Smartphone-Based Assessment of Mobility and Manual Dexterity in Adult People with Spinal Muscular Atrophy.

IF 3.2 4区医学 Q2 CLINICAL NEUROLOGY

Journal of neuromuscular diseases Pub Date : 2024-01-01 DOI:10.3233/JND-240004

Eduardo Arteaga-Bracho, Gautier Cosne, Christoph Kanzler, Angelos Karatsidis, Claudia Mazzà, Joaquin Penalver-Andres, Cong Zhu, Changyu Shen, Kelley Erb M, Maren Freigang, Hanna-Sophie Lapp, Simone Thiele, Stephan Wenninger, Erik Jung, Susanne Petri, Markus Weiler, Christoph Kleinschnitz, Maggie C Walter, René Günther, Nolan Campbell, Shibeshih Belachew, Tim Hagenacker

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引用次数: 0

Abstract

Background: More responsive, reliable, and clinically valid endpoints of disability are essential to reduce size, duration, and burden of clinical trials in adult persons with spinal muscular atrophy (aPwSMA).

Objective: The aim is to investigate the feasibility of smartphone-based assessments in aPwSMA and provide evidence on the reliability and construct validity of sensor-derived measures (SDMs) of mobility and manual dexterity collected remotely in aPwSMA.

Methods: Data were collected from 59 aPwSMA (23 walkers, 20 sitters and 16 non-sitters) and 30 age-matched healthy controls (HC). SDMs were extracted from five smartphone-based tests capturing mobility and manual dexterity, which were administered in-clinic and remotely in daily life for four weeks. Reliability (Intraclass Correlation Coefficients, ICC) and construct validity (ability to discriminate between HC and aPwSMA and correlations with Revised Upper Limb Module, RULM and Hammersmith Functional Scale - Expanded HFMSE) were quantified for all SDMs.

Results: The smartphone-based assessments proved feasible, with 92.1% average adherence in aPwSMA. The SDMs allowed to reliably assess both mobility and dexterity (ICC > 0.75 for 14/22 SDMs). Twenty-one out of 22 SDMs significantly discriminated between HC and aPwSMA. The highest correlations with the RULM were observed for SDMs from the manual dexterity tests in both non-sitters (Typing, ρ= 0.78) and sitters (Pinching, ρ= 0.75). In walkers, the highest correlation was between mobility tests and HFMSE (5 U-Turns, ρ= 0.79).

Conclusions: This exploratory study provides preliminary evidence for the usability of smartphone-based assessments of mobility and manual dexterity in aPwSMA when deployed remotely in participants' daily life. Reliability and construct validity of SDMs remotely collected in real-life was demonstrated, which is a pre-requisite for their use in longitudinal trials. Additionally, three novel smartphone-based performance outcome assessments were successfully established for aPwSMA. Upon further validation of responsiveness to interventions, this technology holds potential to increase the efficiency of clinical trials in aPwSMA.

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基于智能手机的脊髓肌肉萎缩症成人行动能力和手部灵活性评估。

背景：更灵敏、可靠和临床有效的残疾终点对于减少成年脊髓性肌肉萎缩症患者（aPwSMA）临床试验的规模、持续时间和负担至关重要：目的：旨在研究基于智能手机的评估在 aPwSMA 中的可行性，并为远程收集的 aPwSMA 移动性和手部灵活性的传感器衍生测量（SDM）的可靠性和构建有效性提供证据：方法：从 59 名 aPwSMA（23 名步行者、20 名坐着者和 16 名非坐着者）和 30 名年龄匹配的健康对照者（HC）中收集数据。SDM是从五项基于智能手机的测试中提取的，这些测试捕捉了行动能力和手部灵活性，在诊所和日常生活中进行了为期四周的远程测试。对所有 SDMs 的可靠性（类内相关系数，ICC）和结构效度（区分 HC 和 aPwSMA 的能力以及与修订版上肢模块 RULM 和哈默史密斯功能量表 - 扩展版 HFMSE 的相关性）进行了量化：结果：基于智能手机的评估证明是可行的，在 aPwSMA 中的平均依从率为 92.1%。SDMs 可以可靠地评估移动性和灵活性（15/22 个 SDMs 的 ICC > 0.75）。在 22 项 SDM 中，有 21 项能明显区分 HC 和 aPwSMA。在非坐位者（打字，ρ= 0.78）和坐位者（捏，ρ= 0.75）中，手部灵活性测试的 SDM 与 RULM 的相关性最高。在步行者中，活动能力测试与 HFMSE 之间的相关性最高（5 个 U 形转弯，ρ= 0.79）：这项探索性研究提供了初步证据，证明基于智能手机的行动能力和手部灵活性评估在参与者的日常生活中进行远程部署时的可用性。研究证明了在现实生活中远程收集的 SDMs 的可靠性和结构有效性，这也是在纵向试验中使用 SDMs 的前提条件。此外，还成功地为 aPwSMA 建立了三种基于智能手机的新型性能结果评估。在对干预措施的响应性进行进一步验证后，这项技术有望提高 aPwSMA 临床试验的效率。

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 neuromuscular diseases Medicine-Neurology (clinical)

CiteScore

5.10

自引率

6.10%

发文量

102

期刊介绍： The Journal of Neuromuscular Diseases aims to facilitate progress in understanding the molecular genetics/correlates, pathogenesis, pharmacology, diagnosis and treatment of acquired and genetic neuromuscular diseases (including muscular dystrophy, myasthenia gravis, spinal muscular atrophy, neuropathies, myopathies, myotonias and myositis). The journal publishes research reports, reviews, short communications, letters-to-the-editor, and will consider research that has negative findings. The journal is dedicated to providing an open forum for original research in basic science, translational and clinical research that will improve our fundamental understanding and lead to effective treatments of neuromuscular diseases.