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Choice of prophylactic agent for hereditary angioedema (HAE) in specific patient groups

Choice of prophylactic agent for hereditary angioedema (HAE) in specific patient groups
Patient population Preferred agents Alternate agents Agents to be avoided Other notes
Pre-pubertal children (male and female)
  • Plasma-derived C1-INH*
  • Tranexamic acid (less effective but may be sufficient for mild disease)
  • Androgens
Lanadelumab and berotralstat not studied or approved for children under 12 years of age
Adult women not considering pregnancy and post-pubertal girls
  • Plasma-derived C1-INH*
  • Lanadelumab
  • Berotralstat
  • Tranexamic acid (less effective but may be sufficient for mild disease)
  • Androgens (multiple side effects and virilization)
 
Adult men and post-pubertal boys
  • Plasma-derived C1-INH*
  • Lanadelumab
  • Berotralstat
  • Tranexamic acid (less effective but may be sufficient for mild disease)
  • Androgens (multiple side effects but virilization less of an issue for men)
   
Pregnant and lactating women
  • Plasma-derived C1-INH* (has most safety data)
  • Tranexamic acid (less effective but history of safe use)
  • AndrogensΔ
Lanadelumab and berotralstat not recommended because they have not been studied in pregnancy
The choice of which long-term prophylactic agent to use is influenced both by patient characteristics (age, gender, pregnancy/lactation), as shown in the table, as well as regulatory requirements in different countries.
HAE: hereditary angioedema; C1-INH: C1 inhibitor.
* Plasma-derived C1-INH can be given subcutaneously or intravenously. Subcutaneous is more convenient and appears to be more effective based on preliminary evidence.
¶ Androgens are contraindicated in pre-pubertal children because they can cause premature closure of the growth plates.
Δ Androgens are avoided in pregnancy because they can result in virilization of female fetuses, although if a woman with HAE is carrying a male fetus, androgens have been successfully used with supervision by an endocrinologist.
Graphic 127119 Version 2.0