Safety and Usage of C1-Inhibitor in Hereditary Angioedema: Berinert Registry Data
Marc A. Riedl, Anette Bygum, William Lumry, Markus Magerl, Jonathan A. Bernstein, Paula Busse, Timothy Craig, Michael M. Frank, Jonathan Edelman, Debora Williams-Herman, Henrike Feuersenger, Mikhail Rojavin
(+36 others)
2016
Journal of Allergy and Clinical Immunology: In Practice
What is already known about this topic? The safety of plasma-derived C1-inhibitor (C1-INH; Berinert) has been well documented in studies in patients with hereditary angioedema (HAE) treated with recommended doses. Rare cases of thromboembolic events have been reported with C1-INH use, generally off-label and at supratherapeutic doses. What does this article add to our knowledge? This large, international, registry of patients using C1-INH is the most extensive of its kind, providing real-world
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... ata regarding general safety and intentional surveillance for issues of particular interest, including thromboembolism and possible viral transmission. How does this study impact current management guidelines? Recent HAE guidelines consistently recommend selfadministration of C1-INH. The Berinert registry data provide real-world evidence for widespread implementation of this practice and support the feasibility and safety of C1-INH administration outside of a health care setting. BACKGROUND: The plasma-derived, highly purified, nanofiltered C1-inhibitor concentrate (Berinert; "pnfC1-INH") is approved in the United States for treating hereditary angioedema (HAE) attacks and in many European countries for attack treatment and short-term prophylaxis. OBJECTIVE: The objective of this study was to describe safety and usage patterns of pnfC1-INH. METHODS: A multicenter, observational, registry was conducted between 2010 and 2014 at 30 United States and 7 European sites to obtain both prospective (occurring after enrollment) and retrospective (occurring before enrollment) safety and usage data on subjects receiving pnfC1-INH for any reason. RESULTS: Of 343 enrolled patients, 318 received 1 or more doses of pnfC1-INH for HAE attacks (11,848 infusions) or for prophylaxis (3142 infusions), comprising the safety population. Median dosages per infusion were 10.8 IU/kg (attack treatment) and 16.6 IU/kg (prophylaxis). Approximately 95% of infusions were administered outside of a health care setting. No adverse events (AEs) were reported in retrospective data. Among prospective data (n [ 296 subjects; 9148 infusions), 252 AEs were reported in 85 (28.7%) subjects (rate of 0.03 events/infusion); 9 events were considered related to pnfC1-INH. Two thromboembolic events were reported in subjects with thrombotic risk factors. No patient was noted to have undergone viral testing for suspected blood-borne infection during registry participation. CONCLUSIONS: The findings from this large, international patient registry documented widespread implementation of pnfC1-INH self-administration outside of a health care setting and has received lecture fees from Greer, Shire, and Baxalta. M. M. Frank has received consultancy fees from BioCryst Pharma. J. Edelman and D. Williams-Herman are employed by and have stock/stock options in CSL Behring. H. Feuersenger and M. Rojavin are employed by CSL Behring.
doi:10.1016/j.jaip.2016.04.018
pmid:27286778
fatcat:bd2cjaagybffpbydl74zs4dos4
