publication . Article . 2016

Development of a Sensitive Outcome for Economical Drug Screening for Progressive Multiple Sclerosis Treatment

Peter Kosa; Danish Ghazali; Makoto Tanigawa; Chris Barbour; Irene Cortese; William Kelley; Blake Snyder; Joan Ohayon; Kaylan Fenton; Tanya Lehky; ...
Open Access English
  • Published: 01 Aug 2016 Journal: Frontiers in Neurology, volume 7 (issn: 1664-2295, eissn: 1664-2295, Copyright policy)
  • Publisher: Frontiers Media S.A.
Abstract
Therapeutic advance in progressive multiple sclerosis (MS) has been very slow. Based on the transformative role magnetic resonance imaging (MRI) contrast-enhancing lesions had on drug development for relapsing-remitting MS, we consider the lack of sensitive outcomes to be the greatest barrier for developing new treatments for progressive MS. The purpose of this study was to compare 58 prospectively-acquired candidate outcomes in the real-world situation of progressive MS trials, to select and validate the best-performing outcome.The one year pre-treatment period of adaptively-designed IPPoMS (ClinicalTrials.gov #NCT00950248) and RIVITaLISe (ClinicalTrials.gov #N...
Subjects
free text keywords: clinical trial, Neurology. Diseases of the nervous system, outcome measure, outcome measures, multiple sclerosis, Neuroscience, RC346-429, composite scale, Progressive multiple sclerosis, quantitative MRI, disability scale, progressive MS, clinical trials, Original Research
Funded by
NIH| Comprehensive multimodal analysis of patients with neuroimmunological diseases
Project
  • Funder: National Institutes of Health (NIH)
  • Project Code: 1ZIANS003055-07
  • Funding stream: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
,
NIH| From therapeutic mechanisms to unraveling the pathophysiology of MS
Project
  • Funder: National Institutes of Health (NIH)
  • Project Code: 1ZIANS003056-08
  • Funding stream: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
40 references, page 1 of 3

1 Wolinsky JS Narayana PA O’Connor P Coyle PK Ford C Johnson K Glatiramer acetate in primary progressive multiple sclerosis: results of a multinational, multicenter, double-blind, placebo-controlled trial. Ann Neurol (2007) 61(1):14–24.10.1002/ana.21079 17262850 [PubMed] [DOI]

2 Altmann DR Jasperse B Barkhof F Beckmann K Filippi M Kappos LD Sample sizes for brain atrophy outcomes in trials for secondary progressive multi ple sclerosis. Neurology (2009) 72(7):595–601.10.1212/01.wnl.0000335765.55346.fc 19005170 [OpenAIRE] [PubMed] [DOI]

3 Harrison DM Caffo BS Shiee N Farrell JA Bazin PL Farrell SK Longitudinal changes in diffusion tensor-based quantitative MRI in multiple sclerosis. Neurology (2011) 76(2):179–86.10.1212/WNL.0b013e318206ca61 21220722 [OpenAIRE] [PubMed] [DOI]

4 Frost C Kenward MG Fox NC. Optimizing the design of clinical trials where the outcome is a rate. Can estimating a baseline rate in a run-in period increase efficiency?Stat Med (2008) 27(19):3717–31.10.1002/sim.3280 18484598 [PubMed] [DOI]

5 Smith SM Zhang Y Jenkinson M Chen J Matthews PM Federico A Accurate, robust, and automated longitudinal and cross-sectional brain change analysis. Neuroimage (2002) 17(1):479–89.10.1006/nimg.2002.1040 12482100 [PubMed] [DOI]

6 Komori M Lin YC Cortese I Blake A Ohayon J Cherup J Insufficient disease inhibition by intrathecal rituximab in progressive multiple sclerosis. Ann Clin Transl Neurol (2016) 3(3):166–79.10.1002/acn3.293 27042677 [OpenAIRE] [PubMed] [DOI]

7 SAS Institute Inc.SAS/STAT®9.22 User’s Guide. Cary, NC: SAS Institute (2010).

8 Kurtzke JF. Rating neurologic impairment in multiple sclerosis: an expanded disability status scale (EDSS). Neurology (1983) 33(11):1444–52.10.1212/WNL.33.11.1444 6685237 [PubMed] [DOI]

9 Sipe JC Knobler RL Braheny SL Rice GP Panitch HS Oldstone MB. A neurologic rating scale (NRS) for use in multiple sclerosis. Neurology (1984) 34(10):1368–72.10.1212/WNL.34.10.1368 6541311 [PubMed] [DOI]

10 Fischer JS Rudick RA Cutter GR Reingold SC. The multiple sclerosis functional composite measure (MSFC): an integrated approach to MS clinical outcome assessment. National MS society clinical outcomes assessment task force. Mult Scler (1999) 5(4):244–50.10.1177/135245859900500409 10467383 [PubMed] [DOI]

11 Hallett M. Transcranial magnetic stimulation: a primer. Neuron (2007) 55(2):187–99.10.1016/j.neuron.2007.06.026 17640522 [OpenAIRE] [PubMed] [DOI]

12 Duan Q van Gelderen P Duyn J. Improved Bloch-Siegert based B1 mapping by reducing off-resonance shift. NMR Biomed (2013) 26(9):1070–8.10.1002/nbm.2920 23355474 [OpenAIRE] [PubMed] [DOI]

13 Deoni SC. High-resolution T1 mapping of the brain at 3T with driven equilibrium single pulse observation of T1 with high-speed incorporation of RF field inhomogeneities (DESPOT1-HIFI). J Magn Reson Imaging (2007) 26(4):1106–11.10.1002/jmri.21130 17896356 [PubMed] [DOI]

14 Shiee N Bazin PL Ozturk A Reich DS Calabresi PA Pham DL. A topology-preserving approach to the segmentation of brain images with multiple sclerosis lesions. Neuroimage (2010) 49(2):1524–35.10.1016/j.neuroimage.2009.09.005 19766196 [OpenAIRE] [PubMed] [DOI]

15 Chang LC Jones DK Pierpaoli C. RESTORE: robust estimation of tensors by outlier rejection. Magn Reson Med (2005) 53(5):1088–95.10.1002/mrm.20426 15844157 [PubMed] [DOI]

40 references, page 1 of 3
Abstract
Therapeutic advance in progressive multiple sclerosis (MS) has been very slow. Based on the transformative role magnetic resonance imaging (MRI) contrast-enhancing lesions had on drug development for relapsing-remitting MS, we consider the lack of sensitive outcomes to be the greatest barrier for developing new treatments for progressive MS. The purpose of this study was to compare 58 prospectively-acquired candidate outcomes in the real-world situation of progressive MS trials, to select and validate the best-performing outcome.The one year pre-treatment period of adaptively-designed IPPoMS (ClinicalTrials.gov #NCT00950248) and RIVITaLISe (ClinicalTrials.gov #N...
Subjects
free text keywords: clinical trial, Neurology. Diseases of the nervous system, outcome measure, outcome measures, multiple sclerosis, Neuroscience, RC346-429, composite scale, Progressive multiple sclerosis, quantitative MRI, disability scale, progressive MS, clinical trials, Original Research
Funded by
NIH| Comprehensive multimodal analysis of patients with neuroimmunological diseases
Project
  • Funder: National Institutes of Health (NIH)
  • Project Code: 1ZIANS003055-07
  • Funding stream: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
,
NIH| From therapeutic mechanisms to unraveling the pathophysiology of MS
Project
  • Funder: National Institutes of Health (NIH)
  • Project Code: 1ZIANS003056-08
  • Funding stream: NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
40 references, page 1 of 3

1 Wolinsky JS Narayana PA O’Connor P Coyle PK Ford C Johnson K Glatiramer acetate in primary progressive multiple sclerosis: results of a multinational, multicenter, double-blind, placebo-controlled trial. Ann Neurol (2007) 61(1):14–24.10.1002/ana.21079 17262850 [PubMed] [DOI]

2 Altmann DR Jasperse B Barkhof F Beckmann K Filippi M Kappos LD Sample sizes for brain atrophy outcomes in trials for secondary progressive multi ple sclerosis. Neurology (2009) 72(7):595–601.10.1212/01.wnl.0000335765.55346.fc 19005170 [OpenAIRE] [PubMed] [DOI]

3 Harrison DM Caffo BS Shiee N Farrell JA Bazin PL Farrell SK Longitudinal changes in diffusion tensor-based quantitative MRI in multiple sclerosis. Neurology (2011) 76(2):179–86.10.1212/WNL.0b013e318206ca61 21220722 [OpenAIRE] [PubMed] [DOI]

4 Frost C Kenward MG Fox NC. Optimizing the design of clinical trials where the outcome is a rate. Can estimating a baseline rate in a run-in period increase efficiency?Stat Med (2008) 27(19):3717–31.10.1002/sim.3280 18484598 [PubMed] [DOI]

5 Smith SM Zhang Y Jenkinson M Chen J Matthews PM Federico A Accurate, robust, and automated longitudinal and cross-sectional brain change analysis. Neuroimage (2002) 17(1):479–89.10.1006/nimg.2002.1040 12482100 [PubMed] [DOI]

6 Komori M Lin YC Cortese I Blake A Ohayon J Cherup J Insufficient disease inhibition by intrathecal rituximab in progressive multiple sclerosis. Ann Clin Transl Neurol (2016) 3(3):166–79.10.1002/acn3.293 27042677 [OpenAIRE] [PubMed] [DOI]

7 SAS Institute Inc.SAS/STAT®9.22 User’s Guide. Cary, NC: SAS Institute (2010).

8 Kurtzke JF. Rating neurologic impairment in multiple sclerosis: an expanded disability status scale (EDSS). Neurology (1983) 33(11):1444–52.10.1212/WNL.33.11.1444 6685237 [PubMed] [DOI]

9 Sipe JC Knobler RL Braheny SL Rice GP Panitch HS Oldstone MB. A neurologic rating scale (NRS) for use in multiple sclerosis. Neurology (1984) 34(10):1368–72.10.1212/WNL.34.10.1368 6541311 [PubMed] [DOI]

10 Fischer JS Rudick RA Cutter GR Reingold SC. The multiple sclerosis functional composite measure (MSFC): an integrated approach to MS clinical outcome assessment. National MS society clinical outcomes assessment task force. Mult Scler (1999) 5(4):244–50.10.1177/135245859900500409 10467383 [PubMed] [DOI]

11 Hallett M. Transcranial magnetic stimulation: a primer. Neuron (2007) 55(2):187–99.10.1016/j.neuron.2007.06.026 17640522 [OpenAIRE] [PubMed] [DOI]

12 Duan Q van Gelderen P Duyn J. Improved Bloch-Siegert based B1 mapping by reducing off-resonance shift. NMR Biomed (2013) 26(9):1070–8.10.1002/nbm.2920 23355474 [OpenAIRE] [PubMed] [DOI]

13 Deoni SC. High-resolution T1 mapping of the brain at 3T with driven equilibrium single pulse observation of T1 with high-speed incorporation of RF field inhomogeneities (DESPOT1-HIFI). J Magn Reson Imaging (2007) 26(4):1106–11.10.1002/jmri.21130 17896356 [PubMed] [DOI]

14 Shiee N Bazin PL Ozturk A Reich DS Calabresi PA Pham DL. A topology-preserving approach to the segmentation of brain images with multiple sclerosis lesions. Neuroimage (2010) 49(2):1524–35.10.1016/j.neuroimage.2009.09.005 19766196 [OpenAIRE] [PubMed] [DOI]

15 Chang LC Jones DK Pierpaoli C. RESTORE: robust estimation of tensors by outlier rejection. Magn Reson Med (2005) 53(5):1088–95.10.1002/mrm.20426 15844157 [PubMed] [DOI]

40 references, page 1 of 3
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publication . Article . 2016

Development of a Sensitive Outcome for Economical Drug Screening for Progressive Multiple Sclerosis Treatment

Peter Kosa; Danish Ghazali; Makoto Tanigawa; Chris Barbour; Irene Cortese; William Kelley; Blake Snyder; Joan Ohayon; Kaylan Fenton; Tanya Lehky; ...