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Validation of the Investigator Global Assessment of Chronic Hand Eczema (IGA-CHE): a new clinician reported outcome measure of CHE severity.

ABSTRACT: The Investigator Global Assessment of Chronic Hand Eczema (IGA-CHE) is a novel Clinician-Reported Outcome measure that allows investigators to assess cross-sectional CHE global disease severity using clinical characteristics of erythema, scaling, lichenification/hyperkeratosis, vesiculation, oedema, and fissures as guidelines for overall severity assessment. This study aimed to evaluate the psychometric properties of the IGA-CHE for use as an outcome measure in CHE clinical trials and clinical practice. Psychometric analyses were performed using data from a sample of 280 patients with moderate to severe CHE from a phase 3 trial of delgocitinib cream, pooled across treatment groups. Test-retest reliability results were moderate to strong with kappa coefficients ranging from 0.63 to 0.76. Correlations with measures assessing related concepts were moderate or strong (range 0.65-0.72) and exceeded a priori hypotheses, providing evidence of convergent validity. Known-groups validity was supported by statistically significant differences between severity groups (< 0.001). Within-group effect sizes were consistently larger for improved groups compared to stable groups, providing evidence of ability to detect change. Anchor-based analyses generated within-subject meaningful change estimates ranging from - 0.8 to - 2.3. A correlation weighted average suggested a single value of - 1.7 in change from baseline. These findings provide evidence the IGA-CHE scale has strong reliability, construct validity, and ability to detect change, supporting its use as an endpoint in CHE clinical trials and clinical practice. Based on the evidence, 2-level changes in IGA-CHE score are considered a conservative meaningful change threshold; however, findings also indicate 1-level change in IGA-CHE scores reflects a clinically meaningful improvement for patients.Clinical trial registration: NCT04871711.

SUBMITTER: Silverberg JI

PROVIDER: S-EPMC10955004 | biostudies-literature | 2024 Mar

REPOSITORIES: biostudies-literature

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Validation of the Investigator Global Assessment of Chronic Hand Eczema (IGA-CHE): a new clinician reported outcome measure of CHE severity.

Silverberg Jonathan I JI Agner Tove T Baranowski Keith K Plohberger Ursula U Thoning Henrik H Arbuckle Rob R Grant Laura L Skingley George G Bissonnette Robert R

Archives of dermatological research 20240320 4

The Investigator Global Assessment of Chronic Hand Eczema (IGA-CHE) is a novel Clinician-Reported Outcome measure that allows investigators to assess cross-sectional CHE global disease severity using clinical characteristics of erythema, scaling, lichenification/hyperkeratosis, vesiculation, oedema, and fissures as guidelines for overall severity assessment. This study aimed to evaluate the psychometric properties of the IGA-CHE for use as an outcome measure in CHE clinical trials and clinical p ...[more]

PMID: 38507100

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Project description:IntroductionChronic Hand Eczema (CHE) is an inflammatory skin disease of the hands. The Hand Eczema Symptom Diary (HESD) is a new patient-reported outcome measure of worst severity of core CHE signs/symptoms. This study aimed to evaluate content and psychometric validity of the HESD.MethodsThe HESD was developed based on the literature and concept elicitation interviews. Qualitative cognitive debriefing interviews were conducted with CHE patients to assess relevance and understanding of items, response options and recall period. Psychometric properties of the HESD (item performance, dimensionality, reliability, validity, responsiveness and estimation of meaningful change thresholds) were then assessed, first using data from a phase 2b trial (NCT03683719), and confirmed using data from the first 280 participants completing the 16-week treatment phase of a phase 3 trial (NCT04871711).ResultsCognitive debriefing supported item refinement and removal of items and confirmed all items were well understood and relevant to patients. Item properties and dimensionality analyses in the phase 2b data supported removal of additional items, resulting in the 6-item HESD included in the phase 3 trial. Unidimensionality was supported by inter-item correlations (all > 0.70) and Rasch analysis. Internal consistency (Cronbach's alpha = 0.96) and test-retest reliability (Intraclass Correlation Coefficient > 0.89) results were very strong. Construct validity was supported by moderate correlations with concurrent measures (0.53-0.64) and significant differences between severity groups (p < 0.001). Large effect sizes for mean change scores in participants that improved and significant differences between change groups indicated the ability to detect change. Anchor-based analyses supported within-individual responder definitions of ≥ 4-points for improvements in 7-day average HESD scores.ConclusionThe HESD is the first CHE-specific, patient-reported outcome measure of CHE signs/symptoms developed and validated in line with regulatory guidance. This article provides evidence of strong content validity and psychometric validity and shows improvements of ≥ 4 points on 7-day average HESD scores represent clinically meaningful, important changes.Trial registrationNCT03683719, NCT04871711.

Project description:IntroductionChronic Hand Eczema (CHE) is an inflammatory skin disease that causes significant impact on health-related quality of life (HRQoL). The Hand Eczema Impact Scale (HEIS) is a new patient-reported outcome (PRO) measure designed to assess the impact of CHE on key domains of HRQoL. This study aimed to develop and evaluate content and psychometric validity of the HEIS.MethodsThe HEIS was initially developed on the basis of a literature review and concept elicitation interviews. Qualitative cognitive debriefing interviews (n = 20) were conducted with patients with CHE to assess relevance and understanding of items, response options, and recall period. Psychometric properties (item performance, dimensionality, reliability, validity, responsiveness, and estimation of meaningful change thresholds) were then assessed using data (n = 258) from a phase 2b trial (NCT03683719).ResultsCognitive debriefing confirmed all items were understood and relevant to patients. Inter-item correlations (all > 0.50) and confirmatory factor analysis (factor loadings ≥ 0.80) supported unidimensionality of the HEIS score, and mostly provided support for the HEIS Proximal Daily Activity Limitations (PDAL) score, with only one item loading below the prespecified threshold. Item properties and previous qualitative work supported retaining this item in the total score but removed from the HEIS PDAL domain. Internal consistency (Cronbach's alpha ≥ 0.89) and test-retest reliability (intra-class correlation coefficient ≥ 0.79) results were very strong. Strong correlations with concurrent measures (0.66-0.87) and significant differences between severity groups (p < 0.001) supported construct validity. Large effect sizes for mean change scores in participants that improved and significant differences between groups indicated ability to detect change. Anchor-based analyses supported within-individual responder definitions of ≥ 1.3 points for improvements in both HEIS score and HEIS PDAL score (covering three items) and of ≥ 1.5 points for HEIS embarrassment with the appearance of hands (Emb) score (covering two items).ConclusionsThe 9-item HEIS is the first CHE-specific PRO measure developed and validated according to regulatory guidance for assessment of the impact of CHE on key domains of HRQoL. This article provides evidence of strong content and psychometric validity and shows improvements of ≥ 1.3 points in HEIS score and HEIS PDAL score, and improvements of ≥ 1.5 points in HEIS Emb score represent clinically meaningful, important changes.Trial registrationNCT03683719.

Project description:Patient-reported outcome measures (PROMs) provide an important complement to physician-assessed clinical outcome measures in dermatologic diseases such as atopic dermatitis (AD) and chronic hand eczema (CHE). AD and CHE are chronic and relapsing inflammatory skin conditions that often co-occur. While both diseases result in various signs and symptoms that are burdensome and can negatively affect patients' lives, there may be distinct differences in the signs, symptoms, burden, and health-related quality of life (HRQOL) impact of these diseases. The objective of this study was to identify and evaluate PROMs used in studies of AD and CHE. The aim was to explore the assessment of key symptoms and impacts, and identify any gaps in the measures in use. A structured review of the PubMed database was conducted to identify PROMs used or developed for use in AD or CHE. The Dermatology Life Quality Index (DLQI), the Pruritus/Itch Numeric Rating Scale (NRS), the Patient-Oriented Eczema Measure (POEM), and the Quality of Life in Hand Eczema Questionnaire (QOLHEQ) were identified and reviewed in detail. With these measures, the AD and CHE symptoms and impacts most commonly evaluated in the literature include dermatology-related HRQOL in the domains of symptoms and feelings, daily activities, leisure, work and school, personal relationships, and adverse effects; pruritus; sleep disturbance; AD-specific symptoms (dryness, itching, flaking, cracking, bleeding, and weeping/oozing); and CHE-specific symptoms (pain, itch, fissuring, redness, bleeding, and dryness). A review of regulatory labels of drugs approved for AD by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) found that, among the four measures reviewed, the Pruritus NRS was included in the FDA and EMA labels for dupilumab, the DLQI was included in the EMA labels for dupilumab and tacrolimus, and the POEM was included in the EMA label for dupilumab. Key symptoms of AD (e.g. itching, flaking, cracking) and CHE (e.g. pain, itching, fissuring) are increasingly being assessed with PROMs; however, primary endpoints in clinical trials are often based on clinician-reported outcome measures. As therapeutic strategies in dermatology are targeted at specific dermatologic symptoms and diseases affecting specific sites (e.g. CHE), future research should explore patients' experiences with these symptoms and sites and the changes with treatment that are most meaningful to them.

Project description:ImportanceDespite the importance of patient trust in health care, there are no patient-reported outcome measures (PROMs) for trust in their clinician that have been developed empirically in Spanish, which is the second most common language in the US.ObjectiveTo develop and validate a Spanish-language PROM for trust in pregnancy care clinician.Design, setting, and participantsThis cross-sectional study used a national online panel of patients who reported a Spanish language preference and had limited English proficiency and were currently pregnant or had given birth within the 12 months before the survey. Participants resided in the United States, and data were collected from January to May 2024.ExposuresParticipants had clinical interactions during pregnancy and/or postpartum care. Data collected included demographics, Confianza (Trust) Scale candidate items, and 4 measures for concurrent validity evidence: the Trust in Physician Scale (TPS), the Mothers on Respect Index, the Edinburgh Postpartum Depression Scale (EPDS), and the Patient-Reported Outcomes Measurement Information System Global 10.Main outcomes and measuresThe main outcomes were psychometric properties of the Confianza scale and its association with validated scales (validity coefficients). Item response theory (IRT) analyses were conducted to evaluate the psychometric properties of the candidate items, select the best item subset for the Confianza scale, examine its correlation with other measures, and compare scores according to demographic characteristics.ResultsOf the included 204 participants (mean [SD] age, 26 [7] years; 62 participants from South America [30%]; 32 participants from Mexico [16%]), 117 participants were pregnant (57%), and 87 were within 1-year post partum (43%) at the time of survey completion. Four items were removed based on exploratory factor analysis. Using results from IRT analysis on the remaining 12 items, 5 items were selected to represent communication, caring, competency, accompaniment, and overall trust for the final measure. The 5-item Confianza scale had high measurement precision, with reliability above 0.90 across a wide range of the trust continuum. The Confianza scale (mean [SD] score, 21.5 [4.6] out of 25) was positively correlated with the TPS (r = 0.47; 95% CI, 0.36 to 0.57; P < .001) and negatively correlated with the EPDS (r = -0.41; 95% CI, -0.52 to -0.29; P <.001). Higher trust scores were obtained when there was language concordance with clinicians (mean [SD], 23.6 [2.3] vs 20.0 [5.3]; P < .001) and care continuity (mean [SD], 22.3 [3.8] vs 20.9 [5.3]; P = .001).Conclusions and relevanceIn this cross-sectional study of pregnant and postpartum Spanish-speaking individuals, a Spanish-language PROM for trust in pregnancy care clinician had initial validity.

Project description:BackgroundThe Hand Eczema Severity Index (HECSI) is used to measure the severity of hand eczema. It is unclear what HECSI scores mean and what is the minimally important change (MIC). Furthermore, its responsiveness has not been studied.ObjectivesTo study the responsiveness and interpretability of the HECSI.MethodsThis was a prospective study covering two time points: baseline and after 4-12 weeks. Responsiveness was assessed using a criterion approach, with at least one-step improvement on the 'Photographic guide for severity of hand eczema' as the anchor for important improvement. Interpretability of single scores was determined by defining severity bands based on agreement with the anchor. For change scores, the smallest detectable change (SDC) was calculated in patients indicating no change and the MIC was obtained in patients indicating that they had changed using three methods: mean cutoff, receiver operating characteristic (ROC) curve and 95% limit.ResultsIn total, 294 patients participated (160 male, mean age 44·9 years). HECSI scores improved or deteriorated in parallel with the anchor. The area under the ROC curve was 0·86 (95% confidence interval 0·81-0·91). The final severity band for single scores had a κ-coefficient of agreement of 0·694: clear, 0; almost clear, 1-16; moderate, 17-37; severe, 38-116; very severe, ≥ 117. The SDC in 93 unchanged patients was 40·3 points. The obtained MIC values were all smaller than the SDC.ConclusionsThe HECSI has good responsiveness. This study gives meaning to HECSI scores, which can be applied to clinical decision making and the design of clinical trials. We recommend that an improvement of 41 points on the HECSI is regarded as the minimally measurable true change. What's already known about this topic? The Hand Eczema Severity Index (HECSI) is one of the most widely used measurement instruments to measure the severity of hand eczema. The HECSI has good reliability, but its responsiveness and interpretability have not been studied. What does this study add? This study shows good responsiveness of the HECSI. A severity grading for single scores is proposed. The smallest detectable change and minimally important change for improvement are determined. What are the clinical implications of this work? The obtained values help to interpret HECSI scores in daily practice and clinical studies, and may facilitate researchers in the calculation of sample sizes for clinical trials.

Dataset Information

Validation of the Investigator Global Assessment of Chronic Hand Eczema (IGA-CHE): a new clinician reported outcome measure of CHE severity.

Publications

Validation of the Investigator Global Assessment of Chronic Hand Eczema (IGA-CHE): a new clinician reported outcome measure of CHE severity.

Similar Datasets

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