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NEUROMUSCULAR MANAGEMENT - Maintaining strength and function

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

  • Because your son does not have dystrophin his muscles will gradually get weaker.
Some types of exercise and getting tired can make muscles damage worse.
  • The doctor understands the progression of muscle weakness and can help your son get ready for the next step.
  • It is important for the doctor to know how your son muscles are working so that they can start the right therapy as early as possible.

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.
THE BASICS

  • Steroids (also called glucocorticoids or corticosteroids) are the only drugs known to slow the decline in muscle strength and motor function in DMD. The goal of steroid use is to help the child walk independently

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).

  • Prevention and management of steroid side effects needs to be proactive and anticipatory. Interventions should be put in place EARLY in an effort to prevent problems and to make sure they do not become severe. Side effects associated with steroid use vary and are listed in Table 1.

STARTING AND STOPPING STEROIDS

  • The optimal time for starting steroid treatment is when motor function is in a “plateau phase” – when the boy’s motor skills have stopped improving, but have not yet started to get worse. This is normally sometime between the age of 4-6 years. It is not recommended to start steroids in children who are still gaining motor skills, especially if they are under 2 years of age.

*The recommended national vaccination schedule should be complete before steroid treatment is started, and varicella (chicken pox) immunity should be established.

  • Starting steroid treatment in boys/young men who are no longer walking independently is a matter for individual decision and needs to be discussed with the doctor, taking into consideration the effect of pre-existing risk factors.

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:

  • Steroids are only medicines known to help slow down the muscles weakness.
  • Always tell your doctors and other healthcare provides that your son is taking steroids. It is especially important if he is having surgery or has an infection because steroids can suppress the system.
  • Your son should never stop taking steroids suddenly.
  • Your son should have regular visits with a doctor who is skilled in managing steroids. The doctor will explain possible side effects and tell you if your son is at risk of developing them.

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 .

  • Prednisone (prednisolone) and deflazacort are the two types of steroids that are mainly

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.

  • Starting with daily use of a steroid was preferred by the experts to the alternative regimes. Data from ongoing and future studies may modify this recommendation.

DOSES FOR STARTING AND MAINTAINING STEROIDS

  • The recommended starting dose for prednisone is 0.75 mg/kg/day and that of deflazacort is 0.9 mg/kg/day,give in the morning, some children experience short- lived behavioral side effects(hyperactivity, mood swings) for a few hours after the medication is given. For these children administration of the medication in the afternoon may be alleviate some of these difficulties.
  • For ambulatory individuals, the dosage is commonly increased as the child grows until he reaches approximately 40kg in weight. The maximum dose of prednisone is usally capped at approximately 30mg/day, and that of deflazacort at 36mg/day.
  • Non-ambulatory teenagers maintained on long-term steroid therapy are usally above 40kg in weight and the prednisone dosage per kg is often allowed to drift down to the 0.3 to 0.6mg/kg/day range. While this dosage is less than the appromimate 30mg cap, it demonstrates substanitial benefit.
  • Deciding on the maintenance dose of streroids is a balance between growth, how good the response to steroids is and is and the burden of side effects. So this decision needs to be reviewed at every clinic visit based on the result of the tests done and whether or not side effects are a problem that can’t be managed or tolerated.
  • In boys on a relatively low dosage of steroid (less than the starting dose per kg body weight) who start to show functional decline, it is adjustment. The doseage of steroids is increased to the target and individual is then re-evaluated for any benefit in approximately two to three months.
  • There is no consensus on optimal steroid dosage if initiated in the non-ambulatory individual. Nor is it known how effective steroid treatment is in preventing scoilosis or in stablisingg cardiac or respiratory function in this setting. This issue warrants further study.

BOX 3

MANAGEMENT OF STEROID MEDICATION

  • A dose reduction of approximately to 1/3 is suggested if intolerable or non-manageable side effects occur, with reassessment by phone or clinicial visit in one month to assess control side effects.
  • If a daily dosing schedule results in unmanageable and/or intolerable side effects that do not improve when the dose is reduced, then it is appropriate to change to an alternative regime.
  • Steroid therapyshould not be abandoned even if side effects are NOT manageable and/or tolerable until at least one dosage reduction and change to an alternative regime have been pursued. This recommendation holds for both ambulatory and non-ambulatory individuals.

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.

BOX 4
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:

  • The use of oxandrolone, an anabolic steroid, is not recommended.
  • Safety in the use of botox has not been studiedfor treatment or preventation of contractures in individuals with DMD and is not recommmeded.
  • There was no support for the systemic use of creatine. A ramised controlled trial of creatine in DMD did not show a clear benefit. If a person is taking creatine and has evidence of kidney problems, it is necessary to discontinue the supplement.
  • No recommendations can be at this time about other supplements or other drugs that are sometimes used in DMD treatment, including co-enzyme Q10, carnitine, amino acids (glutamine, arginine), anti- inflammatories/anti-oxidants(fish oil. vitamin E, green tea extract,pentoxifyline)and others including herbal or botanical extract. The experts concluded that there is not enough evidence in the published literature.
  • The experts agreed that this is an area where additional research is needed. Active involvement of families in activies that develop further knowledge, such as patient registries and clinal trials, was encouraged.

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
medication.

Tags

Resources

  • THE TREAT NMD GUIDE : Bushby K et al.The diagnosis and management of duchenne muscular dystrophy, part 1: diagnosis and pharmacological and psychosocial management , lancet Neurology 2010,9(1) 77-93.
  • THE TREAT NMD GUIDE : Bushby K et al. The diagnosis and management of duchenne muscular dystrophy, part 2: implementation of multidisciplinary care, lancet Neurology 2010,9(2) 177-189.

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