Intrathecal administration of nusinersen in adolescent and adult SMA type 2 and 3 patients.
Spinal muscular atrophy is a genetic motor neuron disease that leads to progressive muscular atrophy and muscle weakness. In December 2016, the Food and Drug Administration, and in June 2017, the European Medicines Agency approved the antisense oligonucleotide nusinersen for treatment of spinal muscular atrophy. Nusinersen has to be repeatedly administered intrathecally. Due to the clinical features of SMA, the application of the ASO by lumbar puncture can be challenging in symptomatic patients considering the frequently observed scoliosis, previous spine fusion surgeries, joint contractures, and respiratory insufficiency. To evaluate safety and feasibility of the intrathecal treatment in adolescent and adult SMA type 2 and 3 patients, we analyzed 93 lumbar punctures, monitored number of lumbar puncture attempts, duration of the procedure, injection site, and needle length. Oxygen saturation during the intervention, medication for sedation and local anesthesia, adverse events related to lumbar punctures, and macroscopic analysis of CSF were recorded. Moreover, we analyzed the use of CT-scans for performing lumbar punctures and its associated radiation exposure. Performing lumbar puncture for the intrathecal administration of nusinersen in adolescent and adult patients with later-onset SMA is feasible and safe, even in patients with complex spinal anatomies and respiratory insufficiency. To guarantee the quality of the procedure, we recommend establishing an experienced interdisciplinary team consisting of neurologists and/or neuropediatricians, anesthesiologists, orthopedic surgeons, and/or neuroradiologists.
['Wurster|Claudia D|CD|', 'Winter|Benedikt|B|', 'Wollinsky|Kurt|K|', 'Ludolph|Albert C|AC|', 'Uzelac|Zeljko|Z|', 'Witzel|Simon|S|', 'Schocke|Michael|M|', 'Schneider|Ralf|R|', 'Kocak|Tugrul|T|']
D000293:Adolescent D000328:Adult D001688:Biological Products D002648:Child D005260:Female D006801:Humans D007278:Injections, Spinal D008297:Male D008875:Middle Aged D009841:Oligonucleotides D014897:Spinal Muscular Atrophies of Childhood D013129:Spinal Puncture D014057:Tomography, X-Ray Computed D016896:Treatment Outcome D055815:Young Adult
Nusinersen Spinal muscular atrophy Lumbar puncture