Pediatric hereditary angioedema: what the otolaryngologist should know

To review pediatric hereditary angioedema for otolaryngologists, with emphasis on articles within the past 12-18 months.

Biologic therapies are accepted for adult hereditary angioedema (HAE), but have been studied less for pediatric HAE. Recent literature supports expanded use of biologic agents in pediatrics as acute treatment and prophylaxis. Available agents include plasma-derived C1 esterase inhibitors (C1-INH) (Berinert, Haegarda, Cinryze), recombinant C1-INH (Ruconest), bradykinin B2 receptor inhibitor (Icatibant), and kallikrein inhibitors (Ecallantide and lanadelumab). Of these, only Berinert is Food and Drug Administration (FDA) approved for acute therapy for children under 12 years of age. Ruconest is approved for treatment of acute attacks over age 13. Ecallantide also has FDA approval as acute treatment for age 12 and older, while lanadelumab and Haegarda are prophylactic agents for adolescents. Icatibant lacks FDA approval in patients under 18 years of age. Cinryze has FDA approval only for prophylaxis for children as young as 6 years old.

Pediatric HAE is a potentially life-threatening disease. Targeted biologic agents have gained acceptance in treatment of acute attacks, and their use as prophylactic agents is changing the focus of management from acute intervention to preventive management. While intubation or surgical airway management may still be necessary, early intervention or prophylaxis can decrease morbidity and improve quality of life.