Starting corticosteroids earlier helps improve motor function in DMD
Study: Boys were able to run or walk faster, move better
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Starting daily corticosteroids earlier in young children with Duchenne muscular dystrophy (DMD) helps them move better, walk or run faster, and keep their muscles stronger than delaying treatment by about one year, underscoring the importance of early diagnosis for preserving motor function.
These are findings from a Phase 3 clinical study called FOR-DMD (NCT01603407), which included boys with DMD ages 4 to 7 years who were randomly assigned to receive daily prednisone or Emflaza (deflazacort) for one year. They were compared with patients assigned to a placebo in another clinical study, PolarisDMD (NCT03703882), which did not allow corticosteroid use.
Given that children are usually diagnosed around 5 years of age, “these findings underscore the importance of earlier diagnosis and timely discussion of treatment options,” researchers wrote in “Motor Function and Growth Outcomes with Early Corticosteroid Initiation in Duchenne Muscular Dystrophy: An Adjusted Cross-Trial Comparison,” which was published in Muscle & Nerve.
Over 1 year, children on corticosteroids improved in all 3 tests
DMD is a genetic disease that causes muscles to become weaker over time. This happens because the body cannot produce enough dystrophin, a protein that helps protect muscles from damage caused by normal wear and tear. As a result, children with DMD gradually lose the ability to walk and perform everyday movements.
Corticosteroids, which work by reducing inflammation, are the mainstay of treatment for DMD. These medications can slow or stop muscle breakdown and stimulate muscle repair, helping people with DMD maintain strength and mobility for longer.
This study looked at whether starting corticosteroids earlier leads to better outcomes than starting later or not starting at all. It included 114 children with DMD who started daily corticosteroids — either prednisone or Emflaza — and 42 children who did not receive corticosteroids.
Motor function was measured in three main ways. The North Star Ambulatory Assessment (NSAA) is a scale that scores motor skills such as standing, walking, and climbing, with higher scores indicating better motor function. The 10-meter (32.8-feet) walk/run test measures how fast a child can move over a short distance. The rise from supine test measures how quickly a child can stand up from a lying position.
The earlier in life a patient is diagnosed, the more time his caregivers have for informed discussions about corticosteroids and other treatment options, and the greater the opportunity to support meaningful functional gains during early developmental years.
Over one year, children on corticosteroids improved in all three tests, while untreated children generally worsened. Treated children gained an average of 6.9 points on the NSAA compared with untreated children, indicating better overall movement.
They also walked or ran faster and stood up more quickly. Fewer treated children needed more than 5 seconds to stand up (27.4% vs. 62.9%), and more of them showed an improvement of at least three points on the NSAA (67.6% vs. 12.3%).
These benefits were consistent across all ages, though changes varied as children grew. For example, younger children (ages 4 to 5) improved the most with treatment. They showed the smallest declines without treatment, while older children (7 to up to 8 years) tended to show smaller improvements with treatment and the greatest decline in motor function without treatment.
Benefits were also observed regardless of the type of corticosteroid used and across all levels of mobility at the start of treatment, including those who already had more difficulty standing up.
“The earlier in life a patient is diagnosed, the more time his caregivers have for informed discussions about corticosteroids and other treatment options, and the greater the opportunity to support meaningful functional gains during early developmental years,” the researchers concluded.
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