Fluoroscopic guidance for intrathecal delivery of nusinersen in pediatric patients with spinal muscular atrophy and complex spines

IF 2.7 4区医学 Q2 CLINICAL NEUROLOGY

Neuromuscular Disorders Pub Date : 2025-03-01 DOI:10.1016/j.nmd.2025.105336

Shany Lando - Dotan , Eliyahu Perlow , Vered Shkalim Zemer , Hagit Levine , Elchanan Bruckheimer , Yelena Tzeitlin , Tamar Steinberg , Yoram Nevo , Tzipora Shochat , Sharon Aharoni

{"title":"Fluoroscopic guidance for intrathecal delivery of nusinersen in pediatric patients with spinal muscular atrophy and complex spines","authors":"Shany Lando - Dotan , Eliyahu Perlow , Vered Shkalim Zemer , Hagit Levine , Elchanan Bruckheimer , Yelena Tzeitlin , Tamar Steinberg , Yoram Nevo , Tzipora Shochat , Sharon Aharoni","doi":"10.1016/j.nmd.2025.105336","DOIUrl":null,"url":null,"abstract":"<div><div>The introduction of nusinersen revolutionized the treatment of spinal muscular atrophy (SMA). However, nusinersen is administered by interlaminar intrathecal injection which is challenging in patients with severe scoliosis, a common comorbidity of advanced SMA. This study evaluated the technical benefits of fluoroscopic guidance of intrathecal nusinersen administration in complex SMA patients with or without a fixation device. The cohort included 12 patients aged 10–20 years (total 124 injections). The total success rate was 99 %, with failure to complete only one out of 124 procedures. Demographic characteristics were diverse. Mean age at first injection was 14.2 years. Mean duration of radiation exposure was 77 s; mean dose area product was 2.32 Gycm<sup>2</sup>; and mean cumulative air kerma was 20.91mGy. Adverse events included post-dural-puncture headache (4.8 % of procedures), mostly mild and self-limited, and one allergic reaction. Treatment was discontinued in 2 patients because of difficult intrathecal access, and in 2 for reasons unrelated to the injection technique. Fluoroscopy-guided nusinersen administration is a feasible option for patients with SMA and complex access. Success depends on proper patient positioning and expertise of the interventional radiologist. Radiation exposure is lower than with other techniques. Larger prospective studies are needed to confirm these findings.</div></div>","PeriodicalId":19135,"journal":{"name":"Neuromuscular Disorders","volume":"49 ","pages":"Article 105336"},"PeriodicalIF":2.7000,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Neuromuscular Disorders","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S096089662500063X","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}

引用次数: 0

Abstract

The introduction of nusinersen revolutionized the treatment of spinal muscular atrophy (SMA). However, nusinersen is administered by interlaminar intrathecal injection which is challenging in patients with severe scoliosis, a common comorbidity of advanced SMA. This study evaluated the technical benefits of fluoroscopic guidance of intrathecal nusinersen administration in complex SMA patients with or without a fixation device. The cohort included 12 patients aged 10–20 years (total 124 injections). The total success rate was 99 %, with failure to complete only one out of 124 procedures. Demographic characteristics were diverse. Mean age at first injection was 14.2 years. Mean duration of radiation exposure was 77 s; mean dose area product was 2.32 Gycm²; and mean cumulative air kerma was 20.91mGy. Adverse events included post-dural-puncture headache (4.8 % of procedures), mostly mild and self-limited, and one allergic reaction. Treatment was discontinued in 2 patients because of difficult intrathecal access, and in 2 for reasons unrelated to the injection technique. Fluoroscopy-guided nusinersen administration is a feasible option for patients with SMA and complex access. Success depends on proper patient positioning and expertise of the interventional radiologist. Radiation exposure is lower than with other techniques. Larger prospective studies are needed to confirm these findings.

查看原文本刊更多论文

脊髓性肌萎缩症和复杂脊柱患儿鞘内施药的透视指导

nusinersen的引入彻底改变了脊髓性肌萎缩症（SMA）的治疗。然而，nusinersen是通过椎板间鞘内注射给药，这对于严重脊柱侧凸患者是具有挑战性的，脊柱侧凸是晚期SMA的常见合病。本研究评估了带或不带固定装置的复杂SMA患者鞘内给药的透视指导技术效益。该队列包括12例10-20岁的患者（共124次注射）。总成功率为99%，124次手术中只有1次失败。人口统计学特征多种多样。首次注射的平均年龄为14.2岁。辐照时间平均为77 s；平均剂量面积积为2.32 Gycm2；平均累积气温为20.91mGy。不良事件包括硬脊膜穿刺后头痛（4.8%的手术），大多数是轻度和自限性的，以及一次过敏反应。2例患者因难以进入鞘内而停止治疗，2例患者因与注射技术无关的原因而停止治疗。透视引导下的nusinersen给药是SMA患者和复杂通道的可行选择。成功与否取决于病人的正确体位和介入放射科医生的专业知识。辐射暴露比其他技术要低。需要更大规模的前瞻性研究来证实这些发现。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Neuromuscular Disorders 医学-临床神经学

CiteScore

4.60

自引率

3.60%

发文量

543

审稿时长

53 days

期刊介绍： This international, multidisciplinary journal covers all aspects of neuromuscular disorders in childhood and adult life (including the muscular dystrophies, spinal muscular atrophies, hereditary neuropathies, congenital myopathies, myasthenias, myotonic syndromes, metabolic myopathies and inflammatory myopathies). The Editors welcome original articles from all areas of the field: • Clinical aspects, such as new clinical entities, case studies of interest, treatment, management and rehabilitation (including biomechanics, orthotic design and surgery). • Basic scientific studies of relevance to the clinical syndromes, including advances in the fields of molecular biology and genetics. • Studies of animal models relevant to the human diseases. The journal is aimed at a wide range of clinicians, pathologists, associated paramedical professionals and clinical and basic scientists with an interest in the study of neuromuscular disorders.