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Table 1 Current therapeutics for long-term prophylaxis in HAE

From: An open-label study to evaluate the long-term safety and efficacy of lanadelumab for prevention of attacks in hereditary angioedema: design of the HELP study extension

Prophylactic treatment Route of administration Dosing frequency Efficacy Safety
Plasma-derived C1-INH Intravenous Every 3–4 days Effective in reducing attack rate [17, 18]
Breakthrough attacks still reported [17, 18]
Risk of thromboembolic events [19]
No safety concerns in patients treated with up to 2500 U [20]
Androgens Oral Daily Treatment associated with significantly fewer and less severe attacks [4]
Breakthrough attacks still reported [21]
Most common side effects: weight gain, virilization, headaches, myalgia, mood changes, menstrual disorders, liver dysfunction, increased serum lipids [4]
Contraindicated in pediatrics and during pregnancy [1]
Tranexamic acid Oral Daily Not licensed for long-term prophylaxis, although often used for this indication [1]
Efficacy unconfirmed. In 12 patients with HAE, 50% had some improvement in HAE attack rate [22]
Gastrointestinal upset, myalgia, theoretical risk of thrombosis [1]
  1. C1-INH C1 inhibitor, HAE hereditary angioedema