title: Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience.
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.
authors.affiliation: Division of Pediatric Anesthesia, Columbia University Medical Center, New York, NY, USA mh2289@cumc.columbia.edu.
authors.forename: Manon
authors.initials: M
authors.lastname: Haché
authors.affiliation: Department of Neurology, Massachusetts General Hospital, Boston, MA, USA.
authors.forename: Kathryn J
authors.initials: KJ
authors.lastname: Swoboda
authors.affiliation: Department of Anesthesiology, Perioperative and Pain Medicine, Boston Children's Hospital, Boston, MA, USA.
authors.forename: Navil
authors.initials: N
authors.lastname: Sethna
authors.affiliation: Department of Anesthesiology and Pain Management, University of Texas Southwestern Medical School, Dallas, TX, USA.
authors.forename: Alan
authors.initials: A
authors.lastname: Farrow-Gillespie
authors.affiliation: Department of Radiology, Columbia University, New York, NY, USA.
authors.forename: Alexander
authors.initials: A
authors.lastname: Khandji
authors.affiliation: Ionis Pharmaceuticals, Inc., Carlsbad, CA, USA.
authors.forename: Shuting
authors.initials: S
authors.lastname: Xia
authors.affiliation: Ionis Pharmaceuticals, Inc., Carlsbad, CA, USA.
authors.forename: Kathie M
authors.initials: KM
authors.lastname: Bishop
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007278:Injections, Spinal / Q000009:adverse effects; D008297:Male; D009134:Muscular Atrophy, Spinal / Q000188:drug therapy*; D009465:Neuromuscular Agents / Q000008:administration & dosage* / Q000009:adverse effects; D009841:Oligonucleotides / Q000008:administration & dosage* / Q000009:adverse effects; D013129:Spinal Puncture / Q000009:adverse effects
chemical_list: D009465:Neuromuscular Agents; D009841:Oligonucleotides; C000590926:nusinersen
keywords: antisense oligonucleotide; drug delivery; lumbar puncture; spinal muscular atrophy
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