Hereditary Angioedema Treatment

Hereditary Angioedema (HAE) Treatment Overview

• HAE is a rare genetic disorder characterized by recurrent episodes of swelling (angioedema) in various body parts, including the skin, gastrointestinal tract, and upper airway.
• Treatment aims to manage acute attacks, prevent future attacks, and improve patients' quality of life.

Treatment Goals

• Restore a normal quality of life.
• Individualize treatment based on patient-specific factors.

Types of Treatment

• On-Demand Treatment: used when an attack starts, addressing acute symptoms.
• Short-Term Prophylaxis: administered before known triggers (e.g., surgery, dental procedures) to prevent attacks during high-risk situations.
• Long-Term Prophylaxis: prevents recurrent attacks over an extended period.

Long-Term Prophylaxis Options

• Cinryze (Intravenous Plasma-Derived C1-INH Concentrate):
  • Given twice a week.
  • Approved for adolescents and adults.
  • Short half-life (about 40 hours).
• Subcutaneous C1-INH Concentrate:
  • Approved for adolescents and adults.
  • Given twice a week.
• Lanadelumab (Monoclonal Antibody):
  • Inhibits plasma kallikrein.
  • Subcutaneous administration once every 2-4 weeks.
• Berotralstat (Oral Pill):
  • Inhibits plasma kallikrein.
  • Taken once a day.
• Garadacimab (Recombinant Monoclonal Antibody):
  • Targets activated factor XII.
  • Efficacy as a prophylactic agent.

Patient-Specific Considerations

• Treatment choice depends on factors such as patient preference, access to emergency care, and individual needs.

Fibrinolysis Inhibitors and Other Novel Treatments

• ε-Aminocaproic Acid (EACA):
  • Used in children, but limited by severe fatigue and muscle weakness over time.
• Tranexamic Acid:
  • A cyclized analog of EACA, more effective and less toxic.
  • Widely used in Europe, but less so in the United States due to side effects and other treatment options.

FDA-Approved On-Demand Treatments for HAE

• Berinert:
  • A purified C1-inhibitor product administered intravenously (20 U/kg) for acute attacks.
• Ecallantide:
  • A kallikrein inhibitor given subcutaneously for patients aged 16 years and older.
• Icatibant:
  • A bradykinin type 2 receptor antagonist approved for acute treatment in patients aged 18 years and older.
• Recombinant C1-INH:
  • An intravenous product approved for acute attacks in adolescents and adults.

Effectiveness and Timing

• All treatments are most effective when administered early during an attack.
• Noticeable effects typically occur 1-4 hours after treatment.

Hereditary Angioedema (HAE) Treatment Overview

• HAE is a rare genetic disorder characterized by recurrent episodes of swelling (angioedema) in various body parts, including the skin, gastrointestinal tract, and upper airway.
• Treatment aims to manage acute attacks, prevent future attacks, and improve patients' quality of life.

Treatment Goals

• Restore a normal quality of life.
• Individualize treatment based on patient-specific factors.

Types of Treatment

• On-Demand Treatment: used when an attack starts, addressing acute symptoms.
• Short-Term Prophylaxis: administered before known triggers (e.g., surgery, dental procedures) to prevent attacks during high-risk situations.
• Long-Term Prophylaxis: prevents recurrent attacks over an extended period.

Long-Term Prophylaxis Options

• Cinryze (Intravenous Plasma-Derived C1-INH Concentrate):
  • Given twice a week.
  • Approved for adolescents and adults.
  • Short half-life (about 40 hours).
• Subcutaneous C1-INH Concentrate:
  • Approved for adolescents and adults.
  • Given twice a week.
• Lanadelumab (Monoclonal Antibody):
  • Inhibits plasma kallikrein.
  • Subcutaneous administration once every 2-4 weeks.
• Berotralstat (Oral Pill):
  • Inhibits plasma kallikrein.
  • Taken once a day.
• Garadacimab (Recombinant Monoclonal Antibody):
  • Targets activated factor XII.
  • Efficacy as a prophylactic agent.

Patient-Specific Considerations

• Treatment choice depends on factors such as patient preference, access to emergency care, and individual needs.

Fibrinolysis Inhibitors and Other Novel Treatments

• ε-Aminocaproic Acid (EACA):
  • Used in children, but limited by severe fatigue and muscle weakness over time.
• Tranexamic Acid:
  • A cyclized analog of EACA, more effective and less toxic.
  • Widely used in Europe, but less so in the United States due to side effects and other treatment options.

FDA-Approved On-Demand Treatments for HAE

• Berinert:
  • A purified C1-inhibitor product administered intravenously (20 U/kg) for acute attacks.
• Ecallantide:
  • A kallikrein inhibitor given subcutaneously for patients aged 16 years and older.
• Icatibant:
  • A bradykinin type 2 receptor antagonist approved for acute treatment in patients aged 18 years and older.
• Recombinant C1-INH:
  • An intravenous product approved for acute attacks in adolescents and adults.

Effectiveness and Timing

• All treatments are most effective when administered early during an attack.
• Noticeable effects typically occur 1-4 hours after treatment.