Summary of the International Multicenter Prospective Angioedema C1-inhibitor Trials 1 and 2 (IMPACT1 and 2)

Expert Review of Clinical Immunology

Volumn 6, Issue 3, 2010, Pages 327-334

  Krassilnikova, Svetlana ^a   Craig, Ethan T ^b   Craig, Timothy J ^b  

^a PENN STATE COLLEGE OF MEDICINE   (United States)

^b PENNSYLVANIA STATE UNIVERSITY   (United States)

Author keywords

Berinert P; C1 inhibitor; C1 inhibitor deficiency; Hereditary angioedema; IMPACT1; IMPACT2; Pasteurized C1 inhibitor concentrate

Indexed keywords

ANALGESIC AGENT; ANTIEMETIC AGENT; BERINERT P; COMPLEMENT COMPONENT C1S INHIBITOR; FRESH FROZEN PLASMA; PLACEBO; UNCLASSIFIED DRUG;

ANGIONEUROTIC EDEMA; CLINICAL TRIAL; CONNECTIVE TISSUE DISEASE; DIZZINESS; DRUG EFFICACY; DRUG INDUCED HEADACHE; DRUG SAFETY; DRUG TREATMENT FAILURE; FLU LIKE SYNDROME; GASTROINTESTINAL SYMPTOM; HUMAN; INJECTION SITE REACTION; MUSCULOSKELETAL DISEASE; NEUROTOXICITY; REVIEW; RHINOPHARYNGITIS; SYMPTOM; UNITED STATES; UPPER RESPIRATORY TRACT INFECTION; VULVOVAGINITIS; XEROSTOMIA;

ADULT; ANGIOEDEMAS, HEREDITARY; CANADA; CLINICAL TRIALS, PHASE I AS TOPIC; CLINICAL TRIALS, PHASE II AS TOPIC; COMPLEMENT C1 INHIBITOR PROTEIN; EUROPE; FEMALE; HUMANS; MALE; MULTICENTER STUDIES AS TOPIC; RANDOMIZED CONTROLLED TRIALS AS TOPIC; UNITED STATES;

EID: 77952015991     PISSN: 1744666X     EISSN: None     Source Type: Journal    
DOI: 10.1586/ECI.10.19     Document Type: Review

References (36)

1. 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). • Report of C1-inhibitor (C1-INH) deficiency in hereditary angioedema (HAE).
2. Pappalardo E, Cicardi M, Duponchel C et al. Frequent de novo mutations and exon deletions in the C1 inhibitor gene of patients with angioedema. J. Allergy Clin. Immunol. 106 (6), 1147-1154 (2000).
3. Tosi M. Molecular genetics of C1 inhibitor. Immunobiology 199 (2), 358-365 (1998).
4. Rosen FS, Pensky J, Donaldson V et al. Hereditary angioneurotic edema: two genetic variants. Science 148, 957-958 (1965).
5. Bork K, Horstkorte R, Weidemann W. Increasing the sialylation of therapeutic glycoproteins: the potential of the sialic acid biosynthetic pathway. J. Pharma. Sci. 98 (10), 3499-3508 (2009).
6. 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).
7. Frank MM. Hereditary angioedema. J. Allergy Clin. Immunol. 121 (2 Suppl.), S398-S401 (2008).
8. Frank MM, Sergent JS, Kane MA et al. Epsilon aminocaproic acid therapy of hereditary angioneurotic edema. A double-blind study N. Engl. J. Med. 286 (15), 808-812 (1972).
9. Ritchie BC. Protease inhibitors in the treatment of hereditary angioedema. Transfus. Apher. Sci. 29 (3), 259-267 (2003).
10. Banerji A, Sloane DE, Sheffer AL. Hereditary angioedema: a current state-of-the-art review, V: attenuated androgens for the treatment of hereditary angioedema. Ann. Allergy Asthma Immunol. 100 (1 Suppl. 2), S19-S22 (2008).
11. Farkas H, Gyeney L, Gidofalvy E et al. 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).
12. Bork K, Staubach P, Eckardt AJ et al. Symptoms, course, and complications of abdominal attacks in hereditary angioedema due to C1 inhibitor deficiency Am. J. Gastroenterol. 101 (3), 619-627 (2006).
13. Gompels MM, Lock RJ, Abinun M et al. C1 inhibitor deficiency: consensus document. Clin. Exp. Immunol. 141 (1), 189-190 (2005). •• Consensus report on C1-INH as a first-line therapy for acute HAE attacks and short-term prophylaxis.
14. Bowen T, Cicardi M, Bork K et al. Hereditary angiodema: a current state-ofthe-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). •• Consensus report on C1-INH as a first-line therapy for acute HAE attacks and short-term prophylaxis.
15. Bernstein JA. 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 (2008).
16. 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. 302 (10), 542-546 (1980). • The activity of 1 unit of C1-INH in concentrate was defined as activity of C1-INH in 1 ml of plasma.
17. Bork K, Meng G, Staubach P et al. Treatment with C1 inhibitor concentrate in abdominal pain attacks of patients with hereditary angioedema. Transfusion 45 (11), 1774-1784 (2005).
18. Craig TJ, Levy RJ, Wasserman RL et al. Efficacy of human C1 esterase inhibitor concentrate compared with placebo in acute hereditary angioedema attacks. Allergy Clin. Immunol. 124 (4), 801-808 (2009). •• Results of the International Multicenter Prospective Angioedema C1-inhibitor Trial (IMPACT) 1 trial.
19. Craig TJ, Wassermann RL, Levy R et al. C1-esterase inhibitor (C1-INH) - standard of care for the treatment of acute attacks in hereditary angioedema (HAE) : initial results of an ongoing, prospective, open-label study in North America (I. M. P. A. C. T 2). J. Allergy Clin. Immunol. 121, S98-S99 (2008). •• Interim analysis of the IMPACT2 trial presented to the public for the first time.
20. Waytes AT, Rosen FS, Frank MM. Treatment of hereditary angioedema with a vapor-heated C1 inhibitor concentrate. N. Engl. J. Med. 334 (25), 1630-1634 (1996). •• Pivotal trial of C1-INH effcacy over placebo.
21. 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 (6), 540-549 (1998). •• Pivotal trial of C1-INH efficacy over placebo.
22. Bork K, Hardt J, Schicketanz KH et al. Clinical studies of sudden upper airway obstruction in patients with hereditary angioedema due to C1 esterase inhibitor defciency. Arch. Intern. Med. 163 (10), 1229-1235 (2003).
23. Bork K, Staubach P, Hardt J. Treatment of skin swellings with C1-inhibitor concentrate in patients with hereditary angio-oedema. Allergy 63 (6), 751-757 (2008).
24. Farkas H, Harmat G, Fust G et al. Clinical management of hereditary angio-oedema in children. Pediatr. Allergy Immunol. 13 (3), 153-161 (2002).
25. Leimgruber A, Jaques WA, Spaeth PJ. Hereditary angioedema: uncomplicated maxillofacial surgery using short-term C1 inhibitor replacement therapy. Int. Arch. Allergy Immunol. 101 (1), 107-112 (1993).
26. 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 (4), 473-476 (2002).
27. 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 (7), 626-630 (1996).
28. 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).
29. Longhurst HJ, Carr S, Khair K. C1-inhibitor concentrate home therapy for hereditary angioedema: a viable, effective treatment option. Clin. Exp. Immunol. 147 (1), 11-17 (2007).
30. Fabiani JE, Avigliano A, Dupont JC. Hereditary angioedema. Long-term follow-up of 88 patients. Experience of the Argentine Allergy and Immunology Institute. Allergol. Immunopathol. (Madr.) 28 (5), 267-271 (2000).
31. Terpstra FG, Kleijn M, Koenderman AH et al. Viral safety of C1-inhibitor NF. Biologicals 35 (3), 173-181 (2007).
32. Martinez-Saguer I. Pharmacokinetic analysis of C1-inhibitor concentrate in 40 patients with hereditary angio-oedema (HAE) - prospective study. Haemophilia 8, 571-578 (2002).
33. Choi G, Soeters MR, Farkas H et al. Recombinant human C1-inhibitor in the treatment of acute angioedema attacks. Transfusion 47 (6), 1028-1032 (2007).
34. 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. 120 (2), 416-422 (2007).
35. Williams A, Baird LG. DX-88 and HAE: a developmental perspective. Transfus. Apher. Sci. 29 (3), 255-258 (2003).
36. Wagner BR, Knolle J. Absolute bioavailability of subcutaneously administered icatibant, a selective and potent bradykinin B2 receptor antagonist. J. Allergy Clin. Immunol. 119 (1), S274 (2007).