Recombinant human C1 esterase inhibitor in the management of hereditary angioedema.

摘要: Hereditary angioedema (HAE), a rare autosomal dominant genetic disorder, is caused by deficiency in functional C1 esterase inhibitor (C1-INH). This potentially life-threatening condition manifests as recurrent attacks of subcutaneous and submucosal swelling the skin, gastrointestinal tract larynx. The management HAE includes treatment acute episodes, short-term prophylaxis preparation for exposure to known triggers long-term decrease incidence severity attacks. Four products are approved USA HAE, including one human plasma-derived C1-INH therapy, recombinant product (rhC1-INH), plasma kallikrein bradykinin B2 receptor antagonist. In addition, therapy danazol rhC1-INH, extracted from milk transgenic rabbits, glycoprotein 478 amino acids with an identical acid sequence endogenous protein. Population pharmacokinetic analysis rhC1-INH supports intravenous dosing strategy 50 U/kg (maximum 4200 U). safety efficacy patients were demonstrated three randomized, double-blind, placebo-controlled studies two open-label extension studies. pilot study, weekly administration 8 weeks reduced was well tolerated. Administration has not been associated development anti-drug antibodies or anti-host-related impurities.