Dupilumab in Preschool Atopic Dermatitis: Real-World Data on Efficacy and Safety

A growing body of evidence supports the use of dupilumab, a monoclonal antibody targeting IL-4 and IL-13, in pediatric populations with moderate-to-severe atopic dermatitis (AD). And while it’s notable as the first systemic treatment option for children under six years old with severe AD, real-world data on this age group has remained limited.

To help fill this gap, a recent multicenter, retrospective observational study from the Czech Republic provided insight into both the clinical performance and tolerability of dupilumab in these young patients.

Study Design and Patient Population

The study enrolled 24 pediatric patients under the age of six (mean age 4.4 years) with moderate-to-severe AD; they were then all treated with weight-based dupilumab dosing for a minimum of 16 weeks and monitored at regular intervals for up to 96 weeks.

Data were extracted from three tertiary-care dermatology centers and included Eczema Area and Severity Index (EASI) scores, Itch Numeric Rating Scale (NRS), Children's Dermatology Life Quality Index (CDLQI), and adverse event (AE) reporting.

At baseline, the mean EASI score was 26.7 (range 11.2–42.5), and the mean CDLQI was 21, reflecting a high disease burden. None of the patients had a prior history of treatment with biologics or JAK inhibitors.

Clinical Outcomes

EASI Response:

Improvements in disease severity were both rapid and sustained. By week 4, 83.3 percent of patients had achieved EASI-50, with this rate rising to 100 percent by week 16. At that point, 75 percent of patients achieved EASI-75 and 37.5 percent achieved EASI-90.

These responses were maintained through long-term follow-up, as the three patients who remained in the study at week 96 achieved EASI-90.

Quality of Life:

Quality-of-life outcomes improved in parallel. CDLQI scores decreased from a mean of 21 at baseline to 7 by week 16, with only 19 percent of children maintaining scores indicating substantial QoL impairment (CDLQI ≥11) compared to 95 percent at baseline.

This degree of improvement was greater than what was reported in the LIBERTY AD PRESCHOOL phase 3 trial, potentially due to higher baseline CDLQI values in this cohort.

Symptom Burden:

Itch NRS scores decreased from 7 at baseline to 4 by week 16, with further reductions observed in later assessments. By week 24, the majority of patients reported itch scores ≤3.

Sleep disturbance also improved, with average NRS values falling from 7 to 3 within the first 16 weeks.

Safety Profile

Adverse events (AEs) were observed in 50 percent of patients during the initial 16-week treatment period. The most frequently reported events included mild upper respiratory tract infections (20.8 percent), conjunctivitis (8.3 percent), and localized viral infections, including eczema herpeticum (8.3 percent), molluscum (8.3 percent), and varicella (4.2 percent).

Importantly, no serious AEs were reported, and no laboratory abnormalities were detected throughout the treatment period. Conjunctivitis cases were managed with topical therapies and did not lead to treatment discontinuation.

Across the 96-week study duration, at least one AE was reported in 58.3 percent of participants. These findings are consistent with previously published data from both clinical trials and other real-world studies.

Contextualizing with Prior Literature

The study’s outcomes are broadly aligned with results from the LIBERTY AD PRESCHOOL study, which reported EASI-50, -75, and -90 responses of 69 percent, 53 percent, and 25 percent respectively at 16 weeks. Likewise, two other retrospective studies by Berna-Rico et al. and Yang et al. have demonstrated comparable efficacy, albeit with variations in dosing schedules and loading protocols.

While direct comparison is limited by these methodological differences, the consistent trajectory of improvement across studies supports the generalizability of dupilumab's benefits in this age group.

Limitations

This study is limited by its retrospective design, small sample size, and variable duration of follow-up. Additionally, CDLQI responses rely on caregiver interpretation in very young children, which may introduce subjectivity.

However, the consistency of response across multiple validated endpoints strengthens the reliability of these findings.

Conclusions

This real-world observational study validates that dupilumab is both effective and well tolerated in children under six years of age with moderate-to-severe AD. Clinical improvements were observed as early as week 4 and were sustained across nearly two years of follow-up in some patients.

The data support early intervention with dupilumab in this demographic, particularly in cases where conventional therapies are insufficient or carry unacceptable safety risks.

Reference

Rob F, Pinkova B, Sokolova K, Kopuleta J, Jiraskova Zakostelska Z, Cadova J. Real-world efficacy and safety of dupilumab in children with atopic dermatitis under 6 years of age: a retrospective multicentric study. J Dermatolog Treat. 2025;36(1):2460578. doi:10.1080/09546634.2025.2460578