Abstract：
Although there is only symptomatic treatment for Fukuyama congenital muscular dystrophy (FCMD), several reports have suggested that steroid therapy could be effective for FCMD; however, no independent intervention studies have been conducted. This study aimed to evaluate the efficacy of steroid therapy for restoring motor functions in FCMD patients. This study involved 3-to-10-year-old FCMD patients who exhibited a decline in motor functions, requested steroid therapy. Patients with consent started oral administration of 0.5-mg/kg prednisolone every alternate day, which was increased to 1.0 mg/kg if the response was inadequate. We used the Gross Motor Function Measure (GMFM) to evaluate and compare the motor functions of all patients. Wilcoxon signed-rank test (significance level, P ≤ 0.05) was used for statistical analysis. At the onset of steroid therapy, 8.10 years (SD, 2.14 years) was the mean age of FCMD patients. The mean GMFM difference between before and after the steroid therapy was + 1.23 (SD, 1.10), and a P value of 0.015 represented significant improvement in GMFM. Our results indicate that steroid therapy may contribute to the maintenance and improvement of the motor functions of advanced-stage FCMD patients.Clinical Trial Registration Registration Number: UMIN000020715, Registration Date: Feb 1st, 2016 (01/02/2016).
摘要：
尽管福山先天性肌营养不良（FCMD）只有对症治疗，一些报告表明，类固醇治疗可能对FCMD有效；然而，尚未进行独立干预研究.这项研究旨在评估类固醇治疗恢复FCMD患者运动功能的疗效。这项研究涉及3至10岁的FCMD患者，他们表现出运动功能下降，要求类固醇治疗。同意的患者开始每隔一天口服0.5-mg/kg泼尼松龙，如果反应不充分，则增加到1.0mg/kg。我们使用粗大运动功能测量（GMFM）来评估和比较所有患者的运动功能。Wilcoxon符号秩检验(显著性水平，P≤0.05)进行统计学分析。在类固醇治疗开始时，8.10年（SD，2.14岁）是FCMD患者的平均年龄。类固醇治疗前后的平均GMFM差异为+1.23(SD，1.10),0.015的P值代表GMFM的显著改善。我们的结果表明，类固醇治疗可能有助于维持和改善晚期FCMD患者的运动功能。临床试验注册登记号：UMIN000020715，注册日期：2月1日，2016年（2016年01月02日）。
