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Abstract Details

Re-weighting MDS-UPDRS Parts II and III Items to Improve Assessment of Motor Decline in Untreated Parkinson’s Disease
Movement Disorders
P7 - Poster Session 7 (5:00 PM-6:00 PM)
5-014

PARCOMS-Motor was developed using two different datasets (natural history and clinical trial placebo arms) to optimize motor decline assessment in patients with untreated PD.

There is considerable interest in improving the sensitivity of Parkinson’s disease (PD) measures to adequately capture the effect of disease modifying therapies.

Subjects with confirmed PD naïve to dopaminergic treatment from the Parkinson’s Progression Markers Initiative (PPMI) and the Critical Path for Parkinson’s (CPP) datasets were included in the analysis. Items from MDS-UPDRS Parts II and III were selected and weighted based on responsiveness to clinical decline using partial least square regression. The responsiveness of PARCOMS-Motor was measured using a 1-year mean-to-standard-deviation ratio (MSDR), a ratio of signal-to-noise with higher values indicating better sensitivity.

In PARCOMS-Motor, 34 of the 46 items from the MDS-UPDRS Parts II and III were retained in either the CPP or PPMI derived composite scales: 17 (50%) were retained in both the PPMI and the CPP derived composite scales; six (18%) were included only in the CPP composite scale; and 11 (32%) were retained only in the PPMI scale. Items retained in both PPMI and CPP derived scales predominantly measured irregular/involuntary movement (including items capturing bradykinesia, tremor and rigidity) concepts. In both scales, activities of daily living and oral dysfunction items had similar weights. In PARCOMS-Motor there was a 13.1% and 27.5% increase in the MSDR compared to the original scales, for PPMI and CPP respectively.

More responsive scales were derived from the MDS-UPDRS motor items using two distinct data sources. The two PARCOMS-Motor scales were not entirely consistent, which may be explained by differences in disease characteristics of patients enrolled in clinical trials versus natural history datasets.

Authors/Disclosures
Samuel Dickson (Pentara Corporation)
PRESENTER
Mr. Dickson has nothing to disclose.
Basia Rogula Basia Rogula has received personal compensation for serving as an employee of Broadstreet HEOR.
Jordan S. Dubow, MD Dr. Dubow has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Revalesio .
Nick Kozauer (Biohaven Pharmaceuticals) Dr. Kozauer has received personal compensation for serving as an employee of Biohaven. Dr. Kozauer has stock in Biohaven.
Lauren Powell (Broadstreet HEOR) Lauren Powell has nothing to disclose.
Michele Potashman, PhD (Biohaven) Dr. Potashman has received personal compensation for serving as an employee of Biohaven Pharmaceuticals.
Patrick O'Keefe (Pentara) Patrick O'Keefe has received personal compensation for serving as an employee of Pentara.
Ellen Korol (Broadstreet HEOR) Ellen Korol has received personal compensation for serving as an employee of Broadstreet HEOR.
Madeleine Crabtree (Broadstreet HEOR) Madeleine Crabtree has nothing to disclose.
Fernanda Nagase (Broadstreet HEOR) Fernanda Nagase has nothing to disclose.
Vlad Coric No disclosure on file
Liana Rosenthal, MD (Johns Hopkins School of Medicine) Dr. Rosenthal has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biohaven Pharmaceuticals. Dr. Rosenthal has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Reata Pharmaceuticals. Dr. Rosenthal has received research support from NIH. Dr. Rosenthal has received research support from Gordon and Marilyn Macklin Foundation. Dr. Rosenthal has received research support from The Daniel B. and Florence E. Green Foundation. Dr. Rosenthal has received research support from National Ataxia Foundation. Dr. Rosenthal has received research support from Michael J. Fox Foundation. Dr. Rosenthal has received research support from Pfizer. Dr. Rosenthal has received research support from Biohaven Pharmaceuticals. Dr. Rosenthal has a non-compensated relationship as a Medical Director, ex-officio Member of the Board with National Ataxia Foundation that is relevant to AAN interests or activities.
Suzanne Hendrix, PhD (Pentara) Dr. Hendrix has received personal compensation for serving as an employee of Pentara Corporation. Dr. Hendrix has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pentara Corporation. Dr. Hendrix has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Pentara Corporation. The institution of Dr. Hendrix has received personal compensation in the range of $0-$499 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Pentara Corporation. Dr. Hendrix has or had stock in Pentara.
Gilbert J. L'Italien Gilbert J. L'Italien has received personal compensation for serving as an employee of Biohaven Pharmaceuticals. Gilbert J. L'Italien has stock in biohaven pharmaceuticals.