Laboratory monitoring of nusinersen safety.
Muscle Nerve
; 63(6): 902-905, 2021 06.
Article
em En
| MEDLINE
| ID: mdl-33675054
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.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Oligonucleotídeos
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Laboratórios Hospitalares
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Atrofia Muscular Espinal
Tipo de estudo:
Guideline
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Observational_studies
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Prognostic_studies
Limite:
Adolescent
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Adult
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Child
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Child, preschool
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Female
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Humans
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Infant
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Male
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Middle aged
Idioma:
En
Revista:
Muscle Nerve
Ano de publicação:
2021
Tipo de documento:
Article
País de afiliação:
Estados Unidos