Dataset Information

Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema.

ABSTRACT:

Background

There is considerable burden of illness in hereditary angioedema (HAE). However, instruments to assess health-related quality of life (HRQoL) in HAE are limited. The Angioedema Quality of Life Questionnaire (AE-QoL) was developed to measure HRQoL in patients with recurrent angioedema; the validity of the AE-QoL in patients with HAE is described.

Methods

To identify disease-related experiences with a focus on the impact of HAE on HRQoL, interviews were conducted with a group of clinician experts and patients with HAE from Canada, France, Germany, Spain, the United Kingdom, and the United States, along with a targeted literature review. Concepts were mapped to the AE-QoL to assess item relevance, interpretation, and conceptual coverage. Cognitive interviews assessed item clarity and relevance. A psychometric validation was performed using data from a phase 3 trial.

Results

Interviews were conducted with seven clinicians and 40 adult patients. Patients reported 35 unique impacts of HAE on their lives, the most frequent being on work/school, social relationships, physical activities, and emotions, particularly fear/worrying and anxiety. Saturation for these impacts was reached, and all concepts covered in the AE-QoL were reported during the interviews. Patients agreed that the questionnaire items and response options were clear and relevant, and the 4-week recall period was appropriate. The psychometric validation included data from 64 patients. For AE-QoL total scores, excellent internal consistency (Cronbach's alpha > 0.90), test-retest reliability (intraclass coefficient > 0.80), convergent validity with the Sheehan Disability Scale (r = 0.663), divergent validity with the EQ-5D-5L index (r = 0.292) and EQ-VAS (r = 0.337), and known-groups validity (p < 0.0001; ɳ2 = 0.56) were demonstrated.

Conclusions

Qualitative and psychometric analyses showed that the AE-QoL is a reliable and valid instrument for measuring HRQoL in adult patients with HAE from six countries.

SUBMITTER: Vanya M

PROVIDER: S-EPMC10070575 | biostudies-literature | 2023 Apr

REPOSITORIES: biostudies-literature

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Publications

Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema.

Vanya Magdalena M Watt Maureen M Shahraz Saeid S Kosmas Charlotte E CE Rhoten Stephanie S Costa-Cabral Sara S Mendivil Joan J Devercelli Giovanna G Weller Karsten K

Journal of patient-reported outcomes 20230403 1

<h4>Background</h4>There is considerable burden of illness in hereditary angioedema (HAE). However, instruments to assess health-related quality of life (HRQoL) in HAE are limited. The Angioedema Quality of Life Questionnaire (AE-QoL) was developed to measure HRQoL in patients with recurrent angioedema; the validity of the AE-QoL in patients with HAE is described.<h4>Methods</h4>To identify disease-related experiences with a focus on the impact of HAE on HRQoL, interviews were conducted with a g ...[more]

PMID: 37012445

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Project description:BackgroundThe impact of recurrent angioedema can be severely debilitating and remains difficult to quantify. Several standardized patient-reported outcome measures (PROMs), including the Angioedema Activity Score (AAS), Angioedema Quality of Life (AE-QoL) questionnaire, and Angioedema Control Test (AECT), have been developed and translated into different languages. However, these PROMs have yet to be validated in Chinese individuals, and their correlations in the Chinese population remain unknown.ObjectiveOur aim was to validate the Chinese versions of the AAS, AE-QoL questionnaire, and AECT and assess their intercorrelations.MethodsA prospective cohort of 118 Chinese patients with recurrent angioedema at the Angioedema and Urticaria Centre of Reference and Excellence in Hong Kong completed the traditional Chinese versions of the AAS, AE-QoL questionnaire, and AECT. We analyzed the reliability and validity of these PROMs and their correlations with each other as well as with generic PROMs.ResultsThe Chinese AAS, AE-QoL questionnaire, and AECT demonstrated excellent internal consistency (Cronbach α = 0.920, 0.976, and 0.832, respectively; McDonald ω = 0.972, 0.977, and 0.901, respectively). Confirmatory factor analysis for the AE-QoL questionnaire showed an acceptable fit with the 4-dimensional model (comparative fit index = 0.869; Tucker-Lewis index = 0.842). The AECT showed significant correlations with both the AAS and AE-QoL questionnaire (ρ = -0.750 and -0.456 respectively [both P < .05]). The AE-QoL questionnaire was moderately correlated with certain domains of generic PROMs such as the Work Productivity and Activity Impairment Questionnaire: General Health, version 2.0, and the Short Form 12-Item Health Survey, version 2 (all ρ < 0.60).ConclusionThe Chinese AE-QoL questionnaire, AAS, and AECT are valid and reliable tools for use with Chinese patients. More validated tools should be made available to improve patient care and research for all patients with angioedema globally.

Project description:BackgroundHereditary angioedema (HAE) is a rare but serious condition characterized by unpredictable and recurrent attacks affecting the skin and mucosa. HAE has wide-ranging impacts on the health-related quality of life (HRQoL) of patients. This study aims to assess the HRQoL of Chinese patients with HAE using the 36-item Short Form Health Survey (SF-36v2) and to explore potential risk factors for low HRQoL.MethodsA total of 104 patients (47 male and 57 female) over age 18 living in China with a known diagnosis of HAE due to C1-INH deficiency completed the SF-36v2 (generic HRQoL questionnaire). The results were compared to Chinese population norms. Subgroup analysis and logistic regression were used to interpret the data.ResultsSF-36v2 showed a significant reduction in all dimensions of HRQoL (p < 0.001) in patients with HAE compared with the general Chinese population. Female patients reported significantly lower bodily pain (BP) (p = 0.039) and physical component scores (PCSs) (p = 0.027) than male patients. Patients with mucosal edema tended to report lower role-physical (RP) limitations (p = 0.031) than patients with only skin edema. There were no differences between the mean scores of the SF-36 in relation to disease subtype, age, disease severity and long-term prophylaxis. Among female patients on long-term prophylaxis, social functioning (SF) (r = - 0.404, p = 0.010), role-emotional (RE) (r = - 0.320, p = 0.044) and mental component scores (MCSs) (r = - 0.313, p = 0.049) were negatively correlated with danazol dosage. A correlation between decreased disease control and decreased HRQoL scores was found, although the correlation was not significant in terms of RE or mental health (MH) scores. The logistic regression model revealed uncontrolled disease to be a risk factor for a low PCS (odds ratio 10.77, 95% confidence interval [CI] 1.78-65.06; p = 0.010) and laryngeal edema to be a risk factor for a low MCS (odds ratio 4.75, 95% CI 1.09-20.69; p = 0.038).ConclusionsChinese HAE patients reported significantly lower HRQoL scores than the general population. Unsatisfactory disease control is a risk factor for decreased PCSs. Laryngeal edema is a risk factor for decreased MCSs.

Project description:BackgroundQuality of life (QoL) is considered a key treatment outcome in bipolar disorder (BD) across research, clinical, and self-management contexts. Web-based assessment of patient-reported outcomes offer numerous pragmatic benefits but require validation to ensure measurement equivalency. A web-based version of the Quality of Life in Bipolar Disorder (QoL.BD) questionnaire was developed (QoL Tool).ObjectiveThis study aimed to evaluate the psychometric properties of a web-based QoL self-report questionnaire for BD (QoL Tool). Key aims were to (1) characterize the QoL of the sample using the QoL Tool, (2) evaluate the internal consistency of the web-based measure, and (3) determine whether the factor structure of the original version of the QoL.BD instrument was replicated in the web-based instrument.MethodsCommunity-based participatory research methods were used to inform the development of a web-based adaptation of the QoL.BD instrument. Individuals with BD who registered for an account with the QoL Tool were able to opt in to sharing their data for research purposes. The distribution of scores and internal consistency estimates, as indicated by Cronbach alpha, were inspected. An exploratory factor analysis using maximum likelihood and oblique rotation was conducted. Inspection of the scree plot, eigenvalues, and minimum average partial correlation were used to determine the optimal factor structure to extract.ResultsA total of 498 people with BD (349/498, 70.1% female; mean age 39.64, SD 12.54 years; 181/498, 36.3% BD type I; 195/498, 39.2% BD type II) consented to sharing their QoL Tool data for the present study. Mean scores across the 14 QoL Tool domains were, in general, significantly lower than that of the original QoL.BD validation sample. Reliability estimates for QoL Tool domains were comparable with that observed for the QoL.BD instrument (Cronbach alpha=.70-.93). Exploratory factor analysis supported the extraction of an 11-factor model, with item loadings consistent with the factor structure suggested by the original study. Findings for the sleep and physical domains differed from the original study, with this analysis suggesting one shared latent construct.ConclusionsThe psychometric properties of the web-based QoL Tool are largely concordant with the original pen-and-paper QoL.BD, although some minor differences in the structure of the sleep and physical domains were observed. Despite this small variation from the factor structure identified in the QoL.BD instrument, the latent factor structure of the QoL Tool largely reproduced the original findings and theoretical structure of QoL areas relevant to people with BD. These findings underscore the research and clinical utility of this instrument, but further comparison of the psychometric properties of the QoL Tool relative to the QoL.BD instrument is warranted. Future adaptations of the QoL Tool, including the production of an app-based version of the QoL Tool, are also discussed.

Project description:BackgroundTo evaluate the psychometric characteristics of the 1-month recall Uterine Fibroid Symptom and Health-Related Quality of Life questionnaire (UFS-QOL), including the Revised Activities subscale.MethodsVENUS I and II were phase III, randomized, double-blind, placebo-controlled trials of ulipristal acetate in women with uterine fibroids (UF) and abnormal uterine bleeding. Women completed the 1-month recall UFS-QOL at baseline and after 12 weeks' treatment. Uterine bleeding was assessed via a daily diary (both studies); the Patient Global Impression of Improvement scale (PGI-I) was completed in VENUS II. Psychometric analyses examined internal consistency reliability and construct validity of the UFS-QOL; confirmatory factor analysis (CFA) compared model fit of the original and Revised Activities subscales. Analyses were conducted separately for VENUS I and II.ResultsOne hundred and fifty-seven patients in VENUS I and 429 in VENUS II were included. Changes in mean Symptom Severity and health-related quality of life (HRQoL) scale scores indicated symptom burden reductions and HRQoL improvements. Cronbach's alpha coefficients were high at baseline and after 12 weeks' treatment (all ≥0.76, meeting the >0.70 threshold), demonstrating strong internal consistency reliability. Correlations between UFS-QOL scores and bleeding diary responses (range: -0.35 to -0.63), and UFS-QOL scores and PGI-I responses (range: -0.48 to -0.70), ranged from moderate to strong after 12 weeks' treatment (all p < 0.0001). Patients with absence of bleeding or controlled bleeding after 12 weeks' treatment scored significantly better (p < 0.001) on each UFS-QOL scale than patients not achieving those end points, supporting construct validity. CFA confirmed model fit for the Revised Activities subscale.ConclusionsThe 1-month recall UFS-QOL, including the Revised Activities subscale, is a valid, reliable measure to assess UF symptoms and their impact on HRQoL.Trial registrationClinicalTrials.gov, NCT02147197 . Registered May 26, 2014; retrospectively registered. ClinicalTrials.gov, NCT02147158 . Registered May 26, 2014; retrospectively registered.

Project description:BackgroundSelf-reported measures are relevant both for the clinic and for health evaluation because they provide an interpretation of quality parameters. Women who experience labour can express themselves through these measures, identifying indicators that need improvement.ObjectiveThe objective of this study is to adapt the Childbirth Experience Questionnaire to the Portuguese context and to determine its psychometric properties.MethodA methodological study carried out with a convenience sample where the participants were 161 female users of a hospital in southern Portugal. They were aged between 20 and 43 years (M = 31.05, SD = 4.87) and answered a questionnaire approximately 48 h postpartum, preserving the ethical principles. The original instrument, with 22 items, underwent the linguistic and cultural adequacy process.ResultsFactor analysis with Varimax rotation was performed, revealing a set of 19 items with factor weights above .400. The set of items remained four-dimensional as the original, explaining 62.517% of the variance. In the retest, the reliability results showed that similar characteristics to the original study are maintained in the two subscales that express 'Participation' (three items) and 'Professional Support' (four items), with internal consistency values of .807 and .782. The 'Own Performance' and 'Own Threshold' subscales were elaborated from the results of the Varimax rotation, presenting Cronbach's alpha coefficients of .840 and 714, respectively. The total scale showed alpha values of .873 and .823 in the test and retest, respectively. Time stability showed a positive association, with r = .659 (p < .001). Accuracy through the split-half method reached an alpha value of .880 with Spearman-Brown correction. The floor effect was high in the 'Participation' subscale, both in the test and in the retest. Convergent validity between the instrument and the 'Index of Strategies for Pain Relief in Labour' discrete variable showed a Spearman's rho value of .209 (p = .011) in the total scale. In discriminating validity, the Mann-Whitney test reveals that the women who recognize interactions with the midwife have more favourable scores in Childbirth Experience Questionnaire (U = 2748.000; Z = 2.905; p = .004).ConclusionThe current version in European Portuguese suggests that it is a valid and reliable measure. This study may facilitate other validation processes in Lusophony countries.

Project description:RationaleKallikrein-bradykinin-forming cascade is known to cause hereditary angioedema (HAE) acute angioedema (AE) attacks. Further research of HAE attacks is needed to explain disease heterogeneity, predict treatment response and identify biomarkers for monitoring HAE attacks. Differential expression of the microvascular endothelial cell-surface receptors for example, g-C1qR, cytokeratin-1, and plasminogen-activator-urokinase-receptor (PLAUR) were hypothesized as biomarkers of AE attacks.MethodTo understand HAE attacks, the differentially expressed genes (DEGs) in RNAseq and mi-RNAseq data of total RNA extracted from skin biopsies of lesional versus non-lesional skin collected during and between attacks in Type-1 HAE patients (n = 11; F:M = 8:3) were compared. To understand the HAE variants, DEGs in skin biopsies from HAE with normal C1 inhibitor (n = 5, F:M = 5:0), and non-HAE (n = 7; F:M = 3:4) patients were compared. Gene-set enrichment analyses and regulator effects analysis of these DEGs identified biological pathways in HAE attacks and their regulators.ResultsPLAUR gene, encoding urokinase-type plasminogen activator (u-PAR), was constitutively over-expressed in HAE-Type-1 versus non-HAE controls suggestive of overactive u-PAR-mediated signaling via binding to Factor-XII. Baseline PLAUR expression was associated with severe AE (p = 0.05). The 18 significant DEGs investigated between baseline and AE attack samples in Type1-HAE were enriched in beta1/beta3-integrin cell surface interactions and IL-6-mediated signaling. Regulator effects analysis suggests a role for IL-1b in HAE flares. AKT2, the mRNA regulated by the differentially-expressed miR-184A, was also associated with HAE attacks.ConclusionAngiopoetin-activated β1-integrin signaling pathways causing endothelial destabilization, and avid binding of factor XII to u-PAR are possible novel mechanisms for progression of the endothelial kinin-bradykinin-forming cascade in HAE attacks.

Dataset Information

Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema.

Background

Methods

Results

Conclusions

Publications

Content validation and psychometric evaluation of the Angioedema Quality of Life Questionnaire for hereditary angioedema.

Similar Datasets

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