Core Outcome Measures in Effectiveness Trials

Core Outcome Set for Epidermiolysis Bullosa (COSEB)

General Information

Summary:
Epidermolysis bullosa (EB) comprises a group of hereditary skin fragility disorders characterized by skin and mucosal blistering upon minor trauma with a prevalence of 2-5/100.000 inhabitants and an incidence of 10-15 per 100.000 newborns per year. Over 30 clinical subtypes are known, based on the level of separation in the skin and the clinical features. Depending on the mutation and the consequences on protein and tissue level many complications may arise, like scarring, pseudosyndactylie, contractures, esophagus strictures, cornea erosions and skin cancer . Other organs may be involved as well, like the heart and skeletal muscle. In the last thirty years the molecular causes have been identified, and as yet mutations in 19 different genes have been found to underlie the different subtypes of EB. Lately the research field is moving from etiology to therapy. With protein, RNA-based and DNA-based therapies in development it becomes of utmost importance to be able to compare efficacy and safety of these treatments. We aim to develop core outcome set for EB studies.

Contributors:
Dr. M.C. Bolling, principal investigator. University Medical Center Groningen, University of Groningen, Center for Blistering Disease, Department of Dermatology.
Prof. Dr. M.F. Jonkman, University Medical Center Groningen, University of Groningen, Center for Blistering Disease, Department of Dermatology.
Dr. S.J. Gordijn, Supervisor, University Medical Center Groningen, University of Groningen, Department of Obstetrics and Gynaecology.

Further Study Information

Current Stage:
Planning
Date:
January 2018 - January 2019
Funding source(s):
None

Health Area

Disease Category
Skin

Disease Name
Epidermolysis bullosa

Target Population

Age Range
0 - 100

Sex
Either


Nature / type of Intervention
Any

Method(s)

Consensus meeting
Delphi process

We will execute an online Delphi procedure. We anticipate 3 rounds after which we will have a consensus meeting as a satellite meeting to an annual meeting for EB.


Stakeholders Involved

Clinical experts
Consumers (patients)
Families
Methodologists
Patient/ support group representatives

Study Type

COS for clinical trials or clinical research
COS for practice

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