Treatment of hereditary angioedema in the pediatric patient

Hereditary angioedema (HAE) is an autosomal dominant disease, with the expectation that there is a risk of 50%, if a parent has HAE, of inheriting the disease. Manifestations of the disease start early in life for some patients, but symptoms frequently increase during the transition from childhood to adolescence. Unfortunately, most medications are not approved for use in children. This requires off-label use for most of the medications that are U.S. Food and Drug Administration approved for HAE. For this reason, we have sought to identify the use of medications approved and unapproved and used off label for children with HAE.