A cross-sectional study to assess the clinical utility of modern visual function assessments in patients with inherited retinal disease: a mixed methods observational study protocol.
A cross-sectional study to assess the clinical utility of modern visual function assessments in patients with inherited retinal disease: a mixed methods observational study protocol.
Background: treatment options for patients with inherited retinal disease are limited, although research into novel therapies is underway. To ensure the success of future clinical trials, appropriate visual function outcome measures that can assess changes resulting from therapeutic interventions are urgently required. Rod-cone degenerations are the most common type of inherited retinal disease. Visual acuity is a standard measure but is typically preserved until late disease stages, frequently making it an unsuitable visual function marker. Alternative measures are required. This study investigates the clinical utility of a range of carefully selected visual function tests and patient reported outcome measures. The aim is to identify suitable outcome measures for future clinical trials that could be considered for regulatory approval.
Methods: this cross-sectional study involves two participant groups, patients with inherited retinal disease (n = 40) and healthy controls (n = 40). The study has been designed to be flexible and run alongside NHS clinics. The study is split into two parts. Part one includes examining standard visual acuity, low luminance visual acuity, the Moorfields acuity chart visual acuity, mesopic microperimetry and three separate patient reported outcome measures. Part two involves 20 min of dark adaptation followed by two-colour scotopic microperimetry. Repeat testing will be undertaken where possible to enable repeatability analyses. A subset of patients with inherited retinal disease will be invited to participate in a semi-structured interview to gain awareness of participants’ thoughts and feelings around the study and different study tests.
Discussion: the study highlights a need for reliable and sensitive validated visual function measures that can be used in future clinical trials. This work will build on work from other studies and be used to inform an outcome measure framework for rod-cone degenerations. The study is in keeping with the United Kingdom Department of Health and Social Care research initiatives and strategies for increasing research opportunities for NHS patients as part of their NHS care. Trial registration: ISRCTN registry, ISRCTN24016133, Visual Function in Retinal Degeneration, registered on 18th August 2022.
Endpoint, Inherited retinal disease, Low luminance visual acuity, Microperimetry, Moorfields acuity chart, Outcome measure, Patient reported outcome measures, Retinitis pigmentosa, Rod-cone degeneration, Scotopic microperimetry, Visual acuity
Taylor, Laura J.
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Josan, Amandeep S.
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Stratton, Irene
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Jolly, Jasleen K.
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MacLaren, Robert E.
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24 May 2023
Taylor, Laura J.
986a15a2-eceb-4cf1-bc13-587367cac037
Josan, Amandeep S.
859cf422-b9fc-4280-984a-b2301c0120c7
Stratton, Irene
772f25b9-23c0-4240-a3f6-1e76b03b172f
Jolly, Jasleen K.
3196d568-3730-4b67-8418-9b1ef271aba7
MacLaren, Robert E.
c7a2f458-a7b1-4b96-b88e-d376c90ec059
Taylor, Laura J., Josan, Amandeep S., Stratton, Irene, Jolly, Jasleen K. and MacLaren, Robert E.
(2023)
A cross-sectional study to assess the clinical utility of modern visual function assessments in patients with inherited retinal disease: a mixed methods observational study protocol.
BMC Ophthalmology, 23 (1), [234].
(doi:10.1186/s12886-023-02974-6).
Abstract
Background: treatment options for patients with inherited retinal disease are limited, although research into novel therapies is underway. To ensure the success of future clinical trials, appropriate visual function outcome measures that can assess changes resulting from therapeutic interventions are urgently required. Rod-cone degenerations are the most common type of inherited retinal disease. Visual acuity is a standard measure but is typically preserved until late disease stages, frequently making it an unsuitable visual function marker. Alternative measures are required. This study investigates the clinical utility of a range of carefully selected visual function tests and patient reported outcome measures. The aim is to identify suitable outcome measures for future clinical trials that could be considered for regulatory approval.
Methods: this cross-sectional study involves two participant groups, patients with inherited retinal disease (n = 40) and healthy controls (n = 40). The study has been designed to be flexible and run alongside NHS clinics. The study is split into two parts. Part one includes examining standard visual acuity, low luminance visual acuity, the Moorfields acuity chart visual acuity, mesopic microperimetry and three separate patient reported outcome measures. Part two involves 20 min of dark adaptation followed by two-colour scotopic microperimetry. Repeat testing will be undertaken where possible to enable repeatability analyses. A subset of patients with inherited retinal disease will be invited to participate in a semi-structured interview to gain awareness of participants’ thoughts and feelings around the study and different study tests.
Discussion: the study highlights a need for reliable and sensitive validated visual function measures that can be used in future clinical trials. This work will build on work from other studies and be used to inform an outcome measure framework for rod-cone degenerations. The study is in keeping with the United Kingdom Department of Health and Social Care research initiatives and strategies for increasing research opportunities for NHS patients as part of their NHS care. Trial registration: ISRCTN registry, ISRCTN24016133, Visual Function in Retinal Degeneration, registered on 18th August 2022.
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s12886-023-02974-6
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Published date: 24 May 2023
Additional Information:
Funding Information:
The VFIRD study is a cross-sectional prospective study involving two groups of participants: participants with an inherited retinal disease causing a rod-cone degeneration (n = 40), and age and gender matched healthy controls (n = 40). Healthy controls will be recruited from members of the general public through advertisements or from individuals accompanying patients (must be non-blood relatives) to eye hospital appointments. Patient participants will be recruited from the NHS specialist ocular genetics clinic at Oxford Eye Hospital. This publication refers to protocol version 2.1 dated 22/11/2022 and was developed using the STROBE and SPIRIT reporting guidelines where applicable [, ]. The study is sponsored by the University of Oxford. A patient and public involvement (PPI) group was set up with representatives providing feedback which supported the study design. Ethics approval has been obtained from the NHS Health Research Authority Black Country Research Ethics Committee (reference 20/WM/0283).
Funding Information:
This project is funded by the National Institute for Health and Care Research (NIHR) under its Research for Patient Benefit (RfPB) Programme (Grant Reference Number NIHR202821). The views expressed are those of the author(s) and not necessarily those of the NIHR or the Department of Health and Social Care. Specialist equipment such as the MAIA microperimeter is funded by the Emma Salisbury Fight for Sight charitable funds.
Funding Information:
We would like to acknowledge Jon Brett from the Eye Research Group Oxford for his help in creating the test procedure flow diagram used in Fig. 1. The study is sponsored by the University of Oxford, via the Research Governance, Ethics & Assurance Team (RGEA), they provide support and policy for human involving research and ensure compliance and assurance with internal and external policy, standards and regulatory frameworks. RGEA supported the submission of the study documents to the research ethics committee. Address: Research Governance, Ethics & Assurance (University of Oxford) Joint Research Office, Boundary Brook House, Churchill Drive Oxford, OX3 7GB. Email: RGEA.Sponsor@admin.ox.ac.uk.
Publisher Copyright:
© 2023, The Author(s).
Keywords:
Endpoint, Inherited retinal disease, Low luminance visual acuity, Microperimetry, Moorfields acuity chart, Outcome measure, Patient reported outcome measures, Retinitis pigmentosa, Rod-cone degeneration, Scotopic microperimetry, Visual acuity
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Local EPrints ID: 487235
URI: http://eprints.soton.ac.uk/id/eprint/487235
PURE UUID: 9ffd2d29-88e5-4cbc-a0af-30a61837f059
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Date deposited: 16 Feb 2024 13:38
Last modified: 18 Mar 2024 04:01
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Contributors
Author:
Laura J. Taylor
Author:
Amandeep S. Josan
Author:
Irene Stratton
Author:
Jasleen K. Jolly
Author:
Robert E. MacLaren
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