Developing a Core Outcome Set for Netherton Syndrome: An International Multi-Stakeholder e-Delphi Consensus Study

• DOI: 10.1159/000542215
• Publication Date: 2024-11-01T17:00:28Z
• Authors: Anouk E.M. Nouwen; Aviël Ragamin; Maria J. Knol; Hagen Ott; Lisa Weibel; Cristina Has; Alain Hovnanian; Amy S. Paller; Christine Bodemer; Virgil A.S.H. Dalm; Suzanne G.M.A. Pa...; Renske Schappin
• Keywords: Delphi Method; Delphi

Netherton syndrome (NS; OMIM#256500) is a rare and severe disorder of epidermal maturation keratinization caused by pathogenic variants in the serine protease inhibitor Kazal type 5 (SPINK5), leading to skin barrier impairment. Although effective treatment crucial for NS patients, there lack knowledge on what best options are these patients. Large heterogeneity reported outcomes measurement instruments hinders accurate comparison results across studies development guideline. Therefore, we aimed develop core outcome set (COS) that can be used clinical care research.