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Systematic review of outcome measures in pediatric eosinophilic esophagitis treatment trials.

ABSTRACT: BACKGROUND:Heterogeneity 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. METHODS:We 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. RESULTS:Eleven 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. CONCLUSIONS:The 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.

SUBMITTER: Rubin T

PROVIDER: S-EPMC5006498 | biostudies-literature | 2016

REPOSITORIES: biostudies-literature

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Systematic review of outcome measures in pediatric eosinophilic esophagitis treatment trials.

Rubin Tamar T Clayton Jacqueline J Adams Denise D Persad Rabin R Vohra Sunita S

Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology 20160831 1

<h4>Background</h4>Heterogeneity 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 establish ...[more]

PMID: 27582776

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Project description:Background: Eosinophilic esophagitis (EoE) was first described in the 1990s, showing an increasing incidence and prevalence since then, being the leading cause of food impaction and the major cause of dysphagia. Probably, in a few years, EoE may no longer be considered a rare disease. Methods: This article discusses new aspects of the pathogenesis, symptoms, diagnosis, and treatment of EoE according to the last published guidelines. Results: The epidemiological studies indicate a multifactorial origin for EoE, where environmental and genetic factors take part. EoE affects both children and adults and it is frequently associated with atopic disease and IgE-mediated food allergies. In patients undergoing oral immunotherapy for desensitization from IgE-mediated food allergy the risk of developing EoE is 2.72%. Barrier dysfunction and T-helper 2 inflammation is considered to be pathogenetically important factors. There are different patterns of clinical presentation varying with age and can be masked by adaptation habits. Besides, symptoms do not usually correlate with histologic disease activity. The diagnostic criteria for EoE has evolved but mainly requires symptoms of esophageal dysfunction with histologic evidence of a peak value of at least 15 eosinophils per high-power field. Endoscopies have to be repeated in order to diagnose, monitor, and treat EoE. Treatment of EoE can be started either by drugs (PPIs and topical corticosteroids) or elimination diets. The multistage step-up elimination diet management approach of EoE is promising. Endoscopic dilation is used for patients with severe dysphagia/food impaction with inadequate response to anti-inflammatory treatment. Conclusions: Research in recent years has contributed to a better understanding of EoE's pathogenesis, genetic background, natural history, allergy workup, standardization in assessment of disease activity, evaluation of minimally invasive diagnostic tools, and new therapeutic approaches. However, several unmet needs are to be solved urgently, as finding a non-invasive disease-monitoring methods and biomarkers for routine practice, the development or new therapies, novel food allergy testing to detect triggering foods, drug, and doses required for initial therapy and safety issues with long-term maintenance therapy, amongst others. Besides, multidisciplinary management units of EoE, involving gastroenterologists, pediatricians, allergists, pathologists, dietitians, and ENT specialists are needed.

Project description:OBJECTIVES:No formal comparative effectiveness studies have been conducted to evaluate the effect of eosinophilic esophagitis (EoE) treatment choice on long-term growth in pediatric patients. Long-term studies of inhaled corticoid steroids in asthma, however, suggest possible effects on linear growth. The aim of this study was to compare longitudinal, anthropometric growth in children with EoE according to treatment approach. METHODS:We conducted a retrospective, multicenter cohort study of anthropometric growth (height and body mass index [BMI] z scores) in pediatric (<18 years of age) patients newly diagnosed with EoE across 5 clinical sites between 2005 and 2014. We compared differences in growth according to treatment approach over a 12-month period. Modification by sex and age was examined and sensitivity analyses were conducted to assess robustness of results given study assumptions. RESULTS:In the 409 patients identified, the mean age and proportion male differed by treatment (P?=??<?0.01 and P?=?0.04, respectively). Baseline growth measures were associated with slight impairment of height at diagnosis (median baseline height z score of -0.1 [interquartile range -0.9, 0.8]). In general, treatment approach was not associated with any significant increase or decrease in expected growth over a 12-month period. Subtle decrease in linear growth was observed with treatment using a combined elemental and topical steroid (? height z score [adjusted]: -0.04; 95% confidence interval [CI]: -0.08, -0.01). Differences in linear growth differed by sex (P for interaction <0.01). For elemental formula in combination with topical steroids, only girls exhibited a significant decline in linear growth (? height z score [adjusted]: -0.24; 95% CI: -0.32, -0.17). A slight reduction in BMI was observed for patients treated with a combination of elemental diet and dietary elimination (? BMI z score [adjusted]: -0.07; 95% CI: -0.13, -0.01). CONCLUSIONS:Treatment of EoE, in general, is not associated with major anthropometric growth changes in most pediatric patients. Slight linear growth impairment was observed for topical steroid treatment, and sex differences in growth by treatment approach were observed. Future prospective studies should evaluate the effect of treatment on optimal growth and development and over a longer period of follow-up.

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Systematic review of outcome measures in pediatric eosinophilic esophagitis treatment trials.

Publications

Systematic review of outcome measures in pediatric eosinophilic esophagitis treatment trials.

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