PMID- 27286778 OWN - NLM STAT- MEDLINE DCOM- 20171030 LR - 20220330 IS - 2213-2201 (Electronic) VI - 4 IP - 5 DP - 2016 Sep-Oct TI - Safety and Usage of C1-Inhibitor in Hereditary Angioedema: Berinert Registry Data. PG - 963-71 LID - S2213-2198(16)30128-3 [pii] LID - 10.1016/j.jaip.2016.04.018 [doi] AB - 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. CI - Copyright (c) 2016. Published by Elsevier Inc. FAU - Riedl, Marc A AU - Riedl MA AD - Department of Rheumatology, Allergy & Immunology, University of California, San Diego, La Jolla, Calif. FAU - Bygum, Anette AU - Bygum A AD - HAE Center Denmark, Department of Dermatology and Allergy Centre, Odense University Hospital, Odense C, Denmark. FAU - Lumry, William AU - Lumry W AD - AARA Research Center, Dallas, Tex. FAU - Magerl, Markus AU - Magerl M AD - Department of Dermatology and Allergy, Charite - Universitatsmedizin Berlin, Berlin, Germany. FAU - Bernstein, Jonathan A AU - Bernstein JA AD - Department of Internal Medicine/Allergy Section Cincinnati, University of Cincinnati College of Medicine, Cincinnati, Ohio. FAU - Busse, Paula AU - Busse P AD - Division of Clinical Immunology, Mount Sinai, New York, NY. FAU - Craig, Timothy AU - Craig T AD - Department of Medicine and Pediatrics, Penn State University, Hershey Medical Center, Hershey, Pa. FAU - Frank, Michael M AU - Frank MM AD - Department of Pediatrics, Duke University Medical Center, Durham, NC. FAU - Edelman, Jonathan AU - Edelman J AD - Clinical Sciences, CSL Behring, King of Prussia, Pa. FAU - Williams-Herman, Debora AU - Williams-Herman D AD - Clinical Development, CSL Behring, King of Prussia, Pa. FAU - Feuersenger, Henrike AU - Feuersenger H AD - Clinical Sciences, CSL Behring, Marburg, Germany. FAU - Rojavin, Mikhail AU - Rojavin M AD - Clinical Development, CSL Behring, King of Prussia, Pa. Electronic address: mikhail.rojavin@cslbehring.com. CN - Berinert Registry investigators LA - eng PT - Journal Article PT - Multicenter Study PT - Observational Study PT - Research Support, Non-U.S. Gov't DEP - 20160608 PL - United States TA - J Allergy Clin Immunol Pract JT - The journal of allergy and clinical immunology. In practice JID - 101597220 RN - 0 (Complement C1 Inhibitor Protein) SB - IM MH - Adolescent MH - Adult MH - Aged MH - Aged, 80 and over MH - Angioedemas, Hereditary/*drug therapy/prevention & control MH - Child MH - Child, Preschool MH - Complement C1 Inhibitor Protein/adverse effects/*therapeutic use MH - Europe MH - Female MH - Humans MH - Male MH - Middle Aged MH - Registries MH - United States MH - Young Adult OTO - NOTNLM OT - Berinert OT - Dosing OT - On demand OT - Patient registry OT - Plasma-derived C1-INH OT - Prophylaxis OT - Real-world OT - Safety OT - Self-administration OT - Thromboembolic event FIR - Offenberger, Jacob IR - Offenberger J FIR - Levy, Robyn IR - Levy R FIR - Hurewitz, David IR - Hurewitz D FIR - Li, H Henry IR - Li HH FIR - Shapiro, Ralph IR - Shapiro R FIR - Bernstein, Jonathan IR - Bernstein J FIR - Craig, Timothy IR - Craig T FIR - Davis, Aaron IR - Davis A FIR - Rosch, Jeffrey IR - Rosch J FIR - Fox, James IR - Fox J FIR - Janss, Gerti IR - Janss G FIR - Baker, James IR - Baker J FIR - Packer, Flint IR - Packer F FIR - Vegh, Art IR - Vegh A FIR - Frank, Michael IR - Frank M FIR - Sher, Ellen IR - Sher E FIR - Busse, Paula IR - Busse P FIR - Wedner, James H IR - Wedner JH FIR - Riedl, Marc IR - Riedl M FIR - Lumry, William IR - Lumry W FIR - Amrol, David IR - Amrol D FIR - Gower, Richard IR - Gower R FIR - Silber, Glenn IR - Silber G FIR - Portnoy, Jay IR - Portnoy J FIR - Paris, Kenneth IR - Paris K FIR - Darter, Amy IR - Darter A FIR - Mumneh, Nayla IR - Mumneh N FIR - Petrov, Andrej IR - Petrov A FIR - Schneider, Lynda IR - Schneider L FIR - Martinez-Saguer, Inmaculada IR - Martinez-Saguer I FIR - Staubach-Renz, Petra IR - Staubach-Renz P FIR - Maurer, Marcus IR - Maurer M FIR - Bas, Murat IR - Bas M FIR - Aygoren-Pursun, Emel IR - Aygoren-Pursun E FIR - Bygum, Anette IR - Bygum A FIR - Wuillemin, Walter IR - Wuillemin W EDAT- 2016/06/12 06:00 MHDA- 2017/10/31 06:00 CRDT- 2016/06/12 06:00 PHST- 2016/01/27 00:00 [received] PHST- 2016/04/20 00:00 [revised] PHST- 2016/04/26 00:00 [accepted] PHST- 2016/06/12 06:00 [entrez] PHST- 2016/06/12 06:00 [pubmed] PHST- 2017/10/31 06:00 [medline] AID - S2213-2198(16)30128-3 [pii] AID - 10.1016/j.jaip.2016.04.018 [doi] PST - ppublish SO - J Allergy Clin Immunol Pract. 2016 Sep-Oct;4(5):963-71. doi: 10.1016/j.jaip.2016.04.018. Epub 2016 Jun 8.