Reassessing the evidence hierarchy in asthma: Evaluating comparative effectiveness

Current Allergy and Asthma Reports

Volumn 11, Issue 6, 2011, Pages 526-538

  Price, David ^a,b   Chisholm, Alison ^b   Van Der Molen, Thys ^a,c   Roche, Nicolas ^d   Hillyer, Elizabeth V ^b   Bousquet, Jean ^e,f  

^a UNIVERSITY OF ABERDEEN   (United Kingdom)

^b Research in Real Life Ltd   (United Kingdom)

^c UNIVERSITY MEDICAL CENTER GRONINGEN   (Netherlands)

^d HÔTEL DIEU   (France)

^e HÔPITAL ARNAUD DE VILLENEUVE   (France)

^f INSERM   (France)

Author keywords

Asthma; Guidelines; Observational studies; Pragmatic trials; Randomized controlled trials; Real world

Indexed keywords

BECLOMETASONE DIPROPIONATE; BETA 2 ADRENERGIC RECEPTOR STIMULATING AGENT; BRONCHODILATING AGENT; CORTICOSTEROID; FLUTICASONE PROPIONATE; LEUKOTRIENE RECEPTOR BLOCKING AGENT; MOMETASONE FUROATE; PLACEBO; TIOTROPIUM BROMIDE;

ALLERGIC RHINITIS; ARTICLE; ASTHMA; CHRONIC OBSTRUCTIVE LUNG DISEASE; CLINICAL EVALUATION; CLINICAL PRACTICE; CLINICAL PROTOCOL; COMPARATIVE EFFECTIVENESS; COST EFFECTIVENESS ANALYSIS; DIFFERENTIAL DIAGNOSIS; DISEASE CLASSIFICATION; DISEASE CONTROL; DISEASE EXACERBATION; DOSAGE SCHEDULE COMPARISON; DOSE RESPONSE; DRUG EFFICACY; DRUG SAFETY; EMERGENCY CARE; EVIDENCE BASED MEDICINE; FORCED EXPIRATORY VOLUME; HEALTH CARE COST; HUMAN; METERED DOSE INHALER; OUTCOME ASSESSMENT; PATIENT COMPLIANCE; PATIENT SELECTION; PEAK EXPIRATORY FLOW; POWDER INHALER; PRACTICE GUIDELINE; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL (TOPIC); SMOKING HABIT;

EID: 81155123124     PISSN: 15297322     EISSN: 15346315     Source Type: Journal    
DOI: 10.1007/s11882-011-0222-7     Document Type: Article

References (83)

1. British Guideline on the Management of Asthma. A national clinical guideline. May 2008. Revised June 2009. Available at http://www.sign.ac.uk/ guidelines/fulltext/101/index.html.
2. HK Reddel DR Taylor ED Bateman, et al. 2009 An official American Thoracic Society/European Respiratory Society statement: asthma control and exacerbations: standardizing endpoints for clinical asthma trials and clinical practice Am J Respir Crit Care Med 180 59 99 19535666 10.1164/rccm.200801-060ST
3. GINA report, Global Strategy for Asthma Management and Prevention. Available at http://www.ginasthma.org.
4. RA Pauwels CG Lofdahl DS Postma, et al. 1997 Effect of inhaled formoterol and budesonide on exacerbations of asthma. Formoterol and Corticosteroids Establishing Therapy (FACET) International Study Group N Engl J Med 337 1405 1411 9358137 10.1056/NEJM199711133372001 1:CAS:528:DyaK2sXnsVynuro%3D (Pubitemid 27497887)
5. L Bjermer H Bisgaard J Bousquet, et al. 2003 Montelukast and fluticasone compared with salmeterol and fluticasone in protecting against asthma exacerbation in adults: one year, double blind, randomised, comparative trial BMJ 327 891 14563743 10.1136/bmj.327.7420.891 1:CAS:528:DC%2BD3sXptlCis70%3D
6. ED Bateman HA Boushey J Bousquet, et al. 2004 Can guideline-defined asthma control be achieved? The gaining optimal asthma control study Am J Respir Crit Care Med 170 836 844 15256389 10.1164/rccm.200401-033OC (Pubitemid 39346477)
7. RJ Prescott CE Counsell WJ Gillespie, et al. 1999 Factors that limit the quality, number and progress of randomised controlled trials Health Technol Assess 3 1 143 10683591 1:STN:280:DC%2BD3c7kvFartg%3D%3D
8. D Price M Thomas 2006 Breaking new ground: challenging existing asthma guidelines BMC Pulm Med 6 Suppl 1 S6 17140424 10.1186/1471-2466-6-S1-S6 (Pubitemid 352015477)
9. M Thomas J Cleland D Price 2003 Database studies in asthma pharmacoeconomics: uses, limitations and quality markers Expert Opin Pharmacother 4 351 358 12614187 10.1517/14656566.4.3.351 (Pubitemid 36367558)
10. JA Krishnan M Schatz AJ Apter 2011 A call for action: comparative effectiveness research in asthma J Allergy Clin Immunol 127 123 127 20855111 10.1016/j.jaci.2010.08.032 This is a discussion of comparative effectiveness research (and its challenges), how it complements traditional efficacy research in asthma, and how it helps provide a basis for rational decision making about individual patients' care
11. J Clatworthy D Price D Ryan, et al. 2009 The value of self-report assessment of adherence, rhinitis and smoking in relation to asthma control Prim Care Respir J 18 300 305 19562233 10.4104/pcrj.2009.00037
12. M Thomas D Price 2008 Impact of comorbidities on asthma Expert Rev Clin Immunol 4 731 742 20477123 10.1586/1744666X.4.6.731
13. V Giraud N Roche 2002 Misuse of corticosteroid metered-dose inhaler is associated with decreased asthma stability Eur Respir J 19 246 251 11866004 10.1183/09031936.02.00218402 1:STN:280:DC%2BD387jtVOkuw%3D%3D (Pubitemid 34162824)
14. M Molimard C Raherison S Lignot, et al. 2003 Assessment of handling of inhaler devices in real life: an observational study in 3811 patients in primary care J Aerosol Med 16 249 254 14572322 10.1089/089426803769017613 1:STN:280:DC%2BD3svps1KgsA%3D%3D (Pubitemid 37169635)
15. J Haughney D Price A Kaplan, et al. 2008 Achieving asthma control in practice: understanding the reasons for poor control Respir Med 102 1681 1693 18815019 10.1016/j.rmed.2008.08.003
16. R Nadif R Matran J Maccario, et al. 2010 Passive and active smoking and exhaled nitric oxide levels according to asthma and atopy in adults Ann Allergy Asthma Immunol 104 385 393 20486328 10.1016/j.anai.2010.03.013
17. JG Mastronarde NR Anthonisen M Castro, et al. 2009 Efficacy of esomeprazole for treatment of poorly controlled asthma N Engl J Med 360 1487 1499 19357404 10.1056/NEJMoa0806290 1:CAS:528:DC%2BD1MXltlSqsr0%3D
18. J Travers S Marsh M Williams, et al. 2007 External validity of randomised controlled trials in asthma: to whom do the results of the trials apply? Thorax 62 219 223 17105779 10.1136/thx.2006.066837 (Pubitemid 46579801)
19. SL Appleton RJ Adams DH Wilson, et al. 2005 Spirometric criteria for asthma: adding further evidence to the debate J Allergy Clin Immunol 116 976 982 16275363 10.1016/j.jaci.2005.08.034 (Pubitemid 41571775)
20. K Herland JP Akselsen OH Skjonsberg L Bjermer 2005 How representative are clinical study patients with asthma or COPD for a larger "real life" population of patients with obstructive lung disease? Respir Med 99 11 19 15672843 10.1016/j.rmed.2004.03.026 (Pubitemid 40017378)
21. Costa DJ, Amouyal M, Lambert P, et al. How representative are clinical study patients with allergic rhinitis in primary care? J Allergy Clin Immunol 2011; Jan 29 (Epub ahead of print).
22. KL Lavoie SL Bacon M Labrecque, et al. 2006 Higher BMI is associated with worse asthma control and quality of life but not asthma severity Respir Med 100 648 657 16159709 10.1016/j.rmed.2005.08.001 (Pubitemid 43285752)
23. P Saint-Pierre A Bourdin P Chanez, et al. 2006 Are overweight asthmatics more difficult to control? Allergy 61 79 84 16364160 1:STN:280: DC%2BD2Mnns1Cmtg%3D%3D (Pubitemid 43381195)
24. K Hakala B Stenius-Aarniala A Sovijarvi 2000 Effects of weight loss on peak flow variability, airways obstruction, and lung volumes in obese patients with asthma Chest 118 1315 1321 11083680 10.1378/chest.118.5.1315 1:STN:280:DC%2BD3M%2FkvFOnsw%3D%3D
25. MD Althuis M Sexton D Prybylski 1999 Cigarette smoking and asthma symptom severity among adult asthmatics J Asthma 36 257 264 10350222 10.3109/02770909909075409 1:STN:280:DyaK1M3nslOhtw%3D%3D (Pubitemid 29267546)
26. F Gallefoss PS Bakke 2003 Does smoking affect the outcome of patient education and self-management in asthmatics? Patient Educ Couns 49 91 97 12527158 10.1016/S0738-3991(02)00051-4 (Pubitemid 36071314)
27. R Chaudhuri E Livingston AD McMahon, et al. 2003 Cigarette smoking impairs the therapeutic response to oral corticosteroids in chronic asthma Am J Respir Crit Care Med 168 1308 1311 12893649 10.1164/rccm.200304-503OC (Pubitemid 37487324)
28. GW Chalmers KJ Macleod SA Little, et al. 2002 Influence of cigarette smoking on inhaled corticosteroid treatment in mild asthma Thorax 57 226 230 11867826 10.1136/thorax.57.3.226 1:STN:280:DC%2BD387jt1arsg%3D%3D (Pubitemid 34214408)
29. PJ Barnes K Ito IM Adcock 2004 Corticosteroid resistance in chronic obstructive pulmonary disease: inactivation of histone deacetylase Lancet 363 731 733 15001333 10.1016/S0140-6736(04)15650-X 1:CAS:528:DC%2BD2cXhs12jur0%3D (Pubitemid 38296564)
30. J Fauler JC Frolich 1997 Cigarette smoking stimulates cysteinyl leukotriene production in man Eur J Clin Invest 27 43 47 9041376 10.1046/j.1365-2362.1997.650619.x 1:CAS:528:DyaK2sXhsFeqsb0%3D (Pubitemid 27065172)
31. KF Rabe M Adachi CK Lai, et al. 2004 Worldwide severity and control of asthma in children and adults: the global asthma insights and reality surveys J Allergy Clin Immunol 114 40 47 15241342 10.1016/j.jaci.2004.04.042 (Pubitemid 39022491)
32. Lenney W, Perry S, Price D. Clinical trials and tribulations: the MASCOT study. Thorax 2011; doi: 10.1136/thx.2010.156885.
33. Sharek PJ, Bergman DA. Beclomethasone for asthma in children: effects on linear growth. Cochrane Database Syst Rev 2000; CD001282.
34. S Holgate H Bisgaard L Bjermer, et al. 2008 The Brussels Declaration: the need for change in asthma management Eur Respir J 32 1433 1442 19043008 10.1183/09031936.00053108 1:STN:280:DC%2BD1cjnsVChuw%3D%3D
35. •• Rawlins MD, De Testimonio. On the evidence for decisions about the use of therapeutic interventions. In: Harveian Oration. Oct-2008 ed. London: Royal College of Physicians; 2008. As Chairman of the National Institute for Health and Clinical Excellence in the United Kingdom, Rawlins' recognition of the importance of real world studies when considering the totality of the evidence base indicates a paradigm shift.
36. D Schwartz J Lellouch 1967 Explanatory and pragmatic attitudes in therapeutical trials J Chronic Dis 20 637 648 4860352 10.1016/0021-9681(67) 90041-0 1:STN:280:DyaF1c%2FgtVGlsg%3D%3D
37. SG Coca HM Krumholz AX Garg CR Parikh 2006 Underrepresentation of renal disease in randomized controlled trials of cardiovascular disease JAMA 296 1377 1384 16985230 10.1001/jama.296.11.1377 1:CAS:528:DC%2BD28XpvF2lsbw%3D (Pubitemid 44427424)
38. PY Lee KP Alexander BG Hammill, et al. 2001 Representation of elderly persons and women in published randomized trials of acute coronary syndromes JAMA 286 708 713 11495621 10.1001/jama.286.6.708 1:STN:280: DC%2BD3MvmtFeisQ%3D%3D (Pubitemid 33010364)
39. DA Zarin JL Young JC West 2005 Challenges to evidence-based medicine: a comparison of patients and treatments in randomized controlled trials with patients and treatments in a practice research network Soc Psychiatry Psychiatr Epidemiol 40 27 35 15624072 10.1007/s00127-005-0838-9 (Pubitemid 40069169)
40. M Zwarenstein S Treweek JJ Gagnier, et al. 2008 Improving the reporting of pragmatic trials: an extension of the CONSORT statement BMJ 337 a2390 19001484 10.1136/bmj.a2390
41. J Bousquet T Bodez P Gehano, et al. 2009 Implementation of guidelines for allergic rhinitis in specialist practices. a randomized pragmatic controlled trial Int Arch Allergy Immunol 150 75 82 19339805 10.1159/000210383
42. P Demoly R Louis U Soes-Petersen, et al. 2009 Budesonide/formoterol maintenance and reliever therapy versus conventional best practice Respir Med 103 1623 1632 19762222 10.1016/j.rmed.2009.07.018
43. DP Tashkin B Celli S Senn, et al. 2008 A 4-year trial of tiotropium in chronic obstructive pulmonary disease N Engl J Med 359 1543 1554 18836213 10.1056/NEJMoa0805800 1:CAS:528:DC%2BD1cXht1WgsrjJ
44. D Price SD Musgrave L Shepstone, et al. 2011 Leukotriene antagonists as first-line or add-on asthma-controller therapy N Engl J Med. 364 1695 707 21542741 10.1056/NEJMoa1010846 1:CAS:528:DC%2BC3MXlvFymtLw%3D
45. BR Celli NE Thomas JA Anderson, et al. 2008 Effect of pharmacotherapy on rate of decline of lung function in chronic obstructive pulmonary disease: results from the TORCH study Am J Respir Crit Care Med 178 332 338 18511702 10.1164/rccm.200712-1869OC
46. ED Bateman J Bousquet WW Busse, et al. 2008 Stability of asthma control with regular treatment: an analysis of the Gaining Optimal Asthma controL (GOAL) study Allergy 63 932 938 18588561 10.1111/j.1398-9995.2008.01724.x 1:STN:280:DC%2BD1cvhvVOqsg%3D%3D (Pubitemid 351832801)
47. L Bjermer H Bisgaard J Bousquet, et al. 2000 Montelukast or salmeterol combined with an inhaled steroid in adult asthma: design and rationale of a randomized, double-blind comparative study (the IMPACT Investigation of Montelukast as a Partner Agent for Complementary Therapy-trial) Respir Med 94 612 621 10921768 10.1053/rmed.2000.0806 1:STN:280:DC%2BD3czpvVOnuw%3D%3D (Pubitemid 30601338)
48. K Malmstrom G Rodriguez-Gomez J Guerra, et al. 1999 Oral montelukast, inhaled beclomethasone, and placebo for chronic asthma. A randomized, controlled trial. Montelukast/Beclomethasone Study Group Ann Intern Med 130 487 495 10075616 1:CAS:528:DyaK1MXitFChs7c%3D (Pubitemid 29135801)
49. ER Bleecker MJ Welch SF Weinstein, et al. 2000 Low-dose inhaled fluticasone propionate versus oral zafirlukast in the treatment of persistent asthma J Allergy Clin Immunol 105 1123 1129 10856145 10.1067/mai.2000.106043 1:CAS:528:DC%2BD3cXkslynsLk%3D (Pubitemid 30418790)
50. W Busse GD Raphael S Galant, et al. 2001 Low-dose fluticasone propionate compared with montelukast for first-line treatment of persistent asthma: a randomized clinical trial J Allergy Clin Immunol 107 461 468 11240946 10.1067/mai.2001.114657 1:CAS:528:DC%2BD3MXisFSlur0%3D (Pubitemid 32210026)
51. EO Meltzer RF Lockey BF Friedman, et al. 2002 Efficacy and safety of low-dose fluticasone propionate compared with montelukast for maintenance treatment of persistent asthma Mayo Clin Proc 77 437 445 12004993 10.4065/77.5.437 1:CAS:528:DC%2BD38XjvFOitrY%3D (Pubitemid 34465151)
52. GP Currie DK Lee P Srivastava 2005 Long-acting bronchodilator or leukotriene modifier as add-on therapy to inhaled corticosteroids in persistent asthma? Chest 128 2954 2962 16236972 10.1378/chest.128.4.2954 1:CAS:528:DC%2BD2MXhtF2msL3P (Pubitemid 41507655)
53. Ducharme FM, Lasserson TJ, Cates CJ. Long-acting beta2-agonists versus anti-leukotrienes as add-on therapy to inhaled corticosteroids for chronic asthma. Cochrane Database Syst Rev 2006; CD003137.
54. S Joos A Miksch J Szecsenyi, et al. 2008 Montelukast as add-on therapy to inhaled corticosteroids in the treatment of mild to moderate asthma: a systematic review Thorax 63 453 462 18443162 10.1136/thx.2007.081596 1:STN:280:DC%2BD1c3otFWiuw%3D%3D (Pubitemid 351698876)
55. J Bousquet VJ Lund P Van Cauwenberge, et al. 2003 Implementation of guidelines for seasonal allergic rhinitis: a randomized controlled trial Allergy 58 733 741 12859551 10.1034/j.1398-9995.2003.00076.x 1:STN:280: DC%2BD3szotVWquw%3D%3D (Pubitemid 36920462)
56. D Price A Robertson K Bullen, et al. 2010 Improved adherence with once-daily versus twice-daily dosing of mometasone furoate administered via a dry powder inhaler: a randomized open-label study BMC Pulm Med 10 1 20051135 10.1186/1471-2466-10-1
57. TLN Järvinen H Sievänen P Kannus, et al. 2011 The true cost of pharmacological disease prevention BMJ 342 d2175 10.1136/bmj.d2175 21505222 10.1136/bmj.d2175
58. TP van Staa HG Leufkens B Zhang L Smeeth 2009 A comparison of cost effectiveness using data from randomized trials or actual clinical practice: selective cox-2 inhibitors as an example PLoS Med 6 e1000194 19997499 10.1371/journal.pmed.1000194
59. DM Kent G Kitsios 2009 Against pragmatism: on efficacy, effectiveness and the real world Trials 10 48 19580642 10.1186/1745-6215-10-48
60. K-L Resch 2005 Pragmatic trials: propaganda or research tool? Focus Altern Complement Ther 10 181 183 (Pubitemid 41309244)
61. E Ernst PH Canter 2005 Limitations of "pragmatic" trials Postgrad Med J 81 203 15811880 10.1136/pgmj.2004.026807 1:STN:280: DC%2BD2M7nvF2ksg%3D%3D (Pubitemid 40546612)
62. JH Ware MB Hamel 2011 Statistics in medicine, pragmatic trials-guides to better patient care? N Engl J Med 364 1685 7 21542739 10.1056/NEJMp1103502 1:CAS:528:DC%2BC3MXlvFymt7Y%3D
63. R Temple SS Ellenberg 2000 Placebo-controlled trials and active-control trials in the evaluation of new treatments. Part 1: ethical and scientific issues Ann Intern Med 133 455 463 10975964 1:STN:280:DC%2BD3cvktFGktQ%3D%3D
64. SS Ellenberg R Temple 2000 Placebo-controlled trials and active-control trials in the evaluation of new treatments. Part 2: practical issues and specific cases Ann Intern Med 133 464 470 10975965 1:STN:280: DC%2BD3cvktFGkug%3D%3D
65. National Center for Biotechnology Information (NCBI) Bookshelf. Available at http://www.ncbi.nlm.nih.gov/books/NBK10370/.
66. H Jick 1992 Further validation of information recorded on a general practitioner based computerized data resource in the United Kingdom Pharmacoepidemiol Drug Saf 1 347 349 10.1002/pds.2630010607
67. SS Jick JA Kaye C Vasilakis-Scaramozza, et al. 2003 Validity of the general practice research database Pharmacotherapy 23 686 689 12741446 10.1592/phco.23.5.686.32205 (Pubitemid 36539051)
68. YS Punekar A Sheikh 2009 Establishing the incidence and prevalence of clinician-diagnosed allergic conditions in children and adolescents using routinely collected data from general practices Clin Exp Allergy 39 1209 1216 19400899 10.1111/j.1365-2222.2009.03248.x 1:STN:280:DC%2BD1MrgtVKhtw%3D%3D
69. JB Soriano WC Maier G Visick NB Pride 2001 Validation of general practitioner-diagnosed COPD in the UK General Practice Research Database Eur J Epidemiol 17 1075 1080 12530765 10.1023/A:1021235123382 1:STN:280: DC%2BD3s%2Fitlyhug%3D%3D
70. DA Stempel CS Roberts RH Stanford 2004 Treatment patterns in the months prior to and after asthma-related emergency department visit Chest 126 75 80 15249445 10.1378/chest.126.1.75 (Pubitemid 38943120)
71. J Lenney JA Innes GK Crompton 2000 Inappropriate inhaler use: assessment of use and patient preference of seven inhalation devices Respir Med 94 496 500 10868714 10.1053/rmed.1999.0767 1:STN:280:DC%2BD3czisVGltA%3D%3D (Pubitemid 30353008)
72. F Lavorini A Magnan JC Dubus, et al. 2008 Effect of incorrect use of dry powder inhalers on management of patients with asthma and COPD Respir Med 102 593 604 18083019 10.1016/j.rmed.2007.11.003
73. L Kemp J Haughney N Barnes, et al. 2010 Cost-effectiveness analysis of corticosteroid inhaler devices in primary care asthma management: a real world observational study ClinicoEconomics Outcomes Res 2 75 85
74. D Price J Haughney E Sims, et al. 2011 Effectiveness of inhaler types for real-world asthma management: retrospective observational study using the GPRD J Asthma Allergy 4 37 47 21698214 1:CAS:528:DC%2BC3MXmsVegsr4%3D
75. Study of Treatment Effectiveness in Asthma and Rhinitis (STEAR). Merck, data on file.
76. M Thomas J von Ziegenweidt AJ Lee D Price 2009 High-dose inhaled corticosteroids versus add-on long-acting beta-agonists in asthma: an observational study J Allergy Clin Immunol 123 116 121 18986690 10.1016/j.jaci.2008.09.035 1:CAS:528:DC%2BD1MXjvFyktQ%3D%3D e110
77. Price D, Martin RJ, Barnes N, et al. Prescribing practices and asthma control with hydrofluoroalkane-beclomethasone and fluticasone: a real-world observational study. J Allergy Clin Immunol 2010, 126:511-518 e511-510.
78. Barnes N, Price D, Colice G, et al. Asthma control with extrafine-particle HFA-beclomethasone versus CFC-beclomethasone: a real-world observational study. Under consideration. Clin Exp Allergy 2011; Jul 14. doi: 10.1111/j.1365-2222.2011.03820.x. [Epub ahead of print].
79. MJ Radford JM Foody 2001 How do observational studies expand the evidence base for therapy? JAMA 286 1228 1230 11559269 10.1001/jama.286.10.1228 1:STN:280:DC%2BD3MrhvVegtA%3D%3D (Pubitemid 33015553)
80. K Benson AJ Hartz 2000 A comparison of observational studies and randomized, controlled trials N Engl J Med 342 1878 1886 10861324 10.1056/NEJM200006223422506 1:STN:280:DC%2BD3czgt1Chsg%3D%3D (Pubitemid 30419053)
81. E von Elm DG Altman M Egger, et al. 2007 The strengthening the reporting of observational studies in epidemiology (STROBE) statement: guidelines for reporting observational studies Lancet 370 1453 1457 10.1016/S0140-6736(07) 61602-X
82. MK Campbell DR Elbourne DG Altman 2004 CONSORT statement: extension to cluster randomised trials BMJ 328 702 708 15031246 10.1136/bmj.328.7441.702 (Pubitemid 38393415)
83. J Bousquet HJ Schunemann T Zuberbier, et al. 2010 Development and implementation of guidelines in allergic rhinitis - an ARIA-GA2LEN paper Allergy 65 1212 1221 20887423 10.1111/j.1398-9995.2010.02439.x