Laboratory monitoring of nusinersen safety.

Autor: Goedeker NL; Department of Neurology, Washington University School of Medicine, St. Louis, Missouri, USA., Gibbons JL; Department of Neurology, Washington University School of Medicine, St. Louis, Missouri, USA., Varadhachary AS; Department of Neurology, Washington University School of Medicine, St. Louis, Missouri, USA., Connolly AM; Department of Pediatrics, Neurology Division, Nationwide Children's Hospital, Ohio State University, Columbus, Ohio, USA., Zaidman CM; Department of Neurology, Washington University School of Medicine, St. Louis, Missouri, USA.
Jazyk: angličtina
Zdroj: Muscle & nerve [Muscle Nerve] 2021 Jun; Vol. 63 (6), pp. 902-905. Date of Electronic Publication: 2021 Mar 16.
DOI: 10.1002/mus.27217
Abstrakt: 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.
(© 2021 Wiley Periodicals LLC.)
Databáze: MEDLINE
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