Validation of Constant Work Rate Cycling Endurance Time for Use in Chronic Obstructive Pulmonary Disease Clinical Trials.

A COPD Foundation working group sought to identify measures of exercise endurance, a meaningful aspect of physical functioning in everyday life among patients with chronic obstructive pulmonary disease (COPD) that is not fully accepted in regulatory decision making, hampering drug development. To demonstrate, as we previously asserted (Casaburi 2022;9:252), that constant work rate cycling endurance time is an appropriate exercise endurance measure in patients with COPD. To validate this assertion, we assembled an integrated database of endurance time responses, including 8 bronchodilator (2,166 subjects) and 15 exercise training (3,488 subjects) studies (Casaburi 2022;9:520).

Performance of the Cough and Sputum Assessment Questionnaire (CASA-Q) in COPD: Evidence from Clinical and Online Patient Interaction Studies.

Introduction: Patient perception of the burden of chronic bronchitis symptoms in chronic obstructive pulmonary disease (COPD) can be assessed using patient-reported outcome measures (PROMs). The Cough and Sputum Assessment Questionnaire (CASA-Q) was developed and tested for this purpose. This study reviewed the performance of the CASA-Q in published online studies and tested a novel approach to complement traditional methods of qualitative content validation.

Interpreting Evaluating Respiratory Symptoms in COPD Diary Scores in Clinical Trials: Terminology, Methods, and Recommendations.

Accurately interpreting scores on patient-reported outcome (PRO) measures is essential to understanding and communicating treatment benefit. Over the years, terminology and methods for developing recommendations for PRO score interpretation in clinical trials have evolved, leading to some confusion in the field. The phrase "minimal clinically important difference (MCID)" has been simplified to "minimal important difference (MID)" and use of responder thresholds to interpret statistically significant treatment effects has increased.

A Conceptual Framework for Use of Increased Endurance Time During Constant Work Rate Cycle Ergometry as a Patient-Focused Meaningful Outcome in COPD Clinical Trials.

The Chronic Lung Disease Biomarker and Clinical Outcome Assessment Qualification Consortium (CBQC) evaluates the potential of biomarkers and outcome measures as drug development tools. Exercise endurance is an objective indicator of treatment benefit, closely related to daily physical function. Therefore, it is an ideal candidate for an outcome for drug development trials.

Use of the Evaluating Respiratory Symptoms™ in COPD as an Outcome Measure in Clinical Trials: A Rapid Systematic Review.

Rationale: Patients with chronic obstructive pulmonary disease (COPD) struggle with respiratory symptoms that impair their daily activities and quality of life. Understanding a treatment's ability to relieve symptoms requires precise assessment. The Evaluating Respiratory Symptoms in COPD (E-RS:COPD) was developed to quantify respiratory symptoms in clinical trials.

Protocol Summary of the COPD Assessment in Primary Care To Identify Undiagnosed Respiratory Disease and Exacerbation Risk (CAPTURE) Validation in Primary Care Study.

Chronic obstructive pulmonary disease (COPD) often remains undiagnosed and untreated. To date, COPD screening/case finding has not been designed to identify , disease ready for therapies beyond smoking cessation. Herein, we describe the ongoing prospective, pragmatic cluster-randomized controlled trial to assess specificity and sensitivity of the OPD ssessment in rimary Care o Identify ndiagnosed espiratory Disease and xacerbation Risk (CAPTURE) tool consisting of 5 questions and peak expiratory flow.

Responsiveness of PROMIS® to change in chronic obstructive pulmonary disease.

Background: Chronic obstructive pulmonary disease (COPD) is a progressive chronic disease characterized by airflow obstruction that leads to shortness of breath and substantial negative impacts on health-related quality of life (HRQL). The course of COPD includes periodic acute exacerbations that require changes in treatment and/or hospitalizations. This study was designed to examine the responsiveness of Patient-Reported Outcomes Measurement Information System® (PROMIS®) measures to changes associated with COPD exacerbation recovery.

Reliability, validity and responsiveness of E-RS:COPD in patients with spirometric asthma-COPD overlap.

Background: The Evaluating Respiratory Symptoms in Chronic Obstructive Pulmonary Disease (E-RS:COPD) is a patient-reported diary that assesses respiratory symptoms in stable COPD.

Methods: This post hoc analysis of a randomized, double-blind, parallel-arm trial (GSK ID: 200699; NCT02164539) assessed the structure, reliability, validity and responsiveness of the E-RS, and a separate wheeze item, for use in patients with a primary diagnosis of asthma or COPD, but with spirometric characteristics of both (fixed airflow obstruction and reversibility to salbutamol; a subset of patients referred to as spirometric asthma-COPD overlap [ACO]; N = 338).

Results: Factor analysis demonstrated that E-RS included Cough and Sputum, Chest Symptoms, and Breathlessness domains, with a Total score suitable for quantifying overall respiratory symptoms (comparative fit index: 0.

Using the Influenza Patient-reported Outcome (FLU-PRO) diary to evaluate symptoms of influenza viral infection in a healthy human challenge model.

Background: In clinical studies involving a healthy volunteer human challenge model, a valid and reliable measure to assess the evolution of patient-reported symptom type and severity following viral exposure is necessary. This study examines the use of the InFLUenza Patient-Reported Outcome (FLU-PRO) diary as a standardized measure of symptom severity in a healthy volunteer human challenge model.

Methods: Healthy adults admitted to the NIH Clinical Center (Day - 1) underwent a 9-day inpatient quarantine after intranasal challenge with a wild-type influenza A/H1N1pdm virus (Day 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.

Exacerbation recovery patterns in newly diagnosed or maintenance treatment-naïve patients with COPD: secondary analyses of TICARI 1 trial data.

Background: Little is known about the recovery patterns from acute exacerbations of chronic obstructive pulmonary disease (AECOPD) in newly diagnosed or maintenance treatment-naïve patients with COPD. This study describes the course of AECOPD in these patients at the time of treatment for the symptoms of acute respiratory tract infection (RTI).

Methods: This study was a secondary analysis of data from a 12-week, randomized clinical trial (TICARI 1) testing the efficacy and safety of once-daily tiotropium 18 µg maintenance therapy versus placebo in newly diagnosed or maintenance treatment-naïve COPD patients with acute RTI symptoms for ≤7 days.

Effect of Bronchodilation, Exercise Training, and Behavior Modification on Symptoms and Physical Activity in Chronic Obstructive Pulmonary Disease.

Rationale: Bronchodilation and exercise training (ExT) improve exercise tolerance in patients with chronic obstructive pulmonary disease (COPD); however, behavior modification is required to impact daily physical activity (PA).

Objectives: To assess whether tiotropium/olodaterol, with or without ExT, would improve exercise endurance time (EET) and PA compared with placebo in patients participating in a self-management behavior-modification (SMBM) program.

Methods: This was a 12-week, randomized, partially double-blind, placebo-controlled, parallel-group trial in patients with COPD (PHYSACTO; NCT02085161).

The Short-term Impact of Symptom-defined COPD Exacerbation Recovery on Health Status and Lung Function.

This study examined the short-term effects of symptom-defined exacerbation recovery on health status and pulmonary function in moderate to severe chronic obstructive pulmonary disease (COPD) patients. Secondary analyses of pooled data from two 12-week Phase II international, randomized controlled trials using the cerbations of hronic Pulmonary Disease ool (EXACT) to identify symptom-defined exacerbations were conducted. Recovery was categorized as recovered, unrecovered (persistent worsening), or censored.

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.

Respiratory Symptoms Items from the COPD Assessment Test Identify Ever-Smokers with Preserved Lung Function at Higher Risk for Poor Respiratory Outcomes. An Analysis of the Subpopulations and Intermediate Outcome Measures in COPD Study Cohort.

Rationale: Ever-smokers without airflow obstruction scores greater than or equal to 10 on the COPD Assessment Test (CAT) still have frequent acute respiratory disease events (exacerbation-like), impaired exercise capacity, and imaging abnormalities. Identification of these subjects could provide new opportunities for targeted interventions.

Objectives: We hypothesized that the four respiratory-related items of the CAT might be useful for identifying such individuals, with discriminative ability similar to CAT, which is an eight-item questionnaire used to assess chronic obstructive pulmonary disease impact, including nonrespiratory questions, with scores ranging from 0 to 40.

A systematic approach for studying the signs and symptoms of fever in adult patients: the fever assessment tool (FAST).

Background: Although body temperature is one of four key vital signs routinely monitored and treated in clinical practice, relatively little is known about the symptoms associated with febrile states. The purpose of this study was to assess the validity, reliability and feasibility of the Fever Assessment Tool (FAST) in an acute care research setting.

Methods: Qualitative: To assess content validity and finalize the FAST instrument, 12 adults from an inpatient medical-surgical unit at the National Institutes of Health (NIH) Clinical Center participated in cognitive interviews within approximately 12 h of a febrile state (tympanic temperature ≥ 38° Celsius).

Beyond study participants: a framework for engaging patients in the selection or development of clinical outcome assessments for evaluating the benefits of treatment in medical product development.

Background: Patients are participating more actively in health care decision-making with regard to their health, as well as in the broader realm of assessing the value of medical products and influencing decisions about their registration and reimbursement. There is an increasing trend to include patients' perspectives throughout the stages of medical product development by broadening the traditional study-participant role to that of an active partner throughout the process. Including patients in the selection and development of clinical outcome assessments (COAs) to evaluate the benefit of treatment is particularly important.

How Well Does CAPTURE Translate?: An Exploratory Analysis of a COPD Case-Finding Method for Spanish-Speaking Patients.

Background: This study tested the properties of a Spanish translation of CAPTURE (COPD Assessment in Primary Care To Identify Undiagnosed Respiratory Disease and Exacerbation Risk) with selective use of peak expiratory flow (PEF).

Methods: This study comprised analyses of data from the Spanish-speaking cohort of the cross-sectional, case-control study used to develop CAPTURE. Translation procedures included forward and backward translation, reconciliation, and cognitive interviewing to assure linguistic and cultural equivalence, yielding CAPTURE-S.