Identifying patient-valued outcomes for use in early phase trials of ocular surface disease interventions.

Dry eye disease and public involvement Early phase clinical trials Group concept mapping Ocular surface disease Patient reported outcomes

Journal

The ocular surface
ISSN: 1937-5913
Titre abrégé: Ocul Surf
Pays: United States
ID NLM: 101156063

Informations de publication

Date de publication:
07 2023
Historique:
received: 03 07 2023
accepted: 10 07 2023
medline: 4 9 2023
pubmed: 20 7 2023
entrez: 19 7 2023
Statut: ppublish

Résumé

Patient-reported outcomes (PROs) can be used to evaluate the impact of dry eye symptoms (DES) on daily life. Early-phase clinical trials provide an opportunity to evaluate PRO strategies. Existing measures identified through systematic review omitted important concepts that mattered to patients. The aim of our work was to develop a conceptual map of DES and assess the relative importance of identified concepts. Web-based group concept mapping software was used to develop a conceptual map. This semi-quantitative mixed-methods approach consists of three stages 1) statement generation, 2) thematic sorting, 3) rating of statements for importance [1 (not important), 2 (important), 3 (very important)] and relevance [1 (not my experience), 2 (sometimes my experience); 3 (definitely my experience)]. Thirty-nine participants were recruited from two UK-based patient support groups (British Sjögren's Syndrome Association, PemFriends). Three withdrew, two for health reasons and one struggled with the web-based format. 125 statements and six thematic clusters were generated. The Environmental Impacts cluster scored highest for importance (2.45), followed by Pain and Discomfort (2.35), Eye Treatments (2.32), Daily Impact (2.07), Psychosocial Issues (1.78) and Miscellaneous (1.78). Mapping statements against existing PRO measures confirmed a number of important missing issues including the impact of 'UV levels' (2.50), hot dry weather (2.33), the temporal aspects of pain (2.64), and issues with night-driving (2.59). Group concept mapping identified important issues for people living with DES not currently captured by existing PROs, highlighting the need for additional PRO items to be considered for use in clinical trials.

Sections du résumé

BACKGROUND
Patient-reported outcomes (PROs) can be used to evaluate the impact of dry eye symptoms (DES) on daily life. Early-phase clinical trials provide an opportunity to evaluate PRO strategies. Existing measures identified through systematic review omitted important concepts that mattered to patients. The aim of our work was to develop a conceptual map of DES and assess the relative importance of identified concepts.
METHODS
Web-based group concept mapping software was used to develop a conceptual map. This semi-quantitative mixed-methods approach consists of three stages 1) statement generation, 2) thematic sorting, 3) rating of statements for importance [1 (not important), 2 (important), 3 (very important)] and relevance [1 (not my experience), 2 (sometimes my experience); 3 (definitely my experience)]. Thirty-nine participants were recruited from two UK-based patient support groups (British Sjögren's Syndrome Association, PemFriends). Three withdrew, two for health reasons and one struggled with the web-based format.
RESULTS
125 statements and six thematic clusters were generated. The Environmental Impacts cluster scored highest for importance (2.45), followed by Pain and Discomfort (2.35), Eye Treatments (2.32), Daily Impact (2.07), Psychosocial Issues (1.78) and Miscellaneous (1.78). Mapping statements against existing PRO measures confirmed a number of important missing issues including the impact of 'UV levels' (2.50), hot dry weather (2.33), the temporal aspects of pain (2.64), and issues with night-driving (2.59).
CONCLUSIONS
Group concept mapping identified important issues for people living with DES not currently captured by existing PROs, highlighting the need for additional PRO items to be considered for use in clinical trials.

Identifiants

pubmed: 37467894
pii: S1542-0124(23)00091-5
doi: 10.1016/j.jtos.2023.07.005
pii:
doi:

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

550-556

Subventions

Organisme : Department of Health
Pays : United Kingdom
Organisme : Medical Research Council
Pays : United Kingdom

Informations de copyright

Copyright © 2023 The Authors. Published by Elsevier Inc. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of competing interest O.L.A. declares personal fees from Gilead Sciences Ltd., GlaxoSmithKline (GSK) and Merck outside the submitted work. MC has received personal fees from Astellas, Aparito Ltd, CIS Oncology, Daiichi Sankyo, Glaukos, GSK, Halfloop, Merck, Takeda, and the Patient-Centered Outcomes Research Institute (PCORI) outside the submitted work.

Auteurs

Anita L Slade (AL)

Centre for Patient Reported Outcomes Research, Institute of Applied Health Research, University of Birmingham, Edgbaston, Birmingham, United Kingdom; National Institute for Health and Care Research (NIHR) Applied Research Collaboration West Midlands, Birmingham, United Kingdom; NIHR Birmingham Biomedical Research Centre, University of Birmingham, United Kingdom; NIHR Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, United Kingdom.

Alberto Recchioni (A)

Academic Unit of Ophthalmology, Institute of Inflammation and Ageing, University of Birmingham, United Kingdom; Birmingham & Midland Eye Centre, Sandwell and West Birmingham NHS Trust, Birmingham, United Kingdom.

Olalekan L Aiyegbusi (OL)

Centre for Patient Reported Outcomes Research, Institute of Applied Health Research, University of Birmingham, Edgbaston, Birmingham, United Kingdom; National Institute for Health and Care Research (NIHR) Applied Research Collaboration West Midlands, Birmingham, United Kingdom; NIHR Birmingham Biomedical Research Centre, University of Birmingham, United Kingdom; NIHR Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, United Kingdom; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, United Kingdom; NIHR Blood and Transplant Research Unit (BTRU) in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, United Kingdom.

Ameeta Retzer (A)

Centre for Patient Reported Outcomes Research, Institute of Applied Health Research, University of Birmingham, Edgbaston, Birmingham, United Kingdom; National Institute for Health and Care Research (NIHR) Applied Research Collaboration West Midlands, Birmingham, United Kingdom; NIHR Birmingham Biomedical Research Centre, University of Birmingham, United Kingdom.

Laura Nice (L)

Centre for Patient Reported Outcomes Research, Institute of Applied Health Research, University of Birmingham, Edgbaston, Birmingham, United Kingdom; NIHR Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, United Kingdom; Institute of Translational Medicine, Queen Elizabeth Hospital, University Hospital Birmingham NHS Foundation Trust, Birmingham, United Kingdom.

Elizabeth Dancey (E)

Academic Unit of Ophthalmology, Institute of Inflammation and Ageing, University of Birmingham, United Kingdom.

Melanie J Calvert (MJ)

Centre for Patient Reported Outcomes Research, Institute of Applied Health Research, University of Birmingham, Edgbaston, Birmingham, United Kingdom; National Institute for Health and Care Research (NIHR) Applied Research Collaboration West Midlands, Birmingham, United Kingdom; NIHR Birmingham Biomedical Research Centre, University of Birmingham, United Kingdom; NIHR Surgical Reconstruction and Microbiology Research Centre, University of Birmingham, United Kingdom; Birmingham Health Partners Centre for Regulatory Science and Innovation, University of Birmingham, United Kingdom; NIHR Blood and Transplant Research Unit (BTRU) in Precision Transplant and Cellular Therapeutics, University of Birmingham, Birmingham, United Kingdom.

Saaeha Rauz (S)

Academic Unit of Ophthalmology, Institute of Inflammation and Ageing, University of Birmingham, United Kingdom; Birmingham & Midland Eye Centre, Sandwell and West Birmingham NHS Trust, Birmingham, United Kingdom. Electronic address: s.rauz@bham.ac.uk.

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