Outcome Measures Used in Ocular Gene Therapy Trials: A Scoping Review of Current Practice.
Ontology highlight
ABSTRACT:
SUBMITTER: Jolly JK
PROVIDER: S-EPMC6759794 | biostudies-literature | 2019
REPOSITORIES: biostudies-literature
Publications
Outcome Measures Used in Ocular Gene Therapy Trials: A Scoping Review of Current Practice.
Jolly Jasleen K JK Bridge Holly H MacLaren Robert E RE
Frontiers in pharmacology 20190918
Multiple gene therapy trials are occurring for a variety of ophthalmic diseases around the world. The safety of gene therapy in the eye has been established, and the next step is to reliably assess efficacy. This is primarily done through the use of imaging techniques and visual function measures. Standardized visual function assessments, however, were originally developed for a clinical setting and may not be suitable for detecting and quantifying therapeutic changes. This scoping review takes ...[more]
Similar Datasets
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.
| S-EPMC9396127 | biostudies-literature
Project description:BackgroundAnkle fractures cause considerable pain, loss of function and healthcare resource use. High quality randomised controlled trials are required to evaluate the optimal management protocols for ankle fracture. However, there is debate regarding the most appropriate outcome measure to use when assessing patients with ankle fractures. The aim of this systematic review is to identify and summarise primary outcome measure use in clinical trials of non-pharmacological interventions for adults with an ankle fracture.MethodsWe performed comprehensive searches of the Medline, Embase, CINAHL, AMED and Cochrane CENTRAL databases, as well as ISRCTN and ClinicalTrials.gov online clinical trial registries on 19/06/2019 with no date limits applied. The titles and abstracts were initially screened to identify randomised or quasi-randomised clinical trials of non-pharmacological interventions for ankle fracture in adults. Two authors independently screened the full text of any articles which could potentially be eligible. Descriptive statistics we used to summarise the outcome measures collected in these articles including an assessment of trends over time. Secondary analysis included a descriptive summary of the multi-item patient reported outcome measures used in this study type.ResultsThe searches returned a total of 3380 records. Following application of the eligibility criteria, 121 records were eligible for inclusion in this review. The most frequently collected primary outcome measures in this type of publication was the Olerud Molander Ankle Score, followed by radiographic and range of movement assessments. There was a total of 28 different outcome measures collected and five different multi-item, patient reported outcome measures collected as the primary outcome measure. There was a sequential increase in the number of this type of study published per decade since the 1980's.ConclusionThis review demonstrates the wide range of measurement methods used to assess outcome in adults with an ankle fracture. Future research should focus on establishing the validity and reliability of the outcome measures used in this patient population. Formulation of a consensus based core outcome set for adults with an ankle fracture would be advantageous for ensuring homogeneity across studies in order to meta-analyse trial results.
| S-EPMC6712770 | biostudies-literature
Project description:BackgroundMultiple Sclerosis (MS) is associated with impaired gait and a growing number of clinical trials have investigated efficacy of various interventions. Choice of outcome measures is crucial in determining efficiency of interventions. However, it remains unclear whether there is consensus on which outcome measures to use in gait intervention studies in MS.ObjectiveWe aimed to identify the commonly selected outcome measures in randomized controlled trials (RCTs) on gait rehabilitation interventions in people with MS. Additional aims were to identify which of the domains of the International Classification of Functioning, Disability and Health (ICF) are the most studied and to characterize how outcome measures are combined and adapted to MS severity.MethodsPubmed, Cochrane Central, Embase and Scopus databases were searched for RCT studies on gait interventions in people living with MS according to PRISMA guidelines.ResultsIn 46 RCTs, we identified 69 different outcome measures. The most used outcome measures were 6-minute walking test and the Timed Up and Go test, used in 37% of the analyzed studies. They were followed by gait spatiotemporal parameters (35%) most often used to inform on gait speed, cadence, and step length. Fatigue was measured in 39% of studies. Participation was assessed in 50% of studies, albeit with a wide variety of scales. Only 39% of studies included measures covering all ICF levels, and Participation measures were rarely combined with gait spatiotemporal parameters (only two studies).ConclusionsSelection of outcome measures remains heterogenous in RCTs on gait rehabilitation interventions in MS. However, there is a growing consensus on the need for quantitative gait spatiotemporal parameter measures combined with clinical assessments of gait, balance, and mobility in RCTs on gait interventions in MS. Future RCTs should incorporate measures of fatigue and measures from Participation domain of ICF to provide comprehensive evaluation of trial efficacy across all levels of functioning.
| S-EPMC8483298 | biostudies-literature
Project description:It is unclear which patient-reported outcome measures (PROMs) can assess non-operative care for scoliosis. Most existing tools aim to assess the effects of surgery. This scoping review aimed to inventory the PROMs used to assess non-operative scoliosis treatment by population and languages. We searched Medline (OVID) as per COSMIN guidelines. Studies were included if patients were diagnosed with idiopathic scoliosis or adult degenerative scoliosis and used PROMs. Studies without quantitative data or reporting on fewer than 10 participants were excluded. Nine reviewers extracted the PROMs used, the population(s), language(s), and study setting(s). We screened 3724 titles and abstracts. Of these, the full texts of 900 articles were assessed. Data were extracted from 488 studies, in which 145 PROMs were identified across 22 languages and 5 populations (Adolescent Idiopathic Scoliosis, Adult Degenerative Scoliosis, Adult Idiopathic Scoliosis, Adult Spine Deformity, and an Unclear category). Overall, the most used PROMs were the Oswestry Disability Index (ODI, 37.3%), Scoliosis Research Society-22 (SRS-22, 34.8%), and the Short Form-36 (SF-36, 20.1%), but the frequency varied by population. It is now necessary to determine the PROMs that demonstrate the best measurement properties in the non-operative treatment of scoliosis to include in a core set of outcomes.
| S-EPMC9954663 | biostudies-literature
Project description:Introduction:Various methods have been used to interpret the reports of pediatric polypharmacy across the literature. This is the first scoping review that explores outcome measures in pediatric polypharmacy research. Objectives:The aim of our study was to describe outcome measures assessed in pediatric polypharmacy research. Methods:A search of electronic databases was conducted in July 2017, including Ovid Medline, PubMed, Elsevier Embase, Wiley Cochrane Central Register of Controlled Trials (CENTRAL), EBSCO CINAHL, Ovid PsyclNFO, Web of Science Core Collection, ProQuest Dissertations and Thesis A&I. Data were extracted about study characteristics and outcome measures, and also synthesized by harms or benefits mentioned. Results:The search strategy initially identified 8169 titles and screened 4398 using the inclusion criteria after de-duplicating. After the primary screening, a total of 363 studies were extracted for the data analysis. Polypharmacy (prevalence) was identified as an outcome in 31.4% of the studies, prognosis-related outcomes in 25.6%, and adverse drug reactions in 16.5%. A total of 265 articles (73.0%) mentioned harms, including adverse drug reactions (26.4%), side effects (24.2%), and drug-drug interactions (20.9%). A total of 83 studies (22.9%) mentioned any benefit, 48.2% of which identified combination for efficacy, 24.1% combination for treatment of complex diseases, and 19.3% combination for treatment augmentation. Thirty-eight studies reported adverse drug reaction as an outcome, where polypharmacy was a predictor, with various designs. Conclusions:Most studies of pediatric polypharmacy evaluate prevalence, prognosis, or adverse drug reaction-related out-comes, and underscore harms related to polypharmacy. Clinicians should carefully weigh benefits and harms when introducing medications to treatment regimens.
| S-EPMC7123381 | biostudies-literature
Project description:BackgroundRetinoblastoma is a childhood retinal cancer with lifelong consequences such as vision loss and increased risk of second cancer. Patient-reported outcome measures (PROMs) are instruments that measure outcomes related to health directly reported by patients. The purpose of this study was to determine the scope, characteristics and quality of PROMs used in retinoblastoma and related fields of pediatric ophthalmology and pediatric oncology.MethodsDatabases MEDLINE and Embase were searched for studies in the English language that reported on PROMs used in retinoblastoma, pediatric oncology, or pediatric ophthalmology; grey literature and studies reporting on developmental PROM phases were excluded. PROMs were grouped by the construct measured and domains assessed, and classified as condition-specific or generic. A subsequent search was then conducted in MEDLINE and Embase for studies assessing measurement properties of the identified PROMs. PROMs with associated studies were assessed for their methodologic quality using the COnsensus-based standard for the Selection of health Measurement INstruments (COSMIN) strategy.ResultsAmong 110 eligible studies uncovered by the database searches, 143 PROMs were identified: one retinoblastoma-specific, 56 ophthalmology- and 86 oncology-related. The most common construct measured was 'health-related quality of life' and the most common domain assessed was emotional well-being. Of the 143 PROMs, 100 had associated validation studies; the one retinoblastoma-specific PROM was not validated. Quality assessment revealed 34/100 PROMs received a score of sufficient quality in both subcategories of 'overall content validity'; 3/100 received a score of sufficient quality in both subcategories of 'internal structure'; 0/100 received a score of sufficient quality in all three subcategories of 'remaining measurement properties'. The Patient-Reported Outcome Measure Information System (PROMIS) Pediatric Profile-25 was the highest-scoring PROM identified, meeting COSMIN standards for 2/3 measurement property categories (and 5/7 subcategories). Eleven additional PROMs were identified which had sufficient scores in 1/3 measurement property categories (and 5/7 subcategories).ConclusionThe study identified several PROMs from the pediatric ophthalmology and pediatric oncology literature that could be relevant to the retinoblastoma population, but many have limits to their validation. Future development of a retinoblastoma-specific PROM, performed in partnership with retinoblastoma patients to support optimal content validity, could first focus on the selection and definition of the optimal construct to measure, followed potentially by adaptation and further validation of the relevant PROMs with strong methodologic quality identified in this study.
| S-EPMC7415058 | biostudies-literature
Project description:ObjectiveThe aim of this systematic scoping review is to identify and categorize the outcome measures that have been reported in clinical studies, where therapeutic plasma exchange (TPE) has been used as an intervention in any clinical settings, excluding thrombotic thrombocytopenic purpura (TTP).MethodsWe searched electronic databases using a predefined search strategy from inception to October 9, 2020. Two reviewers independently screened and extracted data.ResultsWe included 42 studies (37 RCTs and 5 prospective cohort studies) grouped into six main categories (neurology, immunology, renal, rheumatology, hematology, and dermatology). Primary outcomes were defined in eight studies (19%, 8/42) and were categorized as efficacy (five studies) or patient reported outcomes (three studies). A power calculation was reported in six studies (75%, 6/8): five neurology studies (mainly patient reported outcomes) and a single immunological study (efficacy outcome). Disease-specific efficacy outcomes were dependent on the clinical setting of the population receiving TPE. Most of the trials (43%, 18/42) were undertaken in patients with neurology conditions where clear, disease-specific, clinical outcome measures were used, including neurological disability scales (11/18, 61%), change in neurological examination (9/18, 50%), and functional improvement scores (7/18, 39%). For other conditions, the reporting of disease-specific outcomes was poorly reported. Safety outcomes were mainly related to replacement fluid type rather than being disease-specific. The most common outcome reported was hypotension (19%, 8/42), and this was primarily in patients exchanged with albumin.ConclusionFuture clinical studies to determine which fluid replacement option is most efficacious and safe should use disease-specific outcomes, as a trial in one therapeutic area may not necessarily translate to another therapeutic area. Patient reported outcomes are not universally reported for all disease areas. Safety measures focused primarily on fluid safety.
| S-EPMC9795884 | biostudies-literature
Project description:Stem cells are undifferentiated cells showcasing a remarkable capacity of self-replenishing and differentiating into mature cells. Their ability to proliferate connotes that a designated stem cell source is capable of generating an unrestricted number of mature cells. The ever-increasing comprehension of position, activity, and function of ocular stem cells has led to rapid progress and incessant improvement of possible procedures and therapies. A narrative review was conducted to summarize the current evidence on clinical trials and respective literature, regarding current evolution in the field of ocular regenerative medicine. We tried to ascertain the safety of experimental and clinical procedures, their effectiveness, and the ethical repercussion of their use.
| S-EPMC9453397 | biostudies-literature
Project description:Cannabis use disorder (CUD) is prevalent and demand for treatment is increasing, yet few individuals engage in formal treatment and the efficacy of established interventions for CUD is modest. Existing clinical trials evaluating psychosocial and pharmacological treatments for CUD have incorporated a wide variety of measures for assessing cannabis use outcomes, including abstinence, self-reported frequency and quantity used, withdrawal, use/dependence severity, and other psychosocial outcomes. The heterogeneity of measures and outcomes has limited quantitative analyses of the comparative effectiveness of existing interventions. The purpose of this systematic review is to: 1) identify and characterize approaches for measuring cannabis use in existing CUD intervention trials, including abstinence, frequency and quantity of use, and 2) summarize measures used to assess treatment efficacy in other outcome domains (e.g., cannabis use severity, psychosocial functioning, cannabis withdrawal), and provide a platform for future research to evaluate which outcome measures are most likely to reflect treatment efficacy and clinically significant improvement in other outcome domains.
| S-EPMC7038155 | biostudies-literature
Project description:Because no gold-standard outcome measure or measures exist to allow comparison of pediatric stroke study outcomes in clinical trials, we designed a systematic review of the literature to survey the current use of pediatric stroke outcome measures. Studies that used at least 1 standardized measure to assess the outcome of children with ischemic or hemorrhagic stroke, from full-term newborn to age 18 years, were included. Although 34 studies were included, an additional 36 studies could not be included because ad hoc, author-generated outcome measures were used. Excluding those measures in neuropsychological batteries, 38 unique outcome measures were used. The Wechsler Intelligence Scales, Pediatric Stroke Outcome Measure, and Bayley Scales of Infant Development were among the most used, but 79% of outcome measures were used by no more than 2 studies. Although many measures used have been validated for use in children with other medical conditions or for adults with stroke, only 1 measure has been specifically validated for use in pediatric ischemic stroke. To maximize comparability of future clinical trial results, agreement regarding a preferred pediatric stroke outcome scale or battery of measures is paramount; these measures should be reliable, responsive to change, and specifically validated for use in children with stroke.
| S-EPMC3397390 | biostudies-literature