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
- Be sure to tell all of your health care providers that you are taking steroids (corticosteroids). This is especially important if you are having surgery, have an infection, or have an injury.
- Most neurologists recommend that people with Duchenne begin steroids before the plateau phase (generally around 4-5 years of age).
- Schedule regular visits with your neuromuscular specialist (NMS) to manage steroids. Your NMS should explain the risks and benefits of steroids, and screen for side effects at each visit.
- Do not stop taking steroids until you and your NMS have tried to manage the side effects. Potential remedies may include changing to a different steroid, adjusting the dosage, and changing the schedule or regimen of the steroids.
- Do NOT stop taking steroids suddenly on your own. This puts you at risk for an adrenal crisis.
- If there is increased stress on the body (such as a severe illness or surgery), a stress dose of steroids is needed. Discuss potential situations when you may need a stress dose of steroids with your NMS.
Steroids are used to treat many different conditions. In Duchenne, a type of steroids called corticosteroids are 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 for all patients that can slow down the skeletal muscle damage and weakness caused by Duchenne. Prednisone (prednisolone) and deflazacort (Emflaza, Calcort, etc.) are both forms of steroids prescribed in Duchenne. Steroids may help your heart and lungs, as well as reduce the chance of a curved spine (scoliosis).
Steroids should be started before significant weakness is seen, prior to the “plateau phase” (see below for more information). Steroids are encouraged to be taken lifelong in order to optimize upper body strength and function, and to support the heart and lungs.
Know when to start taking steroids.
Steroids should be discussed near the time of diagnosis with your neuromuscular specialist. Steroids should be started before your child reaches the “plateau phase” and loses any significant strength – this is generally around age 4-5 years. In Duchenne, the plateau phase is when your child:
- Has learned all of their motor skills (crawling, walking, climbing stairs)
- Is having minimal trouble walking
- Can rise from the floor with little to no effort
- Can climb stairs with little to no effort
In other words, when your child is still able to physically do things in a way that is similar to their peers, it is the best time to start steroids.
Below is a visual description of the CDC recommendations of steroid initiation and management across the lifespan included in the Care Considerations.
The recommended starting dose of daily prednisone is 0.75 mg/kg/day. The recommended starting dose of daily deflazacort is 0.9 mg/kg/day. Your neuromuscular specialist (NMS) may increase or decrease the dose based on growth, weight, and other side effects experienced.
Steroids have been prescribed and taken in many doses and using many different schedules. In the US, dosing is either daily or high dose weekends. In some countries, other dosing schedules (i.e, 10 days on, 10 days off, etc.) have been used. The impact that steroids have on the body depends on the dose of the steroid and how often the steroids are taken. People who are taking intermittent doses (i.e., not daily) may have fewer or less severe side effects.
Is there a maximal dosing of steroids in Duchenne?
Due to the effects of daily steroids on the body, as well as the risk of side effects with higher doses, daily steroids are not prescribed in doses higher than around 36 mg/day of prednisone or 40 mg/day of deflazacort. While the FOR-DMD study is looking at dosing, there currently is no information around the maximal dosing of steroids in Duchenne. Generally doses of twice weekly steroids can go up to 250 mg/day of prednisone (or 300 mg/day of deflazacort, which is comparable to the prednisone weekend dose, but has not been studied) given on weekends.
Monitor for side effects of steroids
Taking daily steroids may lead to a variety of undesirable side effects (download). You should monitor for these side effects at home and notify your NMS if you notice major changes. At minimum, discuss these concerns at your regular NMS appointments. Major side effects of steroids include, but are not limited to:
Steroids may increase your appetite, making you feel more hungry and want to eat more. Maintaining a healthy diet will help avoid excessive weight gain.
Steroids can lead to new behavioral issues such as attention or emotional problems. If you or your child already has behavioral issues such as autism or attention deficit disorder (ADD), discuss this with your neuromuscular doctor before starting steroids. Sometimes taking steroids later in the day (after school) can help.
Weak bones (osteoporosis)
Steroid therapy in addition to weakened muscles can lead to thinner, weaker bones (osteoporosis). Ensuring you have enough calcium and vitamin D in your diet (or supplements if required) can help keep your bones healthy and prevent fractures.
Delayed growth and puberty
In Duchenne, steroid therapy can impact hormone levels. Most commonly, the hormones testosterone and growth hormone are affected. These imbalances can lead to delayed pubertal development and short stature. An endocrinologist can help correct these imbalances if indicated.
Stomach problems, such as heartburn (gastroesophageal reflux or GERD)
Steroids can cause stomach issues, such as the overproduction of acid in the stomach (heartburn), the reversal of acid and stomach contents back up into the esophagus (the tube that connects the throat to the stomach; also called “gastroesophageal reflux” or GERD). Taking steroids with food can help prevent stomach irritation.
Steroids can lead to cataracts, or clouding of the eye. It is recommended that you have eye exams yearly to check for cataracts.
High blood pressure
Steroids may cause increases in blood pressure. Blood pressure should be checked at every visit, but it is not necessary to limit salt intake unless you have been recommended to do so by your cardiologist.
Managing side effects of steroids
The most common reason for people to stop taking steroids is because of their side effects. Although these side effects can be serious, you or your child should never abruptly stop taking steroids if they experience undesirable side effects. This puts you or your child at risk of an adrenal crisis, which is a medical emergency.
There are many ways to manage the side effects of steroids. Possible remedies include:
- changing the type of steroid taken (i.e. prednisone to deflazacort)
- changing the prescribed dose
- changing the dosing schedule (i.e. time of day taken, weekend only dosing, etc.)
It is important for you to work with your NMS to make these changes and exhaust all alternatives before the choice is made to discontinue steroids. Currently, steroids are the only medicines known to help maintain strength and function for people living with Duchenne.
If you do make the decision to stop taking steroids, it is important that you do so under the supervision of a medical provider (preferably, the prescribing medical provider) who can advise a slow taper, or reduction of dosing. We recommend following the corticosteroid therapy withdrawal guidelines outlined in the PJ Nicholoff Steroid Protocol (download).
An adrenal crisis is a serious situation that can arise when steroids are suddenly discontinued, doses are missed for more than 24 hours, or stress doses are not given when indicated. Your adrenal glands secrete the hormone cortisol, also known as “stress hormone,” which has a vital role in protecting your body’s overall health. Steroids take the place of cortisol while you are taking them, causing your adrenal gland to stop producing cortisol and shrinks in size over time. If you suddenly stop taking steroids for any reason, your body can be thrown into a dangerous hormone imbalance.
Watch very carefully for signs of life-threatening adrenal crisis during the corticosteroid taper, and for one year post-taper during times of serious injury or illness. More information on adrenal crisis can be found here.
Higher doses (“stress doses”) of corticosteroids are sometimes necessary during illness, surgery, or other stress on your body. Anyone who takes steroids should know what dose to take on “sick days.”
Recommendations for supplemental stress doses are provided in the PJ Nicholoff Steroid Protocol (download). It is important that you share this critical information with your doctor during times of severe illness, surgery, or trauma to avoid life-threatening adrenal crisis.
What should I do if I miss a steroid dose(s)?
Missing doses of daily steroids for more than 24 hours for any reason can also cause a life-threatening adrenal crisis. If you or your child goes to the emergency room or is hospitalized for any reason, it is extremely important you notify the providers that you are on steroids and if you have missed a dose. Additionally, it is important to be mindful of conditions that may impact the amount of steroid being absorbed, such as vomiting for more than 24 hours (food poisoning, stomach bug, etc.).
If oral corticosteroids are missed on days when they are normally given for more than 24 hours, IV doses should be given. Be sure to share the PJ Nicholoff Steroid Protocol (download) with your doctor and create a plan together about what to do in case of a missed dose.
A stress dose is an extra dose of steroids that helps your body to deal with significant stressors. Stresses on the body can be mild, moderate, or severe. The table below, from the PJ Nicholoff Steroid Protocol, will help you to recognize which stressors might require a stress dose of steroids. When in doubt, it is generally safer to give a stress dose (which might not be needed) than to not give a stress dose (which really was needed).
Corticosteroid Stress Doses:
|Medical / Surgical Stress
|Corticosteroid Dosage Day of Surgery (DOS)
|Post-operative Taper Regimen
|Minor (local anesthesia, < 1 hour)
(e.g. inguinal hernia, single tooth extraction, colonoscopy), mild
febrile illness, mild, nausea/vomiting, mild diarrhea)
|25mg or 30-50 mg/m2 po (if able to take po) or IV hydrocortisone (HC) or equivalent
physiologic dose of
hydrocortisone when illness, pain or fever subsides
(e.g. multiple teeth extraction, fracture, pneumonia)
|50mg or 50-75 mg/m2 IV hydrocortisone or equivalent
|25 mg Q 8 or 50-75 mg/m2/day ÷ q 6 hours X 24 hour. Taper to baseline over 1-2 days.
(e.g. Septic shock, multiple trauma/fractures or severe burns, severe systemic infections, major surgery, pancreatitis, orthopedic surgery including open reduction, spinal fusion, etc.)
|100mg or 100 mg/m2/dose IV hydrocortisone or equivalent
|50 mg IV Q 8 or 100 mg/m2/day ÷ q 6 hours X 24-48hours. Taper to baseline over 1-3 days (continue stress dose if the physical stress (fever or pain) continues).
What should I do if I suspect an adrenal crisis?
Always go to the emergency room if you suspect an adrenal crisis — this is a life-threatening condition. It is important to know the risk factors and symptoms of life-threatening adrenal crisis. Keep the PJ Nicholoff Steroid Protocol (download) available to you, and your medical providers, in case of a medical emergency or admission. Alert your neuromuscular team in the event of a medical emergency or admission to help ensure that your/your child’s comprehensive care continues.