Dupilumab Effective for Children with Severe Atopic Dermatitis in Real World Setting

Dupilumab’s efficacy and safety findings among children aged 6 months to 5 years with severe atopic dermatitis are consistent with previous results observed in those with moderate-to-severe disease, new data suggest.1

Audrey Lasek, MD, from the Group Des Hôpitaux Université Catholique De Lille, led a team of investigators in authoring this analysis. Lasek and colleagues noted authorization in France for early access to medications allows patients to be treated with investigational drugs or foreign medicines. It can also allow for access to approved treatments for unapproved indications prior to formal European Medicines Agency (EMA) approvals via the pre-marketing authorization Early Access Program (EAP).

This system, Lasek and coauthors highlighted in the background portion of their analysis, allows for more timely options for medication among patients with diseases such as psoriasis and addresses gaps in drug availability.

“This study reports aggregated data from the French EAP, evaluating the effectiveness and safety of dupilumab treatment in children aged 6 months–5 years with severe [atopic dermatitis] in a real-world setting prior to dupilumab reimbursement in France,” the investigators wrote.1

Study Background and Design Details

There were 108 physicians involved in this expanded access program in France, with infants and young children aged 6 months to 5 years with severe atopic dermatitis being enrolled by these clinicians through 65 clinical sites located across the country. Nearly all of the participating centers were public institutions.

Lasek et al noted 96.9% of the 65 sites were public hospitals and only 2 sites (3.1%) were private healthcare facilities. Individuals deemed eligible met the investigators' predefined criteria, including having a dermatologist-confirmed atopic dermatitis diagnosis, requiring systemic drugs, and having an inadequate response to, intolerance of, or contraindication to the use of topical corticosteroids.

A mix of secondary care centers were used in this program, along with university hospitals, general hospitals, and a small number of private centers. University hospitals made up the majority of sites taking part (72.2%). Prescribing authority was restricted to specialists who were hospital-based and working in allergology, dermatology, or internal medicine.

Several patient populations were defined within the program, including all of those for whom an EAP request was provided, subjects who were formally involved and granted treatment based on criteria for eligibility, and exposed patients who received at least 1 dupilumab dose and provided at least a single initiation, follow-up, or drug cessation form and a documented administration date.

Dupilumab was administered by Lasek and colleagues subcutaneously on an every-4-week basis, with prefilled syringes being implemented. They used a dose of 200 mg for children weighing 5 kg at least but less than 15 kg. Alternatively, a 300 mg syringe would be given to those weighing at least 15 kg but less than 30 kg, in alignment with protocol recommendations. Children who initially weighed under 15 kg but attained this threshold in the middle of the program would be kept on the 200-mg dosing regimen throughout their participation.

The investigative team looked at clinical effectiveness via multiple validated outcome measures. Such measures included the Investigator’s Global Assessment, the Pruritus Numerical Rating Scale, Eczema Area and Severity Index (EASI) with 50%, 75%, and 90% improvement thresholds, and age-appropriate quality-of-life instruments.

Lasek and coauthors used the Infants’ Dermatitis Quality of Life Indexfor childr en who were younger than 4 years of age. They would apply the Children’s Dermatology Life Quality Index to those aged 4 years or older. Evaluations were performed at the point of baseline and at the 1, 3, and 6-month marks. Safety was monitored throughout the study period.

Dupilumab Results in Children with Severe Atopic Dermatitis

There were 198 children recruited within the analysis, with 13.6% being under 2 years of age 86.5% being aged 2 years or older. Notably, among the 116 individuals evaluated by Lasek et al who had at least a single follow-up examination, substantial clinical improvements were seen by the 6-month mark.

94.4%, 75.0%, and 44.4% of subjects in the study met EASI 50, EASI 75, and EASI 90 response criteria, respectively. The investigators also found IGA score attainment of 0 or 1 was seen among 83.7% of those assessed. Additionally, the investigative team found 67.6% of participants reported at least a 4-point reduction in pruritus severity as determined by the P-NRS.

Lasek and the team further noted, from treatment initiation to Month 6, study subjects' mean ± standard deviation reductions were shown to be −17.5 ± 11.6 for EASI, −2.1 ± 1.2 for IGA, and −4.4 ± 2.8 for P-NRS scores. Outcomes related to patients' quality-of-life also improved over the same period, with the team highlighting mean ± SD decreases of −6.9 ± 6.6 in participants' IDQOL scores and −8.8 ± 7.3 in their CDLQI scores.

The investigators' safety findings during the program were also found to have been in line with the previously established safety profile of dupilumab in both pediatric and adult patients with atopic dermatitis.

“The real-world nature of the EAP complements the controlled environment of randomized clinical trials, offering insights into the practical use of dupilumab in routine clinical practice, in children as young as 6 months old,” the team concluded.1 “A longer term follow-up is required to assess the durability of response and long-term safety of dupilumab in this young population.”

References

1. Lasek A, Bellon N, Achour L, et al. Dupilumab treatment in children aged 6 months to 5 years with severe atopic dermatitis. J Eur Acad Dermatol Venereol. 2025; 00: 1–10. https://doi.org/10.1111/jdv.70268.

2. Tarantola A, Otto MH, Jommi C, et al. Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case. J Pharm Policy Pract. 2023 May 17;16(1):67. doi: 10.1186/s40545-023-00570-z. PMID: 37198599; PMCID: PMC10193685.