Observational study of the Amaze™ asthma disease management platform.

Objective: Asthma is often inadequately controlled, which can result in exacerbations that lead to unplanned healthcare visits. Mobile application (app) use could help manage asthma exacerbations. We implemented the Amaze™ asthma disease management platform in clinical practice and assessed user satisfaction, usage, and usability.

Burden of adult atopic dermatitis and unmet needs with existing therapies.

Objective: Patients with atopic dermatitis (AD) have low treatment satisfaction. In this study, we evaluated the humanistic burden, treatment satisfaction, and treatment expectations in patients with AD in the United States.

Methods: Adults with AD recruited through the National Eczema Association and clinical sites completed a web-based survey comprising the Patient-Oriented SCORing Atopic Dermatitis (PO-SCORAD), Dermatology Life Quality Index; Work Productivity and Activity Impairment Questionnaire-Atopic Dermatitis; Treatment Satisfaction Questionnaire for Medication (TSQM); and answered questions on healthcare provider (HCP) visits, treatment history, and treatment goals.

Nocturnal polyuria in women: results from the EpiNP study.

Introduction And Hypothesis: Data from a large US population-based, cross-sectional, epidemiological study (the EpiNP Study) were used to assess the symptoms and bother experienced by women with nocturnal polyuria (NP).

Methods: Consenting participants recruited from an online panel completed the baseline EpiNP survey online (Lower Urinary Tract Symptoms Tool and urological comorbidities). All reporting ≥2 voids/night and a random sample of 100 respondents, each reporting 0 or 1 void/night were asked to complete a 3-day web-based bladder diary recording time, volume, and urgency rating of each void.

The patient burden of nocturnal polyuria in the United States: Results from the epidemiology of nocturnal polyuria (EpiNP) study.

Objectives: To explore the impact of nocturnal polyuria (NP) on health-related quality of life (HRQoL), work productivity, mental health, fatigue, bother, and daytime sleepiness.

Materials And Methods: This large-scale, US population-representative epidemiologic study was conducted in two parts: a web-based survey and 3-day bladder diary. Consenting participants completed the baseline Epidemiology of NP (EpiNP) survey online (Lower Urinary Tract Symptoms [LUTS] Tool, comorbidities, burden, and multiple HRQoL measures).

Dysphagia Days as an Assessment of Clinical Treatment Outcome in Eosinophilic Esophagitis.

Introduction: The aim of this study was to evaluate Dysphagia Days as a measure of symptom improvement in patients with eosinophilic esophagitis from the HEROES study.

Methods: Dysphagia Days, defined as a yes answer to the following question: During any meal today, did food go down slowly or get stuck in your throat or chest? was assessed for cendakimab vs placebo.

Results: A statistically significant reduction in the mean number of Dysphagia Days experienced was observed with cendakimab 360 mg vs placebo at week 16 (-4.

Validation and Meaningful Change Thresholds for an Objective Cough Frequency Measurement in Chronic Cough.

Purpose: Objective cough frequency is used to assess efficacy of chronic cough (CC) treatments. The objective of this study was to explore the relationship between objective cough frequency and cough-specific patient-reported outcomes (PROs) and estimate a clinically meaningful change threshold (MCT) for objective cough frequency.

Methods: Data collected in a phase 2b study in participants with refractory or unexplained CC were used to investigate the relationship between 24-h cough frequency (measured using an ambulatory cough monitor) and cough-specific PROs (i.

Leicester Cough Questionnaire validation and clinically important thresholds for change in refractory or unexplained chronic cough.

Introduction: The Leicester Cough Questionnaire (LCQ), a cough-specific quality-of-life measure, evaluates the impact of cough across physical, psychological, and social domains in patients with chronic cough (CC). This study assessed the psychometric properties of the LCQ.

Methods: Data from a phase IIb, randomized controlled trial of the P2X3-receptor antagonist gefapixant were analyzed (NCT02612610).

Differences in the Prevalence of Nocturnal Polyuria in the U.S. by Definition: Results from the Epidemiology of Nocturnal Polyuria Study.

Purpose: Prevalence data on nocturnal polyuria (NP), nocturia caused by overproduction of urine during sleep, is primarily limited to men and varies by NP definition. This U.S.

Developing a Conceptual Disease Model of Patient Experiences and Identifying Patient-Reported Clinical Outcome Assessments for Use in Trials of Treatments for Focal Onset Seizures.

Purpose: To identify concepts important to understanding the experiences of adults with focal onset seizures (FOS) and evaluate clinical outcome assessments (COAs) for measuring these concepts in clinical trials of treatments for FOS.

Methods: A search of published qualitative research, clinical trials, and approved product labels for FOS treatments was performed to develop a conceptual disease model (CDM) of patients' experience of living with FOS. Concepts of interest (COI) were selected, and a second literature search was conducted to identify COAs measuring these concepts.

The Prevalence of Nocturnal Polyuria in the United States: Results from the Epidemiology of Nocturnal Polyuria Study.

Background: The prevalence of nocturnal polyuria (NP), which is passing large volumes of urine during the main sleep period, has been investigated primarily in middle-aged to older men. There is thus a gap in the NP evidence base for women and for younger individuals.

Objective: To estimate the prevalence of nocturia due to NP in the USA.

Patient-Reported Disease Burden and Unmet Therapeutic Needs in Atopic Dermatitis.

Background: Atopic dermatitis (AD) is a common, chronic, relapsing, inflammatory skin disease causing a variety of dermatologic signs and symptoms, affecting patient’s quality of life. While treatment options are available, they are of variable effectiveness. This study sought to characterize patient-reported AD signs and symptoms, flare, and associated bother, by disease severity and control.

Validation of a visual analog scale for assessing cough severity in patients with chronic cough.

Introduction: Patients with chronic cough experience considerable burden. The cough severity visual analog scale (VAS) records patients' assessment of cough severity on a 100-mm linear scale ranging from "no cough" (0 mm) to "worst cough" (100 mm). Although cough severity scales are widely used in clinical practice and research, their use in patients with refractory or unexplained chronic cough has not been formally validated.

Measuring respiratory symptoms in moderate/severe asthma: evaluation of a respiratory symptom tool, the E-RS®: COPD in asthma populations.

Background: Symptom constructs included in the Evaluating Respiratory Symptoms in Chronic Obstructive Pulmonary Disease (E-RS®: COPD) tool may be relevant to patients with asthma. The purpose of this study was to evaluate content validity and psychometric performance of the E-RS: COPD in moderate/severe asthma patients.

Methods: Content validity of the E-RS: COPD was evaluated in patients with moderate/severe asthma using concept elicitation and cognitive debriefing interviews.

Quantitative measurement properties and score interpretation of the Cough Severity Diary in patients with chronic cough.

Aims: The Cough Severity Diary (CSD) was developed in accordance with the FDA guidance for patient-reported outcome measures and is focused on capturing the patient's perception of cough in terms of frequency, intensity, and disruption due to their cough. The measure includes a series of seven items asking patients to rate the frequency (three items), intensity (two items), and disruptiveness (two items) of their cough. The instrument was designed to be completed daily before bedtime, has a recall period of 'today,' and responses to items are entered on an 11-point numeric rating scale ranging from 0 to 10 with anchors on each end.

Understanding side effects of therapy for myasthenia gravis and their impact on daily life.

Background: Myasthenia gravis is a chronic, autoimmune, neuromuscular junction disorder characterized by skeletal muscle weakness. Current therapies for myasthenia gravis are associated with significant side effects. The objective of this study was to characterize the side effects, and associated health-related quality of life and treatment impacts, of traditional myasthenia gravis treatments.

Patient Preferences for GLP-1 Receptor Agonist Treatment of Type 2 Diabetes Mellitus in Japan: A Discrete Choice Experiment.

Introduction: The incidence and prevalence of type 2 diabetes mellitus (T2D) are increasing in Japan, and glucagon-like peptide-1 receptor agonists (GLP-1 RAs) are commonly used to treat the disease. The objective of this study was to use a discrete choice experiment (DCE) to characterize patient preferences for clinical treatment features of two GLP-1 RAs-dulaglutide 0.75 mg and semaglutide 0.

Can CAPTURE be used to identify undiagnosed patients with mild-to-moderate COPD likely to benefit from treatment?

Background: COPD Assessment in Primary Care To Identify Undiagnosed Respiratory Disease and Exacerbation Risk (CAPTURE™) uses five questions and peak expiratory flow (PEF) thresholds (males ≤350 L/min; females ≤250 L/min) to identify patients with a forced expiratory volume in 1 second (FEV)/forced vital capacity (FVC) <0.70 and FEV <60% predicted or exacerbation risk requiring further evaluation for COPD. This study tested CAPTURE's ability to identify symptomatic patients with mild-to-moderate COPD (FEV 60%-80% predicted) who may also benefit from diagnosis and treatment.

Performance of the inFLUenza Patient-Reported Outcome (FLU-PRO) diary in patients with influenza-like illness (ILI).

Background: The inFLUenza Patient Reported Outcome (FLU-PRO) measure is a daily diary assessing signs/symptoms of influenza across six body systems: Nose, Throat, Eyes, Chest/Respiratory, Gastrointestinal, Body/Systemic, developed and tested in adults with influenza.

Objectives: This study tested the reliability, validity, and responsiveness of FLU-PRO scores in adults with influenza-like illness (ILI).

Methods: Data from the prospective, observational study used to develop and test the FLU-PRO in influenza virus positive patients were analyzed.

Reliability, Validity, and Responsiveness of InFLUenza Patient-Reported Outcome (FLU-PRO©) Scores in Influenza-Positive Patients.

Objectives: To assess the reliability, validity, and responsiveness of InFLUenza Patient-Reported Outcome (FLU-PRO©) scores for quantifying the presence and severity of influenza symptoms.

Methods: An observational prospective cohort study of adults (≥18 years) with influenza-like illness in the United States, the United Kingdom, Mexico, and South America was conducted. Participants completed the 37-item draft FLU-PRO daily for up to 14 days.

Evaluation of a respiratory symptom diary for clinical studies of idiopathic pulmonary fibrosis.

Background: There are no validated patient diaries for evaluating respiratory symptoms in idiopathic pulmonary fibrosis (IPF).

Purpose: To evaluate the performance properties of the chronic obstructive pulmonary disease (COPD) Evaluating Respiratory Symptoms™ (E-RS™: COPD) measure in patients with IPF.

Methods: Concept elicitation and cognitive interviews were conducted with IPF patients to evaluate content validity, including comprehensiveness, relevance, and interpretability of E-RS™ items in this patient population.

Psychometric properties of morning joint stiffness duration and severity measures in patients with moderately to severely active rheumatoid arthritis.

Background: To assess the measurement properties of two single-item patient-reported outcome (PRO) measures that assessed the length of time (in minutes) and severity of morning joint stiffness (MJS) experienced each day.

Methods: Data from two Phase 3, randomized placebo-controlled (and active-controlled [RA-BEAM]), clinical studies assessing the safety and efficacy of baricitinib in adults with moderately to severely active rheumatoid arthritis (RA) were used to evaluate the psychometric properties of the Duration of MJS and Severity of MJS PROs.

Results: Test-retest reliability of Duration of MJS and Severity of MJS was supported through large intraclass correlation coefficients among stable patients (coefficient range for both studies: 0.