Angelo Antonini1, Per Odin2, Peter Schmidt3, Fernando Cubillos4, David G Standaert5, Tove Henriksen6, Joohi Jimenez-Shahed7, Ali Alobaidi8, Yash J Jalundhwala9, Yanjun Bao10, Jorge Zamudio11, Juan Carlos Parra12, Pavnit Kukreja13, Koray Onuk14, Anne M Skalicky15, Leah Kleinman16, Eddie Jones17, Sharon Metz18, Hubert H Fernandez19. 1. Parkinson and Movement Disorders Unit, Center for Neurodegenerative Diseases CESNE, Department of Neuroscience, University of Padova, Padova, Italy. Electronic address: angelo.antonini@unipd.it. 2. University of Lund, Lund, Sweden. Electronic address: per.odin@med.lu.se. 3. Brody School of Medicine, East Carolina University, Greenville, NC, USA. Electronic address: pnschmidt@gmail.com. 4. Scientific Consultant, USA. Electronic address: fer.cubillos@gmail.com. 5. Center for Neurodegeneration and Experimental Therapeutics, University of Alabama at Birmingham, Birmingham, AL, USA. Electronic address: dstandaert@uabmc.edu. 6. Movement Disorder Clinic, University Hospital of Bispebjerg, Copenhagen, Denmark. Electronic address: then0003@bbh.regionh.dk. 7. Department of Neurology, Icahn School of Medicine at Mount Sinai, New York, NY, USA. Electronic address: Joohi.Jimenez-shahed@mountsinai.org. 8. AbbVie Inc., North Chicago, IL, USA; University of Illinois at Chicago, Chicago, IL, USA. Electronic address: ali.alobaidi@abbvie.com. 9. AbbVie Inc., North Chicago, IL, USA. Electronic address: yash.jalundhwala@abbvie.com. 10. AbbVie Inc., North Chicago, IL, USA. Electronic address: carol.bao@abbvie.com. 11. AbbVie Inc., North Chicago, IL, USA. Electronic address: Jorge.zamudio@abbvie.com. 12. AbbVie Inc., North Chicago, IL, USA. Electronic address: juancarlos.parrariaza@abbvie.com. 13. AbbVie Inc., North Chicago, IL, USA. Electronic address: pavnit.kukreja@abbvie.com. 14. AbbVie Inc., North Chicago, IL, USA. Electronic address: Koray.onuk@abbvie.com. 15. Evidera, Bethesda, MD, USA. Electronic address: Anne.Skalicky@evidera.com. 16. Evidera, Bethesda, MD, USA. Electronic address: Leah.Kleinman@evidera.com. 17. Adelphi Real World, Adelphi Mill, Bollington, UK. Electronic address: eddie.jones@adelphigroup.com. 18. Parkinson's Foundation, Miami, FL, USA. Electronic address: smetz@parkinson.org. 19. Center for Neurological Restoration, Cleveland Clinic, Cleveland, OH, USA. Electronic address: FERNANH@ccf.org.
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 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.
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.
Authors: Irene A Malaty; Pablo Martinez-Martin; K Ray Chaudhuri; Per Odin; Matej Skorvanek; Joohi Jimenez-Shahed; Michael J Soileau; Susanna Lindvall; Josefa Domingos; Sarah Jones; Ali Alobaidi; Yash J Jalundhwala; Prasanna L Kandukuri; Koray Onuk; Lars Bergmann; Samira Femia; Michelle Y Lee; Jack Wright; Angelo Antonini Journal: BMC Neurol Date: 2022-01-24 Impact factor: 2.474
Authors: Alfonso Fasano; Victor S C Fung; Klaus Seppi; Zvezdan Pirtosek; Annamária Takáts; Ali Alobaidi; Koray Onuk; Lars Bergmann; Juan Carlos Parra; Bulent Elibol Journal: Acta Neurol Scand Date: 2022-05-24 Impact factor: 3.915
Authors: Diego Santos García; María Álvarez Sauco; Matilde Calopa; Fátima Carrillo; Francisco Escamilla Sevilla; Eric Freire; Rocío García Ramos; Jaime Kulisevsky; Juan Carlos Gómez Esteban; Inés Legarda; María Rosario Isabel Luquín; Juan Carlos Martínez Castrillo; Pablo Martínez-Martin; Irene Martínez-Torres; Pablo Mir; Ángel Sesar Ignacio Journal: Diagnostics (Basel) Date: 2021-12-28