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Developing and validating a multivariable prediction model which predicts progression of intermediate to late age-related macular degeneration-the PINNACLE trial protocol

Developing and validating a multivariable prediction model which predicts progression of intermediate to late age-related macular degeneration-the PINNACLE trial protocol
Developing and validating a multivariable prediction model which predicts progression of intermediate to late age-related macular degeneration-the PINNACLE trial protocol
Aims: age-related macular degeneration (AMD) is characterised by a progressive loss of central vision. Intermediate AMD is a risk factor for progression to advanced stages categorised as geographic atrophy (GA) and neovascular AMD. However, rates of progression to advanced stages vary between individuals. Recent advances in imaging and computing technologies have enabled deep phenotyping of intermediate AMD. The aim of this project is to utilise machine learning (ML) and advanced statistical modelling as an innovative approach to discover novel features and accurately quantify markers of pathological retinal ageing that can individualise progression to advanced AMD.

Methods: the PINNACLE study consists of both retrospective and prospective parts. In the retrospective part, more than 400,000 optical coherent tomography (OCT) images collected from four University Teaching Hospitals and the UK Biobank Population Study are being pooled, centrally stored and pre-processed. With this large dataset featuring eyes with AMD at various stages and healthy controls, we aim to identify imaging biomarkers for disease progression for intermediate AMD via supervised and unsupervised ML. The prospective study part will firstly characterise the progression of intermediate AMD in patients followed between one and three years; secondly, it will validate the utility of biomarkers identified in the retrospective cohort as predictors of progression towards late AMD. Patients aged 55-90 years old with intermediate AMD in at least one eye will be recruited across multiple sites in UK, Austria and Switzerland for visual function tests, multimodal retinal imaging and genotyping. Imaging will be repeated every four months to identify early focal signs of deterioration on spectral-domain optical coherence tomography (OCT) by human graders. A focal event triggers more frequent follow-up with visual function and imaging tests. The primary outcome is the sensitivity and specificity of the OCT imaging biomarkers. Secondary outcomes include sensitivity and specificity of novel multimodal imaging characteristics at predicting disease progression, ROC curves, time from development of imaging change to development of these endpoints, structure-function correlations, structure-genotype correlation and predictive risk models.

Conclusions: this is one of the first studies in intermediate AMD to combine both ML, retrospective and prospective AMD patient data with the goal of identifying biomarkers of progression and to report the natural history of progression of intermediate AMD with multimodal retinal imaging.
0950-222X
1275-1283
Sutton, Janice
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Menten, Martin J.
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Riedl, Sophie
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Bogunović, Hrovje
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Leingang, Oliver
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Anders, Philipp
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Hagag, Ahmed M.
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Waldstein, Sebastian
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Wilson, Amber
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Cree, Angela J.
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Traber, Ghislaine
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Fritsche, Lars G.
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Scholl, Hendrik
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Rueckert, Daniel
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Schmidt-Erfurth, Ursula
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Sivaprasad, Sobha
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Prevost, Toby
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Lotery, Andrew
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Sutton, Janice
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Menten, Martin J.
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Riedl, Sophie
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Bogunović, Hrovje
cd58759c-5291-4550-ad04-158bc2dbd992
Leingang, Oliver
523e1226-ff2d-48bb-b77a-fcc1f7cb3fc7
Anders, Philipp
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Hagag, Ahmed M.
25260e22-2a8b-423b-a8ec-8304e2a83fc7
Waldstein, Sebastian
7dc8b750-c495-459e-8ce2-2bc48f3ed125
Wilson, Amber
ce8d28bb-3f9a-48f5-aeed-733fecce70c3
Cree, Angela J.
6724b71b-8828-4abb-971f-0856c2af555e
Traber, Ghislaine
d045af04-898f-4251-b5b3-4e191a8e55aa
Fritsche, Lars G.
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Scholl, Hendrik
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Rueckert, Daniel
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Schmidt-Erfurth, Ursula
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Sivaprasad, Sobha
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Prevost, Toby
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Lotery, Andrew
5ecc2d2d-d0b4-468f-ad2c-df7156f8e514

Sutton, Janice, Menten, Martin J., Riedl, Sophie, Bogunović, Hrovje, Leingang, Oliver, Anders, Philipp, Hagag, Ahmed M., Waldstein, Sebastian, Wilson, Amber, Cree, Angela J., Traber, Ghislaine, Fritsche, Lars G., Scholl, Hendrik, Rueckert, Daniel, Schmidt-Erfurth, Ursula, Sivaprasad, Sobha, Prevost, Toby and Lotery, Andrew (2022) Developing and validating a multivariable prediction model which predicts progression of intermediate to late age-related macular degeneration-the PINNACLE trial protocol. Eye, 37, 1275-1283, [1750]. (doi:10.1038/s41433-022-02097-0).

Record type: Article

Abstract

Aims: age-related macular degeneration (AMD) is characterised by a progressive loss of central vision. Intermediate AMD is a risk factor for progression to advanced stages categorised as geographic atrophy (GA) and neovascular AMD. However, rates of progression to advanced stages vary between individuals. Recent advances in imaging and computing technologies have enabled deep phenotyping of intermediate AMD. The aim of this project is to utilise machine learning (ML) and advanced statistical modelling as an innovative approach to discover novel features and accurately quantify markers of pathological retinal ageing that can individualise progression to advanced AMD.

Methods: the PINNACLE study consists of both retrospective and prospective parts. In the retrospective part, more than 400,000 optical coherent tomography (OCT) images collected from four University Teaching Hospitals and the UK Biobank Population Study are being pooled, centrally stored and pre-processed. With this large dataset featuring eyes with AMD at various stages and healthy controls, we aim to identify imaging biomarkers for disease progression for intermediate AMD via supervised and unsupervised ML. The prospective study part will firstly characterise the progression of intermediate AMD in patients followed between one and three years; secondly, it will validate the utility of biomarkers identified in the retrospective cohort as predictors of progression towards late AMD. Patients aged 55-90 years old with intermediate AMD in at least one eye will be recruited across multiple sites in UK, Austria and Switzerland for visual function tests, multimodal retinal imaging and genotyping. Imaging will be repeated every four months to identify early focal signs of deterioration on spectral-domain optical coherence tomography (OCT) by human graders. A focal event triggers more frequent follow-up with visual function and imaging tests. The primary outcome is the sensitivity and specificity of the OCT imaging biomarkers. Secondary outcomes include sensitivity and specificity of novel multimodal imaging characteristics at predicting disease progression, ROC curves, time from development of imaging change to development of these endpoints, structure-function correlations, structure-genotype correlation and predictive risk models.

Conclusions: this is one of the first studies in intermediate AMD to combine both ML, retrospective and prospective AMD patient data with the goal of identifying biomarkers of progression and to report the natural history of progression of intermediate AMD with multimodal retinal imaging.

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Accepted/In Press date: 27 April 2022
e-pub ahead of print date: 25 May 2022
Additional Information: A correction has been attached to this output located at https://www.nature.com/articles/s41433-022-02131-1 and https://doi.org/10.1038/s41433-022-02131-1

Identifiers

Local EPrints ID: 484662
URI: http://eprints.soton.ac.uk/id/eprint/484662
ISSN: 0950-222X
PURE UUID: 25b89df4-240e-4e51-bbc4-a69ab2014dc7
ORCID for Angela J. Cree: ORCID iD orcid.org/0000-0002-1987-8900
ORCID for Andrew Lotery: ORCID iD orcid.org/0000-0001-5541-4305

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Date deposited: 20 Nov 2023 17:35
Last modified: 18 Mar 2024 03:02

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Contributors

Author: Janice Sutton
Author: Martin J. Menten
Author: Sophie Riedl
Author: Hrovje Bogunović
Author: Oliver Leingang
Author: Philipp Anders
Author: Ahmed M. Hagag
Author: Sebastian Waldstein
Author: Amber Wilson
Author: Angela J. Cree ORCID iD
Author: Ghislaine Traber
Author: Lars G. Fritsche
Author: Hendrik Scholl
Author: Daniel Rueckert
Author: Ursula Schmidt-Erfurth
Author: Sobha Sivaprasad
Author: Toby Prevost
Author: Andrew Lotery ORCID iD

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