Abstract Number: 273

Which outcome measures are the best for clinical trials in Epidermolysis Bullosa?

C.L. Rogers 1,2, M. Gibson1,2, J.S. Kern3,4, L. Martin5, S. Robertson6, B.S. Daniel1,2, J.C. Su6,7, D.F. Murrell1,2

Meeting: 2021 Dermcoll

Session Information

Date: -

Session Title: Poster Presentations

Session Time: -

Background: The success of clinical trials in Epidermoly- sis 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 Instru- ment for Scoring Clinical Outcomes of Research for Epi- dermolysis Bullosa (iscorEB) were independently developed and validated against the Birmingham Epider- molysis Bullosa Severity Score (BEBS), but have never been directly compared.

Objective: To compare the reliability, convergent validity and discriminate validity of the EBDASI and iscorEB scor- ing tools.

Methods: An observational cohort study was conducted in 15 patients with EB. Each patient was evaluated by six dermatol- ogists with expertise in EB using the EBDASI and iscorEB- clinician (iscorEB-c) scoring tools. Quality of life was assessed using the iscorEB-patient and QOLEB measures.

Results: The intraclass correlation coefficients (ICC) for inter-rater reliability were: EBDASI 0.942 and iscorEB-c
0.852. The ICC for intra-rater reliability was 0.99 for both scores. The two tools demonstrated strong convergent validity with each-other. Both tools could discriminate between EB Simplex (EBS) and Junctional EB, EBS and Recessive Dystrophic EB (RDEB), and Dominant Dys- trophic EB (DDEB) and RDEB. In addition, the EBDASI could discriminate between EBS and DDEB.

Limitations: Limited sample size. Clinical and demo- graphic characteristics were not evenly distributed within the patient cohort. Due to seasonal conditions, EBS patients had mild disease on the day of assessment.

Conclusion: Both scoring tools demonstrate excellent reli- ability. The EBDASI appears to better discriminate between EB types and disease severities.

References

1. Jain, S, Harris, A, Su, J, Orchard, D, Warren, L, McManus, H, Murrell, D, 2017, The Epidermolysis Bullosa Disease Activity and Scarring Index (EBDASI): grading disease severity and assessing responsiveness to clinical change in epidermolysis bullosa, J Eur Acad Dermatol Venereol, vol. 31, no. 4, pp. 692–
698.
2. Bruckner, A, Fairclough, D, Feinstein, J, Lara-Corrales, I, Lucky, A, Tolar, J, Pope, E, 2018, Reliability and validity of the instru- ment for scoring clinical outcomes of research for epidermolysis bullosa (iscorEB), Br J Dermatol, vol. 178, no. 5, pp. 1128–1134.
3. Loh, C, Kim, J, Su, J, Daniel, B, Venugopal, S, Rhodes, M, Intong, L, Law, M, Murrell, D, 2014, Development, reliability, and valid- ity of a novel Epidermolysis Bullosa Disease Activity and Scar- ring Index (EBDASI), J Am Acad Dermatol, vol. 70, no. 1, pp. 89–
97.
4. Schwieger-Briel, A, Chakkittakandiyil, A, Lara-Corrales, I, Aujla, N, Lane, A, Lucky, A, Bruckner, A, Pope, E, 2014, Instrument for Scoring Clinical Outcome of Research for Epidermolysis Bullosa: A consensus-generated clinical research tool, Pediatr Dermatol, vol. 32, no. 1, pp. 41–52.
5. Frew, J, Cepeda-Valdes, R, Fortuna, G, Salas-Alanis, J, Murrell, D, 2013, Cross cultural adaptation of the ‘Quality of Life in Epi- dermolysis Bullosa’ (QOLEB) questionnaire: Validation and results of a Hispanic translation, Australas J Dermatol, vol. 54, no. 2, pp. 39–40.