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

J Child Neurol. 2016 Jun;31(7):899-906. doi: 10.1177/0883073815627882. Epub 2016 Jan 27.


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.

Trial registration: NCT01494701 NCT01780246.

Keywords: antisense oligonucleotide; drug delivery; lumbar puncture; spinal muscular atrophy.

Publication types

  • Clinical Trial, Phase I

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Injections, Spinal / adverse effects
  • Male
  • Muscular Atrophy, Spinal / drug therapy*
  • Neuromuscular Agents / administration & dosage*
  • Neuromuscular Agents / adverse effects
  • Oligonucleotides / administration & dosage*
  • Oligonucleotides / adverse effects
  • Spinal Puncture / adverse effects


  • Neuromuscular Agents
  • Oligonucleotides
  • nusinersen

Associated data