The information on this page represents the recommended standard of care for Duchenne muscular dystrophy. Most of the care recommendations also apply to Becker muscular dystrophy, but at older ages. Most, but not all, people with Duchenne are males—but the care recommendations apply to both males and females with Duchenne.
If you don’t understand any of the medical terms and concepts, ask your healthcare providers. Take notes and ask questions during your clinical visits.
Steroid Facts to Remember
|1. Be sure to tell all
providers that you are taking steroids
This is especially
important if you are having
surgery, have an infection,
or have an injury.
Your neurologist should
start giving steroids when the person with Duchenne reaches
the plateau phase or
You should have
regular visits with a
doctor who is skilled in
managing steroids. Your
doctor should explain
the possible side effects
of steroids and tell you
if you are at risk for
You should not
stop taking steroids until
your doctor has tried to
manage the side effects,
adjusted the dosage,
and changed the way
you are taking your steroids.
Doctors use different types of steroids to treat different conditions. In Duchenne,
a type of steroids called glucocorticosteroids is used to help maintain muscle
strength. They are different from the anabolic steroids that are sometimes misused by athletes who want to become stronger. Steroids are the only medicines currently available that can slow down
the muscle damage and weakness caused by Duchenne. Steroids may help your
son’s heart and lungs as well as reduce the chance of a curved spine (scoliosis).
Prednisone (prednisolone) is the only steroid approved by the U.S. Federal Drug
Administration (FDA) and available in the US. The starting dose of Prednisone is
0.75mg/kg/day. Deflazacort is a steroid available to treat Duchenne outside the US.
The starting dose of deflazacort is 0.9 mg/kg/day. Your child’s doctor may increase or
decrease the dose based on your child’s size, status, and any side effects.
Doctors may prescribe steroids during the "plateau phase" or sooner (see below for more information). Your doctor may
continue to give your child steroids after he starts using a wheelchair full-time to
continue to help his heart and lungs.
Below is the recommended standard care for using steroids. Talk to your doctor about care options that go above standard care.
Know when to start taking steroids.
Your doctor should start giving your child steroids when he reaches the “plateau
phase” or sooner. In Duchenne, the plateau phase is when your son:
- has learned all of his motor skills (crawling, walking, climbing stairs),
- is having minimal trouble walking,
- can rise from the floor with little to no effort, and
- can climb stairs with little to no effort.
In other words, when your child is still similar to his peers physically, that is the time to
intervene with steroids.
After this plateau stage, it will become more difficult for your child to walk, rise from
the floor, and climb stairs. If your doctor does not offer steroids during your child’s
plateau stage, your doctor should offer your child steroids when he starts to be less
Work with healthcare providers to watch for side effects.
Steroids can cause serious side effects, including:
- behavior problems
- thinner, weaker bones (osteoporosis)
- delayed puberty
- stomach problems (gastroesophageal reflux or GERD)
- sensitivity to infections
Your doctor should be careful when starting steroids if your child already has any of
these issues, if he has autism or attention deficit disorder, or if he is overweight.
Although side effects to steroids can be serious, your child should not stop taking
steroids if side effects first appear. You doctor has many ways to manage the side
effects, including lowering the amount of steroids your child takes and changing the
way your child takes the steroids. Make sure your doctor makes changes to try to
decrease side effects. Try out these changes before you and your child decide to stop
taking steroids—though the side effects may be difficult, remember that steroids are
the only medicines known to improve strength in people with Duchenne.
- It is important that people taking corticosteroids not miss their doses for more than 24 hours. You may also need a higher dose, or a “stress dose,” of corticosteroids with extreme stress, such as severe illness, surgery, or trauma.
Not having appropriate levels of cortisol in the body, daily or during episodes of “stress,” can cause the body to experience symptoms of acute adrenal insufficiency, or adrenal crisis, which can be life-threatening. Learn more about stress doses, corticosteroid therapy withdrawal, adrenal crisis symptoms, etc.
|Important Steroid Protocol Topics (Stress Doses, Corticosteroid Therapy Withdrawal, Adrenal Crisis Symptoms, Etc)
It is important that people taking corticosteroid not miss their doses for more than 24 hours. They may also need a higher dose, or a “stress dose,” of corticosteroids with extreme stress, such as severe illness, surgery, or trauma. Not having appropriate levels of cortisol in the body, daily or during episodes of “stress,” can cause the body to experience symptoms of acute adrenal insufficiency, or adrenal crisis, which can be life-threatening.
|Steroids, Salt Intake, & High Blood Pressure
It is well known that taking steroids can cause glucocorticoid-induced hypertension (“hypertension” is the fancy word for high blood pressure). So is it necessary for children who take steroids to limit their sodium/salt intake?
PJ Nicholoff Steroid Protocol (download)
Understanding and Using Glucocorticosteroids (download)
Our family-centered approach is at the heart of everything we do. Learn more.