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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 |

REPOSITORIES: biostudies-literature

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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:BACKGROUND:Among 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. METHODS:Potential 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. RESULTS:Due 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. CONCLUSION:The 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: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.

Project description:Purpose Generalizations can be made about the order in which speech sounds are added to a child's phonemic inventory and the ways that child speech deviates from adult targets in a given language. Developmental and disordered speech patterns are presumed to reflect differences in both phonological knowledge and skilled motor control, but the relative contribution of motor control remains unknown. The ability to differentially control anterior versus posterior regions of the tongue increases with age, and thus, complexity of tongue shapes is believed to reflect an individual's capacity for skilled motor control of speech structures. Method The current study explored the relationship between tongue complexity and phonemic development in children (ages 4-6 years) with and without speech sound disorder producing various phonemes. Using established metrics of tongue complexity derived from ultrasound images, we tested whether tongue complexity incrementally increased with age in typical development, whether tongue complexity differed between children with and without speech sound disorder, and whether tongue complexity differed based on perceptually rated accuracy (correct vs. incorrect) for late-developing phonemes in both diagnostic groups. Results Contrary to hypothesis, age was not significantly associated with tongue complexity in our typical child sample, with the exception of one association between age and complexity of /t/ for one measure. Phoneme was a significant predictor of tongue complexity, and typically developing children had more complex tongue shapes for /ɹ/ than children with speech sound disorder. Those /ɹ/ tokens that were rated as perceptually correct had higher tongue complexity than the incorrect tokens, independent of diagnostic classification. Conclusions Quantification of tongue complexity can provide a window into articulatory patterns characterizing children's speech development, including differences that are perceptually covert. With the increasing availability of ultrasound imaging, these measures could help identify individuals with a prominent motor component to their speech sound disorder and could help match those individuals with a corresponding motor-based treatment approach. Supplemental Material https://doi.org/10.23641/asha.14880039.

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

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