Abstract
: Agents for prophylaxis of hereditary angioedema (HAE) have been available in the United States for several decades, but their usefulness is limited by side effects and they cannot be used at all in some patients. No agents have been available in the United States to specifically treat acute attacks. HAE types I and II are associated with low functional levels of C1 inhibitor, and evidence accumulated over decades suggests that intravenous infusion of C1 inhibitor is useful for terminating angioedema attacks and for prophylaxis. : C1 inhibitor derived from pooled human plasma has been available for decades in Europe, and 2 preparations have been recently introduced into the United States. Both have been efficacious in carefully controlled double-blind studies. One preparation, Cinryze, was approved by the U.S. Food & Drug Administration (FDA) for prophylaxis of HAE attacks in October 2008, and the second, Berinert, was approved by the FDA for treatment of acute attacks in October 2009. A third preparation, the recombinant human C1 inhibitor Rhucin, is completing clinical trials. Although not yet approved by the FDA, preliminary results made available suggest that Rhucin, too, is effective in terminating attacks of HAE. : Americans will now have access to effective acute and prophylactic treatments with C1 inhibitor for hereditary angioedema.
Highlights
OF C1 INHIBITOR INTO THE AMERICAN MARKETSeveral years ago, Behring and Sanquin made the decision to bring their European plasma-derived products to the United States
The author’s own group had access to the American Red Cross preparation and in 1980 reported that C1 inhibitor infusion raised plasma C1 inhibitor levels as expected in 8 patients and, as a consequence of its action in inhibiting activated C1, raised the levels of serum C4.14 We reported that this treatment terminated angioedema attacks in 5 of our patients who were having attacks at the time of their infusion.[14]
The pharmaceutical company Lev was organized in the United States to test and bring to market the Sanquin product
Summary
Agents for prophylaxis of hereditary angioedema (HAE) have been available in the United States for several decades, but their usefulness is limited by side effects and they cannot be used at all in some patients. Key Points: C1 inhibitor derived from pooled human plasma has been available for decades in Europe, and 2 preparations have been recently introduced into the United States Both have been efficacious in carefully controlled double-blind studies. S30 preparation in a double-blind study reported in 1996 and demonstrated that it was effective for both prophylaxis and acute treatment of angioedema attacks[17]; this product is no longer available. Many patients began to recover at about 30 minutes, and by 1 hour after treatment began most patients were well on the road to recovery.[19]
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