Observational studyThe British journal of dermatology2018
Reliability and validity of the instrument for scoring clinical outcomes of research for epidermolysis bullosa (iscorEB).
Observational study in The British journal of dermatology, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 2 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
21 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Keratin-associated epidermolysis bullosa simplex: phenotypes and challenges in clinical trials - a narrative review and systematic update.Orphanet journal of rare diseases · 2025Pooled it
- A systematic literature review of the disease burden in patients with recessive dystrophic epidermolysis bullosa.Orphanet journal of rare diseases · 2021Pooled it
- Bone marrow transplant with post-transplant cyclophosphamide for recessive dystrophic epidermolysis bullosa expands the related donor pool and permits tolerance of nonhaematopoietic cellular grafts.The British journal of dermatology · 2019Trial
- Health-Related Quality of Life of Patients withJournal of market access & health policy · 2026Article
- Eliciting health state utilities for open wounds in dystrophic epidermolysis bullosa.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2026Article
- Health-related quality of life in recessive dystrophic epidermolysis bullosa: findings of the Prospective Epidermolysis Bullosa Longitudinal Evaluation Study (PEBLES).Orphanet journal of rare diseases · 2026Article
- Development and Validation of a Scale to Assess Epidermolysis Bullosa Simplex Severity.JAMA dermatology · 2025Article
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- Article
- Pain in recessive dystrophic epidermolysis bullosa (RDEB): findings of the Prospective Epidermolysis Bullosa Longitudinal Evaluation Study (PEBLES).Orphanet journal of rare diseases · 2024Article
- Facing the complex challenges of people with epidermolysis bullosa in Austria: a mixed methods study on burdens and helpful practices.Orphanet journal of rare diseases · 2024Article
- Costs of UK community care for individuals with recessive dystrophic epidermolysis bullosa: Findings of the Prospective Epidermolysis Bullosa Longitudinal Evaluation Study.Skin health and disease · 2024Article
- Itch in recessive dystrophic epidermolysis bullosa: findings of PEBLES, a prospective register study.Orphanet journal of rare diseases · 2023Article
- Podiatric assessment in children and adults with epidermolysis bullosa: are foot and ankle patient-reported outcome measures (PROMs) able to measure the severity of podiatric involvement among EB patients?International journal of women's dermatology · 2023Review
- A prospective short-term study to evaluate methodologies for the assessment of disease extent, impact, and wound evolution in patients with dystrophic epidermolysis bullosa.Orphanet journal of rare diseases · 2022Observational
- Clinical trial of ABCB5+ mesenchymal stem cells for recessive dystrophic epidermolysis bullosa.JCI insight · 2021Article
- Immune tolerance of allogeneic haematopoietic cell transplantation supports donor epidermal grafting of recessive dystrophic epidermolysis bullosa chronic wounds.The British journal of dermatology · 2021Article
- Article
- Profiling trial burden and patients' attitudes to improve clinical research in epidermolysis bullosa.Orphanet journal of rare diseases · 2020Article
- Occupational therapy for epidermolysis bullosa: clinical practice guidelines.Orphanet journal of rare diseases · 2019Review
Corrections and comments
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Authors and funding
7 authors.
Funding
Abstract
backgroundEpidermolysis bullosa (EB) is a group of rare and currently incurable genetic blistering disorders. As more pathogenic-driven therapies are being developed, there is an important need for EB-specific validated outcomes measures designed for use in clinical trials.
objectivesTo test the reliability and construct validity of an instrument for scoring clinical outcomes of research for EB (iscorEB), a new combined clinician- and patient-reported outcomes tool.
methodsWe conducted an observational study consisting of independent 1-day assessments (six assessors) at two academic hospitals. The assessments consisted of iscorEB clinician (iscorEB-c), Birmingham Epidermolysis Bullosa Severity (BEBS) and global severity assessment for physicians; and iscorEB patient (iscorEB-p), Quality of Life evaluation in Epidermolysis Bullosa and Children's Dermatology Life Quality Index for patients. Construct validity and intraclass correlation coefficients (ICCs) for interobserver, intraobserver and test-retest reliability were calculated.
resultsOverall, 31 patients with a mean age of 19·5 years (1·8-45·2) were included. Disease severity was mild in 42% of cases, moderate in 29% and severe in 29%. The interobserver ICC was 0·96 for both the clinician-reported section of iscorEB-c and BEBS. The ICC for intraobserver reliability was 0·91 and 0·70 for the skin and mucosal domains of iscorEB-c, respectively. Cronbach's alpha for iscorEB-c was 0·89. The test-retest reliability of iscorEB-p was 0·97 and Cronbach's alpha was 0·84. The clinical score differentiated between subjects with mild, moderate and severe disease, and both clinical and patient subscores discriminated between recessive dystrophic EB and other EB subtypes.
conclusionsiscorEB has robust reliability and construct validity, including strong ability to distinguish EB types and severities. Further studies are planned to test its responsiveness to change.
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Registered trials
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