Neuromuscular Disorders ( IF 2.7 ) Pub Date : 2021-08-24 , DOI: 10.1016/j.nmd.2021.08.011 Niveditha Putananickal 1 , Anna-Lena Orsini 2 , Simone Schmidt 1 , Vanya Gocheva 1 , Daniela Rubino 1 , Tanja Haas 3 , Sabine Schädelin 4 , Xeni Deligianni 5 , Oliver Bieri 5 , Dirk Fischer 1 , Patricia Hafner 1
This single-centered, randomized, double-blind, placebo-controlled study reports the results of L-Citrulline treatment for 24 weeks in patients with post-polio syndrome (PPS). Twenty-nine patients were randomized and assigned into receiving a treatment of 15 g L-Citrulline or placebo. The primary endpoint was the change of the 6 min walking distance test. Secondary endpoints included motor function measure, quantitative muscle strength, quantitative MRI and self-reported impairment questionnaires. Patients receiving L-Citrulline walked 17.5 longer in the 6 min walking distance test when compared to placebo group, however not statistically significant (95% CI = -14.69; 49.68, p = 0.298). None of the secondary endpoints showed a statistically significant change in the L-Citrulline group when compared to placebo group. The motor function measure showed a change of -0.78 (95% CI= [-3.39; 1.83] p = 0.563). Muscle degeneration of leg muscles assessed with quantitative MRI indicated no significant change (estimate= -0.01, 95% CI =-0.13; 0.11, p = 0.869). L-Citrulline was safe and well tolerated. In conclusion, administration of 15 g L-Citrulline daily for 24 weeks to patients with PPS showed no beneficial treatment effect in timed muscle function.
中文翻译:
L-瓜氨酸治疗脊髓灰质炎后综合征患者:一项单中心、随机、双盲、安慰剂对照试验
这项单中心、随机、双盲、安慰剂对照研究报告了L-瓜氨酸治疗脊髓灰质炎后综合征 (PPS) 患者 24 周的结果。29 名患者被随机分配接受 15 g L-瓜氨酸或安慰剂治疗。主要终点是6分钟步行距离测试的变化。次要终点包括运动功能测量、定量肌肉力量、定量 MRI 和自我报告的损伤问卷。与安慰剂组相比,接受L-瓜氨酸的患者在 6 分钟步行距离测试中的步行时间长了 17.5,但没有统计学意义(95% CI = -14.69;49.68,p = 0.298)。与安慰剂组相比,L-瓜氨酸组的次要终点均未显示出统计学上的显着变化。运动功能测量显示变化为 -0.78 (95% CI= [-3.39; 1.83] p = 0.563)。用定量 MRI 评估的腿部肌肉的肌肉退化表明没有显着变化(估计 = -0.01,95% CI =-0.13;0.11,p = 0.869)。L-瓜氨酸安全且耐受性良好。总之,PPS 患者每天服用 15 g L-瓜氨酸,持续 24 周,在定时肌肉功能方面没有显示出有益的治疗效果。