200
Views
23
CrossRef citations to date
0
Altmetric
Drug Profile

rhC1INH: a new drug for the treatment of attacks in hereditary angioedema caused by C1-inhibitor deficiency

&
Pages 143-153 | Published online: 10 Jan 2014

References

  • Varga L, Farkas H. Treatment of type I and II hereditary angioedema with Rhucin, a recombinant human C1 inhibitor. Expert Rev. Clin. Immunol.4(6), 653–661 (2008).
  • Donaldson VH, Evans RR. A biochemical abnormality in herediatry angioneurotic edema: absence of serum inhibitor of C´ 1-esterase. Am. J. Med.35, 37–44 (1963).
  • Pappalardo E, Caccia S, Suffritti C, Tordai A, Zingale LC, Cicardi M. Mutation screening of C1 inhibitor gene in 108 unrelated families with hereditary angioedema: functional and structural correlates. Mol. Immunol.45(13), 3536–3544 (2008).
  • Bork K. Diagnosis and treatment of hereditary angioedema with normal C1 inhibitor. Allergy Asthma Clin. Immunol.6(1), 15 (2010).
  • Cicardi M, Zanichelli A. Acquired angioedema. Allergy Asthma Clin. Immunol.6(1), 14 (2010).
  • Davis AE, 3rd. Hereditary angioedema: a current state-of-the-art review, III: mechanisms of hereditary angioedema. Ann. Allergy Asthma Immunol.100(1 Suppl. 2), S7–S12 (2008).
  • Gurewich V, Pannell R. Recombinant human C1-inhibitor prevents non-specific proteolysis by mutant pro-urokinase during optimal fibrinolysis. Thromb. Haemost.102(2), 279–286 (2009).
  • Kaplan AP, Ghebrehiwet B. The plasma bradykinin-forming pathways and its interrelationships with complement. Mol. Immunol.47(13), 2161–2169 (2010).
  • Bork K, Hardt J, Schicketanz KH, Ressel N. Clinical studies of sudden upper airway obstruction in patients with hereditary angioedema due to C1 esterase inhibitor deficiency. Arch. Intern. Med.163(10), 1229–1235 (2003).
  • Agostoni A, Cicardi M. Hereditary and acquired C1-inhibitor deficiency: biological and clinical characteristics in 235 patients. Medicine (Baltimore)71(4), 206–215 (1992).
  • Frank MM, Gelfand JA, Atkinson JP. Hereditary angioedema: the clinical syndrome and its management. Ann. Intern. Med.84(5), 580–593 (1976).
  • Agostoni A, Aygoren-Pursun E, Binkley KE et al. 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–S131 (2004).
  • Bas M, Hoffmann TK, Kojda G, Bier H. [ACE-inhibitor induced angioedema]. Laryngorhinootologie86(11), 804–808, quiz 9–13 (2007).
  • Bork K, Fischer B, Dewald G. Recurrent episodes of skin angioedema and severe attacks of abdominal pain induced by oral contraceptives or hormone replacement therapy. Am. J. Med.114(4), 294–298 (2003).
  • Bork K, Meng G, Staubach P, Hardt J. Hereditary angioedema: new findings concerning symptoms, affected organs, and course. Am. J. Med.119(3), 267–274 (2006).
  • Farkas H, Harmat G, Fay A, Fekete B, Karadi I, Visy B, Varga L. Erythema marginatum preceding an acute oedematous attack of hereditary angioneurotic oedema. Acta Derm. Venereol.81(5), 376–377 (2001).
  • Kemp JG, Craig TJ. Variability of prodromal signs and symptoms associated with hereditary angioedema attacks: a literature review. Allergy Asthma Proc.30(5), 493–499 (2009).
  • Farkas H, Harmat G, Kaposi PN et al. Ultrasonography in the diagnosis and monitoring of ascites in acute abdominal attacks of hereditary angioneurotic oedema. Eur. J. Gastroenterol. Hepatol.13(10), 1225–1230 (2001).
  • Bork K, Staubach P, Eckardt AJ, Hardt J. Symptoms, course, and complications of abdominal attacks in hereditary angioedema due to C1 inhibitor deficiency. Am. J. Gastroenterol.101(3), 619–627 (2006).
  • Neri S, Ierna D, Sfogliano L. Unusual manifestations of hereditary angioedema. Eur. J. Emerg. Med.7(2), 111–112 (2000).
  • Farkas H. Management of upper airway edema caused by hereditary angioedema. Allergy Asthma Clin. Immunol.6(1), 19 (2010).
  • 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), 24 (2010).
  • Cicardi M, Zanichelli A. Angioedema due to C1 inhibitor deficiency in 2010. Intern. Emerg. Med.5(6), 481–486 (2010).
  • Zuraw BL. HAE therapies: past present and future. Allergy Asthma Clin. Immunol.6(1), 23 (2010).
  • Longhurst HJ, Farkas H, Craig T et al. HAE international home therapy consensus document. Allergy Asthma Clin. Immunol.6(1), 22 (2010).
  • Gompels MM, Lock RJ, Abinun M et al. C1 inhibitor deficiency: consensus document. Clin. Exp. Immunol.139(3), 379–394 (2005).
  • Bowen T, Cicardi M, Bork K et al. Hereditary angiodema: a current state-of-the-art review, VII: Canadian Hungarian 2007 International Consensus Algorithm for the Diagnosis, Therapy, and Management of Hereditary Angioedema. Ann. Allergy Asthma Immunol.100(1 Suppl. 2), S30–S40 (2008).
  • Farkas H, Jakab L, Temesszentandrasi G et al. Hereditary angioedema: a decade of human C1-inhibitor concentrate therapy. J. Allergy Clin. Immunol.120(4), 941–947 (2007).
  • Craig TJ, Levy RJ, Wasserman RL et al. Efficacy of human C1 esterase inhibitor concentrate compared with placebo in acute hereditary angioedema attacks. J. Allergy Clin. Immunol.124(4), 801–808 (2009).
  • 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.30(6), 823–829 (2010).
  • 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, e14 (2010).
  • Bernstein JA, Qazi M. Ecallantide: its pharmacology, pharmacokinetics, clinical efficacy and tolerability. Expert Rev. Clin. Immunol.6(1), 29–39 (2010).
  • 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 (2010).
  • Longhurst HJ. Management of acute attacks of hereditary angioedema: potential role of icatibant. Vasc. Health Risk Manag.6, 795–802 (2010).
  • Prematta M, Gibbs JG, Pratt EL, Stoughton TR, Craig TJ. Fresh frozen plasma for the treatment of hereditary angioedema. Ann. Allergy Asthma Immunol.98(4), 383–388 (2007).
  • Birgerson L. Tranexamic acid in the treatment of hereditary angioedema. Am. J. Med.91(1), 102 (1991).
  • Fust G, Farkas H, Csuka D, Varga L, Bork K. Long-term efficacy of danazol treatment in hereditary angioedema. Eur. J. Clin. Invest.41(3), 256–262 (2011).
  • Bork K, Bygum A, Hardt J. Benefits and risks of danazol in hereditary angioedema: a long-term survey of 118 patients. Ann. Allergy Asthma Immunol.100(2), 153–161 (2008).
  • Farkas H, Czaller I, Csuka D et al. Theeffect of long-term danazol prophylaxis on liver function in hereditary angioedema – a longitudinal study. Eur. J. Clin. Pharmacol.66(4), 419–426 (2010).
  • 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 (2010).
  • Cocchio C, Marzella N. Cinryze, a human plasma-derived c1 esterase inhibitor for prophylaxis of hereditary angioedema. Pharm. Therap.34(6), 293–328 (2009).
  • Bork K, Hardt J. Hereditary angioedema: long-term treatment with one or more injections of C1 inhibitor concentrate per week. Int. Arch. Allergy Immunol.154(1), 81–88 (2010).
  • Saito T, Namura O, Honma T, Hayashi J. Supplementation of C1-esterase inhibitor concentrates for a patient suffering from hereditary angioedema undergoing complex open-heart surgery. Eur. J. Cardiothorac. Surg.37(4), 975–977 (2010).
  • Farkas H, Gyeney L, Gidofalvy E, Fust G, Varga L. The efficacy of short-term danazol prophylaxis in hereditary angioedema patients undergoing maxillofacial and dental procedures. J. Oral Maxillofac. Surg.57(4), 404–408 (1999).
  • Van Sickels NJ, Hunsaker RB, JE Van Sickels. Hereditary angioedema: treatment, management, and precautions in patients presenting for dental care. Oral Surg. Oral Med. Oral Pathol. Oral Radiol. Endod.109(2), 168–172 (2010).
  • Bernstein JA, Coleman S, Bonnin AJ. Successful C1 inhibitor short-term prophylaxis during redo mitral valve replacement in a patient with hereditary angioedema. J. Cardiothorac. Surg.5, 86 (2010).
  • Spyridonidou A, Iatrou C, Alexoudis A, Vogiatzaki T, Polychronidis A, Simopoulos C. Peri-operative management of a patient with hereditary angioedema undergoing laparoscopic cholecystectomy. Anaesthesia65(1), 74–77 (2010).
  • Farkas H, Varga L, Szeplaki G, Visy B, Harmat G, Bowen T. Management of hereditary angioedema in pediatric patients. Pediatrics120(3), e713–e22 (2007).
  • Farkas H. Pediatric hereditary angioedema due to C1-inhibitor deficiency. Allergy Asthma Clin. Immunol.6(1), 18 (2010).
  • Davis AE 3rd, Lu F, Mejia P. C1 inhibitor, a multi-functional serine protease inhibitor. Thromb. Haemost.104(5), 886–893 (2010).
  • Carter PE, Dunbar B, Fothergill JE. Genomic and cDNA cloning of the human C1 inhibitor. Intron-exon junctions and comparison with other serpins. Eur. J. Biochem.173(1), 163–169 (1988).
  • Perkins S. Three-dimensional structure and molecular modelling of C1-inhibitor. Behring Inst. Mitt.93, 63–80 (1993).
  • Eldering E, Nuijens JH, Hack CE. Expression of functional human C1 inhibitor in COS cells. J. Biol. Chem.263(24), 11776–11779 (1988).
  • Eldering E, Huijbregts CC, Lubbers YT, Longstaff C, Hack CE. Characterization of recombinant C1 inhibitor P1 variants. J. Biol. Chem.267(10), 7013–7020 (1992).
  • Lamark T, Ingebrigtsen M, Bjornstad C, Melkko T, Mollnes TE, Nielsen EW. Expression of active human C1 inhibitor serpin domain in Escherichia coli. Protein Expr. Purif.22(2), 349–358 (2001).
  • Wolff MW, Zhang F, Roberg JJ et al. Expression of C1 esterase inhibitor by the baculovirus expression vector system: preparation, purification, and characterization. Protein Expr. Purif.22(3), 414–421 (2001).
  • Longhurst H. Rhucin, a recombinant C1 inhibitor for the treatment of hereditary angioedema and cerebral ischemia. Curr. Opin. Investig. Drugs9(3), 310–323 (2008).
  • Koles K, van Berkel PH, Pieper FR et al. N- and O-glycans of recombinant human C1 inhibitor expressed in the milk of transgenic rabbits. Glycobiology14(1), 51–64 (2004).
  • Koles K, van Berkel PH, Mannesse ML, Zoetemelk R, Vliegenthart JF, Kamerling JP. Influence of lactation parameters on the N-glycosylation of recombinant human C1 inhibitor isolated from the milk of transgenic rabbits. Glycobiology14(11), 979–986 (2004).
  • van Doorn MB, Burggraaf J, T van Dam et al. A Phase I study of recombinant human C1 inhibitor in asymptomatic patients with hereditary angioedema. J. Allergy Clin. Immunol.116(4), 876–883 (2005).
  • Relan A, Hack CE, Haase G, Pijpstra R. Mechanistic implications from the treatment of acute angioedema attacks in hereditary angioedema (HAE) with recombinant human C1 inhibitor (rhC1INH) (poster). Presented at: XXIII International Complement Workshop. NY, USA, 1–5 August 2010.
  • Choi G, Soeters MR, Farkas H et al. Recombinant human C1-inhibitor in the treatment of acute angioedema attacks. Transfusion47(6), 1028–1032 (2007).
  • Relan A, Haase G, Hack E, Nuijens J, Giannetti B, Pijpstra R. Dose justification for recombinant human C1INH for the treatment of acute angioedema attacks in patients with hereditary angioedema. Allergy65(s92), S1186 (2010).
  • Moldovan D, Levy RJ, Visscher S, Relan A, Nuijens JH, Hack CE. Interim results from ongoing open-label studies with recombinant C1 inhibitor (Rhucin; rC1INH) for treatment of patients with acute attacks of hereditary angioedema (oral presentation). Presented at: 6th C1 Inhibitor Deficiency Workshop 2009. Budapest, Hungary, 22–24 May 2009.
  • Kusuma A, Relan A, Haase G et al. Clinical value for the treatment with rhC1INH of patients with hereditary angioedema (HAE) suffering from an acute peripheral angioedema attack. Allergy65(S92), 1182 (2010).
  • Varga L, Szeplaki G, Visy B et al. C1-inhibitor (C1-INH) autoantibodies in hereditary angioedema. Strong correlation with the severity of disease in C1-INH concentrate naive patients. Mol. Immunol.44(6), 1454–1460 (2007).
  • Baboeram A, Relan A, Hack E et al. Immunogenicity assessment of recombinant human C1 Inhibitor (rhC1INH). Allergy65(S92), 1184 (2010).
  • Pijpstra R, Hack E, Baboeram A et al. IgE against rabbit allergens and the therapeutic use of recombinant human C1 inhibitor. Allergy65(S92), S1185 (2010).
  • Tillou X, Poirier N, Le Bas-Bernardet S et al. Recombinant human C1-inhibitor prevents acute antibody-mediated rejection in alloimmunized baboons. Kidney Int.78(2), 152–159 (2010).
  • Gesuete R, Storini C, Fantin A et al. Recombinant C1 inhibitor in brain ischemic injury. Ann. Neurol.66(3), 332–342 (2009).

Websites

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.