Analysis Group, Inc., Boston, MA 02199, USA.
Cincinnati Children's Hospital Medical Center & Department of Pediatrics, College of Medicine, University of Cincinnati, Cincinnati, OH 45229, USA.
J Comp Eff Res. 2020 Feb;9(3):177-189. doi: 10.2217/cer-2019-0170. Epub 2020 Jan 10.
To assess outcomes among patients with Duchenne muscular dystrophy receiving deflazacort or prednisone in real-world practice. Clinical data for 435 boys with Duchenne muscular dystrophy from Cincinnati Children's Hospital Medical Center were studied retrospectively using time-to-event and regression analyses. Median ages at loss of ambulation were 15.6 and 13.5 years among deflazacort- and prednisone-initiated patients, respectively. Deflazacort was also associated with a lower risk of scoliosis and better ambulatory function, greater % lean body mass, shorter stature and lower weight, after adjusting for age and steroid duration. No differences were observed in whole body bone mineral density or left ventricular ejection fraction. This single center study adds to the real-world evidence associating deflazacort with improved clinical outcomes.
评估在真实世界实践中接受地夫可特或泼尼松龙治疗的杜氏肌营养不良症患者的结局。 使用生存时间和回归分析对辛辛那提儿童医院医疗中心的 435 名杜氏肌营养不良症男孩的临床数据进行回顾性研究。 分别接受地夫可特和泼尼松龙治疗的患者丧失行走能力的中位年龄分别为 15.6 岁和 13.5 岁。 在调整年龄和类固醇持续时间后，地夫可特还与较低的脊柱侧凸风险和更好的行走功能、更高的瘦体重百分比、更矮的身高和更低的体重相关。 全身骨矿物质密度或左心室射血分数无差异。 这项单中心研究增加了将地夫可特与改善临床结局相关联的真实世界证据。
