karenwalkersmith East Midlands
Andleo South East
Mariemulholland Northern Ireland
SophieO South East
MichaelM North West
fertzy South East
What assessments should be done and why
Your son should have regular checkups with a specialist doctor who has the expertise to monitor how things are going and to understand if there is anything unusual that might need additional evaluation. This is important in order to make decisions about new treatments at the most appropriate time and to anticipate and prevent problems to the
maximum extent possible. It is recommended that your son sees the doctor every 6 months and the specialist physiotherapist and/or occupational therapist about every 4 months if possible. Tests used in different clinics to follow
individuals with DMD may vary. The most important thing is that there is regular review so that interventions can be properly monitored. This regular assessment should include tests that help show how the condition is progressing, including:
STRENGTH: Strength may be measured in a number of different ways to see if the force that can be generated at specific joints is changing.
RANGE OF JOINT MOTION: This is done to monitor if contractures or joint tightening is developing and to help to guide what stretches or interventions will be most helpful.
TIMED TESTS: Many clinics routinely time activities such as the time to get up off the floor, time to walk a certain distance, and time to climb several steps. This gives important information on how the condition is changing and how it is responding to treatment.
MOTOR FUNCTION SCALES: There are a large number of different scales, but your clinic should routinely use the same one to monitor the condition in a systematic way. Different scales may be needed at different times.
ACTIVITIES OF DAILY LIVING: This allows the team to tell if some additional help might be needed to assist independence.
IMPORTANT FACTS TO REMEMBER
Drug treatments for muscle symptoms
There is a lot of research happening at the moment in the area of new drugs for DMD. In this document the experts only gave recommendations where there is already sufficient evidence for a treatment. These recommendations will change in future when new evidence (such as the results of clinical trials) becomes available. The guidelines will
be reviewed as new results become available. Although it is expected that in future a wider range of treatment options will be available, at the present time, the only drug treatment for the musculoskeletal symptoms of DMD
that the experts agreed there was sufficient evidence to be able to recommend is steroid treatment. Steroids are discussed in detail in this section. Drug treatments for other specific symptoms of the disease, such as heart
problems, are discussed later.
Steroid treatment a step by step guide
Steroids are used in many other medical conditions and there is a lot of experience in their use worldwide. There is no doubt they can benefit many boys with DMD but this benefit needs to be balanced with proactive management of possible side effects. Use of steroids is very important in DMD and should be discussed with all families early.
for longer to allow enhanced participation and to later minimise breathing, heart and orthopaedic problems. They can also reduce the risk of scoliosis (curvature of the spine).
STARTING AND STOPPING STEROIDS
*The recommended national vaccination schedule should be complete before steroid treatment is started, and varicella (chicken pox) immunity should be established.
In boys who used steroids when they were walking, many experts recommend continuation of medication after loss of ambulation. The goal in the non-ambulatory person is to preserve upper limb strength, slow the progression of scoliosis, and delay the decline of respiratory and cardiac function.
IMPORTANT FACTS TO REMEMBER:
The different steroid regimes
One of the potentially confusing things in DMD care is that different doctors and different clinics often prescribe different regimens of steroids, which means you will find information about different drugs and different regimes. These guidelines have tried to establish a clear route to use steroids effectively and safely based on regular
assessments of function and side effects .
used in DMD. They are believed to work similarly. Neither one is clearly better. Planned trials of these drugs are important and should help us understand them better in future. The choice of which steroid to use depends upon availability in a particular country, the cost to the family, the way the drug is taken, and the perceived side effects. Prednisone has the advantage of being inexpensive and is available in both tablet and liquid formulation. Deflazacort may be preferred to prednisone for some individuals because there may be a slightly lower risk of weight gain.
DOSES FOR STARTING AND MAINTAINING STEROIDS
MANAGEMENT OF STEROID MEDICATION
Should adjustments to the steroids dosing and/or schedule regimens prove ineffective in making any significant side effects sufficiently manageable and tolerable, then it is necessary to discontinue steroid therapy. These decisions need to be made individually in partnership with the child and the family.steroids should should never be stopped suddenly.
Steroid management and side effects (Boxes 3 and 4 and Table 1)
Attentive management of steroid-related side effects is crucial once a boy has started on long-term steroid therapy. While steroid therapy is currently the mainstay of medication therapy for DMD, it should not be undertaken casually by the doctor or family, and should be undertaken only by doctors with appropriate expertise.
OTHER DRUG AND DIETARY SUPPLEMENTS
The experts considered a range of other drugs and supplements that are known to be used in some cases of DMD treatment. They reviewed published data on these substances to see if there was enough evidence for their safety and efficacy to be able to make recommendations.
The experts concluded the following:
Steroids are the only drugs that the experts have agreed can be recommended. Though some of the drugs mentioned in Box 4 are quite widely used, there is just not enough evidence to say whether these other supplements really work or not. It is important to discuss all medication with your doctor before you think about adding or stopping
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