Xingpeng Fu , Yijie Feng , Yiqin Cui , Xiao Fang , Yicheng Yu , Jin Yu , Jingjing Qian , Feng Gao , Jingjing Ye , Shanshan Mao
{"title":"Echocardiographic evaluation of left ventricular function in children with spinal muscular atrophy before and after nusinersen treatment","authors":"Xingpeng Fu , Yijie Feng , Yiqin Cui , Xiao Fang , Yicheng Yu , Jin Yu , Jingjing Qian , Feng Gao , Jingjing Ye , Shanshan Mao","doi":"10.1016/j.jns.2025.123415","DOIUrl":null,"url":null,"abstract":"<div><h3>Background</h3><div>Spinal muscular atrophy (SMA) is a genetic neuromuscular disease associated with cardiovascular abnormalities. The impact of nusinersen treatment on myocardial function in children with SMA remains unclear. This study aimed to evaluate changes in left ventricular (LV) function in children with SMA before and after nusinersen treatment using echocardiography.</div></div><div><h3>Methods</h3><div>A prospective observational study was conducted on 35 children with SMA who received six doses of nusinersen within 10 months at a tertiary hospital in China. 35 healthy controls were included for comparison. LV function was assessed using echocardiography at baseline and after 10 months of treatment. LV dyssynchrony and myocardial strain were measured using two-dimensional speckle tracking echocardiography.</div></div><div><h3>Results</h3><div>The mean age of the SMA children was 6.58 ± 3.11 years. Before treatment, the global longitudinal strain (GLS) in the SMA group was significantly lower than in the control group (<em>p</em> < 0.001), and LV systolic synchronization was poorer (<em>p</em> < 0.001). Following nusinersen treatment, GLS increased (<em>p</em> < 0.001) and synchrony improved (<em>p</em> = 0.004) in the SMA group. However, even after 10 months of treatment, GLS in the SMA group remained lower than in the control group (<em>p</em> = 0.011), and LV synchronization was still inferior (<em>p</em> = 0.028).</div></div><div><h3>Conclusions</h3><div>Short-term nusinersen treatment improved LV function in children with SMA, as evidenced by changes in LV myocardial strain indicators. Further research is warranted to explore the treatment of myocardial injury in SMA patients.</div></div>","PeriodicalId":17417,"journal":{"name":"Journal of the Neurological Sciences","volume":"470 ","pages":"Article 123415"},"PeriodicalIF":3.2000,"publicationDate":"2025-02-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of the Neurological Sciences","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0022510X25000322","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"CLINICAL NEUROLOGY","Score":null,"Total":0}
引用次数: 0
Abstract
Background
Spinal muscular atrophy (SMA) is a genetic neuromuscular disease associated with cardiovascular abnormalities. The impact of nusinersen treatment on myocardial function in children with SMA remains unclear. This study aimed to evaluate changes in left ventricular (LV) function in children with SMA before and after nusinersen treatment using echocardiography.
Methods
A prospective observational study was conducted on 35 children with SMA who received six doses of nusinersen within 10 months at a tertiary hospital in China. 35 healthy controls were included for comparison. LV function was assessed using echocardiography at baseline and after 10 months of treatment. LV dyssynchrony and myocardial strain were measured using two-dimensional speckle tracking echocardiography.
Results
The mean age of the SMA children was 6.58 ± 3.11 years. Before treatment, the global longitudinal strain (GLS) in the SMA group was significantly lower than in the control group (p < 0.001), and LV systolic synchronization was poorer (p < 0.001). Following nusinersen treatment, GLS increased (p < 0.001) and synchrony improved (p = 0.004) in the SMA group. However, even after 10 months of treatment, GLS in the SMA group remained lower than in the control group (p = 0.011), and LV synchronization was still inferior (p = 0.028).
Conclusions
Short-term nusinersen treatment improved LV function in children with SMA, as evidenced by changes in LV myocardial strain indicators. Further research is warranted to explore the treatment of myocardial injury in SMA patients.
期刊介绍：
The Journal of the Neurological Sciences provides a medium for the prompt publication of original articles in neurology and neuroscience from around the world. JNS places special emphasis on articles that: 1) provide guidance to clinicians around the world (Best Practices, Global Neurology); 2) report cutting-edge science related to neurology (Basic and Translational Sciences); 3) educate readers about relevant and practical clinical outcomes in neurology (Outcomes Research); and 4) summarize or editorialize the current state of the literature (Reviews, Commentaries, and Editorials).
JNS accepts most types of manuscripts for consideration including original research papers, short communications, reviews, book reviews, letters to the Editor, opinions and editorials. Topics considered will be from neurology-related fields that are of interest to practicing physicians around the world. Examples include neuromuscular diseases, demyelination, atrophies, dementia, neoplasms, infections, epilepsies, disturbances of consciousness, stroke and cerebral circulation, growth and development, plasticity and intermediary metabolism.
京公网安备 11010802042870号
京ICP备2023020795号-1
分享
求助内容：
应助结果提醒方式：
扫码关注我们
