A multicenter, double-blind, randomized trial of deflazacort versus prednisone in duchenne muscular dystrophy

Marco Domenico Bonifati, Giampietro Ruzza, Piero Bonometto, Angela Berardinelli, Kseniya Gorni, Simona Orcesi, Giovanni Lanzi, Corrado Angelini

Research output: Contribution to journalArticle

145 Citations (Scopus)

Abstract

We randomized 18 Duchenne muscular dystrophy (DMD) boys whose age ranged from 5,2 to 14.6 years (mean, 7.3 years) for treatment with either deflazacort (0.9 mg/kg/day) or prednisone (0.75 mg/kg/day) on the basis of age and functional score at the onset of treatment. We followed the patients every 3 months for 1 year, evaluating four limb muscles with the Medical Research Council scale and performance of four functions (walking, climbing stairs, Gowers' maneuver, and rising from a chair). Side effects were monitored by a questionnaire and by routine blood examination, and weight and height were recorded at each visit. At 12 months, the effect of both steroids was examined by comparing the status of the treated patients with another group of untreated DMD patients that served as natural history control. The two steroids were equally effective in improving motor function and functional performances. At 9 months, the average weight increase with respect to baseline value was 5% (2 kg) in the deflazacort group but 18% in the prednisone group (P <0.005), and the change remained significant after 12 months (P <0.05). Other minor but nonsignificant side effects were observed. Steroid treatment with deflazacort appears to cause fewer side effects than with prednisone, particularly weight gain, which could be important to maximize motor performances. (C) 2000 John Wiley and Sons, Inc.

Original languageEnglish
Pages (from-to)1344-1347
Number of pages4
JournalMuscle and Nerve
Volume23
Issue number9
DOIs
Publication statusPublished - 2000

Fingerprint

Duchenne Muscular Dystrophy
Prednisone
Steroids
Weights and Measures
Natural History
Weight Gain
Walking
Biomedical Research
Therapeutics
Extremities
Muscles
deflazacort

Keywords

  • Deflazacort
  • Duchenne muscular dystropiny
  • Prednisone
  • Steroid
  • Trial

ASJC Scopus subject areas

  • Clinical Neurology
  • Neuroscience(all)

Cite this

A multicenter, double-blind, randomized trial of deflazacort versus prednisone in duchenne muscular dystrophy. / Bonifati, Marco Domenico; Ruzza, Giampietro; Bonometto, Piero; Berardinelli, Angela; Gorni, Kseniya; Orcesi, Simona; Lanzi, Giovanni; Angelini, Corrado.

In: Muscle and Nerve, Vol. 23, No. 9, 2000, p. 1344-1347.

Research output: Contribution to journalArticle

Bonifati, Marco Domenico ; Ruzza, Giampietro ; Bonometto, Piero ; Berardinelli, Angela ; Gorni, Kseniya ; Orcesi, Simona ; Lanzi, Giovanni ; Angelini, Corrado. / A multicenter, double-blind, randomized trial of deflazacort versus prednisone in duchenne muscular dystrophy. In: Muscle and Nerve. 2000 ; Vol. 23, No. 9. pp. 1344-1347.
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