Abstract：
This systematic review and meta-analysis aimed to assess the efficacy and safety of risdiplam on motor and respiratory function in spinal muscular atrophy (SMA). We systematically searched Medline, Scopus, Web of Science, and the Cochrane Library from inception to March 2023. We included pre-post studies that determined the effect of risdiplam on the Children\'s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), the 32-item Motor Function Measure (MFM32), the Revised Upper Limb Module (RULM), the Hammersmith Functional Motor Scale - Expanded (HFMSE), respiratory function, and the proportion of risdiplam-related adverse events in a population with SMA (phenotypes 1 and 2/3). Meta-analyses were also performed where possible. Eleven studies were included. After 12 months of treatment, 57% of participants with SMA1 achieved a CHOP-INTEND score ≥ 40 points, and more than half were able to feed orally and had head control. In SMA2/3, MFM32, RULM, and HFMSE increased by 2.09 (1.17, 3.01), 1.73 (1.25, 2.20), and 1.00 (0.40, 1.59) points, respectively. Efficacy on respiratory function in SMA2/3 was inconsistent. Finally, 16% of participants experienced adverse events, but serious adverse events could not be quantified due to a lack of cases. The limited available evidence suggests that risdiplam is an effective and safe drug for the treatment of SMA. In addition, long-term clinical benefit may be partly determined by the stage of disease at which treatment is initiated.
摘要：
目的：本系统综述和荟萃分析旨在评估利司普坦对脊髓性肌萎缩症（SMA）运动和呼吸功能的疗效和安全性。
方法：我们系统地搜索了Medline，Scopus,WebofScience,和Cochrane图书馆从成立到2023年3月。我们纳入了确定利司普坦对费城儿童医院神经肌肉疾病婴儿测试（CHOP-INTEND）的影响的pre-post研究，32项电机功能测量(MFM32)，修订的上肢模块(RULM),哈默史密斯功能电机扩展秤(HFMSE)，呼吸功能,和利司普兰相关不良事件在SMA患者中的比例(表型1和2/3).在可能的情况下也进行了荟萃分析。
结果：共纳入11项研究。经过12个月的治疗，57％的SMA1参与者获得CHOP-INTEND得分≥40分，超过一半的人能够口服进食并控制头部。在SMA2/3、MFM32、RULM、HFMSE增加2.09（1.17，3.01），1.73(1.25,2.20),和1.00（0.40，1.59）点，分别。SMA2/3对呼吸功能的疗效不一致。最后,16％的参与者经历了不良事件，但由于缺乏病例,严重不良事件无法量化.
结论：有限的现有证据表明，利司普坦是治疗SMA的有效且安全的药物。此外,长期临床获益可能部分取决于开始治疗的疾病阶段.
方法：我们系统地搜索了Medline，Scopus,WebofScience,和Cochrane图书馆从成立到2023年3月。我们纳入了确定利司普坦对费城儿童医院神经肌肉疾病婴儿测试（CHOP-INTEND）的影响的pre-post研究，32项电机功能测量(MFM32)，修订的上肢模块(RULM),哈默史密斯功能电机扩展秤(HFMSE)，呼吸功能,和利司普兰相关不良事件在SMA患者中的比例(表型1和2/3).在可能的情况下也进行了荟萃分析。
结果：共纳入11项研究。经过12个月的治疗，57％的SMA1参与者获得CHOP-INTEND得分≥40分，超过一半的人能够口服进食并控制头部。在SMA2/3、MFM32、RULM、HFMSE增加2.09（1.17，3.01），1.73(1.25,2.20),和1.00（0.40，1.59）点，分别。SMA2/3对呼吸功能的疗效不一致。最后,16％的参与者经历了不良事件，但由于缺乏病例,严重不良事件无法量化.
结论：有限的现有证据表明，利司普坦是治疗SMA的有效且安全的药物。此外,长期临床获益可能部分取决于开始治疗的疾病阶段.
