Outcome measures in interventional studies of patients with autoimmune bullous diseases: a systematic review

Outcome measurement autoimmune blistering diseases

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

Nature of the research:

Fields of study:

Background / introduction
Autoimmune bullous diseases (AIBDs) are a group of rare, heterogeneous blistering diseases with autoantibodies against structural skin proteins. AIBDs are characterized by varying levels of blistering and erosions of the skin and mucous membranes, which may result in significant morbidity and mortality. Currently, there are no curative therapies available. Therefore, the current mainstay of treatment consists of high potency topical and systemic corticosteroids and immunomodulatory therapies. In the Netherlands, medical specialized care for AIBD patients is provided by the Center for Blistering Diseases at the University Medical Center Groningen (UMCG).
Research within the field of AIBD is increasingly aimed at developing and evaluating interventions in randomized clinical trials to examine the efficacy and safety of promising therapeutic strategies. When evaluating the available research evidence, concerns about the quality of primary interventional studies have been expressed in several systematic reviews. They state the need for consistent reporting of outcomes and randomized controlled trials with high levels of evidence.
These findings warrant a consensus regarding which outcome measures should be used in future clinical trials, to allow results to be compared, contrasted and combined. To aid in this process, a systematic review will be conducted to synthesize the outcome measures reported in AIBD studies and identify the used measurement instruments (e.g. tools, questionnaires). In addition, we will critically evaluate the methodological quality of the identified measurement instruments through application of the COSMIN ‘Good Measurement Property’ checklist .
Research question / problem definition
1. What disease and treatment related outcome measures have been reported in AIBD studies?
2. Which outcome measurement instruments are being used for these outcomes?
3. What is the quality of the measurement properties of the identified measurement instruments?
The workplan of this student research project may include assisting in the literature search, data extraction, data analysis and writing a scientific report on the results. The workplan will be adjusted to the personal learning objectives of the student. The student will be supervised by a PhD-student (daily supervisor) and a medical staff member of the Dermatology department in the UMCG. The project will lead to a scientific publication, which the student would be welcome to co-author. Please feel free to contact us for more information.
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