Other drugs and dietary supplements
The experts considered a range of other drugs and supplements that are known to be used in some cases for 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 studied for the treatment or prevention of contractures in individuals with DMD and is not recommended.
- There was no support for the systematic use of creatine. A randomised 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 this supplement.
- No recommendations can be made at this time about other supplements or other drugs that are sometimes used in DMD treatment, including coenzyme Q10, carnitine, amino acids (glutamine, arginine), anti-inflammatories/anti-oxidants (fish oil, vitamin E, green tea extract, pentoxifylline), and others including herbal or botanical extracts. The experts concluded that there was 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 activities that develop further knowledge, such as patient registries and clinical trials, was encouraged.
Steroids are the only drugs that the experts have agreed can be recommended. Though some of the drugs mentioned above 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.
The steroid side effects table summarises the main side effects of steroids that should be monitored and useful interventions. Factors to take into account in maintaining or increasing dose are response to therapy, weight and growth, and whether side effects are present and manageable.