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Table 1 Prophylactic treatments for HAE

From: Attenuated androgen discontinuation in patients with hereditary angioedema: a commented case series

Drug name Mode of action Administration Self-administration Safety eventsa,b
Plasma-derived C1-INH (pdC1-INH) [7] C1-INH replacement Intravenous (IV) Yes Very common: headache; nausea
Common: hypersensitivity; dizziness; vomiting; rash, erythema, pruritus; injection site rash/erythema, infusion site pain, pyrexia
Uncommon: hyperglycaemia; venous thrombosis, phlebitis, venous burning, hot flush; cough; diarrhoea, abdominal pain; contact dermatitis; joint swelling, arthralgia, myalgia; chest discomfort
pdC1-INH [8] C1-INH replacement Subcutaneous (SC) Yes Rare: development of thrombosis; rise in temperature, injection site reactions; allergic or anaphylactic-type reactions
Lanadelumab [9] Plasma kallikrein inhibition Subcutaneous (SC) Yes Very common: injection site reactions
Common: hypersensitivity; dizziness; maculopapular rash; myalgia; increased alanine aminotransferase, increased aspartate aminotransferase
Berotralstat [10] Plasma kallikrein inhibition Oral Yes Very common: headache; abdominal pain, diarrhoea
Common: vomiting, gastroesophageal reflux, flatulence; rash; alanine aminotransferase increases, aspartate aminotransferase increases
Attenuated androgensc (danazol, oxandrolone) [11,12,13,14,15,16,17,18] Unknown, but potentially through increased C1‑INH levels and/or metabolism of bradykinin Oral Yes Blood and lymphatic disorders: increase in red cell and platelet count; reversible polycythaemia, leucopenia, thrombocytopenia, eosinophilia, and splenic peliosis
Endocrine disorders: androgenic effects (acne, weight gain, increased appetite, seborrhoea, hirsutism, hair loss, voice change, which may take the form of hoarseness, sore throat or instability or deepening of pitch; hypertrophy of the clitoris, fluid retention); other endocrine effects (menstrual disturbances in the form of spotting, alteration of the timing of the cycle and amenorrhoea; flushing, vaginal dryness, changes in libido, vaginal irritation and reduction in breast size; modest reduction in spermatogenesis)
Metabolic and nutrition disorders: increased insulin resistance, increase in plasma glucagon, mild impairment of glucose tolerance; increase in low-density lipoprotein cholesterol, decrease in high-density lipoprotein cholesterol, affecting all subfractions, and decrease in apoliproteins AI and AII; induction of aminolevulinic acid synthetase, and reduction in thyroid binding globulin, T4, with increased uptake of T3, but without disturbance of thyroid stimulating hormone or free levothyroxine index
Psychiatric disorders: emotional lability, anxiety, depressed mood and nervousness
Nervous system disorders: dizziness, headache, vertigo, benign intracranial hypertension, migraine; aggravation of epilepsy, carpal tunnel syndrome
Eye disorders: visual disturbances, such as blurring of vision, difficulty in focusing, difficulty in wearing contact lenses and refraction disorders requiring correction
Respiratory, thoracic and mediastinal disorders: pleuritic pain, interstitial pneumonitis
Gastrointestinal disorders: nausea, epigastric pain
Cardiac disorders: hypertension, palpitations and tachycardia; thrombotic events, including saggital sinus, cerebrovascular thrombosis as well as arterial thrombosis; myocardial infarction
Hepatobiliary disorders: isolated increases in serum transaminase levels, cholestatic jaundice, benign hepatic adenomata and pancreatitis; peliosis hepatitis as well as malignant hepatic tumour observed with long term use; hepatocellular injury, hepatic failure, jaundice hepatocellular, hepatocellular focal nodular hyperplasia
Skin and subcutaneous tissue disorders: rashes, which may be maculopapular, petechial or purpuric and may be accompanied by fever, or may take an urticarial form and may be accompanied by facial oedema; sun-sensitive rash; inflammatory erythematosus nodules, changes in skin pigmentation, exfoliative dermatitis and erythema multiforme
Musculoskeletal and connective tissue disorders: backache and muscle cramps, which can be severe, with elevation of creatine phosphokinase levels; muscle tremors, fasciculation, limb pain, joint pain and joint swelling
Renal and urinary disorders: haematuria with prolonged use in patients with HAE
General disorders and administration site condition: fatigue
  1. a Listed as in summary of product characteristics (SPC)
  2. b Frequencies of events are categorized as: Very common (≥ 1/10); Common (≥ 1/100 – < 1/10); Uncommon (≥ 1/1000 to  < 1/100); Rare (≥ 1/10,000 – < 1/1000); Very rare (< 1/10,000) [7,8,9,10]
  3. c Event frequency not categorized in SPC [18]