Safety and Usage of C1-Inhibitor in Hereditary Angioedema

Berinert Registry Data

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, Jacob Offenberger, Robyn Levy, David Hurewitz, H. Henry Li, Ralph Shapiro, Jonathan Bernstein, Timothy Craig, Aaron Davis & 28 others 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 Michael 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

Research output: Contribution to journalArticle

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.

Original languageEnglish (US)
Pages (from-to)963-971
Number of pages9
JournalJournal of Allergy and Clinical Immunology: In Practice
Volume4
Issue number5
DOIs
StatePublished - Sep 1 2016

Fingerprint

Hereditary Angioedemas
Registries
Safety
Delivery of Health Care
Self Administration
Guidelines
Therapeutics
Infection
Population

Keywords

  • Berinert
  • Dosing
  • On demand
  • Patient registry
  • Plasma-derived C1-INH
  • Prophylaxis
  • Real-world
  • Safety
  • Self-administration
  • Thromboembolic event

ASJC Scopus subject areas

  • Immunology and Allergy

Cite this

Riedl, M. A., Bygum, A., Lumry, W., Magerl, M., Bernstein, J. A., Busse, P., ... Wuillemin, W. (2016). Safety and Usage of C1-Inhibitor in Hereditary Angioedema: Berinert Registry Data. Journal of Allergy and Clinical Immunology: In Practice, 4(5), 963-971. https://doi.org/10.1016/j.jaip.2016.04.018

Safety and Usage of C1-Inhibitor in Hereditary Angioedema : Berinert Registry Data. / Riedl, Marc A.; Bygum, Anette; Lumry, William; Magerl, Markus; Bernstein, Jonathan A.; Busse, Paula; Craig, Timothy; Frank, Michael M.; Edelman, Jonathan; Williams-Herman, Debora; Feuersenger, Henrike; Rojavin, Mikhail; Offenberger, Jacob; Levy, Robyn; Hurewitz, David; Li, H. Henry; Shapiro, Ralph; Bernstein, Jonathan; Craig, Timothy; Davis, Aaron; Rosch, Jeffrey; Fox, James; Janss, Gerti; Baker, James; Packer, Flint; Vegh, Art; Frank, Michael; Sher, Ellen; Busse, Paula; Wedner, James H.; Riedl, Marc; Lumry, William; Amrol, David; Gower, Richard; Silber, Glenn Michael; Portnoy, Jay; Paris, Kenneth; Darter, Amy; Mumneh, Nayla; Petrov, Andrej; Schneider, Lynda; Martinez-Saguer, Inmaculada; Staubach-Renz, Petra; Maurer, Marcus; Bas, Murat; Aygören-Pürsün, Emel; Bygum, Anette; Wuillemin, Walter.

In: Journal of Allergy and Clinical Immunology: In Practice, Vol. 4, No. 5, 01.09.2016, p. 963-971.

Research output: Contribution to journalArticle

Riedl, MA, Bygum, A, Lumry, W, Magerl, M, Bernstein, JA, Busse, P, Craig, T, Frank, MM, Edelman, J, Williams-Herman, D, Feuersenger, H, Rojavin, M, Offenberger, J, Levy, R, Hurewitz, D, Li, HH, Shapiro, R, Bernstein, J, Craig, T, Davis, A, Rosch, J, Fox, J, Janss, G, Baker, J, Packer, F, Vegh, A, Frank, M, Sher, E, Busse, P, Wedner, JH, Riedl, M, Lumry, W, Amrol, D, Gower, R, Silber, GM, Portnoy, J, Paris, K, Darter, A, Mumneh, N, Petrov, A, Schneider, L, Martinez-Saguer, I, Staubach-Renz, P, Maurer, M, Bas, M, Aygören-Pürsün, E, Bygum, A & Wuillemin, W 2016, 'Safety and Usage of C1-Inhibitor in Hereditary Angioedema: Berinert Registry Data', Journal of Allergy and Clinical Immunology: In Practice, vol. 4, no. 5, pp. 963-971. https://doi.org/10.1016/j.jaip.2016.04.018
Riedl, Marc A. ; Bygum, Anette ; Lumry, William ; Magerl, Markus ; Bernstein, Jonathan A. ; Busse, Paula ; Craig, Timothy ; Frank, Michael M. ; Edelman, Jonathan ; Williams-Herman, Debora ; Feuersenger, Henrike ; Rojavin, Mikhail ; Offenberger, Jacob ; Levy, Robyn ; Hurewitz, David ; Li, H. Henry ; Shapiro, Ralph ; Bernstein, Jonathan ; Craig, Timothy ; Davis, Aaron ; Rosch, Jeffrey ; Fox, James ; Janss, Gerti ; Baker, James ; Packer, Flint ; Vegh, Art ; Frank, Michael ; Sher, Ellen ; Busse, Paula ; Wedner, James H. ; Riedl, Marc ; Lumry, William ; Amrol, David ; Gower, Richard ; Silber, Glenn Michael ; Portnoy, Jay ; Paris, Kenneth ; Darter, Amy ; Mumneh, Nayla ; Petrov, Andrej ; Schneider, Lynda ; Martinez-Saguer, Inmaculada ; Staubach-Renz, Petra ; Maurer, Marcus ; Bas, Murat ; Aygören-Pürsün, Emel ; Bygum, Anette ; Wuillemin, Walter. / Safety and Usage of C1-Inhibitor in Hereditary Angioedema : Berinert Registry Data. In: Journal of Allergy and Clinical Immunology: In Practice. 2016 ; Vol. 4, No. 5. pp. 963-971.
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TY - JOUR

T1 - Safety and Usage of C1-Inhibitor in Hereditary Angioedema

T2 - Berinert Registry Data

AU - Riedl, Marc A.

AU - Bygum, Anette

AU - Lumry, William

AU - Magerl, Markus

AU - Bernstein, Jonathan A.

AU - Busse, Paula

AU - Craig, Timothy

AU - Frank, Michael M.

AU - Edelman, Jonathan

AU - Williams-Herman, Debora

AU - Feuersenger, Henrike

AU - Rojavin, Mikhail

AU - Offenberger, Jacob

AU - Levy, Robyn

AU - Hurewitz, David

AU - Li, H. Henry

AU - Shapiro, Ralph

AU - Bernstein, Jonathan

AU - Craig, Timothy

AU - Davis, Aaron

AU - Rosch, Jeffrey

AU - Fox, James

AU - Janss, Gerti

AU - Baker, James

AU - Packer, Flint

AU - Vegh, Art

AU - Frank, Michael

AU - Sher, Ellen

AU - Busse, Paula

AU - Wedner, James H.

AU - Riedl, Marc

AU - Lumry, William

AU - Amrol, David

AU - Gower, Richard

AU - Silber, Glenn Michael

AU - Portnoy, Jay

AU - Paris, Kenneth

AU - Darter, Amy

AU - Mumneh, Nayla

AU - Petrov, Andrej

AU - Schneider, Lynda

AU - Martinez-Saguer, Inmaculada

AU - Staubach-Renz, Petra

AU - Maurer, Marcus

AU - Bas, Murat

AU - Aygören-Pürsün, Emel

AU - Bygum, Anette

AU - Wuillemin, Walter

PY - 2016/9/1

Y1 - 2016/9/1

N2 - 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.

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.

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KW - Dosing

KW - On demand

KW - Patient registry

KW - Plasma-derived C1-INH

KW - Prophylaxis

KW - Real-world

KW - Safety

KW - Self-administration

KW - Thromboembolic event

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