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Efficacy and safety of an intravenous C1-inhibitor concentrate for long-term prophylaxis in hereditary angioedema

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Background:

The plasma-derived, pasteurized, nanofiltered C1-inhibitor concentrate (pnfC1-INH) is approved in the United States as an intravenous (IV) on-demand treatment for hereditary angioedema (HAE) attacks, and, in Europe, as on demand and short-term prophylaxis.

Objective:

This analysis evaluated Berinert Patient Registry data regarding IV pnfC1-INH used as long-term prophylaxis (LTP).

Methods:

The international registry (2010‐2014) collected prospective and retrospective usage, dosing, and safety data on individuals who used pnfC1-INH for any reason.

Results:

The registry included data on 47 subjects (80.9% female subjects; mean age, 44.8 years), which reflected 4082 infusions categorized as LTP and a total of 430.2 months of LTP administration. The median absolute dose of pnfC1-INH given for LTP was 1000 IU (range, 500‐3000 IU), with a median time interval between infusion and a subsequent pnfC1-INH‐treated attack of 72.0 hours (range, 0.0‐166.4 hours). Fifteen subjects (31.9%) had no pnfC1-INH‐treated HAE attacks within 7 days after pnfC1-INH infusion for LTP; 32 subjects (68.1%) experienced 246 attacks, with rates of 0.06 attacks per infusion and 0.57 attacks per month. A total of 81 adverse events were reported in 16 subjects (34.0%) (0.02 events per infusion; 0.19 events per month); only 3 adverse events were considered related to pnfC1-INH (noncardiac chest pain, postinfusion headache, deep vein thrombosis in a subject with an IV port).

Conclusion:

In this international registry, IV pnf-C1-INH given as LTP for HAE was safe and efficacious, with a low rate of attacks that required pnfC1-INH treatment, particularly within the first several days after LTP administration.
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Keywords: Berinert; dosing; efficacy; hereditary angioedema; long-term prophylaxis; plasma-derived C1-inhibitor; registry; safety; self-administration; thromboembolic events

Document Type: Research Article

Affiliations: 1Department of Medicine and Pediatrics, Pennsylvania State University, Hershey Medical Center, Hershey, Pennsylvania, USA

Publication date: 01 March 2017

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