Validation and clinical value of the MANAGE-PD tool: A clinician-reported tool to identify Parkinson's disease patients inadequately controlled on oral medications

Angelo Antonini,Per Odin,Peter Schmidt,Fernando Cubillos,David G Standaert,Tove Henriksen,Joohi Jimenez-Shahed,Ali Alobaidi,Yash J Jalundhwala,Yanjun Bao,Jorge Zamudio,Juan Carlos Parra,Pavnit Kukreja,Koray Onuk,Anne M Skalicky,Leah Kleinman,Eddie Jones,Sharon Metz,Hubert H Fernandez

doi:10.1016/j.parkreldis.2021.10.009

Abstract

IntroductionMaking Informed Decisions to Aid Timely Management of Parkinson's Disease (MANAGE-PD) is a clinician-reported tool designed to facilitate timely identification and management of patients with advancing Parkinson's disease (PD) with suboptimal symptom control while on standard therapy. The objective of this study was to evaluate the validity and clinical value of the tool. MethodsDriven by structured inputs from a steering committee and panel of PD experts, the tool was developed to classify patients into 3 categories. Validity and clinical value were elucidated using a two-pronged approach: (i) hypothetical patient vignettes (n = 10) developed based on the MANAGE-PD tool and rated by 17 PD specialists and 400 general neurologists (GN) and (ii) patients with PD (n = 2546) managed in real-world clinical settings. Vignette validity was based on concordance between PD experts’ clinical judgement and MANAGE-PD vignette categorization. Patient-level data was used for known-group comparisons (validity) and discordant pair analysis (clinical value). ResultsThe tool demonstrated strong validity and clinical value among PD specialists (intraclass coefficient [ICC] 0.843; Fleiss weighted kappa [ƙweighted] 0.79) and GN (ICC 0.690; ƙweighted 0.65) using patient vignettes. MANAGE-PD also demonstrated real-world validity and clinical value based on ability to identify patients with incrementally higher clinical, economic, and humanistic PD burden across categories of the tool (p < 0.01). ConclusionsMANAGE-PD demonstrated robust validity and clinical value in identifying patients with suboptimal PD symptom control. Clinical use of MANAGE-PD may complement treatment decision-making and facilitate timely and comprehensive management of patients with advancing PD.

Highlights

Making Informed Decisions to Aid Timely Management of Parkinson’s Disease (MANAGE-PD) is a clinician-reported tool designed to facilitate timely identification and management of patients with advancing Parkinson’s disease (PD) with suboptimal symptom control while on standard therapy
This study reports on the continued efforts of a global multi-year research program focused on the development, validation, and assess ment of clinical value of Making Informed Decisions to Aid Timely Management of Parkinson’s Disease (MANAGE-PD)
The MANAGE-PD tool allows for the clinical evaluation of a patient to support treatment decision-making by classifying a patient into 1 of 3 categories: 1) controlled on current treatment regimen, 2) inadequately controlled on current treatment regimen but may potentially benefit with further non-device aided treatment optimization, or 3) inade quately controlled on current treatment regimen and may benefit from device-aided therapy

Summary

Introduction

Making Informed Decisions to Aid Timely Management of Parkinson’s Disease (MANAGE-PD) is a clinician-reported tool designed to facilitate timely identification and management of patients with advancing Parkinson’s disease (PD) with suboptimal symptom control while on standard therapy. Validity and clinical value were elucidated using a two-pronged approach: (i) hypothetical patient vignettes (n = 10) developed based on the MANAGE-PD tool and rated by 17 PD spe cialists and 400 general neurologists (GN) and (ii) patients with PD (n = 2546) managed in real-world clinical settings. Results: The tool demonstrated strong validity and clinical value among PD specialists (intraclass coefficient [ICC] 0.843; Fleiss weighted kappa [ƙweighted] 0.79) and GN (ICC 0.690; ƙweighted 0.65) using patient vignettes. MANAGE-PD demonstrated real-world validity and clinical value based on ability to identify patients with incrementally higher clinical, economic, and humanistic PD burden across categories of the tool (p < 0.01). While prevalent in PD, motor fluctuations are under-recognized by routine neurological clinical evaluation [5]

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