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Treatment of Hereditary Angioedema

Caballero T1,2,3

1Allergy Department, Hospital Universitario La Paz, Madrid, Spain
2Hospital La Paz Institute for Health Research (IdiPaz), Madrid, Spain
3Center for Biomedical Research Network on Rare Diseases (CIBERER U754), Madrid, Spain

J Investig Allergol Clin Immunol 2021; Vol 31(1) : 1-16
doi: 10.18176/jiaci.0653

Hereditary angioedema due to C1-esterase inhibitor deficiency (C1-INH-HAE) is a rare autosomal dominant disease.
In the last decade, new drugs and new indications for old drugs have played a role in the management of C1-INH-HAE. This review examines current therapy for C1-INH-HAE and provides a brief summary of drugs that are under development. Increased knowledge of the pathophysiology of C1-INH-HAE has been crucial for advances in the field, with inhibition of the kallikrein-kinin system (plasma kallikrein, activated factor XII) as a key area in the discovery of new drugs, some of which are already marketed for treatment of C1-INH-HAE. Pharmacological treatment is based on 3 pillars: treatment of acute angioedema attacks (on-demand treatment), short-term (preprocedure) prophylaxis, and long-term prophylaxis.
The 4 drugs that are currently available for the treatment of acute angioedema attacks (purified plasma-derived human C1 esterase inhibitor concentrate, icatibant acetate, ecallantide, recombinant human C1 esterase inhibitor) are all authorized for self-administration, except ecallantide.
Purified plasma-derived human C1 esterase inhibitor concentrate is the treatment of choice for short-term prophylaxis.
Tranexamic acid, danazol, intravenous and subcutaneous nanofiltered purified plasma-derived human C1 esterase inhibitor concentrate,
and lanadelumab can be used for long-term prophylaxis. New drugs are being investigated, mainly as long-term prophylaxis, and are aimed at blocking the kallikrein-kinin system by means of antiprekallikrein, antikallikrein, and anti–activated FXII action.

Key words: Hereditary angioedema, C1 inhibitor, Bradykinin, Kallikrein, Treatment