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Evaluating minimal important differences and responder definitions for the asthma symptom diary in patients with moderate to severe asthma.

ABSTRACT: BACKGROUND:The Asthma Symptom Diary was developed to assess severity of symptoms in patients with moderate to severe asthma, and has evidence supporting reliability and validity. Only limited information is available on sensitivity to change and responder definitions for the Asthma Symptom Diary. OBJECTIVES:Main study objectives were to evaluate sensitivity to change and provide responder definitions for clinically meaningful effects for the Asthma Symptom Diary. METHODS:This is a secondary analysis of Phase II clinical trial data in patients with moderate to severe asthma, Asthma Symptom Diary (ASD) was collected daily during the 24-week study. The Asthma Control Questionnaire and the Patient Global Assessment were collected at baseline, and week 12 and 24. Analysis of covariance (ANCOVA) models were used to evaluate sensitivity to change in Asthma Symptom Diary scores after 12 and 24?weeks of treatment. Anchor-based methods, using Asthma Control Questionnaire and Patient Global Assessment defined anchors, were used to identify minimal important differences and various responder criteria for changes in mean 7-day ASD score, symptomatic days, and minimal symptom days. RESULTS:Sample was 59% female, 81% White, with a mean age of 47.3 (SD?=?13.6) years. ANCOVAs demonstrated significant differences in baseline to week 12 and week 24 changes in mean 7-day Asthma Symptom Diary scores and symptomatic days by Asthma Control Questionnaire (all p?

SUBMITTER: Globe G

PROVIDER: S-EPMC6447631 | biostudies-literature | 2019 Apr

REPOSITORIES: biostudies-literature

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Evaluating minimal important differences and responder definitions for the asthma symptom diary in patients with moderate to severe asthma.

Globe Gary G Wiklund Ingela I Mattera Maria M Zhang Hao H Revicki Dennis A DA

Journal of patient-reported outcomes 20190403 1

<h4>Background</h4>The Asthma Symptom Diary was developed to assess severity of symptoms in patients with moderate to severe asthma, and has evidence supporting reliability and validity. Only limited information is available on sensitivity to change and responder definitions for the Asthma Symptom Diary.<h4>Objectives</h4>Main study objectives were to evaluate sensitivity to change and provide responder definitions for clinically meaningful effects for the Asthma Symptom Diary.<h4>Methods</h4>Th ...[more]

PMID: 30945020

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Project description:BackgroundPatient-reported outcome measures that facilitate self-report by children are needed to reduce the bias of proxy report. We previously developed an electronic Pediatric Asthma Symptom Diary (ePASD) to assess the severity of daily asthma symptoms and proximal impacts in children aged 6-11 years with mild to severe asthma. The ePASD, administered via a digital application with visuals, sounds, and text, is uniquely designed to minimize the importance of reading skills on children's ability to self-report accurately. Here, we describe the ePASD's psychometric properties.MethodsNinety-one children aged 6-11 years with mild to severe asthma and their caregivers participated in 2 study visits, which consisted of training on the provisioned device and completing asthma-specific clinical outcome assessment (COA) questionnaires. The children self-completed the ePASD at home twice daily for 8 consecutive days. The scoring of the ePASD was guided by factor analyses, inter-item correlations, and internal consistencies. Reliability, discriminating ability, construct validity, and responsiveness were evaluated for ePASD items and candidate scores.ResultsAll COAs included in the study-the ePASD, Asthma Control Questionnaire (ACQ), Childhood Asthma Control Test, Pediatric Asthma Quality of Life Questionnaire-Standardized (PAQLQ[S]), and global ratings-demonstrated that the children exhibited few asthma-related symptoms and impacts at all timepoints, and consequently, showed little change over time. Internal consistencies (all Cronbach's alphas ≥ 0.52) and test-retest reliabilities (all intraclass correlation coefficients ≥ 0.60) were largely satisfactory. Patterns of convergent and divergent correlations supported the construct validity of ePASD scores. The ePASD symptom scores correlated moderately to strongly with PAQLQ(S) Symptom scores (all correlations ≥ - 0.46) and with ACQ scores (all correlations ≥ 0.42), as predicted. Evidence of the discriminating ability of ePASD items and composite scores was demonstrated by known-groups analyses.ConclusionsThe ePASD is a reliable and valid measure of asthma symptoms and proximal impacts in children aged 6-11 years with mild, moderate, or severe asthma. These results lay the psychometric groundwork for use of the ePASD in future clinical trials for the management of pediatric asthma. An ongoing pediatric asthma treatment trial is anticipated to provide evidence of the ePASD's responsiveness to change.

Project description:BackgroundChildhood asthma is an important unmet need. To date, patient-reported outcome measures (PROMs) for children with asthma have used a combination of caregiver or proxy-reported and self-reported measures. No comprehensive measure is available to assess the severity and impact of daytime and nighttime asthma symptoms and rescue medication use for self-completion by children aged 6-11 years. This study aimed to develop a novel, interactive, electronic Pediatric Asthma Symptom Diary (ePASD) measuring self-reported key symptom severity and proximal impacts of asthma in young children with varying reading ability and disease severity, consistent with US Food and Drug Administration (FDA) PRO guidance and the International Society for Health Economics and Outcomes Research (ISPOR) good research practices.MethodsA targeted literature review and clinician interviews were undertaken to characterize symptoms and impacts experienced by children with mild-to-severe asthma. Concept elicitation interviews (CEIs) were conducted with 44 children and their caregivers (30 US; 14 UK). Following item and digital application development, the ePASD was assessed for relevance, understanding, and interpretability through cognitive debriefing interviews (CDIs) with 21 US children. Face validity/translatability assessments were also performed.ResultsKey measurement concepts included cough, wheeze, difficulty breathing, chest tightness/discomfort, nighttime awakening, and daytime activity limitations. Concept saturation was reached during CEIs for primary asthma-related daytime and nighttime symptoms and core impacts. Most CDI participants found the ePASD items clear, understandable, and comprehensive. Standardized training is anticipated to facilitate reliable child self-report.ConclusionThe ePASD, a novel PROM for children aged 6-11 years with asthma, uses an innovative multimedia approach and has been developed in accordance with FDA PRO guidance and ISPOR good research practices, directly capturing the child's self-reported asthma symptoms, impacts on daily activities and nighttime awakening, and rescue medication use.

Project description:BackgroundSymptom 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.MethodsContent validity of the E-RS: COPD was evaluated in patients with moderate/severe asthma using concept elicitation and cognitive debriefing interviews. Secondary analyses using data from two clinical trials in patients with moderate/severe asthma evaluated the factor structure of the E-RS: COPD plus two supplementary items (wheeze; shortness of breath with strenuous physical activity) and assessed psychometric properties of the tool, which will be referred to as E-RS®: Asthma when used in asthma populations.ResultsQualitative interviews (N = 25) achieved concept saturation for asthma respiratory symptoms. Concepts in the E-RS: COPD were relevant to patients and instructions were understood. Most patients (19/25; 76%) reported experiencing all concepts in the E-RS: COPD; no patients indicated missing symptoms. Secondary analyses of clinical trial data supported the original factor structure (RS-Total and three symptom-specific subscales). The two supplemental items did not fit with this factor structure and were not retained. RS-Total and subscale score reliability was high (internal consistency [α] > 0.70). Validity was demonstrated through significant (P < 0.0001) relationships with the St George's Respiratory Questionnaire (SGRQ) and Asthma Symptom Severity scale. E-RS: Asthma was responsive to change when evaluated using SGRQ, Patient Global Impression of Change and Asthma Quality of Life Questionnaire as anchors (P < 0.0001). Clinically meaningful change thresholds were also identified (RS-Total: - 2.0 units).ConclusionsThe E-RS: Asthma is reliable and responsive for evaluating respiratory symptoms in patients with moderate/severe asthma.

Project description:BackgroundPhysical and psychological symptoms are the hallmark of patients' subjective perception of their illness. The purpose of this analysis was to determine if patients with COPD have distinctive symptom profiles and to examine the association of symptom profiles with systemic biomarkers of inflammation.MethodsWe conducted latent class analyses of three physical (dyspnea, fatigue, and pain) and two psychological symptoms (depression and anxiety) in 302 patients with moderate to severe COPD using baseline data from a longitudinal observational study of depression in COPD. Systemic inflammatory markers included IL1, IL8, IL10, IL12, IL13, INF, GM-CSF, TNF-α (levels >75thcentile was considered high); and CRP (levels >3 mg/L was considered high). Multinominal logistic regression models were used to examine the association between symptom classes and inflammation while adjusting for key socio-demographic and disease characteristics.ResultsWe found that a 4-class model best fit the data: 1) low physical and psychological symptoms (26%, Low-Phys/Low-Psych), 2) low physical but moderate psychological symptoms (18%, Low-Phys/Mod Psych), 3) high physical but moderate psychological symptoms (25%, High-Phys/Mod Psych), and 4) high physical and psychological symptoms (30%, High-Phys/High Psych). Unadjusted analyses showed associations between symptom class with high levels of IL7, IL-8 (p ≤ .10) and CRP (p < .01). In the adjusted model, those with a high CRP level were less likely to be in the High-Phys/Mod-Psych class compared to the Low-Phys/Low-Psych (OR: 0.41, 95%CI 0.19, 0.90) and Low-Phys/Mod-Psych classes (OR: 0.35, 95%CI 0.16, 0.78); elevated CRP was associated with in increased odds of being in the High-Phys/High-Psych compared to the High-Phys/Mod-Psych class (OR: 2.22, 95%CI 1.08, 4.58). Younger age, having at least a college education, oxygen use and depression history were more prominent predictors of membership in the higher symptom classes.ConclusionsPatients with COPD can be classified into four distinct symptom classes based on five commonly co-occurring physical and psychological symptoms. Systemic biomarkers of inflammation were not associated with symptom class. Additional work to test the reliability of these symptom classes, their biological drivers and their validity for prognostication and tailoring therapy in larger and more diverse samples is needed.Trial registrationClinicaltrials.gov, NCT01074515 .

Project description:BACKGROUND:Severe asthma is a chronic disease contributing to disproportionate disease morbidity and mortality. From the year of 2007, many genome-wide association studies (GWAS) have documented a large number of asthma-associated genetic variants and related genes. Nevertheless, the molecular mechanism of these identified variants involved in asthma or severe asthma risk remains largely unknown. METHODS:In the current study, we systematically integrated 3 independent expression quantitative trait loci (eQTL) data (N?=?1977) and a large-scale GWAS summary data of moderate-to-severe asthma (N?=?30,810) by using the Sherlock Bayesian analysis to identify whether expression-related variants contribute risk to severe asthma. Furthermore, we performed various bioinformatics analyses, including pathway enrichment analysis, PPI network enrichment analysis, in silico permutation analysis, DEG analysis and co-expression analysis, to prioritize important genes associated with severe asthma. RESULTS:In the discovery stage, we identified 1129 significant genes associated with moderate-to-severe asthma by using the Sherlock Bayesian analysis. Two hundred twenty-eight genes were prominently replicated by using MAGMA gene-based analysis. These 228 replicated genes were enriched in 17 biological pathways including antigen processing and presentation (Corrected P?=?4.30?×?10-?6), type I diabetes mellitus (Corrected P?=?7.09?×?10-?5), and asthma (Corrected P?=?1.72?×?10-?3). With the use of a series of bioinformatics analyses, we highlighted 11 important genes such as GNGT2, TLR6, and TTC19 as authentic risk genes associated with moderate-to-severe/severe asthma. With respect to GNGT2, there were 3 eSNPs of rs17637472 (PeQTL?=?2.98?×?10-?8 and PGWAS?=?3.40?×?10-?8), rs11265180 (PeQTL?=?6.0?×?10-?6 and PGWAS?=?1.99?×?10-?3), and rs1867087 (PeQTL?=?1.0?×?10-?4 and PGWAS?=?1.84?×?10-?5) identified. In addition, GNGT2 is significantly expressed in severe asthma compared with mild-moderate asthma (P?=?0.045), and Gngt2 shows significantly distinct expression patterns between vehicle and various glucocorticoids (Anova P?=?1.55?×?10-?6). CONCLUSIONS:Our current study provides multiple lines of evidence to support that these 11 identified genes as important candidates implicated in the pathogenesis of severe asthma.

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Evaluating minimal important differences and responder definitions for the asthma symptom diary in patients with moderate to severe asthma.

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Evaluating minimal important differences and responder definitions for the asthma symptom diary in patients with moderate to severe asthma.

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