Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study

Lauren Elman, Bora Youn, Crystal M. Proud, Margaret R. Frey, Senda Ajroud-Driss, M. Eileen McCormick, David Michelson, Michael S. Cartwright, Terry Heiman-Patterson, Joseph M. Choi, Aastha Chandak, Artak Khachatryan, Marta Martinez, Angela D. Paradis*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

7 Scopus citations

Abstract

Limited evidence exists on real-world adherence to nusinersen for the treatment of spinal muscular atrophy (SMA). Data are presented from a multi-site retrospective chart review of 86 adults with SMA initiating nusinersen at nine US centers between January 2017 and February 2019. Seventy-nine (92%) adults remained on nusinersen during the study; 454 (92%) of 493 total nusinersen doses were received on time. Fifty-eight (67%) adults received all nusinersen doses on time. The majority of patients with at least one nonadherent dose resumed nusinersen on time. Most patients followed the dosing schedule across the loading and maintenance dose periods.

Original languageEnglish (US)
Pages (from-to)655-660
Number of pages6
JournalJournal of neuromuscular diseases
Volume9
Issue number5
DOIs
StatePublished - 2022

Keywords

  • Medication adherence
  • adults
  • antisense oligonucleotides
  • neuromuscular diseases
  • retrospective studies
  • spinal muscular atrophy

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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