The use of statistical MUNE in a multicenter clinical trial

Muscle Nerve. 2004 Oct;30(4):463-9. doi: 10.1002/mus.20120.

Abstract

Techniques to estimate motor unit number (MUNE) measure the number of functioning motor units in a muscle. In diseases characterized by progressive motor unit loss, such as amyotrophic lateral sclerosis (ALS), MUNE may be useful to monitor disease progression or beneficial response to treatment. As part of a multicenter, placebo-controlled, randomized, double-blind clinical trial testing the efficacy of creatine in patients with ALS, statistical MUNE was measured in 104 patients tested monthly for 6 months. The objective was to determine whether MUNE was a reliable and sensitive outcome measure in the context of a multicenter trial. Formal training and reliability testing was required for all MUNE evaluators. Testing of normal controls showed a high degree of test-retest reliability. All patient data were combined as the experimental treatment showed no efficacy. There was a 23% decline in MUNE over 6 months. The technique as employed in this trial overemphasized the presence of small motor units; this problem was partially addressed by poststudy data monitoring and censuring. Thus, MUNE can be used reliably as an outcome measure in multicenter clinical trials; specific remedies are suggested for the difficulties encountered in this study.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Action Potentials / physiology
  • Amyotrophic Lateral Sclerosis / drug therapy*
  • Amyotrophic Lateral Sclerosis / pathology*
  • Cell Count
  • Creatine / therapeutic use
  • Data Interpretation, Statistical
  • Disease Progression
  • Electrodiagnosis
  • Electrophysiology
  • Humans
  • Motor Neurons / pathology*
  • Muscle, Skeletal / pathology*
  • Observer Variation
  • Quality Control
  • Reproducibility of Results
  • Research Design*
  • Treatment Outcome

Substances

  • Creatine