The plasma-derived, pasteurized, nanofiltered C1-inhibitor concentrate (pnfC1-INH; Berinert®/CSL Behring) is the only on demand treatment of HAE approved for use in patients across all age groups, including children ≤12 years of age in the European Union. A large international patient registry collected data on the use of pnfC1-INH in patients of all ages, including children.
This observational registry, conducted between 2010 and 2014 at 34 US and 7 European sites, gathered data on a total of 318 subjects, including 18 children (5 to <12y) and 21 adolescents (12 to <17y) with HAE. Data gathered included reason for use, HAE attack characteristics, pnfC1-INH dosing, administration setting, and adverse events (AEs).
The registry documented 796 pnfC1-INH infusions in pediatric patients given as on demand treatment (n=757) or pre-procedural prophylaxis (n=39). Abdominal attacks were most common in both children (74% of attacks) and adolescents (52% of attacks). A majority of infusions were administered outside of a healthcare setting among children (213/275 infusions; 77.5%) and adolescents (425/52 infusions; 81.6%). The mean (SD) pnfC1-INH doses per infusion were 17.3 (4.7) IU/kg for children and 14.3 (5.4) IU/kg for adolescents. There were 3 AEs reported in 3 children (0.01 events per infusion; 0.17 events per patient) and 16 AEs reported in 7 adolescents (0.03 events per infusion; 0.76 events per patient), none of which were serious or considered related to pnfC1-INH.
Registry data regarding pnfC1-INH usage support a favorable safety profile in pediatric HAE patients regardless of administration within or outside of a healthcare facility setting.