Peak functional ability and age at loss of ambulation in Duchenne muscular dystrophy.
Alberto A Zambon, Vandana Ayyar Gupta, Deborah Ridout and 4 others
PMID 35385138WHAT IT FOUND
For boys with Duchenne muscular dystrophy, how they move at age 6 to 7 predicts when they will stop walking.
A rise-from-floor time over 5 seconds or a low functional score at this age strongly predicts earlier loss of ambulation, even with steroid treatment.
Key findings
01A time to rise from the floor of more than 5 seconds at baseline was associated with a 96% higher risk of losing ambulation compared to a time between 3.6 and 5 seconds.
02Boys with a North Star Ambulatory Assessment score of less than 22 at baseline had a 53% greater risk of losing ambulation compared to those scoring 26 to 28.
03Daily corticosteroid regimen at baseline was associated with a 41% reduction in the risk of losing ambulation compared to intermittent regimen.
STILL TO COME
How it was doneWhat they foundWhat it means for PTsWhat it means for OTs
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What it does not show
The study used a pragmatic age window (6 to 7.5 years) to define baseline because the exact peak of NSAA scores could not be precisely determined for many patients. Close to 40% of patients switched their corticosteroid type or regimen after the baseline assessment, which may affect the interpretation of the treatment's long-term impact. The cohort was drawn from UK neuromuscular centers, so the results may not be representative of DMD populations in other countries or ethnic groups. There was some missing data in the NorthStar database, particularly regarding corticosteroid regimen information at baseline for some patients.
Declared interests
The study was supported by non-U.S. government funding. No specific commercial conflicts of interest are detailed in the provided text.
The easy way to misread this
Do not assume that a high NSAA score at age 6 or 7 guarantees walking beyond age 13. Approximately 40% of patients in this study lost ambulation before age 13 despite having very good baseline motor abilities, so prognosis remains uncertain for many children.