Laboratory monitoring of nusinersen safety

Natalie L. Goedeker, Jennifer L. Gibbons, Arun S. Varadhachary, Anne M. Connolly, Craig M. Zaidman

Research output: Contribution to journalArticlepeer-review

7 Scopus citations


Introduction: This retrospective study reports our tertiary care center's experience with intrathecal nusinersen administration in children and adults with spinal muscular atrophy (SMA). Methods: We reviewed safety monitoring laboratory results and need for procedural sedation and fluoroscopy-guidance in all SMA patients receiving nusinersen between February 2017 and March 2020. Results: Fifty-eight patients ages 1 mo- 56 y received 494 nusinersen doses. There were 166 laboratory abnormalities in 45 patients. Most were either mild (145 [87.3%]) or were transient proteinuria (18 [10.8%]). None altered nusinersen treatment. Twenty-eight patients required either general anesthesia (75 doses) or anxiolysis with oral midazolam (133 doses, including 6 patients [23 doses] with SMA type I). Eight patients with complicated spines (45 doses) required fluoroscopic guidance. One treatment-related serious adverse event (emesis leading to intubation) occurred during general anesthesia. Two children had asymptomatic increased intracranial pressure. No patients discontinued treatment due to adverse events. Discussion: Intrathecal nusinersen is generally safe and well-tolerated, including in patients requiring oral anxiolysis, general sedation, and fluoroscopic guidance. Frequent serial laboratory monitoring did not identify any persistent significantly abnormal findings or alter treatment.

Original languageEnglish
Pages (from-to)902-905
Number of pages4
JournalMuscle and Nerve
Issue number6
StatePublished - Jun 2021


  • antisense oligonucleotide
  • intrathecal
  • motor neuron disease
  • nusinersen
  • safety
  • spinal muscular atrophy


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