Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here? (original) (raw)
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ERJ Open Research
BackgroundAn understanding of the experience of patients with progressive fibrosing interstitial lung disease (PF-ILD) is needed to select appropriate patient-reported outcome measures (PROMs) to evaluate treatment effect in clinical trials.MethodsA systematic literature review was conducted to develop a preliminary conceptual model of the symptoms experienced by patients with PF-ILD and the impacts the disease has on them. An online survey and consensus meetings were then conducted with 12–14 stakeholders (patients, clinicians, regulatory and payer advisors) to refine the conceptual model and critically appraise how key concepts should be measured by PROMs. PROMs assessed included: Living with Idiopathic Pulmonary Fibrosis, Living with Pulmonary Fibrosis, King's Brief Interstitial Lung Disease, Cough and Sputum Assessment Questionnaire, Evaluating Respiratory Symptoms, Leicester Cough Questionnaire, Functional Assessment of Chronic Illness Therapy (Dyspnoea/Fatigue) and St. Geo...
Thorax, 2012
Rationale Health status is impaired in patients with interstitial lung disease (ILD). There is a paucity of tools that assess health status in ILD. The objective of this study was to develop and validate the King's Brief Interstitial Lung Disease questionnaire (K-BILD), a new health status measure for patients with ILD. Methods Patients with ILD were recruited from outpatient clinics. The development of the questionnaire consisted of three phases: item generation; item reduction, allocation to domains by factor analysis, Rasch analysis to create unidimensional scales and validation; and repeatability testing. Results 173 patients with ILD (49 with idiopathic pulmonary fibrosis) completed a preliminary 71-item questionnaire. 56 items were removed due to redundancy, low factor loadings or poor fit to the Rasch model. The final version of the K-BILD questionnaire consisted of 15 items and three domains (breathlessness and activities, chest symptoms and psychological). Internal consistency assessed with Cronbach's a coefficient was 0.94 for the K-BILD total score. Concurrent validity of the K-BILD questionnaire was high compared with St George's Respiratory Questionnaire (r¼0.90) and moderate with lung function (vital capacity, r¼0.50). The K-BILD questionnaire was repeatable over 2 weeks (n¼44), with intraclass correlation coefficients for domains and total score 0.86e0.94. The K-BILD construct validity for patients with idiopathic pulmonary fibrosis was similar to that of other ILDs. Conclusion The K-BILD questionnaire is a brief, valid, self-completed health status measure for ILD. It could be used in the clinic to assess ILD from the patients' perspective.
BMC Pulmonary Medicine, 2019
Background Health-related quality of life (HRQL) is impaired in patients with idiopathic pulmonary fibrosis (IPF). The King’s Brief Interstitial Lung Disease questionnaire (K-BILD) is a validated measure of HRQL, but no previous studies have focused on the validity of K-BILD in IPF. Moreover, the relationship between K-BILD and dyspnoea or the 6-min walk test (6MWT) has not been assessed. The aim of this study was to validate K-BILD in the largest cohort of patients with IPF to date and assess how K-BILD correlates to dyspnoea and 6MWT. Methods Firstly, K-BILD was translated into Danish using validated translation procedures. Consecutive patients with IPF were recruited. At baseline, patients completed K-BILD, the IPF-specific version of St. Georges Respiratory Questionnaire, University of California, San Diego Shortness of Breath Questionnaire (SOBQ) Short Form-36, and pulmonary function tests and 6MWT were performed. After 14 days, K-BILD and Global Rating of Change Scales were co...
Frontiers in Pharmacology
Background: The World Health Organization (WHO) introduced the International Classification of Functioning, Disability, and Health (ICF) as a scientific method of disability data collection comprised of >1,200 categories describing the spectrum of impairment types (functional, symptoms-based and anatomical) under the bio-psycho-social model with consideration of environmental and personal factors (pf). ICF Core Sets and ICF Checklists are streamlined disease-specific resources for clinical use, service provision, and for use in health economics and health policy. ICF can disclose strengths and weaknesses across multiple patient-reported outcome measures (PROMs) and help consolidate best-fitting question-items from multiple PROMs. Interstitial lung diseases (ILDs), are generally progressive, with restrictive physiology sometimes occurring in the context of multi-organ autoimmunity/inflammatory conditions such as connective tissue diseases (CTDs). In spite of significant associated...
2020
BACKGROUND Limited data are available on the association between clinically measured disease severity markers and quality of life (QOL) in idiopathic pulmonary fibrosis (IPF). We examined the associations between objective disease severity metrics and QOL in a contemporary IPF population. METHODS We evaluated baseline data from patients enrolled in the multicenter, US-based IPF-PRO Registry between June 2014 and July 2018. Disease severity metrics included forced vital capacity [FVC] % predicted, diffusing capacity for carbon monoxide [DLCO] % predicted, supplemental oxygen use with activity, supplemental oxygen use at rest, and two summary scores: the Gender-Age-Lung Physiology (GAP) Index (based on gender, age, and % predicted values for DLCO and FVC) and the Composite Physiologic Index (CPI) (based on % predicted values for DLCO, FVC, and forced expiratory volume in 1 second). We used multivariable adjusted regression models to examine cross-sectional associations between each se...
Chest, 2019
Limited data are available on the association between clinically measured disease severity markers and patient-perceived well-being in IPF. The purpose of this analysis was to examine associations between established metrics of disease severity and quality of life (QOL) in a contemporary IPF population. METHODS: We evaluated baseline data from patients enrolled in the IPF-PRO Registry between June 2014 and July 2018. The IPF-PRO Registry is a multicenter outpatient registry of patients with IPF that was diagnosed or confirmed at the enrolling center in the past 6 months. Disease severity metrics of interest included four clinical variables (forced vital capacity [FVC], diffusing capacity for carbon monoxide [DLCO], supplemental oxygen use with activity, and supplemental oxygen use at rest). We also evaluated two summary severity scores: the Gender-Age-Lung Physiology (GAP) Index, which assigns points based on gender, age, DLCO, and FVC (range: 0-7 points, with higher score indicating greater severity); and the Composite Physiologic Index (CPI), which assigns points based on DLCO, FVC, and forced expiratory volume (range w5-80, with higher values indicating greater severity). QOL was estimated from the St. George's Respiratory Questionnaire (SGRQ), an instrument designed to measure health impairment in patients with respiratory disease. We used multivariable regression models to examine associations between each continuous severity measure and SGRQ total score at baseline, adjusting for demographics, body mass index, comorbidities and per capita income. RESULTS: Among 859 patients with complete QOL data, the median SGRQ score at study enrollment was 40 (IQR¼26-53; higher scores indicate worse quality of life). Increasing disease severity was associated with worse QOL for all six metrics. Modest SGRQ impairments were observed with increasing GAP score [2.9 (1.8-4.0) per 1 point increase in GAP] and with increasing CPI values [3.0 (2.4-3.6) per 5 point increase in CPI]. QOL deficits were associated with all four clinical variables examined, with substantial SGRQ impairments observed for oxygen use with activity [15.6 (12.9-18.2)], oxygen use at rest [16.2 (13.0-19.4)], decreasing FVC (3.7 (2.9-4.4) per 10% decrease in FVC % predicted] and decreasing DLCO [5.0 (4.0-6.1) per 10% decrease in DLCO % predicted]. CONCLUSIONS: Objective measures of disease severity are associated with worse QOL in contemporary IPF patients, with associations observed for composite severity metrics, physiologic parameters and supplemental oxygen use. CLINICAL IMPLICATIONS: IPF patients with more severe disease, particularly those who use supplemental oxygen, experience meaningful deficits in QOL. Future research is needed to identify the most effective QOL interventions for IPF patients with severe disease, particularly those who require supplemental oxygen.
Respiratory Research, 2021
Rationale The University of California, San Diego Shortness of Breath Questionnaire (UCSDSOBQ) is a frequently used domain-specific dyspnea questionnaire; however, there is little information available regarding its use and minimum important difference (MID) in fibrotic interstitial lung disease (ILD). We aimed to describe the key performance characteristics of the UCSDSOBQ in this population. Methods UCSDSOBQ scores and selected anchors were measured in 1933 patients from the prospective multi-center Canadian Registry for Pulmonary Fibrosis. Anchors included the St. George’s Respiratory Questionnaire (SGRQ), European Quality of Life 5 Dimensions 5 Levels questionnaire (EQ-5D-5L) and EQ visual analogue scale (EQ-VAS), percent-predicted forced vital capacity (FVC%), diffusing capacity of the lung for carbon monoxide (DLCO%), and 6-min walk distance (6MWD). Concurrent validity, internal consistency, ceiling and floor effects, and responsiveness were assessed, followed by estimation of...
The Journal of Rheumatology, 2014
Interstitial lung diseases (ILD), including those related to connective tissue disease (CTD), and idiopathic pulmonary fibrosis (IPF) carry high morbidity and mortality. Great efforts are under way to develop and investigate meaningful treatments in the context of clinical trials. However, efforts have been challenged by a lack of validated outcome measures and by inconsistent use of measures in clinical trials. Lack of consensus has fragmented effective use of strategies in CTD-ILD and IPF, with a history of resultant difficulties in obtaining agency approval of treatment interventions. Until recently, the patient perspective to determine domains and outcome measures in CTD-ILD and IPF had never been applied. Efforts described here demonstrate unequivocally the value and influence of patient involvement on core set development. Regarding CTD-ILD, this is the first OMERACT working group to directly address a manifestation/comorbidity of a rheumatic disease (ILD) as well as a disease...