Dupilumab Safety and Efficacy up to 3 Years in Children Aged 6 Months to 11 Years with Atopic Dermatitis: Results from a Phase III Open-Label Extension Study

Dupilumab previously demonstrated safety and efficacy in randomized controlled trials (RCTs) and long-term open-label extension (OLE) studies for up to 2 years in patients with atopic dermatitis (AD) aged ≥ 6 months. We report safety and efficacy up to 3 years from an ongoing OLE study (LIBERTY PED OLE) in young pediatric patients with moderate-to-severe AD. Children aged ≥ 6 months to 11 years with moderate-to-severe AD from prior dupilumab RCTs received open-label dupilumab every 4 weeks (200 mg: 5 to < 15 kg; 300 mg: 15 to < 30 kg) or every 2 weeks (200 mg: 30 to < 60 kg; 300 mg: ≥ 60 kg). Concomitant topical medications were permitted. Safety, efficacy, and quality of life (QoL) were assessed at parent study baseline or OLE study baseline through 152 weeks. One hundred and eighty patients 6 months to 5 years old and 383 patients 6 to 11 years old were enrolled; 56.1% and 65.3% completed week 152, respectively. The most frequent drug-related treatment-emergent adverse events (TEAEs) included conjunctivitis (2.8% in the 6 months to 5 years group; 2.6% in the 6 to 11 years group), allergic conjunctivitis (2.8%; 2.6%), and nasopharyngitis (1.7%; 2.1%). Dupilumab resulted in persistent efficacy improvements from parent study baseline and OLE study baseline to week 152. ≥ 75%/90% improvement in Eczema Area and Severity Index scores (EASI-75/EASI-90) at week 152 was achieved in 90.3%/60.2% of patients (6 months to 5 years) and 89.1%/61.1% (6 to 11 years). Similar outcomes were observed in other clinical efficacy (SCORing AD score [SCORAD]-50 and body surface area) and QoL (Infants’/Children’s Dermatitis QoL Index) metrics. In this OLE study, long-term treatment with dupilumab for up to 3 years showed an acceptable safety profile and continued efficacy in infants and children with moderate-to-severe AD, supporting its use in pediatric patients [Graphical Abstracct available online]. ClinicalTrials.gov Identifier: NCT02612454. Atopic dermatitis (AD, or eczema) is an inflammatory disease with repeating episodes of skin rash and itch. It is common in children and can last for years or throughout life. Dupilumab is an advanced treatment for AD. We evaluated the benefits and safety of dupilumab treatment for up to 3 years in children aged 6 months to 11 years with AD. LIBERTY PED OLE is an ongoing study for children with AD. Patients receive dupilumab based on body weight and can continue using medicated skin creams. This analysis includes two age groups: 6 months to 5 years (180 children) and 6–11 years (383 children). Among these, 101 and 250 children, respectively, completed the 3-year visit (week 152). The most common side effects were red eye, reported as either “allergic conjunctivitis” or “conjunctivitis not further specified” (each occurring in up to 3% in each age group), and nasopharyngitis (runny nose, sore throat; around 2% in each age group). These side effects are similar to those reported in shorter dupilumab studies. After 3 years, 90% of children 6 months to 5 years old and 89% of children 6–11 years old achieved EASI-75 (at least 75% reduction in disease severity and extent of AD). By week 152, most patients reported substantially less disease impact on daily activities, compared with severe quality-of-life impairment at study start. In conclusion, in children aged 6 months to 11 years, up to 3 years of dupilumab treatment led to sustained improvement of AD with an acceptable safety profile.