Several approved treatments are now available for Hereditary Angioedema (HAE) attacks. A recent article highlights the effectiveness in 46 children who received 2237 infusions of nanofiltered C1-esterase inhibitor (C1 INH-nf) treatment (J Pediatr 2013; 162: 1017-22).
Background: HAE prevalence: 1 in 50,000. Though it is inherited in an autosomal dominant manner, up to 25% of case are de novo. HAE is characterized by recurrent episodic attacks of subcutaneous edema of the skin, GI tract or upper airway. Symptoms often present between 4 and 11 years of age. GI tract symptoms are often recurrent colicky abdominal pain. Triggers for attacks may include infections, emotional stress, and trauma (e.g. bodily contact from play).
This manuscript provides data from 4 prospective clinical trials of C1 INH-nf (Cinryze). Most children in this study responded within 30 minutes compared with 2 hours for placebo. Therapy also reduced the rate of attacks. One interesting aspect of the study was its lack of any mention of cost.
From UptoDate: “Cost of first-line therapies — All first-line therapies for acute attacks of HAE are costly. In the US, the cost of one treatment with C1INHRP,ecallantide, or icatibant ranged from $5000 to $10,000.” Hereditary angioedema: Treatment of acute attacks – UpToDate
From a Hereditary Angioedema – RegenceRx website: “As a preventative therapy, C1 esterase inhibitor (Cinryze) is significantly more costly than other formulary alternatives (~$500,000 per year; ~250 times more than danazol). For acute treatment of an HAE attack, C1 esterase inhibitor (Berinert) and ecallantide (Kalbitor) costs approximately $6,000 to $10,000 per attack. Cost may double if re- treatment is needed within 24 hours.”
Additional reference:
- -Eur J Pediatr 2012; 171: 1339-48. Consensus of therapeutic strategies for HAE.
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