Deflazacort for the treatment of Duchenne Dystrophy: a systematic review.
BACKGROUND
To complete a systematic review and meta-analysis based on the clinical question: Is Deflazacort (DFZ), a prednisolone derivative, an effective therapy for improving strength, with acceptable side effects, in children with Duchenne Dystrophy (DD)?


METHODS
MEDLINE, EMBASE, Current Contents, Dissertation Abstracts, Health Star, PsychINFO and Cochrane, were searched using the following inclusion criteria: 1) A randomized controlled trial comparing DFZ with placebo or another therapy; 2) Male participants age 2-18 years with DD; 3) Outcomes of (a) any form of strength or functional testing, or (b) any form of side effect.


RESULTS
Fifteen studies of potential relevance were identified, with five meeting the inclusion criteria. These five studies included 291 children and were published in English language journals between 1994 and 2000. Two studies compared DFZ versus placebo, two studies compared DFZ with prednisone and one study had both placebo and prednisone comparisions. Two large trials were identified that have not been published in article format. Due to the heterogeneity in outcome measures and the inconsistent reporting of summary statistics a meta-analytic approach could not be taken.


CONCLUSIONS
Examining individual studies it appears that DFZ improves strength and functional outcomes compared to placebo, but it remains unclear if it has a benefit over prednisone on similar outcomes. Two trials found that DFZ causes less weight gain than prednisone.
Campbell
Craig
C
Division of Neurology, Children's Hospital of Eastern Ontario, University of Ottawa, Ottawa, Canada. craig.campbell@lhsc.on.ca
Jacob
Pierre
P
D000293:Adolescent; D000893:Anti-Inflammatory Agents / Q000009:adverse effects / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D009119:Muscle Contraction / Q000187:drug effects; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy*; D011241:Prednisone / Q000009:adverse effects / Q000627:therapeutic use; D011282:Pregnenediones / Q000009:adverse effects / Q000627:therapeutic use*; D016032:Randomized Controlled Trials as Topic / Q000706:statistics & numerical data; D016896:Treatment Outcome; D015430:Weight Gain / Q000187:drug effects
D000893:Anti-Inflammatory Agents; D007166:Immunosuppressive Agents; D011282:Pregnenediones; C021988:deflazacort; D011241:Prednisone