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Recombinant human C1 esterase inhibitor in the management of hereditary angioedema.
Clin Drug Investig 2015; 35(7):407-17CD

Abstract

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

Authors+Show Affiliations

Division of Rheumatology, Allergy, and Immunology, University of California, San Diego, 8899 University Center Lane, Ste 230, San Diego, CA, 92122, USA, mriedl@ucsd.edu.

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

26091744

Citation

Riedl, Marc. "Recombinant Human C1 Esterase Inhibitor in the Management of Hereditary Angioedema." Clinical Drug Investigation, vol. 35, no. 7, 2015, pp. 407-17.
Riedl M. Recombinant human C1 esterase inhibitor in the management of hereditary angioedema. Clin Drug Investig. 2015;35(7):407-17.
Riedl, M. (2015). Recombinant human C1 esterase inhibitor in the management of hereditary angioedema. Clinical Drug Investigation, 35(7), pp. 407-17. doi:10.1007/s40261-015-0300-z.
Riedl M. Recombinant Human C1 Esterase Inhibitor in the Management of Hereditary Angioedema. Clin Drug Investig. 2015;35(7):407-17. PubMed PMID: 26091744.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Recombinant human C1 esterase inhibitor in the management of hereditary angioedema. A1 - Riedl,Marc, PY - 2015/6/21/entrez PY - 2015/6/21/pubmed PY - 2016/8/23/medline SP - 407 EP - 17 JF - Clinical drug investigation JO - Clin Drug Investig VL - 35 IS - 7 N2 - Hereditary angioedema (HAE), a rare autosomal dominant genetic disorder, is caused by a deficiency in functional C1 esterase inhibitor (C1-INH). This potentially life-threatening condition manifests as recurrent attacks of subcutaneous and submucosal swelling of the skin, gastrointestinal tract and larynx. The management of HAE includes treatment of acute episodes, short-term prophylaxis in preparation for exposure to known triggers and long-term prophylaxis to decrease the incidence and severity of HAE attacks. Four products are approved in the USA for the treatment of acute attacks of HAE, including one human plasma-derived C1-INH therapy, a recombinant human C1-INH product (rhC1-INH), a plasma kallikrein inhibitor and a bradykinin B2 receptor antagonist. In addition, one human plasma-derived C1-INH therapy and danazol are approved for prophylaxis of HAE attacks. rhC1-INH, extracted from the milk of transgenic rabbits, is a glycoprotein of 478 amino acids with an identical amino acid sequence to the endogenous human C1-INH protein. Population pharmacokinetic analysis of rhC1-INH supports an intravenous dosing strategy of 50 U/kg (maximum 4200 U). The safety and efficacy of rhC1-INH in the treatment of acute attacks in patients with HAE were demonstrated in three randomized, double-blind, placebo-controlled studies and two open-label extension studies. In a pilot prophylaxis study, weekly administration of rhC1-INH 50 U/kg for 8 weeks reduced the incidence of HAE attacks and was well tolerated. Administration of rhC1-INH has not been associated with the development of anti-drug antibodies or antibodies to anti-host-related impurities. SN - 1179-1918 UR - https://www.unboundmedicine.com/medline/citation/26091744/Recombinant_human_C1_esterase_inhibitor_in_the_management_of_hereditary_angioedema_ L2 - https://dx.doi.org/10.1007/s40261-015-0300-z DB - PRIME DP - Unbound Medicine ER -