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

Study Protocol

Irene Stratton¹  Robert E. MacLaren²  Amandeep S. Josan³  Laura J. Taylor³  Jasleen K. Jolly⁴ 

[1] Centre for Statistics in Medicine, University of Oxford, Oxford, UK;Nuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK;Nuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK;Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, UK;Vision and Eye Research Institute, Anglia Ruskin University, Cambridge, UK;

关键词: Retinitis pigmentosa;    Inherited retinal disease;    Rod-cone degeneration;    Outcome measure;    Endpoint;    Microperimetry;    Scotopic microperimetry;    Visual acuity;    Low luminance visual acuity;    Moorfields acuity chart;    Patient reported outcome measures.;   

DOI  :  10.1186/s12886-023-02974-6

 received in 2023-03-02, accepted in 2023-05-11,  发布年份 2023

来源: Springer

【 摘 要 】

BackgroundTreatment 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.MethodsThis 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.DiscussionThe 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 registrationISRCTN registry, ISRCTN24016133, Visual Function in Retinal Degeneration, registered on 18th August 2022.

【 授权许可】

CC BY   
© The Author(s) 2023

【 图 表 】

Fig. 35

Fig. 1

Fig. 26

40517_2023_256_Article_IEq134.gif