The use of dupilumab in young children with severe atopic dermatitis has demonstrated continued clinical improvements and a consistent safety profile, according to a recent subgroup analysis of an ongoing extension study.
Severe atopic dermatitis carries a substantial disease burden and markedly impairs quality of life. Yet current treatment options for infants and young children with severe atopic dermatitis are limited, particularly due to safety concerns associated with off-label systemic immunosuppressants and the fact that topical corticosteroids can be ineffective.
Therefore, a study published in the journal Pediatric Drugs has investigated the outcomes of long-term therapy with the monoclonal antibody dupilumab in this patient population.
The open-label extension analysis included 121 patients aged six months to five years with severe atopic dermatitis who had an Investigator’s Global Assessment (IGA) score of 4 at baseline.
They had previously participated in a 16-week LIBERTY AD PRESCHOOL parent trial and then continued in the LIBERTY AD PED open-label extension study.
Patients received weight-based dupilumab every four weeks and were followed for up to 104 weeks.
The primary endpoint was the incidence and rate of treatment-emergent adverse events (TEAEs). Secondary endpoints included the incidence and rate of serious TEAEs and discontinuations.
Clinical improvements with dupilumab
TEAEs were reported by 88.4% of patients, most of which were mild or moderate and not considered treatment-related, while serious TEAEs occurred in 14.0% of patients. Of these, only one event was deemed related to treatment, and none led to treatment discontinuation.
Upper respiratory tract infections, nasopharyngitis and cough were common adverse events. Mild-to-moderate conjunctivitis was reported in 19.0% of patients. Injection-site reactions were infrequent (3.3%), and no new safety signals were identified during follow-up.
Eczema Area and Severity Index (EASI) responses, IGA scores, and quality-of-life measures were key efficacy outcomes, and 95.9% of patients had achieved at least a 75% improvement in EASI by week 104.
In addition, 26.5% of patients achieved clear or almost clear skin (IGA ≤1), while 91.8% achieved mild disease or better (IGA ≤2).
A clinically meaningful ≥6-point improvement in the Children’s Dermatology Life Quality Index was achieved in 88.5% of evaluable patients, and all patients assessed with the Infants’ Dermatitis Quality of Life questionnaire reached this threshold.
Patients who initially received placebo in the parent study showed rapid improvement, with outcomes comparable to those of patients previously treated with dupilumab by week four of the extension study, indicating a prompt treatment response.
Limitations and implications for clinical practice
The authors noted that the safety profile was consistent with that observed in older children, adolescents and adults, and with earlier short-term studies, but several study limitations should be considered.
The open-label, non-randomised design and the absence of a comparator group may have led to overestimation of treatment effects. Analyses were based on observed data without imputation, and attrition over time may have introduced bias.
In addition, the small number of patients under two years of age and the predominantly White study population may limit generalisability, the authors added.
For clinical practice, the results support the potential for long-term dupilumab use in infants and young children with severe atopic dermatitis, particularly where disease control is difficult with standard therapies.
However, the authors said that future research should include more diverse populations and a broader age range to confirm these findings.
Reference
Paller AS et al. Long-term safety and efficacy of dupilumab treatment in children aged 6 months to 5 years with severe atopic dermatitis. Pediatr Drugs 2026;doi: 10.1007/s40272-026-00747-4.
This article was originally published by our sister publication Hospital Pharmacy Europe.