/ ENG

Information about Steroid Use 

For Duchenne Muscular Dystrophy

(2023 Version)

This article serves as a basic introduction. Please consult a doctor or a qualified specialist for more information when treatment is started.

 

Contents

What are steroids?

What are the benefits of steroid therapy?

When should I / my child consider taking steroid?

What are the side effects of steroid? How can these side effects be managed?

What are steroids?

What are commonly called “steroids” are synthetic versions of a human hormone called “cortisol” produced by our adrenal glands. Cortisol helps the entire body to respond to stress, reduce inflammation, regulate blood sugar and control blood pressure. It also affects how food is used by the body for energy.

Commonly used steroids medications in Duchenne muscular dystrophy include prednisolone, prednisone and deflazacort. Prednisone is a drug that would be turned into Prednisolone in the body such that they are similar. Both are available as medications as oral tablets or liquid. 

There are also newer medications under studies, such as “Vamorolone”, which may be candidates for future options.

In Duchenne muscular dystrophy, steroids act on the immune system and counteract the inflammatory and metabolic processes that are activated in the damaged skeletal muscles of patients with Duchenne muscular dystrophy.

 

What are the benefits of steroid therapy?

Decades of past studies supported the undisputed effectiveness of steroids as a disease-modifying treatment for Duchenne muscular dystrophy. Steroids have been shown to improve disease outcomes by delaying and reducing the rate of disease progression.
 
 Steroids can provide a wide range of benefits to patients with DMD, including improving muscle strength and preserving the function of the heart, lungs, as well as arms, and legs. Studies have shown the use of steroids can delay the development or slow down the progression of scoliosis, cardiomyopathy and the loss of ambulation.

Duchenne muscular dystrophy manifestations

Body System

How do steroids help?

Heart diseases, such as cardiomyopathy, abnormal rhythm, and heart failure.

Heart

A black background with a black squareDescription automatically generated with medium confidence

Steroids can delay the onset of cardiomyopathy and slow down the progression of heart failure in patients.

Kyphoscoliosis 

Bone

A black background with a black borderDescription automatically generated

Steroids can delay the onset and reduce the progression of the curvature of the kyphoscoliosis, so can reduce the need for scoliosis surgeries. 

 

Breathing problem such as breathing difficulty and respiratory failure.

 

Lung

A black background with a black squareDescription automatically generated with medium confidence

Steroids can preserve respiratory muscle strength, delay the onset of breathing problems , and slow down the deterioration of lung function, so can delay the requirement for ventilation support.

 

 

Progressive skeletal muscle weakness


 Affected children will be expecting wheelchair usage between 8-14 years old.

 

 

 

Skeletal Muscles

A black background with a black squareDescription automatically generated with medium confidence

A black background with a black squareDescription automatically generated with medium confidence A black background with a black squareDescription automatically generated with medium confidence

A black background with a black squareDescription automatically generated with medium confidence A black background with a black squareDescription automatically generated with medium confidence

Steroids can improve muscle strength and delay the deterioration of motor function.


 Steroids, if started no later than 4 years old can improve motor performance more effectively, e.g. able to jump or run better.


 Steroids can prolong independent walking.


 Steroids can prolong the independence in daily activities using both hands, such as feeding oneself during mealtime.

Reduced life expectancy to 22-30 years old.

 

 

General

A black background with a black squareDescription automatically generated with medium confidence

Steroids use together with a good standard of care can contribute to better health and extend survival into the third and fourth decades.

 

 

When should I / my child consider taking steroid?    

Stage of Duchenne muscular dystrophy

Steroid therapy planning

 

1.

Birth/Infancy

Pre-symptomatic

A black background with a black squareDescription automatically generated with medium confidence

Duchenne muscular dystrophy is usually unnoticed until three to four years old, which is the average age of diagnosis. Children have delay in developmental milestone.

 

 

Upon confirming the diagnosis, it is crucial to provide family support, genetic counselling, assessments, and education on the standard of care. This includes discussing the potential benefits of steroid therapy.

 

2.

Early Childhood /

Early Ambulatory

A black background with a black squareDescription automatically generated with medium confidence

Children may have trouble in maintaining balancing due to noticeable muscle weakness. They may have increased risk of falling. They may also struggle with activities such as jumping, getting up from the floor and climbing stairs.

This is the optimal time to begin steroid therapy.

3.

Late Childhood/

Late Ambulatory

A black background with a black squareDescription automatically generated with medium confidence

Children may find it increasingly challenging to maintain balance while walking and may develop fatigue more easily, especially when traveling long distances or climbing stairs.
 They may require additional support for mobility, such as scooter or wheelchair.

 

It is essential to continue steroid therapy and customise the steroid dosing according to the individual child’s needs.

4.

Adolescence/

Early Non-Ambulatory

A black background with a black squareDescription automatically generated with medium confidence

Children require a wheelchair to go around. They maintain good truncal support and can propel the wheelchair effectively using both hands.

 

Steroid therapy remains important at this stage and should be continued but with a lower dosing.

 

5.

Adulthood /

Late Non-Ambulatory

A black background with a black squareDescription automatically generated with medium confidence

The use of arms and hands becomes progressively more difficult.

 

 

Steroid therapy remains important at this stage and should be continued but with a lower dosing.

 

What are the side effects of steroid? How can these side effects be managed?

This table provides examples of the side effects of steroid and their management. 
 This is not an exhaustive list. If you suspect any side effects of steroids, always consult your doctor.

Possible side effects

Management

Acne, Warts, Skin infection.

Abnormal hair growth in parts of the body.

A white line in the darkDescription automatically generated

 

Depending on the specific skin condition, various medications and creams may be prescribed to manage symptoms and facilitate healing.
 
 In some cases, a dermatologist or endocrinologist referral may be necessary for further evaluation and management of the skin condition.

 

 

 

Short stature

A black background with white dotsDescription automatically generated

Regular monitoring of a child's growth is essential. This monitoring typically involves measuring the child's height, weight, and body mass index (BMI) during routine check-ups.  Currently, there is no specific treatment required for short stature.

Puberty delay

A black background with white eyesDescription automatically generated

Puberty characteristics typically  begin between 10 to 15 years of age. If puberty is delayed, your doctor can arrange referrals to an endocrinology specialist for further evaluation and possible treatment with hormonal therapy. 

 

Adrenal insufficiency

Adrenal Crisis

A black background with white dotsDescription automatically generated

 

 

Never abruptly stop steroids use without doctor’s guidance. 

 

Learn about the at-risk situations and symptoms of “adrenal insufficiency” that need immediate medical attention. 

 

If you wish to reduce or stop steroid medications, always consult your doctor.

 

Communicate steroid requirements to all healthcare providers and carry a steroid-alert card to facilitate necessary medical arrangements.

 

If necessary, your doctor can arrange referrals to an endocrinology or metabolic specialist.

Suppression of Immunity and Infections

A black background with a black centerDescription automatically generated

Any minor infections need to be promptly addressed by seeking medical attention.

Be aware of any regional contagious infections and take necessary precautions.

 

Behavioural changes such as irritability, mood swings, aggression, withdrawn, distractions.

A black background with white dotsDescription automatically generated

Altering the timing and adjusting the dosage of steroid treatment can help reduce behavioural side effects.
 If significant behavioural side effects persist, consider switching from Prednisolone to Deflazacort.
 If necessary, your doctor can arrange a referral to child behavioural specialists for further management.

Hypertension from sodium and fluid retention

A black background with white dotsDescription automatically generated

Regularly monitor the blood pressure and follow dietary advice to avoid high salt intake and maintain a healthy weight. If necessary, medications can be prescribed to help control the blood pressure.

 

Increase in appetite leading to excessive weight gain

A white light in the darkDescription automatically generated

Consulting a dietitian can provide valuable guidance on proactive dietary management, and recommendations on how to minimize excessive sugar and salt intake and eating a balanced diet based on individual needs.
 To prevent excessive weight, it is essential to maintain regular physical exercises.
 If weight gain or obesity is affecting sleep quality or causing difficulty in breathing, a sleep study may be recommended.

Glucose intolerance

Insulin resistance

Diabetes Mellitus

A black background with a black squareDescription automatically generated with medium confidence

Increased thirst or increased urination can be symptoms of glucose intolerance or diabetes. If you experience these symptoms, please inform your doctor, who may recommend tests to check your blood sugar levels. Regular monitoring of urine and blood sugar levels can help in the early detection of glucose intolerance and diabetes.

If glucose intolerance is identified, lifestyle modifications and dietary control can help improve blood sugar levels.

If diabetes has developed, your doctor will refer you to an endocrinologist or diabetes specialist for further evaluation and medical treatment.

Gastritis

Gastro-Oesophageal-Reflux 

Peptic ulcer disease

Abdominal Pain

A black background with a black squareDescription automatically generated with medium confidence

Steroids can cause gastrointestinal side effects, including inflammation of the stomach (gastritis), acid reflux symptoms, and in severe cases, damage to the stomach. These side effects can be uncomfortable.
 
 To minimize the side effects, it is advisable to avoid medications that can irritate the stomach, such as aspirin, ibuprofen, naproxen, and NSAIDS (Non-Steroid Anti-Inflammatory Drugs).
 
 If you are experiencing gastrointestinal symptoms related to steroid use, consult your doctor. They may prescribe antacids or other medications to help protect the stomach lining and provide symptomatic relief.
 
 In some cases, if symptoms persist or worsen, your doctor may refer you to a gastroenterologist.

Cataract

Increased eyeball pressure

A black background with a black squareDescription automatically generated with medium confidence

Steroids can cause benign cataracts. In addition to cataracts, steroid use may also increase the risk of elevated intraocular pressure. To monitor these potential complications, it is essential to have a yearly visual check-up. Referral to an ophthalmologist would be necessary if any eye issue occurs.

Lowered bone mineral density

Osteoporosis

Risk of fractures

A black background with a black borderDescription automatically generated

Regular monitoring of bone health, including bone mineral density scans and vitamin D level assessments, is essential. A high calcium diet, as well as vitamin D3 and calcium supplements, can also improve dietary calcium intake.
 Exposure to sunlight can aid in the production of vitamin D from cholesterol within the skin.


 If osteoporosis is diagnosed, it is necessary to refer the patient to an endocrinology specialist for regular bisphosphonate treatment.


 In case of a fracture, the patient should be referred to an orthopaedic surgeon for appropriate management.

 

 

Some of the images of this web article is provided by https://www.flaticon.com/icons