Use of ecallantide in pediatric hereditary angioedema

Pediatrics

Volumn 132, Issue 2, 2013, Pages

  MacGinnitie, Andrew J ^a   Davis Lorton, Mark ^b   Stolz, Leslie E ^c   Tachdjian, Raffi ^d  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b WINTHROP UNIVERSITY HOSPITAL   (United States)

^c Dyax Corp   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Angioedema; C1 inhibitor; Ecallantide; Hereditary angioedema; Pediatric; Plasma kallikrein

Indexed keywords

ECALLANTIDE; PLACEBO;

ADOLESCENT; ALLERGIC REACTION; ANGIONEUROTIC EDEMA; ARTICLE; CHILD; CLINICAL ARTICLE; DISEASE SEVERITY; DRUG EFFICACY; DRUG SAFETY; FEMALE; HUMAN; MALE; OUTCOME ASSESSMENT; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SCHOOL CHILD; TREATMENT OUTCOME; UNSPECIFIED SIDE EFFECT;

EID: 84882256379     PISSN: 00314005     EISSN: 10984275     Source Type: Journal    
DOI: 10.1542/peds.2013-0646     Document Type: Article

References (33)

1. Zuraw BL. Clinical practice. Hereditary angioedema. N Engl J Med. 2008;359(10): 1027-1036
2. Bork K, Hardt J, Witzke G. Fatal laryngeal attacks and mortality in hereditary angioedema due to C1-INH deficiency. J Allergy Clin Immunol. 2012;130(3):692-697
3. Wilson DA, Bork K, Shea EP, Rentz AM, Blaustein MB, Pullman WE. Economic costs associated with acute attacks and longterm management of hereditary angioedema. Ann Allergy Asthma Immunol. 2010; 104(4):314-320
4. Lumry WR, Castaldo AJ, Vernon MK, Blaustein MB, Wilson DA, Horn PT. The humanistic burden of hereditary angioedema: Impact on health-related quality of life, productivity, and depression. Allergy Asthma Proc. 2010;31(5): 407-414
5. Farkas H. Pediatric hereditary angioedema due to C1-inhibitor deficiency. Allergy Asthma Clin Immunol. 2010;6(1):18-27
6. Hsu D, Shaker M. An update on hereditary angioedema. Curr Opin Pediatr. 2012;24(5): 638-646
7. Farkas H, Csuka D, Zotter Z, et al. Treatment of attacks with plasma-derived C1-inhibitor concentrate in pediatric hereditary angioedema patients. J Allergy Clin Immunol. 2013;131 (3):909-911
8. Schneider L, Hurewitz D, Wasserman R, et al. C1-INH concentrate for treatment of acute hereditary angioedema: a pediatric cohort from the I.M.P.A.C.T. studies. Pediatr Allergy Immunol. 2013;24(1):54-60
9. Lumry W, Manning ME, Hurewitz DS, et al. Nanofiltered C1-Esterase Inhibitor for the Acute Management and Prevention of Hereditary Angioedema Attacks due to C1-Inhibitor Deficiency in Children. J Pediatr. 2013;162(5):1017-, e2
10. Cicardi M, Levy RJ, McNeil DL, et al. Ecallantide for the treatment of acute attacks in hereditary angioedema. N Engl J Med. 2010;363(6):523-531
11. Levy RJ, Lumry WR, McNeil DL, et al. EDEMA4: a phase 3, double-blind study of subcutaneous ecallantide treatment for acute attacks of hereditary angioedema. Ann Allergy Asthma Immunol. 2010;104(6): 523-529
12. Lumry WR, Bernstein JA, Li HH, et al. Efficacy and safety of ecallantide in treatment of recurrent attacks of hereditary angioedema: open-label continuation study. Allergy Asthma Proc. 2013;34(2):155-161
13. Vernon MK, Rentz AM, Wyrwich KW, White MV, Grienenberger A. Psychometric validation of two patient-reported outcome measures to assess symptom severity and changes in symptoms in hereditary angioedema. Qual Life Res. 2009;18(7):929-939
14. Centers for Disease Control and Prevention, National Center for Health Statistics. CDC growth charts: United States. Available at: www.cdc.gov/ growthcharts/. Accessed May 30, 2000
15. Sheffer AL, Campion M, Levy RJ, Li HH, Horn PT, Pullman WE. Ecallantide (DX-88) for acute hereditary angioedema attacks: integrated analysis of 2 double-blind, phase 3 studies. J Allergy Clin Immunol. 2011;128 (1):153-159, e4
16. Riedl M, Campion M, Horn PT, Pullman WE. Response time for ecallantide treatment of acute hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2010;105(6):430-436, e2
17. Bernstein JA, Qazi M. Ecallantide: its pharmacology, pharmacokinetics, clinical efficacy and tolerability. Expert Rev Clin Immunol. 2010;6(1):29-39
18. KALBITOR [package insert]. Cambridge, MA: Dyax Corp; December 2009. Available at: www.kalbitor.com/pdf/KalbitorFullPrescribingInformation. pdf. Accessed June 2, 2013
19. Frank MM. 8. Hereditary angioedema. J Allergy Clin Immunol. 2008;121(suppl 2): S398-S401, quiz S419
20. Longhurst H, Cicardi M. Hereditary angiooedema. Lancet. 2012;379(9814):474-481
21. Donaldson VH, Evans RR. A biochemical abnormality in herediatry angioneurotic edema: absence of serum inhibitor of C' 1-esterase. Am J Med. 1963;35:37-44
22. Zuraw BL, Bork K, Binkley KE, et al. Hereditary angioedema with normal C1 inhibitor function: consensus of an international expert panel. Allergy Asthma Proc. 2012;33(suppl 1): S145-S156
23. Bork K, Wulff K, Meinke P, Wagner N, Hardt J, Witzke G. A novel mutation in the coagulation factor 12 gene in subjects with hereditary angioedema and normal C1-inhibitor. Clin Immunol. 2011;141(1):31-35
24. Davis AE III. C1 inhibitor and hereditary angioneurotic edema. Annu Rev Immunol. 1988;6:595-628
25. Davis AE III. The pathophysiology of hereditary angioedema. Clin Immunol. 2005;114 (1):3-9
26. Landerman NS, Webster ME, Becker EL, Ratcliffe HE. Hereditary angioneurotic edema. II. Deficiency of inhibitor for serum globulin permeability factor and/or plasma kallikrein. J Allergy. 1962;33:330-341
27. Bygum A. Hereditary angio-oedema in Denmark: a nationwide survey. Br J Dermatol. 2009;161(5):1153-1158
28. Bork K, Meng G, Staubach P, Hardt J. Hereditary angioedema: new findings concerning symptoms, affected organs, and course. Am J Med. 2006;119(3):267-274
29. Nielsen EW, Johansen HT, Holt J, Mollnes TE. C1 inhibitor and diagnosis of hereditary angioedema in newborns. Pediatr Res. 1994;35(2):184-187
30. Jacobs J. Recurrent attacks of hereditary angioedema: a case of delayed diagnosis. Allergy Asthma Proc. 2011;32(suppl 1):S23-S26
31. Gompels MM, Lock RJ, Abinun M, et al. C1 inhibitor deficiency: consensus document. Clin Exp Immunol. 2005;139(3):379-394
32. Cicardi M, Bork K, Caballero T, et al; HAWK(Hereditary Angioedema International Working Group). Evidence-based recommendations for the therapeutic management of angioedema owing to hereditary C1 inhibitor deficiency: consensus report of an International Working Group. Allergy. 2012;67(2):147-157
33. Lang DM, Aberer W, Bernstein JA, et al. International consensus on hereditary and acquired angioedema. Ann Allergy Asthma Immunol. 2012;109(6):395-402