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Cervical puncture to ship nusinersen in sufferers with spinal muscular atrophy.

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Cervical puncture to ship nusinersen in sufferers with spinal muscular atrophy.

Neurology. 2018 Jul 13;:

Authors: Veerapandiyan A, Pal R, D’Ambrosio S, Younger I, Eichinger Okay, Collins E, Westesson PL, Kwon J, Ciafaloni E

OBJECTIVE: To report our expertise delivering intrathecal nusinersen via cervical puncture in sufferers with spinal muscular atrophy (SMA) with no lumbar entry.
BACKGROUND: SMA is a neuromuscular dysfunction characterised by profound muscle weak spot, atrophy, and paralysis because of degeneration of the anterior horn cells. Nusinersen, the primary Meals and Drug Administration-approved remedy for SMA, is run intrathecally by way of lumbar puncture; nevertheless, many sufferers with SMA have scoliosis or strong spinal fusion with that makes lumbar entry unattainable. Research in primates have demonstrated higher spinal wire tissue focus with intrathecal injections than with intracerebral ventricular injections. Due to this fact we have now used C1/C2 puncture as an alternative choice to administer nusinersen.
METHOD: Retrospective chart evaluation.
RESULTS: Intrathecal nusinersen by way of cervical puncture was given to three sufferers who had thoracic and lumbosacral spinal fusion: a 12-year-old lady with kind 1 SMA and a couple of 17-year-old ladies with kind 2 SMA. Cervical puncture was carried out with out deep sedation beneath fluoroscopic steerage utilizing a 25-G or a 24-G Whitacre needle within the posterior side of C1-C2 interspace and full dose of nusinersen (12 mg/5 mL) was injected after visualizing free CSF circulation. Sufferers accomplished their four loading doses and first upkeep dose of nusinersen, and 15 procedures have been profitable and well-tolerated.
CONCLUSION: Cervical puncture is a possible various supply path to administer intrathecal nusinersen in sufferers with longstanding SMA and backbone anatomy precluding lumbar entry when achieved by suppliers with experience on this process.

PMID: 30006410 [PubMed – as supplied by publisher]

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