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, Bram;Iterbeke, Louise;Huysmans, Lotte;Peeters, Ronald;Claeys, Kristl G;et.al.
(2024) European journal of neurology — Vol. 31, n° 7, p. e16282 [1-12] (2024)

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Authors
  • De Wel, Bramorcid-logo
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  • Iterbeke, Louise
    Author
  • Huysmans, Lotte
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  • Peeters, Ronald
    Author
  • Dubuisson, Nicolasorcid-logoUCLouvain
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  • Van den Bergh, Peterorcid-logoUCLouvain
    Author
  • Van Parys, VincianeUCLouvain
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  • Claeys, Kristl Gorcid-logo
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Abstract
BACKGROUND AND PURPOSE: Because Becker muscular dystrophy (BMD) is a heterogeneous disease and only few studies have evaluated adult patients, it is currently still unclear which outcome measures should be used in future clinical trials. METHODS: Muscle magnetic resonance imaging, patient-reported outcome measures and a wide range of clinical outcome measures, including motor function, muscle strength and timed-function tests, were evaluated in 21 adults with BMD at baseline and at 9 and 18 months of follow-up. RESULTS: Proton density fat fraction increased significantly in 10/17 thigh muscles after 9 months, and in all thigh and lower leg muscles after 18 months. The 32-item Motor Function Measurement (MFM-32) scale (-1.3%, p = 0.017), North Star Ambulatory Assessment (-1.3 points, p = 0.010) and patient-reported activity limitations scale (-0.3 logits, p = 0.018) deteriorated significantly after 9 months. The 6-min walk distance (-28.7 m, p = 0.042), 10-m walking test (-0.1 m/s, p = 0.032), time to climb four stairs test (-0.03 m/s, p = 0.028) and Biodex peak torque measurements of quadriceps (-4.6 N m, p = 0.014) and hamstrings (-5.0 N m, p = 0.019) additionally deteriorated significantly after 18 months. At this timepoint, domain 1 of the MFM-32 was the only clinical outcome measure with a large sensitivity to change (standardized response mean 1.15). DISCUSSION: It is concluded that proton density fat fraction imaging of entire thigh muscles is a sensitive outcome measure to track progressive muscle fat replacement in patients with BMD, already after 9 months of follow-up. Finally, significant changes are reported in a wide range of clinical and patient-reported outcome measures, of which the MFM-32 appeared to be the most sensitive to change in adults with BMD.
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De Wel, B., Iterbeke, L., Huysmans, L., Peeters, R., Goosens, V., Dubuisson, N., Van den Bergh, P., Van Parys, V., Remiche, G., De Waele, L., Maes, F., Dupont, P., & Claeys, K. G. (2024). 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. European journal of neurology, 31(7), e16282 [1-12]. https://doi.org/10.1111/ene.16282 (Original work published 2024)