The New Era of C1-Esterase Inhibitor Deficiency Therapy

Tourangeau, Louanne M.; Zuraw, Bruce L.

doi:10.1007/s11882-011-0213-8

Cited by 10 publications

(5 citation statements)

References 42 publications

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“…Activation of the plasma contact system with generation of bradykinin has been shown to be the major cause of swelling in HAE due to C1INH deficiency, 25,29 -36 and these drugs are thought to interfere with the generation, catabolism, or signaling of bradykinin. [37][38][39] The discovery of F12 mutations linked to the inheritance of HAE with normal C1INH represented a key advance in our understanding of this disease with important pathophysiological implications. Coagulation fXII is a zymogen serine protease that is part of the intrinsic coagulation system and initiates plasma contact system activation when plasma is exposed to a negatively charged surface or a variety of other activating signals.…”

Section: Key Pointsmentioning

confidence: 99%

Hereditary angioedema with normal C1 inhibitor function: Consensus of an international expert panel

Zuraw

Bork²,

Binkley³

et al. 2012

allergy asthma proc

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146

111

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A new form of hereditary angioedema (HAE) with normal C1 inhibitor (C1INH) was first described in 2000. The lack of clear diagnostic criteria, the heterogeneity among affected patients, and the varying names given to this disease have led to substantial confusion among both physicians and patients. This study was designed to bring more clarity to the diagnosis and potential treatment of HAE with normal C1INH. An international symposium of experts was convened to review the field and develop consensus opinions that could help clinicians who evaluate and manage these patients. Criteria were developed for the diagnosis of HAE with normal C1INH in patients with recurrent angioedema in the absence of concurrent urticaria. In addition, potential therapeutic strategies are discussed. The consensus criteria developed during this symposium will allow physicians to better diagnose and treat patients with HAE with normal C1INH.

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Section: Key Pointsmentioning

confidence: 99%

Hereditary angioedema with normal C1 inhibitor function: Consensus of an international expert panel

Zuraw

Bork²,

Binkley³

et al. 2012

allergy asthma proc

Self Cite

146

111

View full text Add to dashboard Cite

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“…This large glycoprotein that belongs to the SERPIN family is primarily used in connection with hereditary angioedema (HAE) (Tourangeau and Zuraw 2011), a genetic disease caused by a deficiency of functional C1-INH and characterized by a swelling of subcutaneous tissue. While C1-INH preparations have been available for the treatment of HAE for several years in Europe and other regions, the introduction of such drugs into the US market happened only recently.…”

Section: 3 a Handyman's Toolbox Of Complement Inhibitionmentioning

confidence: 99%

Progress and Trends in Complement Therapeutics

Ricklin

Lambris

2012

Advances in Experimental Medicine and Biology

108

104

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The past few years have proven to be a highly successful and exciting period for the field of complement-directed drug discovery and development. Driven by promising experiences with the first marketed complement drugs, increased knowledge about the involvement of complement in health and disease, and improvements in structural and analytical techniques as well as animal models of disease, the field has seen a surge in creative approaches to therapeutically intervene at various stages of the cascade. An impressive panel of compounds that show promise in clinical trials is meanwhile being lined up in the pipelines of both small biotechnology and big pharmaceutical companies. Yet with this new focus on complement-targeted therapeutics, important questions concerning target selection, point and length of intervention, safety, and drug delivery emerge. In view of the diversity of the clinical disorders involving abnormal complement activity or regulation, which include both acute and chronic diseases and affect a wide range of organs, diverse yet specifically tailored therapeutic approaches may be needed to shift complement back into balance. This chapter highlights the key changes in the field that shape our current perception of complement-targeted drugs and provides a brief overview of recent strategies and emerging trends. Selected examples of complement-related diseases and inhibitor classes are highlighted to illustrate the diversity and creativity in field.

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“…Therapy for HAE consists of treatment of acute attacks, as well as short‐term and long‐term prophylaxis . Until recently, except in Western Europe, therapeutic options for HAE were limited, and consisted of androgens, narcotics, hydration and fresh frozen plasma (FFP) . Androgens are used for both short and long‐term prophylaxis and are effective, but the long‐term use may have significant adverse events especially when the dose exceeds 200 mg a day of danazol.…”

Section: Introductionmentioning

confidence: 99%

Update on treatment of hereditary angioedema

Buyantseva

Agarwal

et al. 2013

Clin Experimental Allergy

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Hereditary angioedema (HAE) is a rare disease characterized by recurrent, self-limiting episodes of swelling. New research and therapies have recently emerged and are now available; however, many physicians are not aware of the new developments in HAE. To update immunologists and other health care providers on new advances in HAE therapies, a PubMed, OVID and Google literature search were used to develop this manuscript. English language peer-reviewed angioedema articles were selected. High quality clinical trials were reviewed and summarized. Acute therapy in the past often consisted of symptom relief with narcotics, hydration and fresh frozen plasma (FFP). Androgens and FFP are frequently used despite multiple, significant side-effects. Newer therapies include C1-inhibitor - both human plasma derived and recombinant - as well as contact system modulators such as ecallantide and icatibant. These newer products can be used for treatment of acute attacks of HAE, and C1-inhibitors can also be used for prophylaxis. These disease-specific therapies have proven to work by placebo-controlled studies, have minimal adverse effects and can be utilized for the treatment of HAE.

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The New Era of C1-Esterase Inhibitor Deficiency Therapy

Cited by 10 publications

References 42 publications

Hereditary angioedema with normal C1 inhibitor function: Consensus of an international expert panel

Hereditary angioedema with normal C1 inhibitor function: Consensus of an international expert panel

Progress and Trends in Complement Therapeutics

Update on treatment of hereditary angioedema

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