New promise and hope for treating hereditary angioedema

Expert Opinion on Investigational Drugs

Volumn 17, Issue 5, 2008, Pages 697-706

  Zuraw, Bruce L ^a   Christiansen, Sandra C ^a  

^a UNIVERSITY OF CALIFORNIA SAN DIEGO   (United States)

Author keywords

Bradykinin; C1 inhibitor; DX 88; HwAE; Icatibant

Indexed keywords

ANDROGEN; ANTIFIBRINOLYTIC AGENT; BERINERT P; BLOOD CLOTTING FACTOR 11A; BRADYKININ; CINRYZE; COMPLEMENT COMPONENT C1R; COMPLEMENT COMPONENT C1S; COMPLEMENT COMPONENT C1S INHIBITOR; DX 88; ECALLANTIDE; FRESH FROZEN PLASMA; ICATIBANT; KALLIKREIN; KALLIKREIN INHIBITOR; PLACEBO; PROTEIN ANTIBODY; RECOMBINANT HUMAN C1S INHIBITOR; RHUCIN; TRANEXAMIC ACID; RECOMBINANT PROTEIN; SERINE PROTEINASE INHIBITOR; SERPING1 PROTEIN, HUMAN;

ANGIONEUROTIC EDEMA; ANTIBODY BLOOD LEVEL; BLOOD DONOR; BLOOD VESSEL PERMEABILITY; CLINICAL TRIAL; DRUG CLEARANCE; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG FORMULATION; DRUG HALF LIFE; DRUG MECHANISM; DRUG MEGADOSE; DRUG SAFETY; DRUG TOLERABILITY; ENZYME INHIBITION; HEPATITIS C; HUMAN; INFECTION RISK; INJECTION SITE ERYTHEMA; INJECTION SITE PAIN; NONHUMAN; PATIENT ATTITUDE; PHYSICIAN ATTITUDE; PROPHYLAXIS; REVIEW; SINGLE DRUG DOSE; UNSPECIFIED SIDE EFFECT; VIRUS TRANSMISSION; ANIMAL; CHEMISTRY; PHASE 3 CLINICAL TRIAL; PLASMA; PLASMAPHERESIS;

ANGIOEDEMA, HEREDITARY; ANIMALS; CLINICAL TRIALS, PHASE III AS TOPIC; COMPLEMENT C1 INACTIVATOR PROTEINS; HUMANS; PLASMA; PLASMA EXCHANGE; RECOMBINANT PROTEINS; SERPINS;

EID: 44449141099     PISSN: 13543784     EISSN: None     Source Type: Journal    
DOI: 10.1517/13543784.17.5.697     Document Type: Review

References (57)

1. Spaulding WB. Methyltestosterone therapy for hereditary episodic edema (hereditary angioneurotic edema). Ann Intern Med 1960;53:739-45
2. Nilsson IM, Andersson L, Bjorkman SE. Epsilon-aminocaproic acid (E-ACA) as a therapeutic agent based on 5 year's clinical experience. Acta. Med Scand Suppl 1966;448:1-46
3. Sloane DE, Lee CW, Sheffer AL. Hereditary angioedema: safety of long-term stanozolol therapy. J Allergy Clin Immunol 2007;120:654-8
4. Cicardi M, Castelli R, Zingale LC, Agostoni A. Side effects of long-term prophylaxis with attenuated androgens in hereditary angioedema: comparison of treated and untreated patients. J Allergy Clin Immunol 1997;99:194-6
5. Bork K, Pitton M, Harten P, Koch P. Hepatocellular adenomas in patients taking danazol for hereditary angio-oedema. Lancet 1999;353:1066-7
6. Szeplaki G, Varga L, Valentin S, et al. Adverse effects of danazol prophylaxis on the lipid profiles of patients with hereditary angioedema. J Allergy Clin Immunol 2005;115:864-9
7. Monnier N, Ponard D, Duponchel C, et al. Characterisation of a new C1 inhibitor mutant in a patient with hepatocellular carcinoma. Mol Immunol 2006;43:2161-8
8. Donaldson VH, Evans RR. A biochemical abnormality in hereditary angioneurotic edema: absence of serum inhibitor of C1 - esterase. Am J Med 1963;35:37-44
9. Landerman NS, Webster ME, Becker EL, Ratdiffe HE. Hereditary angioneurotic edema. II. Deficiency of inhibitor for serum globulin permeability factor and/or plasma kallikrein. J Allergy 1962;33:330-41
10. Pickering RJ, Good RA, Kelly JR, Gewurz H. Replacement therapy in hereditary angioedema. Successful treatment of two patients with fresh frozen plasma. Lancet 1969;1:326-30
11. Marasini B, Cicardi M, Martignoni GC, Agostoni A. Treatment of hereditary angioedema. Klin Wochenschr 1978;56:819-23
12. Agostoni A, Bergamaschini L, Martignoni G, et al. Treatment of acute attacks of hereditary angioedema with C1-inhibitor concentrate. Ann Allergy 1980;44:299-301
13. Gadek JE, Hosea SW, Gelfand JA, et al. Replacement therapy in hereditary angioedema. Successful treatment of acute episodes of angioedema with partly purified C1 inhibitor. N Engl J Med 1980;302:542-6
14. Bergamaschini L, Cicardi M, Tucci A, et al. C1 INH concentrate in the therapy of hereditiry angioedema. Allergy 1983;38:81-4
15. Logan RA, Greaves MW. Hereditary angio-oedema: treatment with C1 esterase inhibitor concentrate. J R Soc Med 1984;77:1046-8
16. Bork K, Barnstedt SE. Treatment of 193 episodes of laryngeal edema with C1 inhibitor concentrate in patients with hereditary angioedema. Arch Intern Med 2001;161:714-8
17. Kirschfink M, Mollnes TE. C1-inhibitor: an anti-inflammatory reagent with therapeutic potential. Expert Opin Pharmacother 2001;2:10713-83
18. Bork K, Meng G, Staubach P, Hardt J. Treatment with C1 inhibitor concentrate in abdominal pain attacks of patients with hereditary angiocdema. Transfusion 2005;45:1774-84
19. Maves KK, Weiler JM. Tonsillectomy in a patient with hereditary angioedema after prophylaxis with C1 inhibitor concentrate. Ann Allergy 1994;73:435-81
20. Leimgruber A, Jaques WA, Spaeth PJ. Hereditary angioedema: uncomplicated maxillofacial surgery using short-term C1 inhibitor replacement therapy. Int Arch Allergy Immunol 1993;101:107-12
21. Mohr M, Pollok-Kopp B, Gotze O, Burchardi H. The use of a C1-inhibior concentrate for short-term preoperative prophylaxis in two patients with hereditary angioedema. Anaesthesist 1996;45:626-30
22. Lehmann A, Lang J, Boldt J, Saggau W. Successful off-pump coronary artery bypass graft surgery in a patient with hereditary angioedema. J Cardiothorac Vasc Aresth 2002;16:473-6
23. Bork K, Witzke G. Long-term prophylaxis with C1-inhibitor (C1 INH) concentrate in patients with recurrent angioedema caused by hereditary and acquired C1-inhibitor deficiency. J Allergy Clin Immunol 1989;83:677-82
24. De Serres J, Groner A, Lindner J. Safety and efficacy of pasteurized C1 inhibitor concentrate (Berinert P) in hereditary angioedema: a review. Transfus Apheresis Sci 2003;29:247-54
25. Altman AD, Mclaughlin J, Schellenberg R, et al. Hereditary angioedema managed with low-dose danazol and C1 esterase inhibitor concentrate: a case report. J Obstet Gynaecol Can 2006;28:27-31
26. Waytes AT, Rosen FS, Frank MM. Treatment of hereditary angioedema with a vapor-heated C1 inhibitor concentrate. N Engl J Med. 1996;334:1630-4
27. Kunschak M, Engl W, Maritsch F, et al. A randomized, controlled trial to study the efficacy and safety of C1 inhibitor concentrate in treating hereditary angioedema: Transfusion 1998;38:540-9
28. Cicardi M, Zingale L. How do we treat patients with hereditary angioedema. Transfus Apher Sci 2003;29:221-7
29. Bergamaschini L, Cicardi M. Recent advances in the use of C1 inhibitor as a therapeutic agent. Mol Immunol 2003;40:155-8
30. 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 2007;44:1454-60
31. Burnouf T, Radosevich M. Nanofiltration of plasma-derived biopharmaceutical products. Haemophilia 2003;9:24-37
32. Zuraw BL, Schaefer O, Grant JA, et al. Results of a randomized double-blind placebo controlled study of nanofiltered C1-inhibitor for the treatment of HAE attacks. American College of Allergy, Asthma & Immunology. Dallas, TX, USA; November 2007
33. CSL Behring reaches primary endpoint in clinical study of C1-INH for treatment of hereditary angioedema. CSL Behring press release. 26 Nov 2007. Available from: http://www.cslbehring.com/s1/cs/enco/1151517263302/ news/1195505474707/prdetail.htm
34. 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. Glycobiology 2004;14:51-64
35. Van Doorn MB, Burggraaf J, Van Dam T, et al. A phase I study of recombinant human C1 inhibitor in asymptomatic patients with hereditary angioedema. J Allergy Clin Immunol 2005;116:876-83
36. Choi G, Soeters MR, Farkas H, et al. Recombinant human C1-inhibitor in the treatment of acute angioedema attacks. Transfusion 2007;47:1028-32
37. Schapira M, Silver LD, Scott CF, et al. Prekallikrein activation and high- molecular-weight kininogen consumption in hereditary angioedema. N Engl J Med 1983;308:1050-4
38. Zuraw BL, Curd JG. Demonstration of modified inactive first component of complement (C1) inhibitor in the plasmas of C1 inhibitor-deficient patients. JCI 1986;78:567-75
39. Curd JG, Prograis L Jr, Cochrane CG. Detection of active kallikein in induced blister fluids of hereditary angioedema patients. J Exp Med 1980;152:742-7
40. Curd JG, Yelvington M, Burridge N, et al. Generation of Wadykinin during incubation of hereditary angioedema plasma. Mol Immunol 1983;19:1365-5
41. Fields T, Ghebrehiwet B, Kaplan AP. Kinin formation in hereditary angioedema plasma: evidence against kinin derivation from C2 and in support of "spontaneous" formation of bradykinin. J Allergy Clin Immunol 1983;72:54-60
42. Davis AE III. The pathogenesis of hereditary angioedema. Transfus Apheresis Sci 2003;29:195-203
43. Williams A, Baird LG. DX-88 and HAE: a developmental perspective. Transfus Apheresis Sci 2003;29:255-8
44. Han ED, MacFarlane RC, Mulligan AN, et al. Increased vascular permeability in C1 inhibitor-deficient mice mediated by the bradykinin type 2 receptor. J Clin Invest 2002;109:1057-63
45. Schneider L, Lumry W, Vegh A, et al. Critical role of kallikrein in hereditary angioedema pathogenesis: a clinical trial of ecallantide, a novel kallikrein inhibitor. J Allergy Clin Immunol 2007;120:416-22
46. Lumry W, Ritchie B, Beck T, Morrison J. Interim results of EDEMA2, a multicenter, open-label, repeat-dosing study of intravenous and subcutaneous administration of ecallantide (DX-88) in hereditary angioedema. American Academy of Allergy Asthma and Immunology, San Diego, CA, USA; March 2006
47. Levy R, Mcneil D, Li H, et al. Results of a phase 3 double-blind, placebo-controlled trial, EDEMA3: a study of subcutaneous DX-88 (Ecallantide) in patients with hereditary angioedema. American College of Allergy Asthma and Immunology. Dallas, TX, USA; November 2007
48. Caballero T, Lopez-Serrano C. Anaphylactic reaction and antibodies to DX-88 (kallikrein inhibitor) in a patient with hereditary angioedema. J Allergy Clin Immunol 2006;117:476-7
49. Beck TR, Baird LG. Reply: anaphylactic reaction and antibodies to DX-88 (kallikrein inhibitor) in a patient with hereditary angioedema. J Allergy Clin Immunol 2006;117:477
50. Hock FJ, Wirth K, Albus U, et al. Hoe 140 a new potent and long acting bradykinin-antagonist: in vitro studies. Br J Pharmacol 1991;102:769-73
51. Bork K, Frank J, Grundt B, et al. Treatment of acute edema attacks in hereditary angioedema with a bradykinin receptor-2 antagonist (Icatibant). J Allergy Clin Immunol 2007;119:1497-503
52. Bas M, Bier H,, Greve J, et al. Novel pharmacotherapy of acute hereditary angioedema with bradykinin B2-receptor antagonist icatibant. Allergy 2006;61:1490-2
53. 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 2004;114(3 Suppl.):S51-131
54. Farkas H, Jakab L, Temesszentandrasi G, et al. Hereditary angioedema: a decade of human C1-inhibitor concentrate therapy. J Allergy Clin Immunol 2007;120:941-7
55. Burnouf T. Modern plasma fractionation. Transfus Med Rev 2007;21:101-17
56. Levi M, Choi G, Picavet C, Hack CE. Self-administration of C1-inhibitor concentrate in patients with hereditary or acquired angioedema caused by C1-inhibitor deficiency. J Allergy Clin Immunol 2006;117:904-8
57. Available from: www.jerini.com with permission