| Prophylactic therapy | Advantages | Disadvantages |
| C1 inhibitor concentrate – Intravenous | - Well tolerated
- Widely available
| - Requires reliable venous access (but indwelling ports are not recommended)
|
| C1 inhibitor concentrate – Subcutaneous | - Convenient – Subcutaneous injection every 3 to 4 days
- Approved for children aged 6 years and older in the US, approved for adolescents in Europe
| |
| Lanadelumab | - Convenient – Subcutaneous injection every 2 to 4 weeks
- Approved for children aged 2 years and older in Europe and the US
| |
| Berotralstat | - Convenient – Once-daily oral administration
- Effective in the majority of patients
| - Clinical experience is limited
- Not approved for use in children younger than 12 years of age
- Avoid in patients on a P-gp inducer
- Caution in patients on P-gp or BCRP inhibitors or CYP2D6, CYP3A4, or P-gp substrates
|
| Androgens | - Oral administration
- Effective in most patients
- Inexpensive
| - Side effects are prohibitive in some patients (hypercholesterolemia, liver enzyme abnormalities, virilization, hypertension, mood effects, weight gain, others)
- Avoid in prepubertal children (premature closure of growth plate) and in pregnancy (virilization of female fetus)
- Avoid in patients with liver disease or nephrotic syndrome
|
| Antifibrinolytics – Tranexamic acid (preferred) or epsilon aminocaproic acid | - Oral administration
- Safe in children
- Well tolerated by most patients
- Inexpensive
| - Less effective than other therapies
- Avoid in patients with history of thrombosis or thromboembolic disease or risk factor for thrombosis
|
| Treatment for breakthrough symptoms (required in combination with any of the options above) |
| C1 inhibitor concentrate | | |
| Icatibant | - Subcutaneous injection
- Approved for self-administration at home
| |
| Ecallantide | | - Should be given under medical supervision due to possible hypersensitivity reactions usually within one hour
- Only available in the United States
|
| Plasma – Solvent/detergent-treated or fresh frozen | - Widely available
- Inexpensive
| - Efficacy not as well established as other therapies
- Theoretical risk of transmission of blood-borne agents
- Requires visit to hospital or clinic for administration
|
