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INTRODUCTION: Children with chronic conditions have greater health care needs than the general paediatric population but may not receive care that centres their needs and preferences as identified by their families. Clinicians and researchers are interested in developing interventions to improve family-centred care need information about the characteristics of existing interventions, their development and the domains of family-centred care that they address. We conducted a scoping review that aimed to identify and characterize recent family-centred interventions designed to improve experiences with care for children with chronic conditions. METHODS: We searched Medline, Embase, PsycInfo and Cochrane databases, and grey literature sources for relevant articles or documents published between 1 January 2019 and 11 August 2020 (databases) or 7-20 October 2020 (grey literature). Primary studies with ≥10 participants, clinical practice guidelines and theoretical articles describing family-centred interventions that aimed to improve experiences with care for children with chronic conditions were eligible. Following citation and full-text screening by two reviewers working independently, we charted data covering study characteristics and interventions from eligible reports and synthesized interventions by domains of family-centred care. RESULTS: Our search identified 2882 citations, from which 63 articles describing 61 unique interventions met the eligibility criteria and were included in this review. The most common study designs were quasiexperimental studies (n = 18), randomized controlled trials (n = 11) and qualitative and mixed-methods studies (n = 9 each). The most frequently addressed domains of family-centred care were communication and information provision (n = 45), family involvement in care (n = 37) and access to care (n = 30). CONCLUSION: This review, which identified 61 unique interventions aimed at improving family-centred care for children with chronic conditions across a range of settings, is a concrete resource for researchers, health care providers and administrators interested in improving care for this high-needs population. PATIENT OR PUBLIC CONTRIBUTION: This study was co-developed with three patient partner co-investigators, all of whom are individuals with lived experiences of rare chronic diseases as parents and/or patients and have prior experience in patient engagement in research (I. J., N. P., M. S.). These patient partner co-investigators contributed to this study at all stages, from conceptualization to dissemination.
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Assistência Centrada no Paciente , Humanos , Doença Crônica/terapia , Criança , FamíliaRESUMO
Purpose of Review: To discuss the potential biological mechanisms between vitamin D and toxic metals and summarize epidemiological studies examining this association in pregnant women. Recent Findings: We identified four plausible mechanisms whereby vitamin D and toxic metals may interact: nephrotoxicity, intestinal absorption of metals, endocrine disruption, and oxidative stress. Few studies have examined the association between vitamin D and toxic metals in pregnant women. North American studies suggest that higher vitamin D status early in pregnancy are associated with lower blood metals later in pregnancy. However, a trial of vitamin D supplementation in a pregnant population, with higher metal exposures and lower overall nutritional status, does not corroborate these findings. Summary: Given ubiquitous exposure to many toxic metals, nutritional intervention could be a means for prevention of adverse outcomes. Future prospective studies are needed to establish a causal relationship and clarify the directionality of vitamin D and metals. Supplementary Information: The online version contains supplementary material available at 10.1007/s40471-024-00348-0.
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Importance: Ultrasonographic measurement of fetal nuchal translucency is used in prenatal screening for trisomies 21 and 18 and other conditions. A cutoff of 3.5 mm or greater is commonly used to offer follow-up investigations, such as prenatal cell-free DNA (cfDNA) screening or cytogenetic testing. Recent studies showed a possible association with chromosomal anomalies for levels less than 3.5 mm, but extant evidence has limitations. Objective: To evaluate the association between different nuchal translucency measurements and cytogenetic outcomes on a population level. Design, Setting, and Participants: This population-based retrospective cohort study used data from the Better Outcomes Registry & Network, the perinatal registry for Ontario, Canada. All singleton pregnancies with an estimated date of delivery from September 1, 2016, to March 31, 2021, were included. Data were analyzed from March 17 to August 14, 2023. Exposures: Nuchal translucency measurements were identified through multiple-marker screening results. Main Outcomes and Measures: Chromosomal anomalies were identified through all Ontario laboratory-generated prenatal and postnatal cytogenetic tests. Cytogenetic testing results, supplemented with information from cfDNA screening and clinical examination at birth, were used to identify pregnancies without chromosomal anomalies. Multivariable modified Poisson regression with robust variance estimation and adjustment for gestational age was used to compare cytogenetic outcomes for pregnancies with varying nuchal translucency measurement categories and a reference group with nuchal translucency less than 2.0 mm. Results: Of 414 268 pregnancies included in the study (mean [SD] maternal age at estimated delivery date, 31.5 [4.7] years), 359â¯807 (86.9%) had a nuchal translucency less than 2.0 mm; the prevalence of chromosomal anomalies in this group was 0.5%. An increased risk of chromosomal anomalies was associated with increasing nuchal translucency measurements, with an adjusted risk ratio (ARR) of 20.33 (95% CI, 17.58-23.52) and adjusted risk difference (ARD) of 9.94% (95% CI, 8.49%-11.39%) for pregnancies with measurements of 3.0 to less than 3.5 mm. The ARR was 4.97 (95% CI, 3.45-7.17) and the ARD was 1.40% (95% CI, 0.77%-2.04%) when restricted to chromosomal anomalies beyond the commonly screened aneuploidies (excluding trisomies 21, 18, and 13 and sex chromosome aneuploidies). Conclusions and Relevance: In this cohort study of 414 268 singleton pregnancies, those with nuchal translucency measurements less than 2.0 mm were at the lowest risk of chromosomal anomalies. Risk increased with increasing measurements, including measurements less than 3.5 mm and anomalies not routinely screened by many prenatal genetic screening programs.
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Ácidos Nucleicos Livres , Síndrome de Down , Recém-Nascido , Feminino , Gravidez , Humanos , Pré-Escolar , Medição da Translucência Nucal , Estudos de Coortes , Estudos Retrospectivos , Trissomia , Aneuploidia , Análise Citogenética , Ontário/epidemiologiaRESUMO
BACKGROUND: Generating rigorous evidence to inform care for rare diseases requires reliable, sustainable, and longitudinal measurement of priority outcomes. Having developed a core outcome set for pediatric medium-chain acyl-CoA dehydrogenase (MCAD) deficiency, we aimed to assess the feasibility of prospective measurement of these core outcomes during routine metabolic clinic visits. METHODS: We used existing cohort data abstracted from charts of 124 children diagnosed with MCAD deficiency who participated in a Canadian study which collected data from birth to a maximum of 11 years of age to investigate the frequency of clinic visits and quality of metabolic chart data for selected outcomes. We recorded all opportunities to collect outcomes from the medical chart as a function of visit rate to the metabolic clinic, by treatment centre and by child age. We applied a data quality framework to evaluate data based on completeness, conformance, and plausibility for four core MCAD outcomes: emergency department use, fasting time, metabolic decompensation, and death. RESULTS: The frequency of metabolic clinic visits decreased with increasing age, from a rate of 2.8 visits per child per year (95% confidence interval, 2.3-3.3) among infants 2 to 6 months, to 1.0 visit per child per year (95% confidence interval, 0.9-1.2) among those ≥ 5 years of age. Rates of emergency department visits followed anticipated trends by child age. Supplemental findings suggested that some emergency visits occur outside of the metabolic care treatment centre but are not captured. Recommended fasting times were updated relatively infrequently in patients' metabolic charts. Episodes of metabolic decompensation were identifiable but required an operational definition based on acute manifestations most commonly recorded in the metabolic chart. Deaths occurred rarely in these patients and quality of mortality data was not evaluated. CONCLUSIONS: Opportunities to record core outcomes at the metabolic clinic occur at least annually for children with MCAD deficiency. Methods to comprehensively capture emergency care received at outside institutions are needed. To reduce substantial heterogeneous recording of core outcome across treatment centres, improved documentation standards are required for recording of recommended fasting times and a consensus definition for metabolic decompensations needs to be developed and implemented.
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Erros Inatos do Metabolismo Lipídico , Avaliação de Resultados em Cuidados de Saúde , Criança , Humanos , Acil-CoA Desidrogenase , Canadá , Estudos Prospectivos , Pré-EscolarRESUMO
BACKGROUND: Despite the critical importance of clinical trials to provide evidence about the effects of intervention for children and youth, a paucity of published high-quality pediatric clinical trials persists. Sub-optimal reporting of key trial elements necessary to critically appraise and synthesize findings is prevalent. To harmonize and provide guidance for reporting in pediatric controlled clinical trial protocols and reports, reporting guideline extensions to the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT) and Consolidated Standards of Reporting Trials (CONSORT) guidelines specific to pediatrics are being developed: SPIRIT-Children (SPIRIT-C) and CONSORT-Children (CONSORT-C). METHODS: The development of SPIRIT-C/CONSORT-C will be informed by the Enhancing the Quality and Transparency of Health Research Quality (EQUATOR) method for reporting guideline development in the following stages: (1) generation of a preliminary list of candidate items, informed by (a) items developed during initial development efforts and child relevant items from recent published SPIRIT and CONSORT extensions; (b) two systematic reviews and environmental scan of the literature; (c) workshops with young people; (2) an international Delphi study, where a wide range of panelists will vote on the inclusion or exclusion of candidate items on a nine-point Likert scale; (3) a consensus meeting to discuss items that have not reached consensus in the Delphi study and to "lock" the checklist items; (4) pilot testing of items and definitions to ensure that they are understandable, useful, and applicable; and (5) a final project meeting to discuss each item in the context of pilot test results. Key partners, including young people (ages 12-24 years) and family caregivers (e.g., parents) with lived experiences with pediatric clinical trials, and individuals with expertise and involvement in pediatric trials will be involved throughout the project. SPIRIT-C/CONSORT-C will be disseminated through publications, academic conferences, and endorsement by pediatric journals and relevant research networks and organizations. DISCUSSION: SPIRIT/CONSORT-C may serve as resources to facilitate comprehensive reporting needed to understand pediatric clinical trial protocols and reports, which may improve transparency within pediatric clinical trials and reduce research waste. TRIAL REGISTRATION: The development of these reporting guidelines is registered with the EQUATOR Network: SPIRIT-Children ( https://www.equator-network.org/library/reporting-guidelines-under-development/reporting-guidelines-under-development-for-clinical-trials-protocols/#35 ) and CONSORT-Children ( https://www.equator-network.org/library/reporting-guidelines-under-development/reporting-guidelines-under-development-for-clinical-trials/#CHILD ).
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Lista de Checagem , Saúde da Criança , Humanos , Criança , Adolescente , Consenso , Projetos de Pesquisa , Padrões de ReferênciaRESUMO
OBJECTIVE: Gestational weight gain (GWG) outside recommended ranges can negatively impact both the woman and child. The long-term effects of below-recommended or above-recommended GWG on the child are unclear. METHODS: This retrospective cohort study used a population-based birth registry of 258,005 live births to evaluate the relationship between maternal GWG and paediatric health service use. RESULTS: The results suggest below recommended GWG in underweight women in particular is associated with an increased rate of hospitalizations and specialist visits for the child in the first 24 months. CONCLUSION: Findings indicate that GWG may impact paediatric outcomes in ways that depend on pre-pregnancy body mass index, as derived from maternal height and weight measures.
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Ganho de Peso na Gestação , Complicações na Gravidez , Gravidez , Pré-Escolar , Feminino , Criança , Humanos , Aumento de Peso , Resultado da Gravidez , Estudos Retrospectivos , Índice de Massa Corporal , Sobrepeso/complicações , Peso ao NascerRESUMO
INTRODUCTION: Reference intervals and reference curves provide clinicians with a point of reference when evaluating patients' laboratory test results. In practical applications, the 2.5th and 97.5th percentiles of healthy reference population are typically used as lower and upper reference limits. Guidelines outlining analytical and methodological steps involved in reference intervals and curves estimation are available and there have been large-scale world-wide initiatives to provide reference intervals and curves for children. However, there is a lack of synthesised evidence regarding the results of such initiatives in general, but specifically in iron-related biomarkers, ferritin (in serum and plasma) and haemoglobin. Objectives of this review are to identify studies that have produced reference intervals and curves for ferritin and haemoglobin in paediatric populations and to synthesise all available evidence. We also aim to quantify heterogeneity across reference intervals and curves and identify and elucidate sources of heterogeneity, including heterogeneity in the methods employed in their development. METHODS AND ANALYSIS: Using a comprehensive search strategy, we will identify eligible studies. Following electronic databases will be searched from inception: EMBASE, MEDLINE, SCOPUS and The Cochrane Library. We will also perform grey literature search to capture unpublished reference intervals and curves from healthy cohorts. Two researchers will independently screen retrieved citations against eligibility criteria in two stages, focusing first on titles and abstracts and then on full-text articles. Studies that provide reference intervals and curves for ferritin and haemoglobin for paediatric population will be eligible. Data extraction will include study characteristics, characteristics of reference population, methodological and analytical considerations and estimated reference intervals and curves. We will consider narrative synthesis and quantitative synthesis when appropriate. ETHICS AND DISSEMINATION: Ethical approval is not required as data from already published studies will be used. Results will be disseminated through peer-reviewed publications and conference presentations. PROSPERO REGISTRATION NUMBER: CRD42023399802.
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Ferritinas , Hemoglobinas , Humanos , Criança , Revisões Sistemáticas como Assunto , Metanálise como Assunto , Revisão por Pares , Projetos de Pesquisa , Literatura de Revisão como AssuntoRESUMO
OBJECTIVES: Children and families are increasingly involved as equal partners in child health research, however, considerations around authorship have received little attention and there is limited guidance on the topic. Our objective was to determine the frequency and nature of patient partner authorship and/or acknowledgment among articles focused on patient engagement in child health research. STUDY DESIGN AND SETTING: In this umbrella review, we searched MEDLINE, Embase, APA PsycINFO, Cochrane Database of Systematic Reviews, CINAHL, and Web of Science for systematic/scoping reviews on patient engagement in child health research. Individual articles included in eligible reviews comprised the sample of articles for analysis and were examined to identify patient partner authorship. Descriptive statistics were used to quantify patient partner authorship and/or acknowledgment and to summarize article characteristics. RESULTS: Twelve systematic/scoping reviews met eligibility criteria, from which 230 individual articles were examined. In 16/230 (7%) articles, there was at least one patient partner author, and in 6/230 (3%) articles, patient partners were included as group authors. Within article Acknowledgments sections, patient partners were acknowledged by name in 41/230 (18%) articles, and anonymously or as a group in 98/230 (43%) articles. Patient partner authorship and/or acknowledgment was more frequent among articles published more recently (after 2015) and among articles where patient engagement was explicitly reported in the article. CONCLUSION: Patient partners were more likely to be acknowledged than listed as an author on articles on patient engagement in child health research. Understanding patient partner preferences about authorship and acknowledgment, examination of the unique aspects of child and youth authorship and developing supports to empower patient partner authorship are needed.
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Autoria , Saúde da Criança , Criança , Humanos , Adolescente , Prevalência , Revisões Sistemáticas como AssuntoRESUMO
BACKGROUND: Pediatric trials are possible through voluntary participation of children, youth (age ≤ 18 years), and their families. Despite important arguments for trialists to provide trial progress or results, and evidence that participants desire it, this information remains rarely shared with youth and their families. Little guidance exists on how trialists can best communicate trial results back to participants and their families. Guided by Liabo et al.'s framework, we describe how we developed a pediatric-specific, "plain language summary" clinical trial results template called CommuniKIDS with an adult patient partner, family partner (parent), youth advisors, and parent advisors, taking into account their unique knowledge needs and preferences. MAIN TEXT: Patient and Public Involvement (PPI) was integrated in the development of the CommuniKIDS template. In collaboration with Clinical Trials Ontario, we used a generic trial results template as a starting point. The core project leadership team included a patient partner and a family partner from project inception to completion. Five youth (ages 13-18 years) and eight parent advisors were consulted at each point of the development process through three virtual workshops conducted separately; youth workshops were led by a youth facilitator. During these workshops, advisors agreed on the importance and value of sharing trial results, and expressed their preferences on content, format, and timing of sharing trial results. PPI-led improvements included the addition of three new sections to the CommuniKIDS template: "at a glance," "side effects," and "next steps." We reflect on our PPI strategy in the context of five "values" and six "practicalities" identified as good PPI principles, and summarize lessons learned when collaborating with youth and families from this project. CONCLUSION: Involvement of a patient partner, a family partner, youth advisors, and parent advisors in the development of CommuniKIDS was critical to create a clinical trial results template that is useful and relevant to its end-users. To our knowledge, CommuniKIDS is the first to meaningfully engage youth and parents as advisors and partners in developing a plain language summary results template for pediatric trial participants and their families. Our experience of co-developing CommuniKIDS demonstrates that meaningful PPI can be achieved in trial results communication and knowledge translation practices. This report provides resources for those seeking to involve youth and families in their initiatives and in meaningfully sharing trial results.
The voluntary participation of youth aged 18 and under in clinical trials makes it possible for researchers and healthcare providers to study medications and other treatments. However, most youth and their families who take part in clinical trials do not get any information on the trial's progress or results, leaving many to wonder if anything useful came from their participation. There is an ethical obligation to give this information back to youth and their families, who might take risks by participating in trials. The aim of the CommuniKIDS project was to develop a "plain language summary" results template to share trial results back to youth and their families. Working with a patient partner, a family partner, five youth advisors (ages 1318), and eight parent advisors, we set out to understand what youth and parents would like to see in a plain language summary of clinical trial results. The needs and preferences discussed with the advisors were included to create a child/youth health-specific template. The CommuniKIDS project is the first to involve youth and parents as advisors in developing a plain language summary results template for child/youth health trials. Here, we describe how we involved youth and parents in the development of CommuniKIDS, how the template was customized to be youth and family-friendly and reflect on lessons learned.
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BACKGROUND: There are few data on patient and public involvement (PPI) in pragmatic trials. We aimed to describe the prevalence and nature of PPI within pragmatic trials, describe variation in prevalence of PPI by trial characteristics and compare prevalence of PPI reported by trial authors to that reported in trial publications. METHODS: We applied a search filter to identify pragmatic trials published from 2014 to 2019 in MEDLINE. We invited the corresponding authors of pragmatic trials to participate in an online survey about their specific trial. RESULTS: Of 3163 authors invited, 2585 invitations were delivered, 710 (27.5%) reported on 710 unique trials and completed the survey; 334 (47.0%) conducted PPI. Among those who conducted PPI, for many the aim was to increase the research relevance (86.3%) or quality (76.5%). Most PPI partners were engaged at protocol development stages (79.1%) and contributed to the co-design of interventions (70.9%) or recruitment or retention strategies (60.5%). Patient and public involvement was more common among trials involving children, trials conducted in the United Kingdom, cluster randomized trials, those explicitly labelled as "pragmatic" in the study manuscript, and more recent trials. Less than one-quarter of trials (22.8%) that reported PPI in the survey also reported PPI in the trial manuscript. INTERPRETATION: Nearly half of trialists in this survey reported conducting PPI and listed several benefits of doing so, but researchers who did not conduct PPI often cited a lack of requirement for it. Patient and public involvement appears to be significantly underreported in trial publications. Consistent and standardized reporting is needed to promote transparency about PPI methods, outcomes, challenges and benefits.
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PURPOSE: Exome and genome sequencing have rapidly transitioned from research methods to widely used clinical tests for diagnosing rare genetic diseases. We sought to synthesize the topics covered and appraise the development processes of clinical guidance documents generated by genetics professional organizations. METHODS: We conducted a scoping review of guidance documents published since 2010, systematically identified in peer-reviewed and gray literature, using established methods and reporting guidelines. We coded verbatim recommendations by topic using content analysis and critically appraised documents using the Appraisal of Guidelines Research and Evaluation (AGREE) II tool. RESULTS: We identified 30 guidance documents produced by 8 organizations (2012-2022), yielding 611 recommendations covering 21 topics. The most common topic related to findings beyond the primary testing indication. Mean AGREE II scores were low across all 6 quality domains; scores for items related to rigor of development were among the lowest. More recently published documents generally received higher scores. CONCLUSION: Guidance documents included a broad range of recommendations but were of low quality, particularly in their rigor of development. Developers should consider using tools such as AGREE II and basing recommendations on living knowledge syntheses to improve guidance development in this evolving space.
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Exoma , Sociedades , Humanos , Exoma/genética , Mapeamento CromossômicoRESUMO
BACKGROUND: Maternal pre-pregnancy body mass index (BMI) and gestational weight gain (GWG) above or below recommendations have been associated with increased paediatric health service utilization as well as increased risk of adverse birth outcomes, including small for gestational age (SGA) and preterm birth (PTB). SGA and PTB are associated with numerous adverse health outcomes in the child, including delayed growth, motor and cognitive impairment. Previous research has identified birth weight and gestational age on the causal pathway in the association between maternal pre-pregnancy BMI and child hospital admissions, there are no studies to date to quantify this relationship across other areas of health service utilization, nor the impact of gestational weight gain. This study aimed to assess if SGA or PTB partially explain the association between maternal weight and paediatric health service utilization. METHODS: The study population consisted of all women who delivered a singleton, live infant in Ontario between 2012 and 2014, and was assembled from data contained in the provincial birth registry. Health service utilization over the first 24 months following birth was examined by linking data from the registry with other provincial health administrative databases housed at ICES. The mediating roles of PTB and SGA were assessed using the Baron-Kenny method and causal mediation analysis. RESULTS: A total of 204,162 infants were included in the analysis of maternal pre-pregnancy BMI and 171,127 infants were included in the GWG analysis. The small magnitude of association between maternal BMI and paediatric health service utilization impacted our ability to estimate the indirect effect of maternal BMI through adverse birth outcomes (adjusted indirect effect = 0.00). 56.7% of the association between below recommended GWG and increased hospitalizations was attributed to PTB, while 6.8% of the association was attributed to SGA. CONCLUSION: Paediatric hospitalizations may be partially attributable to PTB and SGA in children born to mothers with below-recommended GWG. However, maternal weight also appears to be related to increased paediatric health service utilization independent of PTB and SGA.
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Serviços de Saúde da Criança , Ganho de Peso na Gestação , Complicações na Gravidez , Nascimento Prematuro , Humanos , Feminino , Adulto , Gravidez , Recém-Nascido , Lactente , Obesidade , Retardo do Crescimento Fetal , Peso ao Nascer , Índice de Massa Corporal , Recém-Nascido Pequeno para a Idade Gestacional , Estudos RetrospectivosRESUMO
Pediatric anxiety disorders (AD) are prevalent disorders with an impact on all aspects of a child's life and functioning.1 Although evidence supports commonly used treatments, there are notable concerns with the research to date.2 Heterogeneity in outcome selection, measurement, analysis, and reporting is a contributing factor to the hinderance of the translation of research into clinical practice.3 Recognition for outcome standardization in pediatric mental health disorders is evolving and there are several initiatives of importance, including the International Consortium for Health Outcomes Measurement (ICHOM), which has developed standardized outcome sets for use in the routine clinical mental health treatment of children and adolescents.4 Similarly, the International Alliance of Mental Health Research Funders5 advocate for use of 1 specific outcome measurement instrument (OMI) in the youth mental health research that they fund. Development of a Core Outcome Set (COS), a minimal set of outcomes that should be measured and reported in clinical trials, has been a solution in other areas of medicine to address heterogeneity in outcome selection and measurement across trials.6 The Core Outcomes and Measures in Pediatric Anxiety Clinical Trials (COMPACT) Initiative will develop a harmonized, evidence- and consensus-based COS that is meaningful to youth and families for use in future trials in pediatric AD.
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Transtornos de Ansiedade , Projetos de Pesquisa , Adolescente , Humanos , Criança , Técnica Delphi , Determinação de Ponto Final , Transtornos de Ansiedade/terapia , Avaliação de Resultados em Cuidados de Saúde , Resultado do TratamentoRESUMO
OBJECTIVES: Registry-based randomized controlled trials (RRCTs) have potential to address limitations of traditional clinical trials. To describe their current use, information on planned and published RRCTs was identified and synthesized. STUDY DESIGN AND SETTING: A scoping review of published RRCT protocols and reports was conducted. Articles published between 2010 and 2021 identified from electronic database searching, a recent review of RRCTs, and targeted searching for recent RRCT protocols (2018-2021) were screened. Data on trial data sources, types of primary outcomes, and how these primary outcomes were described, selected, and reported were extracted. RESULTS: Ninety RRCT articles (77 reports; 13 protocols) were included. Forty nine (54%) used or planned to rely on registry data for their trial, 26 (29%) used both registry and additional data, and 15 (17%) used the registry solely for recruitment. Primary outcomes were routinely collected from the registry for 66 articles (73%). Only 28 articles (31%) described any methods to promote outcome data quality during or after data collection. Core outcome sets were not used in any of the trials. CONCLUSION: With improvements in registry design, outcome selection, measurement, and reporting, future RRCTs may deliver on promises of efficient, high-quality trials that address clinically relevant questions.
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Dados de Saúde Coletados Rotineiramente , Humanos , Ensaios Clínicos Controlados Aleatórios como Assunto , Sistema de RegistrosRESUMO
OBJECTIVES: Registry-based randomized controlled trials (RRCTs) are increasingly used, promising to address challenges associated with traditional randomized controlled trials. We identified strengths and limitations reported in planned and completed RRCTs to inform future RRCTs. STUDY DESIGN AND SETTING: We conducted an environmental scan of literature discussing conceptual or methodological strengths and limitations of using registries for trial design and conduct (n = 12), followed by an analysis of RRCT protocols (n = 13) and reports (n = 77) identified from a scoping review. Using framework analysis, we developed and refined a conceptual framework of RRCT-specific strengths and limitations. We mapped and interpreted strengths and limitations discussed by authors of RRCT articles using framework codes and quantified the frequencies at which these were mentioned. RESULTS: Our conceptual framework identified six main RRCT strengths and four main RRCT limitations. Considering implications for RRCT conduct and design, we formulated ten recommendations for registry designers, administrators, and trialists planning future RRCTs. CONCLUSION: Consideration and application of empirically underpinned recommendations for future registry design and trial conduct may help trialists utilize registries and RRCTs to their full potential.
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Ensaios Clínicos Controlados Aleatórios como Assunto , Humanos , Sistema de RegistrosRESUMO
BACKGROUND: Toxic metals, like lead, are risk factors for preterm birth (PTB), but few studies have examined low levels found in most Canadians. Vitamin D, which may have antioxidant activity, protects against PTB. OBJECTIVES: In this study, we investigated the impact of toxic metals (lead, mercury, cadmium and arsenic) on PTB and examined if maternal plasma vitamin D concentrations modify these associations. METHODS: We investigated whether concentrations of metals in whole blood measured in early and late pregnancy were associated with PTB (<37 weeks) and spontaneous PTB in 1851 live births from the Maternal-Infant Research on Environmental Chemicals Study using discrete time survival analysis. We also investigated whether the risk of PTB was modified by first-trimester plasma 25-hydroxyvitamin D (25OHD) concentrations. RESULTS: Of 1851 live births, 6.1% (n = 113) were PTBs and 4.9% (n = 89) were spontaneous PTB. A 1 µg/dL increase in blood lead concentrations during pregnancy was associated with an increased risk of PTB (relative risk [RR] 1.48, 95% confidence interval [CI] 1.00, 2.20) and spontaneous PTB (RR 1.71, 95% CI 1.13, 2.60). The risk was higher in women with insufficient vitamin D concentrations (25OHD <50 nmol/L) for both PTB (RR 2.42, 95% CI 1.01, 5.79) and spontaneous PTB (RR 3.04, 95% CI 1.15, 8.04). However, an interaction on the additive scale was not present. Arsenic was associated with a higher risk of PTB (RR 1.10, 95% CI 1.02, 1.19) and spontaneous PTB (RR 1.11, 95% CI 1.03, 1.20) per 1 µg/L. CONCLUSIONS: Gestational exposure to low levels of lead and arsenic may increase the risk of PTB and spontaneous PTB; individuals with insufficient vitamin D may be more susceptible to the adverse effects of lead. Given our relatively small number of cases, we encourage testing of this hypothesis in other cohorts, especially those with vitamin D-deficient populations.
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Arsênio , Nascimento Prematuro , Gravidez , Recém-Nascido , Feminino , Lactente , Humanos , Nascimento Prematuro/epidemiologia , Arsênio/toxicidade , Chumbo/toxicidade , Canadá/epidemiologia , Vitamina D , VitaminasRESUMO
BACKGROUND: Accurate estimates of gestational age (GA) at birth are important for preterm birth surveillance but can be challenging to obtain in low income countries. Our objective was to develop machine learning models to accurately estimate GA shortly after birth using clinical and metabolomic data. METHODS: We derived three GA estimation models using ELASTIC NET multivariable linear regression using metabolomic markers from heel-prick blood samples and clinical data from a retrospective cohort of newborns from Ontario, Canada. We conducted internal model validation in an independent cohort of Ontario newborns, and external validation in heel prick and cord blood sample data collected from newborns from prospective birth cohorts in Lusaka, Zambia and Matlab, Bangladesh. Model performance was measured by comparing model-derived estimates of GA to reference estimates from early pregnancy ultrasound. RESULTS: Samples were collected from 311 newborns from Zambia and 1176 from Bangladesh. The best-performing model accurately estimated GA within about 6 days of ultrasound estimates in both cohorts when applied to heel prick data (MAE 0.79 weeks (95% CI 0.69, 0.90) for Zambia; 0.81 weeks (0.75, 0.86) for Bangladesh), and within about 7 days when applied to cord blood data (1.02 weeks (0.90, 1.15) for Zambia; 0.95 weeks (0.90, 0.99) for Bangladesh). CONCLUSIONS: Algorithms developed in Canada provided accurate estimates of GA when applied to external cohorts from Zambia and Bangladesh. Model performance was superior in heel prick data as compared to cord blood data.
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Traumatismos do Tornozelo , Traumatismos do Joelho , Nascimento Prematuro , Recém-Nascido , Feminino , Gravidez , Humanos , Idade Gestacional , Estudos Prospectivos , Estudos Retrospectivos , Zâmbia , Algoritmos , Aprendizado de Máquina , OntárioRESUMO
PURPOSE: The collection and use of patient reported outcomes (PROs) in care-based child health research raises challenging ethical and logistical questions. This paper offers an analysis of two questions related to PROs in child health research: (1) Is it ethically obligatory, desirable or preferable to share PRO data collected for research with children, families, and health care providers? And if so, (2) What are the characteristics of a model best suited to guide the collection, monitoring, and sharing of these data? METHODS: A multidisciplinary team of researchers, providers, patient and family partners, and ethicists examined the literature and identified a need for focus on PRO sharing in pediatric care-based research. We constructed and analyzed three models for managing pediatric PRO data in care-based research, drawing on ethical principles, logistics, and opportunities to engage with children and families. RESULTS: We argue that it is preferable to share pediatric PRO data with providers, but to manage expectations and balance the risks and benefits of research, this requires a justifiable data sharing model. We argue that a successful PRO data sharing model will allow children and families to have access to and control over their own PRO data and be engaged in decision-making around how PROs collected for research may be integrated into care, but require support from providers. CONCLUSION: We propose a PRO data sharing model that can be used across diverse research settings and contributes to improved transparency, communication, and patient-centered care and research.
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Saúde da Criança , Qualidade de Vida , Criança , Humanos , Qualidade de Vida/psicologia , Disseminação de Informação , Comunicação , Medidas de Resultados Relatados pelo PacienteRESUMO
OBJECTIVE: To identify impacts of patient and family engagement in child health research on the research process, research teams, and patient and family partners. STUDY DESIGN: A scoping review was conducted using the MEDLINE, Embase, Cumulative Index to Nursing and Allied Health Literature, PsycINFO, and Web of Science databases. English-language studies were included if they described ≥1 impact of patient and family engagement on child health research (age <18 years), researchers, or patient and family partners. Data were retrieved by 2 independent extractors. RESULTS: Of the 7688 studies identified, 25 were included in our analysis. Impacts of patient and family engagement were mostly on the research process (n = 24 studies; 96%), 11 (44%) determined impacts on the research team, and 17 (68%) reported impacts on patient and family partners. Less than one-half (n = 11; 44%) had a primary purpose of determining the impact of patient engagement, and no study used a specific evaluation tool. CONCLUSIONS: Patient and family engagement can strengthen the relevance and feasibility of research and empower researchers and patient partners. Measuring and reporting the impact of engagement is rare. Systematic and standardized evaluation of engagement is needed to understand how, when, and why to engage patients and families.
Assuntos
Saúde da Criança , Participação do Paciente , Criança , Humanos , Adolescente , IdiomaRESUMO
BACKGROUND: Accurate estimates of gestational age in pregnancy are important for the provision of optimal care. Although current guidelines generally recommend estimating gestational age via first-trimester ultrasound measurement of crown-rump length, error associated with this method can range from 3 to 8 days of gestation. In pregnancies resulting from assisted reproductive technology, estimated due date can be calculated on the basis of the age of the embryo and the date of embryo transfer, arguably providing the most accurate estimates possible. We have developed and extensively validated statistical models to estimate gestational age postnatally using metabolomic markers from blood samples in combination with clinical and demographic data. These models have shown high accuracy compared with first-trimester ultrasound, the recommended method for estimating gestational age in spontaneous pregnancies. We hypothesized that gestational age derived from date and stage of embryo at transfer in newborns conceived using assisted reproduction therapy would provide the most accurate reference standard possible to evaluate and compare the accuracy of both first-trimester ultrasound and metabolomic model-based gestational dating. OBJECTIVE: This study aimed to validate both first-trimester ultrasound dating and postnatal metabolomic gestational age estimation models against gestational age derived from date and stage of embryo at transfer in a cohort of newborns conceived via assisted reproductive technology, both overall and in important subgroups of interest (preterm birth, small for gestational age, and multiple birth). STUDY DESIGN: This was a retrospective cohort study of infants born in Ontario, Canada between 2015 and 2017 and captured in the provincial birth registry. Spontaneous conceptions were randomly partitioned into a model derivation sample (80%) and a test sample (20%) for model validation. A cohort of assisted conceptions resulting from fresh embryo transfers was derived to evaluate the accuracy of both ultrasound and model-based gestational dating. Postnatal gestational age estimation models were developed with multivariable linear regression using elastic-net regularization. Gestational age estimates from dating ultrasound and from postnatal metabolomic models were compared with date of embryo transfer reference gestational age in the independent test cohorts. Accuracy was quantified by calculating mean absolute error and the square root of mean squared error. RESULTS: Our model derivation cohort included 202,300 spontaneous conceptions, and the testing cohorts included 50,735 spontaneous conceptions and 1924 assisted conceptions. In the assisted conception cohort, first-trimester dating ultrasound was accurate to within approximately ±1.5 days compared with date of embryo transfer reference overall (mean absolute error, 0.21 [95% confidence interval, 0.20-0.23]). When compared with gestational age derived from date of embryo transfer, the metabolomic estimation models were accurate to within approximately ±5 days overall (0.79 [0.76-0.81] weeks). When ultrasound was used as the reference in validating the metabolomic model, the mean absolute error was slightly higher overall (0.81 [0.78-0.84] weeks). In general, the accuracy of gestational age estimates derived from ultrasound or metabolomic models was highest in term infants and lower in preterm and small-for-gestational-age newborns. CONCLUSION: Our findings support the accuracy of ultrasound as a gestational age dating tool. They also support the potential utility of metabolic gestational age dating algorithms in settings where ultrasound or other accurate methods of estimating gestational age are not available because of lack of infrastructure or specialized training (eg, low-income countries). However, the accuracy of metabolomic model-based dating was generally lower than that of ultrasound.