Dataset Information

Leveraging Visual Outcome Measures to Advance Therapy Development in Neuroimmunologic Disorders.

ABSTRACT: The visual system offers unparalleled precision in the assessment of neuroaxonal damage. With the majority of patients with multiple sclerosis (MS) experiencing afferent and efferent visual dysfunction, outcome measures capturing these deficits provide insight into neuroaxonal injury, even in those with minimal disability. Ideal for use in clinical trials, visual measures are generally inexpensive, accessible, and reproducible. Quantification of visual acuity, visual fields, visual quality of life, and electrophysiologic parameters allows assessment of function, whereas optical coherence tomography (OCT) provides reliable measures of the structural integrity of the anterior afferent visual pathway. The technology of oculomotor biometrics continues to advance, and discrete measures of fixation, smooth pursuit, and saccadic eye movement abnormalities are ready for inclusion in future trials of MS progression. Visual outcomes allow tracking of neuroaxonal injury and aid in distinguishing MS from diseases such as neuromyelitis optica spectrum disorder (NMOSD) or myelin oligodendrocyte glycoprotein antibody-associated diseases (MOGAD). OCT has also provided unique insights into pathophysiology, including the identification of foveal pitting in NMOSD, possibly from damage to Müller cells, which carry an abundance of aquaporin-4 channels. For some study designs, the cost-benefit ratio favors visual outcomes over more expensive MRI outcomes. With the next frontier of therapeutics focused on remyelination and neuroprotection, visual outcomes are likely to take center stage. As an international community of collaborative, committed, vision scientists, this review by the International MS Visual System Consortium (IMSVISUAL) outlines the quality standards, informatics, and framework needed to routinely incorporate vision outcomes into MS and NMOSD trials.

SUBMITTER: Graves JS

PROVIDER: S-EPMC8711076 | biostudies-literature | 2022 Mar

REPOSITORIES: biostudies-literature

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Leveraging Visual Outcome Measures to Advance Therapy Development in Neuroimmunologic Disorders.

Graves Jennifer S JS Oertel Frederike Cosima FC Van der Walt Anneke A Collorone Sara S Sotirchos Elias S ES Pihl-Jensen Gorm G Albrecht Philipp P Yeh E Ann EA Saidha Shiv S Frederiksen Jette J Newsome Scott Douglas SD Paul Friedemann F

Neurology(R) neuroimmunology & neuroinflammation 20211226 2

The visual system offers unparalleled precision in the assessment of neuroaxonal damage. With the majority of patients with multiple sclerosis (MS) experiencing afferent and efferent visual dysfunction, outcome measures capturing these deficits provide insight into neuroaxonal injury, even in those with minimal disability. Ideal for use in clinical trials, visual measures are generally inexpensive, accessible, and reproducible. Quantification of visual acuity, visual fields, visual quality of li ...[more]

PMID: 34955459

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Project description:ObjectivesReporting of research for stroke-related visual impairment is inconsistent. The aim of this study was to define three core outcome sets (COS) and related core outcome measurements (COM) for central visual impairment, visual field loss and ocular motility disorders in stroke research.DesignThe consensus process consisted of an online three-round Delphi survey followed by a consensus meeting of key stakeholders.SettingUK-wide survey.ParticipantsStakeholders included orthoptists, occupational therapists, ophthalmologists, stroke survivors and COS users such as researchers, journal editors and guideline developers.Outcome measuresFor COS development, a list of potentially relevant visual outcomes was created after review of the literature and further grouped into outcome domains. For COM development, a list of potential instruments was created after review of the literature and quality appraised for reliability and validity.ResultsCOS-119 potential outcomes extracted from published literature. Similar assessment outcomes were grouped into 24 outcome domains. Delphi process included 123 participants in round 1, 65 round 2, 51 round 3. Twelve participants attended the consensus meeting with recommended outcome domains for central visual impairment (visual acuity, functional vision, quality of life), visual field loss (visual fields, functional vision, quality of life) and ocular motility disorders (eye alignment, eye movements, functional vision, quality of life). COM-52 test options extracted from the COS outcomes and grouped into 16 domains. Thirteen participants attended the COM consensus meeting. Recommended instruments for measurement of these outcomes include; Logarithm of the Minimal Angle of Resolution visual acuity, cover test, cardinal position eye movement assessments, peripheral visual field perimetry, Visual Function Questionnaire-25.ConclusionsCOS and COM are defined for vision research for stroke survivors. Their use has potential to reduce heterogeneity in routine clinical practice and improve standardisation and accuracy of vision assessment. Future research is required to evaluate the use of these COS and COM.

Project description:BackgroundCore Outcome Sets (COS) are defined as the minimum sets of outcomes that should be measured and reported in all randomised controlled trials to facilitate combination and comparability of research. The aim of this review is to produce an item bank of previously reported outcome measures from published studies in amblyopia, strabismus and ocular motility disorders to initiate the development of COS.MethodsA review was conducted to identify articles reporting outcome measures for amblyopia, strabismus and ocular motility disorders. Using systematic methods according to the COMET handbook we searched key electronic bibliographic databases from 1st January 2011 to 27th September 2016 using MESH terms and alternatives indicating the different subtypes of amblyopia, strabismus and ocular motility disorders in relation to treatment outcomes and all synonyms. We included Cochrane reviews, other systematic reviews, controlled trials, non-systematic reviews and retrospective studies. Data was extracted to tabulate demographics of included studies, primary and secondary outcomes, methods of measurement and their time points.ResultsA total of 142 studies were included; 42 in amblyopia, 33 in strabismus, and 68 in ocular motility disorders (one study overlap between amblyopia and strabismus). We identified ten main outcome measure domains for amblyopia, 14 for strabismus, and ten common "visual or motility" outcome measure domains for ocular motility disorders. Within the domains, we found variable nomenclature being used and diversity in methods and timings of measurements.ConclusionThis review highlights discrepancies in outcome measure reporting within published literature for amblyopia, strabismus and ocular motility and it generated an item bank of the most commonly used and reported outcome measures for each of the three conditions from recent literature to start the process of COS development. Consensus among all stakeholders including patients and professionals is recommended to establish a useful COS.

Project description:BackgroundAmong the available patient-reported outcome measures (PROMs) there is an absence of a PROM with a specific focus on the impact of the wide variety of visual impairments following stroke. Our aim was to develop a patient reported quality of life outcome measure for stroke survivors with visual impairment.MethodsPotential items were sourced from a combination of existing PROMs from a systematic review and qualitative in-depth interviews, duplicates were removed and items shortlisted. The initial pilot instrument was created following a ranking exercise of these potential items and consultation with stroke survivors. Version 1 was piloted with 37 stroke survivors at acute and chronic stages. Version 2 was piloted with 243 stroke survivors with visual impairment at acute and chronic stages. This data was analysed using the Rasch measurement model. Simultaneously, items from Version 2 underwent a Delphi process with stroke survivors and stroke clinicians, to assess the importance of each item. Final consensus decisions on item removal were made using the combined analysis from the Rasch measurement model and Delphi process in a nominal group meeting.ResultsDue to the wide range of rank given to the majority of categories/items, only two items were discarded. Version 1 comprised of 102 items with 5 response categories relating to amount of difficulty. The pilot of Version 1 allowed item reduction based on analysis of floor/ceiling effects and not applicable responses. Version 2 comprised of 62 items. Within the nominal group meeting, the expert panel created a set of rules which aided them with decision making in addition to the Rasch and Delphi analysis data. This resulted in the removal of 43 items and the combination of seven items to create three new items. The expert panel also recommended the rewording of three items.ConclusionThe Brain Injury associated Visual Impairment Impact Questionnaire (BIVI-IQ-15), a 15-item instrument with 4 response categories has been developed for capturing vision-related quality of life of stroke survivors with any of the predominant types of visual impairment, in the presence of other impairments and for both inpatients and outpatients.

Project description:BackgroundBlue Cone Monochromacy (BCM) is an X-linked retinopathy caused by mutations in the OPN1LW / OPN1MW gene cluster, encoding long (L)- and middle (M)-wavelength sensitive cone opsins. Recent evidence shows sufficient structural integrity of cone photoreceptors in BCM to warrant consideration of a gene therapy approach to the disease. In the present study, the vision in BCM is examined, specifically seeking clinically-feasible outcomes for a future clinical trial.MethodsBCM patients (n = 25, ages 5-72) were studied with kinetic and static chromatic perimetry, full-field sensitivity testing, and eye movement recordings. Vision at the fovea and parafovea was probed with chromatic microperimetry.ResultsKinetic fields with a Goldmann size V target were generally full. Short-wavelength (S-) sensitive cone function was normal or near normal in most patients. Light-adapted perimetry results on conventional background lights were abnormally reduced; 600-nm stimuli were seen by rods whereas white stimuli were seen by both rods and S-cones. Under dark-adapted conditions, 500-nm stimuli were seen by rods in both BCM and normals. Spectral sensitivity functions in the superior retina showed retained rod and S-cone functions in BCM under dark-adapted and light-adapted conditions. In the fovea, normal subjects showed L/M-cone mediation using a 650-nm stimulus under dark-adapted conditions, whereas BCM patients had reduced sensitivity driven by rod vision. Full-field red stimuli on bright blue backgrounds were seen by L/M-cones in normal subjects whereas BCM patients had abnormally reduced and rod-mediated sensitivities. Fixation location could vary from fovea to parafovea. Chromatic microperimetry demonstrated a large loss of sensitivity to red stimuli presented on a cyan adapting background at the anatomical fovea and surrounding parafovea.ConclusionsBCM rods continue to signal vision under conditions normally associated with daylight vision. Localized and retina-wide outcome measures were examined to evaluate possible improvement of L/M-cone-based vision in a clinical trial.

Project description:BackgroundAdvance care planning (ACP) is a process that involves discussing a person's goals, values, and preferences; it is particularly important for persons living with dementia (PLWD) given that dementia is incurable and progressive. To ensure results that will impact real-world practices, ACP outcome measures must be psychometrically strong, meaningful to key partners, and pragmatic to collect. Therefore, we conducted a scoping review of outcome measures utilized in ACP randomized controlled clinical trials (RCTs) enrolling PLWD or their care partners and evaluated their pragmatic characteristics.MethodsWe searched MEDLINE/PubMed, EMBASE, CINAHL, PsycINFO, and Web of Science for peer-reviewed ACP RCTs enrolling PLWD or their care partners from 2011 to 2021. We abstracted characteristics of primary and secondary outcome measures, including pragmatic characteristics using an adapted Psychometric and Pragmatic Evidence Rating Scale and ACP outcome domains using the standardized ACP Outcome Framework (i.e., process, action, healthcare, or quality of care).ResultsWe included 21 ACP RCTs. Trials included 103 outcome measures (39 primary and 64 secondary), of which 11% measured process, 14% measured action, 49% measured healthcare, and 26% measured quality of care. Twenty-four (23%) outcome measures were highly pragmatic, the majority of which (67%) reflected healthcare outcome measures. Sixty-one (59%) outcomes were assessed as highly relevant to PLWD or their care partners. Only 20% (n = 21) of outcome measures were embedded into clinical practice. Most (62%) RCTs were conducted in nursing homes, and 33% were focused PLWD with advanced stage disease.ConclusionsIn RCTs testing ACP interventions to support PLWD, only 23% of outcome measures were highly pragmatic, and most of these measured healthcare utilizations. Outcome assessments were rarely integrated into the EHR during routine clinical care. New outcome measures that address the lived experience of PLWD and their care partners plus have high pragmatic characteristics are needed for embedded pragmatic clinical trials.

Project description:ObjectivesTherapy outcome measures (TOMs) in temporomandibular disorders (TMDs) have not been systematically evaluated. We systematically explored the main TOM assessment methods for TMD TOMs used in previous studies.DesignScoping review.Data sourcesAccording to Preferred Reporting Items for Systematic reviews and Meta-Analysis extension for Scoping Review reporting guidelines, we systematically searched five key databases (MEDLINE/PubMed, Web of Science, Embase, Epistemonikos and ClinicalTrials) and thoroughly scanned relevant grey literature using Medical Subject Headings, Emtree and index terms.Eligibility criteriaWe considered primary research papers published from January 2010 to December 2020 that included patients with TMD aged ≥18 years, diagnosed according to the Diagnostic Criteria for Temporomandibular Disorders.Data extraction and synthesisFour reviewers extracted general information and information on study design and setting, target, interventions, and outcome type.ResultsOne hundred and seventy-two of the 3726 screened articles (3704 by search engines and 22 manually) were included. The TOMs analysed included pain (n=161 articles), maximal mouth opening (MMO) (91), jaw function (32), jaw movement (26), joint sound (16), quality of life (QOL) (15), depression/anxiety (14), oral QOL (10) or others (30). Evaluation periods were <4 weeks (111), <8 weeks (62), <12 weeks (59), >12 weeks (75) or 'not mentioned' (12). Pain outcomes (229) included general pain (115), tenderness (45), pain during functioning (44), resting pain (16) and others (8). Pain outcome evaluation methods included Visual Analogue Scale (VAS; 121), Numerical Rating Scale (21) and other methods (21). Pain outcome indicators were binary (10) or continuous (158); only five studies reported the least significant difference in treatment efficacy. MMO evaluation using painless methods (19) and jaw function evaluation using methods assessing mandibular movement range (23) were the most frequent.ConclusionsTMD TOMs are diverse; the major outcomes were pain, MMO, jaw function and jaw movement. Most pain outcomes are evaluated by VAS Score changes.

Dataset Information

Leveraging Visual Outcome Measures to Advance Therapy Development in Neuroimmunologic Disorders.

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Leveraging Visual Outcome Measures to Advance Therapy Development in Neuroimmunologic Disorders.

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