Dupilumab Shows Sustained Efficacy and Safety in Young Children With Severe Atopic Dermatitis: Study
USA: Long-term treatment with dupilumab for up to 2 years demonstrated sustained improvements in signs, symptoms, and quality of life in children aged 6 months to 5 years with severe atopic dermatitis (AD). Children who had previously received placebo experienced rapid improvement after starting dupilumab. The long-term safety profile remained consistent with the parent study, with no new safety signals identified.
The study, published in Paediatric Drugs, was led by Amy S. Paller, Department of Dermatology, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA, and colleagues. The researchers evaluated the long-term efficacy and safety of dupilumab in infants and young children with severe AD, a chronic inflammatory skin disease that can substantially affect sleep, daily activities, and quality of life.
The analysis included children from the ongoing LIBERTY AD PED open-label extension study who had previously participated in the LIBERTY AD PRESCHOOL Part B trial. All participants had severe disease at the beginning of the parent study, defined by an Investigator's Global Assessment (IGA) score of 4.
Children received weight-based dupilumab every 4 weeks for up to 2 years. Those weighing 5 to less than 15 kg received 200 mg, while those weighing 15 to less than 30 kg received 300 mg. Researchers assessed treatment-emergent adverse events along with improvements in eczema severity and quality of life.
The analysis involved 121 children, of whom 50 completed the week-104 assessment.
Key findings included:
- By week 104, 96% of children achieved at least a 75% reduction in Eczema Area and Severity Index (EASI-75) from the parent-study baseline.
- An IGA score of 0 or 1, indicating clear or almost clear skin, was achieved by 27% of patients.
- Overall, 92% reached an IGA score of 2 or lower, corresponding to clear skin or mild AD.
- EASI scores decreased by 89% from baseline by week 104.
- Among children aged 4 years or older, 89% (23 of 26) achieved a clinically meaningful improvement of at least 6 points on the Children's Dermatology Life Quality Index.
- All 3 assessed children younger than 4 years achieved a clinically meaningful improvement on the Infants' Dermatitis Quality of Life questionnaire.
- Children who had initially received placebo showed improvements comparable to those who started dupilumab by week 4 after entering the extension study.
Treatment-emergent adverse events occurred in 88% of participants, mostly mild or moderate. Common events included upper respiratory tract infections, nasopharyngitis, and cough. Conjunctivitis was reported in 19%, while one patient discontinued dupilumab due to severe urticaria. Serious adverse events occurred in 14%, with no serious event leading to treatment discontinuation.
Overall, dupilumab provided sustained improvements in disease severity and quality of life over 2 years, with no new safety concerns identified.
Reference:
Paller AS, Pinter A, Wine Lee L, Aschoff R, Zdybski J, Schnopp C, Khokhar FA, Praestgaard AH, Bansal A, Shumel B, Bastian M. Long-Term Safety and Efficacy of Dupilumab Treatment in Children Aged 6 Months to 5 Years with Severe Atopic Dermatitis. Paediatr Drugs. 2026 May;28(3):307-320. doi: 10.1007/s40272-026-00747-4. Epub 2026 Apr 2. PMID: 41926052; PMCID: PMC13121186.
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