Novel Therapies for Hereditary Angioedema

Immunology and Allergy Clinics of North America

Volumn 26, Issue 4, 2006, Pages 691-708

  Zuraw, Bruce L ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANDROGEN; ANTIFIBRINOLYTIC AGENT; APROTININ; BEHRINERT P; BRADYKININ B2 RECEPTOR; BRADYKININ B2 RECEPTOR ANTAGONIST; COMPLEMENT COMPONENT C1S INHIBITOR; DX 88; ICATIBANT; KALLIKREIN INHIBITOR; PLACEBO; PLASMA DERIVED COMPLEMENT COMPONENT C1S INHIBITOR; PLASMA KALLIKREIN; PLASMIN; RECOMBINANT HUMAN COMPLEMENT COMPONENT C1S INHIBITOR PROTEIN; RECOMBINANT PROTEIN; TISSUE FACTOR PATHWAY INHIBITOR; TRYPSIN; UNCLASSIFIED DRUG;

ALLERGIC REACTION; ANAPHYLAXIS; ANGIONEUROTIC EDEMA; ANTIINFLAMMATORY ACTIVITY; BLOOD VESSEL PERMEABILITY; CLINICAL TRIAL; DISEASE CLASSIFICATION; DISEASE SEVERITY; DRUG CLEARANCE; DRUG CONTRAINDICATION; DRUG EFFECT; DRUG EFFICACY; DRUG INHIBITION; DRUG POTENCY; DRUG SAFETY; DRUG SELF ADMINISTRATION; DRUG TREATMENT FAILURE; HUMAN; IMMUNOTOXICITY; INFECTIOUS COMPLICATION; INJECTION SITE DISCOMFORT; INJECTION SITE ERYTHEMA; LONG TERM CARE; NONHUMAN; PREGNANCY; PRIORITY JOURNAL; PROPHYLAXIS; RETROSPECTIVE STUDY; REVIEW; SHORT COURSE THERAPY; SUBSTITUTION THERAPY; TRANSGENE; TREATMENT PLANNING; VIRUS INFECTION;

ANGIONEUROTIC EDEMA; ANIMALS; BRADYKININ; CLINICAL TRIALS; COMPLEMENT C1 INHIBITOR PROTEIN; HUMANS; KALLIKREINS; RECOMBINANT PROTEINS;

EID: 33750494753     PISSN: 08898561     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.iac.2006.09.007     Document Type: Review

References (64)

1. Nilsson I.M., Andersson L., and Bjorkman S.E. Epsilon-aminocaproic acid (E-ACA) as a therapeutic agent based on 5 years' clinical experience. Acta Med Scand Suppl 448 (1966) 1-46
2. Spaulding W.B. Methyltestosterone therapy for hereditary episodic edema (hereditary angioneurotic edema). Ann Intern Med 53 (1960) 739-745
3. Donaldson V.H., and Evans R.R. A biochemical abnormality in hereditary angioneurotic edema: absence of serum inhibitor of C'1- esterase. Am J Med 35 (1963) 37-44
4. Landerman N.S., Webster M.E., Becker E.L., et al. Hereditary angioneurotic edema. II. Deficiency of inhibitor for serum globulin permeability factor and/or plasma kallikrein. J Allergy 33 (1962) 330-341
5. Spath P.J., Wuthrich B., and Butler R. Quantification of C1-inhibitor functional activities by immunodiffusion assay in plasma of patients with hereditary angioedema-evidence of a functionally critical level of C1-inhibitor concentration. Complement 1 (1984) 147-159
6. Sheffer A.L., Fearon D.T., and Austen K.F. Hereditary angioedema: a decade of management with stanozolol. J Allergy Clin Immunol 80 (1987) 855-860
7. Pickering R.J., Good R.A., Kelly J.R., et al. Replacement therapy in hereditary angioedema. Successful treatment of two patients with fresh frozen plasma. Lancet 1 (1969) 326-330
8. Agostoni A., Bergamaschini L., Martignoni G., et al. Treatment of acute attacks of hereditary angioedema with C1-Inhibitor concentrate. Ann Allergy 44 (1980) 299-301
9. Gadek J.E., Hosea S.W., Gelfand J.A., et al. Replacement therapy in hereditary angioedema. Successful treatment of acute episodes of angioedema with partly purified C1 inhibitor. N Engl J Med 302 (1980) 542-546
10. Marasini B., Cicardi M., Martignoni G.C., et al. Treatment of hereditary angioedema. Klin Wochenschr 56 (1978) 819-823
11. Bergamaschini L., Cicardi M., Tucci A., et al. C1 INH concentrate in the therapy of hereditary angioedema. Allergy 38 (1983) 81-84
12. Bork K., and Barnstedt S.E. Treatment of 193 episodes of laryngeal edema with C1 inhibitor concentrate in patients with hereditary angioedema. Arch Intern Med 161 (2001) 714-718
13. Kirschfink M., and Mollnes T.E. C1-inhibitor: an anti-inflammatory reagent with therapeutic potential. Expert Opin Pharmacother 2 (2001) 1073-1083
14. Logan R.A., and Greaves M.W. Hereditary angio-oedema: treatment with C1 esterase inhibitor concentrate. J R Soc Med 77 (1984) 1046-1048
15. Agostoni A., and Cicardi M. Hereditary and acquired C1-inhibitor deficiency: biological and clinical characteristics in 235 patients. Medicine (Baltimore) 71 (1992) 206-215
16. Bork K., Hardt J., Schicketanz K.H., et al. Clinical studies of sudden upper airway obstruction in patients with hereditary angioedema due to C1 esterase inhibitor deficiency. Arch Intern Med 163 (2003) 1229-1235
17. 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 38 (1998) 540-549
18. Cicardi M., and Zingale L. How do we treat patients with hereditary angioedema?. Transfus Apher Sci 29 (2003) 221-227
19. Bork K., Meng G., Staubach P., et al. Hereditary angioedema: new findings concerning symptoms, affected organs, and course. Am J Med 119 (2006) 267-274
20. Waytes A.T., Rosen F.S., and Frank M.M. Treatment of hereditary angioedema with a vapor-heated C1 inhibitor concentrate. N Engl J Med 334 (1996) 1630-1634
21. De Serres J., Groner A., and Lindner J. Safety and efficacy of pasteurized C1 inhibitor concentrate (Berinert P) in hereditary angioedema: a. review.jean.de.serres@aventis.com. Transfus Apheresis Sci 29 (2003) 247-254
22. US HAE Association. C1-inhibitor (C1INH) personal importation. Available at: http://www.hereditaryangioedema.com/import.html. Accessed October 20, 2006.
23. Bork K., and 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 83 (1989) 677-682
24. Altman A.D., 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 28 (2006) 27-31
25. Lehmann A., Lang J., Boldt J., et al. Successful off-pump coronary artery bypass graft surgery in a patient with hereditary angioedema. J Cardiothorac Vasc Anesth 16 (2002) 473-476
26. Leimgruber A., Jaques W.A., and Spaeth P.J. Hereditary angioedema: uncomplicated maxillofacial surgery using short-term C1 inhibitor replacement therapy. Int Arch Allergy Immunol 101 (1993) 107-112
27. Maves K.K., and Weiler J.M. Tonsillectomy in a patient with hereditary angioedema after prophylaxis with C1 inhibitor concentrate. Ann Allergy 73 (1994) 435-438
28. Mohr M., Pollok-Kopp B., Gotze O., et al. [The use of a C1-inhibior concentrate for short-term preoperative prophylaxis in two patients with hereditary angioedema.]. Anaesthesist 45 (1996) 626-630 [in German]
29. Alsenz J., Lambris J.D., Bork K., et al. Acquired C1 inhibitor (C1-INH) deficiency type II. Replacement therapy with C1-INH and analysis of patients' C1-INH and anti-C1-INH autoantibodies. JCI 83 (1989) 1794-1799
30. Bork K., Barnstedt S.E., Koch P., et al. Hereditary angioedema with normal C1-inhibitor activity in women. Lancet 356 (2000) 213-217
31. Levi M., Choi G., Picavet C., et al. Self-administration of C1-inhibitor concentrate in patients with hereditary or acquired angioedema caused by C1-inhibitor deficiency. J Allergy Clin Immunol 117 (2006) 904-908
32. Bergamaschini L., and Cicardi M. Recent advances in the use of C1 inhibitor as a therapeutic agent. Mol Immunol 40 (2003) 155-158
33. 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 (2007) 1454-1460
34. Burnouf T., and Radosevich M. Nanofiltration of plasma-derived biopharmaceutical products. Haemophilia 9 (2003) 24-37
35. Koles K., van Berkel P.H., Pieper F.R., et al. N- and O-glycans of recombinant human C1 inhibitor expressed in the milk of transgenic rabbits. Glycobiology 14 (2004) 51-64
36. van Doorn M.B., 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 116 (2005) 876-883
37. Porebski G., Bilo B., Obtulowicz K., et al. [Recombinant human C1-inhibitor is effective in the treatment of acute attacks of hereditary angioedema-case report]. Przegl Lek 62 (2005) 317-320 [in Polish]
38. Caliezi C., Wuillemin W.A., Zeerleder S., et al. C1-Esterase inhibitor: an anti-inflammatory agent and its potential use in the treatment of diseases other than hereditary angioedema. Pharmacol Rev 52 (2000) 91-112
39. Cai S., and Davis III A.E. Complement regulatory protein C1 inhibitor binds to selectins and interferes with endothelial-leukocyte adhesion. J Immunol 171 (2003) 4786-4791
40. Cicardi M., Zingale L., Zanichelli A., et al. C1 inhibitor: molecular and clinical aspects. Springer Semin Immunopathol 27 (2005) 286-298
41. Horstick G. C1-esterase inhibitor in ischemia and reperfusion. Immunobiology 205 (2002) 552-562
42. Kirschfink M. C1-inhibitor and transplantation. Immunobiology 205 (2002) 534-541
43. Liu D., Cai S., Gu X., et al. C1 inhibitor prevents endotoxin shock via a direct interaction with lipopolysaccharide. J Immunol 171 (2003) 2594-2601
44. Storini C., Rossi E., Marrella V., et al. C1-inhibitor protects against brain ischemia-reperfusion injury via inhibition of cell recruitment and inflammation. Neurobiol Dis 19 (2005) 10-17
45. Schapira M., Silver L.D., Scott C.F., et al. Prekallikrein activation and high-molecular-weight kininogen consumption in hereditary angioedema. N Engl J Med 308 (1983) 1050-1054
46. Zuraw B.L., and Curd J.G. Demonstration of modified inactive first component of complement (C1) inhibitor in the plasmas of C1 inhibitor-deficient patients. JCI 78 (1986) 567-575
47. Donaldson V.H., Ratnoff O.D., Da Silva W.D., et al. Permeability-increasing activity in hereditary angioneurotic edema plasma. II. Mechanism of formation and partial characterization. JCI 48 (1969) 642-653
48. Donaldson V.H., Rosen F.S., and Bing D.H. Role of the second component of complement (C2) and plasmin in kinin release in hereditary angioneurotic edema (H.A.N.E.) plasma. Trans Assoc Am Physicians 40 (1977) 174-183
49. Curd J.G., Prograis Jr. L.J., and Cochrane C.G. Detection of active kallikein in induced blister fluids of hereditary angioedema patients. J Exp Med 152 (1980) 742-747
50. Curd J.G., Yelvington M., Burridge N., et al. Generation of bradykinin during incubation of hereditary angioedema plasma. Mol Immunol 19 (1983) 1365
51. Fields T., Ghebrehiwet B., and Kaplan A.P. Kinin formation in hereditary angioedema plasma: evidence against kinin derivation from C2 and in support of "spontaneous" formation of bradykinin. J Allergy Clin Immunol 72 (1983) 54-60
52. Juhlin L., and Michaelsson G. Use of a kallikrein inhibitor in the treatment of urticaria and hereditary angioneurotic edema. Acta Derm Venereol 49 (1969) 37-44
53. Bauer J., Futterman S., and Dreiling D.A. Anaphylactic shock secondary to initial Trasylol administration. Am J Gastroenterol 56 (1971) 542-544
54. Proud G., and Chamberlain J. Anaphylactic reaction to aprotinin. [letter]. Lancet 2 (1976) 48-49
55. Williams A., and Baird L.G. DX-88 and HAE: a developmental perspective. Transfus Apheresis Sci 29 (2003) 255-258
56. Han E.D., MacFarlane R.C., Mulligan A.N., et al. Increased vascular permeability in C1 inhibitor-deficient mice mediated by the bradykinin type 2 receptor. J Clin Invest 109 (2002) 1057-1063
57. Storini C., Bergamaschini L., Gesuete R., et al. Selective inhibition of plasma kallikrein protects brain from reperfusion injury. J Pharmacol Exp Ther 318 (2006) 849-854
58. Caballero T., and Lopez-Serrano C. Anaphylactic reaction and antibodies to DX-88 (kallikrein inhibitor) in a patient with hereditary angioedema. J Allergy Clin Immunol 117 (2006) 476-477
59. Beck T.R., and Baird L.G. Reply: anaphylactic reaction and antibodies to DX-88 (kallikrein inhibitor) in a patient with hereditary angioedema. J Allergy Clin Immunol 117 (2006) 477
60. Stewart J.M., Gera L., Hanson W., et al. A new generation of bradykinin antagonists. Immunopharmacology 33 (1996) 51-60
61. Stewart J.M., and Vavrek R.J. Kinin antagonists: design and activities. J Cardiovasc Pharmacol 15 (1990) S69-S74
62. Hock F.J., Wirth K., Albus U., et al. Hoe 140 a new potent and long acting bradykinin-antagonist: in vitro studies. Br J Pharmacol 102 (1991) 769-773
63. Akbary A.M., Wirth K.J., and Schölkens B.A. Efficacy and tolerability of Icatibant (Hoe 140) in patients with moderately severe chronic bronchial asthma. Immunopharmacology 33 (1996) 238-242
64. Moreau M.E., Garbacki N., Molinaro G., et al. The kallikrein-kinin system: current and future pharmacological targets. J Pharmacol Sci 99 (2005) 6-38