Objectives: Amblyopia, strabismus and ocular motility disorders are common conditions with significant impact on visual function, appearance and quality of life. We aimed to establish a core set of outcomes for each of the three conditions for use in clinical trials and routine clinical practice.
Design: A comprehensive databank of outcomes was developed from a systematic review of the literature and a series of focus groups with healthcare professionals, researchers, patients and carers. The databank of outcomes was scored in a two-round Delphi Survey completed by two stakeholder groups: healthcare professionals/researchers and patients/carers. Results of the online Delphi were discussed at a face-to-face consensus meeting where the core outcome sets were finalised.
Setting: UK-wide consultation.
Participants: Researchers, clinicians, patients and carers.
Outcome measures: Core outcome sets.
Results: For amblyopia, strabismus and ocular motility, 40/42/33 participants contributed to both rounds of the Delphi; six/nine/seven members attended consensus meetings, respectively. Consensus was reached on ten core outcomes for both amblyopia and ocular motility and nine for strabismus. All three conditions shared the core outcomes: adverse events, cost, vision-related quality of life and ocular alignment. The strabismus and ocular motility disorder core sets included, in addition, measuring the deviation, binocular vision, ocular movement, patient satisfaction and symptoms. The amblyopia set, distinct from the sets for the other two conditions, included best corrected distance and near visual acuity, spherical and cylindrical refraction, compliance and treatment-related and functionality/long-term impacts.
Conclusions: The study used robust consensus methods to develop a core outcome set for three ophthalmic conditions. Implementation of these core outcome sets in clinical trials and routine clinical practice will ensure that the outcomes being measured and reported are relevant to all stakeholders. This will enhance the relevance of study findings and enable comparison of results from different studies.
Samiya Al-Jabri, Fiona J Rowe, Jamie J Kirkham
Disease Category: Eyes & vision
Disease Name: Strabismus, Amblyopia, Ocular motility disorders
Age Range: 18 - 100
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Consumers (caregivers)
- Consumers (patients)
- Methodologists
- Researchers
- Service providers
- Statisticians
- COS for clinical trials or clinical research
- COS for practice
- Consensus meeting
- Delphi process
- Focus group(s)
- Literature review
- Systematic review
Research question: What are the outcomes reported in the published literature assessing the effectiveness of treatments for amblyopia, strabismus and ocular motility disorders.
Methods: We will conduct an overview of reviews inclusive of narrative and systematic reviews identified from a database search (medline, SCOPUS, Central, EMBASE, PSCHY, etc) and Cochrane systematic reviews (Eyes and Vision Cochrane group currently contains 17 full reviews and 6 protocols of relevance).
Inclusion criteria: Reviews of the literature that include intervention studies and/or diagnostic test accuracy studies for conditons of amblyopia, strabismus and ocular motility disorders. We will include any intervention that aims to improve the conditions of amblyopia, strabismus and ocular motility disorders or alleviate their associated visual symptoms. Interventions may include occlusion, optical penalisation, behavioural vision training, extra oclar muscle surgery, extra ocular muscle injection of botulinum toxin, pharmacology therapy for nystagmus. The review will be reported in accordance with PRISMA guidelines.
Methods: We will engage with patients and carers to determine if any additional items can be identified to supplement those determined from the systematic review. We will conduct semi-structured focus groups of thirty patients and carers (ten amblyopia, ten strabismus and ten ocular motility disorders) using a nominal group technique to gather information from the participants in a facilitated forum. This sample size is considered an appropriate number for qualitative research using focus group design (NIHR James Lind Alliance). Patients and carers will be identified from patient and public involvement groups linked with the participating University and NHS Trusts in addition to national groups linked with professional organisations such as the British and Irish Orthoptic Society (for which the lead applicant is the national Director of Research).
Methods: A survey of key stakeholder opinions using Delphi methodology will be conducted for all three conditions collectively. The list of potential outcomes finalised from Sections 1 and 2 will be formatted into ‘outcome items’ with a response designed to allow the participants to rate each of the outcomes value for potential inclusions into the final core outcome set, with higher scores indicating the importance of inclusion. An online questionnaire will be developed for the Delphi process by the IS Developer using the outcome measures identified from work sections 1 and 2. This will be piloted for each of the key stakeholder groups (patients, carers, clinicians, NHS managers and researchers). Clinicians, researchers and NHS managers will be identified through existent national and international professional bodies and networks. Patients and carers will be identified through patient and public involvement groups linked with the participating University and NHS Trusts in addition to national groups linked with professional organisations. We will aim for 30 participants across each group. Outcome items will be reworded into plain language suitable for each stakeholder groups, with help sought from our PPI collaborator for language suitable for patients. The design of the questionnaires for each group will also be carefully considered with inputs from the advisory group. It is anticipated that the Delphi process will consist of three rounds.