title: Nanofiltered C1 esterase inhibitor (human) for the treatment of acute attacks of hereditary angioedema: an open-label trial.
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.
  
  
  BACKGROUND
  clinicaltrials.gov Identifier: NCT00438815.
authors.affiliation: Department of Clinical Immunology and Allergy, David Geffen School of Medicine, University of California, Los Angeles, California. Electronic address: mriedl@mednet.ucla.edu.
authors.forename: Marc A
authors.initials: MA
authors.lastname: Riedl
authors.affiliation: Department of Medicine, University of Oklahoma Health Science Center-Tulsa, Allergy Clinic of Tulsa, Tulsa, Oklahoma.
authors.forename: David S
authors.initials: DS
authors.lastname: Hurewitz
authors.affiliation: Family Allergy and Asthma Center, Atlanta, Georgia.
authors.forename: Robyn
authors.initials: R
authors.lastname: Levy
authors.affiliation: Department of Medicine-Allergy & Immunology, The Mount Sinai Medical Center, New York, New York.
authors.forename: Paula J
authors.initials: PJ
authors.lastname: Busse
authors.affiliation: Biometrics, ViroPharma Incorporated, Exton, Pennsylvania.
authors.forename: David
authors.initials: D
authors.lastname: Fitts
authors.affiliation: M2G Consulting, New York, New York.
authors.forename: Ira
authors.initials: I
authors.lastname: Kalfus
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D054179:Angioedemas, Hereditary / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D050718:Complement C1 Inhibitor Protein / Q000008:administration & dosage / Q000009:adverse effects* / Q000627:therapeutic use*; D051056:Complement Inactivating Agents / Q000008:administration & dosage / Q000009:adverse effects* / Q000627:therapeutic use*; D005260:Female; D005374:Filtration / Q000379:methods; D006801:Humans; D008297:Male; D008875:Middle Aged; D013997:Time Factors; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D050718:Complement C1 Inhibitor Protein; D051056:Complement Inactivating Agents