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在以人群为基础的杜氏和贝克型肌营养不良男孩队列中使用皮质类固醇。

Use of corticosteroids in a population-based cohort of boys with duchenne and becker muscular dystrophy.

作者信息

Matthews Dennis J, James Katherine A, Miller Lisa A, Pandya Shree, Campbell Kimberly A, Ciafaloni Emma, Mathews Katherine D, Miller Timothy M, Cunniff Christopher, Meaney F John, Druschel Charlotte M, Romitti Paul A, Fox Deborah J

机构信息

Department of Physical Medicine & Rehabilitation, University of Colorado School of Medicine and The Children's Hospital, Denver, CO 80045, USA.

出版信息

J Child Neurol. 2010 Nov;25(11):1319-24. doi: 10.1177/0883073810362762. Epub 2010 Mar 5.

DOI:10.1177/0883073810362762
PMID:20207610
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC5882203/
Abstract

The use of corticosteroids for treatment of Duchenne and Becker muscular dystrophy in clinical practice from 1991 through 2005 was reviewed in a large population-based cohort (MD STARnet) of boys in 4 regional sites and 6 clinics of the United States. Corticosteroid use increased from 20% (11 of 56 individuals) in 1991 to 44% (93 of 218 individuals) in 2005. Average use varied by site and ranged from 15% to 49%. The median age of corticosteroid initiation was 6.9 years (range, 3.7-17.4 years). Dosage and growth information was available for 102 participants and showed a median dose as 0.729 mg/kg for prednisone and 0.831 mg/kg for deflazacort. T. The most common reasons that corticosteroids were discontinued included weight gain, behavioral side effects, and loss of ambulation, resulting in full-time wheelchair use. Substantial variations in clinical practice were identified among study sites.

摘要

对1991年至2005年期间美国4个地区和6个诊所的一个以人群为基础的大型队列(MD STARnet)中的男孩,在临床实践中使用皮质类固醇治疗杜氏和贝克肌营养不良症的情况进行了回顾。皮质类固醇的使用从1991年的20%(56人中的11人)增加到2005年的44%(218人中的93人)。平均使用率因地点而异,范围从15%到49%。开始使用皮质类固醇的中位年龄为6.9岁(范围3.7 - 17.4岁)。有102名参与者提供了剂量和生长信息,显示泼尼松的中位剂量为0.729mg/kg,地夫可特为0.831mg/kg。停用皮质类固醇最常见的原因包括体重增加、行为副作用和失去行走能力,导致需要全天使用轮椅。研究地点之间在临床实践中发现了很大差异。

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本文引用的文献

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2
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Neurology. 2007 May 8;68(19):1607-13. doi: 10.1212/01.wnl.0000260974.41514.83.
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The muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet): surveillance methodology.肌肉萎缩症监测、跟踪与研究网络(MD STARnet):监测方法
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