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. 2021 Jun;63(6):902-905.
doi: 10.1002/mus.27217. Epub 2021 Mar 16.

Laboratory monitoring of nusinersen safety

Natalie L Goedeker  1 , Jennifer L Gibbons  1 , Arun S Varadhachary  1 , Anne M Connolly  2 , Craig M Zaidman  1
Affiliations

Laboratory monitoring of nusinersen safety

Natalie L Goedeker et al. Muscle Nerve. 2021 Jun.

Abstract

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.

Keywords: antisense oligonucleotide; intrathecal; motor neuron disease; nusinersen; safety; spinal muscular atrophy.

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References

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