When your child is first diagnosed with Duchenne muscular dystrophy (DMD), you’ll hear about steroids early and often. These medicines can help kids stay stronger for longer, but they also carry tradeoffs. Understanding both their pros and cons can help you feel more prepared for the decisions ahead.
Why are steroids used to treat DMD?
Steroids, also known as corticosteroids, are medications that reduce inflammation.
People living with DMD cannot make enough dystrophin, a protein that usually protects muscles from damage. Without dystrophin, muscles become progressively weaker and are gradually replaced by scar tissue and fat. By reducing inflammation, steroids help slow this process.
Steroids can also help preserve strength, breathing and heart function. Doctors often recommend starting steroids in early childhood, generally around age 4 or 5. Prednisolone and deflazacort are the two main steroids used to treat Duchenne.
Learn more about DMD treatment and care
Benefits of steroid treatment
Steroids are considered a cornerstone of DMD care. They offer several important benefits for children living with DMD:
- Better daily function: By slowing muscle decline, steroids can help children stay active and independent for longer.
- Reduced risk of a misshapen spine: Steroids can delay the development of scoliosis.
- Delay in loss of walking ability: Many children taking steroids are able to walk for months or years longer than those who do not.
- Support for heart and lung health: Steroids protect the heart muscle and improve breathing over time.
Risks and side effects
Steroids also have important drawbacks. Families should be aware of these before starting treatment:
- Weight gain: Extra weight is common and can make movement harder.
- Bone loss: Steroids can thin the bones, leading to fractures or osteoporosis.
- Growth delays: Children may grow more slowly while taking steroids.
- Mood and behavior changes: Irritability, mood swings or trouble sleeping can occur.
- Vision changes or loss: Steroids increase the risk of developing cataracts.
- Adrenal suppression: Long-term steroid use can reduce the body’s natural production of the stress hormone, cortisol. In the event of missed doses, or during times of stress, illness or surgery, this can lead to a dangerous problem called adrenal crisis. People living with DMD should never stop taking steroids without explicit instruction from their healthcare team.
Because of these risks, doctors carefully monitor growth, weight, blood pressure and bone health during steroid treatment. Tell your child’s doctor about any side effects that are unmanageable, so that they can adjust the dosage.
When starting steroids, any sudden losses in physical ability need to be reported to the doctor; this may also mean dosage adjustments.
Questions to ask your doctor
Every child with DMD is different. Asking your care team the right questions can help you make the best decision for your family. Some examples include:
- When should treatment begin and how is it given?
- How often will my child need check-ups or tests?
- What happens if a dose is missed?
- How do we prepare for surgery, illness, or other emergencies while on steroids?
The bottom line
Steroids remain one of the most important tools for slowing DMD progression, but they come with challenges. By working closely with your child’s neuromuscular team, you can balance the benefits and risks, plan ahead for side effects and make the treatment journey a little smoother.
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