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Core Outcome Set Epidermolysis Bullosa (COSEB)

Condition

Epidermolysis Bullosa

Background

Epidermolysis bullosa (EB) is a group of rare genetic skin disorders characterized by fragility of skin and mucous membranes with blistering following minimal trauma. Patients with EB present a broad spectrum of clinical severity, which ranges from minor palmoplantar blistering, to extra cutaneous involvement and early lethality. Despite developments in novel therapies, there is still no cure available for EB. Therefore, symptom alleviation and disease-modifying therapies are currently the cornerstone of therapy. Due to advances in understanding the pathomechanisms of EB, there is a significant increase in the development and evaluation of treatments in randomized clinical trials to ascertain the efficacy and safety of promising therapeutic strategies. However, the appraisal of these results is hampered due to a lack of uniformity in outcome measurement and reporting. These findings warrant a consensus regarding which outcome domains and outcome measurement instruments should be used and reported in future clinical trials, to allow the results to be compared, contrasted and combined. This will in turn improve the quality and relevance of future research within EB.

Scope of the intended COS

  • Study types: (randomized) clinical trials
  • Region: Global

Methods

Scoping review to identify previously reported outcomes and outcome measurement instruments, systematic review to evaluate quality of identified outcome measurement instruments, consensus workshops, E-Delphi studies

Involvement of stakeholders

EB patients and/or their representatives, health care practitioners including clinicians, nurses and physician assistants involved in EB care, EB researchers and trialists, regulatory agencies, industry representatives.

Current status

COSEB has been registered at COMET: https://comet-initiative.org/Studies/Details/1033. Currently: scoping review.

Funding

No funding

Involved Countries

The Netherlands, Austria, Germany (so far, more to participate)

Date of updates

  • 16/02/2022

Lead

  • Marieke Bolling, MD, PhD.

Project team members

EB Expertise Center, University Medical Center Groningen, The Netherlands:

  • Marieke Bolling, MD, PhD
  • Barbara Horv├íth, MD, PhD
  • Eva Korte, MD

EB Haus, Hospital of the Paracelsus Medical University, Salzburg, Austria:

  • Martin Laimer, MD, PhD
  • Tobias Welponer, MD
  • Verena Wally, PhD

Department of Dermatology, Medical Center-University of Freiburg, Germany:

  • Dimitra Kiritsi, MD, PhD

Medicines Evaluation Board, Utrecht, Netherlands.

  • Marjon Pasmooij, PhD

Responsible member of Methods group (as assigned by CS-COUSIN)

  • Jan Kottner

Contact

Marieke Bolling, MD, PhD
Head of EB Expertise Center
EB Expertise Center, Center for Blistering Diseases
Dermatology Department, University Medical Center Groningen,
Groningen, The Netherlands
m.c.bolling@umcg.nl

Eva Korte, MD
PhD-candidate Blistering Diseases
EB Expertise Center, Center for Blistering Diseases,
Dermatology Department, University Medical Center Groningen,
Groningen, The Netherlands
e.w.h.korte@umcg.nl

Published references

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