Reevaluating measures of disease progression in facioscapulohumeral muscular dystrophy

Jeffrey M. Statland, Michael P. McDermott, Chad Heatwole, William B. Martens, Shree Pandya, E. L. van der Kooi, John T. Kissel, Kathryn R. Wagner, Rabi Tawil

Research output: Contribution to journalArticlepeer-review

27 Scopus citations

Abstract

Recent advances in the understanding of the molecular pathophysiology of facioscapulohumeral muscular dystrophy (FSHD) have identified potential therapeutic targets. Consequently, an accurate understanding of disease progression in FSHD is crucial for the design of future clinical trials. Data from 228 subjects in 3 clinical trials and 1 natural history study were compared to examine disease progression in FSHD. All studies utilized the same techniques for manual muscle testing and maximum voluntary isometric contraction testing. Both techniques yield a total strength score that can be followed over time as an indicator of disease progression. Whereas natural history data showed a decrease in strength over 1. year, there was an apparent increase in strength at 6. months in 2 of the 3 clinical trials in both the placebo and treatment groups, that persisted for up to 1. year for maximum voluntary isometric contraction testing. Variability estimates from the clinical trial data were consistent with those seen in the natural history data. Patients in clinical trials in FSHD may have better outcomes than those in natural history studies, regardless of treatment assignment, emphasizing the importance of placebo groups and the need for caution when interpreting the strength results of controlled and uncontrolled trials.

Original languageEnglish (US)
Pages (from-to)306-312
Number of pages7
JournalNeuromuscular Disorders
Volume23
Issue number4
DOIs
StatePublished - Apr 2013

Keywords

  • All clinical trials
  • All neuromuscular disease
  • Clinical trials methodology/study design
  • Muscle disease
  • Outcome research

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Neurology
  • Clinical Neurology
  • Genetics(clinical)

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