Interlaminar Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal Muscular Atrophy: a Retrospective Analysis of More Than 500 Consecutive Administrations
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 51
- 试验地点
- 1
- 主要终点
- Number of successful administrations
研究概览
简要总结
Nusinersen (Spinraza, Biogen Inc, Boston, MA), the first treatment approved by FDA and EMA for all Spinal Muscular Atrophy (SMA) subtypes, is an antisense oligonucleotide that is administered intrathecally through a lumbar puncture. This procedure can be challenging in some adults with intermediate and late onset SMA (types II-IV) frequently presenting scoliosis secondary to neuromuscular weakness and often treated with spinal instrumentation to prevent worsening deformities.
In such patients, in order to access the intrathecal space, US guidance and/or assistance have been recently proposed as useful and successful tool. The US guidance and/or assistance have been associated to a high success rate, a reduction of number of attempts and needle passes to obtain a successful anesthesia. A reduced risk of adverse events (AEs), such as post dural puncture headache (PDPH) and low back pain (LBP), and low patient satisfaction often associated with multiple needle punctures was also reported.
Aim of this retrospective study was to report the efficacy, evaluated as rate of the successful procedures and subsequent delivery of nusinersen within the subarachnoid space, the number of attempts, the procedure time and the adverse events (AEs) of interlaminar intrathecal nusinersen administration using either ultrasound assistance or the landmark-based technique in a historical cohort of 51 adult SMA patients.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 18 Years 至 —(Adult, Older Adult)
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A genetically confirmed diagnosis of SMA
- •Treatment with nusinersen
排除标准
- •congenital coagulopathy,
- •localized infections,
- •increased intracranial pressure.
结局指标
主要结局
Number of successful administrations
时间窗: During the procedure
Defined as a confirmation of the CSF flow through the spinal needle and subsequent administration of whole drug
Number of attempts
时间窗: During the procedure
Defined as the number of needle insertions through the skin surface
Procedure time
时间窗: During the procedure
The time in minutes from the start of US imaging or the palpation of superior aspect of iliac crest to visualization of CSF flow
Number of technical success procedures
时间窗: During the procedure
Defined as a successful intrathecal administration with equal or less than 4 attempts
Adverse events (AEs)
时间窗: In the first 72 hours after the procedure
Defined as mild AEs (Post Dural Puncture Headache, Low Back Pain) or severe AEs (spinal cord injury/epidural hematoma, abdominal organ lesions)
次要结局
未报告次要终点
