Disease-modifying therapy in MS: A critical review of the literature. Part I: Analysis of clinical trial errors

Journal of Neurology, Supplement

Volumn 251, Issue 5, 2004, Pages

  Goodin, Douglas S ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Analysis errors; Clinical trial; Disease modifying therapy; Multiple sclerosis

Indexed keywords

BETA1A INTERFERON; GLATIRAMER;

CLINICAL TRIAL; CONFERENCE PAPER; DATA ANALYSIS; EPIDEMIOLOGICAL DATA; HUMAN; MEDICAL LITERATURE; MULTIPLE SCLEROSIS; NUCLEAR MAGNETIC RESONANCE IMAGING; PRIORITY JOURNAL; STATISTICS;

BIAS (EPIDEMIOLOGY); CLINICAL TRIALS; DATA INTERPRETATION, STATISTICAL; HUMANS; META-ANALYSIS; MULTIPLE SCLEROSIS; REVIEW LITERATURE;

EID: 5644300578     PISSN: 09391517     EISSN: None     Source Type: Journal    
DOI: 10.1007/s00415-004-1503-z     Document Type: Conference Paper

References (34)

1. Goodman SN (1993) Reviews and commentary: p values, hypothesis tests, and likelihood: implications for epidemiology of a neglected historical debate. Am J Epidemiol 137:485-496
2. Bellhouse DR (1993) Invited commentary: p values, hypothesis tests, and likelihood. Am J Epidemiol 137:497-499
3. Goodman SN (1993) Author's response to: Invited commentary: p values, hypothesis tests, and likelihood. Am J Epidemiol 137:500-501
4. O'Brien PC (1984) Procedures for comparing samples with multiple endpoints. Biometrics 40:1079-1087
5. Thomas DC, Siemiatycki J, Dewar R, Robins J, Goldberg M, Armstrong BG (1985) The problem of multiple inference in studies designed to generate hypotheses. Am J Epidemiol 122:1080-1095
6. Rothman KJ (1986) Significance questing. Ann Int Med 105:445-447
7. Tandon PK (1990) Applications of global statistics in analyzing quality of life data. Stat Med 9:819-827
8. Hochberg Y, Benjamini Y (1990) More powerful procedures for multiple significance testing. Stat Med 9:811-818
9. Cohen J (1988) Statistical power analysis for the behavioral sciences, 2nd edn. Lawrence Erlbaum Associates, Hillsdale, New Jersey
10. Johnson KP, Brooks BR, Cohen JA, Ford CC, Goldstein J, Lisak RP, Myers LW, Panitch HS, Rose JW, Schiffer RB, Vollmer T, Weiner LP, Wolinsky JS and the Copolymer Multiple Sclerosis Study Group (1995) Copolymer 1 reduces relapse rate and improves disability in relapsing-remitting multiple sclerosis: results of a phase III multicenter, double-blind, placebo-controlled trial. Neurology 45:1268-1276
11. Johnson KP, Brooks BR, Cohen JA, Ford CC, Goldstein J, Lisak RP, Myers LW, Panitch HS, Rose JW, Schiffer RB, Vollmer T, Weiner LP, Wolinsky JS and the Copolymer Multiple Sclerosis Study Group (1998) Extended use of glatiramer acetate (Copaxone) is well tolerated and maintains its clinical effect on multiple sclerosis relapse rate and degree of disability. Neurology 50:701-708
12. Summary basis for approval (1996) FDA official document for license of glatiramer acetate (Copaxone). Teva Pharmaceuticals USA, NDA 20-622
13. Boneschi FM, Rovaris M, Johnson KP, Miller A, Wolinsky JS, Ladkani D, Shifroni G, Comi G, Filippi M (2003) Effects of glatiramer acetate on relapse rate and accumulated disability in multiple sclerosis: a meta-analysis of three double-blind, randomized, placebo-controlled clinical trials. Mult Scler 9:349-355
14. Bornstein MB, Miller A, Slagle S, Weitzman M, Crystal H, Drexler E, Keilson M, Merriam A, Wassertheil-Smoller S, Spada V, et al. (1987) A pilot trial of COP 1 in exacerbating-remitting multiple sclerosis. N Engl J Med 317:408-414
15. Comi G, Filippi M, Wolinsky JS (2001) European/Canadian multicenter, double-blind, randomized, placebo-controlled study of the effects of glatiramer acetate on magnetic resonance imaging-measured disease activity and burden in patients with relapsing multiple sclerosis. Ann Neurol 49:290-297
16. Rudick RA, Fisher E, Lee JC, Simon J, Jacobs L and the Multiple Sclerosis Collaborative Research Group (1999) Use of the brain parenchymal fraction to measure whole brain atrophy in relapsing-remitting MS. Neurology 53:1698-1704
17. rd, Priore RL, Pullicino PM, Scherokman BJ, Whitham RH, et al. and the Multiple Sclerosis Collaborative Research Group (MSCRG) (1996) Intramuscular interferon beta-1a for disease progression in exacerbating-remitting multiple sclerosis. Ann Neurol 39:285-294
18. Rudick RA, Cookfair DL, Griffin J, Hauser S, Piantadosi S (2003) Interferons in relapsing remitting multiple sclerosis. Lancet 361:1824
19. Summary basis of approval (1995) FDA official document for license of interferon beta-1a (Avonex). Biogen Inc, on file at www.fda.gov/cber/products/ ifnbbio051796.htm
20. Beck RW, Cleary PA, Anderson MM Jr, Kellner JL, Shults WT, Kaufman DI, Buckley EG, Corbett JJ, Kupersmith MJ, Miller NR, et al. (1992) A randomized controlled trial of corticosteroids in the treatment of acute optic neuritis. N Engl J Med 326:581-588
21. Beck RW, Cleary PA, Trobe JD, Kaufman DI, Kupersmith MJ, Paty DW, Brown CH (1993) The effect of corticosteroids for acute optic neuritis on the subsequent development of multiple sclerosis. N Engl J Med 329:1764-1769
22. Cleary PA, Beck RW, Anderson MM Jr, Kenny DJ, Backhand JY, Gilbert PR (1993) Design, methods, and conduct of the optic neuritis treatment trial. Control Clin Trials 14:123-142
23. Goodin DS (1999) Perils and pitfalls in the interpretation of clinical trials: a reflection on the recent experience in multiple sclerosis. Neuroepidemiology 18:53-63
24. Johnson KP, Brooks BR, Ford CC, Goodman A, Guarnaccia J, Lisak RP, Myers LW, Panitch HS, Pruitt A, Rose JW, Kachuck N, Wolinsky JS and the Copolymer 1 Multiple Sclerosis Study Group (2000) Sustained clinical benefits of glatiramer acetate in relapsing multiple sclerosis patients observed for 6 years. Mult Scler 6:255-266
25. Weinshenker BG, Rice GP, Noseworthy JH, Carriere W, Baskerville J, Ebers GC (1991) The natural history of multiple sclerosis: a geographically based study. 3. Multivariate analysis of predictive factors and models of outcome. Brain 114:1045-1056
26. Johnson KP, Panitch HS, Ford CC, Lisak RP and the Copaxone Study Group (2004) Long-term slowing of disability progression in patients receiving continuous glatiramer acetate compared with those withdrawing from therapy: 10 year results from an ongoing trial. Neurology 62(Suppl 5):A180.3
27. Pittock SJ, Mayr WT, McClelland RL, Jorgensen NW, Weigand SD, Noseworthy JH, Weinshenker BG, Rodriguez M (2004) Change in MS-related disability in a population-based cohort: a 10-year follow-up study. Neurology 62:51-59
28. Kümpfel T, Schumann E, Bergh FT, Gottschalk M, Auer D, Holsboer F, Trenkwalder C (2001) Monthly high-dose methylprednisolone pulse therapy in patients with multiple sclerosis: an MRI controlled study with single crossover design. Neurology 56(Suppl 3):A76
29. The IFNB Multiple Sclerosis Study Group (1993) Interferon beta-1b is effective in relapsing-remitting multiple sclerosis. I. Clinical results of a multicenter, randomized, double-blind, placebo-controlled trial. Neurology 43:655-661
30. PRISMS Study Group (1998) Randomized double-blind placebo-controlled study of Interferon β-1a in relapsing/remitting multiple sclerosis. Lancet 352:1498-1504
31. Hartung HP, Gonsette R, Konig N, Kwiecinski H, Guseo A, Morrissey SP, Krapf H, Zwingers T and the Mitoxantrone in Multiple Sclerosis Study Group (MIMS) (2002) Mitoxantrone in progressive multiple sclerosis (MS): a placebo-controlled, double-blind, randomised multicenter study. Lancet 360:2018-2025
32. Panitch H, Goodin DS, Francis G, Chang P, Coyle PK, O'Connor P, Monaghan E, Li D, Weinshenker B, the EVIDENCE Study Group and the UBC MS/MRI Research Group (2002) Randomized, comparative study of interferon beta-1a treatment regimens in MS: the EVIDENCE trial. Neurology 59:1496-1506
33. Schwid SR, Thorpe J, Sharief M, Sandberg-Wollheim M, Rammohan K, Wendt J, Panitch H, Goodin DS, Li D, Chang P, Francis G, the EVIDENCE Study Group and the UBC MS/MRI Research Group. Enhanced benefit of increasing interferon-1a dose and frequency in relapsing MS: the EVIDENCE trial. Arch Neurol (in press)
34. Ackerman KD, Heyman R, Rabin BS, Anderson BP, Houck PR, Frank E, Baum A (2002) Stressful life events precede exacerbations of multiple sclerosis. Psychosom Med 64:916-920