Clement C H Loh1, Jaehwan Kim1, John C Su2, Benjamin S Daniel3, Supriya S Venugopal4, Lesley M Rhodes5, Lizbeth R A Intong1, Matthew G Law6, Dedee F Murrell7. 1. Department of Dermatology, St George Hospital, Sydney, Australia; Faculty of Medicine, University of New South Wales, Sydney, Australia. 2. Epidermolysis Bullosa Clinic, Royal Children's Hospital, Melbourne, Australia; Department of Pediatrics, University of Melbourne, Melbourne, Australia; Department of Medicine, Monash University, Melbourne, Australia. 3. Department of Medicine, St Vincent's Hospital, Sydney, Australia; Faculty of Medicine, University of New South Wales, Sydney, Australia. 4. Department of Dermatology, Westmead Hospital, Sydney, Australia; Faculty of Medicine, University of New South Wales, Sydney, Australia. 5. Department of Dermatology, St George Hospital, Sydney, Australia. 6. Kirby Institute, University of New South Wales, Sydney, Australia; Faculty of Medicine, University of New South Wales, Sydney, Australia. 7. Department of Dermatology, St George Hospital, Sydney, Australia; Faculty of Medicine, University of New South Wales, Sydney, Australia. Electronic address: d.murrell@unsw.edu.au.
Abstract
BACKGROUND: There is a lack of validated standardized outcome measures for epidermolysis bullosa (EB) that can separate activity from damage. OBJECTIVE: We sought to develop and validate an instrument for inherited EB of all ages and subtypes, the EB Disease Activity and Scarring Index (EBDASI), which scores activity responsive to therapy separately from scarring. METHODS: The EBDASI was validated by comparing its reliability and validity against the Birmingham EB Severity (BEBS) score (partially validated with activity mixed with scarring), using the Physician Global Assessment (PGA) scale as a reference measurement. Sixteen patients with EB (7 EB simplex, 5 dominant dystrophic EB [DDEB], 2 junctional EB, and 2 recessive dystrophic EB) were assessed by 5 EB experts using the EBDASI, BEBS, and PGA, and data from 9 additional patients assessed on an ad hoc basis during routine patient clinic were also included. RESULTS: For interrater reliability, the overall total score intraclass correlation coefficients (95% confidence intervals) were: EBDASI 0.964 (0.929-0.986), BEBS 0.852 (0.730-0.937), and PGA 0.873 (0.765-0.946). For intrarater reliability, the intraclass correlation coefficients were: EBDASI 0.994 (0.976-0.998), BEBS 0.926 (0.748-0.981), and PGA 0.932 (0.764-0.982). The EBDASI had a higher correlation with PGA (ρ = 0.871) than BEBS with PGA (ρ = 0.852). Intraclass correlation coefficients scatterplots showed the EBDASI was better at distinguishing milder forms of EB, with better correlations at higher severity scores than the BEBS. LIMITATIONS: A limited number of patients were recruited for this study. An independent study will be required to demonstrate the responsiveness of the EBDASI. CONCLUSION: The EBDASI demonstrated excellent reliability and validity, as compared with 2 other outcome measures.
BACKGROUND: There is a lack of validated standardized outcome measures for epidermolysis bullosa (EB) that can separate activity from damage. OBJECTIVE: We sought to develop and validate an instrument for inherited EB of all ages and subtypes, the EB Disease Activity and Scarring Index (EBDASI), which scores activity responsive to therapy separately from scarring. METHODS: The EBDASI was validated by comparing its reliability and validity against the Birmingham EB Severity (BEBS) score (partially validated with activity mixed with scarring), using the Physician Global Assessment (PGA) scale as a reference measurement. Sixteen patients with EB (7 EB simplex, 5 dominant dystrophic EB [DDEB], 2 junctional EB, and 2 recessive dystrophic EB) were assessed by 5 EB experts using the EBDASI, BEBS, and PGA, and data from 9 additional patients assessed on an ad hoc basis during routine patient clinic were also included. RESULTS: For interrater reliability, the overall total score intraclass correlation coefficients (95% confidence intervals) were: EBDASI 0.964 (0.929-0.986), BEBS 0.852 (0.730-0.937), and PGA 0.873 (0.765-0.946). For intrarater reliability, the intraclass correlation coefficients were: EBDASI 0.994 (0.976-0.998), BEBS 0.926 (0.748-0.981), and PGA 0.932 (0.764-0.982). The EBDASI had a higher correlation with PGA (ρ = 0.871) than BEBS with PGA (ρ = 0.852). Intraclass correlation coefficients scatterplots showed the EBDASI was better at distinguishing milder forms of EB, with better correlations at higher severity scores than the BEBS. LIMITATIONS: A limited number of patients were recruited for this study. An independent study will be required to demonstrate the responsiveness of the EBDASI. CONCLUSION: The EBDASI demonstrated excellent reliability and validity, as compared with 2 other outcome measures.
Authors: Clare L Rogers; Matthew Gibson; Johannes S Kern; Linda K Martin; Susan J Robertson; Benjamin S Daniel; John C Su; Oliver G C Murrell; Grant Feng; Dedee F Murrell Journal: JAAD Int Date: 2021-01-21
Authors: S V Jain; A G Harris; J C Su; D Orchard; L J Warren; H McManus; D F Murrell Journal: J Eur Acad Dermatol Venereol Date: 2016-10-03 Impact factor: 6.166
Authors: Brendon W H Lee; Jeremy C K Tan; Melissa Radjenovic; Minas T Coroneo; Dedee F Murrell Journal: Orphanet J Rare Dis Date: 2018-05-22 Impact factor: 4.123
Authors: M T Khan; M O'Sullivan; B Faitli; J E Mellerio; R Fawkes; M Wood; L D Hubbard; A G Harris; L Iacobaccio; T Vlahovic; L James; L Brains; M Fitzpatrick; K Mayre-Chilton Journal: Br J Dermatol Date: 2019-10-23 Impact factor: 9.302
Authors: Rocío Maseda; Lucía Martínez-Santamaría; Rosa Sacedón; Nora Butta; María Del Carmen de Arriba; Sara García-Barcenilla; Marta García; Nuria Illera; Isabel Pérez-Conde; Marta Carretero; Eva Jiménez; Gustavo Melen; Alberto M Borobia; Víctor Jiménez-Yuste; Ángeles Vicente; Marcela Del Río; Raúl de Lucas; María José Escámez Journal: Front Med (Lausanne) Date: 2020-11-26