corticosteroids: what parents need to know - by prof. doug biggar
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Corticosteroids - What parents need to knowTRANSCRIPT
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
• stop around 10 years old
No corticosteroids
With corticosteroids
• walk 3-5 years longer
Preserved
Pulmonary Function
PULMONARY FUNCTION
0
20
40
60
80
100
10 15 18
FV
C P
erce
nt
Pre
dic
ted
Untreated
Treated
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
Cardiac Function
Delayed Scoliosis
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
Scoliosis Surgery in DMD
What about bones?
What makes bones healthy?
• Genetics 70-80%
• Physical activity
• 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.
Benefits of must be better than the side effects
The other bottom line:
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.
תודה