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      Responder Threshold for Patient-Oriented Eczema Measure (POEM) and Children’s Dermatology Life Quality Index (CDLQI) in Adolescents with Atopic Dermatitis

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          Abstract

          Introduction

          The Patient-Oriented Eczema Measure (POEM) assesses patient-reported frequency of atopic dermatitis (AD) symptoms, while the Children’s Dermatology Life Quality Index (CDLQI) measures the impact of skin disease on health-related quality of life (HRQoL) in children. There is currently no threshold for clinically meaningful within-person change in POEM or CDLQI scores in adolescents. Here we empirically derive within-person thresholds of meaningful within-person change in POEM and CDLQI scores in adolescents with moderate-to-severe AD.

          Methods

          Data were used from a phase 3, randomized, double-blind, placebo-controlled trial of dupilumab in adolescents (aged ≥ 12 to < 18 years) with moderate-to-severe AD. Anchor-based methods were employed using the mean change in POEM and CDLQI scores from baseline to week 16 linked with a 1-point improvement in Patient Global Assessment of Disease (PGAD), a score of “a little better” on the Patient Global Assessment of Treatment effect (PGAT), a 50–74% improvement from baseline in the Eczema Area and Severity Index (EASI-50–74), and a 1-point improvement in Investigator’s Global Assessment (IGA) score.

          Results

          A mean change of − 7.8 and − 5.6 in the POEM score was associated with PGAD and PGAT anchors, respectively. EASI-50–74 was associated with a mean change in POEM score of − 8.2, while the IGA anchor was associated with a mean change of − 7.9 in POEM score. The mean changes in CDLQI score associated with PGAD and PGAT anchors were − 6.4 and − 6.6, respectively, while CDLQI mean scores changed by − 8.3 and − 8.0 for the EASI and IGA anchors, respectively.

          Conclusion

          In adolescents (aged ≥ 12 to < 18 years) with moderate-to-severe AD, a within-person change of 6–8 points in POEM and CDLQI scores, independently, can be considered a reasonable responder threshold for clinically meaningful change in each of the two scales, respectively.

          Trial Registration

          ClinicalTrials.gov Identifier: NCT03054428.

          Funding

          Sanofi and Regeneron Pharmaceuticals, Inc.

          Electronic supplementary material

          The online version of this article (10.1007/s13555-019-00333-2) contains supplementary material, which is available to authorized users.

          Related collections

          Most cited references9

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          The patient-oriented eczema measure: development and initial validation of a new tool for measuring atopic eczema severity from the patients' perspective.

          To develop a simple, valid, repeatable, and readily understandable patient-oriented assessment measure for monitoring disease activity in children and adults with atopic eczema. Qualitative semistructured patient interviews identified a list of symptoms of atopic eczema. These symptoms were quantitatively analyzed in a larger patient population to identify which symptoms were important to patients and amenable to monitoring as part of a scoring system. The outpatient Department of Dermatology at the Queen's Medical Centre, University Hospital, Nottingham, England, and 5 local general practices. Four hundred thirty-five patients with atopic eczema. Seven symptoms were incorporated into the final patient-oriented eczema measure using a simple 5-point scale of frequency of occurrence during the previous week, with a maximum total score of 28. Validity testing against the Dermatology Life Quality Index, Children's Dermatology Life Quality Index, and patients' global severity assessments showed good correlation (r = 0.78, r = 0.73, and r = 0.81, respectively; P<.001). Internal consistency was high (Cronbach alpha = 0.88), and test-retest reliability was good, with 95% of scores falling within 2.6 points on repeat testing (mean score difference, 0.04; SD, 1.32). Individual variables in the measure demonstrated sensitivity to change during a 4-week in-clinic period and an 18-week randomized controlled clinical trial. The patient-oriented eczema measure is a practical self-assessed measurement tool for monitoring aspects of atopic eczema that are important to patients in routine clinical practice or in the clinical trial setting.
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            Determining the Minimal Clinically Important Difference and Responsiveness of the Dermatology Life Quality Index (DLQI): Further Data

            Aims: To determine the minimal clinically important difference (MCID) of the Dermatology Life Quality Index (DLQI) and its responsiveness to change in inflammatory skin diseases. Methods: A longitudinal study: at stage 1, patients completed the DLQI and a disease severity global question; at stage 2, a global rating of change in quality of life (QoL; Global Rating of Change Questionnaire, GRCQ) was added and used as an anchor to measure the MCID of the DLQI. Results: 192 patients completed stage 1 and 107 completed stage 2. The mean DLQI score at stage 1 was 9.8 and 7.4 at stage 2 with a mean change of 2.4 (p < 0.0001). 31 patients experienced a ‘small change' in their QoL (±3 and ±2) on the GRCQ. The mean corresponding change in DLQI scores was 3.3, which is regarded as the approximate MCID. Conclusions: Previous estimates of the MCID of the DLQI have varied from 3 to 5. Although this study demonstrated a MCID of 3.3, we recommend that the MCID in inflammatory skin diseases should be 4.
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              The Children's Dermatology Life Quality Index (CDLQI): initial validation and practical use

              Skin disease can cause severe disability and handicap in children. Measurement of the impact of skin disease on the quality of life is required to aid clinical decision-making, for clinical research, for audit of paediatric dermatology services, and for political reasons, to aid arguments for more resources for the care of children with skin disease. Adult measures are inappropriate, as the lives of children differ markedly from those of adults. The purpose of this study was to create and initially validate a simple practical questionnaire for use in children. One hundred and sixty-nine children, aged 3-16 years, attending a paediatric dermatology clinic, wrote down, with the help of their parents, all the ways in which their skin disease affected their lives. One hundred and eleven different aspects were identified; 10 questions were composed to cover these aspects, using a structure similar to the Adult Dermatology Life Quality Index. This draft questionnaire was piloted on two series, totalling 40 children, and minor alterations were made to improve clarity. The Children's Dermatology Life Quality Index (CDLQI) questionnaire (maximum score 30) was then given to a further 233 dermatology paediatric out-patients (CDLQI mean = 5.1, SD = 4.9), 47 normal controls (mean 0.4, 0.7) and 55 control patients attending a general paediatric clinic (mean 0.7, 2.5). The CDLQI scores for eczema (mean = 7.7, 5.6, n = 47), psoriasis (5.4, 5.0, n = 25) and acne (5.7, 4.4, n = 40), were all highly significantly greater than for moles and naevi (2.3, 2.9, n = 29). The highest mean score was that for scabies (mean = 9.5, 10.5, n = 6).(ABSTRACT TRUNCATED AT 250 WORDS)
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                Author and article information

                Contributors
                simpsone@ohsu.edu
                Journal
                Dermatol Ther (Heidelb)
                Dermatol Ther (Heidelb)
                Dermatology and Therapy
                Springer Healthcare (Cheshire )
                2193-8210
                2190-9172
                22 October 2019
                22 October 2019
                December 2019
                : 9
                : 4
                : 799-805
                Affiliations
                [1 ]GRID grid.5288.7, ISNI 0000 0000 9758 5690, Department of Dermatology, , Oregon Health & Science University, ; Portland, OR USA
                [2 ]GRID grid.7692.a, ISNI 0000000090126352, University Medical Center Utrecht, ; Utrecht, The Netherlands
                [3 ]GRID grid.417924.d, Sanofi, ; Chilly-Mazarin, France
                [4 ]GRID grid.416262.5, ISNI 0000 0004 0629 621X, RTI Health Solutions, ; Manchester, UK
                [5 ]GRID grid.417924.d, Sanofi, ; Lyon, France
                [6 ]GRID grid.476716.5, ISNI 0000 0004 0407 5050, Sanofi, ; Guildford, UK
                [7 ]GRID grid.418961.3, ISNI 0000 0004 0472 2713, Regeneron Pharmaceuticals, Inc., ; Tarrytown, NY USA
                [8 ]GRID grid.62562.35, ISNI 0000000100301493, RTI Health Solutions, ; Durham, NC USA
                Article
                333
                10.1007/s13555-019-00333-2
                6828922
                31641952
                b955de7f-99a4-4d60-a67f-4f5cf47dc2f7
                © The Author(s) 2019
                History
                : 15 August 2019
                Funding
                Funded by: FundRef http://dx.doi.org/10.13039/100004339, Sanofi;
                Funded by: FundRef http://dx.doi.org/10.13039/100009857, Regeneron Pharmaceuticals;
                Categories
                Brief Report
                Custom metadata
                © The Author(s) 2019

                Dermatology
                adolescents,atopic dermatitis,cdlqi,dupilumab,poem,quality of life,responder threshold,validation
                Dermatology
                adolescents, atopic dermatitis, cdlqi, dupilumab, poem, quality of life, responder threshold, validation

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