Hereditary angioedema: is there a better future for treatment?

Hereditary angioedema is a rare disease that is associated with unpredictable, recurrent attacks of potentially life-threatening angioedema. The goal of treatment is complete control of disease, allowing patients to lead healthy lives. 1 Maurer M Magerl M Betschel S et al. The international WAO/EAACI guideline for the management of hereditary angioedema—the 2021 revision and update. Allergy. 2022; 77: 1961-1990 Crossref PubMed Scopus (107) Google Scholar Over the past 20 years, management of patients with type I and type II hereditary angioedema has substantially improved. Previously, partially effective prophylactic treatments, such as antifibrinolytic drugs, or drugs that induced serious side-effects, such as danazol, were used. 2 Busse PJ Christiansen SC Hereditary angioedema. N Engl J Med. 2020; 382: 1136-1148 Crossref PubMed Scopus (188) Google Scholar Intravenous plasma-derived C1 inhibitors were the first new class of targeted prophylactic therapies for the treatment of hereditary angioedema. In a prospective, double-blind, placebo-controlled, randomised, crossover trial of 24 patients aged 6 years or older, intravenous nanofiltered C1 inhibitor concentrate treatment every 3 or 4 days significantly reduced the number of hereditary angioedema attacks. 3 Zuraw BL Busse PJ White M et al. Nanofiltered C1 inhibitor concentrate for treatment of hereditary angioedema. N Engl J Med. 2010; 363: 513-522 Crossref PubMed Scopus (374) Google Scholar Treatment could be self-administered, including by indwelling ports, for many patients. Subcutaneous treatments have also been approved. 4 Longhurst H Cicardi M Craig T et al. Prevention of hereditary angioedema attacks with a subcutaneous C1 inhibitor. N Engl J Med. 2017; 376: 1131-1140 Crossref PubMed Scopus (164) Google Scholar Efficacy and safety of garadacimab, a factor XIIa inhibitor for hereditary angioedema prevention (VANGUARD): a global, multicentre, randomised, double-blind, placebo-controlled, phase 3 trialMonthly garadacimab administration significantly reduced hereditary angioedema attacks in patients aged 12 years and older compared with placebo and had a favourable safety profile. Our results support the use of garadacimab as a potential prophylactic therapy for the treatment of hereditary angioedema in adolescents and adults. Full-Text PDF