Patterns, temporal trends and methodological associations in the measurement and valuation of childhood health utilities

Purpose To systematically assess patterns and temporal changes in the measurement and valuation of childhood health utilities and associations between methodological factors. Methods Studies reporting childhood health utilities using direct or indirect valuation methods, published by June 2017, were identified through PubMed, Embase, Web of Science, PsycINFO, EconLit, CINAHL, Cochrane Library, and PEDE. The following were explored: patterns in tariff application; linear trends in numbers of studies/samples and paediatric cost-utility analyses (CUAs) and associations between them; changes in proportions of studies/samples within characteristic-based categories over pre-specified periods; impact of National Institute for Health and Care Excellence (NICE) guidance on primary UK research; and associations between valuation method, age and methodological factors. Results 335 studies with 3,974 samples covering all ICD-10 chapters, 23 valuation methods, 12 respondent types, and 42 countries were identified by systematic review. 34.0% of samples using indirect methods compatible with childhood applied childhood-derived tariffs. There was no association between numbers of studies/samples and numbers of CUAs. Compared to 1990-2008, 2009-June 2017 saw a significant fall in the proportion of studies using case series; significant compositional changes across ICD-10 chapters; and significantly higher sample proportions using childhood/adolescent-specific and adult-specific indirect valuation methods, and based on pre-adolescents, self-assessment, self-administration and experienced health states. NICE guidance was weakly effective in promoting reference methods. Associations between valuation method, age and methodological factors were significant. Conclusion 1990-2017 witnessed significant changes in primary research on childhood health utilities. Health technology assessment agencies should note the equivocal effect of methodological guidance on primary research methods

A Microcosting and Cost-Consequence Analysis of Genomic Testing Strategies in Autism Spectrum Disorder

Produced by Technology Assessment at SickKids, Hospital for Sick Children.The primary objective of this study is to estimate costs associated with Chromosomal microarray analysis (CMA), whole exome sequencing (WES) and whole genome sequencing (WGS) tests for a targeted patient population consisting of children with ASD from an institutional payer perspective over 5 years. The secondary objective is to compare the incremental costs and diagnostic yields of CMA, WES and WGS in hypothetical clinical testing scenarios in a cost-consequence analysis.Supported by a Large-Scale Applied Research Project grant from Genome Canada and the Ontario Genomics Institute

Chronic ischemic lesions and presence of patent foramen ovale in young adults with embolic stroke of undetermined source - Results of the Young ESUS Patient Registry.

BACKGROUND Chronic ischemic lesions (CIL) are frequent findings in patients with acute ischemic stroke, but their phenotypes and relevance in young adults with embolic stroke of undetermined source (Y-ESUS) remains uncertain. We aimed to compare Y-ESUS patients with CIL to those without CIL and assessed the association of CIL and its phenotypes with the presence of patent foramen ovale (PFO). METHODS This prospective longitudinal, multicenter cohort study enrolled consecutive patients 50 years and younger with ESUS from 10/2017 to 10/2019 in 41 stroke research centers in 13 countries. Local investigators adjudicated presence and phenotypes of CIL on routine brain imaging (either MRI or CT). RESULTS Overall, 535 patients were enrolled (mean age 40.4 (SD 7.3) years, 238 (44%) female). CIL were present in 76/534 (14.2%) patients with a median count CIL count of 1.0 (IQR: 1 to 2), 42/76 (55%) had at least one cortical phenotype and 38/76 (50%) at least one non-cortical phenotype. Y-ESUS with CIL were less often female (32% vs 47% in non-CIL Y-ESUS), were older (mean 43 vs 40 years), had more often hypertension (42% vs 19%), diabetes (17% vs 7%), and hyperlipidemia (34% vs 18%). CIL Y-ESUS were independently associated with lower stroke recurrence (RR 0.17 (0.05-0.61). In Y-ESUS with PFO, CIL were less frequent in probable pathogenic PFO than with probable non-pathogenic PFO (6.1% vs 30% P<0.001). CONCLUSIONS One in seven Y-ESUS patients has additional CIL. CIL were associated with several vascular risk factors, lower probability of a pathogenic PFO and lower stroke recurrence