Literature DB >> 25384682

Commonalities and challenges in the development of clinical trial measures in neurology.

Jesse M Cedarbaum1, Diane Stephenson, Richard Rudick, Maria C Carrillo, Glenn Stebbins, Douglas Kerr, Jill Heemskerk, Wendy R Galpern, Petra Kaufmann, David Cella, Maria Isaac, Marc K Walton.   

Abstract

As neurologists and neuroscientists, we are trained to evaluate disorders of the nervous system by thinking systematically. Clinically, we think in terms of cognition, behavior, motor function, sensation, balance and co-ordination, and autonomic system function. But when we assess symptoms of neurological disorders for the purpose of drug development, we tend to create disease-specific outcome measures, often using a variety of methods to assess the same types of dysfunction in overlapping, related disorders. To begin to explore the potential to simplify and harmonize the assessment of dysfunction across neurological disorders, a symposium, entitled, "Commonalities in the Development of Outcome Measures in Neurology" was held at the 16th annual meeting of the American Society for Experimental NeuroTherapeutics (ASENT), in February 2014. This paper summarizes the presentations at the symposium. The authors hope that readers will begin to view Clinical Outcome Assessment (COA) development in a new light. We hope that in presenting this material, we will stimulate discussions and collaborations across disease areas to develop common concepts of neurological COA development and construction.

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Year:  2015        PMID: 25384682      PMCID: PMC4322066          DOI: 10.1007/s13311-014-0310-1

Source DB:  PubMed          Journal:  Neurotherapeutics        ISSN: 1878-7479            Impact factor:   7.620


  21 in total

1.  The diagnosis of dementia due to Alzheimer's disease: recommendations from the National Institute on Aging-Alzheimer's Association workgroups on diagnostic guidelines for Alzheimer's disease.

Authors:  Guy M McKhann; David S Knopman; Howard Chertkow; Bradley T Hyman; Clifford R Jack; Claudia H Kawas; William E Klunk; Walter J Koroshetz; Jennifer J Manly; Richard Mayeux; Richard C Mohs; John C Morris; Martin N Rossor; Philip Scheltens; Maria C Carrillo; Bill Thies; Sandra Weintraub; Creighton H Phelps
Journal:  Alzheimers Dement       Date:  2011-04-21       Impact factor: 21.566

2.  Validation of the MDS-UPDRS Part I for nonmotor symptoms in Parkinson's disease.

Authors:  David A Gallagher; Christopher G Goetz; Glenn Stebbins; Andrew J Lees; Anette Schrag
Journal:  Mov Disord       Date:  2011-09-13       Impact factor: 10.338

3.  Using cognitive pretesting in scale development for Parkinson's disease: the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) example.

Authors:  Barbara C Tilley; Nancy R LaPelle; Christopher G Goetz; Glenn T Stebbins
Journal:  J Parkinsons Dis       Date:  2014       Impact factor: 5.568

4.  The effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis.

Authors:  Merit Cudkowicz; Michael E Bozik; Evan W Ingersoll; Robert Miller; Hiroshi Mitsumoto; Jeremy Shefner; Dan H Moore; David Schoenfeld; James L Mather; Donald Archibald; Mary Sullivan; Craig Amburgey; Juliet Moritz; Valentin K Gribkoff
Journal:  Nat Med       Date:  2011-11-20       Impact factor: 53.440

5.  How to identify tremor dominant and postural instability/gait difficulty groups with the movement disorder society unified Parkinson's disease rating scale: comparison with the unified Parkinson's disease rating scale.

Authors:  Glenn T Stebbins; Christopher G Goetz; David J Burn; Joseph Jankovic; Tien K Khoo; Barbara C Tilley
Journal:  Mov Disord       Date:  2013-02-13       Impact factor: 10.338

Review 6.  The Unified Parkinson's Disease Rating Scale (UPDRS): status and recommendations.

Authors: 
Journal:  Mov Disord       Date:  2003-07       Impact factor: 10.338

7.  Expanded and independent validation of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS).

Authors:  Pablo Martinez-Martin; Carmen Rodriguez-Blazquez; Mario Alvarez-Sanchez; Tomoko Arakaki; Alberto Bergareche-Yarza; Anabel Chade; Nelida Garretto; Oscar Gershanik; Monica M Kurtis; Juan Carlos Martinez-Castrillo; Amelia Mendoza-Rodriguez; Henry P Moore; Mayela Rodriguez-Violante; Carlos Singer; Barbara C Tilley; Jing Huang; Glenn T Stebbins; Christopher G Goetz
Journal:  J Neurol       Date:  2012-08-05       Impact factor: 4.849

8.  Movement Disorder Society-sponsored revision of the Unified Parkinson's Disease Rating Scale (MDS-UPDRS): scale presentation and clinimetric testing results.

Authors:  Christopher G Goetz; Barbara C Tilley; Stephanie R Shaftman; Glenn T Stebbins; Stanley Fahn; Pablo Martinez-Martin; Werner Poewe; Cristina Sampaio; Matthew B Stern; Richard Dodel; Bruno Dubois; Robert Holloway; Joseph Jankovic; Jaime Kulisevsky; Anthony E Lang; Andrew Lees; Sue Leurgans; Peter A LeWitt; David Nyenhuis; C Warren Olanow; Olivier Rascol; Anette Schrag; Jeanne A Teresi; Jacobus J van Hilten; Nancy LaPelle
Journal:  Mov Disord       Date:  2008-11-15       Impact factor: 10.338

9.  Evidence of multidimensionality in the ALSFRS-R Scale: a critical appraisal on its measurement properties using Rasch analysis.

Authors:  Franco Franchignoni; Gabriele Mora; Andrea Giordano; Paolo Volanti; Adriano Chiò
Journal:  J Neurol Neurosurg Psychiatry       Date:  2013-03-20       Impact factor: 10.154

10.  Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims: draft guidance.

Authors: 
Journal:  Health Qual Life Outcomes       Date:  2006-10-11       Impact factor: 3.186

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  2 in total

Review 1.  Gaps Between Aims and Achievements in Therapeutic Modification of Neuronal Damage ("Neuroprotection").

Authors:  Heinz Wiendl; Christian Elger; Hans Förstl; Hans-Peter Hartung; Wolfgang Oertel; Heinz Reichmann; Stefan Schwab
Journal:  Neurotherapeutics       Date:  2015-04       Impact factor: 7.620

Review 2.  Targeted Therapies for Parkinson's Disease: From Genetics to the Clinic.

Authors:  S Pablo Sardi; Jesse M Cedarbaum; Patrik Brundin
Journal:  Mov Disord       Date:  2018-04-27       Impact factor: 10.338

  2 in total

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