Magnetic resonance active lesions as individual-level surrogate for relapses in multiple sclerosis

Multiple Sclerosis Journal

Volumn 17, Issue 5, 2011, Pages 541-549

  Sormani, Maria Pia ^a   Stubinski, Bettina ^b   Cornelisse, Peter ^b   Rocak, Sanda ^b   Li, David ^c   De Stefano, Nicola ^d  

^a UNIVERSITY OF GENOA   (Italy)

^b MERCK SERONO S A   (Switzerland)

^c UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^d UNIVERSITY OF SIENA   (Italy)

Author keywords

Interferon (IFN); Surrogacy; Treatment effect

Indexed keywords

BETA1A INTERFERON; BETA INTERFERON; IMMUNOLOGIC FACTOR;

ARTICLE; CONTROLLED CLINICAL TRIAL (TOPIC); FOLLOW UP; HUMAN; MAJOR CLINICAL STUDY; MULTIPLE SCLEROSIS; NUCLEAR MAGNETIC RESONANCE IMAGING; RELAPSE; REMISSION; TREATMENT OUTCOME; TREATMENT RESPONSE; VALIDATION PROCESS; BRAIN; CHI SQUARE DISTRIBUTION; DRUG EFFECT; META ANALYSIS; PATHOLOGY; PREDICTIVE VALUE; RANDOMIZED CONTROLLED TRIAL (TOPIC); RECURRENT DISEASE; REPRODUCIBILITY; TIME;

BRAIN; CHI-SQUARE DISTRIBUTION; HUMANS; IMMUNOLOGIC FACTORS; INTERFERON-BETA; MAGNETIC RESONANCE IMAGING; MULTIPLE SCLEROSIS, CHRONIC PROGRESSIVE; MULTIPLE SCLEROSIS, RELAPSING-REMITTING; PREDICTIVE VALUE OF TESTS; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RECURRENCE; REPRODUCIBILITY OF RESULTS; TIME FACTORS; TREATMENT OUTCOME;

EID: 80053424031     PISSN: 13524585     EISSN: 14770970     Source Type: Journal    
DOI: 10.1177/1352458510391837     Document Type: Article

References (28)

1. IFNB Multiple Sclerosis Study Group. Interferon beta-1b is effective in relapsing-remitting multiple sclerosis. I Clinical results of a multicenter, randomized, doubleblind, placebo-controlled trial. Neurology 1993; 43: 655-661.
2. Jacobs LD, Cookfair DL, Rudick RA, et al. Intramuscular interferon beta-1a for disease progression in relapsing multiple sclerosis. Ann Neurol 1996; 39: 285-294. (Pubitemid 26100751)
3. PRISMS (Prevention of Relapses and disability by Interferon beta-1a Subcutaneously in Multiple Sclerosis) Study Group. Randomised double-blind placebocontrolled study of interferon beta-1a in relapsing/remitting multiple sclerosis. Lancet 1998; 352: 1498-1504.
4. Neema M, Stankiewicz J, Arora A, et al. T1- and T2-based MRI measures of diffuse gray matter and white matter damage in patients with multiple sclerosis. J Neuroimaging 2007; 17(Suppl. 1): 16S-21S. (Pubitemid 46558263)
5. Rovaris M, Judica E, Sastre-Garriga J, et al. Large-scale, multicentre, quantitative MRI study of brain and cord damage in primary progressive multiple sclerosis. Mult Scler 2008; 14: 455-464. (Pubitemid 351943857)
6. Barkhof F, Scheltens P, Frequin S, et al. Relapsing- remitting multiple sclerosis: sequential enhanced MR imaging versus clinical findings in determining disease activity. Am J Roentgenol 1992; 159: 1041-1047.
7. Harris JO, Frank JA, Patronas N, McFarlin DE and McFarland HF. Serial gadolinium-enhanced magnetic resonance imaging scans in patients with early, relapsing- remitting multiple sclerosis: implications for clinical trials and natural history. Ann Neurol 1991; 29: 548-555.
8. Li DK, Li MJ, Traboulsee A, Zhao G, Riddehough A and Paty D. The use of MRI as an outcome measure in clinical trials. Adv Neurol 2006; 98: 203-226.
9. Miller DH. Multiple sclerosis: use of MRI in evaluating new therapies. Semin Neurol 1998; 18: 317-325. (Pubitemid 28488948)
10. Thompson AJ, Miller D, Youl B, et al. Serial gadoliniumenhanced MRI in relapsing/remitting multiple sclerosis of varying disease duration. Neurology 1992; 42: 60-63.
11. Sormani MP, Bonzano L, Roccatagliata L, Cutter GR, Mancardi GL and Bruzzi P. Magnetic resonance imaging as a potential surrogate for relapses in multiple sclerosis: a meta-analytic approach. Ann Neurol 2009; 65: 268-275.
12. Prentice RL. Surrogate endpoints in clinical trials: definition and operational criteria. Stat Med 1989; 8: 431-440. (Pubitemid 19105891)
13. Sormani MP, Bruzzi P, Comi G and Filippi M. MRI metrics as surrogate markers for clinical relapse rate in relapsing-remitting MS patients. Neurology 2002; 58: 417-421. (Pubitemid 34150916)
14. Sormani MP, Bruzzi P, Beckmann K, et al. MRI metrics as surrogate endpoints for EDSS progression in SPMS patients treated with IFN beta-1b. Neurology 2003; 60: 1462-1466. (Pubitemid 36554807)
15. Daumer M, Neuhaus A, Morrissey S, Hintzen R and Ebers GC. MRI as an outcome in multiple sclerosis clinical trials. Neurology 2009; 72: 705-711.
16. Petkau J, Reingold SC, Held U, et al. Magnetic resonance imaging as a surrogate outcome for multiple sclerosis relapses. Mult Scler 2008; 14: 770-778.
17. SPECTRIMS Study Group. Randomized controlled trial of interferon-beta-1a in secondary progressive MS: clinical results. Neurology 2001; 56: 1496-1504.
18. Li DK and Paty DW for the UBC MS/MRI Analysis Research Group, PRISMS Study Group. Magnetic resonance imaging results of the PRISMS trial: a randomized, double-blind, placebo-controlled study of interferonbeta1a in relapsing-remitting multiple sclerosis. Ann Neurol 1999; 46: 197-206. (Pubitemid 29382876)
19. Li DK, Zhao GJ and Paty DW for the The UBC MS/ MRI Study Group, The SPECTRIMS Study Group. Randomized controlled trial of interferon-beta-1a in secondary progressive MS: MRI results. Neurology 2001; 56: 1505-1513. (Pubitemid 32532262)
20. Lin DY, Fleming TR and De Gruttola V. Estimating the proportion of treatment effect explained by a surrogate marker. Stat Med 1997; 16: 1515-1527. (Pubitemid 27322655)
21. Sormani MP, Bruzzi P, Miller DH, Gasperini C, Barkhof F and Filippi M. Modelling MRI enhancing lesion counts in multiple sclerosis using a negative binomial model: implications for clinical trials. J Neurol Sci 1999; 163: 74-80. (Pubitemid 29155420)
22. Sormani MP, Bruzzi P, Rovaris M, et al. Modelling new enhancing MRI lesion counts in multiple sclerosis. Mult Scler 2001; 7: 298-304. (Pubitemid 33026255)
23. Pelletier J, Audoin B, Reuter F and Ranjeva J. Plasticity in MS: from functional imaging to rehabilitation. Int MS J 2009; 16: 26-31.
24. Burzykowski T, Molenberghs G and Buyse M. The evaluation of surrogate endpoints. New York: Springer, 2005.
25. Held U, Heigenhauser L, Shang C, Kappos L and Polman C. Predictors of relapse rate in MS clinical trials. Neurology 2005; 65: 1769-1773.
26. Molenberghs G, Buyse M, Geys H, Renard D, Burzykowski T and Alonso A. Statistical challenges in the evaluation of surrogate endpoints in randomized trials. Control Clin Trials 2002; 23: 607-625. (Pubitemid 36005472)
27. Berger VW. Does the Prentice criterion validate surrogate endpoints? Stat Med 2004; 23: 1571-1578. (Pubitemid 38660409)
28. Freedman LS, Graubard BI and Schatzkin A. Statistical validation of intermediate endpoints for chronic diseases. Stat Med 1992; 11: 167-178.