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Background The Severity of Alopecia Tool (SALT) is a clinician‐reported outcome measure of scalp hair loss in alopecia areata (AA). Objectives To characterise the magnitudes of change in SALT scores corresponding to meaningful treatment benefits from the patient's perspective. Methods Anchor‐based methods for the estimation of meaningful within‐patient change thresholds were applied to pooled data from a randomised, double‐blind trial of ritlecitinib. Anchors included a patient‐reported measure of change in AA severity, the Patient Global Impression of Change (PGI‐C) and three items comprising the Patient Satisfaction with Hair Growth (P‐Sat) questionnaire. After reviewing Pearson correlations between change‐from‐baseline SALT scores and each anchor to confirm adequate association, potential thresholds were computed as mean change‐from‐baseline SALT scores among patients who reported moderate improvement on the PGI‐C and/or moderate satisfaction on each of three P‐Sat items at week 24. Results Six hundred and fifty participants (86% adults, 14% adolescents) had mean (standard deviation) SALT scores of 90.6 (14.3) at baseline, suggesting a sample with primarily severe AA. Correlations between SALT change‐from‐baseline scores and the patient‐reported items supported their use as anchors. Estimates based on patients reporting moderate improvement in AA (n = 102) on the PGI‐C and those reporting moderate satisfaction on the P‐Sat item related to the amount of hair growth at week 24 (n = 122) were −42.2 (26.1) and −43.1 (26.8), respectively. Supportive estimates based on the remaining P‐Sat items were similar in magnitude. Conclusions Among patients with severe AA, SALT change‐from‐baseline scores of 42 or 43 represent meaningful improvements. While the achievement of low SALT scores of ≤10–≤20 have been used to characterise efficacy in clinical trials, the amount of change required to meet this endpoint far exceeds the estimates in this study. The treatment goals of individual patients must be considered when evaluating benefit in both clinical trials and clinical practice. Applying anchor‐based methods, we used pooled data from a randomised, double‐blind trial of ritlecitinib to estimate meaningful within‐patient change thresholds for the Severity of Alopecia Tool (SALT), a clinician‐reported measure of scalp hair loss in alopecia areata (AA). Based on patients reporting moderate improvement in AA on the Patient Global Impression of Change and reporting moderate satisfaction on the Patient Satisfaction with Hair Growth at Week 24, estimates of meaningful SALT change from baseline scores were 42 and 43, respectively.

Background Physicians consider ease of use, satisfaction, and preferences when prescribing an inhaler device. These factors may impact appropriate usage and compliance. Methods The objectives were to quantify the relative importance of inhaler attributes in patients currently using Combivent Respimat by eliciting preferences for performance and convenience attributes assessed by items in the Patient Satisfaction and Preference Questionnaire (PASAPQ). Using a pharmacy database, 19,964 adults in the United States who filled ≥2 Combivent Respimat prescriptions were identified. Of those, 8150 patients were randomly selected to receive invitation letters. The online cross-sectional survey included the PASAPQ and best-worst scaling (BWS) questions. The PASAPQ measures satisfaction with medication attributes across two domains: performance and convenience. BWS questions asked participants to select the most and least important device attributes. A descriptive statistics analysis of the PASAPQ and a random-parameters logit model of BWS responses were conducted. Results The survey was completed by 503 participants. Most were female (57.3%), white (88.5%), and 51–70 years old (67.6%). Approximately 47% reported a chronic obstructive pulmonary disease diagnosis, 21.9% asthma, 8.2% other lung disease, and 23.1% more than one lung disease. PASAPQ scores indicated that the majority were satisfied or very satisfied; up to 20% reported being dissatisfied with Combivent Respimat. The three most important inhaler attributes were Feeling that your medicine gets into your lungs , Inhaler works reliably , and Inhaler makes inhaling your medicine easy. The most important attributes corresponded to six of seven items in the PASAPQ performance domain. Conclusions Most participants reported satisfaction with Combivent Respimat. Performance attributes were more important than convenience attributes.

Background The Respiratory Infection Intensity and Impact Questionnaire (RiiQ™) is a patient-reported outcome measure designed to assess symptoms and impacts of respiratory syncytial virus (RSV) infection. This study evaluated the construct validity, reliability, and responsiveness of the RiiQ™ Respiratory and Systemic Symptoms Scale scores. Methods Prospective data were analyzed from a total of 1795 participants, including from non-hospitalized patients with acute respiratory infection (ARI) and no coinfections enrolled in a Phase 2b RSV vaccine study (RSV-positive: n = 60; RSV-negative: n = 1615), and two observational studies of patients hospitalized with RSV (n = 20; n = 100). Descriptive statistics, confirmatory factor analysis (CFA), test–retest intraclass correlation coefficients (ICCs), construct validity correlations (between a clinician-assessed clinical questionnaire and the RiiQ™ symptoms scale), known-groups validity, and responsiveness (correlations of change scores) were evaluated. Results Mean patient age ranged from 66.5 to 71.5 years and the majority of patients were female. Initial assessments in the vaccine trial (ARI Day 1) were suggestive of less severe illness than in the observational studies with hospitalized patients. CFA loadings (> 0.40) supported summary scores. ICCs exceeding the recommended threshold of 0.70 supported test–retest reliability for Respiratory and Systemic Symptoms, except in the small observational study. At the scale level, correlations were moderate to strong (| r | ≥ 0.3) and positive between the Respiratory Symptoms Scale and the related clinical questionnaire scores, reflecting measurement of similar symptoms in support of convergent validity. Correlations with change in Patient Global Impression of Severity > 0.30 supported responsiveness. Conclusions Psychometric tests applied to the RiiQ™ Symptoms scales provide evidence of its reliability, construct validity, discriminating ability, and responsiveness for use in clinical studies to assess the onset and severity of RSV symptoms.

Chronic obstructive pulmonary disease (COPD) is a progressive disease that impairs both objectively measured lung function and patient-reported health status. In a randomized clinical trial of patients with moderate to severe COPD, we compared changes in health status after adding arformoterol tartrate or placebo to patients’ treatment regimens. In this multicenter, double-blind trial, patients were randomized to receive nebulized arformoterol 15 µg BID (n = 420) or matched placebo (n = 421). Treatment with other COPD medications was permitted, except for long-acting β2-agonists. Inclusion criteria were a forced expiratory volume in 1 second (FEV1) ≤65% of predicted, FEV1 >0.50 L, age ≥40 years, smoking history ≥15 pack-years, and a baseline breathlessness severity grade ≥2. The Clinical COPD Questionnaire (CCQ) was used to measure health status at randomization and at months 3, 6, and 12. CCQ scores range from 0 to 6, with higher scores indicating worse health status, and a decrease from baseline in total score by 0.4 point is considered clinically significant. Outcomes were analyzed by using mixed models for repeated measures. At baseline, patients’ mean age was 63.8 years; 42.9% of patients were female, and 51.4% were current smokers. The mean baseline CCQ total scores were 2.88 and 2.91 for the arformoterol and placebo groups, respectively. A total of 841 patients were randomized to receive either arformoterol (n = 420) or placebo (n = 421); among them, 211 (50.1%) who received placebo and 255 (60.7%) who received arformoterol completed the trial. Arformoterol-treated patients had greater mean improvement from baseline in CCQ total score (−0.18 vs 0.02; P = 0.001), symptoms (−0.21 vs 0.01; P = 0.002), functional state (−0.15 vs 0.02; P = 0.018), and mental state (−0.18 vs 0.02; P = 0.023) than patients receiving placebo. At study end, 38.3% of the arformoterol-treated patients and 30.8% of patients receiving placebo reported clinically significant improvements on the CCQ (P = 0.026). These improvements were only modestly correlated with improvements in FEV1 (r = −0.15; P < 0.01). In this 52-week trial, arformoterol-treated patients had greater improvements in health status than patients receiving placebo. Assessing health status along with lung function seems to provide additional information regarding the effectiveness of COPD maintenance treatments. ClinicalTrials.gov identifier: NCT00909779.

Background This study aimed to develop and provide a psychometric and feasibility pilot evaluation of the Heart Failure (HF) Symptom Tracker (HFaST), a new patient-reported tool designed to facilitate communication between patients and health care providers (HCPs) in routine clinical care. The HFaST enables patients to identify worsening HF symptoms, with a long-term goal of preventing hospitalizations or emergency room visits. Methods The HFaST was developed drawing on evidence from the literature, qualitatively with cognitive interviews (12 patient/caregiver and 8 HCPs), and evaluated quantitatively (psychometric, feasibility assessment). The HFaST was administered for 7 consecutive days to 100 individuals diagnosed with HF during a multisite, non-interventional US pilot study. Health care providers then completed a survey assessing the feasibility and importance of the HFaST in clinical practice. Qualitative development included a literature review and cognitive interviews with patients, caregivers, and HCPs. The psychometric properties of the HFaST were evaluated using classical test theory methods. Descriptive statistics provided insight into HCPs’ perceptions of the feasibility of using the HFaST in clinical practice. Results A preliminary set of 40 items was developed for the symptom tracker and iteratively reduced to 10 items based on the qualitative phase. Test-retest reliability (weighted kappa 0.71–0.97), discriminating validity, and construct validity of the HFaST were acceptable. HCPs rated the HFaST as a good (70%) or excellent (30%) means of tracking HF symptoms. Six HFaST items were ultimately retained, covering concepts of fatigue, shortness of breath (3 items), swelling, and rapid weight gain. Conclusions The 6-item HFaST is an easy-to-use tool designed to raise patients’ awareness of HF symptoms and facilitate communication with HCPs. Future research should evaluate HFaST implementation in clinical practice and effectiveness as an intervention to potentially prevent hospitalizations and emergency room visits.

Symptom severity is the largest factor in determining subjective health in COPD. Symptoms (eg, chronic cough, dyspnea) are associated with decreased health-related quality of life (HRQoL). We evaluated the impact of arformoterol on HRQoL in COPD patients, measured by St George's Respiratory Questionnaire (SGRQ). Post hoc growth mixture model (GMM) analysis examined symptom response profiles. We examined data from a randomized, double-blind, parallel-group, 12-month safety trial of twice-daily nebulized arformoterol 15 µg (n=420) versus placebo (n=421). COPD severity was assessed by Global Initiative for Chronic Obstructive Lung Disease (GOLD) status. GMM analysis identified previously unknown patient subgroups and examined the heterogeneity in response to SGRQ Symptoms scores. SGRQ Total score improved by 4.24 points with arformoterol and 2.02 points with placebo ( =0.006). Significantly greater improvements occurred for arformoterol versus placebo in SGRQ Symptoms (6.34 vs 4.25, =0.031) and Impacts (3.91 vs 0.97, =0.001) scores, but not in Activity score (3.57 vs 1.75, =0.057). GMM identified responders and nonresponders based on the SGRQ Symptoms score. End-of-study mean difference in SGRQ Symptoms scores between these latent classes was 20.7 points ( <0.001; 95% confidence interval: 17.6-23.9). Compared with nonresponders, responders were more likely current smokers (55.52% vs 44.02%, =0.0021) and had more severe COPD (forced expiratory volume in 1 second [FEV ]: 1.16 vs 1.23 L, =0.0419), more exacerbations (0.96 vs 0.69, =0.0018), and worse mean SGRQ Total (59.81 vs 40.57, <0.0001), Clinical COPD Questionnaire (3.29 vs 2.05, <0.0001), and Modified Medical Research Council Dyspnea Scale (3.13 vs 2.75, <0.0001) scores. Arformoterol-receiving responders exhibited significantly greater improvements in FEV (0.09 vs 0.008, =0.03) and fewer hospitalizations (0.13 vs 0.24, =0.02) than those receiving placebo. In this study, arformoterol treatment significantly improved HRQoL reflected by SGRQ. For the analysis performed on these data, arformoterol may be particularly effective in improving lung function and reducing hospitalizations among patients who are unable to quit smoking or present with more severe symptoms.

Measures assessing treatment outcomes in previous CC clinical trials have not met the requirements described in the US Food and Drug Administration's guidance on patient-reported outcomes. Psychometric analyses using data from one Phase IIb study and two Phase III trials of linaclotide for the treatment of chronic constipation (CC) were conducted to document the measurement properties of patient-reported CC Symptom Severity Measures. Each study had a multicenter, randomized, double-blind, placebo-controlled, parallel-group design, comparing placebo to four doses of oral linaclotide taken once daily for 4 weeks in the Phase IIb dose-ranging study (n=307) and to two doses of linaclotide taken once daily for 12 weeks in the Phase III trials (n=1,272). The CC Symptom Severity Measures addressing bowel function (Bowel Movement Frequency, Stool Consistency, Straining) and abdominal symptoms (Bloating, Abdominal Discomfort, Abdominal Pain) were administered daily using interactive voice-response system technology. Intraclass correlations, Pearson correlations, factor analyses, F-tests, and effect sizes were computed. The CC Symptom Severity Measures demonstrated satisfactory test-retest reliability and construct validity. Factor analyses indicated one factor for abdominal symptoms and another for bowel symptoms. Known-groups F-tests substantiated the discriminating ability of the CC Symptom Severity Measures. Responsiveness statistics were moderate to strong, indicating that these measures are capable of detecting change. In large studies of CC patients, linaclotide significantly improved abdominal and bowel symptoms. These psychometric analyses support the reliability, validity, discriminating ability, and responsiveness of the CC Symptom Severity Measures for evaluating treatment outcomes in the linaclotide clinical studies.

Background Cushing’s disease (CD) is a rare disorder of chronic hypercortisolism due to an adrenocorticotropic hormone (ACTH)-secreting pituitary corticotroph adenoma. Because hypercortisolism symptoms are wide ranging, it is important to assess a variety of outcomes including both clinical factors, such as cortisol levels, and health-related quality of life (HR-QOL), to better understand the severity and impact of CD on patients and the potential efficacy of CD treatment. Pasireotide, a somatostatin analog that targets somatostatin receptors on the pituitary adenoma, is under development as a treatment for CD. A phase III clinical trial was conducted to investigate its safety and efficacy in patients with CD. In this trial, HR-QOL was assessed with the Cushing’s Quality-of-Life (CushingQOL) questionnaire, specifically developed and validated in patients with Cushing’s syndrome. Objective Reliability, validity, the ability to detect change, and a minimal important difference (MID) were evaluated for the CushingQOL questionnaire using data from patients diagnosed with CD who participated in the phase III clinical trial designed to assess the safety and efficacy of different doses of pasireotide. Methods Adult patients ( n  = 162) with CD participated in a randomized, double-blind, multinational, phase III clinical trial. Patients received subcutaneous pasireotide (600 μg or 900 μg) twice daily for 3 months (double blind). After 3 months, some patients were unblinded based on their mean urinary free cortisol (mUFC) levels and were given the chance to increase their dosage, while the other patients remained blinded. At month 6, an open-label 6-month period began. The CushingQOL questionnaire was self-administered four times (baseline [ n  = 160], and at months 3 [ n  = 134], 6 [ n  = 113], and 12 [ n  = 76]). A confirmatory factor analysis (CFA) was conducted. Reliability estimates were calculated for internal consistency (coefficient alpha) and test retest (intraclass correlation coefficients [ICCs]) for patients with stable hypercortisolism at month 3 and month 6. Construct validity hypotheses (correlations), mean differences in known groups (ANOVAs), and responsiveness effect sizes (Guyatt’s) were estimated based on measures of cortisol, body mass index (BMI), waist circumference, weight, facial rubor (redness), striae (stretch marks), bruising, supraclavicular fat pad, dorsal fat pad, and results of the Beck Depression Inventory II (BDI-II). The half-standard deviation distribution method was used to estimate MID. Results CFA loadings supported a one-factor solution for the CushingQOL questionnaire items. Internal consistency reliability (0.87–0.88) and ICCs (0.87) were high. Construct validity hypotheses were in the anticipated direction. Changes in CushingQOL scores were moderately correlated with changes in mUFC levels, in BMI, and in weight. Mean scores for minimally depressed patients were significantly higher (indicating better HR-QOL) than for severely depressed patients. Moderate Guyatt’s responsiveness effect sizes were observed for patients who achieved reductions in weight, BMI, and waist circumference. Using the half-standard deviation method, an estimate of the MID was computed as 10.1. Conclusions This study provided evidence within the context of a longitudinal design that the CushingQOL questionnaire is a reliable, valid, and responsive instrument for the assessment of HR-QOL in adults with CD in accordance with recommendations set forth by regulatory agencies in the USA and Europe.

Purpose The objective of this study was to develop and validate a patient-reported outcome instrument to comprehensively assess the consequences of inadequate sleep for use in insomnia-related studies. Methods To inform item development, relevant constructs were identified through patient focus groups, literature review, and expert input. Following a translatability assessment for United States (US) English, US Spanish, and French, the draft items were refined through iterative sets of patient interviews in the United States and France. Psychometric properties were evaluated using patient responses from a validation study including 432 participants with either a diagnosis of primary insomnia or no history of insomnia. Results Psychometric analyses supported item reduction from 38 to 26 items, yielding a unidimensional scale and preserving the original content (mood, tiredness/energy, memory/concentration, motivation, daily performance, social interaction, sexual functioning). Evidence of internal consistency (coefficient α = 0.97), convergent validity, and known-groups validity also was documented. Conclusions The Sleep Functional Impact Scale (SFIS) is a psychometrically sound measure targeting the impact of insomnia on patient functioning. When administered with a sleep diary, this instrument has the ability to provide a more comprehensive assessment of treatment response in clinical studies.