impacts through Day 150 in patients treated with onabotulinumtoxinA for moderate-to-severe CFLs in Chinese subjects. TRIAL REGISTRATION: ClinicalTrials.gov identifier no. NCT02195687.

Erythema of Rosacea Affects Health-Related Quality of Life: Results of a Survey Conducted in Collaboration with the National Rosacea Society.

INTRODUCTION: Persistent facial erythema associated with rosacea may negatively impact quality of life (QoL), self-esteem, and self-confidence. We evaluated burden and health-related QoL (HRQoL) impacts of centrofacial erythema of rosacea. METHODS: A cross-sectional, Web-based survey conducted in collaboration with the National Rosacea Society enrolled adults who self-reported having received a physician diagnosis of rosacea and self-evaluated their current erythema as mild to severe on the validated Subject Self-Assessment for Rosacea Facial Redness. Sociodemographic and clinical characteristics, rosacea symptoms, and their impacts on QoL [validated Impact Assessment for Rosacea Facial Redness (IA-RFR)] and HRQoL [validated Dermatology Life Quality Index (DLQI)] were recorded. RESULTS: A total of 708 eligible respondents completed the survey (white/Caucasian, 93.5%; female, 83.1%; mean age, 52.4 years). Respondents had mild (59.2%), moderate (33.2%), or severe (7.6%) erythema. The most bothersome symptoms were persistent facial erythema (69.2%) and blushing/flushing (60.9%). Mean IA-RFR scores showed negative impacts across all severities of erythema. The mean (standard deviation) total DLQI score was 5.2 (6.0) overall [mild erythema, 3.8 (4.9); moderate, 5.7 (5.4); severe, 13.4 (8.9); P < 0.0001]. CONCLUSION: Centrofacial erythema of rosacea represents a substantial HRQoL burden, especially for those with more severe erythema. FUNDING: Allergan plc, Dublin, Ireland.

Psoriasis treatment patterns: a retrospective claims study.

Objective: The objective was to characterize psoriasis treatment patterns, including estimating persistence and describing subsequent events (i.e. switching and restarting) for all systemic therapies. Methods: This retrospective cohort study utilized Truven MarketScan databases from 1 January 2014 to 31 December 2016 to investigate persistence, switching and restarting in new users of systemic psoriasis medications. Descriptive statistics, time-to-event analyses and a Cox proportional hazards regression were conducted. Results: A total of 5205 patients met inclusion criteria. Regardless of treatment type, >50% lost persistence by 12 months. Patients newly initiating acitretin or non-TNF biologic experienced the highest loss of persistence (85.2%, 73.8%, respectively). Patients initiating a TNF-α inhibitor or apremilast experienced the lowest loss (51.8%, 56.4% respectively). Treatment type had a statistically significant effect on persistence loss (adjusted hazard ratio: 0.86, 95% CI: 0.81, 0.91). Restarting was the most commonly observed event for patients on an oral or biologic (60.2%, 79.9%, respectively). The most common switch from an oral was to a TNF-α inhibitor, while apremilast often followed biologics. Conclusion: Most patients lost persistence on initial treatment by 12 months, and the majority restarted treatment. This may indicate poor compliance or the cyclical nature of psoriasis. More patients switched from an oral to biologic than vice versa, likely due to formulary design and preference for orals. Studies are needed to investigate underlying reasons and patient characteristics that differentiate treatment utilization.

The Impact of Acute Kidney Injury on the Risk of Mortality and Health Care Utilization Among Patients Treated With Polymyxins for Severe Gram-Negative Infections.

BACKGROUND: With the rise of antibiotic resistance, polymyxin use has re-emerged but with a concern of renal toxicity. This study aims to assess mortality, length of stay, and total hospitalization cost associated with acute kidney injury (AKI) among recipients of intravenous (IV) sodium colistimethate (CMS) or IV polymyxin B (PMB). METHODS: We conducted a retrospective database analysis using the Premier database from January 1, 2012, through September 30, 2015. Adults ≥18 years of age who were admitted for inpatient treatment with ≥3 consecutive days of CMS or PMB were included. Generalized linear models compared patients who developed AKI with those who did not. Models were adjusted for patient and clinical characteristics. RESULTS: A total of 4886 patients were included; 4103 patients received CMS, and 783 received PMB. In the multivariable analyses, the presence of AKI was associated with higher in-hospital mortality in both the CMS cohort (adjusted odds ratio [aOR], 2.3; 95% confidence interval [CI], 1.9-2.7; P < .001) and the PMB cohort (aOR, 2.7; 95% CI, 1.8-4.2; P < .001). In both cohorts, patients who developed AKI experienced longer hospital stays (9.7 days and 11.6 days in the CMS and PMB cohorts, respectively; P < .001). The mean total hospitalization costs for patients who developed AKI were $47 820 higher (95% CI, $34 918-$60 722) in the CMS cohort and $35 244 higher (95% CI, $17 561-$52 928) in the PMB cohort. CONCLUSIONS: The clinical and economic burden of AKI in the context of polymyxin use is substantial. The use of effective antibiotics with limited toxicity should remain a priority.

Characterizing the Strength of Evidence in FDA Labels for Pharmacogenomic Biomarker-Guided Medication Use.

There is great heterogeneity in drug treatment response that is thought to be due to individual-level allelic variation in pharmacogenomic biomarkers. FDA Drug Labels provide information to guide pharmacogenomic biomarker use. Yet, the strength of evidence for clinical validity and clinical utility is lacking. We characterized the strength of evidence and treatment recommendations contained in FDA Drug Labels as of December 2015. Pharmacogenomic biomarker information was provided for 137 drugs, involving 49 pharmacogenomic biomarkers, constituting 166 drug-biomarker pairs. Convincing/adequate evidence of clinical validity was found for 46% of pairs, of clinical utility for 29% of pairs, and of both, for 27% of pairs. Despite evidence of convincing/adequate validity/utility, no treatment recommendation was provided for 37% of pairs. Germline biomarkers represented nearly three-quarters of all drug-biomarker pairs, however, only 29% and 16% of pairs had convincing/adequate evidence for clinical validity and clinical utility, respectively. Separately, somatic biomarkers that serve as molecular targets for targeted therapies, had convincing/adequate evidence for 95% of pairs for clinical validity, and for 67% for clinical utility. The strength of evidence for pharmacogenomic biomarker use is low, underscoring the need for additional research to achieve the promise of precision medicine.