Five-year follow-up study on quantitative muscle magnetic resonance imaging in facioscapulohumeral muscular dystrophy: The link to clinical outcome

IF 8.9 1区医学

Journal of Cachexia, Sarcopenia and Muscle Pub Date : 2023-05-23 DOI:10.1002/jcsm.13250

Sanne C.C. Vincenten, Karlien Mul, Dani?l van As, Julia J. Jansen, Linda Heskamp, Arend Heerschap, Baziel G.M. van Engelen, Nicol C. Voermans

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引用次数: 1

Abstract

Background

It is unclear how changes in quantitative muscle magnetic resonance imaging (MRI) relate to changes in clinical outcome in facioscapulohumeral muscular dystrophy (FSHD), although this information is crucial for optimal use of MRI as imaging biomarker in trials. We therefore assessed muscle MRI and clinical outcome measures in a large longitudinal prospective cohort study.

Methods

All patients were assessed by MRI at baseline and at 5-year follow-up, employing 2pt-Dixon and turbo inversion recovery magnitude (TIRM) sequences, after which fat fraction and TIRM positivity of 19 leg muscles were determined bilaterally. The MRI compound score (CoS) was defined as the mean fat fraction of all muscles weighted for cross-sectional area. Clinical outcome measures included the Ricci-score, FSHD clinical score (FSHD-CS), MRC sumscore (MRC-SS), and motor-function-measure (MFM).

Results

We included 105 FSHD patients [mean age 54 ± 14 years, median Ricci-score 7 (range 0–10)]. The median change over 5 years' time in the MRI-CoS was 2.0% (range −4.6 to +12.1; P < 0.001). The median change over 5 years' time in clinical outcome measures was small in all measures, with z-scores ranging from 5.0 to 7.2 (P < 0.001). The change in MRI-CoS correlated with change in FSHD-CS and Ricci-score (ρ = 0.25, respectively; ρ = 0.23, P < 0.05). The largest median increase in MRI-CoS was seen in baseline subgroups with an MRI-CoS 20–40% (6.1%), with ≥2 TIRM positive muscles (3.5%) or with an FSHD-CS 5–10 (3.1%).

Conclusions

This 5-year study showed significant changes in MRI and clinical outcome measures and a significant correlation between changes in MRI-CoS and changes in clinical outcome measures. In addition, we identified subgroups of patients that are most prone to radiological disease progression. This knowledge further establishes quantitative MRI parameters as prognostic biomarkers in FSHD and as efficacy biomarkers in upcoming clinical trials.

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面肩肱骨肌营养不良定量肌肉磁共振成像的五年随访研究:与临床结果的联系

目前尚不清楚定量肌肉磁共振成像(MRI)的变化与面肩肱肌营养不良症(FSHD)临床结果的变化之间的关系，尽管这一信息对于在试验中优化使用MRI作为成像生物标志物至关重要。因此，我们在一项大型纵向前瞻性队列研究中评估了肌肉MRI和临床结果测量。方法对所有患者在基线和5年随访时进行MRI评估，采用2pt-Dixon和涡轮反转恢复幅度(TIRM)序列，随后测定19块腿部肌肉的脂肪含量和TIRM阳性。MRI复合评分(CoS)定义为横截面积加权后所有肌肉的平均脂肪分数。临床结果测量包括ricci评分、FSHD临床评分(FSHD- cs)、MRC评分(MRC- ss)和运动功能测量(MFM)。结果纳入105例FSHD患者[平均年龄54±14岁，ricci评分中位数为7(范围0-10)]。5年内MRI-CoS的中位变化为2.0%(范围为- 4.6至+12.1;P & lt;0.001)。5年临床结果测量的中位变化在所有测量中都很小，z分数范围为5.0至7.2 (P <0.001)。MRI-CoS的变化与FSHD-CS和ricci评分的变化相关(ρ = 0.25;ρ = 0.23, P <0.05)。MRI-CoS中位数增加最大的是基线亚组，MRI-CoS为20-40%(6.1%)，≥2块TIRM阳性肌肉(3.5%)或FSHD-CS为5-10(3.1%)。结论这项为期5年的研究显示，MRI和临床结果指标发生了显著变化，MRI- cos的变化与临床结果指标的变化之间存在显著相关性。此外，我们确定了最容易发生放射学疾病进展的患者亚组。这些知识进一步确立了定量MRI参数作为FSHD的预后生物标志物和即将进行的临床试验的疗效生物标志物。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Journal of Cachexia, Sarcopenia and Muscle Medicine-Orthopedics and Sports Medicine

自引率

12.40%

发文量

期刊介绍： The Journal of Cachexia, Sarcopenia, and Muscle is a prestigious, peer-reviewed international publication committed to disseminating research and clinical insights pertaining to cachexia, sarcopenia, body composition, and the physiological and pathophysiological alterations occurring throughout the lifespan and in various illnesses across the spectrum of life sciences. This journal serves as a valuable resource for physicians, biochemists, biologists, dieticians, pharmacologists, and students alike.