Core Outcome Sets in Child Health

Abstract

ImportanceDeveloping core outcome sets is essential to ensure that results of clinical trials are comparable and useful. A number of core outcome sets in pediatrics have been published, but a comprehensive in-depth understanding of core outcome sets in this field is lacking.ObjectiveTo systematically identify core outcome sets in child health, collate the diseases to which core outcome sets have been applied, describe the methods used for development and stakeholder participation, and evaluate the methodological quality of existing core outcome sets.Evidence ReviewMEDLINE, SCOPUS, Cochrane Library, and CINAHL were searched using relevant search terms, such as clinical trials, core outcome, and children, along with relevant websites, such as Core Outcome Measures in Effectiveness Trials (COMET). Four researchers worked in teams of 2, performed literature screening and data extraction, and evaluated the methodological quality of core outcome sets using the Core Outcome Set–Standards for Development (COS-STAD).FindingsA total of 77 pediatric core outcome sets were identified, mainly developed by organizations or researchers in Europe, North America, and Australia and mostly from the UK (22 [29%]) and the US (22 [29%]). A total of 77 conditions were addressed; the most frequent International Classification of Diseases, 11th Revision category was diseases of the digestive system (14 [18%]). Most of the outcomes in pediatric core outcome sets were unordered (34 [44%]) or presented in custom classifications (29 [38%]). Core outcome sets used 1 or more of 8 development methods; the most frequent combination of methods was systematic review/literature review/scoping review, together with the Delphi approach and consensus for decision-making (10 [14%]). Among the 6 main types of stakeholders, clinical experts were the most frequently involved (74 [100%]), while industry representatives were rarely involved (4 [5%]). Only 6 core outcome sets (8%) met the 12 criteria of COS-STAD.Conclusions and RelevanceFuture quality of pediatric core outcome sets should be improved based on the standards proposed by the COMET initiative, while core outcome sets methodology and reporting standards should be extended to pediatric populations to help improve the quality of core outcome sets in child health. In addition, the COMET outcome taxonomy should also add items applicable to children.

Protocol of a scoping review of outcome domains in dermatology

“…(2) notes and insights will be discussed among the team members, resulting in codes (thematic categories) that formed the basis of a preliminary codebook (list of codes and definitions); (3) the codebook will be used to systematically code the source documents line by line, with each document coded by a minimum of two analysts; ( 4) key themes will be identified and their importance rated to determine the most crucial factors; (5) discrepancies in coding will be resolved during group discussion, and the codebook refined when needed; ( 6) the coding will be done iteratively, with continuous reviews of additional source documents for further refinement of the codebook; (7) data obtained from coding later source documents will be compared with initial ones to identify the point of thematic saturation, where no new themes emerged; (8) following the coding of all documents, a final review will be conducted to ensure no themes were missed; (9) finally, the findings and themes derived from this comprehensive analysis served as the foundation for developing a conceptual model representing the spectrum of outcome domains in dermatology research. 22 The planned start and end dates for the study are January-June 2024.…”

“…The process of developing and implementing COS includes defining both the outcome domains (subdomains) and the instruments ( outcome measurement instruments ) most suitable to measure these 4. The reason for developing COS derives from evidentiary problems that have been documented by the Cochrane Collaboration and other entities that synthesise evidence 5. Specifically, a barrier to developing synthesised evidence such as systematic reviews and meta-analyses is the heterogeneity in outcomes and outcome measurement instruments in clinical trials 6 7.…”

“… 4 The reason for developing COS derives from evidentiary problems that have been documented by the Cochrane Collaboration and other entities that synthesise evidence. 5 Specifically, a barrier to developing synthesised evidence such as systematic reviews and meta-analyses is the heterogeneity in outcomes and outcome measurement instruments in clinical trials. 6 7 Multiple studies may study a similar condition, but each assesses different outcomes, or if they assess similar outcomes, they do so with different outcome measurement instruments.…”

Global research landscape and burden of disease in pediatrics: Identifying the research gaps to set directions for a new general pediatric journal

“…We identified only two purely methodological articles in reviewed issues of all four journals. One was a systematic review to identify core outcome sets for child health published in JAMA Pediatrics 8 and one about the metrics to be used for malnutrition in children affected by HIV published in Global Pediatric Health. 9 Finally, we also reviewed the titles of the 100 most recent preprint articles from the medRxiv repository.…”

Uptake of core outcome sets in pediatric clinical trials: a protocol

“…More than 20 years have passed since the publication of the rst study to guide the selection of outcomes for pediatric clinical trials [4] . Since then, a growing number of pediatric COS have been developed, especially since the Core Outcome Measures in Effectiveness Trials (COMET) Initiative was launched in 2010 to provide more resources.…”