A comparison study of outcome measures for epidermolysis bullosa: Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) and the Instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa (iscorEB).

BEBS, Birmingham Epidermolysis Bullosa Severity Score BMD, bone mineral densitometry DDEB, dominant dystrophic epidermolysis bullosa EB, epidermolysis bullosa EBDASI, Epidermolysis Bullosa Disease Activity and Scarring Index EBS, epidermolysis bullosa simplex Epidermolysis Bullosa Disease Activity and Scarring Index ICC, intraclass correlation coefficient Instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa JEB, junctional epidermolysis bullosa QOLEB, Quality of Life in Epidermolysis Bullosa score QoL, quality of life RDEB, recessive dystrophic epidermolysis bullosa blistering skin disease dermatology epidermolysis bullosa iscorEB, Instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa outcome measure

Journal

JAAD international
ISSN: 2666-3287
Titre abrégé: JAAD Int
Pays: United States
ID NLM: 101774762

Informations de publication

Date de publication:
Mar 2021
Historique:
accepted: 03 12 2020
entrez: 19 8 2021
pubmed: 20 8 2021
medline: 20 8 2021
Statut: epublish

Résumé

The success of clinical trials in Epidermolysis Bullosa (EB) is dependent upon the availability of a valid and reliable scoring tool that can accurately assess and monitor disease severity. The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) and Instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa (iscorEB) were independently developed and validated against the Birmingham Epidermolysis Bullosa Severity Score but have never been directly compared. To compare the reliability, convergent validity, and discriminant validity of the EBDASI and iscorEB scoring tools. An observational cohort study was conducted in 15 patients with EB. Each patient was evaluated using the EBDASI and iscorEB-clinician scoring tools by 6 dermatologists with expertise in EB. Quality of life was assessed using the iscorEB-patient and Quality of Life in EB measures. The intraclass correlation coefficients for interrater reliability were 0.942 for the EBDASI and 0.852 for the iscorEB-clinician. The intraclass correlation coefficients for intrarater reliability was 0.99 for both scores. The two tools demonstrated strong convergent validity with each other. Both scoring tools demonstrate excellent reliability. The EBDASI appears to better discriminate between EB types and disease severities.

Sections du résumé

BACKGROUND BACKGROUND
The success of clinical trials in Epidermolysis Bullosa (EB) is dependent upon the availability of a valid and reliable scoring tool that can accurately assess and monitor disease severity. The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI) and Instrument for Scoring Clinical Outcomes of Research for Epidermolysis Bullosa (iscorEB) were independently developed and validated against the Birmingham Epidermolysis Bullosa Severity Score but have never been directly compared.
OBJECTIVE OBJECTIVE
To compare the reliability, convergent validity, and discriminant validity of the EBDASI and iscorEB scoring tools.
METHODS METHODS
An observational cohort study was conducted in 15 patients with EB. Each patient was evaluated using the EBDASI and iscorEB-clinician scoring tools by 6 dermatologists with expertise in EB. Quality of life was assessed using the iscorEB-patient and Quality of Life in EB measures.
RESULTS RESULTS
The intraclass correlation coefficients for interrater reliability were 0.942 for the EBDASI and 0.852 for the iscorEB-clinician. The intraclass correlation coefficients for intrarater reliability was 0.99 for both scores. The two tools demonstrated strong convergent validity with each other.
CONCLUSION CONCLUSIONS
Both scoring tools demonstrate excellent reliability. The EBDASI appears to better discriminate between EB types and disease severities.

Identifiants

pubmed: 34409361
doi: 10.1016/j.jdin.2020.12.007
pii: S2666-3287(20)30084-5
pmc: PMC8362226
doi:

Types de publication

Journal Article

Langues

eng

Pagination

134-152

Informations de copyright

© 2020 Published by Elsevier on behalf of the American Academy of Dermatology, Inc.

Déclaration de conflit d'intérêts

Dr Dedee F. Murrell developed the EBDASI. Dr Dedee F. Murrell and Authors Daniel and Su were involved in the validation of the EBDASI. Dr Kern and authors Rogers, Gibson, Martin, Robertson, Feng, and Oliver G. C. Murrell have no conflicts of interest to declare.

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Auteurs

Clare L Rogers (CL)

Faculty of Medicine, University of New South Wales, Sydney, New South Wales, Australia.
Department of Dermatology, St George Hospital, Sydney, New South Wales, Australia.
Premier Specialists, Dermatology Trials Australia, Kogarah, New South Wales, Australia.

Matthew Gibson (M)

Faculty of Medicine, University of New South Wales, Sydney, New South Wales, Australia.
Department of Dermatology, St George Hospital, Sydney, New South Wales, Australia.
Premier Specialists, Dermatology Trials Australia, Kogarah, New South Wales, Australia.

Johannes S Kern (JS)

Department of Dermatology, The Royal Melbourne Hospital, Parkville, Victoria, Australia.
Faculty of Medicine, Dentistry and Health Sciences, The University of Melbourne, Melbourne, Victoria, Australia.

Linda K Martin (LK)

Sydney Children's Hospital, Randwick, New South Wales, Australia.

Susan J Robertson (SJ)

Department of Dermatology, The Royal Melbourne Hospital, Parkville, Victoria, Australia.
Department of Dermatology, The Royal Children's Hospital, Melbourne, Victoria, Australia.
Murdoch Children's Research Institute, Melbourne, Victoria, Australia.
Department of Dermatology, Monash Health, Melbourne, Victoria, Australia.

Benjamin S Daniel (BS)

Faculty of Medicine, University of New South Wales, Sydney, New South Wales, Australia.
Department of Dermatology, St George Hospital, Sydney, New South Wales, Australia.

John C Su (JC)

Department of Dermatology, The Royal Children's Hospital, Melbourne, Victoria, Australia.
Murdoch Children's Research Institute, Melbourne, Victoria, Australia.
Department of Dermatology, Monash Health, Melbourne, Victoria, Australia.
Department of Pediatrics, The University of Melbourne, Melbourne, Victoria, Australia.
Department of Medicine, Monash University, Melbourne, Victoria, Australia.

Oliver G C Murrell (OGC)

Premier Specialists, Dermatology Trials Australia, Kogarah, New South Wales, Australia.

Grant Feng (G)

Faculty of Medicine, University of New South Wales, Sydney, New South Wales, Australia.

Dedee F Murrell (DF)

Faculty of Medicine, University of New South Wales, Sydney, New South Wales, Australia.
Department of Dermatology, St George Hospital, Sydney, New South Wales, Australia.
Premier Specialists, Dermatology Trials Australia, Kogarah, New South Wales, Australia.

Classifications MeSH