HAE treatment perspectives

PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting study, entitled ”A phase I study of recombinant human C1 inhibitor in asymptomatic patients with hereditary angioedema“ (J Allergy Clin Immunol. 2005 Oct;116(4):876-83. Epub 2005 Aug 8). Authors are van Doorn MB, Burggraaf J, van Dam T, et al, from the Centre for Human Drug Research, Leiden. Hereditary angioedema (HAE) is a congenital disorder, affecting submucosal tissue, subcutaneous tissue, or both caused by a deficiency of the plasma protein C1 inhibitor (C1 esterase inhibitor [C1INH”>). OBJECTIVE: to evaluate the effects of recombinant human C1INH (rhC1INH) in 12 asymptomatic patients with HAE. METHODS: rhC1INH was intravenously administered. RESULTS: rhC1INH appeared safe and was well tolerated. The course of functional C1INH in plasma showed a full initial recovery and a dose-dependent clearance of rhC1INH. rhC1INH displayed dose-dependent biologic activity by increasing the C4 level, and decreasing levels of cleaved C4. CONCLUSION: The observed safety profile and biologic activity of rhC1INH warrants further clinical studies to assess its efficacy in treating HAE attacks. To access the full abstract of the text study, click here.