Nusinersen for older patients with spinal muscular atrophy: A real-world clinical setting experience
Nusinersen for older patients with spinal muscular atrophy: A real-world clinical setting experience
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DOI：
10.1002/mus.26769
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发表时间：
2019-12-13
期刊：
影响因子：
3.4
通讯作者：
Ciafaloni, Emma
中科院分区：
文献类型：
--
作者：
Veerapandiyan, Aravindhan;Eichinger, Katy;Ciafaloni, Emma
引言
关于在年龄较大的脊髓性肌萎缩症（SMA）患者中使用诺西那生的临床试验数据是缺乏的。我们描述了我们中心在临床环境中对年龄较大患者使用鞘内注射诺西那生的经验。
方法
回顾性研究。
结果
12名患者（12 - 52岁）接受了诺西那生治疗。平均随访时间为17.4个月（范围，4 - 26个月）。所有患者都有脊柱侧凸；10名患者进行了脊柱融合/内固定。所有操作（30次颈椎穿刺和57次腰椎穿刺）在技术上都是成功的。唯一的副作用是术后头痛（9%）和穿刺部位疼痛（5.7%）。功能评估显示，9名患者中有6名病情稳定，3名患者病情改善。12名患者中有8名报告在耐力、手部力量以及对日常生活活动至关重要的延髓功能方面有主观改善。到目前为止，没有患者停止治疗。
讨论
鞘内注射诺西那生可以安全地用于年龄较大的SMA患者。现有的功能结果测量方法不足以捕捉有意义的主观改善。
Introduction Clinical trials data concerning use of nusinersen in older spinal muscular atrophy (SMA) patients is lacking. We describe our center's experience in using intrathecal nusinersen for older patients in the clinical setting. Methods Retrospective study. Results Twelve patients (12-52 years old) were treated with nusinersen. Mean follow-up duration was 17.4 months (range, 4-26 months). All patients had scoliosis; 10 had spinal fusion/instrumentation. All procedures (30 cervical and 57 lumbar punctures) were technically successful. The only side effects were postprocedural headache (9%) and site pain (5.7%). Functional assessments showed stability in 6/9 patients and improvement in 3/9 patients. Subjective improvements in endurance, hand strength, and bulbar functioning critical for activities of daily living were reported in 8/12 patients. None of the patients has discontinued treatment so far. Discussion Intrathecal nusinersen can be safely delivered in older SMA patients. Available functional outcome measures are not adequate to capture meaningful subjective improvements.