Systematic review of outcome measures in pediatric eosinophilic esophagitis treatment trials

Tamar Rubin,Rabin Persad,Jacqueline Clayton,Sunita Vohra,Denise Adams

doi:10.1186/s13223-016-0144-y

Tamar Rubin, Rabin Persad + Show 3 more

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https://doi.org/10.1186/s13223-016-0144-y

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Abstract

BackgroundHeterogeneity has been noted in the selection and reporting of disease-specific, pediatric outcomes in randomized controlled trials (RCTs). The consequence is invalid results or difficulty comparing results across trials. The primary objective of this systematic review was to assess primary outcome and outcome measure selection and reporting, in pediatric eosinophilic esophagitis (EoE) treatment trials. As secondary objectives, we compared trial disease definition to established concensus guidelines, and the efficacy of current EoE treatments.MethodsWe searched MEDLINE, EMBASE, The Cochrane Library, Cochrane Central Register of Controlled Trials (CENTRAL), and CINAHL since 2001. We also searched clinical trial registries (portal.nihr.ac.uk; clinicaltrials.gov; isrctn.com; and anzctr.org.au) and references of included studies. We included RCTs of EoE treatment in patients 0–18 years. Two authors independently assessed articles.ResultsEleven studies met inclusion criteria. All identified primary outcomes, however, of 9 unique primary outcomes, only 2 were used in more than one study. In total, 25 unique primary and secondary outcome measures were employed for pediatric EoE treatment trials. Measurement properties and rationale for their selection was rarely provided. Uptake of consensus-based diagnostic criteria was 25 % in trials initiated after 2011. Due to the small number and heterogeneity of studies obtained, no meta-analysis of treatment efficacy could be undertaken. This SR was limited to exclusively pediatric RCTs.ConclusionsThe results of this study confirm the need for a standardized set of core outcomes that are universally reported in pediatric EoE trials. Consistent disease definition and standardized outcome reporting will facilitate meta-analyses across similar trials and inform future clinical decision-making.Systematic review registration number CRD42013003798

Highlights

Heterogeneity has been noted in the selection and reporting of disease-specific, pediatric outcomes in randomized controlled trials (RCTs)
No studies were published since the original consensus report that could allow diagnosis based on a pathognomonic clinical/histologic feature or biomarker. This systematic review (SR) assessed the heterogeneity of outcome measure selection and reporting in exclusively pediatric eosinophilic esophagitis (EoE) treatment RCTs
While this review demonstrated heterogeneity in outcome measures and disease definition, this was not the focus of the review and these issues were not investigated thoroughly

Summary

Introduction

Heterogeneity has been noted in the selection and reporting of disease-specific, pediatric outcomes in randomized controlled trials (RCTs). Rubin et al Allergy Asthma Clin Immunol (2016) 12:45 reporting is well accepted as a significant impediment to knowledge translation and meta-analysis [7] To this end, initiatives such as the Consolidated Standards of Reporting Trials (CONSORT) have been established to help promote transparent and complete reporting [1, 8]. In order to facilitate outcome measure selection, the consensus-based standards for the selection of health measurement instruments (COSMIN) group developed an international consensus on the terminology and definitions of measurement properties [9]. They identified three domains of measurement properties: reliability, validity, and responsiveness. Other international initiatives aiming to improve selection and reporting of outcome measures include the COMET initiative (Core Outcome Measures in Effectiveness Trials), which is an initiative to develop a core set of outcome measures for each condition [4]

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