4.2 Review

Structural Endpoints and Outcome Measures in Uveitis

Journal

OPHTHALMOLOGICA
Volume 244, Issue 5, Pages 465-479

Publisher

KARGER
DOI: 10.1159/000517521

Keywords

Endpoint; Outcome; Outcome measure; Biomarker; Imaging biomarker; Instrument-based measure; Uveitis; Inflammatory eye diseases

Categories

Funding

  1. BONFOR GEROK Program, Faculty of Medicine, University of Bonn [O-137.0028]
  2. Else Kroener-Fresenius Foundation [EKFS/GSO 16]

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Uveitis is a rare disease responsible for 5-10% of worldwide visual impairment, predominantly affecting working-age individuals. There is a lack of high-level evidence in clinical management, necessitating thorough testing and consensus on available structural outcome measures.
Most uveitis entities are rare diseases but, taken together, are responsible for 5-10% of worldwide visual impairment which largely affects persons of working age. As with many rare diseases, there is a lack of high-level evidence regarding its clinical management, partly due to a dearth of reliable and objective quantitative endpoints for clinical trials. This review provides an overview of available structural outcome measures for uveitis disease activity and damage in an anatomical order from the anterior to the posterior segment of the eye. While there is a multitude of available structural outcome measures, not all might qualify as endpoints for clinical uveitis trials, and thorough testing of applicability is warranted. Furthermore, a consensus on endpoint definition, standardization, and core outcomes is required. As stipulated by regulatory agencies, endpoints should be precisely defined, clinically important, internally consistent, reliable, responsive to treatment, and relevant for the respective subtype of uveitis. Out of all modalities used for assessment of the reviewed structural outcome measures, optical coherence tomography, color fundus photography, fundus autofluorescence, and fluorescein/indocyanine green angiography represent current core modalities for reliable and objective quantification of uveitis outcome measures, based on their practical availability and the evidence provided so far.

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