Paula Busse1, Anette Bygum2, Jonathan Edelman3, William Lumry4, Thomas Machnig5, Inmaculada Martinez-Saguer6, Mikhail Rojavin7. 1. Mount Sinai Hospital, New York, NY. 2. HAE Centre Denmark, Department of Dermatology and Allergy Centre, Odense University Hospital, Odense, Denmark. 3. CSL Behring, King of Prussia, Pa. 4. AARA Research Center, Dallas, Tex. 5. CSL Behring GmbH, Marburg, Germany. 6. Hemophilia Center Rhine Main GmbH, Mörfelden-Walldorf, Germany. 7. CSL Behring, King of Prussia, Pa. Electronic address: mikhail.rojavin@cslbehring.com.
Abstract
BACKGROUND: The plasma-derived, pasteurized C1-inhibitor (C1-INH) concentrate, Berinert has a 4-decade history of use in hereditary angioedema (HAE), with a substantial literature base that demonstrates safety and efficacy. Thromboembolic events have rarely been reported with C1-INH products, typically with off-label use or at supratherapeutic doses. OBJECTIVES: Active surveillance of safety and clinical usage patterns of pasteurized C1-inhibitor concentrate and the more recent pasteurized, nanofiltered C1-INH, with a particular interest in thromboembolic events. METHODS: A registry was initiated in April 2010 at 27 US and 4 EU sites to obtain both prospective and retrospective safety and usage data on subjects who were administered C1-INH (Berinert). RESULTS: As of May 10, 2013, data were available for 135 subjects and 3196 infusions. By subject, 67.4% were using C1-INH as on-demand therapy and 23.0% as both on-demand therapy and prophylactic administration. Approximately half of the infusions (49.5%) were administered for prophylaxis and >90% were given by the patient or a caregiver in the home setting. A total of 299 adverse events were reported, for an overall rate of 0.09 events per infusion with only 6 considered related to C1-INH. Two thromboembolic events were reported, both in patients with prothrombotic risk factors. CONCLUSION: This large pool of real-world clinical usage data in HAE further supports the extensive safety profile of 2 Berinert formulations when used on demand and/or for prophylaxis in both home and health care settings. No evidence was found to suggest that Berinert is an independent, causative risk factor for thromboembolic events.
BACKGROUND: The plasma-derived, pasteurized C1-inhibitor (C1-INH) concentrate, Berinert has a 4-decade history of use in hereditary angioedema (HAE), with a substantial literature base that demonstrates safety and efficacy. Thromboembolic events have rarely been reported with C1-INH products, typically with off-label use or at supratherapeutic doses. OBJECTIVES: Active surveillance of safety and clinical usage patterns of pasteurized C1-inhibitor concentrate and the more recent pasteurized, nanofiltered C1-INH, with a particular interest in thromboembolic events. METHODS: A registry was initiated in April 2010 at 27 US and 4 EU sites to obtain both prospective and retrospective safety and usage data on subjects who were administered C1-INH (Berinert). RESULTS: As of May 10, 2013, data were available for 135 subjects and 3196 infusions. By subject, 67.4% were using C1-INH as on-demand therapy and 23.0% as both on-demand therapy and prophylactic administration. Approximately half of the infusions (49.5%) were administered for prophylaxis and >90% were given by the patient or a caregiver in the home setting. A total of 299 adverse events were reported, for an overall rate of 0.09 events per infusion with only 6 considered related to C1-INH. Two thromboembolic events were reported, both in patients with prothrombotic risk factors. CONCLUSION: This large pool of real-world clinical usage data in HAE further supports the extensive safety profile of 2 Berinert formulations when used on demand and/or for prophylaxis in both home and health care settings. No evidence was found to suggest that Berinert is an independent, causative risk factor for thromboembolic events.
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: Nicole Wentzel; Angelica Panieri; Maryam Ayazi; Sipho Duncan Ntshalintshali; Zahra Pourpak; Di Hawarden; Paul Potter; Michael E Levin; Mohammad Reza Fazlollahi; Jonathan Peter Journal: World Allergy Organ J Date: 2019-10-12 Impact factor: 4.084