Deflazacort in Duchenne muscular dystrophy: a comparison of two different protocols

Neuromuscul Disord. 2004 Sep;14(8-9):476-82. doi: 10.1016/j.nmd.2004.05.001.


We compare the long-term benefits and side effects of deflazacort using two treatment protocols from Naples (N) and Toronto (T). Boys with Duchenne muscular dystrophy between the ages of 8 and 15 years and who had four or more years of deflazacort treatment were reviewed. Diagnostic criteria included males with proximal muscle weakness evident before 5 years, increased serum creatine kinase and genetic testing and/or a muscle biopsy consistent with Duchenne muscular dystrophy. Thirty-seven boys were treated with protocol-N using deflazacort at a dose of 0.6 mg/kg per day for the first 20 days of the month and no deflazacort for the remainder of the month. Boys with osteoporosis received daily vitamin D and calcium. Deflazacort treatment started between 4 and 8 years of age. Thirty-two were treated with protocol-T using deflazacort at a dose of 0.9 mg/kg per day, plus daily vitamin D and calcium. Treatment started between 6 and 8 years of age. All boys were monitored every 4-6 months. The results were compared with age-matched controls in the two groups (19 for protocol-N and 30 for protocol-T). For the boys treated with protocol-N, 97% were ambulatory at 9 years (control, 22%), 35% at 12 years (control, 0%), 25% at 15 years (control, 0%). For the 32 boys treated with protocol-T, 100% were ambulatory at 9 years (control, 48%), 83% at 12 years (control, 0%) and 77% at 15 years (control, 0%). No aids or leg braces were used for ambulation. In boys 13 years and older, a scoliosis of >20 degrees developed in 30% of the boys on protocol-N, 16% on protocol-T and 90% of controls. For protocol-N, no cataracts were observed while in protocol-T, 30% of boys had asymptomatic cataracts that required no treatment. Fractures occurred in 19% (control 16%) of boys on protocol-N and 16% (control, 20%) of boys on protocol-T. This report illustrates: (a) the importance of collaborative studies in developing treatment protocols in Duchenne muscular dystrophy and (b) the long-term beneficial effects of deflazacort treatment in both protocols. However, the protocol-T seems to be more effective and frequently is associated with asymptomatic cataracts.

Publication types

  • Clinical Trial
  • Comparative Study

MeSH terms

  • Adolescent
  • Body Height / drug effects
  • Body Weight / drug effects
  • Calcium / therapeutic use
  • Case-Control Studies
  • Cataract / chemically induced
  • Child
  • Clinical Protocols*
  • Dietary Supplements
  • Drug Administration Schedule
  • Follow-Up Studies
  • Fractures, Bone / chemically induced
  • Humans
  • Immunosuppressive Agents / adverse effects
  • Immunosuppressive Agents / therapeutic use*
  • Male
  • Motor Activity / drug effects
  • Muscular Dystrophy, Duchenne / drug therapy*
  • Pregnenediones / adverse effects
  • Pregnenediones / therapeutic use*
  • Psychomotor Performance / drug effects
  • Scoliosis / chemically induced
  • Treatment Outcome
  • Vitamin D / therapeutic use


  • Immunosuppressive Agents
  • Pregnenediones
  • Vitamin D
  • deflazacort
  • Calcium