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Single-center experience with intrathecal administration of Nusinersen in children with spinal muscular atrophy type 1.

BACKGROUND: Spinal muscular atrophy (SMA) is a neuromuscular disorder mainly characterized by proximal muscle weakness. There have been enormous advances in therapeutic development with the possibility to influence the clinical course of the disease. Nusinersen is the first approved drug to treat SMA. It is administered intrathecally and acts as splicing modifier of the SMN2 gene.

METHODS: Lumbar punctures were performed using a standardized protocol. To evaluate safety and feasibility of the intrathecal treatment, vital signs and the need for sedation, analgesia or mechanical ventilation during the procedure were monitored. Furthermore, the number of puncture attempts, the injection site and the macroscopic appearance of cerebrospinal fluid were documented.

RESULTS: Treatment with Nusinersen was initiated in 20 children aged from 2 to 50 months. Administration of a local anesthetic cream on the puncture site and a peripheral analgesic led to an adequate pain management. We observed a beneficial distraction through the possibility to watch a movie or listen to music during the procedure. In some cases, an additional sedation was necessary. In patients accustomed to non-invasive ventilation, this was used during lumbar punctures. On average, 1.5 ± 1.0 puncture attempts were performed between L 4/5 and L 2/3. If required, the position of the medullary cone was identified by ultrasound to guarantee a safe puncture above L 3/4.

CONCLUSIONS: Lumbar punctures for intrathecal administration of Nusinersen could be performed without any relevant complications. With the described approach lumbar punctures were tolerated well in all investigated age groups.

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