Heterogeneity and Gaps in Reporting Primary Outcomes From Neonatal Trials.

OBJECTIVES: Clear outcome reporting in clinical trials facilitates accurate interpretation and application of findings and improves evidence-informed decision-making. Standardized core outcomes for reporting neonatal trials have been developed, but little is known about how primary outcomes are reported in neonatal trials. Our aim was to identify strengths and weaknesses of primary outcome reporting in recent neonatal trials. METHODS: Neonatal trials including ≥100 participants/arm published between 2015 and 2020 with at least 1 primary outcome from a neonatal core outcome set were eligible. Raters recruited from Cochrane Neonatal were trained to evaluate the trials' primary outcome reporting completeness using relevant items from Consolidated Standards of Reporting Trials 2010 and Consolidated Standards of Reporting Trials-Outcomes 2022 pertaining to the reporting of the definition, selection, measurement, analysis, and interpretation of primary trial outcomes. All trial reports were assessed by 3 raters. Assessments and discrepancies between raters were analyzed. RESULTS: Outcome-reporting evaluations were completed for 36 included neonatal trials by 39 raters. Levels of outcome reporting completeness were highly variable. All trials fully reported the primary outcome measurement domain, statistical methods used to compare treatment groups, and participant flow. Yet, only 28% of trials fully reported on minimal important difference, 24% on outcome data missingness, 66% on blinding of the outcome assessor, and 42% on handling of outcome multiplicity. CONCLUSIONS: Primary outcome reporting in neonatal trials often lacks key information needed for interpretability of results, knowledge synthesis, and evidence-informed decision-making in neonatology. Use of existing outcome-reporting guidelines by trialists, journals, and peer reviewers will enhance transparent reporting of neonatal trials.

Strengthening Reporting of Neonatal Trials.

BACKGROUND AND OBJECTIVES: There is variability in the selection and reporting of outcomes in neonatal trials with key information frequently omitted. This can impact applicability of trial findings to clinicians, families, and caregivers, and impair evidence synthesis. The Neonatal Core Outcomes Set describes outcomes agreed as clinically important that should be assessed in all neonatal trials, and Consolidated Standards of Reporting Trials (CONSORT)-Outcomes 2022 is a new, harmonized, evidence-based reporting guideline for trial outcomes. We reviewed published trials using CONSORT-Outcomes 2022 guidance to identify exemplars of neonatal core outcome reporting to strengthen description of outcomes in future trial publications. METHODS: Neonatal trials including >100 participants per arm published between 2015 to 2020 with a primary outcome included in the Neonatal Core Outcome Set were identified. Primary outcome reporting was reviewed using CONSORT 2010 and CONSORT-Outcomes 2022 guidelines by assessors recruited from Cochrane Neonatal. Examples of clear and complete outcome reporting were identified with verbatim text extracted from trial reports. RESULTS: Thirty-six trials were reviewed by 39 assessors. Examples of good reporting for CONSORT 2010 and CONSORT-Outcomes 2022 criteria were identified and subdivided into 3 outcome categories: "survival," "short-term neonatal complications," and "long-term developmental outcomes" depending on the core outcomes to which they relate. These examples are presented to strengthen future research reporting. CONCLUSIONS: We have identified examples of good trial outcome reporting. These illustrate how important neonatal outcomes should be reported to meet the CONSORT 2010 and CONSORT-Outcomes 2022 guidelines. Emulating these examples will improve the transmission of information relating to outcomes and reduce associated research waste.

Measurement Properties of Patient Reported Outcome Scales: A Systematic Review.

CONTEXT: Recently a standard set for overall pediatric health outcomes in routine care was developed, which includes patient (or proxy) reported outcome measures (PROMs) for global health, cognitive functioning, and self-efficacy. OBJECTIVES: To determine whether the following PROMs have sufficient measurement properties to be used in pediatric routine care: PROMIS Pediatric and Parent Proxy Scale - Global Health 7+2, PROMIS Parent Proxy Short Form - Cognitive Function 7a, and NIH Toolbox Self-Efficacy CAT Ages 13 to 17. DATA SOURCES: Embase, Psych INFO, and Web of Science were searched from year of inception of each PROM to May 25, 2020; Medline to October 24, 2022. STUDY SELECTION: English, full-text peer-reviewed articles that evaluated measurement properties of included PROMs were eligible. DATA EXTRACTION: The COSMIN guideline for systematic reviews was used to appraise eligible studies and synthesize the overall evidence. RESULTS: Screening >4000 titles yielded 4 to 6 eligible empirical studies for each PROM. The PROMIS instruments had sufficient content validity with low-quality evidence and at least low-quality evidence for sufficient structural validity and internal consistency. The NIH Toolbox lacked essential evidence for content validity. LIMITATIONS: Assessments of measurement properties were based on information reported in the included studies; underreporting might have led to less favorable ratings. CONCLUSIONS: The PROMIS instruments assessed in this review measure their intended construct for their targeted age group; clinicians can use these PROMs in pediatric routine care. Additional studies evaluating measurement properties, including content validity, are needed for the NIH Toolbox before it should be recommended for use in clinical practice.

Pediatric core outcome sets had deficiencies and lacked child and family input: A methodological review.

OBJECTIVES: The Core Outcome Set-STAndards for Development (COS-STAD), published in 2017, contains 11 standards (12 criteria) describing minimum design criteria for core outcome set (COS) development. We aimed to identify and appraise all pediatric COS published prior to COS-STAD, and assess methods of child and family involvement in their development. STUDY DESIGN AND SETTING: This methodological review included documents that described the development of pediatric COS up to and including 2017. Reviewers independently assessed each COS against COS-STAD criteria, and methods of involvement were synthesized. RESULTS: A total of 56 pediatric COS were identified, meeting a median of five COS-STAD criteria. Nearly all met criteria on COS scope specification for setting, health condition, and population; 41% met criteria for intervention. Standards were more often met for the involvement of researchers/health professionals (64%) than for patients or their representatives (29%). Few met standards for achieving COS consensus (4-23%). Methods of child and family engagement varied and were limited. CONCLUSION: A large proportion of pediatric COS developed prior to COS-STAD recommendations show gaps in design methodology. Updated and newly developed pediatric COS would benefit from the inclusion of the child and family voice, implementing a priori criteria for COS consensus, and clear reporting.

Guidelines for Reporting Outcomes in Trial Reports: The CONSORT-Outcomes 2022 Extension.

Importance: Clinicians, patients, and policy makers rely on published results from clinical trials to help make evidence-informed decisions. To critically evaluate and use trial results, readers require complete and transparent information regarding what was planned, done, and found. Specific and harmonized guidance as to what outcome-specific information should be reported in publications of clinical trials is needed to reduce deficient reporting practices that obscure issues with outcome selection, assessment, and analysis. Objective: To develop harmonized, evidence- and consensus-based standards for reporting outcomes in clinical trial reports through integration with the Consolidated Standards of Reporting Trials (CONSORT) 2010 statement. Evidence Review: Using the Enhancing the Quality and Transparency of Health Research (EQUATOR) methodological framework, the CONSORT-Outcomes 2022 extension of the CONSORT 2010 statement was developed by (1) generation and evaluation of candidate outcome reporting items via consultation with experts and a scoping review of existing guidance for reporting trial outcomes (published within the 10 years prior to March 19, 2018) identified through expert solicitation, electronic database searches of MEDLINE and the Cochrane Methodology Register, gray literature searches, and reference list searches; (2) a 3-round international Delphi voting process (November 2018-February 2019) completed by 124 panelists from 22 countries to rate and identify additional items; and (3) an in-person consensus meeting (April 9-10, 2019) attended by 25 panelists to identify essential items for the reporting of outcomes in clinical trial reports. Findings: The scoping review and consultation with experts identified 128 recommendations relevant to reporting outcomes in trial reports, the majority (83%) of which were not included in the CONSORT 2010 statement. All recommendations were consolidated into 64 items for Delphi voting; after the Delphi survey process, 30 items met criteria for further evaluation at the consensus meeting and possible inclusion in the CONSORT-Outcomes 2022 extension. The discussions during and after the consensus meeting yielded 17 items that elaborate on the CONSORT 2010 statement checklist items and are related to completely defining and justifying the trial outcomes, including how and when they were assessed (CONSORT 2010 statement checklist item 6a), defining and justifying the target difference between treatment groups during sample size calculations (CONSORT 2010 statement checklist item 7a), describing the statistical methods used to compare groups for the primary and secondary outcomes (CONSORT 2010 statement checklist item 12a), and describing the prespecified analyses and any outcome analyses not prespecified (CONSORT 2010 statement checklist item 18). Conclusions and Relevance: This CONSORT-Outcomes 2022 extension of the CONSORT 2010 statement provides 17 outcome-specific items that should be addressed in all published clinical trial reports and may help increase trial utility, replicability, and transparency and may minimize the risk of selective nonreporting of trial results.

Guidelines for Reporting Outcomes in Trial Protocols: The SPIRIT-Outcomes 2022 Extension.

Importance: Complete information in a trial protocol regarding study outcomes is crucial for obtaining regulatory approvals, ensuring standardized trial conduct, reducing research waste, and providing transparency of methods to facilitate trial replication, critical appraisal, accurate reporting and interpretation of trial results, and knowledge synthesis. However, recommendations on what outcome-specific information should be included are diverse and inconsistent. To improve reporting practices promoting transparent and reproducible outcome selection, assessment, and analysis, a need for specific and harmonized guidance as to what outcome-specific information should be addressed in clinical trial protocols exists. Objective: To develop harmonized, evidence- and consensus-based standards for describing outcomes in clinical trial protocols through integration with the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT) 2013 statement. Evidence Review: Using the Enhancing the Quality and Transparency of Health Research (EQUATOR) methodological framework, the SPIRIT-Outcomes 2022 extension of the SPIRIT 2013 statement was developed by (1) generation and evaluation of candidate outcome reporting items via consultation with experts and a scoping review of existing guidance for reporting trial outcomes (published within the 10 years prior to March 19, 2018) identified through expert solicitation, electronic database searches of MEDLINE and the Cochrane Methodology Register, gray literature searches, and reference list searches; (2) a 3-round international Delphi voting process (November 2018-February 2019) completed by 124 panelists from 22 countries to rate and identify additional items; and (3) an in-person consensus meeting (April 9-10, 2019) attended by 25 panelists to identify essential items for outcome-specific reporting to be addressed in clinical trial protocols. Findings: The scoping review and consultation with experts identified 108 recommendations relevant to outcome-specific reporting to be addressed in trial protocols, the majority (72%) of which were not included in the SPIRIT 2013 statement. All recommendations were consolidated into 56 items for Delphi voting; after the Delphi survey process, 19 items met criteria for further evaluation at the consensus meeting and possible inclusion in the SPIRIT-Outcomes 2022 extension. The discussions during and after the consensus meeting yielded 9 items that elaborate on the SPIRIT 2013 statement checklist items and are related to completely defining and justifying the choice of primary, secondary, and other outcomes (SPIRIT 2013 statement checklist item 12) prospectively in the trial protocol, defining and justifying the target difference between treatment groups for the primary outcome used in the sample size calculations (SPIRIT 2013 statement checklist item 14), describing the responsiveness of the study instruments used to assess the outcome and providing details on the outcome assessors (SPIRIT 2013 statement checklist item 18a), and describing any planned methods to account for multiplicity relating to the analyses or interpretation of the results (SPIRIT 2013 statement checklist item 20a). Conclusions and Relevance: This SPIRIT-Outcomes 2022 extension of the SPIRIT 2013 statement provides 9 outcome-specific items that should be addressed in all trial protocols and may help increase trial utility, replicability, and transparency and may minimize the risk of selective nonreporting of trial results.

Opioid dose and postoperative respiratory adverse events after adenotonsillectomy in medically complex children.

STUDY OBJECTIVES: Obstructive sleep-disordered breathing is commonly treated with adenotonsillectomy. Our study objective was to describe perioperative opioid dosing in children with a range of medical complexity evaluated for obstructive sleep-disordered breathing undergoing adenotonsillectomy and to investigate its association with postoperative respiratory adverse events (PRAEs). METHODS: A retrospective chart review of children who underwent adenotonsillectomy and had preoperative polysomnography performed was conducted. PRAEs included requiring oxygen, jaw thrust, positive airway pressure, or mechanical ventilation. Multivariable logistic regression was performed to examine for associations between covariates and PRAEs. RESULTS: The cohort included 374 children with obstructive sleep-disordered breathing, median (interquartile range) age 6.1 (3.9, 9.3) years; 344 (92%) had obstructive sleep apnea (apnea-hypopnea index > 1 events/h) while 30 (8%) had a normal polysomnogram (apnea-hypopnea index < 1 events/h). The median (interquartile range) postoperative morphine-equivalent dose administered was 0.17 (0.09, 0.25) mg/kg. Sixty-six (17.6%) experienced at least 1 PRAE. Multivariable modeling identified the following predictors of PRAE: younger age at surgery (odds ratio 0.90, 95% confidence interval 0.83, 0.98), presence of cardiac comorbidity (odds ratio 2.07, 95% confidence interval 1.09, 3.89), and presence of airway anomaly (odds ratio 3.48, 95% confidence interval 1.30, 8.94). Higher total apnea-hypopnea index and morphine-equivalent dose were associated with PRAE risk, and an interaction between these variables was detected (P = .01). CONCLUSIONS: This study identified opioid dose in morphine equivalents to be a strong predictor of PRAE. Additionally, severity of obstructive sleep apnea and postoperative morphine-equivalent dose contributed together and independently to the occurrence of PRAEs. Attention to opioid dosing, particularly among medically complex children with obstructive sleep-disordered breathing, is required to mitigate risk of PRAEs. CITATION: Tsampalieros A, Murto K, Barrowman N, et al. Opioid dose and postoperative respiratory adverse events after adenotonsillectomy in medically complex children. J Clin Sleep Med. 2022;18(10):2405-2413.

Systematic Review: The Measurement Properties of the Children's Depression Rating Scale-Revised in Adolescents With Major Depressive Disorder.

OBJECTIVE: To systematically appraise existing evidence of the measurement properties of the Children's Depression Rating Scale-Revised (CDRS-R) in adolescents with major depressive disorder (MDD). The CDRS-R is the most commonly used scale in adolescent depression research, yet was originally designed for use in children 6 to 12 years old. METHOD: Seven databases were searched for studies that evaluated the measurement properties of the CDRS-R in adolescents (ages 12-18 years). Of 65 studies screened by full-text, 6 were included. Measurement properties were appraised using the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guidelines. The COSMIN minimum requirements for recommending the use of an outcome measurement instrument are (1) evidence for sufficient content validity (any level of evidence), and (2) at least low-quality evidence for sufficient internal consistency. RESULTS: Four studies assessed an English-language version of the CDRS-R; the other 2 assessed German and Korean versions, respectively. No study assessed content validity, cross-cultural validity/measurement invariance, or measurement error of the CDRS-R in adolescents with MDD. Low-quality evidence was found for sufficient construct validity (n = 4 studies) and responsiveness (n = 2 studies) assessed via comparator instruments. Very-low-quality evidence was found for sufficient interrater reliability (n = 2 studies). The results for structural validity (n = 3 studies) and internal consistency (n = 5 studies) were inconclusive. CONCLUSION: It remains unclear whether the CDRS-R appropriately measures depressive symptom severity in adolescent MDD. Before use of the CDRS-R in adolescent MDD research can be recommended, evidence of sufficient psychometric properties in adolescents with MDD is needed.

Outcome reporting recommendations for clinical trial protocols and reports: a scoping review.

BACKGROUND: Clinicians, patients, and policy-makers rely on published evidence from clinical trials to help inform decision-making. A lack of complete and transparent reporting of the investigated trial outcomes limits reproducibility of results and knowledge synthesis efforts, and contributes to outcome switching and other reporting biases. Outcome-specific extensions for the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT-Outcomes) and Consolidated Standards of Reporting Trials (CONSORT-Outcomes) reporting guidelines are under development to facilitate harmonized reporting of outcomes in trial protocols and reports. The aim of this review was to identify and synthesize existing guidance for trial outcome reporting to inform extension development. METHODS: We searched for documents published in the last 10 years that provided guidance on trial outcome reporting using: an electronic bibliographic database search (MEDLINE and the Cochrane Methodology Register); a grey literature search; and solicitation of colleagues using a snowballing approach. Two reviewers completed title and abstract screening, full-text screening, and data charting after training. Extracted trial outcome reporting guidance was compared with candidate reporting items to support, refute, or refine the items and to assess the need for the development of additional items. RESULTS: In total, 1758 trial outcome reporting recommendations were identified within 244 eligible documents. The majority of documents were published by academic journals (72%). Comparison of each recommendation with the initial list of 70 candidate items led to the development of an additional 62 items, producing 132 candidate items. The items encompassed outcome selection, definition, measurement, analysis, interpretation, and reporting of modifications between trial documents. The total number of documents supporting each candidate item ranged widely (median 5, range 0-84 documents per item), illustrating heterogeneity in the recommendations currently available for outcome reporting across a large and diverse sample of sources. CONCLUSIONS: Outcome reporting guidance for clinical trial protocols and reports lacks consistency and is spread across a large number of sources that may be challenging to access and implement in practice. Evidence and consensus-based guidance, currently in development (SPIRIT-Outcomes and CONSORT-Outcomes), may help authors adequately describe trial outcomes in protocols and reports transparently and completely to help reduce avoidable research waste.

Systematic scoping review identifies heterogeneity in outcomes measured in adolescent depression clinical trials.

OBJECTIVES: The objective of this review was to identify outcomes reported in adolescent major depressive disorder trials and quantify outcome heterogeneity. STUDY DESIGN AND SETTING: Three databases were searched to identify trials evaluating therapies for major depressive disorder in adolescents published from 2008 to 2017. Identified outcomes were thematically grouped and mapped into predefined outcome core areas (physiological/clinical, life impact, resource use, adverse events, and death). Outcome heterogeneity was quantified using descriptive analyses. RESULTS: Of 2,686 articles yielded from the search, 42 articles describing 32 trials were included. A total of 434 outcomes measured using 118 different outcome measurement instruments were grouped into 86 unique outcome terms. Most outcome terms mapped to the physiological/clinical core area (62%), followed by the life impact (27%). Nearly half (45%) were reported in only a single trial each. Of 18 primary outcomes reported, 13 (72%) were each only reported in a single trial. "Depressive symptom severity", reported in 30 trials (94%), was measured using 19 different outcome measurement instruments. CONCLUSION: Heterogeneity exists in the outcomes and outcome measurement instruments used in adolescent depression trials. To enable reproducibility, comparison, and synthesis of trial results, a standard set of agreed-on outcomes and methods of measurement is needed.

Primary outcome reporting in adolescent depression clinical trials needs standardization.

BACKGROUND: Evidence-based health care is informed by results of randomized clinical trials (RCTs) and their syntheses in meta-analyses. When the trial outcomes measured are not clearly described in trial publications, knowledge synthesis, translation, and decision-making may be impeded. While heterogeneity in outcomes measured in adolescent major depressive disorder (MDD) RCTs has been described, the comprehensiveness of outcome reporting is unknown. This study aimed to assess the reporting of primary outcomes in RCTs evaluating treatments for adolescent MDD. METHODS: RCTs evaluating treatment interventions in adolescents with a diagnosis of MDD published between 2008 and 2017 specifying a single primary outcome were eligible for outcome reporting assessment. Outcome reporting assessment was done independently in duplicate using a comprehensive checklist of 58 reporting items. Primary outcome information provided in each RCT publication was scored as "fully reported", "partially reported", or "not reported" for each checklist item, as applicable. RESULTS: Eighteen of 42 identified articles were found to have a discernable single primary outcome and were included for outcome reporting assessment. Most trials (72%) did not fully report on over half of the 58 checklist items. Items describing masking of outcome assessors, timing and frequency of outcome assessment, and outcome analyses were fully reported in over 70% of trials. Items less frequently reported included outcome measurement instrument properties (ranging from 6 to 17%), justification of timing and frequency of outcome assessment (6%), and justification of criteria used for clinically significant differences (17%). The overall comprehensiveness of reporting appeared stable over time. CONCLUSIONS: Heterogeneous reporting exists in published adolescent MDD RCTs, with frequent omissions of key details about their primary outcomes. These omissions may impair interpretability, replicability, and synthesis of RCTs that inform clinical guidelines and decision-making in this field. Consensus on the minimal criteria for outcome reporting in adolescent MDD RCTs is needed.

Predictors of postoperative respiratory complications in children undergoing adenotonsillectomy.

STUDY OBJECTIVES: Obstructive sleep apnea (OSA) is commonly treated with adenotonsillectomy (AT), bringing risk of perioperative respiratory adverse events (PRAEs). We aimed to concurrently identify clinical and polysomnographic predictors of PRAEs in children undergoing AT. METHODS: Retrospective study of children undergoing AT at a tertiary-care pediatric hospital, with prior in-hospital polysomnography, January 2010 to December 2016. PRAEs included those requiring oxygen, jaw thrust, positive airway pressure, or mechanical ventilation. Relationships of PRAEs to preoperative comorbidities or polysomnography results were examined with univariable logistic regression. Variables with P < .1 and age were included in backward stepwise multivariable logistic regression. Predictive performance (area under the curve, AUC) was validated with bootstrap resampling. RESULTS: Analysis included 374 children, median age 6.1 years; 286 (76.5%) had ≥ 1 comorbidity. 344 (92.0%) had sleep-disordered breathing; 232 (62.0%) moderate-severe; 66 (17.6%) had ≥ 1 PRAE. PRAEs were more frequent in children with craniofacial, genetic, cardiac, airway anomaly, or neurological conditions, AHI ≥ 5 events/h and oxygen saturation nadir ≤ 80% on preoperative polysomnography. Prediction modeling identified cardiac comorbidity (odds ratio [OR] 2.09 [1.11, 3.89]), airway anomaly (OR 3.19 [1.33, 7.49]), and younger age (OR < 3 years: 4.10 (1.79, 9.26; 3 to 6 years: 2.21 [1.18, 4.15]) were associated with PRAEs (AUC 0.74; corrected AUC 0.68). CONCLUSIONS: Prediction modeling concurrently evaluating comorbidities and polysomnography metrics identified cardiac disease, airway anomaly, and young age as independent predictors of PRAEs. These findings suggest that medical comorbidity and age are more important factors in predicting PRAEs than PSG metrics in a medically complex population.

Core outcome set for children with neurological impairment and tube feeding.

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.

CONJUNTO BÁSICO DE RESULTADOS PARA NIÑOS CON DETERIORO NEUROLÓGICO Y SONDA DE ALIMENTACIÓN: OBJETIVO: Desarrollar un conjunto básico de resultados (COS) para evaluar el impacto de la sonda de gastrostomía/gastro-yeyunostomía en niños con discapacidad neurológica. MÉTODO: Los proveedores/investigadores y cuidadores de salud calificaron la importancia de los resultados de los candidatos en una escala Likert de 5 puntos. Los resultados fueron calificados como "algo importantes" o "muy importantes" por la mayoría de los encuestados (85%) quienes votaron durante una reunión de consenso. Los resultados que llegaron a un consenso para la inclusión fueron ratificados y asignados a las medidas de resultado en las áreas centrales del filtro de reumatología. El COS fue validado en un grupo separado de cuidadores. RESULTADOS: Doce resultados fueron seleccionados de 120 candidatos para formar el COS. Estos incluyeron cinco resultados de "Impacto en la vida", tres resultados de "Manifestaciones patológicas", dos resultados de "uso de recursos", un resultado de "Crecimiento y desarrollo" y un resultado de "Muerte". INTERPRETACIÓN: Desarrollamos un COS basado en evidencia y basado en el consenso para su uso en estudios de sonda de alimentación por gastrostomía/gastro yeyunostomía en niños con discapacidad neurológica. La implementación de este COS ayudará a reducir la heterogeneidad entre los estudios y facilitará la toma de decisiones basadas en la evidencia.

ITENS PRINCIPAIS PARA CRIANÇAS COM DEFICIÊNCIA NEUROLÓGICA E TUBO DE ALIMENTAÇÃO: OBJETIVO: Desenvolver um conjunto de itens principais (CIP) para avaliar o impacto do tubo de gastrostomia/gastrojejunostomia em crianças com deficiência neurológica. MÉTODO: Pesquisadores, profissionais da saúde, e cuidadores pontuaram a importância dos desfechos candidatos em uma escala Likert de 5 pontos. Os desfechos pontuados como "algo importante"ou "muito importante" pela maioria '(≥85%) dos respondentes foram votados durante um encontro para consenso. Os desfechos que obtiveram consenso foram ratificados e incluídos no filtro de itens principais das Medidas de Resultados em Reumatologia. O CIP foi validado em um grupo separado de cuidadores. RESULTADOS: Doze resultados foram selecionados a partir de 120 resultados candidatos para formar o CIP. Estes incluíram cinco resultados de "Impacto na vida", três de Manifestações Patofisiológicas, um de "Crescimento e Desenvolvimento", e um sobre "Morte". INTERPRETAÇÃO: Desenvolvemos um CIP baseado em evidência e baseado em consenso para uso em estudos de alimentação por tubo de gastrostomia/gastrojejunostomia em crianças com deficiência neurológica. A implementation deste CIP irá ajudar a reduzir a heterogeneidade entre estudos e facilitar a tomada de decisões baseada em evidências.

Treatment and persistence/recurrence of sleep-disordered breathing in children with Down syndrome.

OBJECTIVE: Sleep-disordered breathing (SDB) is common in children with Down syndrome, but the trajectory and long-term outcomes are not well-described. In a retrospective longitudinal cohort of children with Down syndrome, study objectives were to (1) characterize polysomnography (PSG), treatments received, and persistence/recurrence of SDB and (2) explore predictors of SDB persistence/recurrence. METHODS: A retrospective cohort study was conducted of children who underwent PSGs between 2004 and 2014. SDB was defined as obstructive sleep apnea (OSA)-mixed (apnea-hypopnea index [AHI] >5 events/hour), central sleep apnea or hypoventilation. PSGs, interventions, and trajectory of SDB were described. Age, body mass index (BMI) Z-score and AHI at first SDB diagnosis were evaluated as predictors of persistent/recurrent SDB. RESULTS: Of 506 children, 120 had ≥1 PSG; 54 had subsequent PSGs. Children with ≥2 PSGs were more likely to have higher total AHI (P = .02) and obstructive-mixed AHI (P = .01). Thirty-five of fifty-four (65%) were initially diagnosed with OSA-mixed SDB. After first PSG, 67 of 120 had OSA-mixed SDB, of whom 25 (37.3%) underwent adenotonsillectomy (T&A), 13 (19.4%) received positive airway pressure (PAP). Those who underwent T&A after PSG were significantly younger than those who received PAP (median age 6.2 vs 12.5 years; P = .005). OSA-mixed SDB persisted/recurred in 33 of 54 (73.3%) with ≥2 PSGs. Persistence/recurrence was not associated with age, AHI or BMI Z-score at first SDB. CONCLUSION: Children with Down syndrome undergoing T&A for SDB were significantly younger than those treated with PAP. SDB persisted/recurred in three of four and was not predicted by age, SDB severity or BMI Z-score. Longitudinal PSG assessment for persistence/recurrence of SDB is required in this population.

Improving outcome reporting in clinical trial reports and protocols: study protocol for the Instrument for reporting Planned Endpoints in Clinical Trials (InsPECT).

BACKGROUND: Inadequate and poor quality outcome reporting in clinical trials is a well-documented problem that impedes the ability of researchers to evaluate, replicate, synthesize, and build upon study findings and impacts evidence-based decision-making by patients, clinicians, and policy-makers. To facilitate harmonized and transparent reporting of outcomes in trial protocols and published reports, the Instrument for reporting Planned Endpoints in Clinical Trials (InsPECT) is being developed. The final product will provide unique InsPECT extensions to the SPIRIT (Standard Protocol Items: Recommendations for Interventional Trials) and CONSORT (Consolidated Standards of Reporting Trials) reporting guidelines. METHODS: The InsPECT SPIRIT and CONSORT extensions will be developed in accordance with the methodological framework created by the EQUATOR (Enhancing the Quality and Transparency of Health Research Quality) Network for reporting guideline development. Development will consist of (1) the creation of an initial list of candidate outcome reporting items synthesized from expert consultations and a scoping review of existing guidance for reporting outcomes in trial protocols and reports; (2) a three-round international Delphi study to identify additional candidate items and assess candidate item importance on a 9-point Likert scale, completed by stakeholders such as trial report and protocol authors, systematic review authors, biostatisticians and epidemiologists, reporting guideline developers, clinicians, journal editors, and research ethics board representatives; and (3) an in-person expert consensus meeting to finalize the set of essential outcome reporting items for trial protocols and reports, respectively. The consensus meeting discussions will be independently facilitated and informed by the empirical evidence identified in the primary literature and through the opinions (aggregate rankings and comments) collected via the Delphi study. An integrated knowledge translation approach will be used throughout InsPECT development to facilitate implementation and dissemination, in addition to standard post-development activities. DISCUSSION: InsPECT will provide evidence-informed and consensus-based standards focused on outcome reporting in clinical trials that can be applied across diverse disease areas, study populations, and outcomes. InsPECT will support the standardization of trial outcome reporting, which will maximize trial usability, reduce bias, foster trial replication, improve trial design and execution, and ultimately reduce research waste and help improve patient outcomes.

Outcomes reported in randomised clinical trials of major depressive disorder treatments in adolescents: a systematic scoping review protocol.

INTRODUCTION: Major depressive disorder (MDD) is a common mental health condition in adolescents. Randomised clinical trials (RCTs) are the gold standard for assessing the safety and efficacy of interventions in this population. Heterogeneity in the outcomes measured and reported between RCTs limits the ability to compare, contrast, and combine trial results in a clinically meaningful way. There is currently no core outcome set (COS) available for use in RCTs evaluating interventions in adolescents with MDD. We will conduct a systematic scoping review of outcomes reported in adolescent depression RCTs to assess the variability of trial outcomes and to inform the development of a COS for adolescent MDD. METHODS AND ANALYSIS: We will apply methods based on the Joanna Briggs Institute scoping review methods manual. RCTs evaluating any treatment intervention for adolescent MDD published in the last 10 years will be located using an electronic bibliographic database search (MEDLINE, PsycINFO and Cochrane Central Register of Controlled Trials). Title and abstract screening, full-text screening, and data charting of eligible studies will be performed in duplicate. Outcomes identified will be mapped to an outcome-domain framework. Data analysis will include summary statistics of the characteristics of the included trials and outcomes. ETHICS AND DISSEMINATION: The results of this review will inform the development of a COS for adolescent MDD. The development and implementation of a COS for RCTs evaluating interventions in adolescents with MDD promise to help reduce variability in trial outcome selection, definition, measurement and reporting, ultimately facilitating evidence synthesis that will help to identify the best treatment practices for adolescents with MDD.

Guidance for reporting outcomes in clinical trials: scoping review protocol.

INTRODUCTION: Patients, families and clinicians rely on published research to help inform treatment decisions. Without complete reporting of the outcomes studied, evidence-based clinical and policy decisions are limited and researchers cannot synthesise, replicate or build on existing research findings. To facilitate harmonised reporting of outcomes in published trial protocols and reports, the Instrument for reporting Planned Endpoints in Clinical Trials (InsPECT) is under development. As one of the initial steps in the development of InsPECT, a scoping review will identify and synthesise existing guidance on the reporting of trial outcomes. METHODS AND ANALYSIS: We will apply methods based on the Joanna Briggs Institute scoping review methods manual. Documents that provide explicit guidance on trial outcome reporting will be searched for using: (1) an electronic bibliographic database search; (2) a grey literature search; and (3) solicitation of colleagues for guidance documents using a snowballing approach. Reference list screening will be performed for included documents. Search results will be divided between two trained reviewers who will complete title and abstract screening, full-text screening and data charting. Captured trial outcome reporting guidance will be compared with candidate InsPECT items to support, refute or refine InsPECT content and to assess the need for the development of additional items. Data analysis will explore common features of guidance and use quantitative measures (eg, frequencies) to characterise guidance and its sources. ETHICS AND DISSEMINATION: A paper describing the review findings will be published in a peer-reviewed journal. The results will be used to inform the InsPECT development process, helping to ensure that InsPECT provides an evidence-based tool for standardising trial outcome reporting.

Learning best-practices in journalology: course description and attendee insights into the inaugural EQUATOR Canada Publication School.

BACKGROUND AND PURPOSE: Dissemination of research results is a key component of the research continuum and is commonly achieved through publication in peer-reviewed academic journals. However, issues of poor quality reporting in the research literature are well documented. A lack of formal training in journalology (i.e., publication science) may contribute to this problem. To help address this gap in training, the Enhancing the QUAlity and Transparency Of health Research (EQUATOR) Canada Publication School was developed and facilitated by internationally-renowned faculty to train researchers and clinicians in reporting and publication best practices. This article describes the structure of the inaugural course and provides an overview of attendee evaluations and perspectives. KEY HIGHLIGHTS: Attendees perceived the content of this two-day intensive course as highly informative. They noted that the course helped them learn skills that were relevant to academic publishing (e.g., using reporting guidelines in all phases of the research process; using scholarly metrics beyond the journal impact factor; open-access publication models; and engaging patients in the research process). The course provided an opportunity for researchers to share their challenges faced during the publication process and to learn skills for improving reproducibility, completeness, transparency, and dissemination of research results. There was some suggestion that this type of course should be offered and integrated into formal training and course curricula. IMPLICATIONS: In light of the importance of academic publishing in the scientific process, there is a need to train and prepare researchers with skills in Journalology. The EQUATOR Canada Publication School provides an example of a successful program that addressed the needs of researchers across career trajectories and provided them with resources to be successful in the publication process. This approach can be used, modified, and/or adapted by curriculum developers interested in designing similar programs, and could be incorporated into academic and clinical research training programs.