Intrathecal delivery of nusinersen in individuals with complicated spines

Muscle Nerve. 2020 Jul;62(1):114-118. doi: 10.1002/mus.26899. Epub 2020 Apr 30.

Abstract

Background: The treatment of spinal muscular atrophy (SMA) with nusinersen requires intrathecal medication administration, which can be challenging in individuals with complicated spines. This retrospective case series reviews the nusinersen treatment experience at one academic medical center with children and adults with SMA and complicated spines.

Methods: Twenty medical records of individuals receiving nusinersen were reviewed and administration methods summarized and assessed.

Results: Ten children and 10 adults were treated, and 55% had complicated spines. In total, 163 treatments were given, 91 in those with complicated spines. In the complicated spines, 74% of treatments were done by means of fluoroscopic lumbar puncture, 22% by means of intrathecal Ommaya reservoir, 3% by means of palpation, and < 1% by means of computed tomography-guided transforaminal approach.

Conclusions: A large majority of individuals with complicated spines can receive intrathecal nusinersen using fluoroscopic guidance in the lumbar region. Other delivery methods are available but less frequently used.

Keywords: fluoroscopy; lumbar; nusinersen; shunt; spinal muscular atrophy; transforaminal.

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Drug Delivery Systems / methods*
  • Female
  • Fluoroscopy / methods
  • Humans
  • Infant
  • Injections, Spinal / methods
  • Male
  • Middle Aged
  • Muscular Atrophy, Spinal / diagnostic imaging*
  • Muscular Atrophy, Spinal / drug therapy*
  • Oligonucleotides / administration & dosage*
  • Retrospective Studies
  • Young Adult

Substances

  • Oligonucleotides
  • nusinersen