Evidence for the treatment of hereditary angioedema (HAE) in younger children is growing, with expanding treatment options.
A pasteurized formulation of purified C1 inhibitor (Berinert) was approved in 2009 for use in patients ages 12 years and older with acute HAE attacks, while prophylactic treatment with pasteurized, nanofiltered C1 inhibitor concentrate (Cinryze) has been available for adolescents and adults since 2008.
"Together, these developments mark a major turning point in the management of patients with HAE," wrote William R. Lumry, MD, of the AARA Research Center in Dallas, in a review in the American Journal of Managed Care. "In many cases, new treatments have been life altering, allowing people with the condition to pursue academic, professional, and personal interests for the first time in their lives."
The indication for those early agents expanded down to children as young as 2 years in acute treatment and as young as 6 years in prevention in 2017 and 2018, respectively, and others have followed.
However, a 2022 Cochrane review on long-term prevention agents lamented the dearth of evidence for children ages 10 years and under. Those researchers found only one study reporting on children specifically, with just 12 participants who were 6 to 11 years old; none of the studies that included both children and adults presented results by age. One post‐hoc analysis of kids ages 12 to 17 years included just nine randomized to lanadelumab (Takhzyro) or placebo -- "too low to make definitive statements," the authors wrote, adding that they "cannot be sure if the drugs are more or less effective in ... children versus adults."
