Objective: To synthesize published literature about patient-reported symptoms (PRS) or changes from normal in prodromal and early motor stages of Parkinson’s disease (PD) and develop a conceptual framework for scale development.

Background: Incorporating the patient voice has become key for clinical research in PD.[1]  Currently, a PD diagnosis is based on the clinical assessment of core motor features of parkinsonism,[2] which may not reflect the complete impact of the variety of motor and non-motor PD symptoms as experienced by patients.[3] Regulatory bodies recognize this limitation and have focused on the importance of patient voice in developing high-quality patient centered data to support drug labeling claims.[4]

Method: We probed published literature using MEDLINE, EMBASE, SCOPUS and CINAHL databases (inception – May 15^th, 2022). We reviewed all qualitative and quantitative studies that reported on prodromal or early-motor stages of PD (clinical diagnosis <2 years) populations, regardless of study design. We only included studies published in English or French.

Results: From a total of 2687 citations, we included 59 articles reporting on 64 individual cohorts, of which 31% (n=20) related to a prodromal stage and 69% (n=40) to an early-motor PD stage. 461 reported symptoms were identified, corresponding to 85 standardized reported symptoms (SRSs). Overall, the ten most frequent PRS were non-motor. Study ad-hoc questionnaires (30%) and the MDS-UPDRS (parts I and II for Non-Motor and Motor Aspects of Experiences of Daily Living, 26%) were the most frequently used assessments in the prodromal and early-motor PD cohorts,[GS1] respectively. We standardized the reported PRS and groped them in seven symptom domains (Behavioral, Cognition, Dysautonomia, Motor, Sensory, Sleep, Others) and four functional domains: ADL, Communication, Sexual, and Social Impairment.

Conclusion: By probing the available literature, we have identified the most frequent changes from normal in prodromal and early-motor PD stages from a patient’s perspective. This review together with ongoing focus groups will inform the development of new clinical rating scale to measure the earliest changes in PD as reported by people with the lived experience.

References: 1. Mahlknecht P, Marini K, Werkmann M, Poewe W, Seppi K. Prodromal Parkinson’s disease: hype or hope for disease-modification trials? Transl Neurodegener. 2022;11(1):11. doi:10.1186/s40035-022-00286-1
2. Postuma RB, Berg D, Stern M, et al. MDS clinical diagnostic criteria for Parkinson’s disease: MDS-PD Clinical Diagnostic Criteria. Mov Disord. 2015;30(12):1591-1601. doi:10.1002/mds.26424
3. Zolfaghari S, Thomann AE, Lewandowski N, et al. Self‐Report versus Clinician Examination in Early Parkinson’s Disease. Mov Disord. 2022;37(3):585-597. doi:10.1002/mds.28884
4. Mercieca-Bebber R, King MT, Calvert MJ, Stockler MR, Friedlander M. The importance of patient-reported outcomes in clinical trials and strategies for future optimization. Patient Relat Outcome Meas. 2018;Volume 9:353-367. doi:10.2147/PROM.S156279

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MDS Abstracts - https://www.mdsabstracts.org/abstract/patient-reported-changes-from-normal-in-individuals-with-prodromal-and-early-motor-parkinsons-disease-a-scoping-review/