Deflazacort in Duchenne muscular dystrophy: a comparison of two different protocols.
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.
Biggar
W D
WD
Bloorview MacMillan Children's Centre, 150 Kilgour Road, Toronto, Ont. M4G 1R8, Canada. dbiggar@bloorviewmacmillan.on.ca
Politano
L
L
Harris
V A
VA
Passamano
L
L
Vajsar
J
J
Alman
B
B
Palladino
A
A
Comi
L I
LI
Nigro
G
G
D000293:Adolescent; D001827:Body Height / Q000187:drug effects; D001835:Body Weight / Q000187:drug effects; D002118:Calcium / Q000627:therapeutic use; D016022:Case-Control Studies; D002386:Cataract / Q000139:chemically induced; D002648:Child; D002985:Clinical Protocols; D019587:Dietary Supplements; D004334:Drug Administration Schedule; D005500:Follow-Up Studies; D050723:Fractures, Bone / Q000139:chemically induced; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D009043:Motor Activity / Q000187:drug effects; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy*; D011282:Pregnenediones / Q000009:adverse effects / Q000627:therapeutic use*; D011597:Psychomotor Performance / Q000187:drug effects; D012600:Scoliosis / Q000139:chemically induced; D016896:Treatment Outcome; D014807:Vitamin D / Q000627:therapeutic use
D007166:Immunosuppressive Agents; D011282:Pregnenediones; D014807:Vitamin D; C021988:deflazacort; D002118:Calcium