Abstract：
UNASSIGNED: 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).
UNASSIGNED: 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.
UNASSIGNED: 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.
UNASSIGNED: 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 15/22 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).
UNASSIGNED: 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.
UNASSIGNED: 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.
UNASSIGNED: 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.
UNASSIGNED: 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 15/22 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).
UNASSIGNED: 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.
摘要：
■反应更灵敏，可靠,临床上有效的残疾终点对缩小体型至关重要，持续时间,和成人脊髓性肌萎缩症（aPwSMA）的临床试验负担。
■目的是研究基于智能手机的评估在aPwSMA中的可行性，并提供关于在aPwSMA中远程收集的移动性和手动灵活性的传感器衍生度量（SDM）的可靠性和构造有效性的证据。
■数据来自59个aPwSMA（23个步行者，20名保姆和16名非保姆）和30名年龄匹配的健康对照（HC）。SDM是从五项基于智能手机的测试中提取的，这些测试捕获了移动性和手动灵活性，在临床和日常生活中远程给药四周。可靠性(组内相关系数，ICC)和构建效度(区分HC和aPwSMA的能力以及与修订的上肢模块的相关性,RULM和Hammersmith功能量表-扩展的HFMSE）对所有SDM进行了量化。
■基于智能手机的评估被证明是可行的，aPwSMA平均依从性为92.1%。SDM允许可靠地评估移动性和灵活性(15/22SDM的ICC>0.75)。22个SDM中有21个在HC和aPwSMA之间有明显区别。在两个非保姆的手动灵活性测试中，SDM与RULM的相关性最高(分型，ρ=0.78)和坐席(Pinching，ρ=0.75）。在步行者中，最高的相关性是流动性测试和HFMSE(5个U-Turns，ρ=0.79）。
■这项探索性研究为在参与者\'日常生活中远程部署时，基于智能手机对aPwSMA中的移动性和手动灵活性进行评估的可用性提供了初步证据。证明了在现实生活中远程收集的SDM的可靠性和构造有效性，这是他们在纵向试验中使用的先决条件。此外,成功为aPwSMA建立了三个新颖的基于智能手机的性能结果评估。在进一步验证对干预措施的反应后，这项技术具有提高aPwSMA临床试验效率的潜力.
■目的是研究基于智能手机的评估在aPwSMA中的可行性，并提供关于在aPwSMA中远程收集的移动性和手动灵活性的传感器衍生度量（SDM）的可靠性和构造有效性的证据。
■数据来自59个aPwSMA（23个步行者，20名保姆和16名非保姆）和30名年龄匹配的健康对照（HC）。SDM是从五项基于智能手机的测试中提取的，这些测试捕获了移动性和手动灵活性，在临床和日常生活中远程给药四周。可靠性(组内相关系数，ICC)和构建效度(区分HC和aPwSMA的能力以及与修订的上肢模块的相关性,RULM和Hammersmith功能量表-扩展的HFMSE）对所有SDM进行了量化。
■基于智能手机的评估被证明是可行的，aPwSMA平均依从性为92.1%。SDM允许可靠地评估移动性和灵活性(15/22SDM的ICC>0.75)。22个SDM中有21个在HC和aPwSMA之间有明显区别。在两个非保姆的手动灵活性测试中，SDM与RULM的相关性最高(分型，ρ=0.78)和坐席(Pinching，ρ=0.75）。在步行者中，最高的相关性是流动性测试和HFMSE(5个U-Turns，ρ=0.79）。
■这项探索性研究为在参与者\'日常生活中远程部署时，基于智能手机对aPwSMA中的移动性和手动灵活性进行评估的可用性提供了初步证据。证明了在现实生活中远程收集的SDM的可靠性和构造有效性，这是他们在纵向试验中使用的先决条件。此外,成功为aPwSMA建立了三个新颖的基于智能手机的性能结果评估。在进一步验证对干预措施的反应后，这项技术具有提高aPwSMA临床试验效率的潜力.
