超声辅助及基于体表标志的诺西那生钠给药在成年脊髓性肌萎缩症患者中的应用：一项回顾性分析。
Ultrasound-assisted and landmark-based nusinersen delivery in spinal muscular atrophy adults: A retrospective analysis.
作者信息
Zanfini Bruno Antonio, Patanella Agata Katia, Vassalli Francesco, Catarci Stefano, Pane Marika, Frassanito Luciano, Biancone Matteo, Di Muro Mariangela, Bravetti Chiara, Mercuri Eugenio Maria, Sabatelli Mario, Draisci Gaetano
机构信息
Department of Emergency, Anesthesiological and Reanimation Sciences Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy.
Centro Clinico NeMO Adulti Fondazione Policlinico Universitario Agostino Gemelli IRCCS Rome Italy.
出版信息
Australas J Ultrasound Med. 2024 Jul 15;27(4):210-217. doi: 10.1002/ajum.12401. eCollection 2024 Nov.
INTRODUCTION/PURPOSE: Nusinersen, the first treatment approved for all spinal muscular atrophy (SMA) types, is administered intrathecally through lumbar puncture. We used ultrasound assistance or a landmark-based technique to access the lumbar intrathecal space in adult SMA patients. This study aimed to evaluate the technical success and adverse events (AEs) in such patients using either technique over a long observation period.
METHODS
Fifty-one adult patients received 507 consecutive interlaminar nusinersen administrations. Patients presented with both 'uncomplicated spines' or 'complicated spines'; two patients had previous back surgery. Technical success and AEs were recorded using either technique. A generalised linear mixed model was applied to evaluate predictors of technical success and complications.
RESULTS
An overall success rate of 99.6%, with only two procedures failing to reach the intrathecal space, and an overall optimal procedure rate of 90.3% have been reported. A total of 455 procedures (89.7%) were uneventfully performed. One (0.2%) case of severe AE (puncture of a bulky abdominal annexal cyst) was recorded. Twenty-seven episodes (5.3%) of post-dural puncture headache (PDPH) and 24 episodes (4.7%) of radicular or back pain, both successfully treated with medical therapy, have also been reported. Technical success was significantly associated with 'complicated spines' (P = 0.022) and the use of ultrasound assistance (P = 0.01), and the use of ultrasound was the only independent predictor of uncomplicated procedures (P = 0.007).
DISCUSSION
In adult patients with SMA both landmark-based and ultrasound-assisted techniques are safe and effective even in the long term. The use of assistance is associated with technical success and can predict uncomplicated procedures.
CONCLUSION
Our results support the use of ultrasonography in order to improve the success and reduce the burden of nusinersen intrathecal administration.
引言/目的：诺西那生钠是首个被批准用于治疗所有类型脊髓性肌萎缩症（SMA）的药物，通过腰椎穿刺鞘内给药。我们在成年SMA患者中采用超声辅助或基于体表标志的技术进入腰段鞘内间隙。本研究旨在通过长期观察评估采用这两种技术的此类患者的技术成功率和不良事件（AE）。
方法
51例成年患者连续接受了507次椎板间诺西那生钠给药。患者既有“脊柱情况不复杂”的，也有“脊柱情况复杂”的；2例患者曾接受过背部手术。采用两种技术记录技术成功率和AE。应用广义线性混合模型评估技术成功和并发症的预测因素。
结果
报告的总体成功率为99.6%，仅2例操作未能进入鞘内间隙，总体最佳操作率为90.3%。总共455例操作（89.7%）顺利完成。记录到1例（0.2%）严重AE（穿刺到一个巨大的腹部附件囊肿）。还报告了27例（5.3%）硬膜穿刺后头痛（PDPH）和24例（4.7%）神经根性或背痛，均通过药物治疗成功治愈。技术成功与“脊柱情况复杂”（P = 0.022）和使用超声辅助（P = 0.01）显著相关，并且使用超声是操作无并发症的唯一独立预测因素（P = 0.007）。
讨论
在成年SMA患者中，即使从长期来看，基于体表标志的技术和超声辅助技术都是安全有效的。使用辅助技术与技术成功相关，并且可以预测操作无并发症。
结论
我们的结果支持使用超声检查以提高诺西那生钠鞘内给药的成功率并减轻其负担。
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