PURPOSE OF THE STUDY.
To determine the efficacy of nanofiltered C1 inhibitor concentrate in the management of hereditary angioedema (HAE).
Subjects were from 2 studies; the lowest age was 6 years (median age: 36 years).
Both studies compared an intravenous dose of 1000 U of nanofiltered C1 inhibitor concentrate in the management of HAE. The first study end point was the time to unequivocal relief of symptoms during an acute attack of HAE. The second study was a crossover trial that involved 22 subjects; prophylactic twice-weekly infusions of drug (1000 U or placebo) was given during two 12-week periods. The primary end point in the second study was the number of attacks in the 12-week treatment period compared with placebo.
In the first study, the mean time of the onset of unequivocal relief of an attack was 2 hours in treated subjects compared with 4 hours in those given placebo (P = .02). In the second study, the number of attacks per 12-week period was 6.2 with active drug compared with 12.73 with placebo (P < .001). The treated subjects also had significant reduction in both the severity and duration of the attacks.
When given as treatment, the nanofiltered C1 inhibitor concentrate shortened the duration of acute attacks. When used for prophylaxis, the treatment reduced the frequency of acute attacks.
This treatment allows for at least a 50% reduction in the frequency and duration of HAE attacks. In patients with less-severe clinical presentation for whom continuous treatment is not necessary, 2 preparations that are available for as-needed use are reported in the same issue of the journal (Cicardi M, Levy RJ, McNeil DL, et al. N Engl J Med. 2010;363:523–531; and Cicardi M, Banerji A, Bracho F, et al. N Engl J Med. 2010;363:532–541). Both are subcutaneous preparations given within 8 hours of attack onset. Both preparations showed significant efficacy with a <5-hour onset for relief of acute symptoms. An accompanying editorial provided important perspective regarding the efficacy of these treatments (Morgan BP. N Engl J Med. 2010;363:581–583). These 2 studies allowed for therapy of HAE specific for the burden of the illness for each individual patient in a rare but potentially lethal disease.
- Copyright © 2011 by the American Academy of Pediatrics