Issuu on Google+

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 • stop around 10 years old

With corticosteroids • walk 3-5 years longer


Preserved Pulmonary Function


PULMONARY FUNCTION FVC Percent Predicted

100 80 60

Untreated Treated

40 20 0 10

15 Age in years

18


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


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? •

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.


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.


‫תודה‬


Corticosteroids: What parents need to know - by Prof. Doug Biggar