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患有尼曼-匹克C型病的儿科患者鞘内给药相关并发症。

Complications associated with intrathecal drug delivery in a paediatric patient with Niemann-Pick type C.

作者信息

Rodriguez Ciancio Jose Ignacio Rodriguez, Aquilina Kristian

机构信息

Metabolic Medicine, Great Ormond Street Hospital for Children, London, UK

Neurosurgery, Great Ormond Street Hospital for Children, London, UK.

出版信息

BMJ Case Rep. 2021 May 7;14(5):e241786. doi: 10.1136/bcr-2021-241786.

Abstract

We report on a male subject with a diagnosis of Niemann-Pick type C (NPC). He received an experimental medicinal product intrathecally initially via lumbar puncture (LP) and eventually via intrathecal drug delivery device. Shortly after implantation, the device catheter migrated outside of the intrathecal space and coiled subcutaneously. The treatment continued via LP after removal of the device. A subdural haematoma developed after repeated LPs. It was surgically evacuated and the patient recovered with sequelae. Surgically implanted drug delivery devices are designed to bypass the blood-brain barrier and deliver a medicinal product directly into the cerebrospinal fluid circulation. Their use has extended into the field of neurodegenerative disorders. Significant adverse events can occur at any given time after implantation including neurological injury, dislodgement or displacement of any of its components, infection and drug-related complications; all can significantly affect the quality of life of patients. Repeated LPs also carry significant risk.

摘要

我们报告了一名被诊断为尼曼-匹克C型(NPC)的男性患者。他最初通过腰椎穿刺(LP)鞘内注射一种实验性药物产品,最终通过鞘内给药装置进行注射。植入后不久,该装置的导管迁移到鞘外空间并在皮下盘绕。移除该装置后,通过腰椎穿刺继续进行治疗。反复腰椎穿刺后发生了硬膜下血肿。通过手术将其清除,患者康复但留有后遗症。外科植入的给药装置旨在绕过血脑屏障,将药物产品直接输送到脑脊液循环中。它们的应用已扩展到神经退行性疾病领域。植入后任何时候都可能发生重大不良事件,包括神经损伤、其任何组件的移位或错位、感染以及与药物相关的并发症;所有这些都可能严重影响患者的生活质量。反复腰椎穿刺也有很大风险。

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