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10:32
Assessing Functional Performance in the Mdx Mouse Model
Published on: March 27, 2014
Functional outcome measures as clinical trial endpoints in ALS
B J Traynor1, H Zhang, J M Shefner
1Neurology Clinical Trials Unit, Department of Neurology, Massachusetts General Hospital, Boston, MA, USA. btraynor@partners.org
Neurology
|November 24, 2004
Summary
This study found that the decline in forced vital capacity (FVC) and ALS functional rating scale (ALSFRS) predicted survival in ALS patients. Declines in muscle strength measures did not predict survival.
Area of Science:
- Neurology
- Clinical Trials
- Biostatistics
Background:
- Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease.
- Understanding prognostic factors is crucial for managing ALS patients.
Purpose of the Study:
- To evaluate the prognostic value of different outcome measures in ALS patients.
- To assess the rate of decline in functional and strength measures over 12 months.
Main Methods:
- A 12-month randomized placebo-controlled trial involving 97 ALS patients.
- Regular assessment of forced vital capacity (FVC%), ALS functional rating scale (ALSFRS), and muscle strength (MVIC-arm, MVIC-grip Z scores) at 3-month intervals.
Main Results:
- Linear decline observed in FVC% and ALSFRS.
- Curvilinear decline observed in MVIC-arm and MVIC-grip Z scores.
- Rates of FVC% and ALSFRS decline were independent predictors of survival.
Conclusions:
- Forced vital capacity and ALS functional rating scale are significant predictors of survival in ALS.
- Muscle strength measures (MVIC-arm, MVIC-grip) are not independent predictors of survival in this cohort.
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