Prospective study of rapid relief provided by C1 esterase inhibitor in emergency treatment of acute laryngeal attacks in hereditary angioedema.
BACKGROUND
Hereditary angioedema (HAE) is a rare disorder characterized by C1 esterase inhibitor (C1-INH) deficiency, resulting in periodic attacks of acute edema that can be life-threatening if they occur in the laryngeal region. We assessed the efficacy of C1-INH concentrate in the emergency treatment of rarely occurring acute laryngeal HAE attacks in a prospective, open-label clinical study.


METHODS
Acute laryngeal attacks were each treated with C1-INH concentrate (Berinert) at a single dose of 20 U/kg body weight. Efficacy endpoints included time to onset of symptom relief and time to complete resolution of all symptoms, each based on the patient's assessment.


RESULTS
All 39 laryngeal attacks in 16 patients were treated successfully. The median time to onset of symptom relief was 15 min. The median time to complete resolution of all symptoms was 8.25 h. No treatment-related serious adverse events occurred, and the treatment was well tolerated. The administration of C1-INH concentrate was not associated with any viral infections.


CONCLUSIONS
C1-INH concentrate is an effective and safe emergency treatment for providing reliable and rapid relief from the potentially life-threatening symptoms of laryngeal HAE attacks.
Craig
Timothy J
TJ
Asthma and Respiratory Research, Penn State University, 500 University Drive, Hershey, PA 17033, USA. tcraig@hmc.psu.edu
Wasserman
Richard L
RL
Levy
Robyn J
RJ
Bewtra
Againdra K
AK
Schneider
Lynda
L
Packer
Flint
F
Yang
William H
WH
Keinecke
Heinz-Otto
HO
Kiessling
Peter C
PC
D000293:Adolescent; D000328:Adult; D054179:Angioedemas, Hereditary / Q000188:drug therapy* / Q000235:genetics / Q000276:immunology / Q000503:physiopathology; D050718:Complement C1 Inhibitor Protein / Q000008:administration & dosage* / Q000009:adverse effects / Q000235:genetics; D018572:Disease-Free Survival; D004487:Edema; D004632:Emergency Medical Services; D005240:Feasibility Studies; D005260:Female; D006801:Humans; D007818:Laryngeal Diseases / Q000188:drug therapy* / Q000235:genetics / Q000276:immunology / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D011795:Surveys and Questionnaires
D050718:Complement C1 Inhibitor Protein