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JACI Highlights - October 2009A dosing recommendation for C1 inhibitor used to treat acute attacks in hereditary angioedemaHereditary angioedema (HAE) is an autosomal dominant disease caused by a functional or a quantitative deficiency in C1 esterase inhibitor (C1-INH) protein. This protein controls the contact system, and its deficiency leads to episodic activation of plasma kallikrein and factor XIIa, with a subsequent release of bradykinin and increase in vascular permeability. Patients experience these episodes as unpredictable and often painful bouts of well-circumscribed edema that can affect the abdomen, the face, the extremities, and rarely also the larynx. Where licensed, the treatment of choice is to replace the missing functional plasma protein with C1-INH concentrate, which has been used successfully for treating acute HAE attacks for more than 30 years. However, an effective weight-based dosing recommendation has not been available on the basis of a placebo-controlled study. In the October 2009 issue of The Journal of Allergy and Clinical Immunology, Craig and colleagues report a study comparing the efficacy and safety of C1-INH at doses of 10 and 20 U/kg to placebo in the treatment of acute abdominal and facial attacks. The patients reported efficacy outcomes in terms of onset of symptom relief and complete resolution of all symptoms. The researchers found that the 20-U/kg dose provided significantly more rapid relief (within 30 minutes) than placebo (90 minutes), which was not the case with the 10-U/kg dose (72 minutes). A similar trend was seen for the time to complete resolution and other secondary measures. The authors therefore concluded that 20 U/kg is a statistically secure dosing recommendation for C1-INH therapy to ensure rapid relief from the symptoms of acute attacks in HAE. “Efficacy of Human C1 Esterase Inhibitor Concentrate Compared to Placebo in Acute Hereditary Angioedema Attacks” by Craig et al. (JACI October 2009 Volume 124 No. 4) |
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