Intrathecal nusinersen treatment for SMA in a dedicated neuromuscular clinic: an example of multidisciplinary and integrated care

Neurol Sci. 2019 Feb;40(2):327-332. doi: 10.1007/s10072-018-3622-9. Epub 2018 Nov 15.

Abstract

Nusinsersen is now available in Italy for all SMA types. We describe the experience with intrathecal treatment with nusinersen in 50 patients with SMA at the NEMO Center (NEuroMuscular Omniservice Clinical Center) in Milan, a neuromuscular patient-centered clinic hosted within Niguarda Hospital, a National Public General Hospital. Our results indicate that the pathway of care described outweighs the burden due to the repeated intrathecal injections. Irrespective of age and severity, the treatment is feasible, accessible, and replicable provided that there is a multidisciplinary team having experience and training in SMA.

Keywords: Integrated care; Multidisciplinary; Nusinersen; Spinal muscular atrophy; Spinraza.

MeSH terms

  • Adolescent
  • Child
  • Child, Preschool
  • Delivery of Health Care, Integrated* / methods
  • Family
  • Geography, Medical
  • Humans
  • Infant
  • Injections, Spinal
  • Muscular Atrophy, Spinal / complications
  • Muscular Atrophy, Spinal / diagnosis
  • Muscular Atrophy, Spinal / drug therapy*
  • Neuroprotective Agents / administration & dosage*
  • Neuroprotective Agents / adverse effects
  • Oligonucleotides / administration & dosage*
  • Oligonucleotides / adverse effects
  • Patient Care Team
  • Patient Dropouts
  • Scoliosis / complications
  • Scoliosis / diagnostic imaging
  • Spinal Puncture
  • Spine / diagnostic imaging

Substances

  • Neuroprotective Agents
  • Oligonucleotides
  • nusinersen