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A Decade of Change: Recent Developments in Pharmacotherapy of Hereditary Angioedema (HAE).
Clin Rev Allergy Immunol 2016; 51(2):183-92CR

Abstract

Hereditary angioedema (HAE) due to C1 esterase inhibitor (C1-INH) deficiency (HAE-C1-INH) is a rare but medically significant disease that can be associated with considerable morbidity and mortality. Research into the pathogenesis of HAE-C1-INH has expanded greatly in the last six decades and has led to new clinical trials with novel therapeutic agents and treatment strategies. Mechanisms of pharmacotherapy include (a) supplementing C1-INH, the missing serine-protease inhibitor in HAE; (b) inhibiting the activation of the contact system and the uncontrolled release of proteases in the kallikrein-kinin system, by blocking the production/function of its components; (c) inhibiting the fibrinolytic system by blocking the production/function of its components; and (d) inhibiting the function of bradykinin at the endothelial level. Strategies for managing HAE-C1-INH are aimed at treating acute attacks, or preventing attacks, through the use of prophylactic treatment. Available agents for treating acute attacks include plasma-derived C1-INH concentrates, a recombinant C1-INH, a bradykinin B2 receptor antagonist, and a plasma kallikrein inhibitor. Long-term prophylactic treatments include attenuated androgens, plasma-derived C1-INH concentrates, and anti-fibrinolytics. Plasma-derived C1-INH and a bradykinin B2 receptor antagonist are already approved for self-administration at home. The number of management options for HAE-C1-INH has increased considerably within the past decade, thus helping to alleviate the burden of this rare disease.

Authors+Show Affiliations

Department of Dermatology, Johannes Gutenberg University, Langenbeckstr 1, 55131, Mainz, Germany. bork@hautklinik.klinik.uni-mainz.de.

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

27207174

Citation

Bork, Konrad. "A Decade of Change: Recent Developments in Pharmacotherapy of Hereditary Angioedema (HAE)." Clinical Reviews in Allergy & Immunology, vol. 51, no. 2, 2016, pp. 183-92.
Bork K. A Decade of Change: Recent Developments in Pharmacotherapy of Hereditary Angioedema (HAE). Clin Rev Allergy Immunol. 2016;51(2):183-92.
Bork, K. (2016). A Decade of Change: Recent Developments in Pharmacotherapy of Hereditary Angioedema (HAE). Clinical Reviews in Allergy & Immunology, 51(2), pp. 183-92. doi:10.1007/s12016-016-8544-9.
Bork K. A Decade of Change: Recent Developments in Pharmacotherapy of Hereditary Angioedema (HAE). Clin Rev Allergy Immunol. 2016;51(2):183-92. PubMed PMID: 27207174.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - A Decade of Change: Recent Developments in Pharmacotherapy of Hereditary Angioedema (HAE). A1 - Bork,Konrad, PY - 2016/5/22/entrez PY - 2016/5/22/pubmed PY - 2017/2/7/medline KW - C1 inhibitor KW - C1-INH deficiency KW - Hereditary angioedema KW - Prophylaxis KW - Treatment for acute attacks SP - 183 EP - 92 JF - Clinical reviews in allergy & immunology JO - Clin Rev Allergy Immunol VL - 51 IS - 2 N2 - Hereditary angioedema (HAE) due to C1 esterase inhibitor (C1-INH) deficiency (HAE-C1-INH) is a rare but medically significant disease that can be associated with considerable morbidity and mortality. Research into the pathogenesis of HAE-C1-INH has expanded greatly in the last six decades and has led to new clinical trials with novel therapeutic agents and treatment strategies. Mechanisms of pharmacotherapy include (a) supplementing C1-INH, the missing serine-protease inhibitor in HAE; (b) inhibiting the activation of the contact system and the uncontrolled release of proteases in the kallikrein-kinin system, by blocking the production/function of its components; (c) inhibiting the fibrinolytic system by blocking the production/function of its components; and (d) inhibiting the function of bradykinin at the endothelial level. Strategies for managing HAE-C1-INH are aimed at treating acute attacks, or preventing attacks, through the use of prophylactic treatment. Available agents for treating acute attacks include plasma-derived C1-INH concentrates, a recombinant C1-INH, a bradykinin B2 receptor antagonist, and a plasma kallikrein inhibitor. Long-term prophylactic treatments include attenuated androgens, plasma-derived C1-INH concentrates, and anti-fibrinolytics. Plasma-derived C1-INH and a bradykinin B2 receptor antagonist are already approved for self-administration at home. The number of management options for HAE-C1-INH has increased considerably within the past decade, thus helping to alleviate the burden of this rare disease. SN - 1559-0267 UR - https://www.unboundmedicine.com/medline/citation/27207174/A_Decade_of_Change:_Recent_Developments_in_Pharmacotherapy_of_Hereditary_Angioedema__HAE__ L2 - https://dx.doi.org/10.1007/s12016-016-8544-9 DB - PRIME DP - Unbound Medicine ER -