Ariane K. Kawata

Migraine imposes significant burden on patients, their families and health care systems. In this study, we compared episodic to chronic migraine sufferers to determine if migraine status predicted headache-related disability, health-related quality of life (HRQoL) and health care resource utilization.A Web-based survey was administered to panelists from nine countries. Participants were classified as having chronic migraine (CM), episodic migraine (EM) or neither using a validated questionnaire. Data collected and then analyzed included sociodemographics, clinical characteristics, Migraine Disability Assessment, Migraine-Specific Quality of Life v2.1, Patient Health Questionnaire and health care resource utilization.Of the respondents, 5.7% had CM and 94.3% had EM, with CM patients reporting significantly more severe disability, lower HRQoL, higher levels of anxiety and depression and greater health care resource utilization compared to those with EM.These results provide evidence that will enhance our understanding of the factors driving health care costs and will contribute to development of cost-effective health care strategies.

Our objective was to characterize patterns of preventive medication use in persons with episodic migraine (EM) and chronic migraine (CM).Several classes of medications are used both on- and off-label for the prevention of migraine, including β-blockers (eg, propranolol, timolol), tricyclic antidepressants (eg, amitriptyline), anti-epileptic drugs (eg, topiramate, valproic acid), and neurotoxins (eg, onabotulinumtoxinA).Preventive medication use and reasons for discontinuation were collected in an international, Web-based, cross-sectional survey of adults with migraine during 2010. Descriptive analyses were conducted on demographics and headache-related disability as measured by the Migraine Disability Assessment Scale, stratified by use of preventive medication, and EM or CM. Univariate and multivariate logistic regression models were constructed to assess predictors of preventive medication use.One thousand one hundred and sixty-five respondents completed the survey. Only 28.3% of EM and 44.8% of CM respondents were currently using preventive medication; any use of prophylaxis (prior or current) was reported by 43.4% of those with EM and 65.9% with CM. The mean number of prophylactic medications ever used was 2.92 for EM and 3.94 for CM. Antidepressants were used most frequently (EM 60.9%; CM 54.7%), followed by β-blockers (EM 35.4%; CM 36.8%) and anti-epileptics (EM 28.6%; CM 36.3%). Odds of preventive medication use were higher among CM than EM, adjusting for age, gender, race, years of daily headache, and country (odds ratio 2.72; 95% confidence interval 2.15 to 3.57). Greater headache-related disability and older age were also associated with greater odds of ever having used prophylaxis, regardless of headache frequency.Less than half the persons with EM and CM were currently using preventive medication for migraine, with treatment rates being higher for CM, as expected. Those with CM tried more medications than those with EM, possibly reflecting higher levels of treatment need.

The relationship between self-reported quality of life and disability and disease severity was evaluated in subjects with treatment-failure gout (n = 110) in a prospective, 52-week, observational study.Subjects had symptomatic crystal-proven gout of at least 2 years' duration and intolerance or refractoriness to conventional urate-lowering therapy. Serum uric acid (sUA) concentration, swollen and tender joint counts, frequency and severity of gout flares, tophus assessments, comorbidities, and patient-reported outcomes data [Medical Outcomes Study Short Form-36 (SF-36), Health Assessment Questionnaire-Damage Index] were collected. Analyses included correlations of patient-reported outcomes with clinical variables and changes in clinical status.Mean age of study subjects was 59 years. Mean scores on SF-36 physical functioning subscales were 34.2-46.8, analogous to persons aged >or= 75 years in the general population. Subjects with more severe gout at baseline had worse health-related quality of life (HRQOL) in all areas (p < 0.02 for all measures), compared to patients with mild-moderate disease. Number of flares reported in past year, number of tender joints, swollen joints, and tophi correlated significantly with some or all HRQOL and disability measures. sUA was not significantly correlated with any HRQOL or disability measure. Subjects with comorbidities experienced worse physical, but not mental, functioning.Severe gout is associated with poor HRQOL and disability, especially for patients who experience more gout flares and have a greater number of involved joints. Subject perceptions of gout-related functioning and pain severity appear to be highly sensitive indicators of HRQOL and disability.

Preference-based health index scores provide a single summary score assessing overall health-related quality of life and are useful as an outcome measure in clinical studies, for estimating quality-adjusted life years for economic evaluations, and for monitoring the health of populations. We predicted EuroQoL (EQ-5D) index scores from patient-reported outcomes measurement information system (PROMIS) global items and domain item banks. This was a secondary analysis of health outcome data collected in an internet survey as part of the PROMIS Wave 1 field testing. For this study, we included the 10 global items and the physical function, fatigue, pain impact, anxiety, and depression item banks. Linear regression analyses were used to predict EQ-5D index scores based on the global items and selected domain banks. The regression models using eight of the PROMIS global items (quality of life, physical activities, mental health, emotional problems, social activities, pain, and fatigue and either general health or physical health items) explained 65% of the variance in the EQ-5D. When the PROMIS domain scores were included in a regression model, 57% of the variance was explained in EQ-5D scores. Comparisons of predicted to actual EQ-5D scores by age and gender groups showed that they were similar. EQ-5D preference scores can be predicted accurately from either the PROMIS global items or selected domain banks. Application of the derived regression model allows the estimation of health preference scores from the PROMIS health measures for use in economic evaluations.

Background:The 6-minute walk test distance (6MWD) predicts mortality in COPD.Whether variability in study type (observational versus interventional), region performed limits use of the test as a stratification tool or outcome measure for therapeutic trials is unclear.Methods: Original data from 14,497 COPD patients from 6 observational (n = 9641) and 5 interventional (n=4856) studies larger than 100 patients and longer than 6 months in duration were included.The geographical, anthropometrics, FEV 1 , dyspnea, co-morbidities and health status scores were measured.Associations between 6MWD with mortality, hospitalizations and exacerbations adjusted by study type, age and gender were evaluated.Thresholds for outcome prediction were calculated using receiver-operating curves.The change in 6MWD after inhaled bronchodilator treatment and surgical lung volume reduction (LVRS) were analyzed to evaluate the responsiveness of the test as an outcome measure. Results:The 6MWD was significantly lower in non-survivors, those hospitalized or who exacerbated compared with those without events at 6, 12 and > 12 months.At these time points, the 6MWD ROC-AUC to predict mortality was 0.71, 0.70 and 0.68 and for hospitalizations was 0.61, 0.60 and 0.59.After treatment, the 6MWD was not different between placebo or bronchodilators but increased after LVRS compared with medical therapy.Variation across study types (observational or therapeutic) or regions did not confound the ability of 6MWD to predict outcome. Conclusions:The 6MWD can be used to stratify COPD patients for clinical trials and interventions aimed at modifying exacerbations, hospitalizations or death.

Abstract Background Family caregivers comprise a critical component in the care of Alzheimer's disease (AD) patients. Among their many tasks, caregivers are responsible for administering and managing medications. Effective interventions incorporate the needs of both the AD patient and the caregiver, and understanding treatment preferences may maximize intervention effectiveness. Transdermal patches may offer advantages over conventional oral formulations. Methods A 24‐week randomized controlled trial compared the rivastigmine patch to the rivastigmine capsule and placebo in patients with probable AD. At baseline and Weeks 8 and 24, the AD Caregiver Preference Questionnaire (ADCPQ) was used to evaluate caregiver expectations, preferences and satisfaction with treatment. Double‐dummy treatment blinding ensured that caregiver preference for the patch or capsule was not confounded by perceptions of efficacy or tolerability. Reasons for preference were also elicited. The analytic sample included caregivers who completed the ADCPQ at Weeks 8 and/or 24. Results One thousand and fifty‐nine caregivers completed the ADCPQ. More than 70% of caregivers preferred the rivastigmine patch to the capsule. The patch was significantly preferred to the capsule with respect to ease of following the schedule and ease of use. Caregivers indicated greater satisfaction overall, greater satisfaction with administration, and less interference with daily life with the patch versus the capsule (all p ≤ 0.01). Conclusion Caregivers of AD patients preferred the patch to the capsule for drug delivery. Preference for the rivastigmine patch could potentially lead to improved compliance and improved clinical benefits. Copyright © 2007 John Wiley &amp; Sons, Ltd.

To evaluate the performance and score interpretability of the Headache Impact Test (HIT-6) questionnaire in a headache patient population.The HIT-6 is a standardized questionnaire designed to assess headache-related disability. Norms have been established using randomly sampled respondents from the general population. This study evaluated the psychometric properties of the HIT-6 scale among new adult patients who presented for treatment at a university headache-specialty practice.Participants were 309 new patients who presented for treatment at a headache-specialty clinic in an academic medical center.Respondents ranged in age from 18 to 91 years, with an average age of 41 years (SD = 13 years) and were predominantly female (77%). Over half (57%) experienced chronic daily headache. Scores ranged from 38 to 78, with an average of 65.6 (SD = 7.0) and the majority (87%) had "severe impact" (60 or higher). The scale showed high reliability (alpha= 0.87) and an exploratory factor analysis showed large loadings from |0.57| to |0.86| and suggested a single disability factor. Scores were moderately negatively correlated with Short Form Health Survey (SF-36) subscales, ranging from -0.22 for mental health to -0.57 for social functioning. Item response theory analyses showed that half of the items functioned well across the disability continuum, while the remaining items discriminated between low and high levels of headache impact.These findings suggest that the HIT-6 is useful for assessing headache-related disability among patients who seek headache-specialty care.

To describe a survey methodology to evaluate headache characteristics and burden and to present baseline characteristics for the international cohort of survey participants.A targeted, web-based methodology was used to recruit and survey subjects with migraine in 10 countries. Based on reported symptoms, subjects meeting ICHD-2 criteria for migraine were included; eligible subjects were classified as chronic (≥ 15 headache days per month) or episodic (< 15 headache days per month). Outcomes included sociodemographic and clinical characteristics, resource utilization, disability, health-related quality of life, anxiety, depression and productivity.Of 23,312 survey respondents, 11,897 were eligible and 9715 (81.7%) completed the survey; subjects were 81.5% female; 5.7% (n = 555) had chronic migraine.This is the first large international cohort of persons with chronic and episodic migraine studied using a web-based approach, a methodology well suited to the study of the burden of migraines.

Abstract Background Calcitonin gene‐related peptide (CGRP) inhibitors were introduced in the United States (US) in 2018. To understand the changing patterns of preventive treatment following the introduction of these new agents, we must first characterize the patterns which preceded their introduction. Objective To characterize the burden, unmet need, and treatment patterns in patients with migraine initiating preventive migraine medications before the introduction of CGRP inhibitors in the US. Methods Between March 2016 and October 2017, we enrolled episodic (EM) and chronic migraine (CM) patients initiating or changing preventive treatment at primary care or neurology clinic visits in the US, in a real‐world observational study using a prospective cohort design. At baseline and monthly thereafter for 6 months, we collected data from study sites and patients on migraine frequency, treatment modifications, migraine impact on functioning, and work productivity for a descriptive analysis of migraine patient experience and treatment patterns. Results From the sample of 234 completers, 118 had EM (50.4%) and 116 had CM (49.6%). Mean age at enrollment was 41 years (SD = 12) and mean age at first migraine diagnosis was 22 years (SD = 11). Most participants were females ( n = 204/234; 87.2%) and white ( n = 178/234; 76.1%). The majority ( n = 164/234; 70.1%) had not used preventive migraine treatment in the 5 years prior to enrollment (treatment naïve). At baseline, mean monthly migraine days were 9.6 days (SD = 5.0) for the preventive treatment naïve group and 12.4 days (SD = 7.0) for treatment experienced patients. The majority had severe Migraine Disability Assessment (Grade IV, total score ≥21), including 67.1% ( n = 110/164) of the preventive treatment naïve and 77.1% ( n = 54/70) of the preventive treatment experienced patients. Headache Impact Test total scores indicating severe impairment (score &gt;59) occurred in 88.4% ( n = 145/164) of the treatment naïve and 88.6% ( n = 62/70) of treatment experienced patients. Mean work productivity loss as measured by the Work Productivity and Activity Impairment questionnaire in the subsample of employed patients was 53.3% loss. The most used acute medications at baseline were nonsteroidal anti‐inflammatory agents ( n = 124/234; 53.0%), acetaminophen‐based products ( n = 112/234; 47.9%), and triptans ( n = 105/234; 44.9%). The most commonly initiated preventive treatments were topiramate ( n = 100/234; 42.7%), tricyclic antidepressants ( n = 39/234; 16.7%), beta‐blockers ( n = 26/234; 11.1%), and onabotulinumtoxinA ( n = 24/234; 10.3%). Over the 6‐month follow‐up period, almost half of patients ( n = 116/234, 49.6%) modified their preventive treatment and discontinued treatment ( n = 88/312 total modifications; 28.2%) or modified their pattern of use by increasing, decreasing, or skipping doses ( n = 224/312 total modifications; 71.8%), often without seeking medical advice. Avoiding side effects was the main reason reported among patients who discontinued ( n = 52/88; 59.1%), decreased frequency or dose ( n = 37/89; 41.6%), and skipped doses ( n = 29/86; 33.7%). Perceived lack of efficacy was another frequent reason reported among those who discontinued ( n = 20/88; 22.7%), decreased frequency or dose ( n = 15/89; 16.9%), and skipped doses ( n = 18/86; 20.9%). Despite initiation of preventive treatment and improvements observed in number of headache and migraine days, migraine patients continued to experience substantial disability, headache impact, and reduced productivity throughout the 6‐month follow‐up period. Conclusions Prior to 2018, the burden of migraine was high for patients initiating preventive treatments. Despite having more than 9 days of migraine per month on average, the majority (70.1%) of patients initiating prevention had been treatment naïve, indicating underuse of preventive treatments. The preventive treatments used in this study were poorly tolerated and were reported by patients to lack efficacy, resulting in suboptimal adherence. The high discontinuation rates suggest that the preventive medications being offered during the period of the study did not meet the treatment needs of patients. In addition, the decisions by about half of patients to alter their prescribed treatment plan without consulting their provider can pose substantial health risks. These findings pertain to the broad set of preventive treatments initiated in this study and do not support inferences about individual preventive treatments, due to limitations in sample size. These findings suggest the need for more effective and better tolerated preventive treatment options.

Limited data are available on racial differences in clinical characteristics and burden in adult female acne. The objective was to describe racial differences in clinical characteristics, psychosocial impact, perceptions, behaviors, and treatment satisfaction in facial adult female acne.Cross-sectional, web-based survey.Diverse sample of United States women.Women between the ages of 25 and 45 years with facial acne (≥25 visible lesions).Outcomes included sociodemographic characteristics, psychosocial impacts, perceptions, behaviors, and treatment satisfaction. Racial differences were evaluated using descriptive statistics and t-test/chi-square analyses.208 females participated (mean age 35±6 years); 51.4 percent were White/Caucasian and 48.6 percent were non-White/Caucasian women [Black/African American (n=51); Hispanic/Latina (n=23); Asian (n=16); Other (n=ll)]. Age of acne onset (mean 14.8±5 vs. 17.0±8 years, p<0.05) and acne concern occurred earlier (16.6±7 vs. 19.3±9 years, p<0.05) in White/Caucasian than non-White/Caucasian subjects. Facial acne primarily presented on chin (28.0%) and cheeks (30.8%) for White/Caucasian women versus cheeks (58.4%) for non-White/Caucasian women. Non-White/Caucasian women experienced more postinflammatory hyperpigmentation than White/Caucasian women (p<0.0001). Facial acne negatively affected quality of life (QoL) in both groups, and most participants (>70%) reported some depression/anxiety symptoms. More White/Caucasian than non-White/Caucasian women were troubled by facial acne (88.8% vs. 76.2%, p<0.05). Lesion clearance was most important to White/Caucasian women (57.9 vs. non-White/Caucasian 31.7%, p<0.001); non-White/Caucasian females focused on postinflammatory hyperpigmentation clearance (41.6% vs. Caucasian 8.4%, p<0.0001).RESULTS highlight racial differences in participant-reported clinical characteristics, attitudes, behaviors, and treatment satisfaction. These findings may inform clinicians about racial differences in facial adult female acne and guide treatment recommendations toward improving care.

Objective The objective of this study was to evaluate the measurement properties of the Migraine Physical Function Impact Diary (MPFID), a novel patient‐reported outcome (PRO) measure for assessing the impact of migraine on physical functioning. Methods In a prospective, observational study, adults with episodic migraine (EM) or chronic migraine (CM) used an eDiary to complete the MPFID (assessing daily impacts of migraine on physical function) and a headache diary (capturing migraine days, migraine pain intensity, and migraine interference) each day, and other PRO instruments related to migraine. Item‐level evaluation, item response theory (IRT), and exploratory factor analysis (EFA) methods were applied to identify domains, select final MPFID items, and develop scoring procedures. Psychometric properties of the final 13‐item MPFID were evaluated using confirmatory factor analysis and tests of reliability (Cronbach's α for internal consistency and intra‐class correlation [ICC] for test–retest) and validity (convergent and known‐groups). Results The study enrolled 569 adults with chronic or episodic migraine, mean (SD) age 39.9 (12.0) years and 87.2% female. Item‐level analyses based on interim data informed selection of a set of 13 items for the MPFID, through evaluation of floor/ceiling effects, item‐to‐item correlations, factor loadings, and IRT‐based fit/misfit statistics. Two domain scores (EA: Impact on Everyday Activities; PI: Physical Impairment) and a global item score for impact on everyday activities were identified. EA and PI domains exhibited high internal consistency ( α = 0.97; α = 0.93) and good test–retest reliability among stable subjects (ICCs = 0.74 and 0.77). Convergent validity was demonstrated by moderate correlations ( r = ±0.50‐0.68; P &lt; .0001) between MPFID domain scores and number of migraine days, headache days, bed days, and other migraine‐related PRO instruments. EA and PI scores differentiated between groups who varied by number of migraine days, migraine interference levels, migraine pain intensity, and median split groups of scores based on other PROs instruments ( P &lt; .05). Conclusions The MPFID has robust psychometric properties (ie, reliability and validity). Findings supported two distinct domains about the impact of migraine on physical functioning: Impact on Everyday Activities and Physical Impairment. Both domain scores showed evidence of excellent reliability and construct validity in assessing the impacts of migraine on physical functioning.

Many medications present special risks when used by older adults (ie, those aged > or = 65 years) and are considered potentially inappropriate for this population. The Beers criteria are often used to identify such medications. Past research has documented that use of Beers drugs is common among older adults.The aim of this work was to examine factors associated with potentially inappropriate drug use among rural community-dwelling older adults using a conceptual framework adapted from the Andersen-Newman behavioral model of health service use.This was a population-based, cross-sectional survey. Data were collected via face-to-face home interviews between 2002 and 2004. Rural community-dwelling older adults residing in a single county in North Carolina were eligible. Potentially inappropriate drug use was operationalized using the Beers criteria. Data concerning predisposing (ie, age, sex, race, education, and marital status), enabling (ie, social support and insurance status), need (ie, disability and history of major depression, hypertension, osteoarthritis, back problems, or other comorbidities), and utilization factors (ie, number of medications used) were collected.Data were gathered from 892 people, with information on medication use available for 800. Two hundred thirteen of these 800 participants (26.6%) used > or = 1 Beers drug. Compared with individuals who used no Beers drugs, those who used > or = 1 Beers drug reported lower levels of social support (odds ratio [OR], 0.94; 95% CI, 0.90-0.99) and higher levels of disability (OR, 1.48; 95% CI, 1.11-1.97), used more medications (OR, 1.07; 95% CI, 1.01-1.13), and were more likely to have a history of major depression (OR, 1.67; 95% CI, 1.05-2.66), hypertension (OR, 1.58; 95% CI, 1.07-2.33), osteoarthritis (OR, 1.58; 95% CI, 1.09-2.29), and back problems (OR, 1.72; 95% CI, 1.19-2.47).As suggested by the Andersen-Newman model, the risk of potentially inappropriate drug use is highest among those with the greatest medication needs, as evidenced by poorer health status in this sample of rural community-dwelling older patients.

Typically regarded as an adolescent condition, acne among adult females is also prevalent. Limited data are available on the clinical characteristics and burden of adult female acne. The study objective was to describe clinical characteristics and psychosocial impact of acne in adult women.Cross-sectional, web-based survey.Data were collected from a diverse sample of United States females.Women ages 25 to 45 years with facial acne (≥25 visible lesions).Outcomes included sociodemographic and clinical characteristics, perceptions, coping behaviors, psychosocial impact of acne (health-related quality of life using acne-specific Quality of Life questionnaire and psychological status using Patient Health Questionnaire), and work/productivity.A total of 208 women completed the survey (mean age 35±6 years), comprising White/Caucasian (51.4%), Black/African American (24.5%), Hispanic/Latino (11.1%), Asian (7.7%), and Other (5.3%). Facial acne presented most prominently on cheeks, chin, and forehead and was characterized by erythema, postinflammatory hyperpigmentation, and scarring. Average age of adult onset was 25±6 years, and one-third (33.7%) were diagnosed with acne as an adult. The majority (80.3%) had 25 to 49 visible facial lesions. Acne was perceived as troublesome and impacted self-confidence. Makeup was frequently used to conceal acne. Facial acne negatively affected health-related quality of life, was associated with mild/moderate symptoms of depression and/or anxiety, and impacted ability to concentrate on work or school.RESULTS highlight the multifaceted impact of acne and provide evidence that adult female acne is under-recognized and burdensome.

Acne impairs quality of life, but its effect on different races/ethnicities is unclear. This study evaluated racial/ethnic differences in acne-related quality of life and psychological symptoms among female adults.A Web-based survey was conducted with U.S. female adults (25-45 years old) with facial acne (≥25 visible lesions). Outcomes included sociodemographics, clinical characteristics, acne-related quality of life (Acne-Specific Quality of Life Questionnaire), psychological symptoms (Patient Health Questionnaire), and work/school productivity. Racial/ethnic differences were evaluated using descriptive statistics and analysis of variance/chi-square analyses.Three-hundred twelve subjects (Black = 30.8%, Hispanic = 17.6%, Asian/other = 17.3%, White = 34.3%) completed the survey (mean age = 35.3 ± 5.9 years). Acne negatively impacted quality of life for all subjects. Black subjects reported significantly less negative impact on self-perception versus Asian/other (Black = 12.6 ± 9.9, Asian/other = 8.4 ± 8.6; p = .05). Social functioning was less negatively impacted in White and Black subjects versus Asian/other (White = 12.7 ± 7.5, Asian/other = 8.4 ± 7.8, p < .05; Black = 12.1 ± 9.2, Asian/other = 8.4 ± 7.8, p = .06). Over one third (total sample = 40.7%, Black = 31.3%, Hispanic = 36.4%, Asian/other = 50.0%, White = 46.7%) reported moderate/severe anxiety/depression symptoms. Acne also impacted ability to concentrate on work/school.Racial/ethnic differences were observed in acne-related quality of life and psychological symptoms in female adults; acne negatively impacted self-perceptions and social/emotional functioning.

Objective: Data on associations between facial aging and smoking or alcohol consumption are generally derived from small studies, and therefore, vary. The aim of this large multinational study was to determine more accurately which clinical signs of skin- and volume-related facial aging are associated with tobacco and alcohol use in women. Design: This was a subanalysis of a global, cross-sectional, Internet-based survey of self-reported facial aging. Participants: Women aged 18 to 75 years old (n=3,267) from the United States, Australia, Canada, and the United Kingdom who described themselves as white, Asian, black, or Hispanic were included. Measurements: Using a mirror, participants determined their own aging severity on photonumeric rating scales for 11 facial characteristics. Linear regressions were used to assess associations between each feature's severity and smoking status (never vs. current and former smoker); smoking pack years (0 versus 1-10, 11-20, and >20 years); alcohol use (none vs. moderate and heavy); and alcoholic beverage type, after controlling for body mass index, country, age, and race. Results: Smoking was associated with an increased severity of forehead, crow's feet, and glabellar lines; under-eye puffiness; tear-trough hollowing; nasolabial folds; oral commissures; perioral lines; and reduced lip fullness (p≤0.025) but not midface volume loss or visible blood vessels. Heavy alcohol use (≥8 drinks/week) was associated with increased upper facial lines, under-eye puffiness, oral commissures, midface volume loss, and blood vessels (p≤0.042). Conclusion: Smoking and alcohol consumption significantly but differentially impact skin and volume-related facial aging.

To assess the reliability and validity of age-specific versions of the Fibrodysplasia Ossificans Progressiva Physical Function Questionnaire (FOP-PFQ), developed to measure the impact of FOP on physical function and activities of daily living. FOP-PFQ development included a literature review, two iterative phases of qualitative work involving individuals with FOP, and clinical expert review. The analysis used pooled FOP-PFQ data from an FOP natural history study (NCT02322255), a patient registry (NCT02745158), and phase II trials (NCT02190747; NCT02279095; NCT02979769). Item-level and factor analysis informed item retention and determined factor structure. Reliability was evaluated using Cronbach's alpha and intraclass correlation coefficients. Convergent validity was assessed by comparing scores with age, the Cumulative Analogue Joint Involvement Scale (CAJIS), the Patient-Reported Outcomes Measurement Information System Global Health Scale (PROMIS), and heterotopic ossification (HO) volume. Known-groups validity assessment used age, CAJIS, and HO volume. Factor analysis confirmed a two-factor solution: Mobility and Upper Extremity. Results reflected high internal consistency and were supportive of test-retest reliability; correlation coefficients >0.90 demonstrated FOP-PFQ scores were stable over a one- to three-week period. The majority of scores were moderately (r = 0.30–0.50) to highly (r ≥ 0.50) correlated with CAJIS and HO volume, supporting convergent validity. With the exception of some age-based and functional groups, FOP-PFQ scores were significantly worse in groups with more severe disease, demonstrating known-groups validity. The FOP-PFQ was demonstrated to be a reliable, valid measure that may be responsive to change in individuals with FOP, although some results were inconclusive for pediatric versions.

Chronic obstructive pulmonary disease (COPD) is a leading cause of morbidity and mortality worldwide. COPD is characterized by poor treatment adherence, and patient medication preferences may contribute to adherence.A discrete choice experiment with an internet panel drawn from the USA was used to evaluate preference and willingness to pay (WTP) of COPD patients for long-acting maintenance medications. Key attributes derived from earlier qualitative research (brief literature review and focus groups) with COPD patients on maintenance therapy included symptom relief, speed of feeling medication start to work, inhaler ease of use, rescue medication use, side effects, and monthly out-of-pocket co-pay. Patients were presented with hypothetical medications with different profiles and asked which they preferred. Utilities and marginal WTP in monthly co-pay dollars were estimated for all patients and by severity.Utilities for 515 participants were in the expected direction and highest for the most favorable attribute levels. Each attribute evaluated was important, and participants were willing to pay a premium to obtain each benefit. On average, WTP was as high as $US64 for complete symptom relief, $US59 for no side effects, $US32 to rarely use rescue medication, $US16 for a quick and easy to use inhaler, and $US13 for feeling medication work quickly (within 5 min; average WTP $US18/month for patients with severe/very severe COPD).As expected, efficacy and safety were most valued by patients; however, this study showed that other COPD medication attributes, such as rescue medication, ease of use, and feeling medication work quickly, are also important in patient preferences.

Objective. Our study evaluated the impaired health status of clinical trial patients with systemic lupus erythematosus (SLE) and explored the relationship between changes in fatigue and pain and their effect on overall health status. Methods. Pooled treatment and placebo data from a phase Ib clinical trial of adults with moderate/severe SLE were analyzed. Measures included patient-reported Medical Outcome Study Short Form-36 Survey, Version 2 (SF-36v2), Fatigue Severity Scale, and numeric rating scales (NRS) for pain and global health assessment and clinician-reported global assessment of disease activity (MDGA). Disease burden was compared to the US general population. Health status of responders and nonresponders on pain or fatigue were compared. Results. The sample included 161 patients with SLE, predominantly female (96%) and white (72%), with average age of 43 ± 11 years. Mean SF-36v2 component summary scores reflected overall problems with physical [physical component summary (PCS); 35.2 ± 9.7] and mental health (mental component summary; 40.9 ± 12.9). Patients with SLE had worse health status on all SF-36v2 subscales than the US general population and comparable age and sex norms (effect size −0.51 to −2.15). Pain and fatigue responders had greater improvements on SF-36v2 scores (bodily pain, physical functioning, social functioning, PCS), patient global health assessment NRS, and MDGA than nonresponders. There was moderate agreement in responder status, based on global assessments by patients and clinicians (68.1%), with some discrepancy between patients who were MDGA responders but patient assessment nonresponders (27.7%). Conclusion. Improvements in patient-reported pain or fatigue correlated with improvements in overall health. Patient assessments offer a unique perspective on treatment outcomes. Patient-reported outcomes add value in understanding clinical trial treatment benefits.

Background Migraine is a chronic neurologic disease that can be associated with significant migraine‐related impact, disability, and burden. Patient‐reported outcome measures (PRO) are included in clinical trials of migraine interventions to capture treatment effects from a patient perspective. Clinical and regulatory guidelines also encourage use of PROs in trials. The Migraine Functional Impact Questionnaire (MFIQ) is a novel PRO measure, assessing the impact of migraine on Physical Function (PF), Usual Activities (UA), Social Function (SF), and Emotional Function (EF), in the past 7 days. Scientific methods recommended to meet the requirements of the U.S. Food and Drug Administration were followed, to ensure that the MFIQ content included outcomes that were relevant to adults with migraine and were clinically relevant, specifically to evaluate preventive treatments for migraine. Objective The objective of this study was to conduct item analyses informing item reduction and scoring, and to evaluate the psychometric properties of the MFIQ. Methods In a prospective, observational study, adults with migraine completed the MFIQ as well as additional clinical and PRO instruments, including the Headache Impact Test (HIT‐6 TM ), Patient‐Reported Outcomes Measurement Information System Physical Function Short Form 10a (PROMIS‐PF), Migraine‐Specific Quality‐of‐Life Questionnaire (MSQ), and Patient Global Rating of Change (PGIC). Item‐level evaluation, item response theory (IRT), and factor analysis were used to select final MFIQ items, identify domains, and inform scoring. Psychometric properties of the MFIQ were evaluated to assess reliability (internal consistency and test–retest), validity (construct and known‐groups), and responsiveness. Results The study enrolled 569 adults with migraine. Subjects had an average age of 39.9 years (SD 12.0), 87.2% were female, and 80.8% were white. Five items were dropped from the draft version based on results of item‐level analyses reviewed in the context of previous qualitative research to produce the final 26‐item MFIQ (v.2). Four domain scores (PF, UA, SF, and EF) and a global item score for impact on UA were identified. Higher scores on a 0‐100 scale represent greater impact. All scores exhibited high internal consistency ( α ≥ 0.89) and moderate test–retest reliability among stable subjects (ICCs ≥ 0.47). Construct validity was demonstrated by significant correlations (all P &lt; .0001) between MFIQ domain scores, related PRO scores, and the frequency of migraine days and headache days. All domain scores differentiated between subgroups (“known groups”) defined based on established levels of clinical severity: number of monthly migraine and headache days, migraine interference levels and scores on other PRO instruments ( P &lt; .05). Improvements in MFIQ scores corresponded with clinical improvement (percent reduction in monthly migraine days), improvement in migraine interference with daily activities, and related improvements in PRO scores ( P &lt; .05), demonstrating that the MFIQ was responsive to changes in migraine impact. Conclusions The MFIQ is a reliable and valid measure that can be used to collect data about migraine impact. The MFIQ is being used to evaluate outcomes of migraine interventions in clinical trials and observational studies. It could potentially also be used in clinical practice both for initial and ongoing assessments for monitoring outcomes and to enhance communication between patients and healthcare professionals for the management of migraine.

The Sleep Diary Questionnaire (SDQ), a modified version of the Consensus Sleep Diary, is a 17-item sleep diary for assessing subjective total sleep time (sTST: total time spent asleep at night) and other sleep parameters in insomnia trials. sTST is a key parameter of efficacy in insomnia trials; however, the magnitude of improvement in this parameter that people with insomnia disorder consider clinically meaningful is unclear.The aim of this study was to estimate meaningful within-patient change for sTST using clinical trial data.Data were from an open-label trial of zolpidem and pooled data from a phase III placebo-controlled trial of daridorexant. In both trials, adults with moderate to severe insomnia completed the SDQ daily. Meaningful change in sTST was estimated in an anchor-based analysis using outcome measures that were correlated with change in weekly average sTST (Spearman correlation coefficient ≥ 0.30): the Insomnia Severity Index, patient global assessments and impressions of severity and change in daytime and night-time symptoms (PGA-S, PGI-S, PGI-C), and clinician global impressions of severity and change in patients' daytime symptoms (CGI-S, CGI-C). Meaningful within-patient change estimates were 'triangulated' to identify a value where they converged.In the open-label trial (N = 114), subjects with a 1-point or 1-step improvement on the anchors had mean increases in sTST of 60.1-83.2 min at day 8 and 55.5-68.2 min at day 15. For subjects with a 2-point or 2-step improvement on the anchors, mean increases in sTST were 79.6-81.4 min at day 8 and 80.1-93.5 min at day 15. In the phase III trial (N = 930), weekly average increases in sTST for subjects with a 1-point or 1-step improvement on the anchors were 39.3-46.7 min at month 1 and 47.3-58.3 min at month 3. For subjects with a 2-point or 2-step improvement on the anchors, mean increases in sTST were 60.7-76.2 min at month 1 and 70.1-87.7 min at month 3. Triangulation of these values supported a meaningful within-patient change threshold starting at 55 min.Increasing sTST is an important treatment outcome for people with insomnia. An increase in sleep time of approximately 55 min is meaningful to patients.NCT03056053 (17 February 2017) and NCT03545191 (4 June 2018).

The Insomnia Daytime Symptoms and Impacts Questionnaire (IDSIQ) is a new validated 14-item patient-reported outcome (PRO) instrument for evaluating daytime functioning in people with insomnia. It comprises three domains: Alert/Cognition, Mood, and Sleepiness.The aim of this analysis was to estimate the minimum within-patient change for IDSIQ scores that an adult patient with insomnia would consider meaningful.Data were from a randomized, double-blind, placebo-controlled, phase III clinical trial of daridorexant in adults with insomnia. Subjects completed the IDSIQ daily in the evening, with a recall period of 'today', throughout the 3-month double-blind treatment period. Scores were calculated as a weekly average. Each IDSIQ item was scored on an 11-point numeric rating scale ranging from 0 (not at all/none at all) to 10 (very/a lot), with a higher score indicating a greater severity or impact. PRO measures with correlation coefficients ≥0.30 were included in a subsequent anchor-based analysis. For the IDSIQ total score and each IDSIQ domain, meaningful within-patient change was estimated as the minimum score change patients would consider meaningful in an anchor-based analysis using data from PRO instruments capturing daytime and night-time insomnia symptoms (the Insomnia Severity Index [four items, each scored 0-4, with a higher score indicating greater symptom severity; assessed at screening, baseline, month 1 and month 3], Patient Global Assessment of Disease Severity [6-point scale from 'none' to 'very severe'; assessed weekly], Patient Global Impression of Severity [4-point scale from 'none' to 'severe'; assessed weekly], and Patient Global Impression of Change [7-point scale from 'very much better' to 'very much worse'; assessed weekly for night-time and daytime symptoms separately]). A supplemental distribution-based analysis was also conducted to support the anchor-based analysis.The analysis included 930 subjects aged 18-88 years. Spearman correlation coefficients for the relationships between score changes/ratings for anchors and the IDSIQ (0.36-0.44 at month 1, 0.45-0.57 at month 3) were all above the prespecified threshold of 0.30. Mean IDSIQ score changes at months 1 and 3 based on the different anchors supported meaningful within-patient change estimates starting at 17 points for the IDSIQ total score, 9 points for the Alert/Cognition domain, and 4 points for the Mood and Sleepiness domains.This analysis demonstrates the meaningful within-patient change for the IDSIQ total score and domain scores, that the instrument is sensitive to changes in the patient experience of insomnia, and that it can be used in clinical trials to evaluate changes in daytime functioning.NCT03545191 (4 June 2018).

Background: Limited data are available on acne treatment patterns, expectations, and satisfaction in the adult female subpopulation, particularly among different racial and ethnic groups. Objective: Describe acne treatment patterns and expectations in adult females of different racial/ethnic groups and analyze and explore their potential effects on medication compliance and treatment satisfaction. Methods: A cross-sectional, Web-based survey was administered to US females (25–45 years) with facial acne (≥25 visible lesions). Data collected included sociodemographics, self-reported clinical characteristics, acne treatment use, and treatment expectations and satisfaction. Results: Three hundred twelve subjects completed the survey (mean age, 35.3±5.9 years), comprising black (30.8%), Hispanic (17.6%), Asian/other (17.3%), and white (34.3%). More than half of the subjects in each racial group recently used an acne treatment or procedure (black, 63.5%; Hispanic, 54.5%; Asian/other, 66.7%; white, 66.4%). Treatment use was predominantly over-the-counter (OTC) (47.4%) versus prescription medications (16.6%). OTC use was highest in white subjects (black, 42.7%; Hispanic, 34.5%; Asian/other, 44.4%; white, 59.8%; P <0.05). The most frequently used OTC treatments in all racial/ethnic groups were salicylic acid (SA) (34.3%) and benzoyl peroxide (BP) (32.1%). Overall, compliance with acne medications was highest in white versus black (57.0±32.4 vs 42.7±33.5 days, P >0.05), Hispanic (57.0±32.4 vs 43.2±32.9 days, P >0.05), and Asian/other (57.0±32.4 vs 46.9±37.2 days, P >0.05) subjects. Most subjects expected OTC (73.7%) and prescription (74.7%) treatments to work quickly. Fewer than half of the subjects were satisfied with OTC treatment (BP, 47.0%; SA, 43.0%), often due to skin dryness (BP, 26.3%; SA, 44.3%) and flakiness (BP, 12.3%; SA, 31.1%). No statistically significant differences were observed among racial/ethnic groups in their level of satisfaction with OTC or prescription acne treatments. Conclusion: Racial/ethnic differences were observed in acne treatment patterns in adult females, while treatment expectations were similar. Results indicate that treatment patterns and expectations may impact treatment satisfaction and medication compliance. Keywords: acne, ethnicity, patient satisfaction, race, women

Introduction. A five-item Self-Assessment of Treatment (SAT) was developed to assess improvement and satisfaction with treatment associated with the application of a novel high concentration 8% capsaicin topical patch in clinical trials in patients with postherpetic neuralgia (PHN). This study evaluated the item performance and psychometric properties of the SAT. Methods. The SAT, Brief Pain Inventory, SF-36v2, Short-Form McGill Pain Questionnaire, and Patient and Clinician Global Impression of Change (PGIC; CGIC) scores were measured in two 12-week Phase 3 clinical trials. Factor analysis assessed the underlying factor structure, followed by examination of the reliability and validity of the multi-item domain. Results. Pooled data from 698 patients completing SAT after 12 weeks of treatment were analyzed. A one-factor model combining three of the five items emerged as the optimal solution. Internal consistency reliability of this treatment efficacy factor was high (Cronbach's alpha = 0.89). Construct validity was demonstrated by moderate to high correlations with change in other study endpoints. SAT mean scores consistently discriminated between patient change groups defined by PGIC and CGIC. Conclusions. The measurement properties of the three-item version of SAT are valid and reliable for assessment of treatment with a high concentration capsaicin patch among patients with PHN.

Aims: Health utilities summarize a patient’s overall health status. This study estimated utilities based on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (QLQ-C30), a widely used measure of health-related quality-of-life (HRQoL) in oncology, using published mapping algorithms.Materials and methods: Data were from the Anaplastic Lymphoma Kinase (ALK) in Lung Cancer Trial of brigatinib (ALTA; NCT02094573), an open-label, international, phase 2 study. ALTA evaluated the efficacy and safety of two randomized dosing regimens of brigatinib in patients with locally advanced or metastatic ALK + non-small cell lung cancer (NSCLC) that had progressed on prior therapy with crizotinib. QLQ-C30 scores were mapped to European Quality-of-Life-5 Dimensions (EQ-5D) utility scores using two published algorithms (Khan et al. for EQ-5D-5L; Longworth et al. for EQ-5D-3L). The impact of brigatinib treatment on health utilities over time was assessed.Results: The analysis included 208 subjects. Mean baseline utility scores for both algorithms ranged between 0.60 − 0.71 and increased to 0.78 by cycle 5. Utility improvements were sustained during most of the treatment, before disease progression. Minor variations were observed between utility scores; Khan et al. estimates were approximately 0.01 or 0.02 points lower than Longworth et al. estimates.Limitations: Algorithms considered were limited to those available in the published literature at the time of the study. This utility analysis was exploratory, and the ALTA trial did not include an internal control group (i.e. standard of care) and was not powered to detect differences in QoL/utility outcomes between treatment arms.Conclusions: Converting QLQ-C30 scores into utilities in trials using established mapping algorithms can improve evaluation of medicines from the patient perspective. Both algorithms suggested that brigatinib improved health utility in crizotinib-refractory ALK + NSCLC patients, and improvements were maintained during most of the treatment.Clinicaltrials.gov identifier: NCT02094573

This article describes data from 4,111 males and 4,085 females participating in 10 HIV/AIDS service demonstration projects. The sample was diverse in age, gender, ethnicity, HIV status, and risk for HIV transmission. Logistic regression was used to determine the attributes that best predict substance abuse. Males who were younger; HIV positive; homeless; involved in the criminal justice system; had a sexually transmitted disease (STD); engaged in survival sex; and participated in risky sex with men, women, and drug injectors were most likely to have a substance abuse history. For females, the same predictors were significant, with the exception of having an STD. Odds ratios as high as 6 to 1 were associated with the predictors. Information about sexual and other risk factors also was highly predictive of substance abuse issues among youth.

To describe the psychosocial impact of facial aging in adult females and males. Participants aged 18-75 were recruited from a pool of internet panelists in Australia, Canada, United Kingdom, and United States. Psychosocial impact of facial aging was evaluated using the Self-Perception of Age (SPA) measure, the Facial Line Satisfaction Questionnaire (FLSQ), and the Facial Line Outcomes (FLO-11) Questionnaire. Quotas were used to ensure representation of all age, gender, and race categories of interest. Descriptive statistics were reported. A total of 4,086 respondents completed the survey. Mean age was 47.7 years (SD=16.1) and 80.0% of respondents were female. Nearly all participants (95.6%) perceived themselves as looking their current age or younger (females: mean 9.7 [SD=6.8]; males: mean 9.6 [SD=6.4] years younger). Participants aged 40-49 perceived themselves as looking slightly younger than any other cohort. Black participants perceived themselves as looking greater years younger than White participants (in all age cohorts) and Asian and Hispanic participants (in select age cohorts). Over half of participants (53.8%) were ‘mostly/very satisfied’ with the appearance of their face; satisfaction was higher in younger cohorts (18-49 years) and decreased slightly in older cohorts (50-75 years). White and Black participants reported the greatest negative self-perception and most favorable self-perception, respectively, based on the FLO-11. Females reported greater negative impact of facial lines than males, with the greatest negative impact among females aged 40-59; among males, White participants and participants aged 40-49 reported the greatest negative impact. This is the first multinational study to describe the psychosocial impact associated with facial lines. Participants generally reported a high overall positive outlook on their facial appearance, with Black participants reporting the highest levels of satisfaction. Data from this study can be used as a basis for future research on the impact of facial aging.

The Hypoglycaemia Perspectives Questionnaire (HPQ) is a patient-reported outcomes (PRO) instrument assessing diabetic patients’ experience and perceptions of hypoglycaemia. The aim of this study is to evaluate the factor structure and psychometric characteristics of the HPQ in type 2 diabetic patients (T2DM). HPQ was administered to adults with T2DM in a clinical sample from Cyprus and a community sample in the US from 2011 US National Health and Wellness Survey. Demographic and clinical data were collected. Participants completed the Audit of Diabetes Dependent Quality of Life (ADDQoL-19), treatment satisfaction items, and EuroQol-5 Dimensions (EQ-5D) (Cyprus only). HPQ items assess hypoglycaemia attitudes and behaviours on an 11-point numeric rating scale (NRS). Item performance and factor structure were examined and measurement properties (reliability, construct validity, known-groups validity) evaluated. Cyprus (n=500) and US (n=1,257) T2DM samples were of similar age (Cyprus 61.0±10 years, US 59.9±11 years). Cyprus had more males (67.4% vs. 54.2%) and fewer obese subjects (BMI ≥30) 45.6% vs. 67.8%) than the US. More US subjects reported hypoglycaemia events in the past seven days (27.7% vs. 16.6% with ≥1 event). Prescription oral diabetes medications were used by 90.3% of Cyprus participants and 83.6% of US participants, and insulin by 32.9% and 25.3%, respectively. Analyses supported three HPQ domains: Symptom Concern (six items), Compensatory Behavior (five items), and Worry (five items). Internal consistency was high for all three domains (all ≥0.75), supporting reliability. Convergent validity was supported by moderate correlations between HPQ domain scores and ADDQoL-19 total score. Patients with recent hypoglycaemia events had significantly higher HPQ scores supporting known-group validity. HPQ is a valid and reliable measure capturing the experience and impact of hypoglycaemia and can be useful in clinical trial and community-based settings.

The purpose of this study was to examine changes in the functional impact of migraine following treatment with erenumab, as measured by the Migraine Functional Impact Questionnaire (MFIQ).The MFIQ, a novel patient-reported outcome (PRO) measuring the impact of migraine on four domains (physical function, social function, and emotional function [PF, SF, and EF]; usual activities [UAs]) and a single item assessing overall impact on UA, was included in phase III trials evaluating erenumab 70 and 140 mg monthly for migraine prevention among people with episodic migraine (EM).In the ARISE study, 577 patients with EM were randomized to erenumab 70 mg or placebo. In the STRIVE study, 955 patients with EM were randomized to erenumab, 70 mg or 140 mg or placebo. Pairwise comparisons of least-squares mean (LSM) change from baseline in MFIQ scores (with associated 95% confidence interval [CI]) were assessed for each active treatment versus placebo.In ARISE, greater reductions from baseline to month 3 were observed for 70 mg versus placebo for PF (LSM [95% CI]: -3.2 [-6.4 to -0.1]; p = 0.046) and EF (-4.0 [-7.3 to -0.7]; p = 0.019) domain scores. In STRIVE, between-group differences also reflected reductions from baseline to the average of months 4-6 that favored erenumab on all four MFIQ domain scores. Reductions in impact for 70 mg compared to placebo were -4.3 (95% CI: -6.8 to -1.7; p < 0.001) for PF, -4.0 (-6.3 to -1.7; p < 0.001) for UA, -3.7 (-6.1 to -1.2; p = 0.003) for SF, and -5.3 (-7.9 to -2.6; p < 0.001) for EF domain scores. Improvements were also observed for 140 mg versus placebo with between-group differences of -5.7 (95% CI: -8.2 to -3.2; p < 0.001) in PF, -5.1 (-7.5 to -2.8; p < 0.001) in UA, -5.0 (-7.4 to -2.6; p < 0.001) in SF, and -7.2 (-9.9 to -4.5; p < 0.001) in EF domain scores. There were also greater improvements in the overall impact on UA score for 70 mg (LSM [95% CI]: -4.3 [-7.0 to -1.7]; p = 0.001) and 140 mg (-5.3 [-8.5 to -3.2]; p < 0.001) versus placebo.The MFIQ measures the frequency of impacts and level of difficulty on multiple functional domains that provide a more complete picture of the effects of migraine. MFIQ scores showed that in comparison with placebo, patients treated with erenumab had greater reductions in the functional impact of migraine, providing insight into treatment benefits that extend beyond improvements in clinical status and health-related quality of life previously reported based on clinical end points and other PROs.

Abstract Introduction The Insomnia Daytime Symptoms and Impacts Questionnaire (IDSIQ) is a new validated patientreported outcome (PRO) instrument evaluating daytime functioning in people with insomnia. It comprises 14 items grouped into 3 domains: Alert/Cognition, Mood, and Sleepiness. To further explore the ability of the IDSIQ to capture clinically meaningful changes in daytime functioning resulting from treatment, we estimated withinsubject changes in IDSIQ scores using phase 3 trial data. Methods A randomized double-blind placebocontrolled trial of daridorexant in adults with insomnia (NCT03545191), in which subjects completed the IDSIQ daily during treatment, provided data for blinded analyses. Spearman correlations were calculated for changes in IDSIQ scores and potential anchors: Insomnia Severity Index, Patient Global Assessment of Disease Severity, Patient Global Impression of Severity, and Patient Global Impression of Change, applying a prespecified threshold of 0.30 (moderate association). Anchor-based analyses of weekly average IDSIQ total and domain scores were used to estimate responder definitions (RDs). The various RD estimates were triangulated to identify values where they converged. Distribution-based and receiver operating characteristic analyses calculated standard error of measurement (SEM), 0.5 standard deviation (SD), and Youden’s index as supportive evidence for anchor-based RD estimates. Results The analysis included 930 subjects (18-88 years). Score change correlations for the potential anchors and IDSIQ at month 1 (0.36–0.44) and month 3 (0.45–0.57) were all &amp;gt;0.30. Triangulation of mean IDSIQ score changes in subjects with clinically relevant improvement on the different anchors supported RD thresholds for clinically meaningful change of 17 points for the IDSIQ total score, 9 points for the Alert/Cognition domain, 4 points for the Mood domain, and 4 points for the Sleepiness domain. SEM and 0.5 SD values were within the ranges of anchor-based IDSIQ score changes, and Youden’s index was maximized or near-maximized when the RD estimates were used as thresholds for identifying responders based on the anchors. Conclusion The IDSIQ is sensitive to changes in patients who experience daytime impacts of insomnia and can be used to assess treatment efficacy on daytime functioning in patients with insomnia. Support (If Any) This work was funded by Idorsia Pharmaceuticals Ltd.

April 24, 2017April 18, 2017Free AccessEvaluating the Measurement Properties of a New Instrument-the Migraine Physical Function Impact Diary (MPFID) (P2.156)Ariane K. Kawata, Ray Hsieh, Asha Hareendran, Randall Bender, Shannon Shaffer, Sandhya Sapra, Pooja Desai, Katherine Widnell, Martha Bayliss, Dawn C. Buse, and Dennis RevickiAuthors Info & AffiliationsApril 18, 2017 issue88 (16_supplement)https://doi.org/10.1212/WNL.88.16_supplement.P2.156 Letters to the Editor

Issue InformationFree Access Issue Information First published: 17 September 2019 https://doi.org/10.1111/head.13372AboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onFacebookTwitterLinkedInRedditWechat Volume59, Issue8September 2019 RelatedInformation

Abstract The Web site hosted by The Measurement Group uses the Internet as a tool to disseminate information about innovative HIV/AIDS prevention and treatment models funded by the Health Resources and Service Administration (HRSA) Special Projects of National Significance (SPNS) Program and highlight their effectiveness as models for HIV/AIDS care. The site features project descriptions, reports, presentations, evaluation designs/tools, resources, and conference paper abstracts. Users from major universities, governmental agencies, large computer networks, and foreign countries have accessed the site. The Measurement Group Web site has attracted the attention of many users throughout the world in its goal of disseminating new information about innovative models of HIV services to a wide audience.

To explore the dimensionality of the PF construct when HAQ-DI and PF10 items are combined into a single instrument. Item Response Theory (IRT) was used to analyze data from a phase 3, randomized, placebo-controlled study in patients with RA. Dimensionality of the combined instrument was explored by factor analyses (exploratory and confirmatory). Two approaches were taken to address the multi-factor/dimensionality issue identified through factor analysis. The first approach was to fit a sequence of unidimensional Rasch and generalized partial credit (GPC) models to each factor. Model comparisons and diagnoses were performed to assess the adequacy of model fitting and to detect any violation of the underlying assumptions. The second approach was to fit a multi-dimensional IRT model to these items. The factor analysis suggested there were three underlying factors in the combined PF10 and HAQ-DI. These three factors can be best characterized as hygiene (including 4 items from HAQ-DI and 1 from PF10), lower body activity (including 8 items from HAQ-DI and 6 from PF10) and upper body activity (including 7 items from HAQ-DI and 3 from PF10). Overall, the GPC model significantly outperformed the Rasch model across all factors (all p-value<0.0001), implying divergent discriminability among items within each factor. The multi-dimensional IRT improved the model fit but substantially increased the computation effort and sacrificed model interpretability. The pooled HAQ-DI and PF10 items represent multiple domains of physical functioning. To explain the combination of these items, a multi-dimensional IRT model decreased model interpretability and increased computation intensity compared to unidimensional models.

<h3>Background</h3> The SF-36® 10 items Physical Functioning domain (PF10) and the Health Assessment Questionnaire (HAQ) are widely used measurements to evaluate rheumatoid arthritis (RA) treatments. However, there are competing theories about the dimensionality in the overall latent Physical Functioning (PF) construct when combining PF10 and HAQ items. <h3>Objectives</h3> To explore the dimensionality of the PF construct when PF10 and HAQ items are combined into a single instrument and then evaluate the psychometric properties of the combined instrument. <h3>Methods</h3> Item Response Theory (IRT)[1] was used to analyze data from a phase 3, randomized, placebo-controlled study in patients with RA. Dimensionality of the combined instrument was explored by factor analyses (exploratory and confirmatory). A sequence of unidimensional Rasch [1] and generalized partial credit (GPC)[1,2] models were fitted to address the multi-factor/dimensionality issue identified through factor analyses. Model comparisons and diagnoses were performed to assess the adequacy of model fitting and to detect any violation of the underlying assumptions. In addition to building IRT models for individual factor, another IRT model that includes all 30 items was built to derive a total score. Pearson correlation was used to assess the validity of the IRT-based health status scores, whereas analysis of covariance was used to assess sensitivity of response change (defined by the American College of Rheumatology [ACR] 20% improvement criteria [3]). <h3>Results</h3> The factor analysis suggested there were three underlying factors in the combined PF10 and HAQ. These three factors can be best characterized as hygiene (including 1 item from PF10 and 4 from HAQ), lower body activity (including 6 items from PF10 and 8 from HAQ), and upper body activity (including 3 items from PF10 and 7 from HAQ). In all models considered, the GPC model significantly outperformed the Rasch model counterpart (all <i>p</i>-value&lt;0.0001), implying divergent discriminability among items. In addition to the factor analysis-based IRT scales, IRT-based scales derived from all the 30 combined items also show strong convergent validity (all <i>p</i>-value&lt;0.0001) and strong sensitivity (all <i>p</i>-values&lt;0.0001) when comparing across ACR groups. <h3>Conclusions</h3> The pooled PF10 and HAQ represent multiple domains of physical functioning. The superior performance of the GPC model over Rasch model suggested divergent discriminability among items. Strong convergent validity and sensitivity were demonstrated for all IRT-based scales. <h3>References</h3> Edelen, MO and Reeve, BB (2007) Applying item response theory (IRT) modeling to questionnaire development, evaluation, and refinement. Qual. Life Res. 16 Suppl 1: 5-18. Li, Y and Baser, R (2012) Using R and WinBUGS to fit a generalized partial credit model for developing and evaluating patient-reported outcomes assessments. Statist. Med. 31: 2010-2026 Felson, D and American College of Rheumatology Committee to Reevaluate Improvement Criteria (2007) A proposed revision to the ACR20: the hybrid measure of American College of Rheumatology response. Arthritis Rheum 57: 193-202 <h3>Disclosure of Interest</h3> : None declared <h3>DOI</h3> 10.1136/annrheumdis-2014-eular.5254

The Hypoglycemia Perspectives Questionnaire (HPQ) was developed with clinician and patient input to assess symptoms, behaviors, and impact of hypoglycemia on diabetic patients. The HPQ was administered to adult patients with type 2 diabetes mellitus (T2DM) on antidiabetic treatment as part of a cross-sectional, epidemiological study evaluating hypoglycemia and health-related quality of life (HRQoL) in Cyprus. Demographic and clinical data were collected. Patients also completed the Audit of Diabetes Dependent Quality of Life (ADDQoL-19), treatment satisfaction questionnaire, and EuroQol-5 Dimensions (EQ-5D). The original HPQ consisted of 45 items rating current status or behavior related to hypoglycemia on an 11-point numeric rating scale (NRS) and 7 additional descriptive hypoglycemia event frequency items. Analyses included examination of HPQ item performance, item reduction, and factor structure. Measurement properties (reliability, construct validity, known-groups validity) of the final HPQ were evaluated. A total of 500 T2DM patients completed the HPQ with a mean age of 61±10 years; 32.6% women. Based on item evaluation, the original HPQ item pool was reduced to 22 items. Exploratory and confirmatory factor analysis identified 21 items contributing to 3 hypoglycemia domains (Symptoms [8 items], Compensatory Behaviors [7 items], Worry [6 items]) and a single-item of global symptom awareness. HPQ domains had high internal consistency reliability (Cronbach’s alpha=0.78-0.92). Construct validity was demonstrated by significant correlations between HPQ scores with HRQoL, treatment satisfaction, and health status. HPQ also demonstrated ability to discriminate between known groups. Compensatory behaviors and symptom awareness were higher for patients with a recent low blood sugar event (p<0.001) and high symptom awareness corresponded to less concern about experiencing symptoms of low blood sugar and worry (p<0.05). These results provide preliminary evidence that HPQ is reliable and valid for assessing the experience and impact of hypoglycemia on T2DM patients.

To describe survey methodology used to evaluate the burden of illness and racial differences in adult female acne (AFA). A targeted, web-based survey was used to recruit a racially diverse sample of US adult females (25-45 years) with facial acne vulgaris from an existing pool of internet panelists. Subjects who self-reported ≥25 visible facial pimples at screening were eligible. Recruitment was stratified by age (50% 25-35; 50% 36-45 years) and race (50% White; 50% Non-White [25% Black/African American; 25% Hispanic/Asian/Other]). Survey outcomes included: sociodemographic and clinical characteristics; resource utilization; treatment satisfaction; quality of life; perceptions; coping behaviors; work environment/productivity; anxiety/depression symptoms; and skin-specific treatment preferences (non-White females only). Validation rules were pre-programmed into the survey to improve data quality. Descriptive statistics summarize results in the total sample; racial differences (White vs. non-White) were evaluated using descriptive statistics and t-test/chi-square analyses. The survey was fielded online from Oct-Nov 2011. A total of 7245 panelists received survey invitations via email. Of 4112 survey respondents, 208 (5.1%) were eligible and completed the survey. Mean age of sample was 35±6 years; 48.6% non-White [Black (n=51); Hispanic (n=23); Asian (n=16); Other (n=11)]. Most females (80.3%) reported 25-49 visible pimples, followed by 50-75 (13.5%) and >75 (6.3%). Median survey completion time was 24.5 minutes. Web-based survey methodology with an existing pool of panelists permitted focused recruitment of respondents to reach targeted sample sizes in each age and race/ethnicity stratum. This method enabled recruitment of a targeted subset of patients, including non-White females and those with greater acne severity (≥50 visible pimples). Web-based surveys are an effective method for collecting patient-reported data for stratified patient cohorts, while minimizing both time and cost. This method was well suited for studying the burden of AFA in a real-world cohort.

PURPOSE: The goal of this qualitative study was to increase our understanding of the importance of various attributes of the medications used in the maintenance treatment of chronic obstructive pulmonary disease (COPD).

Abstract Introduction The Sleep Diary Questionnaire (SDQ) is a new content-valid 17-item sleep diary adapted from the Consensus Sleep Diary. It assesses key sleep parameters including total time asleep the previous night or “subjective total sleep time” (sTST). People with insomnia value increasing sTST as a key treatment outcome. We estimated meaningful withinpatient change for sTST from two clinical trials in adults with insomnia. Methods Data from a 2-week, phase 2 open-label trial of zolpidem (NCT03056053) and blinded data from a 3-month, phase 3 randomized placebo-controlled trial of daridorexant (NCT03545191) were used. In both trials, subjects completed the SDQ daily before and during treatment. Changes in weekly average sTST were calculated using anchor-based analyses that included patient and clinicianreported outcome measures whose correlations with change in weekly average sTST were at least moderate (Spearman correlation coefficient ≥|0.3|). The outcome measures were Insomnia Severity Index, Patient Global Assessment of Disease Severity, Patient Global Impression of Severity, Patient Global Impression of Change, Clinician Global Impression of Severity, and Clinician Global Impression of Change. Distribution-based analyses calculated standard error of measurement (SEM) as supportive evidence. Change estimates from the anchor and distributionbased analyses were “triangulated” to identify a value where they converged. Results In the phase 2 trial (N=114), mean increases in sTST from baseline in subjects with meaningful improvements on the anchors were 60.1–83.2 min at day 8 and 55.5–93.5 min at day 15. SEM was 51.1 min at day 8 and 55.5 min at day 15. In the phase 3 trial (N=930, pooled across treatment arms), mean increases in sTST were 36.5–76.2 min at month 1 and 47.3–87.7 min at month 3. SEM was 43.2 min at month 1 and 53.3 min at month 3. Triangulation of these results supported a meaningful change threshold of 55 min. Conclusion Our findings support the importance of using sTST to assess insomnia from the patient’s perspective and provide useful information that an increase in sleep time of almost 1 hour is meaningful to patients. Support (If Any) This work was funded by Idorsia Pharmaceuticals Ltd.