Zhang Yani, Zheng Kelu, Liang Cuili, Zheng Ruidan, Chen Jinghui, Jiang Minyan, Zhou Zhizi, Zhao Yuan, Rao Min, Yang Sida, Chen Wenxiong, Liu Li
Department of Neurology, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, China.
Department of Endocrinology and Genetic Metabolism, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangzhou, China.
Front Neurol. 2023 Sep 21;14:1234442. doi: 10.3389/fneur.2023.1234442. eCollection 2023.
Nusinersen, an extremely expensive biologic drug (around 100,000 US$ per dose) that needs to be administered intrathecally, is approved for the treatment of 5q-spinal muscular atrophy (SMA). Because of the low muscle tone of the back muscles of pediatric SMA patients, especially type 1 SMA patients, the safe, effective, and fast execution of sheath injection is needed. Therefore, a modified intrathecal injection method was developed accordingly. This paper aims to describe the applicability and safety of this modified method.
The modified intrathecal injection method (MIIM) mainly includes a septal needle-free closed infusion connector between the lumbar puncture needle and the syringe, besides the procedures of routine lumbar puncture. Its applicability and safety were evaluated through clinical observation.
A total of 92 children with SMA have successfully received nusinersen treatment at our hospital using the modified method since 2019 without obvious adverse events related to the modified injection method. Based on the clinical feedback of operators, the advantages of the modified method include successfully injecting the total dose of nusinersen with constant injection rate and a more stable fixation of the puncture needle, as well as making the operator more relaxed. However, compared with the routine method, the procedure of the modified method has additional steps.
The modified intrathecal injection method is an effective and safe method to inject nusinersen when weighing the pros and cons, and it may also be used for administering intrathecal injections of other expensive medicines or for patients with other strict requirements for intrathecal injection.
诺西那生钠是一种极其昂贵的生物药物（每剂约10万美元），需要鞘内注射，已被批准用于治疗5q型脊髓性肌萎缩症（SMA）。由于小儿SMA患者，尤其是1型SMA患者背部肌肉肌张力低，需要安全、有效且快速地进行鞘内注射。因此，相应地开发了一种改良的鞘内注射方法。本文旨在描述这种改良方法的适用性和安全性。
改良鞘内注射方法（MIIM）除常规腰椎穿刺步骤外，主要包括在腰椎穿刺针与注射器之间使用隔片无针密闭输液接头。通过临床观察评估其适用性和安全性。
自2019年以来，共有92例SMA患儿在我院使用改良方法成功接受了诺西那生钠治疗，未出现与改良注射方法相关的明显不良事件。根据操作人员的临床反馈，改良方法的优点包括以恒定注射速率成功注射诺西那生钠的总剂量、穿刺针固定更稳定以及使操作人员更轻松。然而，与常规方法相比，改良方法的操作步骤更多。
权衡利弊后，改良鞘内注射方法是注射诺西那生钠的一种有效且安全的方法，它也可用于其他昂贵药物的鞘内注射或对鞘内注射有其他严格要求的患者。
