MFM and MRI of the thighs are the assessment tools most sensitive to changes in BMD

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In order to define parameters for monitoring and evaluating Becker muscular dystrophy (BMD) that could be used in clinical trials, a Belgian team conducted a study in 21 BMD patients over 18 months.

  • Changes in muscle magnetic resonance imaging (MRI), patient-reported criteria (ActivLim, SF36), numerous clinical evaluation criteria, including motor function measurement (MFM), muscle strength and timed tests were assessed at inclusion, 9 and 18 months.
  • MRI was sensitive enough to detect a significant increase in thigh muscle fat fraction as early as nine months.
  • MFM-32, North Star Ambulatory Assessment (NSAA) and ActivLim scores had all deteriorated significantly by nine months.
  • MFM domain 1 showed the greatest sensitivity in detecting motor changes.

 

Lessons for future clinical trials in adults with Becker muscular dystrophy: Disease progression detected by muscle magnetic resonance imaging, clinical and patient-reported outcome measures. De Wel B, Iterbeke L, Huysmans L et al. Eur J Neurol. 2024 Mar 20:e16282.

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