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      • Record: found
      • Abstract: found
      • Article: found
      Is Open Access

      MACUSTAR: Development and Clinical Validation of Functional, Structural, and Patient-Reported Endpoints in Intermediate Age-Related Macular Degeneration

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          Abstract

          Purpose: Currently, no outcome measures are clinically validated and accepted as clinical endpoints by regulatory agencies for drug development in intermediate age-related macular degeneration (iAMD). The MACUSTAR Consortium, a public-private research group funded by the European Innovative Medicines Initiative intends to close this gap. Procedures: Development of study protocol and statistical analysis plan including predictive modelling of multimodal endpoints based on a review of the literature and expert consensus. Results: This observational study consists of a cross-sectional and a longitudinal part. Functional outcome measures assessed under low contrast and low luminance have the potential to detect progression of visual deficit within iAMD and to late AMD. Structural outcome measures will be multimodal and investigate topographical relationships with function. Current patient-reported outcome measures (PROMs) are not acceptable to regulators and may not capture the functional deficit specific to iAMD with needed precision, justifying development of novel PROMs for iAMD. The total sample size will be n = 750, consisting mainly of subjects with iAMD ( n = 600). Conclusions: As clinical endpoints currently accepted by regulators cannot detect functional loss or patient-relevant impact in iAMD, we will clinically validate novel candidate endpoints for iAMD.

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          Estimating classification error rate: Repeated cross-validation, repeated hold-out and bootstrap

          Ji Hyun Kim (2009)
          Article 0 comments Cited 174 times – based on 0 reviews     Review now
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            Geographic atrophy: clinical features and potential therapeutic approaches.

            Frank G. Holz, Erich C Strauss, Steffen Schmitz-Valckenberg … (2014)
            In contrast to wet age-related macular degeneration (AMD), where loss of vision is typically acute and treatment leads to a relatively rapid reduction in retinal fluid and subsequent improvements in visual acuity (VA), disease progression and vision loss in geographic atrophy (GA) owing to AMD are gradual processes. Although GA can result in significant visual function deficits in reading, night vision, and dark adaptation, and produce dense, irreversible scotomas in the visual field, the initial decline in VA may be relatively minor if the fovea is spared. Because best-corrected VA does not correlate well with GA lesions or progression, alternative clinical endpoints are being sought. These include reduction in drusen burden, slowing the enlargement rate of GA lesion area, and slowing or eliminating the progression of intermediate to advanced AMD. Among these considerations, slowing the expansion of the GA lesion area seems to be a clinically suitable primary efficacy endpoint. Because GA lesion growth is characterized by loss of photoreceptors, it is considered a surrogate endpoint for vision loss. Detection of GA can be achieved with a number of different imaging techniques, including color fundus photography, fluorescein angiography, fundus autofluorescence (FAF), near-infrared reflectance, and spectral-domain optical coherence tomography. Previous studies have identified predictive characteristics for progression rates including abnormal patterns of FAF in the perilesional retina. Although there is currently no approved or effective treatment to prevent the onset and progression of GA, potential therapies are being evaluated in clinical studies.
            Article 0 comments Cited 116 times – based on 0 reviews     Review now
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              Prevalence of age-related maculopathy in older Europeans: the European Eye Study (EUREYE).

              Cristina Augood, Johannes Vingerling, Paulus de Jong … (2006)
              To estimate the prevalence of age-related maculopathy in an older population from 7 European countries. Randomly sampled people 65 years and older were invited to an eye examination in centers across 7 European countries (Norway, Estonia, United Kingdom, France, Italy, Greece, and Spain). Fundus images of each eye were graded at a single reading center. Prevalence rates were calculated for stage of age-related maculopathy with 95% confidence intervals (CIs) estimated for clustered data. Of 5040 participants (45% response rate), 4753 (2128 men and 2625 women) had gradable fundus images. The prevalences were grade 0, 47.59% (95% CI, 43.53%-51.65%); grade 1, 36.48% (95% CI, 32.66%-40.30%); grade 2, 10.14% (95% CI, 8.92% to 11.37%); grade 3, 2.46% (95% CI, 1.79%-3.13%); and grade 4 (age-related macular degeneration [AMD]), 3.32% (95% CI, 2.52%-4.13%) and large drusen only (> or = 125 microm), 15.41% (95% CI, 13.61%-17.21%). The prevalence of geographic atrophic AMD was 1.2% (95% CI, 0.8%-1.7%) and of neovascular AMD, 2.3% (95% CI, 1.7%-2.9%). The prevalence of bilateral AMD was 1.4% (95% CI, 1.0%-1.8%). Age-specific prevalences of age-related maculopathy in the European Eye Study (EUREYE) are similar to other population-based studies in Western populations.
              Article 0 comments Cited 100 times – based on 0 reviews     Review now
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                Author and article information

                Journal
                Journal ID (publisher-id): OPH
                Journal ID (nlm-ta): Ophthalmologica
                Journal ID (doi): 10.1159/issn.0030-3755
                Title: Ophthalmologica
                Publisher: S. Karger AG
                ISSN (Print): 0030-3755
                ISSN (Electronic): 1423-0267
                Publication date Subscription-year: 2019
                Publication date (Print): January 2019
                Publication date (Electronic): 28 August 2018
                Volume: 241
                Issue: 2
                Pages: 61-72
                Affiliations
                [_a] aDepartment of Ophthalmology, University of Bonn, Bonn, Germany
                [_b] bInstitute for Medical Biometry, Epidemiology and Informatics, University Hospital Bonn, Bonn, Germany
                [_c] cUCL Institute of Ophthalmology, University College London, London, United Kingdom
                [_d] dMoorfields Eye Hospital, London, United Kingdom
                [_e] eDivision of Optometry and Visual Science, School of Health Sciences, City, University of London, London, United Kingdom
                [_f] fDiagnostic Image Analysis Group, Radboud University Medical Center, Nijmegen, The Netherlands
                [_g] gNovartis Pharma AG, Basel, Switzerland
                [_h] hCarl Zeiss Meditec AG, Dublin, California, USA
                [_i] iRoche Pharmaceutical Research and Early Development, Translational Medicine Ophthalmology, Roche Pharma Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland
                [_j] jAssociation for Innovation and Biomedical Research on Light and Image, Coimbra, Portugal
                [_k] kDevelopment Pharmaceuticals, Therapeutic Areas PAO, Bayer AG, Berlin, Germany
                Author notes
                *Robert P. Finger, Department of Ophthalmology, University of Bonn, Ernst-Abbe-Strasse 2, DE–53127 Bonn (Germany), E-Mail robert.finger@ukbonn.de
                Article
                Publisher ID: 491402 Pmcid ID: PMC6482983 Other ID: Ophthalmologica 2019;241:61–72
                DOI: 10.1159/000491402
                PMC ID: PMC6482983
                PubMed ID: 30153664
                SO-VID: 915fa2cb-1377-4097-9cf5-66430288aa43
                Copyright © © 2018 S. Karger AG, Basel
                License:

                This article is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND). Usage and distribution for commercial purposes as well as any distribution of modified material requires written permission. Drug Dosage: The authors and the publisher have exerted every effort to ensure that drug selection and dosage set forth in this text are in accord with current recommendations and practice at the time of publication. However, in view of ongoing research, changes in government regulations, and the constant flow of information relating to drug therapy and drug reactions, the reader is urged to check the package insert for each drug for any changes in indications and dosage and for added warnings and precautions. This is particularly important when the recommended agent is a new and/or infrequently employed drug. Disclaimer: The statements, opinions and data contained in this publication are solely those of the individual authors and contributors and not of the publishers and the editor(s). The appearance of advertisements or/and product references in the publication is not a warranty, endorsement, or approval of the products or services advertised or of their effectiveness, quality or safety. The publisher and the editor(s) disclaim responsibility for any injury to persons or property resulting from any ideas, methods, instructions or products referred to in the content or advertisements.

                History
                Date received : 26 March 2018
                Date accepted : 21 June 2018
                Page count
                Figures: 1, Tables: 2, Pages: 12
                Categories
                Subject: Research Article

                ScienceOpen disciplines: Vision sciences,Ophthalmology & Optometry,Pathology
                Keywords: Patient-reported outcomes,Structure-function correlation,Intermediate age-related macular degeneration,Clinical endpoint,Disease progression
                Data availability:
                ScienceOpen disciplines: Vision sciences, Ophthalmology & Optometry, Pathology
                Keywords: Patient-reported outcomes, Structure-function correlation, Intermediate age-related macular degeneration, Clinical endpoint, Disease progression

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