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The following is a summary of “Developing a core outcome set for Netherton syndrome: An international multi-stakeholder e-Delphi consensus study,” published in the November 2024 issue of Dermatology by Nouwen et al. 

Netherton syndrome (NS), a rare genetic skin disorder resulting from serine protease inhibitor Kazal type 5 (SPINK5) gene mutations, leading to severe skin barrier impairment.   

Researchers conducted a retrospective study to invent a core outcome set (COS) for NS to be used in clinical care and research.   

They followed the Core Outcome Measures in Effectiveness Trials (COMET) initiative guidelines and identified the outcomes from the literature, classified them using the International Classification of Functioning and COMET taxonomies, organized discussion groups at the 2nd International Netherton Congress 2022 to finalize the provisional outcome list. Through a 2-round e-Delphi, 41 stakeholders (patients and family members, professionals, and industry representatives) from 14 countries rated outcome importance on a 9-point Likert scale. An online consensus meeting with 14 stakeholders finalized the COS.  

The results showed that the COS for NS comprised 21 outcomes across 10 domains with 4 skin, 2 sensation, 2 side-effects of treatment, 1 vitality, 1 emotional functioning, 2 physical development, 2 nutrition, 2 infections, 2 allergies, and 3 assessment results outcomes.  

Investigators concluded that consensus reached on 21 outcomes for the NS COS, emphasizing the need for a multidisciplinary approach to NS care and research. 

Source: karger.com/drm/article/doi/10.1159/000542215/915442/Developing-a-core-outcome-set-for-Netherton