Mouse Nerve Growth Factor Injection and Progression Rate in Patients With Amyotrophic Lateral Sclerosis: An Observational Study.
Mouse Nerve Growth Factor Injection and Progression Rate in Patients With Amyotrophic Lateral Sclerosis: An Observational Study.
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DOI：
10.3389/fneur.2022.829569
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发表时间：
2022
影响因子：
3.4
通讯作者：
Chen XJ
中科院分区：
文献类型：
--
作者：
Li JT;Dong SQ;Qian T;Yang WB;Chen XJ
肌萎缩侧索硬化症（amyotrophiclateralsclerosis，ALS）是一种进行性、致死性疾病，目前尚无有效的治疗方法.本研究旨在分析使用小鼠神经生长因子（mNGF）治疗的患者的ALS进展，以及治疗的效果，副作用和不良事件。进行了一项回顾性观察性研究，包括2020年7月至2021年7月的94名ALS患者。其中32例在常规使用利鲁唑的基础上接受至少一个疗程的mNGF治疗，其余62例仅接受利鲁唑治疗。比较两组患者的体质指数（BMI）下降率和ALS功能评定量表修订版（ALSFRS-R）评分，以判断ALS进展情况。两个队列之间没有观察到由BMI和ALSFRS-R评分下降率指示的ALS进展的显著差异。mNGF第一个疗程前后ALS进展也没有明显差异。然而，我们注意到，当比较mNGF +利鲁唑治疗与仅利鲁唑治疗时，BMI和ALSFRS-R运动分项评分的下降率分别中度降低62.7%和25.1%。mNGF治疗总体安全且耐受性良好，mNGF注射后报告了一例罕见的腹泻病例。我们的研究表明，mNGF治疗ALS患者总体安全且耐受性良好。应用mNGF联合常规利鲁唑治疗对延缓ALS进展无明显临床效果。为了得出更有说服力的结论，需要进行基于更大队列和更长随访时间的前瞻性队列研究和随机临床试验。
Amyotrophic lateral sclerosis (ALS) is a progressive, fatal disease with no curative treatment up to now. This study aims to analyze ALS progression of patients treated with mouse nerve growth factor (mNGF), as well as the effects, side effects, and adverse events of the therapy. A retrospective, observational study was performed including 94 patients with ALS from July 2020 to July 2021. Thirty-two of them were treated with at least one course of mNGF on a regular riluzole use, and the rest 62 were treated with riluzole only. The declining rates of body mass index (BMI) and ALS Functional Rating Scale-Revised (ALSFRS-R) scores were compared between the two groups to indicate ALS progression. No significant differences in ALS progression indicated by the declining rates of BMI and ALSFRS-R score were observed between the two cohorts. ALS progression before and after the first treatment course of mNGF also showed no discernible difference. However, we noticed a moderate 62.7 and 25.1% reduction in the declining rate of BMI and ALSFRS-R motor subscore when comparing mNGF + riluzole treatment to riluzole only. The mNGF treatment was overall safe and well-tolerated, and a rare case of diarrhea was reported after mNGF injection. Our study revealed that mNGF treatment was overall safe and well-tolerated in patients of ALS. Application of mNGF combined with regular riluzole treatment had no significant clinical effects on delaying ALS progression. Prospective cohort studies and randomized clinical trials based on larger cohorts and longer follow-up times are needed to make a more convincing conclusion.
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影响因子：
3.4
作者：
Gustafsson, E;Andsberg, G;Kokaia, Z
通讯作者：
Kokaia, Z
影响因子：
4.4
作者：
Cedarbaum, JM;Stambler, N;Nakanishi, A
通讯作者：
Nakanishi, A
DOI：
10.1073/pnas.0706192104
发表时间：
2007-08-21
影响因子：
11.1
作者：
Cui, Bianxiao;Wu, Chengbiao;Chu, Steven
通讯作者：
Chu, Steven
影响因子：
4.2
作者：
Murdoch, I;Perry, EK;Dewar, D
通讯作者：
Dewar, D
影响因子：
6
作者：
Lunetta, Christian;Moglia, Cristina;Calvo, Andrea
通讯作者：
Calvo, Andrea