Corticosteroids: What parents need to know!
“Little Steps”, Israel, 2011 Doug Biggar, MD Toronto, Canada
The Case for Corticosteroids • Are they really the gold standard?
• Some of the risks and benefits
• The need for more studies
Why Corticosteroids in DMD? We have known for more that 30 years that they: • reduce muscle weakness and • preserve motor function but …..taken daily is best and • side effects common
Corticosteroids in DMD
References: The Diagnosis and Management of DMD. K. Bushby and the DMD Care Working Group/CDC Lancet Neurology Jan/Feb 2010.
Corticosteroids in DMD So we all agree that: • Corticosteroids help muscle strength • They slow the progression of muscle weakness • Best to start when boys are walking • Best to give once a day, every day if you can • Balanced discussion of benefits and risks • More studies are needed
What are the benefits? Short-term • Some boys, but not all have more energy Long-term • Walking • Breathing • Heart • Spine • Arm strength
What are the Risks? Variable and can include: • Weight gain • Behavior • Shorter
What are the Risks?
What are the Risks? Variable and can include: • Weight gain • Behavior • Shorter • Bone health • Cataracts • Delayed puberty • And more….
Preserved Muscle Function
Walking No corticosteroids â€˘ stop around 10 years old
With corticosteroids â€˘ walk 3-5 years longer
Preserved Pulmonary Function
PULMONARY FUNCTION FVC Percent Predicted
100 80 60
40 20 0 10
15 Age in years
Pulmonary Function At 18 years, my son on steroids should have:
A better cough • Fewer lung infections
• Fewer admissions to hospital for pneumonia • Very few need night time ventilation
Preserved Cardiac Function
Scoliosis Surgery in DMD
No steroids •
90% boys done around 15 years
Need help feeding after surgery
10% of spines stay straight
Scoliosis Surgery in DMD Steroid treated: •
Only 10%, not 90%, of boys done at 15-
17 years •
Can self feed after surgery
What about bones?
What makes bones healthy? •
Other things: calcium, vitamin D etc
What about my son’s bones? • Even young boys have reduced bone density • Worse with less walking
• ~35% break a long bone (leg or arm), with or without steroids • Vertebral (back bone) fractures only occur on steroids
Soooo….. Probably not worse with deflazacort Risk • • • •
of fractures: long bones vertebrae Can be treated, if needed More research needed
So what is the bottom line?
This is one bottom line.
The other bottom line: Benefits of must be better than the side effects Major benefits: • Skeletal muscles preserved • Pulmonary • cardiac
• Scoliosis • Upper extremity
Bottom Line: Common side effects: • • • • • •
Weight Height Cataracts Delayed puberty Vertebral fragility fractures Difficult behaviour
Now it is your choice….
Steroids aren’t perfect but they are the best we have.
Corticosteroids - What parents need to know