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Clinical Reviews in Allergy & Immunology

Published in:

01-08-2011

Therapeutic Approaches in Hereditary Angioedema

Author: Sabina Antonela Antoniu

Published in: Clinical Reviews in Allergy & Immunology | Issue 1/2011

Abstract

Hereditary angioedema (HAE) is characterized by acute attacks of edema with multiple localizations, the laryngeal angioedema being the most potentially lethal. In HAE, C1-INH impairments cause episodic increase in kallikrein activity leading to attacks of angioedema. Several therapies have recently become available to treat or to prevent HAE attacks, and others are under evaluation for this indication. Plasma-derived C1-INH, bradykinin receptor antagonists (icatibant), kallikrein inhibitors (ecallantide), or recombinant C1-INH is authorized on the market for HAE attack therapy or prophylaxis. Some of these compounds can be used exclusively to treat HAE attacks, whereas others can also be used as prophylactic therapies. Such therapies, although not available worldwide, can improve disease outcome due to their different mechanisms of action.

Bowen T, Cicardi M, Farkas H et al (2010) International consensus algorithm for the diagnosis, therapy and management of hereditary angioedema. Allergy Asthma Clin Immunol 6(1):24PubMedCrossRef

Zuraw BL HAE therapies: past present and future. Allergy Asthma Clin Immunol 6(1):23

Kaplan AP Enzymatic pathways in the pathogenesis of hereditary angioedema: The role of C1 inhibitor therapy. J Allergy Clin Immunol

Cugno M, Zanichelli A, Foieni F, Caccia S, Cicardi M (2009) C1-inhibitor deficiency and angioedema: molecular mechanisms and clinical progress. Trends Mol Med 15(2):69–78PubMedCrossRef

Craig T, Riedl M, MS Dykewicz et al (2009) When is prophylaxis for hereditary angioedema necessary? Ann Allergy Asthma Immunol 102(5):366–372PubMedCrossRef

AE Davis (2005) 3RD: the pathophysiology of hereditary angioedema. Clin Immunol 114(1):3–9CrossRef

Quastel M, Harrison R, Cicardi M, CA Alper, FS Rosen (1983) Behavior in vivo of normal and dysfunctional C1 inhibitor in normal subjects and patients with hereditary angioneurotic edema. J Clin Invest 71(4):1041–1046PubMedCrossRef

Cugno M, Nuijens J, Hack E et al (1990) Plasma levels of C1- inhibitor complexes and cleaved C1- inhibitor in patients with hereditary angioneurotic edema. J Clin Invest 85(4):1215–1220PubMedCrossRef

Pappalardo E, LC Zingale, Terlizzi A et al (2002) Mechanisms of C1-inhibitor deficiency. Immunobiology 205(4–5):542–551PubMedCrossRef

10.

Pappalardo E, LC Zingale, Cicardi M (2004) C1 inhibitor gene expression in patients with hereditary angioedema: quantitative evaluation by means of real-time RT-PCR. J Allergy Clin Immunol 114(3):638–644PubMedCrossRef

11.

ED Han, RC Macfarlane, AN Mulligan, Scafidi J, AE Davis (2002) 3RD: increased vascular permeability in C1 inhibitor-deficient mice mediated by the bradykinin type 2 receptor. J Clin Invest 109(8):1057–1063

12.

AP Kaplan, Joseph K, Silverberg M (2002) Pathways for bradykinin formation and inflammatory disease. J Allergy Clin Immunol 109(2):195–209CrossRef

13.

Joseph K, BG Tholanikunnel, AP Kaplan (2002) Heat shock protein 90 catalyzes activation of the prekallikrein-kininogen complex in the absence of factor XII. Proc Natl Acad Sci USA 99(2):896–900PubMedCrossRef

14.

KD Bhoola, CD Figueroa, Worthy K (1992) Bioregulation of kinins: kallikreins, kininogens, and kininases. Pharmacol Rev 44(1):1–80

15.

Marcondes S, Antunes E (2005) The plasma and tissue kininogen-kallikrein-kinin system: role in the cardiovascular system. Curr Med Chem Cardiovasc Hematol Agents 3(1):33–44PubMedCrossRef

16.

Longhurst HJ Management of acute attacks of hereditary angioedema: potential role of icatibant. Vasc Health Risk Manag 6:795–802

17.

Cugno M, Nussberger J, Cicardi M, Agostoni A (2003) Bradykinin and the pathophysiology of angioedema. Int Immunopharmacol 3(3):311–317PubMedCrossRef

18.

Nussberger J, Cugno M, Amstutz C et al (1998) Plasma bradykinin in angio-oedema. Lancet 351(9117):1693–1697PubMedCrossRef

19.

Cugno M, Cicardi M, Bottasso B et al (1997) Activation of the coagulation cascade in C1-inhibitor deficiencies. Blood 89(9):3213–3218PubMed

20.

FJ Hock, Wirth K, Albus U et al (1991) Hoe 140 a new potent and long acting bradykinin-antagonist: in vitro studies. Br J Pharmacol 102(3):769–773

21.

Wirth K, FJ Hock, Albus U et al (1991) Hoe 140 a new potent and long acting bradykinin-antagonist: in vivo studies. Br J Pharmacol 102(3):774–777PubMed

22.

JR Cockcroft, PJ Chowienczyk, SE Brett, Bender N, JM Ritter (1994) Inhibition of bradykinin-induced vasodilation in human forearm vasculature by icatibant, a potent B2-receptor antagonist. Br J Clin Pharmacol 38(4):317–321

23.

http://www.ema.europa.euFirazyr: EPAR-product information (2008)

24.

Bork K, Frank J, Grundt B et al (2007) Treatment of acute edema attacks in hereditary angioedema with a bradykinin receptor-2 antagonist (Icatibant). J Allergy Clin Immunol 119(6):1497–1503PubMedCrossRef

25.

Cicardi M, Banerji A, Bracho F et al. Icatibant, a new bradykinin-receptor antagonist, in hereditary angioedema. N Engl J Med 363(6):532–541

26.

JA Bernstein (2008) Hereditary angioedema: a current state-of-the-art review, VIII: current status of emerging therapies. Ann Allergy Asthma Immunol 100(1 Suppl 2):S41–S46

27.

Bernstein JA, Qazi M Ecallantide: its pharmacology, pharmacokinetics, clinical efficacy and tolerability. Expert Rev Clin Immunol 6(1):29–39

28.

Schneider L, Lumry W, Vegh A, AH Williams, Schmalbach T (2007) Critical role of kallikrein in hereditary angioedema pathogenesis: a clinical trial of ecallantide, a novel kallikrein inhibitor. J Allergy Clin Immunol 120(2):416–422PubMedCrossRef

29.

Cicardi M, Levy RJ, Mcneil DL et al. Ecallantide for the treatment of acute attacks in hereditary angioedema. N Engl J Med 363(6):523–531

30.

Levy RJ, Lumry WR, Mcneil DL et al. EDEMA4: a phase 3, double-blind study of subcutaneous ecallantide treatment for acute attacks of hereditary angioedema. Ann Allergy Asthma Immunol 104(6):523–529

31.

Davis AE, 3RD, Lu F, Mejia P C1 inhibitor, a multi-functional serine protease inhibitor. Thromb Haemost 104(5)

32.

C1 esterase inhibitor (human) P T 35(7 Section 2):2–3

33.

ED Hanlee, Pappalardo E, Scafidi J, AE Davis (2003) 3RD: approaches toward reversal of increased vascular permeability in C1 inhibitor deficient mice. Immunol Lett 89(2–3):155–160

34.

TJ Craig, RJ Levy, RL Wasserman et al (2009) Efficacy of human C1 esterase inhibitor concentrate compared with placebo in acute hereditary angioedema attacks. J Allergy Clin Immunol 124(4):801–808CrossRef

35.

Bernstein JA, Ritchie B, LEVY RJ et al. Population pharmacokinetics of plasma-derived C1 esterase inhibitor concentrate used to treat acute hereditary angioedema attacks. Ann Allergy Asthma Immunol 105(2):149–154

36.

Craig TJ, Wasserman RL, Levy RJ et al. Prospective study of rapid relief provided by C1 esterase inhibitor in emergency treatment of acute laryngeal attacks in hereditary angioedema. J Clin Immunol

37.

Cocchio C, Marzella N (2009) Cinryze, a human plasma-derived C1 esterase inhibitor for prophylaxis of hereditary angioedema. P T 34(6):293–328PubMed

38.

Zuraw BL, Busse PJ, White M et al. Nanofiltered C1 inhibitor concentrate for treatment of hereditary angioedema. N Engl J Med 363(6):513–522

39.

Eldering E, JH Nuijens, CE Hack (1988) Expression of functional human C1 inhibitor in COS cells. J Biol Chem 263(24):11776–11779PubMed

40.

Lamark T, Ingebrigtsen M, Bjornstad C et al (2001) Expression of active human C1 inhibitor serpin domain in escherichia coli. Protein Expr Purif 22(2):349–358PubMedCrossRef

41.

van Doorn MB, Burggraaf J, van Dam T et al (2005) A phase I study of recombinant human C1 inhibitor in asymptomatic patients with hereditary angioedema. J Allergy Clin Immunol 116(4):876–883PubMedCrossRef

42.

Choi G, MR Soeters, Farkas H et al (2007) Recombinant human C1-inhibitor in the treatment of acute angioedema attacks. Transfusion 47(6):1028–1032PubMedCrossRef

43.

Zuraw B, Cicardi M, Levy RJ et al. Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol 126(4):821–827 e814

44.

http://www.ema.europa.euCommittee for medicinal products for human use positive summary of opinion for ruconest (2010)

45.

IG Bos, EC Debruin, YA Karuntu et al (2003) Recombinant human C1-inhibitor produced in Pichia pastoris has the same inhibitory capacity as plasma C1-inhibitor. Biochim Biophys Acta 1648(1–2):75–83

46.

Agostoni A, Aygoren-Pursun E, KE Binkley et al (2004) Hereditary and acquired angioedema: problems and progress: proceedings of the third C1 esterase inhibitor deficiency workshop and beyond. J Allergy Clin Immunol 114(3 Suppl):S51–S131PubMedCrossRef

47.

MC Owen, SO Brennan, JH Lewis, RW Carrell (1983) Mutation of antitrypsin to antithrombin. alpha 1-antitrypsin Pittsburgh (358 Met leads to Arg), a fatal bleeding disorder. N Engl J Med 309(12):694–698CrossRef

48.

Schapira M, MA Ramus, Jallat S, Carvallo D, Courtney M (1986) Recombinant alpha 1-antitrypsin Pittsburgh (Met 358––Arg) is a potent inhibitor of plasma kallikrein and activated factor XII fragment. J Clin Invest 77(2):635–637PubMedCrossRef

49.

PA Patston, Roodi N, JA Schifferli et al (1990) Reactivity of alpha 1-antitrypsin mutants against proteolytic enzymes of the kallikrein-kinin, complement, and fibrinolytic systems. J Biol Chem 265(18):10786–10791

50.

Sulikowski T, BA Bauer, PA Patston (2002) Alpha(1)-Proteinase inhibitor mutants with specificity for plasma kallikrein and C1s but not C1. Protein Sci 11(9):2230–2236PubMedCrossRef

Title: Therapeutic Approaches in Hereditary Angioedema
Author: Sabina Antonela Antoniu
Publication date: 01-08-2011
Publisher: Humana Press Inc
Published in: Clinical Reviews in Allergy & Immunology / Issue 1/2011
Print ISSN: 1080-0549
Electronic ISSN: 1559-0267
DOI: https://doi.org/10.1007/s12016-011-8254-2

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