What are the CDC guidelines for pharmacologic therapy for Duchenne muscular dystrophy (DMD)?

Updated: Aug 17, 2020
  • Author: Twee T Do, MD; Chief Editor: Jeffrey D Thomson, MD  more...
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Answer

The CDC guidelines recommend considering glucocorticoid therapy in all patients who have DMD, based on findings that glucocorticoids are the only medication available that effectively slows decline in muscle strength and function, offers reduced risk of scoliosis, and stabilizes pulmonary function. [72]

Additional recommendations for initiation of steroid therapy include the following:

  • Timing of initiation of glucocorticoid therapy must be individualized on the basis of functional state, age, and pre-existing risk factors for adverse effects
  • Recommended immunizations should be completed and varicella immunity established before therapy is initiated
  • Glucocorticoid therapy is not recommended for children still gaining motor skills, especially those under the age of 2 years
  • Once motor skills have plateaued (usually between ages 4-8), children should be started on steroids unless there are substantial reasons (eg, a major pre-existing risk factor for adverse effects) to delay therapy until motor skills decline
  • Initiation of steroids when the child’s condition is in full decline may provide limited benefits but is still recommended

Further CDC guidance on glucocorticoid regimes and dosing includes the following [72] :

  • Daily use is preferred over alternative regimens
  • Neither prednisone nor deflazacort has been proved superior in altering the decline in motor, respiratory, or cardiac functioning, but deflazacort may be preferred for some patients because of the lower risk of weight gain
  • Starting doses: Prednisone, 0.75 mg/kg daily; deflazacort, 0.9 mg/kg daily; higher doses have not been found more effective
  • The minimum effective dose that shows some benefit is believed to be 0.3 mg/kg daily for prednisone
  • The dose is increased as the child grows, as long as adverse effects are manageable and tolerable, until a weight of 40 kg is reached
  • The maximum daily dose is 40 mg for prednisone and 36-39 mg for deflazacort
  • For children who experience transient behavioral effects, administration of the medication after school may be preferred
  • Use of the anabolic steroid oxandrolone was not considered appropriate either with or without glucocorticoid therapy

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