Abstract
Background
Hereditary angioedema (HAE) is a rare, potentially life-threatening autosomal dominant
disease characterized by recurrent angioedema attacks that affect the skin, gastrointestinal
tract, and airway, including the larynx. Pharmacologic developments in HAE treatment
have culminated in the recent introduction of 4 new HAE-specific therapies in the
United States.
Objectives
In light of these new therapeutic options, this commentary outlines historical US
HAE therapy choices, discusses the potential effect of the 4 recently approved HAE
treatments, and considers strategies for optimizing their use in line with international
treatment recommendations.
Discussion
Treatment options for HAE in the United States have been limited to attenuated androgens
and antifibrinolytic agents for long-term prophylaxis and FFP and supportive therapy
for the management of acute attacks. The 4 new therapies that have recently become
available (ie, 2 plasma-derived C1 esterase inhibitor (C1-INH) concentrates, the kallikrein
inhibitor ecallantide, and the bradykinin β2-antagonist icatibant) have provided an opportunity to change routine HAE treatment.
In 2009, despite the availability of 2 of the new treatments (ie, the plasma-derived
C1-INH concentrates), a large survey of US physicians suggested that wide variability
still existed in the treatment of patients with HAE. Since this survey was undertaken,
clinical experience with all 4 new treatments has increased significantly, and because
3 of these agents (ie, 2 plasma-derived C1-INH concentrates and icatibant) can be
self-administered by trained patients, physicians can now provide individualized care
that is proven effective and more aligned with international guidance.
Key words
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Article info
Publication history
Published online: March 05, 2012
Accepted:
February 3,
2012
Identification
Copyright
© 2012 Elsevier HS Journals, Inc. Published by Elsevier Inc. All rights reserved.
