Key result
Higher baseline T2 H2O (>40.6 ms) in the right vastus medialis was associated with an 11-point greater deterioration on the NSAAD and an additional 86 m lost on the 6-minute walk over 3 years.
Why the study?
Water T2 mapping is increasingly used to assess active muscle damage and suggested as a clinical trial outcome measure, but its prognostic utility to identify changes in muscle function over time required investigation.
Does baseline Water T2 mapping predict functional decline over 3 years in patients with dysferlinopathy?
Population
18 patients with genetically confirmed dysferlinopathy from two sites
Comparison
Higher vs lower than median baseline water T2 in bilateral muscles
Design
Two-site cohort study
Follow-up
3 years
Authors
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Higher baseline T2 H2O may identify faster progressors in dysferlinopathy; leaves open its utility for trial enrichment pending validation.
Cohort (n=18)
Yes
Does baseline Water T2 mapping predict functional decline over 3 years in patients with dysferlinopathy?
Baseline Water T2 mapping can predict the rate of functional decline in patients with dysferlinopathy, suggesting its utility for prognostication and patient selection in clinical trials.
Moore et al. (2022) conducted a cohort in Dysferlinopathy (n=18). Higher baseline Water T2 (T2 H2O) vs. Lower baseline Water T2 (T2 H2O) was evaluated on Degree of deterioration on four functional tests over 3 years. Higher baseline T2 H2O (>40.6 ms) in the right vastus medialis was associated with an 11-point greater deterioration on the NSAAD and an additional 86 m lost on the 6-minute walk over 3 years.