Design of a virtual longitudinal observational study in Parkinson's disease (AT-HOME PD)

被引：19

作者：

Schneider, Ruth B. ^{[1
,2
]}

Omberg, Larsson ^{[3
]}

Macklin, Eric A. ^{[4
,5
]}

Daeschler, Margaret ^{[6
]}

Bataille, Lauren ^{[6
]}

Anthwal, Shalini ^{[2
]}

Myers, Taylor L. ^{[2
]}

Baloga, Elizabeth ^{[2
]}

Duquette, Sidney ^{[2
]}

Snyder, Phil ^{[3
]}

Amodeo, Katherine ^{[1
]}

Tarolli, Christopher G. ^{[1
,2
]}

Adams, Jamie L. ^{[1
,2
]}

Callahan, Katherine F. ^{[7
]}

Gottesman, Joshua ^{[6
]}

Kopil, Catherine M. ^{[6
]}

Lungu, Codrin ^{[8
]}

Ascherio, Alberto ^{[9
]}

Beck, James C. ^{[10
]}

Biglan, Kevin ^{[1
,11
]}

Espay, Alberto J. ^{[12
]}

Tanner, Caroline ^{[13
]}

Oakes, David ^{[14
]}

Shoulson, Ira ^{[1
,2
,15
]}

Novak, Dan ^{[10
]}

Kayson, Elise ^{[1
,2
]}

Dorsey, Earl Ray ^{[1
,2
]}

Mangravite, Lara ^{[3
]}

Schwarzschild, Michael A. ^{[7
]}

Simuni, Tanya ^{[16
]}

机构：

[1] Univ Rochester, Med Ctr, Dept Neurol, Rochester, NY 14642 USA

[2] Univ Rochester, Med Ctr, Ctr Hlth Technol, Rochester, NY USA

[3] Sage Bionetworks, Seattle, WA USA

[4] Massachusetts Gen Hosp, Biostat Ctr, Boston, MA 02114 USA

[5] Harvard Med Sch, Boston, MA 02115 USA

[6] Michael J Fox Fdn Parkinsons Res, New York, NY USA

[7] Massachusetts Gen Hosp, Dept Neurol, Boston, MA 02114 USA

[8] NINDS, Div Clin Res, Bethesda, MD USA

[9] Harvard TH Chan Sch Publ Hlth, Dept Nutr, Boston, MA USA

[10] Parkinsons Fdn, New York, NY USA

[11] Eli Lilly & Co, Indianapolis, IN 46285 USA

[12] Univ Cincinnati, Dept Neurol, Cincinnati, OH USA

[13] Univ Calif San Francisco, Dept Neurol, Weill Inst Neurosci, San Francisco Vet Affairs Hlth Care Syst, San Francisco, CA USA

[14] Univ Rochester, Dept Biostat, Med Ctr, Rochester, NY USA

[15] Grey Matter Technol, Sarasota, FL USA

[16] Northwestern Univ, Dept Neurol, Feinberg Sch Med, Chicago, IL USA

来源：

ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY | 2021年 / 8卷 / 02期

基金：

美国国家卫生研究院;

关键词：

CLINICAL-TRIALS; EVALUATE;

D O I：

10.1002/acn3.51236

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Objective: The expanding power and accessibility of personal technology provide an opportunity to reduce burdens and costs of traditional clinical site-centric therapeutic trials in Parkinson's disease and generate novel insights. The value of this approach has never been more evident than during the current COVID-19 pandemic. We sought to (1) establish and implement the infrastructure for longitudinal, virtual follow-up of clinical trial participants, (2) compare changes in smartphone-based assessments, online patient-reported outcomes, and remote expert assessments, and (3) explore novel digital markers of Parkinson's disease disability and progression. Methods: Participants from two recently completed phase III clinical trials of inosine and isradipine enrolled in Assessing Tele-Health Outcomes in Multiyear Extensions of Parkinson's Disease trials (AT-HOME PD), a two-year virtual cohort study. After providing electronic informed consent, individuals complete annual video visits with a movement disorder specialist, smartphone-based assessments of motor function and socialization, and patient-reported outcomes online. Results: From the two clinical trials, 226 individuals from 42 states in the United States and Canada enrolled. Of these, 181 (80%) have successfully downloaded the study's smartphone application and 161 (71%) have completed patient-reported outcomes on the online platform. Interpretation: It is feasible to conduct a large-scale, international virtual observational study following the completion of participation in brick-and-mortar clinical trials in Parkinson's disease. This study, which brings research to participants, will compare established clinical endpoints with novel digital biomarkers and thereby inform the longitudinal follow-up of clinical trial participants and design of future clinical trials.

引用

页码：308 / 320

页数：13

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