Steroids (Corticosteroids)

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.

Forms of steroids prescribed in Duchenne include:

  • Prednisone (also called prednisolone in its liquid form)
  • Deflazacort (Emflaza, Calcort, etc.)
  • Agamree (vamorolone)

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.

Important Steroid Facts to Remember

  1. 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.
  2. Most neurologists recommend that people with Duchenne begin steroids before the plateau phase (generally around 4-5 years of age).
  3. 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.
  4. 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.
  5. Do NOT stop taking steroids suddenly on your own. This puts you at risk for an adrenal crisis.
  6. If there is increased stress on the body (such as a severe illness, injury, or surgery), a stress dose of steroids is needed. Recommendations for supplemental stress doses are provided in the PJ Nicholoff Steroid Protocol (download). Discuss potential situations when you may need a stress dose of steroids with your NMS.

Recommended Care

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.

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.

Understanding steroid dosing

  • 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. The recommended starting dose of daily Agamree (vamorolone) is 2mg-6mg/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.
  • Vamorolone should not be used for intermittent or weekend-only treatment.
  • 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, or 300mg/day of vamorolone.
  • 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.

What else do I need to know about steroid dosing?

Adrenal suppression, or adrenal crisis, is a potentially life-threatening complication of steroid use. It is important to remember the following information:

  1. Do not stop taking steroids suddenly.
  2. Do not miss daily steroid doses for more than 24 hours (i.e. accidentally missing a dose, vomiting more than 24 hours, hospitalization without continuation of medication, etc.).
  3. Know when “stress doses” should be given (severe illness, surgery, or trauma).
    *Vamorolone cannot be used for stress dosing and it is unknown if deflazacort is appropriate for stress dosing. Refer to the  PJ Nicholoff Steroid Protocol (download) for stress dosing guidelines.

More information on these important topics can be found here.

Monitoring 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:

Weight gain

  • 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.

Behavior issues

  • 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.

Cataracts

  • 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

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. Guidance for switching steroid medications and dosages can be found in the PJ Nicholoff Steroid Protocol (download).

Additional Resources:

Discontinuing steroids

  • 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.
    • Watch very carefully for signs of life-threatening adrenal crisis during the corticosteroid taper, and for up to one year post-taper during times of serious injury or illness. You should continue to follow your stress dosing plan during this time.
  • 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. If you suddenly stop taking steroids for any reason, your body can be thrown into a dangerous hormone imbalance.

Stress dosing

  • 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

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.

Symptoms of adrenal crisis can include any of the following:

  • Abdominal pain
  • Shock
  • Confusion or disorientation 
  • Dehydration
  • Dizziness or light-headedness
  • Fatigue
  • Flank pain
  • Headache
  • High fever
  • Loss of appetite
  • Loss of consciousness
  • Low blood pressure
  • Low blood sugar
  • Nausea
  • Profound weakness
  • Rapid heart rate
  • Rapid respiratory rate (see tachypnea)
  • Slow, sluggish movement
  • Unusual and excessive sweating on face or palms
  • Vomiting

Additional Resources:

Risk for Adrenal Crisis – Download PJ’s Protocol

Adrenal suppression, or adrenal crisis, is a potentially life-threatening complication of steroid use. It is important to know that you may be at risk of an adrenal crisis when:

  • Steroids are stopped suddenly
  • Doses are missed for any reason (accidentally, vomiting more than 24 hours, hospitalization without continuation of medication, etc.)
  • The body is under extreme “stress” (severe illness, surgery, or trauma)

If you experience any signs of adrenal crisis, go to the Emergency Room and alert your neuromuscular team. Keep the PJ Nicholoff Steroid Protocol (download) available to you, and your medical providers, in case of a medical emergency or admission.

Risks and how to prevent adrenal crisis.

FAQ: Choosing a Steroid Drug, Dose, and Regimen

Considerations in Decision Making

What are the current steroids approved by the FDA that may be prescribed for the treatment of Duchenne?

  • Prednisone/Prednisolone is a widely utilized steroid for many medical conditions, and may be prescribed for patients with Duchenne. 
  • EMFLAZA, also called deflazacort in its generic form, is approved and commercially available for treatment of Duchenne. 
  • AGAMREE (vamorolone) has been approved by the FDA for treatment of Duchenne.

What should I consider when deciding which steroid drug and regimen to initiate for my child, or if I am considering switching from one steroid drug or regimen to another?

  • These decisions require an in-depth conversation between you and your neuromuscular team. Dr. Kathy Mathews developed this summary snapshot of steroids used in Duchenne that may act as a helpful introduction to steroid options and associated factors to consider.

Why are individuals living with Duchenne prescribed different drugs and regimens than other patients with the same diagnosis?

  • There is not one best steroid choice that applies to every situation. It is important to work in partnership with your neuromuscular team to make the best choice for you/your child and revisit this decision based on side effects.

Steps Toward Starting a New Medication

  • You will be asked to complete an enrollment, or “start,” form, which is specific to the brand drug and manufacturer where you will consent to provide information such as demographics, insurance information, etc.

How do I know whether my steroid choice is covered by insurance?

  • There will be a benefits investigation (BI) to review your medical and pharmacy benefits provided by your insurance, whether private or government funded. Your neuromuscular team and/or the drug manufacturer will work with your insurer on a prior authorization process to determine criteria for eligibility for coverage and whether it is approved or not.

Can I receive specialty medications through any pharmacy?

  • Typically, specialty medications are dispensed through a limited distribution specialty pharmacy, which is licensed and accredited to dispense specific drugs. Medications will typically need to be mailed to you from one or more locations and would not typically be available to pick up at a physical location.

Brand vs. Generic Drugs

I heard EMFLAZA lost patent exclusivity, what does this mean and why is it happening?

  • The patent and exclusivity period for the brand medication (EMFLAZA) recently expired. This means other companies can make a generic version of the medication deflazacort. The FDA determines the amount of time a particular drug receives exclusivity, which expired on February 9th, 2024.

How are generic medications different from brand medications?

  • Generic and brand medications have the same active ingredient, but are made by different companies and may have different “inert” or inactive ingredients.

Is it safe to switch from a brand medication to a generic?

  • Yes, it is safe to switch. Manufacturers have to perform extensive testing to prove to the FDA that their generic drug shows therapeutic equivalence, meaning it has equal effect and no difference when substituted for the brand.

Does generic medication work the same as brand?

  • Yes! It has the same medication in the same amounts so it will have the same effect on the body.

Will the generic medication look the same?

  • Generic deflazacort may look slightly different because it will be made by a different company. The tablets may be a different color or shape. The liquid may have a different flavor, color, or bottle

How do I switch to generic deflazacort?

  • You will need to discuss this change with your neuromuscular team, and if making a decision to switch to generic deflazacort, a neuromuscular provider will need to write a new prescription and have it sent to your pharmacy. It is possible that a specialty pharmacy will still be required for medication distribution.

Can we choose to stay on EMFLAZA and not switch to generic deflazacort?

  • At this time, PTC Therapeutics will continue to produce the brand drug EMFLAZA. If you and your neuromuscular team decide remaining on EMFLAZA is the best choice for you/your child, they will include a “Dispense as Written” (DAW) code on the prescription so it may not be substituted for a generic.

Will my insurance coverage of deflazacort change when generic deflazacort becomes available?

  • This will depend on your individual insurance plan.
  • Insurance may require the brand or generic.
  • Typically, insurance coverage of a medication improves when a generic becomes available. Insurance may also cover generic deflazacort if they had not previously covered brand Emflaza.
  • Insurance cannot make you change the medication. The decision to switch medications is between you and your provider. However, your insurance may choose not to cover brand EMFLAZA or may increase your cost-sharing.

Will my cost responsibility change?

  • This is dependent on your individual insurance plan. Typically when medications are available as generic, patient and societal costs decrease.
  • If you switch to generic deflazacort, you may not have the same access to the manufacturer copay assistance programs. This may not be necessary if insurance is covering the medication or cost-sharing responsibility is lower.

Who is the best person to talk to about my questions and concerns?

Your neuromuscular provider!

Watch: Navigating Steroid Choices [January 2024]

In recent months we have learned there will soon be more steroid options clinically available to people living with Duchenne. We invited the University of Iowa team to walk us through considerations for initiating a new steroid drug and/or regimen, and the different processes for obtaining access to these medications.