Long-term off-label dupilumab in pediatric atopic dermatitis: A case series

Background/Objectives Clinical trials of dupilumab have shown efficacy in treating moderate-to-severe atopic dermatitis (AD) in adult patients. While a phase 2 trial of dupilumab has shown efficacy and safety in children, the medication awaits Food and Drug Administration (FDA) approval, and off-label use is limited by dosing currently available to adults. We present this case series to describe the efficacy and safety profile of off-label dupilumab use in six pediatric patients treated by one provider in a private practice. Methods We conducted a retrospective chart review of all patients under the age of 18 receiving dupilumab for AD. Two dosing regimens were used as follows: adult dose for patients >= 40 kg and half dose for those < 40 kg. We recorded the investigator's global assessment (IGA) and body surface area (BSA) prior to dupilumab initiation and following treatment. All patients were warned of potential side effects including injection site reaction, conjunctivitis, increased risk of infection, and lack of information about use in children. Results Six pediatric patients were identified with an average age of 10.8 years (range: 7-15). All patients had a decrease in IGA of at least 2 points using biweekly 300 or 150 milligram (mg) doses for an average treatment duration of 8.5 months (range: 6-11). Three patients (50%) had an IGA of 1 after treatment. No side effects were reported. Conclusions Dupilumab appears to be an effective treatment for AD in pediatric patients. Optimal dose and delivery have yet to be determined.