Objective: To characterize the lived experience of individuals at a prodromal stage of PD or with a recent clinical diagnosis.

Background: There is no validated clinical outcome assessment (COA) for assessing functional impairment for people with Parkinson (PwP) in the earliest clinical stages. Development of such a COA would improve identification of PD patients who might benefit from early treatments, and the evaluation of the therapeutic effects for novel intervention in the setting of early disease modification trials.

Method: Mixed methods approach with focus groups followed by a survey about experience of symptoms, degree of bothersomeness, importance, and functional impact. We used purposive sampling for the focus group sessions (each session, n=10) considering the following criteria: Clinical diagnosis of PD AND on dopaminergic medication <1 year; clinical diagnosis of PD < 1yr ; prodromal condition (RBD or hyposmia), and care partners and/or knowledge informants. We conducted content analyses, with identification of symptoms and corresponding functional impacts, and descriptive statistics on the results of the post-focus group survey. A consensus was obtained using a modified Delphi panel technique.

Results: We identified functional impacts related to motor and non-motor symptoms from prodromal to clinical PD groups. The focus group sessions with caregivers highlighted large discrepancies between the changes noted by the individual and care partners, with very few additional changes reported by the latter. The post-focus group survey suggested that both motor and non-motor changes from normal were more commonly rated as bothersome but lacking significant functional impact, with exception of the “treated PD” group reporting greater impacts. The reported changes were grouped in the following domains after consensus: Tremor, Gross Motor/Mobility, Fine Motor/Dexterity, Sensory Changes, Cognition, Fatigue, Sleep, Behavioral/Mood Changes, Bulbar changes, Dysautonomia, Activities of Daily Living (for functional Impacts that span symptom domains).

Conclusion: The earliest lived experience of PwP includes a comprehensive set of motor and non-motor symptoms with functional impacts, even prior to a clinical diagnosis. The results support the development of a PRO on early functional impacts and informs a comprehensive item bank

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MDS Abstracts - https://www.mdsabstracts.org/abstract/lived-experiences-in-early-clinical-pd-developing-a-framework-of-a-novel-patient-reported-outcome-pro-fit-for-purpose-for-disease-modification-therapeutic-trials/