What HAE acute treatment has the most documented use in children?
There have been several studies on the main dose, but the most decisive have been the IMPACT-1 and its extension the IMPACT-2. In the IMPACT-1 included 124 patients (aged 6-72 years) treated in every crisis of angioedema (abdominal or facial) with 10 U/kg, 20 U/kg or placebo.
How is C1 esterase inhibitor deficiency diagnosed?
HAE is diagnosed by the finding of low C1 esterase inhibitor level or function….Present with:
- Stridor or respiratory distress due to laryngeal oedema.
- Other signs of potential airway compromise eg hoarse voice, persistent cough, dysphagia or tongue swelling.
- Severe abdominal pain or vomiting.
Why are ACE inhibitors contraindicated in C1 esterase deficiency?
C1 esterase is a protein inhibitor; it inhibits the complement system to prevent spontaneous activation of the complement system. Without the C1 esterase inhibitor, there is unchecked activation of C1, C2, and C4 complement proteins before other inhibitors can stop the cascade.
What is C1 esterase inhibitor deficiency?
Hereditary angioedema, otherwise known as C1 esterase deficiency, is defined by recurrent episodes of angioedema without urticaria or pruritus. These skin conditions typically involve the legs, hands, face, upper respiratory tract, as well as gastrointestinal tract.
What is the treatment for hereditary angioedema?
In HAE types I and II, the treatment of choice in acute attacks consists of replacement with commercially available C1 inhibitor (C1-INH) concentrates or kallikrein inhibitor or, if those are unavailable, fresh-frozen plasma. In HAE with normal C1 inhibitor levels, infusion of C1-INH has proven to be ineffective.
What is C1 esterase inhibitor used for?
C1 esterase inhibitor is used to treat or prevent hereditary angioedema (HAE).
What does C1 esterase inhibitor do?
C1 esterase inhibitor is used to treat or prevent hereditary angioedema (HAE). HAE is a rare disease that causes swelling of the face, hands, feet, throat, stomach, bowels, or genitals. People who have HAE have low levels of C1 esterase inhibitor in their body.
What is hereditary angioedema?
Hereditary angioedema is a disorder characterized by recurrent episodes of severe swelling (angioedema). The most common areas of the body to develop swelling are the limbs, face, intestinal tract, and airway. Minor trauma or stress may trigger an attack, but swelling often occurs without a known trigger.
What is C1 esterase inhibitor deficiency (C1e)?
Background C1 esterase inhibitor deficiency [hereditary or acquired (HAE/AAE)] is characterized by the occurrence of subcutaneous and submucosal swellings in any part of the skin and the respiratory and gastrointestinal tracts. In the hereditary form, symptoms usually appear early in life and are normally accompanied by a family history.
What is the treatment for C1 esterase inhibitor deficiency with laryngeal edema?
Specific therapy for C1 esterase inhibitor deficiency with laryngeal edema involves replacing the deficient enzyme. The easiest way is to give human plasma, which contains C1 esterase inhibitor. An initial dose of human plasma is 2 units.
Is C1 inhibitor concentrate effective for long-term replacement in children?
C1 inhibitor concentrate in children C1 inhibitor concentrate has been used successfully for long-term replacement in selected adult patients [124], and more recently it has been shown to be superior to a placebo in a double-blind controlled study [76].
What is the role of C-1 inhibitors in hereditary angioedema?
C-1 inhibitor: evidence for decreased hepatic synthesis in hereditary angioedema. Science. 1971;173:553–4. [PubMed] [Google Scholar]