Department of Physical Medicine & Rehabilitation, School of Medicine, University of California, Davis, 4860 Y Street, Suite 3850, Sacramento, California 95817, USA.
Muscle Nerve. 2013 Jul;48(1):32-54. doi: 10.1002/mus.23807. Epub 2013 May 16.
Contemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to assess care recommendations and aid in planning future trials.
The Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 individuals, aged 2-28 years, with DMD in a longitudinal, observational study at 20 centers. Assessments obtained every 3 months for 1 year, at 18 months, and annually thereafter included: clinical history; anthropometrics; goniometry; manual muscle testing; quantitative muscle strength; timed function tests; pulmonary function; and patient-reported outcomes/health-related quality-of-life instruments.
Glucocorticoid (GC) use at baseline was 62% present, 14% past, and 24% GC-naive. In those ≥6 years of age, 16% lost ambulation over the first 12 months (mean age 10.8 years).
Detailed information on the study methodology of the CINRG DMD-NHS lays the groundwork for future analyses of prospective longitudinal natural history data. These data will assist investigators in designing clinical trials of novel therapeutics.
为评估护理建议和辅助未来试验规划，需要有杜氏肌营养不良症（DMD）的当代自然病史数据。
合作国际神经肌肉研究组（CINRG）DMD 自然病史研究（DMD-NHS）在 20 个中心开展了一项纵向观察性研究，纳入了 340 名年龄在 2 至 28 岁之间的 DMD 患者。在 1 年、18 个月和之后每年，进行以下评估：临床病史、人体测量学、关节角度测量、徒手肌肉测试、定量肌肉力量、计时功能测试、肺功能和患者报告的结果/健康相关生活质量工具。
基线时使用糖皮质激素（GC）的比例为 62%，过去使用的比例为 14%，GC 初治的比例为 24%。在≥6 岁的患者中，有 16%在最初 12 个月内丧失了步行能力（平均年龄为 10.8 岁）。
CINRG DMD-NHS 的研究方法的详细信息为未来分析前瞻性纵向自然病史数据奠定了基础。这些数据将有助于研究人员设计新型治疗药物的临床试验。
