No serious or severe adverse events were considered by the investigator to be related to Cinryze, and no adverse events led to treatment discontinuation. There was no evidence of human immunodeficiency virus (HIV) or viral hepatitis transmission or development of clinically relevant anti-C1 INH antibodies in these studies.
Design of Sub-Group Analysis of Prospective DataForty-six children and adolescents ranging in age from two to 17 years received a total of 2,237 infusions of Cinryze across two randomized, placebo-controlled studies and their open-label extensions. One of the studies and its extension evaluated the use of Cinryze in acute HAE attacks, and the other evaluated its use in prophylaxis. In the pivotal acute-attack treatment trial, patients who presented to the study site within 4 hours after onset of a moderate or severe acute attack of the abdomen, face, or external genitalia were randomly assigned to intravenous infusions of placebo or Cinryze 1000 U. The pivotal prophylaxis trial consisted of 2 consecutive 12-week treatment periods during which patients received study medication for preventing HAE attacks. Patients were randomly assigned to receive intravenous infusions of 1000 U Cinryze or placebo every 3 to 4 days.
"Through an analysis of the largest compilation of pediatric patient data from prospective HAE clinical studies, we were able to determine that the safety and efficacy profile of Cinryze in this pediatric subpopulation were similar to those seen in the overall trial population," commented Dr. William Lumry, Medical Director, Asthma and Allergy Research Associates in Dallas, Tx. "Though no drugs are currently approved for children with HAE, taken together, these data offer a substantive body of evidence supporting the clinical utility of Cinryze in pediatric patients with hereditary angioedema."
About Cinryze ® (C1 esterase inhibitor [human]) Cinryze is a highly purified, pasteurized and nanofiltered plasma-derived C1 esterase inhibitor product. In the U.S., Cinryze is approved by the FDA for routine prophylaxis against angioedema attacks in adolescent and adult patients with HAE. In the E.U., the product is approved by the EMA for the treatment and pre-procedure prevention of angioedema attacks in adults and adolescents with hereditary angioedema (HAE), and routine prevention of angioedema attacks in adults and adolescents with severe and recurrent attacks of hereditary angioedema (HAE), who are intolerant to or insufficiently protected by oral prevention treatments or patients who are inadequately managed with repeated acute treatment. Cinryze is for intravenous use only.Severe hypersensitivity reactions to Cinryze may occur. Thrombotic events have occurred in patients receiving Cinryze, and in patients receiving off-label high dose C1 inhibitor therapy. Monitor patients with known risk factors for thrombotic events. With any blood or plasma derived product, there may be a risk of transmission of infectious agents, e.g. viruses and, theoretically, the CJD agent. The risk has been reduced by screening donors for prior exposure to certain virus infections and by manufacturing steps to reduce the risk of viral transmission including pasteurization and nanofiltration.
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