Efficacy assessments in randomized controlled studies of acute therapy for hereditary angioedema

Journal of Clinical Immunology

Volumn 32, Issue 6, 2012, Pages 1204-1212

  Caballero, Teresa ^a  

^a HOSPITAL UNIVERSITARIO LA PAZ IDIPAZ   (Spain)

Author keywords

C1 esterase inhibitor; Hereditary angioedema; patient reported outcome; symptom severity

Indexed keywords

COMPLEMENT COMPONENT C1S INHIBITOR; ECALLANTIDE; ICATIBANT; PLACEBO; KALLIKREIN; PEPTIDE;

ANGIONEUROTIC EDEMA; ARTICLE; CLINICAL ASSESSMENT TOOL; DISEASE SEVERITY; DRUG EFFICACY; HUMAN; OUTCOME ASSESSMENT; PATIENT ASSESSMENT; PRIORITY JOURNAL; PROSPECTIVE STUDY; RANDOMIZED CONTROLLED TRIAL (TOPIC); RETROSPECTIVE STUDY; TREATMENT RESPONSE; VALIDATION PROCESS; VISUAL ANALOG SCALE; DRUG ANTAGONISM; METABOLISM; METHODOLOGY; PAIN ASSESSMENT; PATHOPHYSIOLOGY; PSYCHOLOGICAL ASPECT; REVIEW; SEVERITY OF ILLNESS INDEX; TIME; TREATMENT OUTCOME;

COMPLEMENT C1 INHIBITOR PROTEIN; HEREDITARY ANGIOEDEMA TYPES I AND II; HUMANS; KALLIKREINS; OUTCOME ASSESSMENT (HEALTH CARE); PAIN MEASUREMENT; PEPTIDES; PROSPECTIVE STUDIES; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; RETROSPECTIVE STUDIES; SEVERITY OF ILLNESS INDEX; TIME FACTORS; TREATMENT OUTCOME;

EID: 84878248199     PISSN: 02719142     EISSN: 15732592     Source Type: Journal    
DOI: 10.1007/s10875-012-9734-8     Document Type: Article

References (28)

1. Gompels MM, Lock RJ, Abinun M, et al. C1 inhibitor deficiency: consensus document. Clin Exp Immunol. 2005;139(3):379-94.
2. Zuraw BL. Clinical practice. Hereditary angioedema. N Engl J Med. 2008;359(10):1027-36.
3. Caballero T, Baeza ML, Cabañas R, et al. Consensus statement on the diagnosis, management, and treatment of angioedema mediated by bradykinin. Part I. Classification, epidemiology, pathophysiology, genetics, clinical symptoms, and diagnosis. J Investig Allergol Clin Immunol. 2011;21(5):333-47.
4. Frank MM. Hereditary angioedema: a current state-of-the-art review, VI: novel therapies for hereditary angioedema. Ann Allergy Asthma Immunol. 2008;100(1 Suppl 2):S23-9.
5. Caballero T, Baeza ML, Cabañas R, et al. Consensus statement on the diagnosis, management, and treatment of angioedema mediated by bradykinin. Part II. Treatment, follow-up, and special situations. J Investig Allergol Clin Immunol. 2011;21(6):422-41.
6. 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(4):801-8.
7. Zuraw BL, Busse PJ, White M, et al. Nanofiltered C1 inhibitor concentrate for treatment of hereditary angioedema. N Engl J Med. 2010;363(6):513-22.
8. Zuraw B, Cicardi M, Levy RJ, et al. Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010;126(4):821-7. e14.
9. Cicardi M, Banerji A, Bracho F, et al. Icatibant, a new bradykinin-receptor antagonist, in hereditary angioedema. N Engl J Med. 2010;363(6):532-41.
10. Lumry WR, Li HH, Levy RJ, et al. Randomized placebo-controlled trial of the bradykinin B(2) receptor antagonist icatibant for the treatment of acute attacks of hereditary angioedema: the FAST-3 trial. Ann Allergy Asthma Immunol. 2011;107(6):529-37.
11. 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):523-31.
12. 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):523-9.
13. Bork K, Meng G, Staubach P, Hardt J. Hereditary angioedema: new findings concerning symptoms, affected organs, and course. Am J Med. 2006;119(3):267-74.
14. U. S. Department of Health and Human Services, Food and Drug Administration. Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), Center for Devices and Radiological Health (CDRH). Guidance for Industry. Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims; 2009.
15. European Medicines Agency. Committee for Medicinal Products for Human Use (CHMP). Reflection Paper on the Regulatory Guidance for the Use of Health-Related Quality of Life (HRQL) Measures in the Evaluation of Medicinal Products. London: European Medicines Agency; 2005.
16. Herschorn S, Swift S, Guan Z, et al. Comparison of fesoterodine and tolterodine extended release for the treatment of overactive bladder: a head-to-head placebo-controlled trial. BJU Int. 2010;105(1):58-66.
17. Mathew NT, Jaffri SF. A double-blind comparison of onabotulinumtoxina (BOTOX) and topiramate (TOPAMAX) for the prophylactic treatment of chronic migraine: a pilot study. Headache. 2009;49(10):1466-78.
18. Pincus T, Chung C, Segurado OG, Amara I, Koch GG. An index of patient reported outcomes (PRO-Index) discriminates effectively between active and control treatment in 4 clinical trials of adalimumab in rheumatoid arthritis. J Rheumatol. 2006;33(11):2146-52.
19. Propert KJ, Alexander RB, Nickel JC, Chronic Prostatitis Collaborative Research Network, et al. Design of a multicenter randomized clinical trial for chronic prostatitis/chronic pelvic pain syndrome. Urology. 2002;59(6):870-6.
20. Rothman M, Vallow S, Damaraju CV, Hewitt DJ. Using the patient global assessment of the method of pain control to assess new analgesic modalities in clinical trials. Curr Med Res Opin. 2009;25(6):1433-43.
21. Shikiar R, Bresnahan BW, Stone SP, Thompson C, Koo J, Revicki DA. Validity and reliability of patient reported outcomes used in psoriasis: results from two randomized clinical trials. Health Qual Life Outcomes. 2003;1:53.
22. Shikiar R, Willian MK, Okun MM, Thompson CS, Revicki DA. The validity and responsiveness of three quality of life measures in the assessment of psoriasis patients: results of a phase II study. Health Qual Life Outcomes. 2006;4:71.
23. Toglia MR, Serels SR, Laramée C, et al. Solifenacin for overactive bladder: patient-reported outcomes from a large placebo-controlled trial. Postgrad Med. 2009;121(5):151-8.
24. 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-S131.
25. 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):929-39.
26. Bernstein JA, Ritchie B, Levy RJ, et al. Hereditary angioedema: Validation of the end point time to onset of relief by correlation with symptom intensity. Allergy Asthma Proc. 2011;32(1):36-42.
27. 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):430-6.
28. Roche O, Blanch A, Caballero T, Sastre N, Callejo D, Lopez-Trascasa M. Hereditary angioedema due to C1 inhibitor deficiency: patient registry and approach to the prevalence in Spain. Ann Allergy Asthma Immunol. 2005;94(4):498-503.