Safety and Usage of C1-Inhibitor in Hereditary Angioedema: Berinert Registry Data.
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.
Department of Rheumatology, Allergy & Immunology, University of California, San Diego, La Jolla, Calif.
Marc A
MA
Riedl
HAE Center Denmark, Department of Dermatology and Allergy Centre, Odense University Hospital, Odense C, Denmark.
Anette
A
Bygum
AARA Research Center, Dallas, Tex.
William
W
Lumry
Department of Dermatology and Allergy, Charité - Universitätsmedizin Berlin, Berlin, Germany.
Markus
M
Magerl
Department of Internal Medicine/Allergy Section Cincinnati, University of Cincinnati College of Medicine, Cincinnati, Ohio.
Jonathan A
JA
Bernstein
Division of Clinical Immunology, Mount Sinai, New York, NY.
Paula
P
Busse
Department of Medicine and Pediatrics, Penn State University, Hershey Medical Center, Hershey, Pa.
Timothy
T
Craig
Department of Pediatrics, Duke University Medical Center, Durham, NC.
Michael M
MM
Frank
Clinical Sciences, CSL Behring, King of Prussia, Pa.
Jonathan
J
Edelman
Clinical Development, CSL Behring, King of Prussia, Pa.
Debora
D
Williams-Herman
Clinical Sciences, CSL Behring, Marburg, Germany.
Henrike
H
Feuersenger
Clinical Development, CSL Behring, King of Prussia, Pa. Electronic address: mikhail.rojavin@cslbehring.com.
Mikhail
M
Rojavin
D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D054179:Angioedemas, Hereditary / Q000188:drug therapy* / Q000517:prevention & control; D002648:Child; D002675:Child, Preschool; D050718:Complement C1 Inhibitor Protein / Q000009:adverse effects / Q000627:therapeutic use*; D005060:Europe; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D012042:Registries; D014481:United States; D055815:Young Adult
D050718:Complement C1 Inhibitor Protein
Berinert; Dosing; On demand; Patient registry; Plasma-derived C1-INH; Prophylaxis; Real-world; Safety; Self-administration; Thromboembolic event