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

Antonini, Angelo ; Odin, Per LU orcid ; Schmidt, Peter ; Cubillos, Fernando ; Standaert, David G. ; Henriksen, Tove ; Jimenez-Shahed, Joohi ; Alobaidi, Ali ; Jalundhwala, Yash J. and Bao, Yanjun , et al. (2021) In Parkinsonism and Related Disorders 92. p.59-66
Abstract

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. The objective of this study was to evaluate the validity and clinical value of the tool. Methods: Driven 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... (More)

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. The objective of this study was to evaluate the validity and clinical value of the tool. Methods: Driven 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). 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 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). Conclusions: MANAGE-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.

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@article{cf15f09a-fa25-42e0-8dfb-81101f7f06f2,
  abstract     = {{<p>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. The objective of this study was to evaluate the validity and clinical value of the tool. Methods: Driven 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). Results: The tool demonstrated strong validity and clinical value among PD specialists (intraclass coefficient [ICC] 0.843; Fleiss weighted kappa [ƙ<sub>weighted</sub>] 0.79) and GN (ICC 0.690; ƙ<sub>weighted</sub> 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 &lt; 0.01). Conclusions: MANAGE-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.</p>}},
  author       = {{Antonini, Angelo and Odin, Per and Schmidt, Peter and Cubillos, Fernando and Standaert, David G. and Henriksen, Tove and Jimenez-Shahed, Joohi and Alobaidi, Ali and Jalundhwala, Yash J. and Bao, Yanjun and Zamudio, Jorge and Parra, Juan Carlos and Kukreja, Pavnit and Onuk, Koray and Skalicky, Anne M. and Kleinman, Leah and Jones, Eddie and Metz, Sharon and Fernandez, Hubert H.}},
  issn         = {{1353-8020}},
  keywords     = {{Clinician decision-making; Clinician-reported; Continuous subcutaneous apomorphine infusion (CSAI); Deep brain stimulation (DBS); Dyskinesia; Levodopa-carbidopa intestinal gel (LCIG); MANAGE-PD; Motor fluctuations; Parkinson disease; Patient identification; Wearing-off}},
  language     = {{eng}},
  pages        = {{59--66}},
  publisher    = {{Elsevier}},
  series       = {{Parkinsonism and Related Disorders}},
  title        = {{Validation and clinical value of the MANAGE-PD tool : A clinician-reported tool to identify Parkinson's disease patients inadequately controlled on oral medications}},
  url          = {{http://dx.doi.org/10.1016/j.parkreldis.2021.10.009}},
  doi          = {{10.1016/j.parkreldis.2021.10.009}},
  volume       = {{92}},
  year         = {{2021}},
}