Recombinant C1-INH Is Effective for Hereditary Angioedema

For patients with hereditary angioedema (HAE) and frequent attacks, available prophylactic options include attenuated androgens, antifibrinolytics, and human plasma–derived C1 esterase–inhibitor (C1-INH) concentrate. Twice-weekly C1-INH intravenous infusions (1000 units) can reduce attacks by 50%, whereas subcutaneous twice-weekly injections (60 units/kg) can reduce attacks by as much as 95% (NEJM JW Gen Med May 1 2017 and N Engl J Med 2017; 376:1131); the subcutaneous form recently was FDA approved. In this international industry-sponsored crossover trial, researchers examined whether a recombinant version of C1-INH — already FDA approved for treating acute attacks — also is effective for prevention.

Twenty-six patients with HAE and frequen…