Podcast
Questions and Answers
What is a common side effect of ε-Aminocaproic Acid (EACA) over time?
What is a common side effect of ε-Aminocaproic Acid (EACA) over time?
Which agent is a kallikrein inhibitor given subcutaneously for patients aged 16 years and older?
Which agent is a kallikrein inhibitor given subcutaneously for patients aged 16 years and older?
What is the age range for which Icatibant is approved for acute treatment?
What is the age range for which Icatibant is approved for acute treatment?
When are all treatments for HAE most effective?
When are all treatments for HAE most effective?
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How long after treatment do noticeable effects typically occur?
How long after treatment do noticeable effects typically occur?
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What is a characteristic of tranexamic acid compared to ε-Aminocaproic Acid (EACA)?
What is a characteristic of tranexamic acid compared to ε-Aminocaproic Acid (EACA)?
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Which of the following is NOT administered intravenously?
Which of the following is NOT administered intravenously?
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What is the purpose of Berinert?
What is the purpose of Berinert?
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What is the main difference between tranexamic acid and ε-Aminocaproic Acid (EACA)?
What is the main difference between tranexamic acid and ε-Aminocaproic Acid (EACA)?
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How does the timing of treatment affect its effectiveness?
How does the timing of treatment affect its effectiveness?
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Study Notes
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.
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Description
Learn about the comprehensive treatment approach for Hereditary Angioedema, a rare genetic disorder characterized by recurrent episodes of swelling. Understand the goals and management of acute attacks and prevention of future attacks.
