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.

Psychometric properties of the PSWQ in a sample of pregnant and postpartum women.

BACKGROUND: Generalized anxiety disorder (GAD)-characterised by excessive and uncontrollable worry-is the most frequently diagnosed anxiety disorder during pregnancy and the postpartum period. Identification of GAD often relies on assessment of its cardinal feature, pathological worry. The Penn State Worry Questionnaire (PSWQ) is the most robust measure of pathological worry to date but has not been extensively evaluated for use during pregnancy and the postpartum period. This study evaluated the internal consistency, construct validity, and diagnostic accuracy of the PSWQ in a sample of pregnant and postpartum women with and without a principal GAD diagnosis. METHODS: One hundred forty-two pregnant and 209 postpartum women participated in this study. Sixty-nine pregnant and 129 postpartum participants met criteria for a principal diagnosis of GAD. RESULTS: The PSWQ demonstrated good internal consistency and converged with measures assessing similar constructs. Pregnant participants with principal GAD scored significantly higher on the PSWQ than those with no psychopathology and postpartum participants with principal GAD scored significantly higher than those with principal mood disorders, other anxiety and related disorders, and no psychopathology. A cut-off score of 55 and 61 or greater was determined for detecting probable GAD during pregnancy and the postpartum period, respectively. Screening accuracy of the PSWQ was also demonstrated. CONCLUSIONS: This study underscores the robustness of the PSWQ as a measure of pathological worry and probable GAD and supports its use in the detection and monitoring of clinically significant worry symptoms during pregnancy and postpartum period.

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.

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.