Masahide Goto1, Hirofumi Komaki2, Eri Takeshita1, Yoshiki Abe3, Akihiko Ishiyama1, Kenji Sugai1, Masayuki Sasaki1, Yu-Ichi Goto4, Ikuya Nonaka5. 1. Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan. 2. Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, Tokyo, Japan. Electronic address: komakih@ncnp.go.jp. 3. Faculty of Medicine, University of Tsukuba, Tsukuba, Japan. 4. Department of Mental Retardation and Birth Defect Research, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan. 5. Department of Neuromuscular Research, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
Abstract
INTRODUCTION: Corticosteroids are effective for improving motor function in patients with Duchenne muscular dystrophy (DMD), but there is no consensus on a regimen that balances efficacy and side effects. METHODS: Data from three groups of DMD patients were retrospectively analyzed: those treated with 0.75mg/kg/day prednisolone every day (daily group, n=51); those treated with 1mg/kg/day prednisolone on alternate days (intermittent group, n=36), and those not treated with steroids (nontreatment group, n=42). RESULTS: Although the age of ambulation loss did not differ significantly among the groups, the hazard ratios for ambulation loss relative to the nontreatment group were 0.24 (95% confidence interval [CI]: 0.11-0.54) in the daily group and 0.34 (95% CI: 0.19-0.62) in the intermittent group. The percentage of predicted forced vital capacity increased until 9.6years of age (to 94.1%) in the daily group, until 8.8years of age (to 96.9%) in the intermittent group, and until 7.2years of age (to 87.6%) in the nontreatment group. Weight gain was the most frequently observed side effect in the treated groups. Height was significantly lower in the daily than in the nontreatment group. Other side effects were observed, but no patient discontinued therapy. There were no marked differences in benefits and side effects between the two treated groups. DISCUSSION: This is the first assessment of long-term outcomes of different steroid therapy regimens in Japanese DMD patients. Benefits and side effects, except height, did not differ significantly between steroid regimens.
INTRODUCTION: Corticosteroids are effective for improving motor function in patients with Duchenne muscular dystrophy (DMD), but there is no consensus on a regimen that balances efficacy and side effects. METHODS: Data from three groups of DMDpatients were retrospectively analyzed: those treated with 0.75mg/kg/day prednisolone every day (daily group, n=51); those treated with 1mg/kg/day prednisolone on alternate days (intermittent group, n=36), and those not treated with steroids (nontreatment group, n=42). RESULTS: Although the age of ambulation loss did not differ significantly among the groups, the hazard ratios for ambulation loss relative to the nontreatment group were 0.24 (95% confidence interval [CI]: 0.11-0.54) in the daily group and 0.34 (95% CI: 0.19-0.62) in the intermittent group. The percentage of predicted forced vital capacity increased until 9.6years of age (to 94.1%) in the daily group, until 8.8years of age (to 96.9%) in the intermittent group, and until 7.2years of age (to 87.6%) in the nontreatment group. Weight gain was the most frequently observed side effect in the treated groups. Height was significantly lower in the daily than in the nontreatment group. Other side effects were observed, but no patient discontinued therapy. There were no marked differences in benefits and side effects between the two treated groups. DISCUSSION: This is the first assessment of long-term outcomes of different steroid therapy regimens in Japanese DMDpatients. Benefits and side effects, except height, did not differ significantly between steroid regimens.
Authors: Tatiana M Fontelonga; Brennan Jordan; Andreia M Nunes; Pamela Barraza-Flores; Nicholas Bolden; Ryan D Wuebbles; Lesley Mathews Griner; Xin Hu; Marc Ferrer; Juan Marugan; Noel Southall; Dean J Burkin Journal: Hum Mol Genet Date: 2019-07-01 Impact factor: 6.150
Authors: Erika Finanger; Krista Vandenborne; Richard S Finkel; H Lee Sweeney; Gihan Tennekoon; Sabrina Yum; Maria Mancini; Pradeep Bista; Andrew Nichols; Hanlan Liu; Angelika Fretzen; Joanne M Donovan Journal: J Neuromuscul Dis Date: 2019