Validating candidate endpoints for intermediate age-related macular degeneration trials in a multi-centre setting—lessons from the MACUSTAR study
Eye, vol. 39, pp. 1031–1039
Abstract
For the conduct of future interventional age-related macular degeneration (AMD) trials, the availability of clinical study endpoints is key. However, no endpoints have been accepted by regulators for evaluation of treatment for intermediate (i) AMD, i.e. the AMD stage at highest risk of developing irreversible geographic atrophy or macular neovascularization. The European MACUSTAR consortium has recruited more than 700 individuals to develop and validate structural, functional and patient-reported endpoints, enabling future iAMD trials based on a prospective observational, multi-centre cohort study. Reliably assessing candidate endpoints in a setting that involves multiple clinical sites across countries comes with a plurality of challenges in the study set-up, quality of data, recruitment of participants and study conduct. Therefore, the MACUSTAR consortium has established a framework that successfully addresses these topics, provides relevant insights into the natural history of iAMD and its sub-phenotypes, and will open new regulatory pathways. The MACUSTAR study is registered on ClinicalTrials.gov under NCT03349801.
Authors 79
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Affiliation as printed
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
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Moorfields Eye Hospital NHS Foundation Trust · University College London
Affiliation as printed
Moorfields Eye Hospital NHS Foundation Trust, London, UK
UCL Institute of Ophthalmology, University College London, London, United Kingdom
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University of Utah · University Hospital Bonn
Affiliation as printed
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
GRADE Reading Center, Bonn, Germany
John A. Moran Eye Center, Department of Ophthalmology and Visual Sciences, University of Utah, Salt Lake City, UT, USA
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University of Bonn · Institut für Medizinische Biometrie, Informatik und Epidemiologie
Affiliation as printed
Institute for Medical Biometry, Informatics and Epidemiology, Medical Faculty, University of Bonn, Bonn, Germany
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Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
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Affiliation as printed
Roche Pharmaceutical Research and Early Development, Translational Medicine Ophthalmology, Roche Innovation Center Basel, Basel, Switzerland
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Affiliation as printed
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
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Affiliation as printed
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
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Affiliation as printed
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
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Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
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University of Bonn · Institut für Medizinische Biometrie, Informatik und Epidemiologie
Affiliation as printed
Institute for Medical Biometry, Informatics and Epidemiology, Medical Faculty, University of Bonn, Bonn, Germany
Novartis Institutes for Biomedical Research, Cambridge, MA, USA
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Affiliation as printed
Novartis Institutes for Biomedical Research, Cambridge, MA, USA
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Moorfields Eye Hospital NHS Foundation Trust · University College London
Affiliation as printed
Novartis Institutes for Biomedical Research, Cambridge, MA, USA
Moorfields Eye Hospital NHS Foundation Trust, London, UK
UCL Institute of Ophthalmology, University College London, London, United Kingdom
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Moorfields Eye Hospital NHS Foundation Trust · City, University of London · University College London
Affiliation as printed
Moorfields Eye Hospital NHS Foundation Trust, London, UK
UCL Institute of Ophthalmology, University College London, London, United Kingdom
Division of Optometry and Visual Science, School of Health Sciences, City, University of London, London, UK
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Affiliation as printed
Division of Optometry and Visual Science, School of Health Sciences, City, University of London, London, UK
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City, University of London · Bayer (Switzerland)
Affiliation as printed
Division of Optometry and Visual Science, School of Health Sciences, City, University of London, London, UK
Bayer Consumer Care AG, Basel, Switzerland
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University Hospital Bonn · Bayer (Switzerland)
Affiliation as printed
Bayer Consumer Care AG, Basel, Switzerland
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
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Heidelberg University · University Hospital Bonn · University Medical Centre Mannheim · Medizinische Fakultät Mannheim
Affiliation as printed
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
Department of Ophthalmology, University Hospital Mannheim & Medical Faculty Mannheim, University of Heidelberg, Mannheim, Germany
GRADE Reading Center, Bonn, Germany
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Frank Gerhard Holz corresponding
University of Bonn · University of Freiburg · University Hospital Bonn · University Medical Center Freiburg
Affiliation as printed
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany. frank.holz@ukbonn.de
GRADE Reading Center, Bonn, Germany. frank.holz@ukbonn.de
Department of Ophthalmology, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg im Breisgau, Germany
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University of Freiburg · University Medical Center Freiburg · University Hospital Cologne
Affiliation as printed
Department of Ophthalmology, Medical Center, Faculty of Medicine, University of Freiburg, Freiburg im Breisgau, Germany
Department of Ophthalmology, University Hospital of Cologne, Cologne, Germany
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Vita-Salute San Raffaele University · IRCCS Ospedale San Raffaele · University Hospital Cologne
Affiliation as printed
Department of Ophthalmology, University Hospital of Cologne, Cologne, Germany
Scientific Institute San Raffaele, University Vita Salute, Milan, Italy
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Centre hospitalier national d'ophtalmologie des Quinze-Vingts · Sorbonne Université · Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Centre hospitalier national d’ophtalmologie des Quinze-Vingts, Sorbonne University, Paris, France
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
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Vita-Salute San Raffaele University · IRCCS Ospedale San Raffaele · European Clinical Research Infrastructure Network
Affiliation as printed
Scientific Institute San Raffaele, University Vita Salute, Milan, Italy
European Clinical Research Infrastructure Network, Paris, France
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Copenhagen University Hospital · Rigshospitalet · European Clinical Research Infrastructure Network
Affiliation as printed
European Clinical Research Infrastructure Network, Paris, France
Department of Ophthalmology, Rigshospitalet, Copenhagen University Hospital, Glostrup, Denmark
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City, University of London · Copenhagen University Hospital · Rigshospitalet
Affiliation as printed
Department of Ophthalmology, Rigshospitalet, Copenhagen University Hospital, Glostrup, Denmark
Division of Optometry and Visual Science, School of Health Sciences, City, University of London, London, UK
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University Hospital Bonn · Leiden University Medical Center · Institut für Medizinische Biometrie, Informatik und Epidemiologie
Affiliation as printed
Institute for Medical Biometry, Epidemiology and Informatics, University Hospital Bonn, Bonn, Germany
Department of Ophthalmology, Leiden University Medical Center, Leiden, The Netherlands
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City, University of London · Bayer (Germany)
Affiliation as printed
Division of Optometry and Visual Science, School of Health Sciences, City, University of London, London, UK
Bayer, Clinical Sciences Experimental Medicine, Wuppertal, Germany
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Leiden University Medical Center · University of Sheffield
Affiliation as printed
Department of Ophthalmology, Leiden University Medical Center, Leiden, The Netherlands
School of Health and Related Research, University of Sheffield, Sheffield, UK
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Institut de la Vision · Bayer (Germany)
Affiliation as printed
Bayer, Clinical Sciences Experimental Medicine, Wuppertal, Germany
Institut de la Vision, Paris, France
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Affiliation as printed
School of Health and Related Research, University of Sheffield, Sheffield, UK
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Institut de la Vision · Hospital de São João
Affiliation as printed
Institut de la Vision, Paris, France
Department of Ophthalmology, Centro Hospitalar Universitário de São João, Porto, Portugal
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Novartis (Switzerland) · University of Sheffield
Affiliation as printed
School of Health and Related Research, University of Sheffield, Sheffield, UK
Novartis Pharma AG, Basel, Switzerland
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Hospital de São João · Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Department of Ophthalmology, Centro Hospitalar Universitário de São João, Porto, Portugal
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
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Novartis (Switzerland) · Carl Zeiss (Germany)
Affiliation as printed
Novartis Pharma AG, Basel, Switzerland
ZEISS Medical Technology, Carl Zeiss AG, Oberkochen, Germany
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University of Milan · Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
Department of Biomedical and Clinical Sciences, Universita degli Studi di Milano, Milan, Italy
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Carl Zeiss (Germany) · City, University of London
Affiliation as printed
ZEISS Medical Technology, Carl Zeiss AG, Oberkochen, Germany
Division of Optometry and Visual Science, School of Health Sciences, City, University of London, London, UK
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University of Milan · Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Department of Biomedical and Clinical Sciences, Universita degli Studi di Milano, Milan, Italy
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
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Association for Innovation and Biomedical Research on Light and Image · University College London
Affiliation as printed
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
UCL Institute of Ophthalmology, University College London, London, United Kingdom
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University Hospital Bonn · University Hospital Cologne
Affiliation as printed
Department of Ophthalmology, University Clinic Köln, Köln, Germany
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
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Cheltenham General Hospital · Gloucestershire Hospitals NHS Foundation Trust · Radboud University Medical Center
Affiliation as printed
Department of Ophthalmology, Cheltenham General Hospital, Gloucestershire Hospitals NHS Foundation Trust, Cheltenham, UK
Department of Ophthalmology, Radboud University Medical Center, Nijmegen, The Netherlands
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University of London · City, University of London · St. Franziskus Hospital
Affiliation as printed
Augenzentrum am St. Franziskus-Hospital, Münster, Germany
Optometry and Visual Sciences, School of Health Sciences, City, University of London, London, UK
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University Hospital Bonn · Radboud University Medical Center
Affiliation as printed
Department of Ophthalmology, Radboud University Medical Center, Nijmegen, The Netherlands
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
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University of London · Carl Zeiss (Germany) · City, University of London
Affiliation as printed
Optometry and Visual Sciences, School of Health Sciences, City, University of London, London, UK
ZEISS Medical Technology, Carl Zeiss AG, Oberkochen, Germany
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Radboud University Medical Center · Royal Victoria Hospital
Affiliation as printed
Centre for Experimental Medicine, Royal Victoria Hospital, Belfast, Northern Ireland, UK
Department of Ophthalmology, Radboud University Medical Center, Nijmegen, The Netherlands
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Radboud University Medical Center · Donders Institute for Brain, Cognition and Behaviour · University of Tübingen
Affiliation as printed
Department of Ophthalmology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, The Netherlands
Department of Ophthalmology, Radboud University Medical Center, Nijmegen, The Netherlands
Department of Ophthalmology, University clinic Tübingen, Tübingen, Germany
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Universität Ulm · Copenhagen University Hospital · Rigshospitalet
Affiliation as printed
Department of Ophthalmology, University of Ulm, Ulm, Germany
Department of Ophthalmology, Rigshospitalet, Copenhagen University Hospital, Glostrup, Denmark
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Bayer (Switzerland) · Bayer (Germany)
Affiliation as printed
Bayer AG, Leverkusen, Germany
Bayer Consumer Care AG, Basel, Switzerland
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Radboud University Medical Center · Donders Institute for Brain, Cognition and Behaviour · University of Tübingen
Affiliation as printed
Department of Ophthalmology, University clinic Tübingen, Tübingen, Germany
Department of Ophthalmology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, The Netherlands
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Copenhagen University Hospital · Rigshospitalet
Affiliation as printed
Department of Ophthalmology, Rigshospitalet, Copenhagen University Hospital, Glostrup, Denmark
Ophthalmology Translational Medicine, Novartis Institutes for Biomedical Research, Cambridge, MA, USA
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University Hospital Bonn · Radboud University Medical Center · Donders Institute for Brain, Cognition and Behaviour
Affiliation as printed
Department of Ophthalmology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, The Netherlands
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
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Affiliation as printed
Ophthalmology Translational Medicine, Novartis Institutes for Biomedical Research, Cambridge, MA, USA
ZEISS Medical Technology, Carl Zeiss AG, Oberkochen, Germany
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University Hospital Bonn · Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
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Carl Zeiss (Germany) · University Hospital Bonn
Affiliation as printed
ZEISS Medical Technology, Carl Zeiss AG, Oberkochen, Germany
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
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Novartis (Switzerland) · Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
Novartis Pharma AG, Basel, Switzerland
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University of Southern California · Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
Novartis Pharma, University of Southern California, Los Angeles, CA, USA
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Institut de la Vision · University Hospital Bonn · Sorbonne Université
Affiliation as printed
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
Institut de la Vision, Sorbonne Universités, Paris, France
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University of Southern California · Fondazione G.B. Bietti
Affiliation as printed
Novartis Pharma, University of Southern California, Los Angeles, CA, USA
IRCCS-Fondazione Bietti, Rome, Italy
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Institut de la Vision · Hospital de São João · Sorbonne Université
Affiliation as printed
Institut de la Vision, Sorbonne Universités, Paris, France
Department of Ophthalmology, Centro Hospitalar Universitário de São João, Porto, Portugal
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IRCCS Ospedale San Raffaele · University of Tübingen
Affiliation as printed
Ospedale San Raffaele, Milan, Italy
Department of Ophthalmology, University clinic Tübingen, Tübingen, Germany
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Queen's University Belfast · Fondazione G.B. Bietti
Affiliation as printed
IRCCS-Fondazione Bietti, Rome, Italy
Centre for Public Health, School of Medicine, Dentistry and Biomedical Science, Queen's University Belfast, Northern Ireland, UK
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Hospital de São João · Ludwig-Maximilians-Universität München
Affiliation as printed
Department of Ophthalmology, Centro Hospitalar Universitário de São João, Porto, Portugal
Department of Ophthalmology, Ludwig-Maximilians-University, Munich, Germany
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Novartis (Switzerland) · University of Tübingen
Affiliation as printed
Department of Ophthalmology, University clinic Tübingen, Tübingen, Germany
Novartis Pharma AG, Basel, Switzerland
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Queen's University Belfast · Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Centre for Public Health, School of Medicine, Dentistry and Biomedical Science, Queen’s University Belfast, Northern Ireland, UK
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
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Ludwig-Maximilians-Universität München · University of Sheffield
Affiliation as printed
Department of Ophthalmology, Ludwig-Maximilians-University, Munich, Germany
Sheffield Centre for Health and Related Research, University of Sheffield, Sheffield, England, UK
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Novartis (Switzerland) · University College London
Affiliation as printed
Novartis Pharma AG, Basel, Switzerland
Department of Visual Neuroscience and Function, University College London Institute of Ophthalmology, London, UK
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Radboud University Nijmegen · Radboud University Medical Center · University of Sheffield
Affiliation as printed
Sheffield Centre for Health and Related Research, University of Sheffield, Sheffield, England, UK
Diagnostic Image Analysis Group, Radboud University Medical Center, Nijmegen, The Netherlands
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Novartis (Switzerland) · University College London
Affiliation as printed
Department of Visual Neuroscience and Function, University College London Institute of Ophthalmology, London, UK
Novartis Pharma AG, Basel, Switzerland
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Institut de la Vision · Inserm · University Hospital Bonn · Sorbonne Université · Fondation Voir & Entendre
Affiliation as printed
Institut Hospitalo-Universitaire FOReSIGHT, Sorbonne Universite, Inserm, Quinze-Vingts Hopital de la Vision, Paris, France
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
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Radboud University Nijmegen · University Hospital Bonn · Radboud University Medical Center · Institut für Medizinische Biometrie, Informatik und Epidemiologie
Affiliation as printed
Diagnostic Image Analysis Group, Radboud University Medical Center, Nijmegen, The Netherlands
Institute for Medical Biometry, Epidemiology and Informatics, University Hospital Bonn, Bonn, Germany
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Novartis (Switzerland) · University of Utah
Affiliation as printed
Novartis Pharma AG, Basel, Switzerland
John A. Moran Eye Center, Department of Ophthalmology and Visual Sciences, University of Utah, Salt Lake City, UT, USA
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Université Paris-Est Créteil · University of Utah · University Hospital Bonn · Hôpital Intercommunal de Créteil
Affiliation as printed
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
GRADE Reading Center, Bonn, Germany
John A. Moran Eye Center, Department of Ophthalmology and Visual Sciences, University of Utah, Salt Lake City, UT, USA
Department of Ophthalmology, Hospital Intercommunal de Creteil, University Paris Est Creteil, Creteil, France
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Luigi Sacco Hospital · Ludwig-Maximilians-Universität München
Affiliation as printed
Department of Ophthalmology, Ludwig-Maximilians-University, Munich, Germany
University Eye Clinic Luigi Sacco Hospital, Milan, Italy
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Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
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City, University of London · Université Paris-Est Créteil · Hôpital Intercommunal de Créteil
Affiliation as printed
Department of Ophthalmology, Hospital Intercommunal de Creteil, University Paris Est Creteil, Creteil, France
Division of Optometry and Visual Science, School of Health Sciences, City, University of London, London, UK
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University Hospital Bonn · Luigi Sacco Hospital
Affiliation as printed
University Eye Clinic Luigi Sacco Hospital, Milan, Italy
Department of Ophthalmology, University Hospital Bonn, Bonn, Germany
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Moorfields Eye Hospital NHS Foundation Trust · Association for Innovation and Biomedical Research on Light and Image
Affiliation as printed
Association for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
Moorfields Eye Hospital NHS Foundation Trust, London, UK
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City, University of London · University of Basel
Affiliation as printed
Division of Optometry and Visual Science, School of Health Sciences, City, University of London, London, UK
Department of Ophthalmology, University of Basel, Basel, Switzerland
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Affiliation as printed
Department of Ophthalmology, University of Basel, Basel, Switzerland
Ophthalmology Translational Medicine, Novartis Institutes for Biomedical Research, Cambridge, MA, USA
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Affiliation as printed
IRCCS-Fondazione Bietti, Rome, Italy
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