Establishing long-term efficacy in chronic disease: Use of recursive partitioning and propensity score adjustment to estimate outcome in MS

PLoS ONE

Volumn 6, Issue 11, 2011, Pages

  Goodin, Douglas S ^a   Jones, Jason ^b   Li, David ^c   Traboulsee, Anthony ^c   Reder, Anthony T ^d   Beckmann, Karola ^e   Konieczny, Andreas ^f   Knappertz, Volker ^g  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b INTERMOUNTAIN HEALTHCARE   (United States)

^c UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^d UNIVERSITY OF CHICAGO   (United States)

^e BAYER PHARMA AG   (Germany)

^f Lampe and Company   (Germany)

^g BAYER CORPORATION   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA1A INTERFERON; GLATIRAMER; INTERFERON BETA SERINE; BETA INTERFERON;

ADULT; ALGORITHM; ARTICLE; CONTROLLED STUDY; DISABILITY; DISEASE DURATION; DISEASE SEVERITY; DRUG EXPOSURE; EPIDEMIOLOGY; EXPANDED DISABILITY STATUS SCALE; FEMALE; FOLLOW UP; HUMAN; MAJOR CLINICAL STUDY; MALE; MULTIPLE SCLEROSIS; OUTCOME ASSESSMENT; PREDICTIVE VALUE; PROGNOSIS; PROPENSITY SCORE; RANDOMIZED CONTROLLED TRIAL (TOPIC); RECURSIVE PARTITIONING ALGORITHM; SENSITIVITY ANALYSIS; CHRONIC DISEASE; METHODOLOGY; PROPORTIONAL HAZARDS MODEL; TIME;

ADULT; ALGORITHMS; CHRONIC DISEASE; FEMALE; FOLLOW-UP STUDIES; HUMANS; INTERFERON-BETA; MALE; MULTIPLE SCLEROSIS; OUTCOME ASSESSMENT (HEALTH CARE); PROPENSITY SCORE; PROPORTIONAL HAZARDS MODELS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; TIME FACTORS;

EID: 82355181854     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0022444     Document Type: Article

References (45)

1. Goodin DS, Frohman EM, Garmany GP, Halper J, Likosky WH, et al. (2002) Disease modifying therapies in multiple sclerosis: report of the Therapeutics and Technology Assessment Subcommittee of the American Academy of Neurology and the MS Council for Clinical Practice Guidelines. Neurology 58: 169-178.
2. 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.
3. Paty DW, Li DK, UBC MS/MRI Study Group, the IFNB Multiple Sclerosis Study Group (1993) Interferon beta-1b is effective in relapsing-remitting multiple sclerosis. II. MRI analysis results of a multicenter, randomized, double-blind, placebo-controlled trial. Neurology 43: 662-667.
4. Frohman EM, Havrdova E, Lublin F, Barkhoff F, Achiron A, et al. (2006) Most patients with multiple sclerosis or a clinically isolated demyelinating syndrome should be treated at the time of diagnosis. Arch Neurol 63: 614-619.
5. European Study Group on interferon beta-1b in secondary progressive MS (1998) Placebo-controlled multicentre randomised trial of interferon beta-1b in treatment of secondary progressive multiple sclerosis. Lancet 352: 1491-1497.
6. Kappos L, Polman CH, Freedman MS, Edan G, Hartung HP, et al. (2006) Treatment with interferon beta-1b delays conversion to clinically definite and McDonald MS in patients with clinically isolated syndromes. Neurology 67: 242-1249.
7. Jacobs LD, Beck RW, Simon JH, Kinkel RP, Brownscheidl CM, et al. (2000) Intramuscular interferon beta-1a therapy initiated during a first demyelinating event in multiple sclerosis. CHAMPS Study Group. N Engl J Med 343: 898-904.
8. Comi G, Filippi M, Barkhof F, Druelli L, Early Treatment of Multiple Sclerosis Study Group (2001) Effect of early interferon treatment on conversion to definite multiple sclerosis: a randomised study. Lancet 357: 1576-1582.
9. Mikol DD, Barkhof F, Chang P, Coyle PK, Schwid SR, et al. (2008) Comparison of subcutaneous interferon beta-1a with glatiramer acetate in patients with relapsing multiple sclerosis (the REbif vs Glatiramer Acetate in Relapsing MS Disease [REGARD] study): a multicentre, randomised, parallel, open-label trial. Lancet Neurol 7: 903-914.
10. O'Connor P, Fillipi M, Arnason B, Comi G, Cook S, et al. (2009) 250 or 500 ug Interferon beta-1b versus 20 mg glatiramer Acetate in relapsing-remitting multiple sclerosis: a prospective, randomized, multicentre study. Lancet Neurol 8: 889-897.
11. Comi G, Martinelli V, Rodegher M, Moiola L, Bajenaru O, et al. (2009) Effect of glatiramer acetate on conversion to clinically definite multiple sclerosis in patients with clinically isolated syndrome (PreCISe study): a randomised, double-blind, placebo-controlled trial. Lancet 374: 1503-11.
12. Goodin DS, Bates D, (2009) Treatment of early multiple sclerosis: the value of treatment initiation after a first clinical episode. Multiple Sclerosis 15: 1133-1134.
13. Ebers G, Reder A, Traboulsee A, Langdon D, Goodin DS, et al. (2009) Long-term follow-up of the original interferon-β1b trial in multiple sclerosis: design and lessons from the 16-year observational study. Clinical Therapeutics 31: 1724-1736.
14. Reder AT, Ebers GC, Traboulsee A, Li D, Langdon D, et al. Cross-sectional study assessing long-term safety of interferon-β-1b for relapsing-remitting MS. Neurology 2010; 74: 1877-1885.
15. Langdon D, Reder AT, Traboulsee A, Li D, Goodin DS, et al. Early disability and MRI predicts cognitive status of multiple sclerosis patients after 16 years (submitted), 2011.
16. Goodin DS, Traboulsee A, Knappertz V, Reder AT, Li D, et al. Relationship between early clinical characteristics and long-term disability outcomes: 16-year follow-up of the pivotal IFNB-1b study in MS. (submitted), 2011.
17. Roxburgh RSHR, Seaman SR, Masterman T, Hensiek AE, Sawcer SJ, et al. (2005) Multiple sclerosis severity score: Using disability and disease duration to rate disease severity. Neurology 64: 1144-1151.
18. Sclar DA, Chin A, Skaer TL, Okamoto MP, Nakahiro RK, et al. (1991) Effect of health education in promoting prescription refill compliance among patients with hypertension. Clin Ther 13: 489-495.
19. Sikka R, Xia F, Aubert RE, (2005) Estimating medication persistency using administrative claims data. Am J Manag Care 11: 449-457.
20. Siannis F, Copas J, Lu G, (2005) Sensitivity analysis for informative-censoring in parametric survival models. Biostatistics 6: 77-91.
21. Hothorn T, Hornik K, Zeileis A, (2006) Unbiased recursive partitioning: A conditional inference framework. J Computational Graphical Statistics 15: 651-674.
22. Ciampi A, Hogg SA, McKinney S, Thiffault J, (1988) RECPAM: a computer program for recursive partition and amalgamation for censored survival data and other situations frequently occurring in biostatistics. I Methods and program features. Computer Methods Programs Biomed 26: 239-256.
23. Bass FM, (1969) A new product growth for model consumer durables. Management Sci 15: 215-227.
24. Party Package (2008) Available: http://cran.rproject.org/web/packages/party/vignettes/party.pdf. Accessed 7/16/10.
25. R Platform (2008) Available: http://www.r-project.org/ v2.4.1. Accessed 7/16/10.
26. Ernst MD, (2004) Permutation methods: A basis for exact inference. Stat Sci 19: 676-685.
27. Senn S, Graf E, Caputo A, (2007) Stratification for the propensity score compared with linear regression techniques to assess the effect of treatment or exposure. Stat Med 26: 5529-5544.
28. Rosenbaum PR, Rubin DB, (1983) The central role of the propensity score in observational studies for causal effects. Biometrica 70: 41-55.
29. D'Agostino RB, (1998) Tutorial in biostatistics: Propensity score methods for bias reduction in the comparison of a treatment to a non-randomized control group. Stat Med 17: 2265-2281.
30. Lunceford JK, Davidian M, (2004) Stratification and weighting via the propensity score in estimation of causal treatment effects: A comparative study. Stat Med 23: 2937-2960.
31. Trojano M, Pellegrini F, Fuiani A, Paolicelli D, Zipoli V, et al. (2007) New natural history of interferon-beta-treated relapsing multiple sclerosis. Ann Neurol 61: 300-306.
32. Novikov I, Kalter-Leibovici O, (2007) Analytic approaches to observational studies with treatment-selection bias. JAMA 297: 2077-2078.
33. Heagerty PJ, Zheng Y, (2005) Survival model predictive accuracy and ROC curves. Biometrics 61: 92-105.
34. Johnson KP, Brooks BR, Cohen JA, Ford CC, Goldstein J, et al. (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. The Copolymer 1 Multiple Sclerosis Study Group. Neurology 45: 1268-1276.
35. Jacobs LD, Cookfair DL, Rudick RA, Herndon RM, Richert JR, et al. (1996) Intramuscular interferon beta-1a for disease progression in relapsing multiple sclerosis. The Multiple Sclerosis Collaborative Research Group (MSCRG). Ann Neurol 39: 285-294.
36. PRISMS (Prevention of Relapses and Disability by Interferon beta-1a Subcutaneously in Multiple Sclerosis) Study Group (1998) Randomised double-blind placebo-controlled study of interferon beta-1a in relapsing/remitting multiple sclerosis. Lancet 352: 1498-1504.
37. The Once Weekly Interferon for MS Study Group (1999) Evidence of interferon beta-1a dose response in relapsing-remitting MS: the OWIMS Study. Neurology 53: 679-686.
38. Panitch H, Goodin DS, Francis G, Chiang P, Coyle PK, et al. (2002) Randomized, comparative study of interferon beta-1a treatment regimens in MS: The EVIDENCE Trial. Neurology 59: 1496-1506.
39. Durelli L, Verdun E, Barbero P, Bergli M, Versino E, et al. (2002) Every-other-day interferon beta-1b versus once-weekly interferon beta-1a for multiple sclerosis: results of a 2-year prospective randomised multicentre study (INCOMIN). Lancet 359: 1453-1460.
40. Polman CH, O'Connor PW, Havrdova E, Hutchinson M, Kappos L, et al. (2006) A randomized, placebo-controlled trial of natalizumab for relapsing multiple sclerosis. N Engl J Med 354: 899-910.
41. Rudick RA, Stuart WH, Calabresi PA, Confavreux C, Giovannoni G, et al. (2006) Natalizumab plus interferon beta-1a for relapsing multiple sclerosis. N Engl J Med 354: 911-923.
42. Coles AJ, Compston DAS, Selmaj KW, Lake SL, the CAMMS223 Trial Investigators (2008) Alemtuzumab vs. Interferon Beta-1a in early multiple sclerosis. N Engl J Med 359: 1786-1801.
43. Giovannoni G, Comi G, Cook S, Rammohan K, the CLARITY Study Group (2010) A Placebo-Controlled Trial of Oral Cladribine for Relapsing Multiple Sclerosis. N Engl J Med 362: 416-426.
44. Cohen JA, Barkof F, Comi G, Hartung HP, the TRANSFORMS Study Group (2010) Oral fingolimod or intramuscular interferon for relapsing multiple sclerosis. N Engl J Med 62: 402-415.
45. Kappos L, Radue EW, O'Connor P, Polman C, the FREEDOMS Study Group (2010) A Placebo-Controlled Trial of Oral Fingolimod in Relapsing Multiple Sclerosis. N Engl J Med 362: 387-401.