Longitudinal Evaluation of the Parkinson’s Disease-health Index: A Novel, Disease-specific Patient-reported Outcome Measure
Jamison Seabury1, Charlotte Engebrecht1, Jennifer Weinstein1, Anika Varma1, Spencer Rosero2, Shaweta Khosa1, Christina Shupe1, Charlotte Irwin1, John Heatwole3, Abigail Arky4, Christine Zizzi1, Nuran Dilek5, Jennifer Purks6, Jamie Adams6, E. Ray Dorsey1, Chad Heatwole1
1University of Rochester Center for Health + Technology, 2University of Utah Spencer Fox Eccles School of Medicine, 3Cornell University College of Agriculature and Life Science, 4Des Moines University of Medicine, 5University of Rochester Department of Neurology, 6University of Rochester Medical Center
Objective:

To assess and optimize the longitudinal responsiveness and usability of a regulatory-grade patient-reported outcome (PRO) measure for Parkinson’s disease (PD) known as the Parkinson’s Disease-Health Index (PD-HI).

Background:

We previously developed the PD-HI using large-scale patient-reported data and validated it in accordance with FDA guidelines for use in clinical trials. The present study implemented the PD-HI in a natural history study of adults with PD to determine longitudinal performance metrics of the instrument, optimize its usability, and obtain PD natural history data.

Design/Methods:

Adults with PD are participating in a remote, 2-year natural history study. Participants are scheduled to complete PROs at baseline and every 6 months. PROs include the PD-HI, the Neuro-QOL, and the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale parts Ib and II. Participants are also completing a survey preference questionnaire and a global impression of change form. This data will provide longitudinal performance metrics of the PD-HI, inform participant preferences with using the various PROs, identify which symptomatic domains progress the fastest in PD, and determine which subgroups are at risk of disease progression.

Results:

To date, 255 adults with PD have enrolled in this study, and 128 have completed all baseline assessments. Participants range in age from 40-85 years, are 53.4% female, and represent 9 countries. Nearly half of participants have a master’s or doctorate degree (48.7%), 8.9% carry a genetic diagnosis of PD, 27.3% report gait freezing, and 15.2% use a deep brain stimulator.  Longitudinal data regarding disease progression as measured by the PD-HI is forthcoming. 

Conclusions:
The PD-HI is a novel PRO designed to measure clinically-meaningful changes in disease burden over time. Ongoing analysis will determine which baseline characteristics are associated with slower or faster disease progression and further characterize the responsiveness of the PD-HI for detecting therapeutic gains during interventional trials.
10.1212/WNL.0000000000206168