A randomised trial of adaptive pacing therapy, cognitive behaviour therapy, graded exercise, and specialist medical care for chronic fatigue syndrome (PACE): Statistical analysis plan

Trials

Volumn 14, Issue 1, 2013, Pages

  Walwyn, Rebecca ^a,f   Potts, Laura ^a   McCrone, Paul ^a   Johnson, Anthony L ^b,c   DeCesare, Julia C ^e   Baber, Hannah ^e   Goldsmith, Kimberley ^a   Sharpe, Michael ^d   Chalder, Trudie ^a   White, Peter D ^e  

^a KING'S COLLEGE LONDON   (United Kingdom)

^b MRC BIOSTATISTICS UNIT   (United Kingdom)

^c MRC CLINICAL TRIALS UNIT   (United Kingdom)

^d WARNEFORD HOSPITAL   (United Kingdom)

^e QUEEN MARY UNIVERSITY OF LONDON   (United Kingdom)

^f UNIVERSITY OF LEEDS   (United Kingdom)

Author keywords

chronic fatigue syndrome; myalgic encephalomyelitis; PACE trial; randomised controlled trial; Statistical analysis plan

Indexed keywords

ADAPTIVE PACING THERAPY; ARTICLE; CHRONIC FATIGUE SYNDROME; COGNITIVE THERAPY; CONTROLLED STUDY; COST EFFECTIVENESS ANALYSIS; COST UTILITY ANALYSIS; GRADED EXERCISE THERAPY; HEALTH CARE COST; HUMAN; INFORMATION PROCESSING; KINESIOTHERAPY; MEDICAL SPECIALIST; OUTCOME ASSESSMENT; PATIENT COMPLIANCE; PHYSICAL ACTIVITY; RANDOMIZED CONTROLLED TRIAL; STATISTICAL ANALYSIS; STRATIFICATION; STUDY DESIGN; TEACHING;

ADAPTATION, PHYSIOLOGICAL; COGNITIVE THERAPY; COMBINED MODALITY THERAPY; COST-BENEFIT ANALYSIS; DATA COLLECTION; EXERCISE THERAPY; FATIGUE SYNDROME, CHRONIC; HUMANS; OUTCOME ASSESSMENT (HEALTH CARE); RESEARCH DESIGN; SAMPLE SIZE;

EID: 84887388738     PISSN: None     EISSN: 17456215     Source Type: Journal    
DOI: 10.1186/1745-6215-14-386     Document Type: Article

References (80)

1. Horton R. Pardonable revisions and protocol reviews. Lancet 1997, 349:6. 10.1016/S0140-6736(05)62158-7, 8988113.
2. McNamee D. Review of clinical protocols at The Lancet. Lancet 1820, 2001:357.
3. Roy LP. Review of research protocols. Lancet 1999, 353:428. 10.1016/S0140-6736(98)00229-3, 9989711.
4. Summerskill W, Collingridge D, Frankish H. Protocols, probity, and publication. Lancet 2009, 373:992. 10.1016/S0140-6736(09)60590-0, 19304003.
5. Chalmers I, Altman DG. How can medical journals help prevent poor medical research? Some opportunities presented by electronic publishing. Lancet 1999, 353:490-493. 10.1016/S0140-6736(98)07618-1, 9989737.
6. Godlee F. Publishing study protocols: Making them visible will improve registration, reporting and treatment. BMC News Views 2001, 2:4.
7. Jones G, Abbasi K. Trial protocols at the BMJ. BMJ 2004, 329:1360. 10.1136/bmj.329.7479.1360, 535440, 15591544.
8. Chan A-W. Bias, spin, and misreporting: Time for full access to trial protocols and results. PLoS Med 2008, 5:e230. 10.1371/journal.pmed.0050230, 2586359, 19067481.
9. Finfer S, Bellomo R. Why publish statistical analysis plans?. Crit Care Resusc 2009, 11:5-6.
10. Finfer S, Cass A, Gallagher M, Lee J, Su S, Bellomo R,. On behalf of the RENAL Study Investigators The RENAL (randomised evaluation of normal vs. Augmented level of replacement therapy) study: statistical analysis plan. Crit Care Resusc 2009, 11:58-66. On behalf of the RENAL Study Investigators.
11. Finfer S, Heritier SR,. On behalf of the NICE Study Management Committee and SUGAR Study Executive Committee The NICE-SUGAR (normoglycaemia in intensive care evaluation and survival using glucose algorithm regulation) study: statistical analysis plan. Crit Care Resusc 2009, 11:46-57. On behalf of the NICE Study Management Committee and SUGAR Study Executive Committee.
12. ICH Expert Working Group E9 - Statistical Principles for Clinical Trials. [http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E9/Step4/E9_Guideline.pdf], ICH Expert Working Group.
13. White PD, Sharpe MC, Chalder T, DeCesare JC, Walwyn R,. The PACE trial group Protocol for the PACE trial: A randomised controlled trial of adaptive pacing, cognitive behaviour therapy, and graded exercise as supplements to standardised specialist medical care versus standardised specialist medical care alone for patients with the chronic fatigue syndrome/myalgic encephalomyelitis or encephalopathy. BMC Neurol 2007, 7:6. 10.1186/1471-2377-7-6, 2147058, 17397525, The PACE trial group.
14. White PD, Goldsmith KA, Johnson AL, Potts L, Walwyn R, DeCesare JC, Baber HL, Burgess M, Clark LV, Cox DL, Bavinton J, Angus BJ, Murphy G, Murphy M, O'Dowd H, Wilks D, McCrone P, Chalder T, Sharpe M,. On behalf of the PACE trial management group Comparison of adaptive pacing therapy, cognitive behaviour therapy, graded exercise, and specialist medical care for chronic fatigue syndrome (PACE): A randomised trial. Lancet 2011, 377:823-836. 10.1016/S0140-6736(11)60096-2, 3065633, 21334061, On behalf of the PACE trial management group.
15. McCrone P, Sharpe M, Chalder T, Knapp M, Johnson AL, Goldsmith KA, White PD. Adaptive pacing, cognitive behaviour therapy, graded exercise, and specialist medical care for chronic fatigue syndrome: a cost-effectiveness analysis. PloS One 2012, 7:e40808. 10.1371/journal.pone.0040808, 3411573, 22870204.
16. White PD, Goldsmith KA, Johnson AL, Walwyn R, Baber HL, Chalder T, Sharpe M,. On behalf of the coauthors The PACE trial in chronic fatigue syndrome - Authors' reply. Lancet 2011, 377:1834-1835. On behalf of the coauthors.
17. Giakoumakis J. The PACE trial in chronic fatigue syndrome. Lancet 1831, 2011:377.
18. Mitchell JT. The PACE trial in chronic fatigue syndrome. Lancet 1831, 2011:377.
19. Feehan SM,. Behalf of the Liverpool ME Support Group The PACE trial in chronic fatigue syndrome. Lancet 2011, 377:1831-1832. Behalf of the Liverpool ME Support Group.
20. Kewley AJ. The PACE trial in chronic fatigue syndrome. Lancet 1832, 2011:377.
21. Stouten B, Goudsmit EM, Riley N. The PACE trial in chronic fatigue syndrome. Lancet 2011, 377:1832-1833.
22. Kindlon T. The PACE trial in chronic fatigue syndrome. Lancet 1833, 2011:377.
23. Shinohara M. The PACE trial in chronic fatigue syndrome. Lancet 2011, 377:1833-1834.
24. Vlaeyen JWS, Karsdorp P, Gatzounis R, Ranson S, Schrooten M. The PACE trial in chronic fatigue syndrome. Lancet 1834, 2011:377.
25. Roberts C, Walwyn R. Design and analysis of non-pharmacological treatment trials with multiple therapists per patient. Stat Med 2013, 32:81-98. 10.1002/sim.5521, 22865729.
26. CPMP/EWP/908/99 Points to consider on multiplicity issues in clinical trials. [http://www.tga.gov.au/pdf/euguide/ewp090899en.pdf], CPMP/EWP/908/99.
27. Ioannidis JP, Evans SJ, Gøtzsche PC, O'Neill RT, Altman DG, Schulz K, Moher D,. The CONSORT Group Better reporting of harms in randomized trials: An extension of the CONSORT statment. Ann Int Med 2004, 141:781-788. 10.7326/0003-4819-141-10-200411160-00009, 15545678, The CONSORT Group.
28. Boutron I, Moher D, Altman DG, Schulz KF, Ravaud P,. The CONSORT Group Extending the CONSORT statement to randomized trials of nonpharmacologic treatment: Explanation and elaboration. Ann Int Med 2008, 148:295-309. 10.7326/0003-4819-148-4-200802190-00008, 18283207, The CONSORT Group.
29. Chalder T, Berelowitz G, Pawlikowska T, Watts L, Wessely S, Wright D, Wallace EP. Development of a fatigue scale. J Psycho Res 1993, 37:147-153.
30. Ware JE, Sherbourne CD. The MOS 36-item short-form health survey (SF-36). I. Conceptual franework and item selection. Med Care 1992, 30:473-483. 10.1097/00005650-199206000-00002, 1593914.
31. Guy W. ECDEU Assessment Manual for Psychopharmcology 1976, Rockville, MD: NIMH.
32. Zigmond AS, Snaith RP. The hospital anxiety and depression scale. Acta Psychiatr Scand 1983, 67:361-370. 10.1111/j.1600-0447.1983.tb09716.x, 6880820.
33. Butland RJ, Pang J, Gross ER, Woodcock AA, Geddes DM. Two, six, and 12-minute walking tests in respiratory disease. Brit Med J 1982, 284:1607-1608.
34. Mundt JC, Marks IM, Shear MK, Greist JM. The work and social adjustment scale: A simple measure of impairment in functioning. Brit J Psychiatry 2002, 180:461-464.
35. Reeves WC, Lloyd A, Vernon SD, Klimas N, Jason LA, Bleijenberg G, Evengard B, White PD, Nisenbaum R, Unger ER,. The International Chronic Fatigue Syndrome Study Group The identification of ambiguities in the 1994 chronic fatigue syndrome research case definition and recommendations for resolution. BMC Health Serv Res 2003, 3:25. 10.1186/1472-6963-3-25, 317472, 14702202, The International Chronic Fatigue Syndrome Study Group.
36. Jenkins CD, Stanton BA, Niemcryk SJ, Rose RM. A scale for the estimation of sleep problems in clinical research. J Clin Epidemiol 1988, 41:313-321. 10.1016/0895-4356(88)90138-2, 3351539.
37. Beecham J, Knapp M. Costing psychiatric interventions. Measuring Mental Health Needs 2001, 200-224. London: Gaskell, Thornicroft G.
38. Brooks R. EuroQol: the current state of play. Health Policy 1996, 37:53-72. 10.1016/0168-8510(96)00822-6, 10158943.
39. Deale A, Chalder T, Marks I, Wessely S. Cognitive behaviour therapy for chronic fatigue syndrome: A randomized controlled trial. Am J Psychiatry 1997, 154:408-414.
40. Sharpe MC, Hawton K, Simkin S, Surawy C, Hackmann A, Klimes I, Peto T, Warrell D, Seagroatt V. Cognitive behaviour therapy for the chronic fatigue syndrome: A randomised controlled trial. Brit Med J 1996, 312:22-26. 10.1136/bmj.312.7022.22, 2349693, 8555852.
41. Prins JB, Bleijenberg G, Bazelmans E, Elving LD, de Boo TM, Severens JL, van der Wilt GJ, Spinhoven P, van der Meer JWM. Cognitive behaviour therapy for chronic fatigue syndrome: a multicentre randomised controlled trial. Lancet 2001, 357:841-847. 10.1016/S0140-6736(00)04198-2, 11265953.
42. Wearden AJ, Morriss RK, Mullis R, Strickland PL, Pearson DJ, Appleby L, Campbell IT, Morris JA. Randomised, double-blind, placebo-controlled treatment trial of fluoxetine and graded exercise for chronic fatigue syndrome. Brit J Psychiatry 1998, 172:485-490.
43. Fulcher KY, White PD. Randomised controlled trial of graded exercise in patients with the chronic fatigue syndrome. Brit Med J 1997, 314:1647-1652. 10.1136/bmj.314.7095.1647, 2126868, 9180065.
44. Petrella RJ, Koval JJ, Cunningham DA, Paterson DH. A self-paced step test to predict aerobic fitness in older adults in the primary care clinic. J Am Geriatrics Soc 2001, 49:632-638.
45. White PD, Naish VAB. Graded exercise therapy for chronic fatigue syndrome: an audit. Physiotherapy 2001, 87:285-288.
46. Machin D, Campbell M, Fayers P, Pinol A. Sample Size Tables for Clinical Studies 1997, Oxford: Blackwell Science.
47. Pocock S, Simon R. Sequential treatment assignment with balancing for prognostic factors in the controlled clinical trial. Biometrics 1975, 31:103-115. 10.2307/2529712, 1100130.
48. Lachin JM. Statistical considerations in the intent-to-treat principle. Controlled Clin Trials 2000, 21:167-189. 10.1016/S0197-2456(00)00046-5, 10822117.
49. CPMP Points to consider on adjustment for baseline covariates. Stat Med 2004, 23:701-709. CPMP.
50. Agresti A, Hartzel J. Strategies for comparing treatments on a binary response with multi-centre data. Stat Med 2000, 19:1115-1139. 10.1002/(SICI)1097-0258(20000430)19:8<1115::AID-SIM408>3.0.CO;2-X, 10790684.
51. Goldstein H, Browne W, Rasbash J. Multilevel modeling of medical data. Stat Med 2002, 21:3291-3315. 10.1002/sim.1264, 12375305.
52. Guo G, Zhao H. Multilevel modelling for binary data. Ann Rev Sociol 2000, 26:441-462.
53. Lindsey JK, Lambert P. On the appropriateness of marginal models for repeated measurements in clinical trials. Stat Med 1998, 17:447-469. 10.1002/(SICI)1097-0258(19980228)17:4<447::AID-SIM752>3.0.CO;2-G, 9496722.
54. Walwyn R, Roberts C. Therapist variation within randomised trials of psychotherapy: Implications for precision, internal and external validity. Stat Methods Med Res 2010, 19:291-315. 10.1177/0962280209105017, 19608603.
55. Browne WJ, Goldstein H, Rasbash J. Multiple membership multiple classification (MMMC) models. Stat Modell 2001, 1:103-124.
56. Fayers PM, Curran D, Machin D. Incomplete quality of life data in randomized trials: Missing items. Stat Med 1998, 17:676-696.
57. Curran D, Molenberghs G, Fayers PM, Machin D. Incomplete quality of life data in randomized trials: Missing forms. Stat Med 1998, 17:697-709. 10.1002/(SICI)1097-0258(19980315/15)17:5/7<697::AID-SIM815>3.0.CO;2-Y, 9549817.
58. Kenward MG, Carpenter J. Multiple imputation: current perspectives. Stat Methods Med Res 2007, 16:199-218. 10.1177/0962280206075304, 17621468.
59. Schafer JL. Multiple imputation: a primer. Stat Methods Med Res 1999, 8:3-15. 10.1191/096228099671525676, 10347857.
60. White IR, Royston P, Wood AM. Multiple imputation using chained equations: Issues and guidance for practice. Stat Med 2011, 30:377-399. 10.1002/sim.4067, 21225900.
61. White IR, Thompson SG. Adjusting for partially missing baseline measurements in randomized trials. Stat Med 2005, 24:993-1007. 10.1002/sim.1981, 15570623.
62. Kenward MG, Molenberghs G. Likelihood based frequentist inference when data are missing at random. Stat Sci 1998, 13:236-247.
63. Carpenter JR, Kenward MG, White IR. Sensitivity analysis after multiple imputation under missing at random: a weighting approach. Stat Methods Med Res 2007, 16:259-275. 10.1177/0962280206075303, 17621471.
64. Michiels B, Molenberghs G, Bijnens L, Vangeneugden T, Thijs H. Selection models and pattern-mixture models to analyse longitudinal quality of life data subject to drop out. Stat Med 2002, 21:1023-1041. 10.1002/sim.1064, 11933032.
65. Proschan MA, Waclawiw MA. Practical guidelines for multiplicity adjustment in clinical trials. Controlled Clin Trials 2000, 21:527-539. 10.1016/S0197-2456(00)00106-9, 11146147.
66. Elkin I. A major dilemma in psychotherapy outcome research: disentangling therapists from therapies. Clin Psychol-Sci Prac 1999, 6:10-32.
67. Eldridge SM, Ashby D, Kerry S. Sample size for cluster randomized trials: effect of coefficient of variation of cluster size and analysis method. Int J Epidemiol 2006, 35:1292-1300. 10.1093/ije/dyl129, 16943232.
68. Kerry SM, Bland JM. Unequal cluster sizes for trials in English and welsh general practice: implications for sample size calculations. Stat Med 2001, 20:377-390. 10.1002/1097-0258(20010215)20:3<377::AID-SIM799>3.0.CO;2-N, 11180308.
69. Assmann SF, Pocock SJ, Enos LE, Kasten LE. Subgroup analysis and other (mis)uses of baseline data in clinical trials. Lancet 2000, 355:1064-1069. 10.1016/S0140-6736(00)02039-0, 10744093.
70. Altman DG. Comparability of randomised groups. J Royal Stat Soc Series D 1985, 34:125-136.
71. Senn S. Testing for baseline balance in clinical trials. Stat Med 1993, 13:1715-1726.
72. Fitzmaurice GM, Laird NM, Zahner GE, Daskalakis C. Bivariate logistic regression analysis of childhood psychopathology ratings using multiple informants. Am J Epidemiol 1995, 142:1194-1203.
73. Have TRT, Morabia A. Mixed effects models with bivariate and univariate association parameters for longitudinal bivariate binary response data. Biometrics 1999, 55:85-93. 10.1111/j.0006-341X.1999.00085.x, 11318182.
74. Ribaudo HJ, Thompson SG. The analysis of repeated multivariate binary quality of life data: A hierarchical model approach. Stat Methods Med Res 2002, 11:69-83. 10.1191/0962280202sm272ra, 11923995.
75. Curtis L. Unit Costs of Health and Social Care 2008, PSSRU: Canterbury.
76. Dolan P, Gudex C, Kind P, Williams A. A Social Tariff for EuroQol: Results from a UK general population survey 1995, York: University of York.
77. Briggs AH. A Bayesian approach to stochastic cost-effectiveness analysis. Health Econ 1999, 8:257-261. 10.1002/(SICI)1099-1050(199905)8:3<257::AID-HEC427>3.0.CO;2-E, 10348420.
78. Microsoft Access. [http://office.microsoft.com/en-gb/], Microsoft.
79. StataCorp Stata Statistical Software: Release 10 2007, College Station, TX: StataCorp LP, StataCorp.
80. Rasbash J, Charlton C, Browne WJ, Healy M, Cameron B. MLwiN Version 2.02 2005, Centre for Multilevel Modelling: University of Bristol.