Abstract：
BACKGROUND: Glucocorticoid steroids are standard of care in Duchenne Muscular Dystrophy (DMD) to slow disease course. Use of glucocorticoids in other muscular dystrophies, including Becker (BMD) and Limb Girdle (LGMD), has been less explored. Recently, preclinical studies conducted in DMD and LGMD mouse models showed once-weekly prednisone was associated with improved muscle performance without activation of muscle atrophy genes.
OBJECTIVE: To determine safety and tolerability of once-weekly prednisone in patients with LGMD and BMD.
METHODS: We conducted an open label, exploratory single center study of of once-weekly prednisone at 0.75-1 mg/Kg in LGMD (n = 19) and BMD (n = 1) (mean age 35, range 18-60). The LGMD participants represented multiple different LGMD subtypes, and the study included ambulatory and non-ambulatory participants. Participants were assessed at baseline and 24 weeks for vital signs, blood biomarkers, and for patient-reported side effects. As secondary endpoints, functional muscle testing and body composition were measured.
RESULTS: Over the 24-week study, there were no significant changes in blood pressure, HgbA1C, or lipid profiles. We observed a reduction in serum creatine kinase over the study interval. Whole body DEXA scanning suggested a possible increase in lean mass and a reduction in adiposity. Functional measures suggested trends in improved muscle performance.
CONCLUSIONS: In this single center, open label pilot study, once-weekly prednisone was safe and well tolerated. Additional investigation of once-weekly prednisone in a larger cohort and for a longer period of time is warranted.
OBJECTIVE: To determine safety and tolerability of once-weekly prednisone in patients with LGMD and BMD.
METHODS: We conducted an open label, exploratory single center study of of once-weekly prednisone at 0.75-1 mg/Kg in LGMD (n = 19) and BMD (n = 1) (mean age 35, range 18-60). The LGMD participants represented multiple different LGMD subtypes, and the study included ambulatory and non-ambulatory participants. Participants were assessed at baseline and 24 weeks for vital signs, blood biomarkers, and for patient-reported side effects. As secondary endpoints, functional muscle testing and body composition were measured.
RESULTS: Over the 24-week study, there were no significant changes in blood pressure, HgbA1C, or lipid profiles. We observed a reduction in serum creatine kinase over the study interval. Whole body DEXA scanning suggested a possible increase in lean mass and a reduction in adiposity. Functional measures suggested trends in improved muscle performance.
CONCLUSIONS: In this single center, open label pilot study, once-weekly prednisone was safe and well tolerated. Additional investigation of once-weekly prednisone in a larger cohort and for a longer period of time is warranted.
摘要：
背景：糖皮质激素类固醇是Duchenne肌营养不良症（DMD）治疗的标准治疗方法，以减缓病程。在其他肌营养不良中使用糖皮质激素，包括贝克尔（BMD）和四肢腰带（LGMD），很少探索。最近,在DMD和LGMD小鼠模型中进行的临床前研究显示,每周一次泼尼松与改善的肌肉性能相关,而不激活肌肉萎缩基因.
目的：确定LGMD和BMD患者每周一次泼尼松的安全性和耐受性。
方法：我们进行了开放标签，在LGMD（n=19）和BMD（n=1）（平均年龄35，范围18-60）中每周一次的0.75-1mg/Kg泼尼松的探索性单中心研究。LGMD参与者代表了多个不同的LGMD亚型，研究包括非门诊和非门诊参与者.在基线和24周评估参与者的生命体征，血液生物标志物，以及患者报告的副作用。作为次要终点，测量功能性肌肉测试和身体成分。
结果：在为期24周的研究中，血压没有明显变化,HgbA1C,或脂质分布。我们观察到在研究间隔内血清肌酸激酶的减少。全身DEXA扫描表明瘦体重可能增加，肥胖减少。功能测量表明肌肉性能改善的趋势。
结论：在这个单一中心，开放标签试点研究，泼尼松每周一次安全且耐受性良好.在更大的队列和更长的时间内，每周一次泼尼松的额外调查是必要的。
目的：确定LGMD和BMD患者每周一次泼尼松的安全性和耐受性。
方法：我们进行了开放标签，在LGMD（n=19）和BMD（n=1）（平均年龄35，范围18-60）中每周一次的0.75-1mg/Kg泼尼松的探索性单中心研究。LGMD参与者代表了多个不同的LGMD亚型，研究包括非门诊和非门诊参与者.在基线和24周评估参与者的生命体征，血液生物标志物，以及患者报告的副作用。作为次要终点，测量功能性肌肉测试和身体成分。
结果：在为期24周的研究中，血压没有明显变化,HgbA1C,或脂质分布。我们观察到在研究间隔内血清肌酸激酶的减少。全身DEXA扫描表明瘦体重可能增加，肥胖减少。功能测量表明肌肉性能改善的趋势。
结论：在这个单一中心，开放标签试点研究，泼尼松每周一次安全且耐受性良好.在更大的队列和更长的时间内，每周一次泼尼松的额外调查是必要的。
