Work

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

Year: 2024

Type: article

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.

Source: Journal of Clinical Immunology

DOI: 10.1007/s10875-024-01837-z

Authors: Yannik Vollmuth, Narjes Abdulhameed Alelq, Franziska Sattler, Susanne Schmidt, Fabian Hauck

Institutions: Ludwig-Maximilians-Universität München, LMU Klinikum, Ministry of Health

Language: English

Topic: Immunodeficiency and Autoimmune Disorders

Subfield: Immunology

Field: Immunology and Microbiology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $4,990