6533b836fe1ef96bd12a13a3

RESEARCH PRODUCT

Deflazacort in Duchenne dystrophy: Study of long-term effect

Corrado AngeliniC. CostaE TurellaElena PegoraroMt IntinoAntonella Pini

subject

medicine.medical_specialtyPatient DropoutsTime Factorsmedicine.drug_classPhysiologyDuchenne muscular dystrophymedicine.medical_treatmentMotor ActivityPlaceboMuscular Dystrophieslaw.inventionCellular and Molecular NeuroscienceDouble-Blind MethodRandomized controlled trialPregnenedioneslawPhysiology (medical)medicineHumansChildGaitChemotherapybusiness.industryMusclesAnti-Inflammatory Agents Non-SteroidalBody Weightmedicine.diseaseSurgeryClinical trialDeflazacortAnesthesiaCorticosteroidNeurology (clinical)medicine.symptombusinessWeight gainFollow-Up Studiesmedicine.drug

description

A randomized double-blind controlled trial of deflazacort was conducted in 28 Duchenne muscular dystrophy patients either treated with deflazacort 2.0 mg/kg alternate-day therapy or placebo. The deflazacort group showed significant improvement in climbing stairs (P < 0.01), in rising from a chair, Gower's maneuver, and walking (P < 0.0025) after 6 months of treatment. After 1 year, all the above changes remained significantly improved and the MRC index was significantly better (P < 0.05) in the treated group. After 2 years, a significant change was found in the MRC index: higher scores in walking, chair rising (P < 0.02), and grade and time of Gower's maneuver (P < 0.05) were found. The mean time for loss of ambulation for the treated group after we started the trial was 33.2 +/- 9 months; for the placebo group it was 20.5 +/- 11 months (deflazacort vs. placebo group, P < 0.05) [corrected]. Our treated patients lost their ambulation at a median age of 11.8 years vs. 10.5 years in the placebo group. Side effects were mild, consisting of moderate weight gain and slight behavioral changes.

yearjournalcountryeditionlanguage
1994-04-01Muscle &amp; Nerve
https://doi.org/10.1002/mus.880170726