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脑脊液淀粉样蛋白-β42 作为一种有前途的生物标志物,能否预测成人脊髓性肌萎缩症患者对 nusinersen 的反应?

Is cerebrospinal fluid amyloid-β42 a promising biomarker of response to nusinersen in adult spinal muscular atrophy patients?

机构信息

Neurology Unit, Department of Basic Medical Sciences, Neurosciences and Sense Organs, University of Bari "Aldo Moro,", Bari, Italy.

Neurophysiopathology Unit, Department of Basic Medical Sciences, Neurosciences and Sense Organs, University of Bari "Aldo Moro,", Bari, Italy.

出版信息

Muscle Nerve. 2021 Jun;63(6):905-909. doi: 10.1002/mus.27212. Epub 2021 Mar 13.

Abstract

INTRODUCTION

Nusinersen was approved as the first treatment for all types of spinal muscular atrophy (SMA), including adults with SMA types 2 and 3. Robust biomarkers of treatment response in SMA adults are lacking. Our aim was to examine cerebrospinal fluid (CSF) amyloid-β40 (Aβ40) and amyloid-β42 (Aβ42) peptides as biomarkers of treatment response.

METHODS

Eight patients with SMA types 2 and 3 were recruited consecutively in a single-center study. CSF was sampled at baseline, after a loading dose, and after three maintenance doses. Levels of Aβ42 and Aβ40 were evaluated for each CSF sampling. Wilcoxon matched-pairs signed-rank test was used to detect longitudinal changes.

RESULTS

CSF levels of Aβ42 increased from baseline to day 420 (95% confidence interval, P = .018), with a significant increase at days 180 and 420 compared with days 0 and 300, respectively (95% confidence interval, P = .012 and P = .018).

DISCUSSION

The maintenance and promotion of wellness of residual motor neurons mediated by the restored level of SMN protein due to nusinersen could result in an increased level of amyloid peptides.

摘要

简介

依地膦酸钠已被批准用于治疗所有类型的脊髓性肌萎缩症(SMA),包括 2 型和 3 型 SMA 成人患者。但目前缺乏用于评估 SMA 成人患者药物应答的生物标志物。本研究旨在探讨脑脊液(CSF)中的淀粉样β肽 40(Aβ40)和淀粉样β肽 42(Aβ42)作为治疗应答的生物标志物。

方法

本研究为单中心、连续入组的研究,共纳入 8 例 2 型和 3 型 SMA 患者。在基线时、负荷剂量后以及维持剂量后 3 次分别采集 CSF。评估每次 CSF 采样时 Aβ42 和 Aβ40 的水平。采用 Wilcoxon 配对符号秩检验来检测纵向变化。

结果

CSF 中 Aβ42 水平从基线增加到 420 天(95%置信区间,P =.018),与第 0 天和第 300 天相比,第 180 天和第 420 天的水平分别显著升高(95%置信区间,P =.012 和 P =.018)。

讨论

依地膦酸钠通过恢复 SMN 蛋白水平来维持和促进残存运动神经元的健康,可能导致淀粉样肽水平升高。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/c291/8251706/6fcf6447e276/MUS-63-905-g001.jpg

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