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Core outcomes for geographic atrophy trials
  1. Aniela Krystyna Krezel1,2,
  2. Ruth Hogg1,2,
  3. Lynne Lohfeld2,3,
  4. Usha Chakravarthy1,2,
  5. Augusto Azuara-Blanco1,2
  1. 1Centre for Experimental Medicine, Queen’s University Belfast, Belfast, UK
  2. 2Centre for Public Health, Queen’s University Belfast, Belfast, UK
  3. 3The Eye Hospital, School of Ophthalmology and Optometry, Wenzhou Medical University, Wenzhou, China
  1. Correspondence to Professor Augusto Azuara-Blanco, Centre for Public Health, Queen’s University Belfast, Belfast BT12 6BA, UK; a.azuara-blanco{at}qub.ac.uk

Abstract

Background/Aims Ongoing and recent clinical trials for geographic atrophy (GA) have used different outcomes. The goal of this study was to identify a core outcome set (COS) important for patients, clinicians and researchers, and to propose the use of COS in the design of future GA trials.

Methods Five-component project including: Delphi method with patients and experts, focus groups and interviews with patients, relatives and workers supporting patients. Three hundred and one patients (301) with age-related macular degeneration participated in round 1 of a Delphi exercise. Most subjects had GA; 183 patients (61%) were females and the median (range) age was 77 (50–99) years. In round 2, of the 301 of the first round, 100 participants were randomly selected of whom 76 agreed to take part. In a parallel Delphi exercise, panellists comprised a mix of non-clinical scientists and clinicians (43 in the initial and 21 in the final round). In addition, interviews and focus groups consisting of patients (n=20), family members (n=4) and support workers (n=5) were undertaken.

Results Core outcomes identified as important for age-related macular degeneration trials were the health of the outer retina, multimodal estimation of lesion size, reading speed, best corrected distance and near acuity, low luminance visual acuity, patient reported visual performance and safety.

Conclusion This study identified a set of core outcomes that should be used in GA trials. The COS include patient-reported outcome measures, near visual acuity, reading speed and assessment of the outer retina.

  • clinical trial
  • macula
  • retina
  • treatment medical
  • treatment other
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Footnotes

  • Twitter Ruth Hogg @ruth_hogg

  • Contributors AKK and RH: conception, acquisition, analysis, interpretation of data, drafting the work, final approval. LL, UC and AA-B: conception, analysis, interpretation of data, drafting the work, final approval. All authors have agreed to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.

  • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

  • Competing interests None declared.

  • Patient consent for publication Not required.

  • Ethics approval The School of Medicine Research Ethics Committee at Queen’s University and appropriate Research Ethics Committees: Cambridge Central, London-Westminster and West Midlands/Coventry-Warwick approved this study.

  • Provenance and peer review Not commissioned; externally peer reviewed.

  • Data availability statement Data are available upon request.

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