Development and Initial Validation Analyses of the Living with Idiopathic Pulmonary Fibrosis Questionnaire.
Ontology highlight
ABSTRACT:
SUBMITTER: Swigris JJ
PROVIDER: S-EPMC7737580 | biostudies-literature |
REPOSITORIES: biostudies-literature
Similar Datasets
Project description: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 completed. Internal consistency, concurrent validity, test-retest reliability and known groups validity were assessed. Analyses were also performed in subgroups of patients with different time since diagnosis. RESULTS:At baseline, 150 patients with IPF completed the questionnaires, and 139 patients completed the questionnaires after 14?days. K-BILD had a high internal consistency (Cronbach's ??=?0.92). The concurrent validity was strong compared to SOBQ (r?=?-?0.66) and moderate compared to 6MWT (r?=?0.43). Intraclass correlation coefficients (ICC?=?0.91) and a Bland Altman plot demonstrated a good reliability. K-BILD was also able to discriminate between patients with different stages of disease (p?<?0.002, ?score >?7.4) and most results were comparable in patients with different time since diagnosis. CONCLUSION:K-BILD is a valid and reliable instrument in patients with IPF and in patients with different time since diagnosis. To a major extent, K-BILD scores reflected the impact of dyspnoea on HRQL and the impact of physical functional capacity measured by the 6MWT to a moderate degree. Compared to PFTs alone, K-BILD provides additional information on the burden of living with IPF, and importantly, K-BILD is simple to implement in both research and clinical contexts. TRIAL REGISTRATION:Clinicaltrials.org (NCT02818712) on 30 June 2016.
| S-EPMC6924069 | biostudies-literature
Project description:This SuperSeries is composed of the SubSeries listed below.
2022-07-01 | GSE175459 | GEO
Project description:The concept of rule-governed behavior (RGB) has been used in the behavior-analytic literature as a way to analyze complex human behavior, including thinking and problem-solving. Relational frame theory suggests the existence of two main functional types of RGB termed pliance and tracking. In this paper, we describe the development of the Generalized Tracking Questionnaire (GTQ) and the preliminary evaluation of its psychometric properties and validity through three studies, with a total of 1155 participants. In Study 1, a pool of items describing the main characteristics of generalized tracking was designed and evaluated by experts on the RFT account of RGB. The resulting 11 items were administered to 460 undergraduates to examine the understandability and psychometric quality of the items. The exploratory factor analysis indicated that the GTQ can be seen as a unidimensional scale, with all items exhibiting high factor loadings and corrected item-total correlations. In Study 2, the GTQ was administered online to a sample of 464 non-clinical participants and a clinical sample of 125 participants. The one-factor model of the GTQ obtained a good fit in the conducted confirmatory factor analysis. The GTQ showed measurement invariance across gender and clinical and nonclinical participants. It also obtained excellent internal consistency and correlated in theoretically coherent ways with other constructs. In Study 3, the GTQ and a neuropsychological battery of executive functions were administered to 105 participants. The GTQ showed statistically significant, medium-size correlations with working memory tests, verbal fluency, planning, and behavioral inhibition. In conclusion, the GTQ seems to be a promising measure to advance in the empirical analysis of functional classes of RGB.
| S-EPMC7289427 | biostudies-literature
Project description:Misophonia is characterized by decreased tolerance and accompanying defensive motivational system responding to certain aversive sounds and contextual cues associated with such stimuli, typically repetitive oral (e. g., eating sounds) or nasal (e.g., breathing sounds) stimuli. Responses elicit significant psychological distress and impairment in functioning, and include acute increases in (a) negative affect (e.g., anger, anxiety, and disgust), (b) physiological arousal (e.g., sympathetic nervous system activation), and (c) overt behavior (e.g., escape behavior and verbal aggression toward individuals generating triggers). A major barrier to research and treatment of misophonia is the lack of rigorously validated assessment measures. As such, the primary purpose of this study was to develop and psychometrically validate a self-report measure of misophonia, the Duke Misophonia Questionnaire (DMQ). There were two phases of measure development. In Phase 1, items were generated and iteratively refined from a combination of the scientific literature and qualitative feedback from misophonia sufferers, their family members, and professional experts. In Phase 2, a large community sample of adults (n = 424) completed DMQ candidate items and other measures needed for psychometric analyses. A series of iterative analytic procedures (e.g., factor analyses and IRT) were used to derive final DMQ items and scales. The final DMQ has 86 items and includes subscales: (1) Trigger frequency (16 items), (2) Affective Responses (5 items), (3) Physiological Responses (8 items), (4) Cognitive Responses (10 items), (5) Coping Before (6 items), (6) Coping During (10 items), (7) Coping After (5 items), (8) Impairment (12 items), and Beliefs (14 items). Composite scales were derived for overall Symptom Severity (combined Affective, Physiological, and Cognitive subscales) and Coping (combined the three Coping subscales). Depending on the needs of researchers or clinicians, the DMQ may be use in full form, individual subscales, or with the derived composite scales.
| S-EPMC8511674 | biostudies-literature
Project description:BACKGROUND:The impact of cutaneous leishmaniasis (CL) on the quality of life of patients has been neglected in research studies worldwide. The few reported studies have used non-specific questionnaires for the disease, which represents a limitation since generic instruments may not address specific aspects of the disease, compromising the evaluation of its real impact. The aim of this paper is to describe the development and the initial validation of an instrument for evaluating the impact of CL, named the Cutaneous Leishmaniasis Impact Questionnaire. METHODOLOGY:The formulation and validation of the instrument consisted of the following steps: (1) literature review; (2) conceptual framework construction and initial item generation; (3) tool analysis by health professionals (experts); (4) tool evaluation performed by the patients; and (5) a pilot study with 100 patients with localized CL, evaluated at a reference ambulatory facility in Belo Horizonte, in the state of Minas Gerais, Brazil. The structure of the proposed instrument was analyzed using hierarchical cluster analysis (ICLUST). RESULTS:Twenty-seven items were initially proposed by the researchers to compose the questionnaire. Content validity (evaluates if the instrument fully assesses the construct of interest) was evaluated by the panel of experts, while face validity (evaluates how potential participants interpret the items) was evaluated by the target population. In this step, some items were excluded, reformulated and/or included. After evaluating a factorial structure of the items in accordance with the cluster analysis, we assembled a questionnaire with 25 items (alpha = 0.86), with high reliability and homogeneity, which address the following: 1) the general impact of the disease (alpha = 0.91, beta = 0.67) and 2) the evaluation of the perception about the treatment and health services (alpha = 0.72, beta = 0.51). CONCLUSIONS:The Cutaneous Leishmaniasis Impact Questionnaire, developed with contributions from patients and experts, was confirmed, in this first validation, as a useful and reliable instrument.
| S-EPMC6117079 | biostudies-literature
Project description:Background: Historically, idiopathic pulmonary fibrosis (IPF) was considered a chronic inflammation disorder, but this conception was reassessed in the past decades. Our understanding of the role of inflammation in IPF and its association with clinical significance remained incomplete. Methods: We downloaded mRNA expression data of peripheral blood mononuclear cells (PBMCs) from the Gene Expression Omnibus (GEO) repository. Inflammation-related genes (IRGs) expressed differently between IPF and control (CTRL) were determined. In this study, we systemically analyzed the expression of differently expressed IRGs by comprehensive bioinformatic analysis, and then investigated their potential prognostic values. The related prognostic gene expressions were verified in our cohort. Results: 110 differently expressed IRGs were identified in this study, including 64 upregulated and 46 downregulated IRGs. Three IRGs (S100A12, CCR7, and TNFSF4) were identified as potential hub genes for prognosis. Those genes were subsequently subjected to the construction of the prognostic models. In the results, IPF patients categorized as high risk demonstrated a poor overall survival rate compared to patients categorized as low risk. Based on this prognostic model, the area under the curve (AUC) of the survival-dependent receiver operator characteristic (ROC) for 1-year, 2-year, and 3-year survival rates was 0.611, 0.695, and 0.681, respectively, in the GSE28042 cohort. These observations were validated in the GSE27957 cohort, confirming the good prognostic effect of this model. The expression of the three genes was validated in our cohort. We also conducted a nomogram based on the three IRGs' mRNA for quantitative IPF prognosis. Conclusion: Three IRGs (S100A12, CCR7, and TNFSF4) were identified as potential markers for the prognosis of IPF.
| S-EPMC8339426 | biostudies-literature
Project description:Rationale: To identify functionally relevant common genetic risk variants associated with idiopathic pulmonary fibrosis (IPF), we performed expression quantitative trait loci (eQTL) and methylation quantitative trait loci (mQTL) mapping, followed by co-localization of eQTL and mQTL with genetic association signals as well as mediation analysis. Methods: Illumina MEGA genotyping arrays, mRNA sequencing, and Illumina 850k methylation arrays were performed on lung tissue of participants with IPF (234 RNA and 345 DNA samples) and non-diseased control (188 RNA and 202 DNA samples). Transcriptome and methylome datasets were normalized for unknown batch effects using Probabilistic Estimation of Expression Residuals (PEER). eQTL, cell type-interaction eQTL, and mQTL analyses were performed in FastQTL and co-localization analysis in eCAVIAR separately for cases and controls. Benjamini-Hochberg false discovery rate was used for adjustment for multiple comparisons. Results: After appropriate adjustment, we identified 4,745 genes with significant eQTLs in controls and 6,047 in cases (FDR-adjusted p<0.05). Focusing on genetic variants within 10 primary IPF-associated genetic loci, we identified 27 eQTLs in controls and 24 eQTLs in cases (FDR-adjusted p<0.05). Among these signals, we identified association of rs35705950 with expression of MUC5B (Chr11 locus) and rs2076295 with expression of DSP (Chr6 locus) in both cases and controls. To address cell specificity, we performed cell type-interaction eQTL analysis and identified an association of rs2076295 with expression of DSP in smooth muscle cells. mQTL analysis identified CpGs in gene bodies of MUC5B (cg17589883) and DSP (cg08964675) associated with the lead variants in these two loci. eCAVIAR demonstrated strong co-localization of eQTL/mQTL and genetic signal in MUC5B (rs35705950) and DSP (rs2076295) in both cases and controls. Mediation analysis demonstrated partial mediation of the effect of common variants in MUC5B and DSP loci on disease risk though MUC5B and DSP gene expression. Functional validation of the mQTL in MUC5B demonstrates that the CpG is within a putative internal repressor element that interacts through a 3D loop with the enhancer containing the rs35705950 genetic variant. Conclusions: Using lung eQTL/mQTL, co-localization, and mediation analyses, we have established the functional validation of the common IPF genetic risk variants for MUC5B (rs35705950) and DSP (rs2076295). These results provide additional evidence that both MUC5B and DSP are involved in the etiology of IPF.
2022-07-01 | GSE175457 | GEO
Project description:Idiopathic pulmonary fibrosis (IPF) is a disease related to AT2 cell. We used flow cytometry to analyze the epithelial component of donor and IPF lungs. From the live cells, we first excluded the CD31PosCD45Pos and then selected the EPCAMPos cells for further analysis using the human AT2 cell marker HTll-280 and the surface marker PD-L1. Our data indicate that, the bona fide differentiated AT2 cells (HTll-280High PD-L1Neg), were drastically reduced in the context of IPF. More interestingly, the number of HTll-280Low/Neg PD-L1High was drastically increased, suggesting that HTll-280Low PD-L1High epithelial cells could represent a pool of progenitors linked to the deficient AT2 lineage. The aim of this experiment is further characterization of AT2 and PDL1+ cells in donor and IPF.
2023-01-01 | GSE195770 | GEO
Project description:BackgroundIdiopathic pulmonary fibrosis (IPF) is a progressive interstitial lung disease that often causes disabling dyspnea. In IPF and other lung diseases, patient-reported outcomes (PROs)-questionnaires designed to gather information from the patient's perspective-can determine whether therapies affect dyspnea or other outcomes meaningful to patients. Before a PRO can be used confidently as an outcome measure in a longitudinal trial, studies must demonstrate the PRO's ability to capture change over time in the target population. Our goal in this study was to examine whether the UCSD Shortness of Breath Questionnaire does so in patients with IPF.MethodsWe used data from the Sildenafil Trial of Exercise Performance in Idiopathic Pulmonary Fibrosis (STEP-IPF) to perform analyses that examined associations between UCSD scores and five external measures (anchors) at baseline and over time. Anchors included the Activity domain from St. George's Respiratory Questionnaire (SGRQ-A), the Physical Functioning domain from the SF-36 (SF36-PF), forced vital capacity (FVC), diffusing capacity of the lung for carbon monoxide (DLCO), and distance walked during a timed walk test (6MWD). Linear regression models were used to examine relationships between UCSD scores and anchors over time.ResultsAt baseline, UCSD scores were weakly correlated with percent predicted FVC (-0.21, p = 0.005) and percent predicted DLCO (-0.20, p = 0.008), moderately correlated with 6MWD (-0.39, p < 0.0001) and strongly correlated with SGRQ-A (0.79, p < 0.0001) and SF36-PF (-0.72, p < 0.0001). Change over time in UCSD scores was associated with change in FVC (estimate = 2.54, standard error [SE] = 1.23, p = 0.04), SGRQ-A (estimate = 7.94, SE = 1.11, p < 0.0001), SF36-PF (estimate = 6.00, SE = 1.13, p < 0.0001), and 6MWD (estimate = 4.23, SE = 1.18, p = 0.0004) but not DLCO (estimate = 0.33, SE = 1.33, p = 0.80).ConclusionsThese results support the validity of the UCSD to assess change in dyspnea over time in patients with IPF.
| S-EPMC3441137 | biostudies-literature
Project description:BackgroundThe randomized placebo-controlled IFIGENIA-trial demonstrated that therapy with high-dose N-acetylcysteine (NAC) given for one year, added to prednisone and azathioprine, significantly ameliorates (i.e. slows down) disease progression in terms of vital capacity (VC) (+9%) and diffusing capacity (DLco) (+24%) in idiopathic pulmonary fibrosis (IPF). To better understand the clinical implications of these findings we performed additional, explorative analyses of the IFGENIA data set.MethodsWe analysed effects of NAC on VC, DLco, a composite physiologic index (CPI), and mortality in the 155 study-patients.ResultsIn trial completers the functional indices did not change significantly with NAC, whereas most indices deteriorated with placebo; in non-completers the majority of indices worsened but decline was generally less pronounced in most indices with NAC than with placebo. Most categorical analyses of VC, DLco and CPI also showed favourable changes with NAC. The effects of NAC on VC, DLco and CPI were significantly better if the baseline CPI was 50 points or lower.ConclusionThis descriptive analysis confirms and extends the favourable effects of NAC on lung function in IPF and emphasizes the usefulness of VC, DLco, and the CPI for the evaluation of a therapeutic effect. Most importantly, less progressed disease as indicated by a CPI of 50 points or lower at baseline was more responsive to therapy in this study.
| S-EPMC2774307 | biostudies-literature