-
PharmacoEconomics Dec 2023Missing data in costs and/or health outcomes and in confounding variables can create bias in the inference of health economics and outcomes research studies, which in...
BACKGROUND
Missing data in costs and/or health outcomes and in confounding variables can create bias in the inference of health economics and outcomes research studies, which in turn can lead to inappropriate policies. Most of the literature focuses on handling missing data in randomized controlled trials, which are not necessarily always the data used in health economics and outcomes research.
OBJECTIVES
We aimed to provide an overview on missing data issues and how to address incomplete data and report the findings of a systematic literature review of methods used to deal with missing data in health economics and outcomes research studies that focused on cost, utility, and patient-reported outcomes.
METHODS
A systematic search of papers published in English language until the end of the year 2020 was carried out in PubMed. Studies using statistical methods to handle missing data for analyses of cost, utility, or patient-reported outcome data were included, as were reviews and guidance papers on handling missing data for those outcomes. The data extraction was conducted with a focus on the context of the study, the type of missing data, and the methods used to tackle missing data.
RESULTS
From 1433 identified records, 40 papers were included. Thirteen studies were economic evaluations. Thirty studies used multiple imputation with 17 studies using multiple imputation by chained equation, while 15 studies used a complete-case analysis. Seventeen studies addressed missing cost data and 23 studies dealt with missing outcome data. Eleven studies reported a single method while 20 studies used multiple methods to address missing data.
CONCLUSIONS
Several health economics and outcomes research studies did not offer a justification of their approach of handling missing data and some used only a single method without a sensitivity analysis. This systematic literature review highlights the importance of considering the missingness mechanism and including sensitivity analyses when planning, analyzing, and reporting health economics and outcomes research studies.
Topics: Humans; Data Interpretation, Statistical; Bias; Research Design; Cost-Benefit Analysis; Outcome Assessment, Health Care
PubMed: 37490207
DOI: 10.1007/s40273-023-01297-0 -
Journal of Clinical Periodontology May 2023To evaluate outcome measures, methods of assessment, and analysis in clinical studies on the rehabilitation of full-arch edentulism with implant-supported fixed or... (Review)
Review
Rehabilitation of full-arch edentulism with fixed or removable dentures retained by root-form dental implants: A systematic review of outcomes and outcome measures used in clinical research in the last 10 years.
AIM
To evaluate outcome measures, methods of assessment, and analysis in clinical studies on the rehabilitation of full-arch edentulism with implant-supported fixed or removable prostheses.
MATERIALS AND METHODS
A protocol-oriented search was conducted in MEDLINE via PubMed, EMBASE, and Cochrane Library (PROSPERO registration CRD42021265179) from 1 January 2011 to 27 July 2021 to identify longitudinal studies reporting on the rehabilitation of edentulous patients with implant-supported prostheses. The primary aim of this study was the collection of all objectively reported outcomes in each study. Secondary aims included the description of the methods of assessment and analytical methods. Risk of bias was applied according to the study design (randomized controlled trial, cohort study, or descriptive pre-post study). Individual study data were extracted into an outcomes matrix. Outcomes were grouped into domains and descriptively analysed. A network diagram was generated to establish relationships between domains. The present review follows the PRISMA statement.
RESULTS
The screening and selection processes resulted in the identification of 491 publications, corresponding to 421 different studies (cohorts of patients). Only 24% of the studies reported the use of EQUATOR network guidelines. Implant failure/survival was the most reported outcome (270 studies), but the criterion to determine implant failure and/or survival was frequently not described or was ambiguous. Implant success was much less frequently reported (88) and was based on several heterogenous composite definitions. Marginal bone levels (233 studies), technical complications (158), and clinical outcomes (150), including peri-implant soft tissue and implant stability assessment, were also frequently reported. Patient-reported outcome measures (PROMs) (145) and function-based outcomes (40) were predominantly reported in isolation from other outcomes and most frequently in removable restorations. While quality of life was evaluated using valid instruments, patient satisfaction was evaluated based on a common sense concept of satisfaction. Economic outcomes were under-reported (13).
CONCLUSIONS
There is great heterogeneity in the criteria to define implant failure or survival and implant success, which prevents the comparison of rates across studies. Even though studies frequently report multiple outcomes, PROMs are usually reported in isolation from other outcomes. It would be valuable to have a set of core outcome variables and standardized methods of measurement for future studies.
Topics: Humans; Dental Implants; Cohort Studies; Quality of Life; Dentures; Outcome Assessment, Health Care; Dental Prosthesis, Implant-Supported
PubMed: 35815425
DOI: 10.1111/jcpe.13616 -
Clinical Oral Implants Research May 2023To evaluate outcome measures, methods of assessment, and analysis in clinical studies on the rehabilitation of full-arch edentulism with implant-supported fixed or... (Review)
Review
Rehabilitation of full-arch edentulism with fixed or removable dentures retained by root-form dental implants: A systematic review of outcomes and outcome measures used in clinical research in the last 10 years.
AIM
To evaluate outcome measures, methods of assessment, and analysis in clinical studies on the rehabilitation of full-arch edentulism with implant-supported fixed or removable prostheses.
MATERIALS AND METHODS
A protocol-oriented search was conducted in MEDLINE via PubMed, EMBASE, and Cochrane Library (PROSPERO registration CRD42021265179) from 1 January 2011 to 27 July 2021 to identify longitudinal studies reporting on the rehabilitation of edentulous patients with implant-supported prostheses. The primary aim of this study was the collection of all objectively reported outcomes in each study. Secondary aims included the description of the methods of assessment and analytical methods. Risk of bias was applied according to the study design (randomized controlled trial, cohort study, or descriptive pre-post study). Individual study data were extracted into an outcomes matrix. Outcomes were grouped into domains and descriptively analysed. A network diagram was generated to establish relationships between domains. The present review follows the PRISMA statement.
RESULTS
The screening and selection processes resulted in the identification of 491 publications, corresponding to 421 different studies (cohorts of patients). Only 24% of the studies reported the use of EQUATOR network guidelines. Implant failure/survival was the most reported outcome (270 studies), but the criterion to determine implant failure and/or survival was frequently not described or was ambiguous. Implant success was much less frequently reported (88) and was based on several heterogenous composite definitions. Marginal bone levels (233 studies), technical complications (158), and clinical outcomes (150), including peri-implant soft tissue and implant stability assessment, were also frequently reported. Patient-reported outcome measures (PROMs) (145) and function-based outcomes (40) were predominantly reported in isolation from other outcomes and most frequently in removable restorations. While quality of life was evaluated using valid instruments, patient satisfaction was evaluated based on a common sense concept of satisfaction. Economic outcomes were under-reported (13).
CONCLUSIONS
There is great heterogeneity in the criteria to define implant failure or survival and implant success, which prevents the comparison of rates across studies. Even though studies frequently report multiple outcomes, PROMs are usually reported in isolation from other outcomes. It would be valuable to have a set of core outcome variables and standardized methods of measurement for future studies.
Topics: Humans; Dental Implants; Cohort Studies; Quality of Life; Dentures; Outcome Assessment, Health Care; Dental Prosthesis, Implant-Supported
PubMed: 35815423
DOI: 10.1111/clr.13931 -
Nutrients Mar 2024People are increasingly encouraged to reduce animal food consumption and shift towards plant-based diets; however, the implications for children's health are unclear. In... (Review)
Review
People are increasingly encouraged to reduce animal food consumption and shift towards plant-based diets; however, the implications for children's health are unclear. In this narrative review of research in high-income settings, we summarize evidence on the increasing consumption of plant-based diets in children and update an earlier systematic review regarding their associations with children's health outcomes. The evidence indicates that vegan, but not vegetarian, diets can restrict growth relative to omnivorous children and increase the risk of being stunted and underweight, although the percentage affected is relatively small. Bone mineral content is reduced in vegetarian and, in particular, vegan children, compared to omnivores. Both vegetarian and vegan children who do not use vitamin B12 supplements manifest with B12 deficiency; however, supplementation rectifies this problem. Both vegetarians and vegans have lower concentrations of 25(OH)D if unsupplemented, and lower body iron stores, but usually have normal iron metabolism markers. Both groups are at risk of iodine deficiency, and this might affect thyroid health. Children consuming a vegan diet have a more favorable lipid profile than omnivorous children; however, the results for a vegetarian diet are inconsistent and vary by outcome. Based on the same scientific evidence, national and international dietary recommendations are heterogeneous, with some countries supporting plant-based diets among infants, children, and adolescents, and others discouraging them. We offer a research roadmap, highlighting what is needed to provide adequate evidence to harmonize dietary recommendations for plant-based diets in children. A number of measures should urgently be introduced at international and national levels to improve the safety of their use in children.
Topics: Child; Infant; Adolescent; Animals; Humans; Diet, Plant-Based; Diet; Diet, Vegetarian; Vegetarians; Diet, Vegan; Iron; Outcome Assessment, Health Care
PubMed: 38474851
DOI: 10.3390/nu16050723 -
BMC Geriatrics Apr 2022Comprehensive geriatric assessment (CGA) addresses the bio-psycho-social needs of older adults through multidimensional assessments and management. Synthesising evidence...
Health outcomes and implementation barriers and facilitators of comprehensive geriatric assessment in community settings: a systematic integrative review [PROSPERO registration no.: CRD42021229953].
BACKGROUND
Comprehensive geriatric assessment (CGA) addresses the bio-psycho-social needs of older adults through multidimensional assessments and management. Synthesising evidence on quantitative health outcomes and implementation barriers and facilitators would inform practice and policy on CGA for community-dwelling older adults.
METHODS
We systematically searched four medical and social sciences electronic databases for quantitative, qualitative, and mixed methods studies published from 1 January 2000 to 31 October 2020. Due to heterogeneity of articles, we narratively reviewed the synthesis of evidence on health outcomes and implementation barriers and facilitators.
RESULTS
We screened 14,151 titles and abstracts and 203 full text articles, and included 43 selected articles. Study designs included controlled intervention studies (n = 31), pre-post studies without controls (n = 4), case-control (n = 1), qualitative methods (n = 3), and mixed methods (n = 4). A majority of articles studied populations aged ≥75 years (n = 18, 42%). CGAs were most frequently conducted in the home (n = 25, 58%) and primary care settings (n = 8, 19%). CGAs were conducted by nurses in most studies (n = 22, 51%). There was evidence of improved functional status (5 of 19 RCTs, 2 of 3 pre-post), frailty and fall outcomes (3 of 6 RCTs, 1 of 1 pre-post), mental health outcomes (3 of 6 RCTs, 2 of 2 pre-post), self-rated health (1 of 6 RCTs, 1 of 1 pre-post), and quality of life (4 of 17 RCTs, 3 of 3 pre-post). Barriers to implementation of CGAs involved a lack of partnership alignment and feedback, poor acceptance of preventive work, and challenges faced by providers in operationalising and optimising CGAs. The perceived benefits of CGA that served to facilitate its implementation included the use of highly skilled staff to provide holistic assessments and patient education, and the resultant improvements in care coordination and convenience to the patients, particularly where home-based assessments and management were performed.
CONCLUSION
There is mixed evidence on the quantitative health outcomes of CGA on community-dwelling older adults. While there is perceived positive value from CGA when carried out by highly skilled staff, barriers such as bringing providers into a partnership, greater acceptance of preventive care, and operational issues could impede its implementation.
Topics: Accidental Falls; Aged; Geriatric Assessment; Humans; Independent Living; Outcome Assessment, Health Care; Quality of Life
PubMed: 35488198
DOI: 10.1186/s12877-022-03024-4 -
European Journal of Physical and... Aug 2021Comorbidities in people with traumatic brain injury (TBI) may negatively impact injury recovery course and result in long-term disability. Despite the high prevalence of...
INTRODUCTION
Comorbidities in people with traumatic brain injury (TBI) may negatively impact injury recovery course and result in long-term disability. Despite the high prevalence of several categories of comorbidities in TBI, little is known about their association with patients' functional outcomes. We aimed to systematically review the current evidence to identify comorbidities that affect functional outcomes in adults with TBI.
EVIDENCE ACQUISITION
A systematic search of Medline, Cochrane Central Register of Controlled Trials, Embase and PsycINFO was conducted from 1997 to 2020 for prospective and retrospective longitudinal studies published in English. Three researchers independently screened and assessed articles for fulfillment of the inclusion criteria. Quality assessment followed the Quality in Prognosis Studies tool and the Scottish Intercollegiate Guidelines Network methodology recommendations.
EVIDENCE SYNTHESIS
Twenty-two studies of moderate quality discussed effects of comorbidities on functional outcomes of patients with TBI. Cognitive and physical functioning were negatively affected by comorbidities, although the strength of association, even within the same categories of comorbidity and functional outcome, differed from study to study. Severity of TBI, sex/gender, and age were important factors in the relationship. Due to methodological heterogeneity between studies, meta-analyses were not performed.
CONCLUSIONS
Emerging evidence highlights the adverse effect of comorbidities on functional outcome in patients with TBI, so clinical attention to this topic is timely. Future research on the topic should emphasize time of comorbidity onset in relation to the TBI event, to support prevention, treatment, and rehabilitation. PROSPERO registration (CRD 42017070033).
Topics: Brain Injuries, Traumatic; Comorbidity; Disability Evaluation; Humans; Outcome Assessment, Health Care; Prognosis
PubMed: 33541041
DOI: 10.23736/S1973-9087.21.06491-1 -
European Urology Oct 2023In the oncology setting, patient-reported outcome measures (PROMs) provide important data that help to ensure patient-relevant endpoints are captured and reported. Use... (Review)
Review
CONTEXT
In the oncology setting, patient-reported outcome measures (PROMs) provide important data that help to ensure patient-relevant endpoints are captured and reported. Use of this information for treatment decision-making by clinicians and patients in real-world settings is facilitated by consistent and transparent reporting of trial methods.
OBJECTIVE
To identify and compare PROMs used in advanced renal cell carcinoma (RCC) trials in terms of the rationale for the choice of measure, endpoint hierarchy (primary, secondary, exploratory), assessment time points, statistical methods, and statistical metrics for interpretation.
EVIDENCE ACQUISITION
A systematic literature review via searches of four online databases (2016-2021) and recent conference abstracts (2019-2021) identified 2616 articles, of which 33 were included in the review according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines.
EVIDENCE SYNTHESIS
Among the 33 clinical studies included, 19 different PROMs were identified: three kidney cancer-specific scales, two cancer-specific scales, two generic scales, and 12 symptom-specific scales. The endpoint hierarchy for patient reported outcome (PRO) assessment was reported in 42% of the studies; one study included PROs as a primary endpoint. Reporting of time points, minimal important differences, and statistical analyses was highly heterogeneous.
CONCLUSIONS
A diverse range of PROMs have been included in clinical studies for patients with advanced/metastatic RCC. Prespecified analyses for PRO assessments were generally not stated, while analytical methods and reporting varied. An improvement in alignment across studies would better inform regulatory, market-access, reimbursement, and clinical decision-making to improve patient care.
PATIENT SUMMARY
We reviewed how the impact of cancer therapies on health outcomes from the patient's point of view is being measured in clinical trials for kidney cancer. The techniques and reporting varied across trials. Standardisation of how these data are captured and reported may improve care and decision-making for patients with kidney cancer.
Topics: Humans; Carcinoma, Renal Cell; Patient Reported Outcome Measures; Kidney Neoplasms; Patient Outcome Assessment; Research Design
PubMed: 37550153
DOI: 10.1016/j.eururo.2023.07.006 -
European Spine Journal : Official... Apr 2023Adolescent idiopathic scoliosis (AIS) is the most common type of scoliosis that affects children aged 10-18 years old, manifesting in a three-dimensional spinal... (Review)
Review
PURPOSE
Adolescent idiopathic scoliosis (AIS) is the most common type of scoliosis that affects children aged 10-18 years old, manifesting in a three-dimensional spinal deformity. This study aimed to explore outcome measures used in defining AIS treatment success. Particularly, analyzing the extent of qualitative and quantitative (radiographic and quality of life domains) measures to evaluate AIS and whether AIS treatment approaches (surgical, bracing and physiotherapy) influences outcomes used as proxies of treatment success.
METHODS
EMBASE and MEDLINE databases were used to conduct a systematic scoping review with 654 search queries. 158 papers met the inclusion criteria and were screened for data extraction. Extractable variables included: study characteristics, study participant characteristics, type of study, type of intervention approach and outcome measures.
RESULTS
All 158 studies measured quantitative outcomes. 61.38% of papers used radiographic outcomes whilst 38.62% of papers used quantitative quality of life outcomes to evaluate treatment success. Irrespective of treatment intervention utilized, the type of quantitative outcome measure recorded were similar in proportion. Moreover, of the radiographic outcome measures, the subcategory Cobb angle was predominantly used across all intervention approaches. For quantitative quality of life measures, questionnaires investigating multiple domains such as SRS were primarily used as proxies of AIS treatment success across all intervention approaches.
CONCLUSION
This study identified that no articles employed qualitative measures of describing the psychosocial implications of AIS in defining treatment success. Although quantitative measures have merit in clinical diagnoses and management, there is increasing value in using qualitative methods such as thematic analysis in guiding clinicians to develop a biopsychosocial approach for patient care.
Topics: Child; Humans; Adolescent; Scoliosis; Quality of Life; Treatment Outcome; Outcome Assessment, Health Care; Research Design
PubMed: 36847911
DOI: 10.1007/s00586-023-07592-w -
Critical Care (London, England) Nov 2023CONCISE is an internationally agreed minimum set of outcomes for use in nutritional and metabolic clinical research in critically ill adults. Clinicians and researchers... (Meta-Analysis)
Meta-Analysis
A systematic review and meta-analysis of the clinimetric properties of the core outcome measurement instruments for clinical effectiveness trials of nutritional and metabolic interventions in critical illness (CONCISE).
BACKGROUND
CONCISE is an internationally agreed minimum set of outcomes for use in nutritional and metabolic clinical research in critically ill adults. Clinicians and researchers need to be aware of the clinimetric properties of these instruments and understand any limitations to ensure valid and reliable research. This systematic review and meta-analysis were undertaken to evaluate the clinimetric properties of the measurement instruments identified in CONCISE.
METHODS
Four electronic databases were searched from inception to December 2022 (MEDLINE via Ovid, EMBASE via Ovid, CINAHL via Healthcare Databases Advanced Search, CENTRAL via Cochrane). Studies were included if they examined at least one clinimetric property of a CONCISE measurement instrument or recognised variation in adults ≥ 18 years with critical illness or recovering from critical illness in any language. The COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) checklist for systematic reviews of Patient-Reported Outcome Measures was used. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses were used in line with COSMIN guidance. The COSMIN checklist was used to evaluate the risk of bias and the quality of clinimetric properties. Overall certainty of the evidence was rated using a modified Grading of Recommendations, Assessment, Development and Evaluation approach. Narrative synthesis was performed and where possible, meta-analysis was conducted.
RESULTS
A total of 4316 studies were screened. Forty-seven were included in the review, reporting data for 12308 participants. The Short Form-36 Questionnaire (Physical Component Score and Physical Functioning), sit-to-stand test, 6-m walk test and Barthel Index had the strongest clinimetric properties and certainty of evidence. The Short Physical Performance Battery, Katz Index and handgrip strength had less favourable results. There was limited data for Lawson Instrumental Activities of Daily Living and the Global Leadership Initiative on Malnutrition criteria. The risk of bias ranged from inadequate to very good. The certainty of the evidence ranged from very low to high.
CONCLUSIONS
Variable evidence exists to support the clinimetric properties of the CONCISE measurement instruments. We suggest using this review alongside CONCISE to guide outcome selection for future trials of nutrition and metabolic interventions in critical illness.
TRIAL REGISTRATION
PROSPERO (CRD42023438187). Registered 21/06/2023.
Topics: Adult; Humans; Hand Strength; Critical Illness; Activities of Daily Living; Treatment Outcome; Outcome Assessment, Health Care
PubMed: 37986015
DOI: 10.1186/s13054-023-04729-7 -
Developmental Medicine and Child... Feb 2020To develop a core outcome set (COS) for evaluating gastrostomy/gastrojejunostomy tube impact in children with neurological impairment.
AIM
To develop a core outcome set (COS) for evaluating gastrostomy/gastrojejunostomy tube impact in children with neurological impairment.
METHOD
Healthcare providers/researchers and caregivers rated the importance of candidate outcomes on a 5-point Likert scale. Outcomes rated 'somewhat important' or 'very important' by most (≥85%) respondents were voted on during a consensus meeting. Outcomes that reached consensus for inclusion were ratified and assigned to Outcome Measures in Rheumatology filter core areas. The COS was validated in a separate group of caregivers.
RESULTS
Twelve outcomes were selected from 120 candidate outcomes to form the COS. These included five 'Life Impact' outcomes, three 'Pathophysiological Manifestations' outcomes, two 'Resource Use' outcomes, one 'Growth and Development' outcome, and one 'Death' outcome.
INTERPRETATION
We developed an evidence-informed and consensus-based COS for use in studies of gastrostomy/gastrojejunostomy tube feeding in children with neurological impairment. Implementation of this COS will help reduce heterogeneity between studies and facilitate evidence-based decision-making.
WHAT THE PAPER ADDS
Caregivers, healthcare providers, and researchers ranked the importance of 120 outcomes. Twelve core outcomes were identified as essential to measure in future clinical research studies.
Topics: Adolescent; Child; Clinical Studies as Topic; Enteral Nutrition; Gastrostomy; Humans; Jejunostomy; Nervous System Diseases; Outcome Assessment, Health Care; Primary Health Care
PubMed: 31372989
DOI: 10.1111/dmcn.14326