Clinical outcome measures in epidermolysis bullosa: a scoping review

epidermolysis bullosa Outcome measurement

dr. B. Horvath
drs. E.W.H. Korte
dr. M.C. Bolling

Nature of the research:

Fields of study:

Background / introduction
Epidermolysis bullosa (EB) is a group of rare genetic skin disorders characterized by fragility of skin and mucous membranes. Despite developments in novel treatments, there is still no cure available for EB. Therefore, research is increasingly directed at symptom alleviation and disease-modifying therapies to improve quality of life. In the Netherlands, the medical specialized care for EB patients is provided by the national EB Expertise Center in the University Medical Center Groningen (UMCG).
A relatively large number of clinical trials are currently conducted and aim to ascertain the clinical efficacy of various therapies in EB. A key focus in the evaluation of efficacy is to have an accurate way of measuring the clinical outcomes of these therapies. The number of clinical outcome measurements has grown exponentially and there are numerous questionnaires available for assessing a multitude of domains in patients with EB.
Currently, there is no general consensus on the reporting of outcomes in clinical trials in the field of EB. Reaching consensus on a “core set of outcomes” would improve the consistency and relevance of new findings and enable the results of trials to be compared and combined, which improves quality and relevance of future research leading to better informed healthcare decisions. This scoping review will be the first step in developing a set of core outcomes, by collating and mapping the literature on existing and previously used clinical outcomes in EB research.
Research question / problem definition
What clinical outcome measures have been used in studies of patients with EB?
- What is the content (items/domains) of the used clinical outcomes?
- How are the clinical outcomes reported (e.g. self-reported, proxy, interview)?
- What are the measurement properties of the clinical outcomes (e.g. validity, reliability, responsiveness)?
The workplan will be adjusted to the personal learning objectives of the student. The workplan may include assisting in the literature search, data extraction, data analysis and writing a scientific report on the results. During this research project, the student will be supervised by a PhD-student (daily supervisor) and a medical staff member of the Dermatology department in the UMCG. Participating in this project will be an excellent introduction to evidence based research, suitable for both bachelor students or master students with or without experience in conducting research. The project is part of an on-going PhD-programme of the daily supervisor and the aim is to publish the review, which will result in co-authorship of the student in the published article.
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