Real-World Data Back Dupilumab's Long-Term Use in Pediatric AD

Dupilumab (Dupixent; Sanofi and Regeneron) kept moderate to severe atopic dermatitis (AD) under control for most pediatric patients through 3 years of real-world treatment, but children younger than 6 years continued to report more pruritus and worse quality of life than their older peers, according to a new cohort study published in JAMA Dermatology.¹

The findings, drawn from the Dutch BioDay Registry, followed 309 pediatric patients with moderate to severe AD who started dupilumab between November 2019 and October 2025 across 4 academic and 3 nonacademic hospitals in the Netherlands. The cohort spanned 3 age bands (6 months to 5 years, 6 to 11 years, and 12 to 16 years), reflecting how the drug's pediatric indication rolled out in stages, first approved for adolescents in 2019 before expanding to children aged 6 to 11 in 2020 and finally to children as young as 6 months in 2022.^2-4

The results add long-term, real-world weight to a growing evidence base. Phase 3 open-label extension data have previously shown dupilumab holding up well through 52 weeks in children and adolescents, with incremental gains the longer patients stayed on it.⁵ More recently, an interim analysis of the LIBERTY AD PRESCHOOL trial (NCT03346434) found that even children who didn't hit the trial's strictest response threshold still saw meaningful symptom relief, suggesting standard efficacy measures may understate the drug's real-world benefit in very young children.⁶

“Because AD is a chronic disease that often requires long-term treatment, it is important to evaluate its effectiveness and safety in a clinical setting across different pediatric age groups,” the authors explained.¹

What the 3-Year Data Show

Across the full cohort, mean Eczema Area and Severity Index (EASI) scores fell from 16.3 at baseline to 4.6 at 16 weeks and 3.4 at 3 years, with 89.1% of children reaching an EASI score of 7 or lower by year 3. Itch and quality-of-life scores similarly improved, and roughly a quarter of patients were able to stretch their dosing interval, with the majority (74.4%) of patients who prolonged dose intervals reporting controlled disease as the reason. However, 14.1% and 11.5% of those who prolonged intervals did so because of adverse events (AEs) and administration problems, respectively.

Serum levels of thymus and activation-regulated chemokine (TARC), a biomarker of AD severity, decreased significantly among the patients who had baseline TARC measurements after 1 year of treatment. TARC levels remained decreased during later follow-ups.

Nearly two-thirds (63.1%) of children had at least 1 AE, although most (97.4%) were mild to moderate. The most commonly reported AE was dupilumab-associated ocular surface disease, which affected 36.6% of patients. Only 4.9% of the cohort experienced AEs serious enough to end treatment.

Younger Children Face Added Challenges

Children aged 6 months to 5 years reported significantly higher pruritus and lower quality-of-life scores than older children throughout follow-up, even though their skin-clearance scores improved just as much. The study authors suggested this gap may partly reflect the fact that younger children's outcomes are typically reported by caregivers rather than the child, and proxy reports don't always capture a child's actual symptom burden.

That same age group also drove a specific pattern in treatment discontinuation. Of the 50 patients (16.2%) who stopped dupilumab, ineffectiveness (38.0%) and AEs (30.0%) were the most common reasons. However, administration problems, including injection-related pain or refusal, accounted for 22.0% of discontinuations and were concentrated almost entirely among children age 6 months to 5 years, the authors noted.

With needle phobia being documented among other chronic diseases in childhood, the authors highlighted the importance of clinicians being aware of the potential for needle phobia to develop and “implementing procedural comfort care to minimize the pain and distress associated with these injections.”

References

1. Vroman F, Bacoş-Cosma OI, Loman L, et al. Long-term effectiveness, safety, and survival of dupilumab in pediatric atopic dermatitis. JAMA Dermatol. Published online September 16, 2026. doi:10.1001/jamadermatol.2026.3548
2. FDA approves Dupixent (dupilumab) for moderate-to-severe atopic dermatitis in adolescents. News release. Sanofi. March 11, 2019. Accessed September 24, 2026. https://www.news.sanofi.us/2019-03-11-FDA-approves-Dupixent-R-dupilumab-for-moderate-to-severe-atopic-dermatitis-in-adolescents
3. FDA approves Dupixent (dupilumab) as first biologic medicine for children aged 6 to 11 years with moderate-to-severe atopic dermatitis. News release. Sanofi. May 26, 2020. Accessed September 24, 2026. https://www.sanofi.com/en/media-room/press-releases/2020/2020-05-26-15-40-00-2038798
4. FDA approves Dupixent (dupilumab) as first biologic medicine for children aged 6 months to 5 years with moderate-to-severe atopic dermatitis. News release. Sanofi. June 7, 2022. Accessed September 24, 2026. https://www.sanofi.com/en/media-room/press-releases/2022/2022-06-07-20-45-00-2458243
5. Gavidia M. Dupilumab clinically effective long term for moderate to severe pediatric atopic dermatitis. AJMC^®. May 17, 2022. Accessed September 24, 2026. https://www.ajmc.com/view/dupilumab-clinically-effective-long-term-for-moderate-to-severe-pediatric-atopic-dermatitis
6. Shaw ML. Pediatric patients see benefit in short-, long-term treatment for AD. AJMC. March 22, 2023. Accessed September 24, 2026. https://www.ajmc.com/view/pediatric-patients-see-benefit-in-short--long-term-treatment-for-ad
7. Gavidia M. Drug survival of dupilumab affected by immunosuppressive therapy, older age in AD. AJMC. August 18, 2022. Accessed September 24, 2026. https://www.ajmc.com/view/drug-survival-of-dupilumab-affected-by-immunosuppressive-therapy-older-age-in-ad