Title |
Safety and Usage of C1-Inhibitor in Hereditary Angioedema: Berinert Registry Data
|
---|---|
Published in |
The Journal of Allergy and Clinical Immunology: In Practice, June 2016
|
DOI | 10.1016/j.jaip.2016.04.018 |
Pubmed ID | |
Authors |
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, Berinert Registry investigators, Jacob Offenberger, Robyn Levy, David Hurewitz, H. Henry Li, Ralph Shapiro, Jonathan Bernstein, Timothy Craig, Aaron Davis, Jeffrey 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 |
Abstract |
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. The objective of this study was to describe safety and usage patterns of pnfC1-INH. 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. 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. 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. |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
Unknown | 61 | 100% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Bachelor | 9 | 15% |
Researcher | 8 | 13% |
Professor | 5 | 8% |
Student > Master | 4 | 7% |
Other | 4 | 7% |
Other | 14 | 23% |
Unknown | 17 | 28% |
Readers by discipline | Count | As % |
---|---|---|
Medicine and Dentistry | 27 | 44% |
Nursing and Health Professions | 4 | 7% |
Immunology and Microbiology | 3 | 5% |
Pharmacology, Toxicology and Pharmaceutical Science | 2 | 3% |
Computer Science | 1 | 2% |
Other | 5 | 8% |
Unknown | 19 | 31% |