Marc A Riedl1, Anette Bygum2, William Lumry3, Markus Magerl4, Jonathan A Bernstein5, Paula Busse6, Timothy Craig7, Michael M Frank8, Jonathan Edelman9, Debora Williams-Herman10, Henrike Feuersenger11, Mikhail Rojavin12. 1. Department of Rheumatology, Allergy & Immunology, University of California, San Diego, La Jolla, Calif. 2. HAE Center Denmark, Department of Dermatology and Allergy Centre, Odense University Hospital, Odense C, Denmark. 3. AARA Research Center, Dallas, Tex. 4. Department of Dermatology and Allergy, Charité - Universitätsmedizin Berlin, Berlin, Germany. 5. Department of Internal Medicine/Allergy Section Cincinnati, University of Cincinnati College of Medicine, Cincinnati, Ohio. 6. Division of Clinical Immunology, Mount Sinai, New York, NY. 7. Department of Medicine and Pediatrics, Penn State University, Hershey Medical Center, Hershey, Pa. 8. Department of Pediatrics, Duke University Medical Center, Durham, NC. 9. Clinical Sciences, CSL Behring, King of Prussia, Pa. 10. Clinical Development, CSL Behring, King of Prussia, Pa. 11. Clinical Sciences, CSL Behring, Marburg, Germany. 12. Clinical Development, CSL Behring, King of Prussia, Pa. Electronic address: mikhail.rojavin@cslbehring.com.
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.
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.
Authors: Marcus Maurer; Markus Magerl; Stephen Betschel; Werner Aberer; Ignacio J Ansotegui; Emel Aygören-Pürsün; Aleena Banerji; Noémi-Anna Bara; Isabelle Boccon-Gibod; Konrad Bork; Laurence Bouillet; Henrik Balle Boysen; Nicholas Brodszki; Paula J Busse; Anette Bygum; Teresa Caballero; Mauro Cancian; Anthony J Castaldo; Danny M Cohn; Dorottya Csuka; Henriette Farkas; Mark Gompels; Richard Gower; Anete S Grumach; Guillermo Guidos-Fogelbach; Michihiro Hide; Hye-Ryun Kang; Allen P Kaplan; Constance H Katelaris; Sorena Kiani-Alikhan; Wei-Te Lei; Richard F Lockey; Hilary Longhurst; William Lumry; Andrew MacGinnitie; Alejandro Malbran; Inmaculada Martinez Saguer; Juan José Matta Campos; Alexander Nast; Dinh Nguyen; Sandra A Nieto-Martinez; Ruby Pawankar; Jonathan Peter; Grzegorz Porebski; Nieves Prior; Avner Reshef; Marc Riedl; Bruce Ritchie; Farrukh Rafique Sheikh; William B Smith; Peter J Spaeth; Marcin Stobiecki; Elias Toubi; Lilian Agnes Varga; Karsten Weller; Andrea Zanichelli; Yuxiang Zhi; Bruce Zuraw; Timothy Craig Journal: World Allergy Organ J Date: 2022-04-07 Impact factor: 5.516
Authors: Timothy Craig; Ralph Shapiro; Arthur Vegh; James W Baker; Jonathan A Bernstein; Paula Busse; Markus Magerl; Inmaculada Martinez-Saguer; Marc A Riedl; William Lumry; Debora Williams-Herman; Jonathan Edelman; Henrike Feuersenger; Thomas Machnig; Mikhail Rojavin Journal: Allergy Rhinol (Providence) Date: 2017-03-01
Authors: Anna Valerieva; Maria T Staevska; Vesna Grivcheva-Panovska; Milos Jesenak; Kinga Viktória Kőhalmi; Katarina Hrubiskova; Andrea Zanichelli; Luca Bellizzi; Anurag Relan; Roman Hakl; Henriette Farkas Journal: World Allergy Organ J Date: 2021-04-22 Impact factor: 4.084