Improving diagnosis and care in people affected by progressive supranuclear palsy (PSP) and

corticobasal degeneration (CBD)

CSO/PSP Association Clinical Research Fellowship

Dr Diane Swallow

NHS Research Scotland Annual Conference, 26th October 2016

Overview

“…to improve the evidence base on routes to diagnosis and care of people living with progressive supranuclear palsy (PSP) and

corticobasal degeneration (CBD)…”

• Background

• Study aims and overview

• Relevance to the NHS and future directions

• Benefits of the fellowship

Background PSP and CBD

• Progressive neurodegenerative diseases causing physical and dementia-like symptoms

• Significant disability with an average life expectancy of 5 – 8 years

• Misdiagnosis and delayed diagnosis common- Reasons for misdiagnosis and delayed diagnosis?- Screening tools to improve diagnosis?

• Prognosis and care- Implementation and access to multidisciplinary care?- Prognosis other than survival?- Impact on carers?

Study aims

1 Describe & improve diagnostic pathways in PSP/CBD

2 Validate & develop new diagnostic tools in PSP/CBD

- Edinburgh Cognitive and Edinburgh Motor Assessment Scales

- MRI (3T structural)

3 Describe & improve the quality of care of PSP/CBD patients from diagnosis to end-of-life

4 Evaluate activity limitations as a measure of disease progression, and assess carer outcomes

5 Establish the prevalence of PSP/CBD in Scotland using new diagnostic criteria

Study population and setting

• Existing cohorts of PSP/CBD patients and carers• PINE study

• Tracking Parkinson’s study

• New national cohort of PSP/CBD patients and carers

The PINE Study (Parkinsonism in the North East of Scotland)

PSP/CBD patients and their carers recruited from 2002-09 (n=30)

PSP/CBD patients initially thought to have Parkinson’s (n~5)

Annual assessments: motor and cognitive function, activity limitation, mood, quality of life and carer mood and care burden

6 monthly assessments: diagnostic features, risk factors, motor and non-motor features, cognition, quality of life, olfaction testing

Tracking Parkinson’s (PRoBaND)

Existing cohorts

New cohort: case finding

Existing patients with PSP/CBD over 2 to 2.5 year recruitment period:

• National- ICD-10 diagnostic coding from Scottish Morbidity Record

- Referrals from hospital specialists

- Patient self-referral

• Grampian (to determine true prevalence rate)- Referrals from GP’s

- Search of outpatient referral letters: neurology, medicine for the elderly and old age psychiatry

Inclusion criteria

Fulfil diagnostic criteria for possible or probable PSP, or

CBD

Subtypes considered to be possible allied syndromes (e.g.

PSP-parkinsonism)

New cohort: staged consent

Patients invited to consent (or assent sought) to any/all of:

I. access to medical records and linkage to health and social care registers

II. a one-off assessment visit

III. a six month postal follow-up

IV. inclusion in a new Scottish PSP/CBD register

V. 3T MR brain imaging

VI. DNA/serum for the PROgressive Supranuclear Palsy CorTicoBasal Syndrome Multiple System Atrophy Longitudinal Study UK (PROSPECT-M-UK) biomarker study

Carers invited to return questionnaires about their health, carer burden, and a proxy assessment of patients capabilities

Methods 1: Describe & improve diagnostic pathways in PSP/CBD

Using health records and interviews assess:

• Time delays (patient, primary, secondary care)

• Frequency and type of incorrect initial diagnoses

• Reasons for misdiagnosis

• Location of secondary care speciality referrals

• Potential missed diagnostic opportunities

Compare delays and error rates with age-sex matched Parkinson’s disease patients

Methods 2: Validate & develop new diagnostic tools

Edinburgh Cognitive Assessment Scale (ECAS) and Edinburgh Motor

Assessment Scale (EMAS)

Compare with established cognitive (ACE-III) and motor assessments (UPDRS Part 3)

Compare scores of PSP/CBD cases with PD cases to determine their ability to discriminate between PSP/CBD and PD

Imaging 3T MRI

Imaging features which discriminate between PSP/CBD and other parkinsonian or dementia conditions (e.g. midbrain:pons ratio)

Additional MRI scans from PINE and local imaging studies.

Methods 3: Describe & improve the quality of care from diagnosis to end-of-life

Using research records, clinical assessments and questionnaires:

• Describe clinical features (e.g. motor, cognitive, mood) at study onset and 6 months to define disease stage and care requirements

• Evaluate health service interventions and care pathway co-ordination to determine if these meet recommended standards

• Include patient/carer self report of interactions and their perceived effectiveness

Aim 4 Patient and carer prognosis, and functional activity limitations

Evaluation of activity limitation/dependency

• Existing functional scales

• Extended list of basic and instrumental ADLs- Frequency of limitation- Order of loss of independence- Activities particularly affecting quality of life

Carer prognosis

• Matched to PSP/CBD patient’s symptoms and health needs

• Compared to carers of people with PD

Future outcomes and benefits of the fellowship

Diagnosis: targeted education and adoption new tools

Care: the right interventions, to the right patients, at the right time

Prognosis: improved communication, care planning and provision, carer support

Prevalence: resource allocation for care planning

Future: earlier diagnosis, national register, well characterised clinical trial ready cohort, ongoing follow-up

Fellowship benefits: time, funding and opportunity to work in an area of clinical and academic interest, and to be equipped for a future independent academic career

Acknowledgements

Funders: Chief Scientist Office and PSP Association

Supervisors: Dr Carl Counsell, Dr Donald Grosset, Dr Thomas Bak