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Hereditary Angioedema Patients Report: C1-Esterase Inhibitor Concentrate Effective in Treating Acute Attacks, Helps Them Avoid Emergency Care
Date:11/7/2009

ement 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 attack
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SOURCE CSL Behring
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1. CSL Behring Reaches Primary Endpoint in Clinical Study of C1-INH for Treatment of Hereditary Angioedema
2. Arriva Pharmaceuticals Moves Forward to Phase 2b Development of Lead Inhaled Protein rAAT Replacement Therapy for Hereditary Emphysema
3. CSL Behring Submits BLA Requesting Approval of C1-Esterase Inhibitor for the Treatment of Hereditary Angioedema
4. ViroPharma Announces Presentation of Cinryze(TM) Data in Acute Treatment of Hereditary Angioedema
5. FDA Grants Priority Review of a Supplemental Biologics License Application for Cinryze(TM) C1 Inhibitor (Human) as Treatment for Acute Attacks of Hereditary Angioedema (HAE)
6. Changing Paradigms in Hereditary Angioedema: A Focus on Prompt Diagnosis and New Therapies
7. CSL Behring Announces FDA Approval of Berinert(R), First and Only Therapy Approved for the Treatment of Acute Abdominal and Facial Attacks of Hereditary Angioedema in U.S.
8. Prodromal Symptoms Warn of Hereditary Angioedema Attacks, Give Patients Time to Start Therapy and Decrease Morbidity
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