Hereditary angioedema (HAE) results from a genetic deficiency of C1-inhibitor. C1-inhibitor has been produced in glycosylated form in the milk of transgenic rabbits.
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New therapies for hereditary angioedema (HAE) (click to enlarge the image).
Angioedema (AE) can be allergic or non-allergic.
There are 5 types of non-allergic angioedema (AE):
- acquired AE
- hereditary AE (HAE)
- ACE-inhibitor induced AE
- idiopathic AE, can occur with chronic urticaria
- pseudoallergic AE, e.g. reaction to NSAIDs
There are 3 types of HAE that are differentiated by C4 and C1-INH levels
- type I HAE - low C4, low C1-INH function, low C1-INH antigen level
- type II HAE - low C4, low C1-INH function, normal C1-INH antigen level
- type III HAE - all normal
Patients with an eligible attack were randomized to a single intravenous dose of recombinant human C1-inhibitor (rhC1INH) or saline.
Therapeutic failure occurred in 59% of the saline group compared with 0% of the 50 U/kg group and 10% of the 100 U/kg group. No postexposure antibody responses against rhC1INH were observed.
Administration of rhC1INH at 100 or 50 U/kg was highly effective as a treatment of acute attacks in patients with HAE. It was also safe and well tolerated.
Treatment of acute HAE attacks
- C1-INH, 20 units/kg, IV infusion
- Icatibant, 30 mg SC, bradykinin B2 receptor antagonist
- Ecallantide, 30 mg SC, kallikrein receptor antagonist
Prophylaxis of HAE attacks
- C1-INH, 1,000 units, IV infusion every 3-4 days
- attenuated androgen, e.g. danocrine 200 mg PO TID
References
New Directions in the Treatment of Angioedema. Medscape, 2012.
Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010 Oct;126(4):821-827.e14.
HAE: annual drug cost alone for prophylactic C1 esterase inhibitor is $450k - nearly $5 mln for every decade of life http://goo.gl/BCVtu
Optimal efficacy of C1INH therapy in HAE is achieved at doses ≥50 U/kg, target level ≥0.7 U/ml (70% of normal) http://goo.gl/HJM4X
.jpg)
New therapies for hereditary angioedema (HAE) (click to enlarge the image).
Angioedema (AE) can be allergic or non-allergic.
There are 5 types of non-allergic angioedema (AE):
- acquired AE
- hereditary AE (HAE)
- ACE-inhibitor induced AE
- idiopathic AE, can occur with chronic urticaria
- pseudoallergic AE, e.g. reaction to NSAIDs
There are 3 types of HAE that are differentiated by C4 and C1-INH levels
- type I HAE - low C4, low C1-INH function, low C1-INH antigen level
- type II HAE - low C4, low C1-INH function, normal C1-INH antigen level
- type III HAE - all normal
Patients with an eligible attack were randomized to a single intravenous dose of recombinant human C1-inhibitor (rhC1INH) or saline.
Therapeutic failure occurred in 59% of the saline group compared with 0% of the 50 U/kg group and 10% of the 100 U/kg group. No postexposure antibody responses against rhC1INH were observed.
Administration of rhC1INH at 100 or 50 U/kg was highly effective as a treatment of acute attacks in patients with HAE. It was also safe and well tolerated.
Treatment of acute HAE attacks
- C1-INH, 20 units/kg, IV infusion
- Icatibant, 30 mg SC, bradykinin B2 receptor antagonist
- Ecallantide, 30 mg SC, kallikrein receptor antagonist
Prophylaxis of HAE attacks
- C1-INH, 1,000 units, IV infusion every 3-4 days
- attenuated androgen, e.g. danocrine 200 mg PO TID
References
New Directions in the Treatment of Angioedema. Medscape, 2012.
Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010 Oct;126(4):821-827.e14.
HAE: annual drug cost alone for prophylactic C1 esterase inhibitor is $450k - nearly $5 mln for every decade of life http://goo.gl/BCVtu
Optimal efficacy of C1INH therapy in HAE is achieved at doses ≥50 U/kg, target level ≥0.7 U/ml (70% of normal) http://goo.gl/HJM4X