Dupilumab in a 9-week-old with Netherton Syndrome Leads to Deep Symptom Control

Vollmuth, Yannik; Alelq, Narjes Abdulhameed; Sattler, Franziska; Schmidt, Susanne und Hauck, Fabian (15. November 2024): Dupilumab in a 9-week-old with Netherton Syndrome Leads to Deep Symptom Control. In: Journal of Clinical Immunology, Bd. 45, 42

Abstract

Purpose

Netherton syndrome (NS) is a rare inborn error of immunity (IEI) with an incidence of approximately 1:200,000 and the phenotypic triad of trichorrhexis invaginate (bamboo hair), congenital ichthyosiform erythroderma, and multiple atopic manifestations. Treatment options especially in infants are scarce and generally not licensed.

Methods

Case report of a 9-week-old infant with NS treated with dupilumab off-label.

Results

We report rapid and sustained resolution of allergic inflammation, deep symptom control including normalization of the skin microbiome, and catch-up somatic and psychomotor development without adverse drug reactions.

Conclusion

Due to the high complication rate of NS, especially in the first years of life, we recommend treatment with dupilumab off-label immediately after the diagnosis has been established.

• Dokumententyp: Zeitschriftenartikel
• Fakultät: Medizin > Klinikum der LMU München > Kinderchirurgische Klinik und Poliklinik im Dr. von Haunerschen Kinderspital
• Themengebiete: 600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin und Gesundheit
• ISSN: 0271-9142
• Sprache: Englisch