Muscle & nerve · 2021
Laboratory monitoring of nusinersen safety.
СтатьяНазвание и аннотация: Перевод в очереди
Обработка статьи · этапы, ошибки и управление
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Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Goedeker Natalie L, Gibbons Jennifer L, Varadhachary Arun S, Connolly Anne M, Zaidman Craig M
Аннотация
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.
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.