ACTION DUCHENNE INTERNATIONAL CONFERENCE 2015 Steroids in Duchenne muscular dystrophy 1. The use of steroids in DMD. 2. FOR-DMD steroid trial, does it still matter? 3. Research update on steroids Dr Henriette van Ruiten Clinical Research Associate The John Walton Muscular Dystrophy Research Centre Newcastle upon Tyne
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ACTION DUCHENNE INTERNATIONAL CONFERENCE 2015 Steroids in Duchenne muscular dystrophy 1.The use of steroids in DMD. 2.FOR-DMD steroid trial, does it still.
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ACTION DUCHENNE INTERNATIONAL CONFERENCE
2015
Steroids in Duchenne muscular dystrophy
1. The use of steroids in DMD.2. FOR-DMD steroid trial, does it still matter?3. Research update on steroids
Dr Henriette van RuitenClinical Research Associate The John Walton Muscular Dystrophy Research CentreNewcastle upon Tyne
• Affects 1 in 3500 newborns
• In UK,
• 100 boys are born with DMD each year
• There are 2500 patients with DMD
• Caused by mutation in the dystrophin gene,
• Absence of dystrophin in muscle fibres
• Clinically characterised by progressive muscle wasting
Duchenne Muscular Dystrophy (DMD)
Current treatments
Pharmacological treatments
Treatment of complications
Respiratory care
Cardiac management
Orthotic and orthopaedic approach
Pharmacological treatments
Steroids• Also called corticosteroids or
glucocorticoids. • Man made version of hormones normally
produced by the adrenal glands• Steroids are mainly used to reduce
inflammation and suppress immune system
• Used in conditions such as asthma, arthritis, eczema etc)Why are steroids used in DMD?
• Only medication currently available for all which slows the decline in muscle strength in DMD
Side effects: Behaviour problems Weight gain Cushingoid appearance Vertebral fractures Immunosuppression Short stature Delayed puberty Cataracts Hypertension GI upset
Side effects of steroids
Are significant Require proactive
management
• Behavioural guidance• Prophylaxis/treatment of
osteoporosis• Regular follow up (BP,
glycosuria, eye check, physical exam)
• Flue jabs• Avoid live vaccines
Considerable but predictable
Starting steroids
Around the plateau phase of motor function. This usually happens around the age of 4-5yrs old.
With time steroid dose is adjusted according to benefits and side effects
Try and prevent/treat side effects before reducing dose
In older less ambulant boys steroids may still be of benefit; eg in improving upper body strength, reducing the risk of scoliosis and stabilizing respiratory function.
The age to stop steroids needs to be decided individually. It may be necessary to change or stop the steroids because of side effects. In other situations, they can be used for many years, beyond the age at loss of ambulation.
FOR DMD trial - update Recruitment target = 225 boys End of recruitment is august 2016 Current no of boys enrolled = 156 Boys can be in exon skipping trial AND FOR DMD Results expected 2018 Results will provide evidence based guidance on
best steroid treatment in DMD Standardise steroid treatment Important for current and future clinical trials