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 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 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
|
Steroids can delay the onset of cardiomyopathy and slow down the progression of heart failure in patients. |
Kyphoscoliosis |
Bone
|
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
|
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
|
Skeletal Muscles
|
Steroids can improve muscle strength and delay the deterioration of motor function.
|
Reduced life expectancy to 22-30 years old.
|
General
|
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
|
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
|
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
|
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.
|
It is essential to continue steroid therapy and customise the steroid dosing according to the individual child’s needs. |
4. Adolescence/ Early Non-Ambulatory
|
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
|
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.
|
Depending on the specific skin condition, various medications and creams may be prescribed to manage symptoms and facilitate healing.
|
Short stature
|
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
|
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
|
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
|
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.
|
Altering the timing and adjusting the dosage of steroid treatment can help reduce behavioural side effects. |
Hypertension from sodium and fluid retention
|
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
|
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. |
Glucose intolerance Insulin resistance Diabetes Mellitus
|
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
|
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. |
Cataract Increased eyeball pressure
|
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
|
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.
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