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, Jacob Offenberger, Robyn Levy, David Hurewitz, H. Henry Li, Ralph Shapiro, Jonathan Bernstein, Timothy Craig, Aaron DavisJeffrey Rosch, James Fox, Gerti Janss, James Baker, Flint Packer, Art Vegh, Michael Frank, Ellen Sher, Paula Busse, James H. Wedner, Marc Riedl, William Lumry, David Amrol, Richard Gower, Glenn Silber, Jay Portnoy, Kenneth Paris, Amy Darter, Nayla Mumneh, Andrej Petrov, Lynda Schneider, Inmaculada Martinez-Saguer, Petra Staubach-Renz, Marcus Maurer, Murat Bas, Emel Aygören-Pürsün, Anette Bygum, Walter Wuillemin

Research output: Contribution to journalArticlepeer-review

29 Scopus citations

Abstract

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 consistent with current HAE guidelines. These real-world data revealed pnfC1-INH usage for a variety of reasons in patients with HAE and showed a high level of safety regardless of administration setting or reason for use.

Original languageEnglish (US)
Pages (from-to)963-971
Number of pages9
JournalJournal of Allergy and Clinical Immunology: In Practice
Volume4
Issue number5
DOIs
StatePublished - Sep 1 2016

All Science Journal Classification (ASJC) codes

  • Immunology and Allergy

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