Evidence map›Paper›PMID 39054521›Full record

ArticleBMC pediatrics2024

Safety analysis of laboratory parameters in paediatric patients with spinal muscular atrophy treated with nusinersen.

Xiaomei Zhu, Hui Li, Chaoping Hu, Min Wu, Shuizhen Zhou, Yi Wang, Wenhui Li

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In one paragraph

Article in BMC pediatrics, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

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1 · What the graph read from it

What it found

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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

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3 · Its place in the literature

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0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

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5 · Who and what money

Authors and funding

7 authors.

Xiaomei ZhuDepartment of Neurology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Hui LiDepartment of Rehabilitation, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Chaoping HuDepartment of Neurology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Min WuDepartment of Neurology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Shuizhen ZhouDepartment of Neurology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China. szzhou@shmu.edu.cn.
Yi WangDepartment of Neurology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China. yiwang@shmu.edu.cn.
Wenhui LiDepartment of Neurology, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China. wenhuili@fudan.edu.cn.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundSpinal muscular atrophy (SMA) is a progressive neurodegenerative disorder that can be treated with intrathecal nusinersen, an antisense oligonucleotide. In addition to efficacy, safety is a determining factor in the success of any therapy. Here, we aim to assess the safety of nusinersen therapy in paediatric patients with SMA.

methodsLaboratory data of paediatric patients with SMA who received nusinersen between October 2019 and May 2022 were retrospectively analysed.

resultsDuring the observation period, 46 infants and children aged 2.9 months to 13.6 years received a total of 213 nusinersen doses without safety concerns. Inflammatory markers were stable throughout the study. International normalized ratio was increased by 0.09 per injection. Urea levels were increased by 0.108 mmol/L, and cystatin C decreased by 0.029 mg/L per injection. There were no significant changes in platelet count, activated partial thrombin time, creatinine levels or liver enzyme levels during treatment. The cerebrospinal fluid (CSF) leukocyte count remained stable, and total protein increased by 24.038 mg/L per injection.

conclusionOur data showed that nusinersen therapy is generally safe in children with SMA. Laboratory monitoring did not identify any persistent or significantly abnormal findings. CSF protein should be monitored to gain more insights.

Indexed as

OligonucleotidesAdolescentChildChild, PreschoolFemaleHumansInfantInjections, SpinalInternational Normalized RatioMaleMuscular Atrophy, SpinalRetrospective StudiesnusinersenOligonucleotidesLaboratory parametersNusinersenSafetySide effectsSpinal muscular atrophy

Identifiers

PMID39054521
PMCPMC11270951

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.