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 journalArticle

81 Citations (Scopus)

Abstract

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
Volume31
Issue number7
DOIs
StatePublished - 2015

Fingerprint

Spinal Muscular Atrophy
Spinal Injections
Spinal Puncture
Clinical Trials
Spinal Muscular Atrophies of Childhood
Needles
Antisense Oligonucleotides
Back Pain
Headache
Pharmaceutical Preparations

Keywords

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

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health

Cite this

Intrathecal Injections in Children with Spinal Muscular Atrophy : Nusinersen Clinical Trial Experience. / Haché, Manon; Swoboda, Kathryn J.; Sethna, Navil; Farrow-Gillespie, Alan; Khandji, Alexander; Xia, Shuting; Bishop, Kathie M.

In: Journal of Child Neurology, Vol. 31, No. 7, 2015, p. 899-906.

Research output: Contribution to journalArticle

Haché, Manon ; Swoboda, Kathryn J. ; Sethna, Navil ; Farrow-Gillespie, Alan ; Khandji, Alexander ; Xia, Shuting ; Bishop, Kathie M. / Intrathecal Injections in Children with Spinal Muscular Atrophy : Nusinersen Clinical Trial Experience. In: Journal of Child Neurology. 2015 ; Vol. 31, No. 7. pp. 899-906.
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