Page Alex, Yung Norman, Auinger Peggy, Venuto Charles, Glidden Alistair, Macklin Eric, Omberg Larsson, Schwarzschild Michael A, Dorsey E Ray
Center for Health + Technology, University of Rochester Medical Center, Rochester, New York, USA.
Department of Neurology, University of Rochester Medical Center, Rochester, New York, USA.
Digit Biomark. 2022 Jan 10;6(1):1-8. doi: 10.1159/000521232. eCollection 2022.
Smartphones can generate objective measures of Parkinson's disease (PD) and supplement traditional in-person rating scales. However, smartphone use in clinical trials has been limited.
This study aimed to determine the feasibility of introducing a smartphone research application into a PD clinical trial and to evaluate the resulting measures.
A smartphone application was introduced part-way into a phase 3 randomized clinical trial of inosine. The application included finger tapping, gait, and cognition tests, and participants were asked to complete an assessment battery at home and in clinic alongside the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS).
Of 236 eligible participants in the parent study, 88 (37%) consented to participate, and 59 (27 randomized to inosine and 32 to placebo) completed a baseline smartphone assessment. These 59 participants collectively completed 1,292 batteries of assessments. The proportion of participants who completed at least one smartphone assessment was 61% at 3, 54% at 6, and 35% at 12 months. Finger tapping speed correlated weakly with the part III motor portion ( = -0.16, left hand; = -0.04, right hand) and total ( = -0.14) MDS-UPDRS. Gait speed correlated better with the same measures ( = -0.25, part III motor; = -0.34, total). Over 6 months, finger tapping speed, gait speed, and memory scores did not differ between those randomized to active drug or placebo.
Introducing a smartphone application midway into a phase 3 clinical trial was challenging. Measures of bradykinesia and gait speed correlated modestly with traditional outcomes and were consistent with the study's overall findings, which found no benefit of the active drug.
智能手机可生成帕金森病（PD）的客观测量数据，并补充传统的当面评定量表。然而，智能手机在临床试验中的应用一直有限。
本研究旨在确定将智能手机研究应用引入PD临床试验的可行性，并评估由此产生的测量数据。
在一项关于肌苷的3期随机临床试验中途引入一款智能手机应用。该应用包括手指敲击、步态和认知测试，参与者被要求在家中和诊所完成一组评估，同时完成运动障碍协会统一帕金森病评定量表（MDS-UPDRS）。
在母研究的236名符合条件的参与者中，88名（37%）同意参与，59名（27名随机分配到肌苷组，32名分配到安慰剂组）完成了基线智能手机评估。这59名参与者共完成了1292组评估。在3个月时完成至少一次智能手机评估的参与者比例为61%，6个月时为54%，12个月时为35%。手指敲击速度与MDS-UPDRS第三部分运动部分（左手r = -0.16；右手r = -0.04）及总分（r = -0.14）的相关性较弱。步态速度与相同测量指标的相关性更好（第三部分运动r = -0.25；总分r = -0.34）。在6个月期间，随机分配到活性药物组或安慰剂组的参与者在手指敲击速度、步态速度和记忆分数方面没有差异。
在3期临床试验中途引入智能手机应用具有挑战性。运动迟缓及步态速度测量指标与传统结果的相关性一般，并与该研究的总体结果一致（该研究未发现活性药物有任何益处）
