Friday 11 December 2009
World’s leading Duchenne experts agree comprehensive treatment guidelines
Listen to the podcast on the Nationwide Children's Hospital website which interviews Prof. Kate Bushby about this research.
Comprehensive guidelines to improve the multidisciplinary care of individuals with Duchenne muscular dystrophy have been published after a three-year consultation process that involved 84 of the world's leading Duchenne muscular dystrophy experts. The new guidelines are needed because health care for boys with Duchenne muscular dystrophy has been reported to be variable and inconsistent. The guidelines will carry more weight than similar guidelines previously produced by smaller groups of authors or individual patient advocacy groups. The guidelines will be used as the basis for a comprehensive family guide, to be published in early 2010.
Contents
- How were the guidelines created?
- What do the guidelines cover?
- What does this mean for patients?
- Further information and links
How were the guidelines created?
The new guidelines were largely based on the collective judgement of experts, in addition to data from randomised controlled trials where available. The experts were presented with patient care scenarios and then asked to rate various interventions or assessments as 'necessary', 'appropriate' or 'inappropriate'. Areas of disagreement or uncertainty are highlighted in the guidelines, as are areas where more research is needed before recommendations can be made.
What do the guidelines cover?
The guidelines cover nearly every field of treatment involved in the care of individuals with Duchenne muscular dystrophy, including diagnosis, corticosteroid management, physical and occupational therapy, mental and social wellbeing, speech therapy, diet, and care of the heart, gut, bones, joints, tendons and lungs.
For example, in the guidelines doctors are urged to consider corticosteroid therapy (often known as 'steroid treatment') in all patients with Duchenne muscular dystrophy, with recommendations made regarding the timing and doses to be used at different stages of the condition. How to treat the side-effects of corticosteroids is also outlined, and treatments are recommended for all of the symptoms of the condition such as contractures and breathing difficulties. Also, the guidelines recommend that interventions to aid mental and social wellbeing be put at the centre of management.
The guidelines stress the importance of a multidisciplinary approach in the care of individuals with Duchenne muscular dystrophy and encourage patients and families to actively engage with the medical professional who coordinates their clinical care.
What does this mean for patients?
By providing recognised benchmarks for the care of patients with Duchenne muscular dystrophy, this review will help raise the standard of care that patients receive. Of course they will never replace an experienced clinician, but the review acts as a guide for physicians, patients and families so they know what treatment people with Duchenne muscular dystrophy should receive at every stage of the disease.
The new guidelines will be a valuable tool for people lobbying at a national level to improve the standards of care received by people with Duchenne muscular dystrophy. Furthermore, if the guidelines are used to standardise patient care worldwide, this will make it easier to conduct international clinical trials for Duchenne muscular dystrophy because it will be easier to compare trial participants in different countries.
Looking to the future, the Muscular Dystrophy Campaign will be working with TREAT-NMD, healthcare professionals and health authorities to establish the best ways of implementing these recommendations and ensuring that everyone with Duchenne muscular dystrophy has access to best-practice care.
Professor Kate Bushby, Managing Editor for the review, said:
Care for Duchenne muscular dystrophy is patchy and not always delivered as it should be. This problem was recognised on both sides of the Atlantic, with support for the generation of these guidelines coming from the Centers for Disease Control in the USA as well as the TREAT-NMD network in Europe.
The generation of these guidelines brought together a group of experts of unprecedented size from the whole range of different specialties to present a full picture of what is needed to look after someone with Duchenne muscular dystrophy. Their dissemination and implementation will hopefully raise standards across the board. We look forward to working with the Muscular Dystrophy Campaign and other organisations to ensure they are appropriately applied across the UK.
Further information and links
More information on the new guidelines from TREAT-NMD.
The two full original review papers were published in the Lancet Neurology. The articles are written in technical language with no summary in layman's terms. You can access the papers via the TREAT-NMD website.
More information about Duchenne muscular dystrophy.
