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Study Shows Efficacy of C1-Esterase Inhibitor Concentrate in Treating Acute Attacks of Hereditary Angioedema in Children and Adolescents
Date:11/5/2011

BOSTON, Nov. 5, 2011 /PRNewswire/ -- 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 the 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
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SOURCE CSL Behring
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