Selection Of Appropriate Outcomes Research Articles

Scores and sores: Exploring patient-reported outcomes for knee evaluation in orthopaedics, sports medicine and rehabilitation.

Recognizing and addressing the controversies surrounding using patient-reported outcome measures (PROMs) is crucial for enhancing evaluation standards in clinical studies in orthopedics, sports medicine, and rehabilitation. The article comprehensively described the challenges of using PROMs to evaluate knee conditions in these fields. Apart from defining and characterizing patient-reported outcomes and their measures, the article discussed controversies around them, such as using them as primary outcomes. It highlighted the importance of standardizing and validating PROMs. Several initiatives taken to improve the selection of appropriate outcomes for clinical research purposes were described. Additionally, the potential of technology, mainly digital health tools and mobile applications, was mentioned in the context of enhancing the collection and analysis of PROMs. The article also raised the issue of the readability of PROMs, defined as the ease with which they can be read and understood by patients. The article concluded that adopting a complementary approach to treatment evaluation by integrating subjective and objective measures is imperative for accurately assessing efficacy. This comprehensive approach provides a more holistic understanding of patient outcomes, forms the foundation for evidence-based medicine, and informs future healthcare policies. Proactive measures are urgently needed to address concerns and improve the reliability and validity of PROMs for clinical practice and research. LEVEL OF EVIDENCE: level V.

Long-term cognitive and behavioral outcomes following early exposure to general anesthetics.

Nearly 100 clinical studies have been published evaluating neurodevelopmental outcomes in children following surgery and anesthesia. These studies have reported mixed results, likely attributable at least in part to significant heterogeneity in their study designs, types and numbers of exposures, patient populations evaluated, and most importantly, the outcomes that were assessed. This review aims to summarize the results from clinical studies evaluating behavioral outcomes in children exposed to surgery and anesthesia. Children with early exposure to surgery and anesthesia were found to have limited to no differences in intelligence when compared with unexposed children. However, several studies have reported more behavioral problems in children exposed to general anesthesia. An increased incidence of attention-deficit hyperactivity disorder has also been reported in anesthetic exposed children, particularly after multiple exposures. Nearly all clinical studies of anesthetic neurotoxicity are observational in nature, so the associations between anesthetic exposure and behavioral deficits cannot yet be directly attributed to the anesthetic medication. However, the finding of deficits in some neurodevelopmental domains and not others will help guide the selection of appropriate outcomes in future studies of anesthetic neurotoxicity that can further evaluate whether anesthetic medications have an impact on neurodevelopment in children.

Development of a core outcome set for multimorbidity trials in low/middle-income countries (COSMOS): study protocol

Introduction‘Multimorbidity’ describes the presence of two or more long-term conditions, which can include communicable, non-communicable diseases, and mental disorders. The rising global burden from multimorbidity is well documented, but trial...

Orthodontic trial outcomes: Plentiful, inconsistent, and in need of uniformity? A scoping review.

The selection of appropriate outcomes that matter to both patients and operators is increasingly appreciated, with core outcome sets in clinical trials gaining in popularity. The first step in core outcome set development is the generation of a list of possible important outcomes based on a scoping literature review. Moreover, outcome heterogeneity is known to detract from the findings of systematic reviews and meta-analyses. The aim of this study was to identify the range of outcome domains and specific outcome measures in contemporary orthodontic research. Multiple electronic databases were searched from December 31, 2012, to December 31, 2016, to identify clinical trials of orthodontic interventions, with no language restrictions. Abstracts, eligible full texts, and reference lists were screened, and all reported primary and nonprimary outcomes and methods of measurement were recorded. The search identified 1267 abstracts, of which 189 full-text articles were retrieved, and 164 studies were included in the analysis. A total of 54 outcomes were identified and categorized into 14 outcome domains. The most frequently measured outcomes were patient-reported pain, periodontal health, tooth angulation/inclination changes, and treatment duration, followed by rate of tooth movement and skeletal changes. Outcomes that followed the overall course of treatment were assessed in only 14 studies. Patient perspectives are increasingly being accounted for in orthodontic trials; however, there is little consistency in outcome selection among them. The identified list of outcomes will be used to inform a ranking exercise with service users and providers to establish an agreed core outcome set for future orthodontic clinical trials.

Creation of a core outcome set for clinical trials of people with shoulder pain: a study protocol

BackgroundThe selection of appropriate outcomes or domains is crucial when designing clinical trials, to appreciate the effects of different interventions, pool results, and make valid comparisons between trials. If the findings are to influence policy and practice, then the chosen outcomes need to be relevant and important to key stakeholders, including patients and the public, healthcare professionals and others making decisions about health care. There is a growing recognition that insufficient attention has been paid to the outcomes measured in clinical trials. Recent reviews of the measurement properties of patient-reported outcome measures for shoulder disorders revealed a large selection of diverse measures, many with questionable validity, reliability, and responsiveness. These issues could be addressed through the development and use of an agreed standardized collection of outcomes, known as a core outcome set (COS), which should be measured and reported in all trials of shoulder disorders. The purpose of the present project is to develop and disseminate a COS for clinical trials in shoulder disorders.Methods/DesignThe methods for the COS development will include 3 phases: (1) a comprehensive review of the core domains used in shoulder disorder trials; (2) an international Delphi study involving relevant stakeholders (patients, clinicians, scientists) to define which domains should be core; and (3) an international focus group informed by the evidence identified in phases 1 and 2, to determine which measurement instruments best measure the core domains and identification of any evidence gaps that require further empiric evidence.DiscussionThe aim of the current proposal is to convene several meetings of international experts and patients to develop a COS for clinical trials of shoulder disorders and to develop an implementation strategy to ensure rapid uptake of the core set of outcomes in clinical trials. There would be an expectation that the core set of outcomes would always be collected and reported, but it would not preclude use of additional outcomes in a particular trial.

Outcome Measures in Glaucoma: A Systematic Review of Cochrane Reviews and Protocols.

In clinical trials, the selection of appropriate outcomes is crucial for the assessment of whether one intervention is better than another. Selection of inappropriate outcomes can compromise the utility of a trial. However, the process of selecting the most suitable outcomes to include can be complex. Ideally, glaucoma trials aim to evaluate important outcomes for clinicians and patients. A high variability in the selection of outcomes suggests that there is no consensus on how best to evaluate the effect of glaucoma interventions. Further, it makes evidence synthesis difficult. The purpose of this review is to determine the extent of clinical outcome measures used in published glaucoma Cochrane Reviews and Protocols. A systematic review was conducted (up to February 2012) of all Cochrane Reviews and Protocols related to glaucoma interventions and published in English language. All clinical, patient-reported, as well as economic outcomes were included. In the Cochrane Library there were 12 Reviews and 9 Protocols on glaucoma. A total of 118 clinical outcomes were reported. Intraocular pressure was the most commonly used clinical outcome (n=40), and it was used in 11 Reviews and 5 Protocols. Intraocular pressure was evaluated in many different ways; the most common one was a composite definition of success. Safety outcomes were also frequently reported. Visual field progression or change was reported in 6 reviews and 3 protocols, but in 13 different ways. Patient-reported quality-of-life measures were chosen as main outcome measure in 1 Review. There is a large variability in outcomes selected in glaucoma Cochrane Reviews and Protocols. This heterogeneity in outcome selection impairs the ability for evidence synthesis. There is an urgent need for standardization of outcomes used in glaucoma trials.

Consensus on the need for a hierarchical list of patient-reported pain outcomes for meta-analyses of knee osteoarthritis trials

The selection of appropriate outcomes is crucial when designing, and subsequently interpreting clinical trials, in order to directly compare the effects of different interventions in ways that minimize bias. The same is likely to apply for systematic reviews and meta-analyses. Although protocol registration for systematic reviews is still not mandatory, reviewers should be strongly encouraged to register the protocol, in order to identify - a priori - the proposed methodological approach, including all outcomes of interest. This will help to minimize the likelihood of biased post hoc decisions in review methods, such as selective outcome reporting. A group of international experts convened to address issues regarding the need to develop hierarchical lists of outcome measurement instruments for a particular outcome for meta-analyses. Meta-analysis of knee osteoarthritis (OA) trials, and the assessment of pain as an outcome, was used as an exemplar to assess how ‘Outcome Measures in Rheumatology’ (OMERACT) and other international initiatives might contribute in this area. The meeting began with formal presentations of background topics, empirical evidence from the literature, and a brief introduction to two existing hierarchical lists of pain outcome measures recommended for meta-analyses of knee OA trials. After discussions most participants agreed that there is a need to develop a methodology for generation of hierarchical lists of outcome instruments for use to guide meta-analyses. Tools that could be used to steer development of such a prioritized list are the COSMIN checklist and the OMERACT filter 2.0. For future research we suggest that among outcome instruments frequently reported in trials for the same domain, those with the best measurement properties (e.g., validity and reliability) would achieve high, if not the highest rankings for use on a hierarchical list.

Do orthodontic research outcomes reflect patient values? A systematic review of randomized controlled trials involving children

The selection of appropriate outcomes is a fundamental part of the design of clinical trials. Orthodontic treatment aims to improve a person's dentofacial appearance, and research outcomes should therefore reflect the perspectives of both clinicians and patients. In this study, we aimed to identify which outcomes were measured in recent orthodontic trials and to explore whether any relevant outcome domains were underrepresented. Five electronic databases were searched to identify all randomized controlled trials of orthodontic treatment interventions in children published in the last 5 years. Abstracts and eligible full-text articles were screened independently and in duplicate by 2 reviewers. Outcome measures were identified and categorized into 6 predetermined outcome domains. The search identified 650 abstracts, of which 244 eligible articles were retrieved in full. One hundred thirty-three studies met the inclusion criteria and were included. Morphologic features of malocclusion were measured in 84 studies (63%); health resource utilization in 43 (32%); adverse effects of orthodontic treatment in 43 (32%); quality of life in 12 (9%); functional status in 10 (8%); and physical consequences of malocclusion in 3 (2%). There was no consistency in the outcomes selected among the trials to measure these domains. Most of the outcomes used in orthodontic research are concerned with measuring morphologic changes of treatment and do not reflect patient perspectives. Five of the 6 domains were infrequently evaluated, and outcomes were heterogenous. A core set of outcomes for clinical trials of orthodontic treatment interventions would help to overcome these issues.

Variation of clinical outcomes used in glaucoma randomised controlled trials: a systematic review

PurposeIn randomised clinical trials (RCTs) the selection of appropriate outcomes is crucial to the assessment of whether one intervention is better than another. The purpose of this review is to...

Developing core outcome sets for clinical trials: issues to consider

The selection of appropriate outcomes or domains is crucial when designing clinical trials in order to compare directly the effects of different interventions in ways that minimize bias. If the findings are to influence policy and practice then the chosen outcomes need to be relevant and important to key stakeholders including patients and the public, health care professionals and others making decisions about health care. There is a growing recognition that insufficient attention has been paid to the outcomes measured in clinical trials. These issues could be addressed through the development and use of an agreed standardized collection of outcomes, known as a core outcome set, which should be measured and reported, as a minimum, in all trials for a specific clinical area. Accumulating work in this area has identified the need for general guidance on the development of core outcome sets. Key issues to consider in the development of a core outcome set include its scope, the stakeholder groups to involve, choice of consensus method and the achievement of a consensus.

A Systematic Review of Studies That Aim to Determine Which Outcomes to Measure in Clinical Trials in Children

BackgroundIn clinical trials the selection of appropriate outcomes is crucial to the assessment of whether one intervention is better than another. Selection of inappropriate outcomes can compromise the utility of a trial. However, the process of selecting the most suitable outcomes to include can be complex. Our aim was to systematically review studies that address the process of selecting outcomes or outcome domains to measure in clinical trials in children.Methods and FindingsWe searched Cochrane databases (no date restrictions) in December 2006; and MEDLINE (1950 to 2006), CINAHL (1982 to 2006), and SCOPUS (1966 to 2006) in January 2007 for studies of the selection of outcomes for use in clinical trials in children. We also asked a group of experts in paediatric clinical research to refer us to any other relevant studies. From these articles we extracted data on the clinical condition of interest, description of the method used to select outcomes, the people involved in the selection process, the outcomes selected, and limitations of the method as defined by the authors. The literature search identified 8,889 potentially relevant abstracts. Of these, 70 were retrieved, and 25 were included in the review. These studies described the work of 13 collaborations representing various paediatric specialties including critical care, gastroenterology, haematology, psychiatry, neurology, respiratory paediatrics, rheumatology, neonatal medicine, and dentistry. Two groups utilised the Delphi technique, one used the nominal group technique, and one used both methods to reach a consensus about which outcomes should be measured in clinical trials. Other groups used semistructured discussion, and one group used a questionnaire-based survey. The collaborations involved clinical experts, research experts, and industry representatives. Three groups involved parents of children affected by the particular condition.ConclusionsVery few studies address the appropriate choice of outcomes for clinical research with children, and in most paediatric specialties no research has been undertaken. Among the studies we did assess, very few involved parents or children in selecting outcomes that should be measured, and none directly involved children. Research should be undertaken to identify the best way to involve parents and children in assessing which outcomes should be measured in clinical trials.

Effect size in clinical studies of patients with rheumatoid arthritis. EULAR guidelines and OMERACT core-sets

Clinical trials in rheumatology are confronted with new challenges. The pharmaceutical industry must be more efficient in identifying and marketing new drugs, regulatory authorities require additional evidence about the effectiveness of new compounds, and the consumer including physicians require more transparency in the selection and use of appropriate outcomes in clinical trials. These challenges find their common denominator in a further standardization of clinical trials that extends beyond the application of Good Clinical Practice. For years, national and international organizations have developed recommendations for the selection of appropriate outcomes in RA clinical trials. However, these recommendations have rarely been data driven or evidence based. The international informal collaboration Outcome Measures in Rheumatoid Arthritis Clinical Trials (OMERACT) has contributed enormously towards the application of a standardized methodology in the development of recommendations to enhance the quality of research in clinical rheumatology. The scope of this initiative goes now far beyond rheumatoid arthritis and now covers all major rheumatic conditions.