Progression of atypical parkinsonian syndromes: PROSPECT-M-UK study implications for clinical trials.

Male Humans Middle Aged Aged Female Parkinsonian Disorders / diagnostic imaging Supranuclear Palsy, Progressive / diagnostic imaging Multiple System Atrophy / diagnostic imaging Magnetic Resonance Imaging United Kingdom
clinical trials corticobasal syndrome multiple system atrophy progressive supranuclear palsy sample size

Journal

Brain : a journal of neurology
ISSN: 1460-2156
Titre abrégé: Brain
Pays: England
ID NLM: 0372537

Informations de publication

Date de publication:
01 08 2023
Historique:
received: 24 11 2022
revised: 11 02 2023
accepted: 21 02 2023
medline: 3 8 2023
pubmed: 29 3 2023
entrez: 28 3 2023
Statut: ppublish

Résumé

The advent of clinical trials of disease-modifying agents for neurodegenerative disease highlights the need for evidence-based end point selection. Here we report the longitudinal PROSPECT-M-UK study of progressive supranuclear palsy (PSP), corticobasal syndrome (CBS), multiple system atrophy (MSA) and related disorders, to compare candidate clinical trial end points. In this multicentre UK study, participants were assessed with serial questionnaires, motor examination, neuropsychiatric and MRI assessments at baseline, 6 and 12 months. Participants were classified by diagnosis at baseline and study end, into Richardson syndrome, PSP-subcortical (PSP-parkinsonism and progressive gait freezing subtypes), PSP-cortical (PSP-frontal, PSP-speech and language and PSP-CBS subtypes), MSA-parkinsonism, MSA-cerebellar, CBS with and without evidence of Alzheimer's disease pathology and indeterminate syndromes. We calculated annual rate of change, with linear mixed modelling and sample sizes for clinical trials of disease-modifying agents, according to group and assessment type. Two hundred forty-three people were recruited [117 PSP, 68 CBS, 42 MSA and 16 indeterminate; 138 (56.8%) male; age at recruitment 68.7 ± 8.61 years]. One hundred and fifty-nine completed the 6-month assessment (82 PSP, 27 CBS, 40 MSA and 10 indeterminate) and 153 completed the 12-month assessment (80 PSP, 29 CBS, 35 MSA and nine indeterminate). Questionnaire, motor examination, neuropsychiatric and neuroimaging measures declined in all groups, with differences in longitudinal change between groups. Neuroimaging metrics would enable lower sample sizes to achieve equivalent power for clinical trials than cognitive and functional measures, often achieving N < 100 required for 1-year two-arm trials (with 80% power to detect 50% slowing). However, optimal outcome measures were disease-specific. In conclusion, phenotypic variance within PSP, CBS and MSA is a major challenge to clinical trial design. Our findings provide an evidence base for selection of clinical trial end points, from potential functional, cognitive, clinical or neuroimaging measures of disease progression.

Identifiants

DOI: 10.1093/brain/awad105 PMID: 36975168 PMC: PMC10393398
pubmed: 36975168
pii: 7091433
doi: 10.1093/brain/awad105
pmc: PMC10393398
mid: EMS173240
doi:

Types de publication

Multicenter Study Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

3232-3242

Subventions

Organisme : Medical Research Council
ID : MR/M008525/1
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/Y008219/1
Pays : United Kingdom
Organisme : Wellcome Trust
ID : 220258
Pays : United Kingdom
Organisme : Medical Research Council
ID : MR/T046015/1
Pays : United Kingdom
Organisme : Wellcome Trust
ID : 103838
Pays : United Kingdom

Informations de copyright

© The Author(s) 2023. Published by Oxford University Press on behalf of the Guarantors of Brain.

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Auteurs

Duncan Street (D)

University of Cambridge Department of Clinical Neurosciences and Cambridge University Hospitals NHS Trust, Cambridge, CB2 OQQ, UK.
ORCID: 0000-0003-2168-2242

Edwin Jabbari (E)

Department of Clinical and Movement Neurosciences, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Movement Disorders Centre, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
ORCID: 0000-0001-6844-882X

Alyssa Costantini (A)

Department of Clinical and Movement Neurosciences, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Movement Disorders Centre, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.

P Simon Jones (PS)

University of Cambridge Department of Clinical Neurosciences and Cambridge University Hospitals NHS Trust, Cambridge, CB2 OQQ, UK.

Negin Holland (N)

University of Cambridge Department of Clinical Neurosciences and Cambridge University Hospitals NHS Trust, Cambridge, CB2 OQQ, UK.

Timothy Rittman (T)

University of Cambridge Department of Clinical Neurosciences and Cambridge University Hospitals NHS Trust, Cambridge, CB2 OQQ, UK.
ORCID: 0000-0003-1063-6937

Marte T Jensen (MT)

Department of Clinical and Movement Neurosciences, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Movement Disorders Centre, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.

Viorica Chelban (V)

Department of Neuromuscular Diseases, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Neurobiology and Medical Genetics Laboratory, 'Nicolae Testemitanu' State University of Medicine and Pharmacy, Chisinau 2004, Republic of Moldova.
ORCID: 0000-0002-7797-0756

Yen Y Goh (YY)

Department of Neuromuscular Diseases, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.

Tong Guo (T)

Department of Clinical and Movement Neurosciences, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.

Amanda J Heslegrave (AJ)

Department of Neurodegenerative Disease, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
UK Dementia Research Institute, University College London, London, W1T 7NF, UK.

Federico Roncaroli (F)

Geoffrey Jefferson Brain Research Centre, Division of Neuroscience, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, M6 8HD, UK.

Johannes C Klein (JC)

Wellcome Centre for Integrative Neuroimaging, Oxford Centre for Functional MRI of the Brain, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, OX3 9DU, UK.
Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, OX3 9DU, UK.

Olaf Ansorge (O)

Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, OX3 9DU, UK.

Kieren S J Allinson (KSJ)

University of Cambridge Department of Clinical Neurosciences and Cambridge University Hospitals NHS Trust, Cambridge, CB2 OQQ, UK.

Zane Jaunmuktane (Z)

Department of Clinical and Movement Neurosciences, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Queen Square Brain Bank for Neurological Disorders, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Reta Lila Weston Institute, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.

Tamas Revesz (T)

Queen Square Brain Bank for Neurological Disorders, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Reta Lila Weston Institute, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.

Thomas T Warner (TT)

Queen Square Brain Bank for Neurological Disorders, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Reta Lila Weston Institute, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
ORCID: 0000-0001-6195-6995

Andrew J Lees (AJ)

Queen Square Brain Bank for Neurological Disorders, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Reta Lila Weston Institute, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
ORCID: 0000-0002-2476-4385

Henrik Zetterberg (H)

Department of Neurodegenerative Disease, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
UK Dementia Research Institute, University College London, London, W1T 7NF, UK.
Clinical Neurochemistry Laboratory, Sahlgrenska University Hospital, 431 30 Mölndal, Sweden.
Department of Psychiatry and Neurochemistry, Institute of Neuroscience and Physiology, The Salhgrenska Academy at the University of Gothenburg, 413 45 Goteborg, Sweden.
Hong Kong Center for Neurodegenerative Diseases, Hong Kong Science Park, Shatin, N.T., Hong Kong, China.

Lucy L Russell (LL)

Department of Neurodegenerative Disease, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.

Martina Bocchetta (M)

Centre for Cognitive and Clinical Neuroscience, Division of Psychology, Department of Life Sciences, College of Health, Medicine and Life Sciences, Brunel University London, London, UB8 3PH, UK.
Dementia Research Centre, Department of Neurodegenerative Disease, UCL Queen Square Institute of Neurology, University College London, London, WC1N 3BG, UK.
ORCID: 0000-0003-1814-5024

Jonathan D Rohrer (JD)

Department of Neurodegenerative Disease, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.

David J Burn (DJ)

Faculty of Medical Sciences, Newcastle University, Newcastle, NE2 4HH, UK.

Nicola Pavese (N)

Clinical Ageing Research Unit, Newcastle University, Newcastle, NE4 5PL, UK.

Alexander Gerhard (A)

Division of Neuroscience, Wolfson Molecular Imaging Centre, University of Manchester, Manchester, N20 3LJ, UK.
Departments of Geriatric Medicine and Nuclear Medicine, Center for Translational Neuro- and Behavioral Sciences, University Medicine Essen, 45356 Essen, Germany.
ORCID: 0000-0002-8071-6062

Christopher Kobylecki (C)

Division of Neuroscience, Wolfson Molecular Imaging Centre, University of Manchester, Manchester, N20 3LJ, UK.
Department of Neurology, Manchester Academic Health Science Centre, Northern Care Alliance NHS Foundation Trust, Salford, M13 9NQ, UK.

P Nigel Leigh (PN)

Department of Neuroscience, Brighton and Sussex Medical School, Brighton, BN1 9PX, UK.

Alistair Church (A)

Department of Neurology, Royal Gwent Hospital, Newport, NP20 2UB, UK.

Michele T M Hu (MTM)

Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, OX3 9DU, UK.
Department of Physiology, Anatomy and Genetics, Oxford Parkinson's Disease Centre, University of Oxford, Oxford, OX1 3QU, UK.

Henry Houlden (H)

Department of Clinical and Movement Neurosciences, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Movement Disorders Centre, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Department of Neuromuscular Diseases, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
ORCID: 0000-0002-2866-7777

Huw Morris (H)

Department of Clinical and Movement Neurosciences, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
Movement Disorders Centre, University College London, Queen Square Institute of Neurology, London, WC1N 3BG, UK.
ORCID: 0000-0002-5473-3774

James B Rowe (JB)

University of Cambridge Department of Clinical Neurosciences and Cambridge University Hospitals NHS Trust, Cambridge, CB2 OQQ, UK.
Medical Research Council Cognition and Brain Sciences Unit, University of Cambridge, Cambridge, CB2 7EF, UK.

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