Objective: To aid the development of curative treatments, we need to develop better methods for diagnosing Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD) and Multiple System Atrophy (MSA) early and to track disease progression. The PROSPECT study will capture longitudinal standardised clinical data and create a biobank of samples from patients with these conditions.

Background: PSP, CBD and MSA are rare which means that collaborative multi-centre research studies will be needed. The PROSPECT study comprises seven UK centres who are recruiting patients with atypical Parkinsonian syndromes. The primary outcome measure for the study is mortality; we will determine how baseline clinical and biologic factors determine disease progression. Secondary outcome measures include self/carer reported quality of life and attainment key disease milestones.

Methods: We will recruit cross-sectional and longitudinal clinical cohorts and a control group. We will also review patients with uncertain diagnoses who are at risk of converting to one of the core syndromes over time (Atypical Parkinsonian syndromes group – APS). The cross-sectional study involves a DNA analysis and completion of questionnaires providing details of clinical history and quality of life. The longitudinal study targets recently diagnosed patients. Core measures include blood sample collection for DNA, RNA, plasma and serum, completion of questionnaires and cognitive and functional measures. Deep phenotyping of patients will be performed in some patients at baseline and after 12 months including: a skin biopsy for developing fibroblast cell lines, cerebrospinal fluid sample collection, eye movement exam and a brain MRI. Patients will then be followed up annually for a further 5 years.

Results: So far, we have recruited 70 patients (31 PSP, 5 CBD, 32 MSA, 1 APS) in the cross-sectional study with an average age of onset of 62.5 ± 7.4y and disease duration of 5.5 ± 2.7y. We have recruited 14 in the longitudinal study (6 PSP, 5 CBD, 1 MSA, 3 APS) with an average age of onset of 67.0 ± 7.4y and disease duration 2.9 ± 1.8y. Nine controls have been recruited (Age: 66.8 ± 9.0y). Two patients have died. A cognitive/behavioural prodrome was reported in 20%of patients.

Conclusions: The PROSPECT study will advance our understanding of these diseases and establish a platform for future therapeutic and biologic studies in PSP, CBD and MSA.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/prospect-a-uk-based-longitudinal-observational-study-of-psp-cbd-msa-and-atypical-parkinsonism-syndromes/