Steroids in Duchenne muscular dystrophy--deflazacort trial.
We conducted a double blind controlled trial in 28 Duchenne muscular dystrophy (DMD) patients with Deflazacort (DF), an oxazoline derivative of prednisolone which reduces its side-effects. Myometric muscle strength measurements, Scott Score and timed tests showed statistically significant improvement for the treated group (P less than 0.05). Side-effects after 9 months of treatment included mild cushingoid appearance in four patients (28%) and moderate in only one (7%), increased appetite in seven (50%), increased body hair in four (28%), irritability and hyperactivity in three (21%). Increased body weight was not prominent and was controlled with dietary measures. No patient had to be withdrawn from medication. More research and long-term follow-up are needed in order to establish the mechanism of improvement and the consequences of long-term steroid administration in DMD. In this regard DF appears as an alternative to prednisone preserving its benefits but with fewer side-effects.
Mesa
L E
LE
Sección de Enfermedades Neuromusculares, Hospital Frances, Buenos Aires, Argentina.
Dubrovsky
A L
AL
Corderi
J
J
Marco
P
P
Flores
D
D
D000893:Anti-Inflammatory Agents / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D004311:Double-Blind Method; D006801:Humans; D009136:Muscular Dystrophies / Q000188:drug therapy*; D011239:Prednisolone / Q000009:adverse effects* / Q000037:antagonists & inhibitors; D011282:Pregnenediones / Q000009:adverse effects / Q000627:therapeutic use*
D000893:Anti-Inflammatory Agents; D011282:Pregnenediones; D011239:Prednisolone; C021988:deflazacort