1. 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.
- Author
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De Wel, Bram, Iterbeke, Louise, Huysmans, Lotte, Peeters, Ronald, Goosens, Veerle, Dubuisson, Nicolas, van den Bergh, Peter, Van Parijs, Vinciane, Remiche, Gauthier, De Waele, Liesbeth, Maes, Frederik, Dupont, Patrick, and Claeys, Kristl G.
- Subjects
BECKER muscular dystrophy ,MAGNETIC resonance imaging ,ADULTS ,CLINICAL trials ,MUSCLE strength testing ,HAMSTRING muscle injuries - 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. [ABSTRACT FROM AUTHOR]
- Published
- 2024
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