杜氏肌营养不良症中皮质类固醇治疗的新方法。
Novel approaches to corticosteroid treatment in Duchenne muscular dystrophy.
作者信息
Hoffman Eric P, Reeves Erica, Damsker Jesse, Nagaraju Kanneboyina, McCall John M, Connor Edward M, Bushby Kate
机构信息
Center for Genetic Medicine Research, Children's National Medical Center, Washington, DC 20010, USA.
出版信息
Phys Med Rehabil Clin N Am. 2012 Nov;23(4):821-8. doi: 10.1016/j.pmr.2012.08.003.
Abstract
Although prednisone has never been formally approved for use in Duchenne muscular dystrophy (DMD) by regulatory agencies, its efficacy has been confirmed in trials dating from the 1980s. There is a strong need for optimization of both specific type of glucocorticoid (eg, prednisone, vs deflazacort or others) and the dosing regimen. Ideally an optimized regimen would maximize efficacy while minimizing side-effect profiles. A new trial, FOR-DMD, aims to address this gap in knowledge. In parallel, there has been progress in the area of "dissociative steroids," drugs that are able to better separate efficacy and side effects, providing a broader therapeutic window.
摘要
尽管泼尼松从未被监管机构正式批准用于杜氏肌营养不良症（DMD），但其疗效在20世纪80年代以来的试验中得到了证实。对于优化糖皮质激素的特定类型（如泼尼松与地夫可特或其他药物）和给药方案有强烈需求。理想情况下，优化方案应在使副作用最小化的同时最大化疗效。一项名为FOR-DMD的新试验旨在填补这一知识空白。与此同时，“解离类固醇”领域取得了进展，这类药物能够更好地分离疗效和副作用，提供更宽的治疗窗口。
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