Nanofiltered C1 inhibitor concentrate for treatment of hereditary angioedema

New England Journal of Medicine

Volumn 363, Issue 6, 2010, Pages 513-522

  Zuraw, Bruce L ^a,b   Busse, Paula J ^d   White, Martha ^f   Jacobs, Joshua ^c   Lumry, William ^h   Baker, James ^j   Craig, Timothy ^k   Grant, J Andrew ⁱ   Hurewitz, David ^l   Bielory, Leonard ^m   Cartwright, William E ⁿ   Koleilat, Majed ^o   Ryan, Walter ^p   Schaefer, Oren ^q   Manning, Michael ^r   Patel, Pragnesh ^s   Bernstein, Jonathan A ^t   Friedman, Roger A ^u   Wilkinson, Robert ^v   Tanner, David ^w   Kohler, Gary ^y   Gunther, Glenne ^aa   Levy, Robyn ^x   McClellan, James ^ab   Redhead, Joseph ^z   Guss, David ^a   Heyman, Eugene ^g   Blumenstein, Brent A ^ac   Kalfus, Ira ^e   Frank, Michael M ^ad   more..

^a UNIVERSITY OF CALIFORNIA   (United States)

^b VA SAN DIEGO HEALTHCARE SYSTEM   (United States)

^c Allergy and Asthma Clinical Research Inc   (United States)

^d MOUNT SINAI SCHOOL OF MEDICINE   (United States)

^e M2G Consulting   (United States)

^f Institute for Asthma and Allergy   (United States)

^g ERHStats   (United States)

^h Allergy Associates Research   (United States)

ⁱ UNIVERSITY OF TEXAS MEDICAL BRANCH   (United States)

^j Baker Allergy   (United States)

^k PENN STATE COLLEGE OF MEDICINE   (United States)

^l Allergy Clinic of Tulsa   (United States)

^m UNIVERSITY OF MEDICINE AND DENTISTRY OF NEW JERSEY   (United States)

ⁿ Cornerstone Healthcare St Joseph's Healthcare System ^*  (United States)

^o Welborn Clinic   (United States)

^p Allergy and Asthma Center   (United States)

^q UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^r Allergy and Immunology Associates   (United States)

^s Allergy Asthma Specialists   (United States)

^t UNIVERSITY OF CINCINNATI   (United States)

^u Buckeye Allergy and Asthma   (United States)

^v PACIFIC HEALTH RESEARCH INSTITUTE   (United States)

^w Atlanta Allergy and Asthma Clinic   (United States)

^x Family Allergy and Asthma Center   (United States)

^y Lake Charles Memorial Hospital   (United States)

^z Nephrology Associates of Baton Rouge   (United States)

^aa Libby Clinic   (United States)

^ab Grand Traverse Allergy   (United States)

^ac Trial Architecture Consulting   (United States)

^ad DUKE UNIVERSITY   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENT COMPONENT C1S INHIBITOR; PLACEBO;

ACUTE DISEASE; ADULT; ANGIONEUROTIC EDEMA; ARTICLE; CHEMOPROPHYLAXIS; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CROSSOVER PROCEDURE; DIZZINESS; DRUG EFFICACY; DRUG SAFETY; FEMALE; FEVER; GENETIC DISORDER; HUMAN; INJECTION; INJECTION SITE RASH; MAJOR CLINICAL STUDY; MALE; NANOFILTRATION; OPEN STUDY; PRIORITY JOURNAL; PRURITUS; RANDOMIZED CONTROLLED TRIAL; RASH; SWELLING; TREATMENT OUTCOME;

EID: 77955299623     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa0805538     Document Type: Article

References (29)

1. Bork K, Meng G, Staubach P, Hardt J. Hereditary angioedema: new findings concerning symptoms, affected organs, and course. Am J Med 2006;119:267-74. (Pubitemid 43255068)
2. Frank MM, Gelfand JA, Atkinson JP. Hereditary angioedema: the clinical syndrome and its management. Ann Intern Med 1976;84:580-93.
3. Rosen FS, Alper CA, Pensky J, Klemperer MR, Donaldson VH. Genetically determined heterogeneity of the C1 esterase inhibitor in patients with hereditary angioneurotic edema. J Clin Invest 1971; 50:2143-9.
4. Rosen FS, Pensky J, Donaldson V, Charache P. Hereditary angioneurotic edema: two genetic variants. Science 1965; 148:957-8.
5. Bissler JJ, Aulak KS, Donaldson VH, et al. Molecular defects in hereditary angioneurotic edema. Proc Assoc Am Physicians 1997;109:164-73.
6. Davis AE III. C1 inhibitor and hereditary angioneurotic edema. Annu Rev Immunol 1988;6:595-628.
7. Verpy E, Biasotto M, Brai M, Misiano G, Meo T, Tosi M. Exhaustive mutation scanning by fluorescence-assisted mismatch analysis discloses new genotype-phenotype correlations in angioedema. Am J Hum Genet 1996;59:308-19. (Pubitemid 26266346)
8. Zuraw BL, Herschbach J. Detection of C1 inhibitor mutations in patients with hereditary angioedema. J Allergy Clin Immunol 2000;105:541-6. (Pubitemid 30169913)
9. Frank MM, Sergent JS, Kane MA, Alling DW. Epsilon aminocaproic acid therapy of hereditary angioneurotic edema: a double-blind study. N Engl J Med 1972; 286:808-12.
10. Gelfand JA, Sherins RJ, Alling DW, Frank MM. Treatment of hereditary angioedema with danazol: reversal of clinical and biochemical abnormalities. N Engl J Med 1976;295:1444-8.
11. 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. (Pubitemid 27091567)
12. Széplaki G, Varga L, Valentin S, et al. 12. Adverse effects of danazol prophylaxis on the lipid profiles of patients with hereditary angioedema. J Allergy Clin Immunol 2005;115:864-9. (Pubitemid 40460866)
13. 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. (Pubitemid 32200225)
14. Cicardi M, Zingale L. How do we treat patients with hereditary angioedema. Transfus Apher Sci 2003;29:221-7. (Pubitemid 37315586)
15. 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. (Pubitemid 47531846)
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 1980;302: 542-6.
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 1998;38:540-9. (Pubitemid 28317229)
18. 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.
19. 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 2009;124:801-8.
20. Bowen T, Cicardi M, Bork K, et al. He reditary 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 2008;100:Suppl 2: S30-S40.
21. Gompels MM, Lock RJ, Abinun M, et al. C1 inhibitor deficiency: consensus document. Clin Exp Immunol 2005;139: 379-94.
22. Gooley TA, Leisenring W, Crowley J, Storer BE. Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Stat Med 1999;18:695-706. (Pubitemid 29126745)
23. Zeger SL, Liang KY, Albert PS. Models for longitudinal data: a generalized estimating equation approach. Biometrics 1988;44:1049-60. [Erratum, Biometrics 1989;45:347.]
24. Zuraw BL. Hereditary angioedema. N Engl J Med 2008;359:1027-36.
25. De Serres J, Gröner A, Lindner J. Safety and efficacy of pasteurized C1 inhibitor concentrate (Berinert P) in hereditary angioedema: a review. Transfus Apher Sci 2003;29:247-54. (Pubitemid 37321355)
26. Prematta M, Gibbs JG, Pratt EL, Stoughton TR, Craig TJ. Fresh frozen plasma for the treatment of hereditary angioedema. Ann Allergy Asthma Immunol 2007;98:383-8. (Pubitemid 46582131)
27. 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. (Pubitemid 19094619)
28. 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.
29. Burnouf T, Radosevich M. Nanofiltration of plasma-derived biopharmaceutical products. Haemophilia 2003;9:24-37.