Management of Hereditary Angioedema in Childhood

Background: Hereditary angioedema (HAE) is a rare disease characterized by recurrent, self-limiting attacks of subcutaneous and submucosal edema. Though a majority of patients will experience symptom onset before 20 years of age, there is a paucity of published literature regarding the management of HAE in children and adolescents. Methods: A comprehensive literature review regarding the management of pediatric HAE due to C1-esterase inhibitor deficiency (C1 INH) was performed. Results: A collection of case reports and case series suggest antifibrinolytics and attenuated androgens at low dose are safe and effective options for short- and long-term prophylaxis in pediatric HAE. Plasma-derived C1 INH preparations are available for both on-demand and prophylactic therapy in the United States, and post-hoc analyses of the pediatric patients enrolled in the larger clinical trials support their use in pediatric HAE. Ecallantide and icatibant are kinin-pathway modulators, but only ecallantide is currently approved for use in children 12 years of age and older in the United States. There was no available literature regarding the use of icatibant in pediatric HAE, but ecallantide's efficacy and tolerability were demonstrated in a post-hoc analysis of pediatric patients that participated in four larger, prospective studies. Conclusions: Within the limits of the present literature review, the currently available and approved therapies used in the treatment of adults with HAE also appear safe and effective for use in children and adolescents at appropriate doses. Options available in the United States include plasma-derived C1-INH, attenuated androgens, and antifibrinolytics for prophylactic prevention and plasma-derived C1 INH and ecallantide for on-demand, acute therapy.