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Longitudinal reliability of outcome measures in patients with Duchenne muscular dystrophy
Author(s) -
Nagy Sara,
Schädelin Sabine,
Hafner Patricia,
Bonati Ulrike,
Scherrer Delia,
Ebi Selina,
Schmidt Simone,
Orsini AnnaLena,
Bieri Oliver,
Fischer Dirk
Publication year - 2020
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.26690
Subject(s) - duchenne muscular dystrophy , medicine , clinical trial , muscular dystrophy , sample size determination , physical therapy , physical medicine and rehabilitation , radiological weapon , surgery , statistics , mathematics
The definition of reliable outcome measures is of increasing interest in patients with Duchenne muscular dystrophy (DMD). Methods In this retrospective study, we analyzed the longitudinal reliability of clinical and radiological endpoints in 29 ambulant patients with DMD. Clinical outcome measures included motor function measure (MFM) and timed function tests, while quantitative MRI data were mean fat fraction (MFF) and T2 relaxation time of thigh muscles. Statistical analysis was based on 3‐, 6‐, and 12‐month follow‐up data. Results Quantitative MRI using the MFF was the most sensitive and powerful marker of disease progression with a sample size of four at 1‐year follow‐up, followed by the D1 domain of MFM (standing and transfer function) with a sample size of 12. Discussion Our data support the longitudinal design of clinical trials over at least 12 months and the combinational use of clinical and radiological surrogate outcome measures.