Marc A Riedl1, David S Hurewitz2, Robyn Levy3, Paula J Busse4, David Fitts5, Ira Kalfus6. 1. Department of Clinical Immunology and Allergy, David Geffen School of Medicine, University of California, Los Angeles, California. Electronic address: mriedl@mednet.ucla.edu. 2. Department of Medicine, University of Oklahoma Health Science Center-Tulsa, Allergy Clinic of Tulsa, Tulsa, Oklahoma. 3. Family Allergy and Asthma Center, Atlanta, Georgia. 4. Department of Medicine-Allergy & Immunology, The Mount Sinai Medical Center, New York, New York. 5. Biometrics, ViroPharma Incorporated, Exton, Pennsylvania. 6. M2G Consulting, New York, New York.
Abstract
BACKGROUND: Hereditary angioedema (HAE) is a rare disease caused by C1INH gene mutations, which leads to a deficiency or dysfunction of C1 inhibitor (C1 INH), resulting in recurrent episodes of severe and potentially life-threatening edema. OBJECTIVE: To evaluate the efficacy and safety of repeat use of nanofiltered C1 esterase inhibitor (human) (C1 INH-nf) for the short-term treatment of HAE attacks. METHODS: In this open-label study, patients received C1 INH-nf, 1,000 U intravenously, for the treatment of HAE attacks. Efficacy end points included the proportion of attacks with unequivocal relief of the defining symptom within 1 and 4 hours after receiving study drug and time to beginning of relief of the defining symptom. Safety was monitored through adverse event reporting, vital signs measurements, and laboratory testing. RESULTS: A total of 113 patients were enrolled in the study from September 21, 2006, through March 31, 2009, and received 885 doses of C1 INH-nf. A total of 609 HAE attacks were treated with C1 INH-nf, and the numbers of attacks achieving unequivocal relief of the defining symptom within 1 and 4 hours after the start of the first dose were 412 (68%) and 529 (87%), respectively. Of 101 patients treated for an attack during the study period, 80 achieved unequivocal relief of their first attack within 4 hours after study medication (response rate, 79%); median time to the beginning of unequivocal relief was 0.75 hour. C1 INH-nf was safe and well tolerated. CONCLUSIONS: This open-label study demonstrates the efficacy and safety of C1 INH-nf for short-term treatment of HAE attacks. TRIAL REGISTRATION: clinicaltrials.gov Identifier: NCT00438815.
BACKGROUND:Hereditary angioedema (HAE) is a rare disease caused by C1INH gene mutations, which leads to a deficiency or dysfunction of C1 inhibitor (C1 INH), resulting in recurrent episodes of severe and potentially life-threatening edema. OBJECTIVE: To evaluate the efficacy and safety of repeat use of nanofiltered C1 esterase inhibitor (human) (C1 INH-nf) for the short-term treatment of HAE attacks. METHODS: In this open-label study, patients received C1 INH-nf, 1,000 U intravenously, for the treatment of HAE attacks. Efficacy end points included the proportion of attacks with unequivocal relief of the defining symptom within 1 and 4 hours after receiving study drug and time to beginning of relief of the defining symptom. Safety was monitored through adverse event reporting, vital signs measurements, and laboratory testing. RESULTS: A total of 113 patients were enrolled in the study from September 21, 2006, through March 31, 2009, and received 885 doses of C1 INH-nf. A total of 609 HAE attacks were treated with C1 INH-nf, and the numbers of attacks achieving unequivocal relief of the defining symptom within 1 and 4 hours after the start of the first dose were 412 (68%) and 529 (87%), respectively. Of 101 patients treated for an attack during the study period, 80 achieved unequivocal relief of their first attack within 4 hours after study medication (response rate, 79%); median time to the beginning of unequivocal relief was 0.75 hour. C1 INH-nf was safe and well tolerated. CONCLUSIONS: This open-label study demonstrates the efficacy and safety of C1 INH-nf for short-term treatment of HAE attacks. TRIAL REGISTRATION: clinicaltrials.gov Identifier: NCT00438815.
Authors: H Farkas; I Martinez-Saguer; K Bork; T Bowen; T Craig; M Frank; A E Germenis; A S Grumach; A Luczay; L Varga; A Zanichelli Journal: Allergy Date: 2016-09-08 Impact factor: 13.146
Authors: Stephen Betschel; Jacquie Badiou; Karen Binkley; Rozita Borici-Mazi; Jacques Hébert; Amin Kanani; Paul Keith; Gina Lacuesta; Susan Waserman; Bill Yang; Emel Aygören-Pürsün; Jonathan Bernstein; Konrad Bork; Teresa Caballero; Marco Cicardi; Timothy Craig; Henriette Farkas; Anete Grumach; Connie Katelaris; Hilary Longhurst; Marc Riedl; Bruce Zuraw; Magdelena Berger; Jean-Nicolas Boursiquot; Henrik Boysen; Anthony Castaldo; Hugo Chapdelaine; Lori Connors; Lisa Fu; Dawn Goodyear; Alison Haynes; Palinder Kamra; Harold Kim; Kelly Lang-Robertson; Eric Leith; Christine McCusker; Bill Moote; Andrew O'Keefe; Ibraheem Othman; Man-Chiu Poon; Bruce Ritchie; Charles St-Pierre; Donald Stark; Ellie Tsai Journal: Allergy Asthma Clin Immunol Date: 2019-11-25 Impact factor: 3.406