Study Shows Efficacy of C1-Esterase Inhibitor Concentrate in Treating Acute Attacks of Hereditary Angioedema in Children and Adolescents
Data presented at the 2011 ACAAI Annual Meeting reveal comparability of treatment outcomes for children, adolescents and adults
Boston, MA — 05 November 2011
New findings demonstrate that treatment with C1-esterase inhibitor (C1-INH) concentrate is effective in treating acute swelling attacks in children and adolescents with type I or type II hereditary angioedema (HAE), a rare and serious genetic disorder. Study results show that the outcomes of treatment with C1-INH during acute HAE attacks in children and adolescents are comparable to the outcomes observed in adults. Results of the analysis will be presented today at the 2011 American College of Allergy, Asthma & Immunology (ACAAI) Annual Meeting.
"Hereditary angioedema is a debilitating and potentially life-threatening disease that can be passed from parent to child and impact entire families," said Lynda Schneider, M.D., Associate Professor of Pediatrics at Children's Hospital Boston and lead investigator. "While the benefits of C1-esterase inhibitor have been clearly demonstrated in treating adults with acute attacks of HAE, limited research exists regarding the use of the therapy in pediatric patients. Our findings show that treatment with C1-INH concentrate is consistent across age groups, with the average time to onset of symptom relief and the average time for complete resolution of symptoms comparable in children, adolescents and adults."
HAE is a genetic disorder caused by a deficiency of C1-INH and is inherited in an autosomal dominant manner. Symptoms of HAE include episodes of edema or swelling in face, abdomen, larynx and extremities. Patients who have abdominal attacks of HAE can experience episodes of severe pain, diarrhea, nausea and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face can cause painful distortion and painful swelling. Diagnosis of HAE requires a blood test to confirm low or abnormal levels of C1-INH.
Study Design and Key Findings
In this retrospective, consecutive, cohort analysis of pediatric and adolescent HAE patients enrolled in the International Multicenter Prospective Angioedema C1-INH Trials (I.M.P.A.C.T.) 1 and 2, patients 6 years of age or older but younger than 18 years of age who had type I or II HAE were evaluated. Following an acute attack, patients received a single injection of 20 U/kg C1-INH concentrate and were assessed for up to 4 hours. Efficacy endpoints were the times from start of drug administration to onset of symptom relief and to complete resolution of all symptoms.
During the study period, seven patients each experienced one attack (I.M.P.A.C.T. 1) and nine patients experienced a total of 115 attacks (I.M.P.A.C.T. 2). In I.M.P.A.C.T.1, the median times to complete resolution of symptoms were 8.08 hours in pediatric patients and 4.92 hours for all patients receiving C1-INH concentrate. In I.M.P.A.C.T. 2, the median time to onset of symptom relief in the pediatric group was 0.49 hours, which was comparable to the median time for the overall population (0.46 hours). Median times to complete resolution of symptoms were also comparable.
For further information about HAE, please visit the website of the International Patient Organization for C1-Inhibitor Deficiencies,
www.HAEI.org and the disease information website
About CSL Behring
CSL Behring is a global leader in the plasma protein biotherapeutics industry. Passionate about improving the quality of patients' lives, CSL Behring manufactures and markets a range of safe and effective plasma-derived and recombinant products and related services. The company's therapies are used in the treatment of immune deficiency disorders, hereditary angioedema, haemophilia, von Willebrand disease, other bleeding disorders and inherited emphysema. Other products are used for the prevention of hemolytic diseases in the newborn, in cardiac surgery, organ transplantation and in the treatment of burns. The company also operates one of the world's largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited, a biopharmaceutical company with headquarters in Melbourne, Australia. For more information, visit
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