Neuromuscular Center, Department of Neurosciences, University of Padova, Italy.
Neurology. 2013 Jun 4;80(23):2095-8. doi: 10.1212/WNL.0b013e318295d766. Epub 2013 May 3.
To test the efficacy and tolerability of clenbuterol in patients with spinal and bulbar muscular atrophy (SBMA).
Twenty patients with a diagnosis of SBMA were given oral clenbuterol (0.04 mg/d) for 12 months. The primary efficacy end point was the change from baseline of the walking distance covered in 6 minutes at 12 months. Secondary end points included the change over time in muscle strength assessed with the Medical Research Council scale, the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), and forced vital capacity values. Safety was assessed by a series of laboratory and instrumental tests, as well as reporting of adverse events.
Sixteen patients completed the study. There was a significant and sustained increase in walking distance covered in 6 minutes and forced vital capacity between the baseline and the 12-month assessments (p < 0.001). No differences were recorded in Medical Research Council or ALSFRS-R scores between baseline and follow-up assessments. Serious side effects, including those on heart function, were absent. A significant increase in serum creatine kinase levels was observed.
Our findings suggest a positive effect of clenbuterol on SBMA disease progression.
This study provides Class IV evidence that clenbuterol is effective in improving motor function in SBMA.
检验克仑特罗治疗脊髓性肌萎缩症（SBMA）的疗效和耐受性。
20 名 SBMA 患者接受为期 12 个月的口服克仑特罗（0.04mg/d）治疗。主要疗效终点为 12 个月时 6 分钟步行距离较基线的变化。次要终点包括肌力量随时间的变化，用改良的 Medical Research Council 量表、修订后的肌萎缩侧索硬化功能评定量表（ALSFRS-R）和用力肺活量值评估。安全性通过一系列实验室和仪器检查以及不良事件报告来评估。
16 名患者完成了研究。6 分钟步行距离和用力肺活量在基线和 12 个月评估时均有显著和持续的增加（p＜0.001）。基线和随访评估时，Medical Research Council 或 ALSFRS-R 评分无差异。未记录到严重的副作用，包括心脏功能方面的副作用。血清肌酸激酶水平显著升高。
我们的研究结果表明，克仑特罗对 SBMA 疾病进展有积极作用。
本研究提供了 IV 级证据，表明克仑特罗可有效改善 SBMA 的运动功能。
