Intrathecal Injections in Children with Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience

Manon Haché, Kathryn J. Swoboda, Navil Sethna, Alan Farrow-Gillespie, Alexander Khandji, Shuting Xia, Kathie M. Bishop

Research output: Contribution to journalArticlepeer-review

158 Scopus citations


Nusinersen (ISIS-SMNRx or ISIS 396443) is an antisense oligonucleotide drug administered intrathecally to treat spinal muscular atrophy. We summarize lumbar puncture experience in children with spinal muscular atrophy during a phase 1 open-label study of nusinersen and its extension. During the studies, 73 lumbar punctures were performed in 28 patients 2 to 14 years of age with type 2/3 spinal muscular atrophy. No complications occurred in 50 (68%) lumbar punctures; in 23 (32%) procedures, adverse events were attributed to lumbar puncture. Most common adverse events were headache (n = 9), back pain (n = 9), and post-lumbar puncture syndrome (n = 8). In a subgroup analysis, adverse events were more frequent in older children, children with type 3 spinal muscular atrophy, and with a 21- or 22-gauge needle compared to a 24-gauge needle or smaller. Lumbar punctures were successfully performed in children with spinal muscular atrophy; lumbar puncture-related adverse event frequency was similar to that previously reported in children.

Original languageEnglish (US)
Pages (from-to)899-906
Number of pages8
JournalJournal of child neurology
Issue number7
StatePublished - Jun 2016


  • antisense oligonucleotide
  • drug delivery
  • lumbar puncture
  • spinal muscular atrophy

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Clinical Neurology


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