Development of outcome measures for autoimmune dermatoses.
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ABSTRACT:
SUBMITTER: Gaines E
PROVIDER: S-EPMC2117335 | biostudies-other | 2008 Jan
REPOSITORIES: biostudies-other
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Project description:Purpose:To determine if immunological markers (1) are significantly different between autoimmune retinopathy (AIR) patients and controls and (2) correlate with disease progression in AIR patients. Methods:We enrolled patients with a possible AIR diagnosis, as well as control participants without eye disease, autoimmunity, or cancer. Immunological markers were tested in all participants. In addition, AIR patients had up to three blood draws for testing over their disease course. For AIR patients, clinical measures, including visual acuity (VA) and Goldmann visual field (GVF) area, were recorded at each draw. We used the Mann-Whitney U test to compare the immunological markers between AIR patients and controls. We used multilevel mixed-effect regression to investigate the correlation between markers and clinical parameters over time in AIR patients. Results:Seventeen patients with AIR and 14 controls were included. AIR patients had a higher percent of monocytes (Z = 3.076, P = 0.002). An increase in immunoglobulin G against recoverin was correlated with a VA decrease (? = 0.0044, P < 0.0001). An increase in monocyte proportion was correlated with a decrease in GVF area (? = -7.27, P = 0.0021). Several markers of B-cell depletion were correlated with GVF improvement. Conclusions:Monocytes may play a role in AIR pathophysiology and be a disease activity marker. B-cell depletion markers correlated with clinical parameter improvement, particularly GVF. Translational Relevance:This work elucidates immunologic markers that may improve the accuracy of diagnosis and treatment of AIR.
| S-EPMC7414616 | biostudies-literature
Project description:Interventions: Group 1: The patients are interviewed on the basis of an internationally recognized questionnaire (EORTC QLQ-30 and QLQ-C29) on their general well-being and physical fitness, on mental health problems and on physical problems. The survey takes place at two measuring times: before therapy and once again after 12 months. The total duration of the study is three years.
Primary outcome(s): health-related quality of life measured by EORTC QLQ-C30 and QLQ-CR29; pretherapeutically and once again posttherapeutically after 12 months
Study Design: Allocation: ; Masking: ; Control: ; Assignment: ; Study design purpose: other
| 2431299 | ecrin-mdr-crc
Project description: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.
| S-EPMC8711076 | biostudies-literature
Project description:ObjectivesTo develop and evaluate the psychometric properties of child- and parent-proxy measures of oral health aesthetics.MethodsItems that describe children's perceptions of their oral attractiveness and its impact on social, emotional, and behavioural functioning were developed based on a systematic review of existing measures, clinician feedback (n = 13) and child semi-structured interviews (n = 27). The tools' content validity was assessed in cognitive interviews with 21 children. Items were administered to socio-demographically diverse samples of 998 children aged 8-17 years and 626 parents of children aged 5-17 years. Psychometric methods were used to finalize and calibrate item banks, generate short questionnaire forms, and evaluate the tools' reliability, precision and validity.ResultsThe item banks and their short forms provide precise measurement across a wide range of oral health aesthetic states. They measure relevant and meaningful positive and negative experiences using terminology that most children as young as 8 years of age can understand. Known-group comparisons and convergence with existing measures of oral health-related quality of life, global health and body image provide evidence of construct validity. The scores are interpretable relative to the US general population.ConclusionsThe oral health aesthetic item banks and short forms provide precise and valid assessments of children's satisfaction with their oral appearance. They may be useful for targeting and evaluating paediatric dental and orthodontic care in clinical practice and research settings.
| S-EPMC8629137 | biostudies-literature
Project description:Introduction and hypothesisStandardized measures enable the comparison of outcomes across providers and treatments giving valuable information for improving care quality and efficacy. The aim of this project was to define a minimum standard set of outcome measures and case-mix factors for evaluating the care of patients with overactive bladder (OAB).MethodsThe International Consortium for Health Outcomes Measurement (ICHOM) convened an international working group (WG) of leading clinicians and patients to engage in a structured method for developing a core outcome set. Consensus was determined by a modified Delphi process, and discussions were supported by both literature review and patient input.ResultsThe standard set measures outcomes of care for adults seeking treatment for OAB, excluding residents of long-term care facilities. The WG focused on treatment outcomes identified as most important key outcome domains to patients: symptom burden and bother, physical functioning, emotional health, impact of symptoms and treatment on quality of life, and success of treatment. Demographic information and case-mix factors that may affect these outcomes were also included.ConclusionsThe standardized outcome set for evaluating clinical care is appropriate for use by all health providers caring for patients with OAB, regardless of specialty or geographic location, and provides key data for quality improvement activities and research.
| S-EPMC5705742 | biostudies-literature
Project description:The purpose of this study was to perform a comprehensive systematic review of the literature on voice-related patient-reported outcome (PRO) measures in adults and to evaluate each instrument for the presence of important measurement properties.MEDLINE, the Cumulative Index of Nursing and Allied Health Literature, and the Health and Psychosocial Instrument databases were searched using relevant vocabulary terms and key terms related to PRO measures and voice. Inclusion and exclusion criteria were developed in consultation with an expert panel. Three independent investigators assessed study methodology using criteria developed a priori. Measurement properties were examined and entered into evidence tables.A total of 3,744 studies assessing voice-related constructs were identified. This list was narrowed to 32 PRO measures on the basis of predetermined inclusion and exclusion criteria. Questionnaire measurement properties varied widely. Important thematic deficiencies were apparent: (a) lack of patient involvement in the item development process, (b) lack of robust construct validity, and (c) lack of clear interpretability and scaling.PRO measures are a principal means of evaluating treatment effectiveness in voice-related conditions. Despite their prominence, available PRO measures have disparate methodological rigor. Care must be taken to understand the psychometric and measurement properties and the applicability of PRO measures before advocating for their use in clinical or research applications.
| S-EPMC5533561 | biostudies-other
Project description:BackgroundPatient-reported outcome measures (PROMs) measure patients' perspectives on health outcomes and are increasingly used in health care. To capture the patient's perspective, it is essential that patients are involved in PROM development OBJECTIVE: This article reviews in what ways and to what extent patients are involved in PROM development and whether patient involvement has increased over time.Search strategyLiterature was searched in PubMed, EMBASE, MEDLINE and the Cochrane Methodology Register.Inclusion criteriaStudies were included if they described a new PROM development.Data extractionBasic information and information regarding patient involvement in development phases was recorded.Main resultsA total of 189 studies, describing the development of 193 PROMs, were included. Most PROMs were meant for chronic disease patients (n = 59) and measured quality of life (n = 28). In 25.9% of the PROM development studies, no patients were involved. Patients were mostly involved during item development (58.5%), closely followed by testing for comprehensibility (50.8%), while patient involvement in determining which outcome to measure was minimal (10.9%). Some patient involvement took place in the development of most PROMs, but in only 6.7% patients were involved in all aspects of the development. Patient involvement did not increase with time.ConclusionsAlthough patient involvement in PROM development is essential to develop valid patient-centred PROMs, patients are not always involved. When patients are involved, their level of involvement varies considerably. These variations suggest that further attention to building and/or disseminating consensus on requirements for patient involvement in PROM development is necessary.
| S-EPMC5217930 | biostudies-literature
Project description:The patient-rated Core Outcome Measures Index (COMI) assesses the multidimensional impact of back problems on the sufferer. The brevity and comprehensibility of the tool make it practical for use in clinical and research settings. Although the COMI has been cross-culturally adapted in various languages worldwide, there is currently no Japanese version. The aim of this study was to develop a Japanese version of the COMI by: (1) performing a cross-cultural adaptation of the English version and (2) evaluating the psychometric properties of the Japanese version of the COMI in Japanese volunteers with chronic back problems.The English version of the COMI was cross-culturally adapted for the Japanese language using established guidelines. The pre-final version was pilot-tested in five Japanese-speaking patients with low back pain (LBP) and a history of spine surgery. The psychometric properties of the Japanese COMI were tested in a group of 1052 individuals with chronic LBP (LBP ?3 months), aged 20-69 years, who were recruited through a web-based survey. The psychometric properties that were evaluated included convergent and known-group validity, using the following reference questionnaires: EuroQol 5 Dimension, Roland Morris Disability Questionnaire, Short Form 8™ Health Survey, and the Keele STarT Back Screening Tool.The pre-final version of the cross-culturally adapted Japanese COMI was completed without any major problems of understanding or acceptability. For the evaluation of its psychometric properties, tests for convergent validity showed moderate correlations between COMI items and the respective reference questionnaires for symptom-specific well-being [-?0.33--0.48] and disability domains [0.48] and strong correlations (>?0.5) for the other domains and the COMI summary score. The analysis of known-group validity showed a linear trend for the COMI score in relation to prognostic risk (P?<?0.001).The Japanese COMI retained conceptual equivalence to the original using comprehensible and acceptable Japanese expressions. We developed a Japanese version of the COMI that displayed qualities that support its convergent and known-group validity. The availability of a Japanese version of the COMI should allow for improved documentation of the care provided to patients with back problems.
| S-EPMC5834844 | biostudies-other
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.
| S-EPMC6368710 | biostudies-literature
Project description:Purpose of reviewTakayasu's arteritis (TAK) is a large-vessel vasculitis with a chronic, indolent course affecting the aorta and its main branches. This review will describe the recent studies to develop validated outcome measures to assess TAK.Recent findingsTAK is traditionally assessed with a physician's global assessment including symptoms and signs of inflammation and vascular insufficiency, acute-phase reactants (APRs), and imaging including conventional digital subtraction angiography, computerized tomographic, and magnetic resonance angiography, and recently 18-FDG-PET. Recent attempts to develop a validated tool for disease assessment include the Indian Takayasu Clinical Activity Score (ITAS2010), which incorporates clinical signs and symptoms with APRs in a simplified and weighted adoption of the Birmingham Vasculitis Activity Score. Among biomarkers to assess clinical activity, pentraxin-3 is perhaps the most promising, but its validity and superiority against APRs in clinical practice need to be demonstrated. Patient-reported outcomes (PROs) are increasingly recognized as of substantial importance to measure in clinical trials; in addition to so-called 'generic' tools such as the SF-36 or measures of fatigue, disease-specific instruments would likely help capture aspects of TAK not measured by generic quality-of-life assessments or physician-based tools.SummaryAlthough outcome measures for TAK are not sufficiently validated, progress in the assessment of TAK is reflected in recent studies with new tools such as ITAS2010, new biomarkers, and a variety of PROs.
| S-EPMC7217332 | biostudies-literature