Objective: To establish a threshold for meaningful progression on MDS-UPDRS Part III in an early-stage Parkinson’s disease (PD) population (Hoehn & Yahr [H&Y] Stage I–II) through clinical expert consensus via a modified Delphi panel (mDp).

Background: MDS-UPDRS Part III is a clinician-rated assessment of motor signs commonly used in clinical trials, practice and studies. Establishing a threshold for meaningful progression allows for the use of MDS-UPDRS Part III in progressor-based (including time-to-event) endpoints. Previously, anchor-based analyses have been conducted to estimate a threshold, suggesting a threshold of 5 points. To support these findings, clinical expert consensus was sought via an mDp.

Method: A sponsor/researcher-blind panel of expert clinicians (n=13) were recruited. The mDp had 8–40 years’ experience in PD (mean=19 years), an H-index of 20–119 (mean=40), and represented seven countries across North America and Europe. The mDp received an online survey in two rounds and provided responses anonymously. Round 1 explored initial opinions on the threshold for meaningful progression, and differences between medication state (ON vs OFF), disease stage (H&Y Stage I vs II) and treatment status (naïve vs stable symptomatic). Round 2 was conducted in two parts: 1) an aggregated overview of Round 1 followed by a question on agreement that the threshold of meaningful progression lies within 4–6 points (OFF medication state); and 2) an overview of prior anchor-based analyses followed by an evaluation of agreement with the threshold of 5 points for meaningful progression (OFF medication state). A threshold of 70% was set for establishing consensus.

Results: In Round 1, a range of 2–10 points (median=5) was suggested as a threshold for meaningful progression, with n=11 (85%) reporting that the threshold depends on medication state, n=8 (62%) reporting no difference by H&Y Stage, and n=9 (69%) reporting no difference by treatment status. In Round 2, n=12 (92%) agreed that the threshold lies within 4–6 points and n=12 (92%) agreed with the use of 5 points to define a clinically meaningful threshold of progression.

Conclusion: The mDp proved to be a useful method to support previous findings and establish a threshold of 5 points for clinically meaningful progression on MDS-UPDRS Part III (OFF medication state) in an early-stage PD population.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/a-modified-delphi-panel-to-establish-a-threshold-of-meaningful-progression-on-mds-updrs-part-iii/