Ultrasound‐assisted and landmark‐based nusinersen delivery in spinal muscular atrophy adults: A retrospective analysis

Q3 Medicine
B. Zanfini, Agata Katia Patanella, Francesco Vassalli, S. Catarci, Marika Pane, L. Frassanito, M. Biancone, Mariangela Di Muro, Chiara Bravetti, Eugenio Maria Mercuri, Mario Sabatelli, G. Draisci
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引用次数: 0

Abstract

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.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.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).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.Our results support the use of ultrasonography in order to improve the success and reduce the burden of nusinersen intrathecal administration.
成人脊髓性肌肉萎缩症患者的超声辅助和地标式努西那生给药:回顾性分析
Nusinersen是首款获准用于所有脊髓性肌萎缩症(SMA)类型的治疗药物,通过腰椎穿刺进行鞘内给药。我们使用超声辅助或基于地标的技术对成年 SMA 患者进行腰椎腔内穿刺。这项研究旨在评估在长期观察期内,使用这两种技术对此类患者的技术成功率和不良事件(AEs)。51名成年患者连续接受了507次层间纽西奈森治疗,患者既有 "不复杂的脊柱",也有 "复杂的脊柱";其中两名患者曾接受过背部手术。两种技术均记录了技术成功率和不良反应。应用广义线性混合模型评估了技术成功率和并发症的预测因素。据报道,总体成功率为 99.6%,仅有两次手术未能到达鞘内间隙,总体最佳手术率为 90.3%。共有 455 例手术(89.7%)顺利完成。记录到一例(0.2%)严重AE(穿刺到腹部巨大附件囊肿)。此外,还报告了 27 例(5.3%)硬膜穿刺后头痛(PDPH)和 24 例(4.7%)根痛或背痛,均通过药物治疗成功治愈。技术成功与 "复杂脊柱"(P = 0.022)和使用超声辅助(P = 0.01)明显相关,而使用超声是不复杂手术的唯一独立预测因素(P = 0.007)。我们的研究结果支持使用超声波检查来提高努西那生鞘内给药的成功率并减轻其负担。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Australasian Journal of Ultrasound in Medicine
Australasian Journal of Ultrasound in Medicine Medicine-Radiology, Nuclear Medicine and Imaging
CiteScore
1.90
自引率
0.00%
发文量
40
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