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.
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。