title: Efficacy of Different Medical Therapies for the Treatment of Acute Laryngeal Attacks of Hereditary Angioedema due to C1-esterase Inhibitor Deficiency.
abstract: BACKGROUND
  Hereditary angioedema (HAE) is a rare disease characterized by C1-esterase inhibitor (C1-INH) deficiency, resulting in periodic attacks of acute edema, which can be life-threatening if they occur in the upper airway. No head-to-head comparisons of different treatment options for acute HAE attacks are available. Because immediate symptom relief is critical for potentially life-threatening laryngeal attacks, it is important to determine the treatment option that provides optimal treatment response.
  
  
  OBJECTIVE
  Review and compare data from clinical studies that evaluated the efficacy and safety of treatments for laryngeal HAE attacks.
  
  
  METHODS
  We conducted an indirect comparison of clinical outcomes from prospective studies for treatment of 881 acute laryngeal attacks with plasma-derived C1-INH concentrate (pdC1-INH) at fixed doses (500 or 1000 U) or a body weight-adjusted dose (20 U/kg), recombinant C1-INH concentrate at a fixed dose (2100 U), or a body weight-adjusted dose (50 U/kg), icatibant (30 mg), or ecallantide (30 mg). Comparisons included time to onset of symptom relief and need for re-dosing or emergency procedures.
  
  
  RESULTS
  The median time to onset of symptom relief ranged between 15 min and approximately 2 h, and was shortest with body weight-adjusted doses of pdC1-INH. The proportion of laryngeal attacks with re-dosing ranged between 0% and 72%. No re-dosing was needed after treatment with a single body weight-adjusted dose of pdC1-INH (48 attacks).
  
  
  CONCLUSIONS
  Available data suggest that among different HAE treatments, body weight-adjusted pdC1-INH (20 U/kg) provides the most reliable treatment response for treatment of laryngeal HAE attacks.
authors.affiliation: Department of Dermatology, University of Mainz, Mainz, Germany.
authors.forename: Konrad
authors.initials: K
authors.lastname: Bork
authors.affiliation: University of Cincinnati Medical Center and Bernstein Clinical Research Center, Cincinnati, Ohio.
authors.forename: Jonathan A
authors.initials: JA
authors.lastname: Bernstein
authors.affiliation: CSL Behring GmbH, Marburg, Germany.
authors.forename: Thomas
authors.initials: T
authors.lastname: Machnig
authors.affiliation: Penn State University College of Medicine, Hershey, Pennsylvania.
authors.forename: Timothy J
authors.initials: TJ
authors.lastname: Craig
mesh_terms: D054179:Angioedemas, Hereditary / Q000188:drug therapy* / Q000201:enzymology*; D050718:Complement C1 Inhibitor Protein / Q000627:therapeutic use*; D006801:Humans; D007818:Laryngeal Diseases / Q000188:drug therapy* / Q000201:enzymology*; D016896:Treatment Outcome
chemical_list: D050718:Complement C1 Inhibitor Protein
keywords: C1-INH; HAE; efficacy; laryngeal; re-dosing