The 6-Minute Walk Distance (6MWD) is an effective tool in detecting a motor decline in patients with late-onset Pompe disease who have been treated with enzyme replacement therapy, according to results of a new study published in Cells.
The 6MWD should be included as an outcome measure in late-onset Pompe disease clinical trials, the authors said. In the present study, a team led by Christophe E. Depuydt, MSc, PhD, studied several potential outcome measures in 12 patients with late-onset Pompe disease who could still walk and were treated with enzyme replacement therapy.
These measures included isometric muscle strength using a Biodex® dynamometer, Medical Research Council sum score (MRC-SS), handgrip strength, 10-Meter Walk Test, Timed Up and Go Test, and 6MWD.
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The researchers tested the patients every 6 months for 2 years and compared them to 12 age- and sex-matched controls without the disease.
The results showed that there was a significant decline in the mean isometric muscle strength of the right and left knee extensors in healthy patients after 1 year and in patients with Pompe disease after 1.5 years. After 2 years, there were significant declines in both controls and patients.
When the researchers analyzed the 6MWD, they found that there was a significant decrease in the distance patients could walk in 6 minutes after 2 years. More precisely, the average distance decreased from 451.9 m to 368.1 m. In controls, on the other hand, there was a significant increase in the 6MWD after 6 months and 18 months.
There were no significant differences in the MRC-SS, handgrip test, 10-Meter Walk Test, and Timed Up and Go Test between healthy controls and patients with late-onset Pompe disease.
“We conclude that the 6MWD is a useful outcome measure to detect motor decline in treated [late-onset Pompe disease] patients,” the researchers wrote.
Claeys KG, D’Hondt A, Fache L, Peers K, Depuydt CE. Six-minute walk distance is a useful outcome measure to detect motor decline in treated late-onset Pompe disease patients. Cells. 2022;11(3):334. doi:10.3390/cells11030334