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

Table 1 Current therapeutics for long-term prophylaxis in HAE

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]

ISSN: 2045-7022