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Table 3 Summary of prophylactic treatments

From: Assessment and management of disease burden and quality of life in patients with hereditary angioedema: a consensus report

Generic name (trade name) Dosage Mechanism Approval status Efficacya Potential adverse events
Plasma-derived C1-INH (Cinryze®) [35, 85] Adults and adolescents: 1000 U IV every 3 or 4 days
Children: 500 U IV every 3 or 4 days
C1-INH Approved for prophylaxis in adults, adolescents, and pediatric patients aged ≥ 6 years Normalized attack rate was 6.26 attacks/12 weeks compared with 12.73 with placebo Rash, lightheadedness, fever
Plasma-derived C1-INH (Haegarda®) [34, 87] 60 IU/kg SC twice weekly C1-INH Approved for prophylaxis in adults and adolescents Mean number of attacks/month was 0.52 compared with 4.03 with placebo Injection site reaction, hypersensitivity, nasopharyngitis, dizziness
Lanadelumab (Takhzyro®) [33, 88] 300 mg SC Q2W Dosing Q4W may be considered in patients with favorable response after 6 months Plasma kallikrein inhibitor (monoclonal antibody) Approved for prophylaxis in adults and adolescents Mean number of attacks/month was 0.26 compared with 1.97 with placebo Injection site reaction, dizziness
Berotralstat (Orladeyo®) [36, 89] 150 mg oral QD Plasma kallikrein inhibitor Approved for prophylaxis in adults and pediatric patients aged ≥ 12 years Mean attack rate of 1.31 attacks/month compared with 2.35 attacks/month with placebo Abdominal pain, vomiting, diarrhea, back pain
Danazol (Danocrine®) [95, 96] 200 mg oral QD Dose should be titrated to the lowest clinically effective dose 17-alpha-alkylated androgen, Mechanism unknown Approved for the prevention of attacks of angioedema in adults Attacks occurred in 2.2% of danazol courses compared with 93.6% of placebo courses Weight gain, virilization, acne, menstrual abnormalities, muscle pains, headaches, fatigue, nausea, hypertension
Tranexamic acid (Lysteda™) [28, 97, 98] 30–50 mg/kg QD Antifibrinolytic Not FDA approved Of the 12 patients with C1-INH treated with tranexamic acid over 6 months, 6 experienced no reduction in HAE attacks, 3 experienced a moderate reduction, and 3 experienced a large reduction (> 75%) Gastrointestinal events, myalgia/creatine kinase elevation, risk of thrombosis
  1. C1-INH, C1-esterase inhibitor; FDA, Food and Drug Administration; IV, intravenous; NDA, new drug application; Q2W, every 2 weeks; Q4W, every 4 weeks; QD, once daily; SC, subcutaneous. aDifferences in trial design and populations limit cross-trial comparisons