Importance of long-term motor function evaluation after prednisolone treatment for Duchenne muscular dystrophy.
Hitomi Nishizawa, Naoko Shiba, Akinori Nakamura
PMID 30214127WHAT IT FOUND
In three boys with Duchenne muscular dystrophy, motor tests improved over long-term follow-up after prednisolone dose increases, except one rising-from-floor result.
Because only treated cases were reported, prednisolone cannot be shown to have caused the improvement.
Key findings
01Case 1 had better mean 10-meter running time, mean time to rise from the floor, and mean North Star Ambulatory Assessment score after the prednisolone dose increase than before it.
02Case 2 had better mean 10-meter running time and mean North Star Ambulatory Assessment score after the dose increase, while time to rise from the floor did not show a clear difference.
03Case 3 had better mean 10-meter running time, mean time to rise from the floor, and mean North Star Ambulatory Assessment score after the dose increase, although the mother reported no obvious motor improvement.
STILL TO COME
How it was doneWhat they foundWhat it means for PTs
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What it does not show
Only three boys were reported, so the results cannot show what will happen for most children with Duchenne muscular dystrophy. Only boys receiving prednisolone were reported, so there is no untreated comparison group. The observation periods were not the same for all participants, and the timing of evaluation differed relative to prednisolone start and dose increase. Parent reports did not always match test results, and diagnoses of autism spectrum disorder or attention deficit/hyperactivity disorder may have affected what parents observed.
Declared interests
The authors declared no competing interests.
The easy way to misread this
Do not conclude that prednisolone caused the motor improvements. The report describes only three boys receiving prednisolone, so there is no untreated comparison group.