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.

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