MIAMI BEACH, Fla., Nov. 7 /PRNewswire/ -- Patients reported C1-esterase inhibitor (C1-INH) concentrate is an effective therapy in treating acute swelling attacks at any body location for HAE, a rare and serious genetic disorder, according to data presented today at the 2009 American College of Allergy, Asthma & Immunology (ACAAI) Annual Meeting. The results come from a self-reported survey of patients conducted in parallel with the ongoing, prospective, open label International Multi-center Prospective Angioedema C1-Inhibitor Trial (I.M.P.A.C.T. 2) for C1-INH concentrate. Of the patients surveyed, 98.3 percent reported satisfaction with the treatment, with 37 percent responding that without C1-INH they would have sought emergency treatment and 43 percent would have deferred any medical therapy.
Twenty-eight patients participated in the self-reported survey with responses collected via home computer or telephone voice response. Before the study, these patients reported an annual average of three emergency room visits and four hospital days due to acute attacks. Over 29 months, participants reported 384 attacks (261 abdominal, 71 extremities, eight facial, six genitourinary, 17 laryngeal, and 21 other) with a median of three per patient. C1-INH was used to treat approximately 71 percent of reported attacks. From the start of the attack to C1-INH dosing, the mean time was 12.1 hours (ranging from 0.0 to 189.8 hours) and the mean time from start of treatment to onset of symptom relief was 1.07 hours (ranging from 0.0 to 25.3 hours). The mean time to complete resolution was 18.4 hours (ranging from 1.22 to 471.7 hours).
"Recognizing an acute attack in its early stages is vital in successfully managing the disease," said Robyn J. Levy, M.D., of the Family Allergy & Asthma Center in Atlanta, GA, and the lead investigator of the study. "This survey shows that patients found C1-INH replacement therapy to be highly effective for the treatment of acute HAE attacks and without it some patients may have sought emergency treatment or deferred treatment altogether."
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 and the abdomen. 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. There are estimates of 6,000 to 10,000 or more people with HAE in the U.S.
As part of the same survey, patients could also complete a health-related quality of life (HRQoL) section to compare their opinions about HAE treatment before and after C1-INH. The SF-12 generic measure of health status was supplemented with questions about the effects of C1-INH. Eighteen patients provided HRQoL feedback on 55 attacks with more than 57 percent of respondents reporting very good or excellent general health and 98.1 percent indicating few or no physical health-related limitations on activities. Three-quarters reported no or little pain interference with work and life. About two-thirds reported a positive change in outlook and "much better" feeling of security about treating future attacks.
About I.M.P.A.C.T. 2
Findings of I.M.P.A.C.T. 2 were based on treatment with 20 U/kg bodyweight of C1-INH in 640 episodes of HAE attacks at any body location in 57 patients. The main study end-points were: time to onset of symptom relief; complete resolution of all symptoms; and safety.
The median times to complete resolution of all symptoms were reported as early as 8 hours for laryngeal attacks, 11 hours for abdominal, 24 hours for facial and 25 hours for peripheral attacks. No drug-related serious adverse events have been reported to date, nor were any rebound effects observed following C1-INH administration.
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, hemophilia, 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 www.cslbehring.com.
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