A systematic review finds variable use of the intention-to-treat principle in musculoskeletal randomized controlled trials with missing data

Journal of Clinical Epidemiology

Volumn 68, Issue 1, 2015, Pages 15-24

  Joseph, Royes ^a   Sim, Julius ^a   Ogollah, Reuben ^a   Lewis, Martyn ^a  

^a KEELE UNIVERSITY   (United Kingdom)

Author keywords

Dropout; Intention to treat; Missing data; Musculoskeletal conditions; Randomized controlled trial; Sensitivity analysis; Systematic review

Indexed keywords

DATA EXTRACTION; DISEASE CLASSIFICATION; FEASIBILITY STUDY; FOLLOW UP; HUMAN; INTENTION TO TREAT ANALYSIS; META ANALYSIS; MUSCULOSKELETAL SYSTEM; OUTCOME ASSESSMENT; PUBLICATION; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; RHEUMATIC DISEASE; SENSITIVITY ANALYSIS; SYSTEMATIC REVIEW; MUSCULOSKELETAL DISEASES; PATIENT DROPOUTS; PROCEDURES; STANDARDS; STATISTICAL ANALYSIS; STATISTICS AND NUMERICAL DATA;

DATA INTERPRETATION, STATISTICAL; HUMANS; INTENTION TO TREAT ANALYSIS; MUSCULOSKELETAL DISEASES; PATIENT DROPOUTS; PUBLICATIONS; RANDOMIZED CONTROLLED TRIALS AS TOPIC;

EID: 84915747719     PISSN: 08954356     EISSN: 18785921     Source Type: Journal    
DOI: 10.1016/j.jclinepi.2014.09.002     Document Type: Review

References (54)

1. A. Chan, J.M. Tetzlaff, P.C. Gøtzsche, D.G. Altman, H. Mann, and J.A. Berlin SPIRIT 2013 explanation and elaboration: guidance for protocols of clinical trials BMJ 346 2013 e7586
2. V.M. Montori, and G.H. Guyatt Intention-to-treat principle CMAJ 165 10 2001 1339 1341
3. S.R. Heritier, V.J. Gebski, and A.C. Keech Inclusion of patients in clinical trial analysis: the intention-to-treat principle Med J Aust 179 2003 438 440
4. D.G. Altman Missing outcomes in randomized trials: addressing the dilemma Open Med 3 2 2009 e51 e53
5. T.R. Fleming Addressing missing data in clinical trials Ann Intern Med 154 2011 113 117
6. J.A. Lewis, and D. Machin Intention to treat - who should use ITT? Br J Cancer 68 1993 647 650
7. M. Zwarenstein, S. Treweek, J.J. Gagnier, D.G. Altman, S. Tunis, and B. Haynes Improving the reporting of pragmatic trials: an extension of the CONSORT statement BMJ 337 2008 a2390
8. J.M. Lachin Statistical consideration in the intention-to-treat principle Control Clin Trials 21 2000 167 189
9. Higgins JPT, Altman DG. Chapter 8: assessing risk of bias in included studies. In: Higgins JPT, Green S, Eds. Cochrane handbook for systematic reviews of interventions, Version 5.1.0: The Cochrane Collaboration. Available at www.cochrane-handbook.org. Accessed June 8, 2014.
10. J.P. Ioannidis, S.J. Evans, P.C. Gotzsche, R.T. O'Neill, D.G. Altman, and K. Schulz Better reporting of harms in randomized trials: an extension of the CONSORT statement Ann Intern Med 141 2004 781 788
11. L. Abraha, and A. Montedori Modified intention to treat reporting in randomised controlled trials: systematic review BMJ 340 2010 c2697
12. K.F. Schulz, D.G. Altman, and D. Moher CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials BMJ 340 2010 c332
13. R. McNamee Intention to treat, per protocol, as treated and instrumental variable estimators given non-compliance and effect heterogeneity Stat Med 28 2009 2639 2652
14. I.R. White, N.J. Horton, J. Carpenter, and S.J. Pocock Strategy for intention to treat analysis in randomised trials with missing outcome data BMJ 342 2011 d40
15. R. Crutzen, W. Viechtbauer, D. Kotz, and M. Spigt No differential attrition was found in randomized controlled trials published in general medical journals: a meta-analysis J Clin Epidemiol 66 2013 948 954
16. N.J. Horton, and S.R. Lipsitz Multiple imputation in practice: comparison of software packages for regression models with missing data Am Stat 55 2001 244 254
17. European Medicines Agency Guideline on missing data in confirmatory clinical trials 2010 Committee for Medical Products for Human Use London, UK Available at http://www.ema.europa.eu/docs/en-GB/document-library/Scientific-guideline/2010/09/WC500096793.pdf Accessed September 20, 2013
18. D.B. Rubin Inference and missing data Biometrika 63 3 1976 581 592
19. R.J.A. Little, and D.B. Rubin Statistical analysis with missing data 2nd ed. 2002 Wiley Hoboken, NJ
20. C.C. Wright, and J. Sim Intention-to-treat approach to data from randomized controlled trials: a sensitivity analysis J Clin Epidemiol 56 2003 833 842
21. S. Fielding, P.M. Fayers, and C.R. Ramsay Investigating the missing data mechanism in quality of life outcomes: a comparison of approaches Health Qual Life Outcomes 7 2009 57
22. G. Molenberghs, and M.G. Kenward Missing data in clinical studies 2007 Wiley Chichester, UK
23. National Research Council The prevention and treatment of missing data in clinical trials. Panel on handling missing data in clinical trials. Committee on National Statistics, Division of Behavioral and Social Sciences and Education 2010 National Academies Press Washington, DC
24. S. Hollis, and F. Campbell What is meant by intention to treat analysis? Survey of published randomised controlled trials BMJ 319 1999 670
25. R.L. Kruse, B.S. Alper, C. Reust, J.J. Stevermer, S. Shannon, and R.H. Williams Intention-to-treat analysis: Who is in? Who is out? J Fam Pract 51 2002 969 971
26. A.M. Wood, I.R. White, and S.G. Thompson Are missing outcome data adequately handled? A review of published randomized controlled trials in major medical journals Clin Trials 1 2004 368 376
27. J. Gravel, L. Opatrny, and S. Shapiro The intention-to-treat approach in randomized controlled trials: are authors saying what they do and doing what they say? Clin Trials 4 2007 350 356
28. S. Fielding, G. Maclennan, J.A. Cook, and C.R. Ramsay A review of RCTs in four medical journals to assess the use of imputation to overcome missing data in quality of life outcomes Trial 9 2008 51
29. G. Baron, I. Boutron, B. Giraudeau, and P. Ravaud Violation of the intent-to-treat principle and rate of missing data in superiority trials assessing structural outcomes in rheumatic diseases Arthritis Rheum 52 2005 1858 1865
30. N. Henschke, T. Kuijpers, S.M. Rubinstein, M. van Middelkoop, R. Ostelo, and A. Verhagen Trends over time in the size and quality of randomised controlled trials of interventions for chronic low-back pain Eur Spine J 21 3 2012 375 381
31. B.W. Koes, A. Malmivaara, and M.W. van Tulder Trend in methodological quality of randomised clinical trials in low back pain Best Pract Res Clin Rheumatol 19 4 2005 529 539
32. Food and Drug Administration Guidance for sponsors, clinical investigators, and IRBs: data retention when subjects withdraw from FDA-regulated clinical trials 2008 Available at http://www.fda.gov/downloads/RegulatoryInformation/Guidances/ucm126489.pdf Accessed October 17, 2013
33. A. Cnaan, N.M. Laird, and P. Slasor Using the general linear mixed model to analyse unbalanced repeated measures and longitudinal data Stat Med 16 1997 2349 2380
34. J.W. Hardin, and J.M. Hilbe Generalized estimating equations 2003 Chapman & Hall/CRC Boca Raton, FL
35. J.S. Preisser, K.K. Lohman, and P.J. Rathouz Performance of weighted estimating equations for longitudinal binary data with drop-outs missing at random Stat Med 21 2002 3035 3054
36. A. Mackinnon The use and reporting of multiple imputation in medical research - a review J Intern Med 268 6 2010 586 593
37. Akai M. Musculoskeletal disorders. Pharmaceutical sciences encyclopedia 2010:1-23.
38. S. Hopewell, A. Hirst, G. Collins, S. Mallett, L. Yu, and D. Altman Reporting of participant flow diagrams in published reports of randomized trials Trials 12 2011 253
39. I.R. White, J. Carpenter, and N.J. Horton Including all individuals is not enough: lessons for intention-to-treat analysis Clin Trials 9 2012 396 407
40. D. Moher, K.F. Schulz, and D.G. Altman The CONSORT statement: revised recommendations for improving the quality of reports of parallel-group randomised trials Lancet 357 2001 1191 1194
41. D. Moher, S. Hopewell, F.S. Kenneth, V. Montori, C.G. Peter, and P.J. Devereaux CONSORT 2010 explanation and elaboration: updated guidelines for reporting parallel group randomised trials BMJ 340 2010 c869
42. R.J. Little, M.L. Cohen, K. Dickersin, S.S. Emerson, J.T. Farrar, and J.D. Neaton The design and conduct of clinical trials to limit missing data Stat Med 31 2012 3433 3443
43. P. Lane Handling drop-out in longitudinal clinical trials: a comparison of the LOCF and MMRM approaches Pharm Stat 7 2 2008 93 106
44. M. Kim Statistical methods in arthritis and rheumatism: current trends Arthritis Rheum 54 2006 3741 3749
45. S.B. Woolley, A.A. Cardoni, and J.W. Goethe Last-observation-carried-forward imputation method in clinical efficacy trials: review of 352 antidepressant studies Pharmacotherapy 29 12 2009 1408 1416
46. J.A.C. Sterne, I.R. White, J.B. Carlin, M. Spratt, P. Royston, and M.G. Kenward Multiple imputation for missing data in epidemiological and clinical research: potential and pitfalls BMJ 338 2009 b2393
47. J. Beaudreuil, S. Lasbleiz, P. Richette, G. Seguin, C. Rastel, and M. Aout Assessment of dynamic humeral centering in shoulder pain with impingement syndrome: a randomised clinical trial Ann Rheum Dis 70 9 2011 1613 1618
48. B.E. Thorn, M.A. Day, J. Burns, M.C. Kuhajda, S.W. Gaskins, and K. Sweeney Randomized trial of group cognitive behavioral therapy compared with a pain education control for low-literacy rural people with chronic pain Pain 152 12 2011 2710 2720
49. S. Genevay, S. Viatte, A. Finckh, P. Zufferey, F. Balagué, and C. Gabay Adalimumab in severe and acute sciatica: a multicenter, randomized, double-blind, placebo-controlled trial Arthritis Rheum 62 2010 2339 2346
50. R.E. Fary, G.J. Carroll, T.G. Briffa, and N.K. Briffa The effectiveness of pulsed electrical stimulation in the management of osteoarthritis of the knee: results of a double-blind, randomized, placebo-controlled, repeated-measures trial Arthritis Rheum 63 2011 1333 1342
51. H. Bliddal, A.R. Leeds, L. Stigsgaard, A. Astrup, and R. Christensen Weight loss as treatment for knee osteoarthritis symptoms in obese patients: 1-year results from a randomised controlled trial Ann Rheum Dis 70 10 2011 1798 1803
52. J. Twisk, M. de Boer, W. de Vente, and M. Heymans Multiple imputation of missing values was not necessary before performing a longitudinal mixed-model analysis J Clin Epidemiol 66 2013 1022 1028
53. K. Liang, and S.L. Zeger Longitudinal data analysis using generalized linear models Biometrika 73 1 1986 13 22
54. T. Birhanu, G. Molenberghs, C. Sotto, and M.G. Kenward Doubly robust and multiple-imputation-based generalized estimating equations J Biopharm Stat 21 2 2011 202 225