Dataset Information

Lower extremity outcome measures: considerations for clinical trials in spinal cord injury.

ABSTRACT:

Study design

This is a focused review article.

Objectives

To identify important concepts in lower extremity (LE) assessment with a focus on locomotor outcomes and provide guidance on how existing outcome measurement tools may be best used to assess experimental therapies in spinal cord injury (SCI). The emphasis lies on LE outcomes in individuals with complete and incomplete SCI in Phase II-III trials.

Methods

This review includes a summary of topics discussed during a workshop focusing on LE function in SCI, conceptual discussion of corresponding outcome measures and additional focused literature review.

Results

There are a number of sensitive, accurate, and responsive outcome tools measuring both quantitative and qualitative aspects of LE function. However, in trials with individuals with very acute injuries, a baseline assessment of the primary (or secondary) LE outcome measure is often not feasible.

Conclusion

There is no single outcome measure to assess all individuals with SCI that can be used to monitor changes in LE function regardless of severity and level of injury. Surrogate markers have to be used to assess LE function in individuals with severe SCI. However, it is generally agreed that a direct measurement of the performance for an appropriate functional activity supersedes any surrogate marker. LE assessments have to be refined so they can be used across all time points after SCI, regardless of the level or severity of spinal injury.

Publications

Lower extremity outcome measures: considerations for clinical trials in spinal cord injury.

Bolliger Marc M Blight Andrew R AR Field-Fote Edelle C EC Musselman Kristin K Rossignol Serge S Barthélemy Dorothy D Bouyer Laurent L Popovic Milos R MR Schwab Jan M JM Boninger Michael L ML Tansey Keith E KE Scivoletto Giorgio G Kleitman Naomi N Jones Linda A T LAT Gagnon Dany H DH Nadeau Sylvie S Haupt Dirk D Awai Lea L Easthope Chris S CS Zörner Björn B Rupp Ruediger R Lammertse Dan D Curt Armin A Steeves John J

Spinal cord 20180427 7

<h4>Study design</h4>This is a focused review article.<h4>Objectives</h4>To identify important concepts in lower extremity (LE) assessment with a focus on locomotor outcomes and provide guidance on how existing outcome measurement tools may be best used to assess experimental therapies in spinal cord injury (SCI). The emphasis lies on LE outcomes in individuals with complete and incomplete SCI in Phase II-III trials.<h4>Methods</h4>This review includes a summary of topics discussed during a work ...[more]

PMID: 29700477

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Project description:STUDY DESIGN: Systematic review. STUDY RATIONALE: According to current estimates, there are more than 1 million people living with a spinal cord injury (SCI) in the United States alone. Given the potentially devastating impact of SCI on health-related quality of life (QoL), we sought to gain an improved understanding of QoL outcomes in SCI. OBJECTIVE: To identify and describe common QoL outcomes measures in patients with SCI. METHODS: A systematic review of the English-language literature was undertaken for articles published from 1998 through December 2010. Electronic databases and reference lists of key articles were searched to identify measures or indices used to evaluate QoL outcomes in patients with SCI. The titles and abstracts of the SCI peer-reviewed literature were searched to determine which of these outcome measures were most commonly used to evaluate QoL in patients with SCI. RESULTS: We identified 27 outcome measures used to evaluate QoL in patients with SCI. In SCI literature, the six most commonly used objective outcome measures were the Short-form 36 (SF-36); Craig Handicap Assessment and Reporting Technique (CHART); Short-form 12 (SF-12); Sickness Impact Profile (SIP68); Reintegration to Normal Living Index (RNL); and Community Integration Questionnaire (CIQ). The six subjective measures that were most frequently used were the Satisfaction with Life Scale (SWLS); Quality of Life Index (QLI); Life Satisfaction Questionnaire (LISAT-9/-1); World Health Organization Quality of Life-BREF scale (WHOQOL-BREF); Perceived Quality of Life (PQOL); and global QoL. All six objective measures have been validated in an SCI population, and four of the six subjective measures have been similarly validated. Three of each of the objective and subjective measures have been reliability tested in a population with SCI. CONCLUSION: In addition to neurological and functional changes after SCI, QoL outcomes should be routinely assessed. Choice of appropriate QoL measure should be influenced by the study objectives and design, as well as the psychometric properties of the particular measure within the context of SCI.

Project description:BACKGROUND:Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease characterized by progressive muscle weakness, multiple system involvement and premature mortality. Effective treatments for DMD through clinical trials and natural history studies are currently underway. Clinical trials in DMD typically include several outcome measures of motor function. Research sites and studies have been found to have slightly different operational definitions for a given functional outcome resulting in different procedures and protocols for these measurements. OBJECTIVE:The goal of this study is to establish agreement among experts in the field around best practices in collecting functional outcome data in DMD providing researchers and clinicians with guidance on best practices. METHODS:A group of 30 experts in Duchenne Muscular Dystrophy (DMD) with experience in the development and/ or execution of lower extremity outcome measures for this population met face to face to identify incongruences in the collection of this data. This effort was based in the United States (US) and sponsored by Parent Project Muscular Dystrophy. Several discrepancies were categorized for each outcome which included: 6-minute walk test, 10-meter walk/run, supine to stand, ascend 4 stairs, sit to stand, and the NorthStar Ambulatory Assessment. Following this meeting an additional 32 experts in DMD (28 from the United States and 11 international participants) consented to participate in a Delphi Survey to reach consensus on the protocols and execution of lower extremity outcomes. RESULTS:Round one: 70 operationally defined questions were surveyed with 45 (64%) reaching >70% consensus. Round two: 27 questions were operational, with 20 (74%) reaching >70% consensus. Those questions that did not reach consensus appear minor. CONCLUSION:With minor modifications in the collection of data across sites, outcomes could potentially be normalized across research studies. This would reduce excessive training for evaluators in trials and produce minimal differences between protocols. Consistency in protocols will promote more efficient study start up, less errors between administration of items across studies, and ultimately improve quality and reliability of the functional outcomes. The authors strongly advocate for the establishment of a "research network library" that could be utilized by all those performing clinical assessments and trials in DMD.

Dataset Information

Lower extremity outcome measures: considerations for clinical trials in spinal cord injury.

Study design

Objectives

Methods

Results

Conclusion

Sponsors

Publications

Lower extremity outcome measures: considerations for clinical trials in spinal cord injury.

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