Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience.
Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience.
复制标题
脊柱肌肉萎缩儿童的鞘内注射：Nusinersen临床试验经验。
DOI：
10.1177/0883073815627882
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发表时间：
2016-06
影响因子：
1.9
通讯作者：
Bishop KM
中科院分区：
文献类型：
--
作者：
Haché M;Swoboda KJ;Sethna N;Farrow-Gillespie A;Khandji A;Xia S;Bishop KM
Nusinersen（ISIS-SMNRx 或 ISIS 396443）是一种鞘内注射的反义寡核苷酸药物，用于治疗脊髓性肌萎缩症。我们总结了 nusinersen 及其扩展的 1 期开放标签研究中脊髓性肌萎缩症儿童的腰椎穿刺经验。研究期间，对 28 名 2 至 14 岁 2/3 型脊髓性肌萎缩症患者进行了 73 次腰椎穿刺。 50 次（68%）腰椎穿刺未出现并发症；在 23 例 (32%) 手术中，不良事件归因于腰椎穿刺。最常见的不良事件是头痛（n = 9）、背痛（n = 9）和腰椎穿刺后综合征（n = 8）。在亚组分析中，与 24 号或更小的针头相比，年龄较大的儿童、患有 3 型脊髓性肌萎缩症的儿童以及使用 21 号或 22 号针头的不良事件更常见。成功对脊髓性肌萎缩症患儿进行腰椎穿刺；腰椎穿刺相关不良事件发生频率与之前报告的儿童相似。
Nusinersen (ISIS-SMNRx or ISIS 396443) is an antisense oligonucleotide drug administered intrathecally to treat spinal muscular atrophy. We summarize lumbar puncture experience in children with spinal muscular atrophy during a phase 1 open-label study of nusinersen and its extension. During the studies, 73 lumbar punctures were performed in 28 patients 2 to 14 years of age with type 2/3 spinal muscular atrophy. No complications occurred in 50 (68%) lumbar punctures; in 23 (32%) procedures, adverse events were attributed to lumbar puncture. Most common adverse events were headache (n = 9), back pain (n = 9), and post–lumbar puncture syndrome (n = 8). In a subgroup analysis, adverse events were more frequent in older children, children with type 3 spinal muscular atrophy, and with a 21- or 22-gauge needle compared to a 24-gauge needle or smaller. Lumbar punctures were successfully performed in children with spinal muscular atrophy; lumbar puncture–related adverse event frequency was similar to that previously reported in children.