From the Guidelines
Hereditary angioedema (HAE) management requires both acute treatment of attacks and long-term prophylaxis, with a focus on reducing morbidity, mortality, and improving quality of life. For acute attacks, first-line treatments include C1 esterase inhibitor concentrates, icatibant, or ecallantide, which should be administered as early as possible during an attack 1.
Acute Treatment
- C1 esterase inhibitor concentrates (Berinert or Cinryze, 20 units/kg IV)
- Icatibant (Firazyr, 30 mg subcutaneously)
- Ecallantide (Kalbitor, 30 mg subcutaneously)
Long-term Prophylaxis
- Lanadelumab (Takhzyro, 300 mg subcutaneously every 2 weeks)
- Berotralstat (Orladeyo, 150 mg orally daily)
- C1 inhibitor concentrates (Cinryze 1000 units IV twice weekly or Haegarda 60 units/kg subcutaneously twice weekly)
Workup for HAE
- C4 levels (typically low in HAE)
- C1 inhibitor antigen levels
- C1 inhibitor functional assays
- Genetic testing for mutations in factor XII, plasminogen, or angiopoietin-1 for HAE with normal C1 inhibitor
Patient Education and Follow-up
- All patients should have an emergency action plan and carry on-demand medication
- Education about attack triggers, including stress, trauma, infections, and certain medications like ACE inhibitors and estrogen-containing products
- Regular follow-up every 6-12 months to assess disease control and medication effectiveness 1
The goal of HAE management is to reduce the frequency and severity of attacks, and to improve the patient's quality of life, by using the most effective and safe treatments available, based on the latest evidence and guidelines.
From the FDA Drug Label
The safety and efficacy of KALBITOR to treat acute attacks of hereditary angioedema in adolescents and adults were evaluated in 2 randomized, double-blind, placebo-controlled trials (EDEMA4 and EDEMA3) in 168 patients with HAE Patients having an attack of hereditary angioedema, at any anatomic location, with at least 1 moderate or severe symptom, were treated with 30 mg subcutaneous KALBITOR or placebo. KALBITOR should only be administered by a healthcare professional with appropriate medical support to manage anaphylaxis and hereditary angioedema.
The management and workup for Hereditary Angioedema (HAE) involves:
- Diagnosis: Identifying patients with HAE who are experiencing an acute attack with at least 1 moderate or severe symptom.
- Treatment: Administering 30 mg of KALBITOR subcutaneously, which can be repeated within a 24-hour period if the attack persists.
- Monitoring: Closely monitoring patients for hypersensitivity reactions, including anaphylaxis, due to the similarity in symptoms between hypersensitivity reactions and HAE.
- Medical Support: Ensuring that KALBITOR is administered in a setting equipped to manage anaphylaxis and HAE, with appropriate medical support available 2, 2, 2.
From the Research
Management of Hereditary Angioedema (HAE)
The management of HAE due to C1-esterase inhibitor deficiency (C1-INH-HAE) involves several approaches, including:
- Treatment of acute angioedema attacks (on-demand treatment)
- Short-term (preprocedure) prophylaxis
- Long-term prophylaxis 3, 4, 5
Treatment of Acute Angioedema Attacks
The following drugs are available for the treatment of acute angioedema attacks:
- Purified plasma-derived human C1 esterase inhibitor concentrate
- Icatibant acetate
- Ecallantide
- Recombinant human C1 esterase inhibitor 3, 4, 5 These drugs are all authorized for self-administration, except ecallantide 3.
Short-term Prophylaxis
Purified plasma-derived human C1 esterase inhibitor concentrate is the treatment of choice for short-term prophylaxis 3, 4.
Long-term Prophylaxis
The following drugs can be used for long-term prophylaxis:
- Tranexamic acid
- Danazol
- Intravenous and subcutaneous nanofiltered purified plasma-derived human C1 esterase inhibitor concentrate
- Lanadelumab 3, 4, 6 Attenuated androgens, such as stanozolol, oxandrolone, and danazol, are also commonly used for long-term prophylaxis 4, 5, 6.
Other Treatment Options
Fresh frozen plasma is also available for treating acute attacks, particularly in emergency situations where other treatments are not available 7.