Patient-Reported Outcome Measures and Clinical Outcomes in Children with Foregut Anomalies.

Isabel I Sreeram,Johannes M Schnater,Joost Van Rosmalen,Hanneke Ijsselstijn,Saskia J Gischler,André B Rietman,René M H Wijnen,Chantal A Ten Kate

doi:10.3390/children8070587

Isabel I Sreeram, Johannes M Schnater + Show 6 more

Open Access

https://doi.org/10.3390/children8070587

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Abstract

Increasing numbers of children and adults with chronic disease status highlight the need for a value-based healthcare system. Patient-reported outcome measures (PROMs) are essential to value-based healthcare, yet it remains unclear how they relate to clinical outcomes such as health and daily functioning. We aimed to assess the added value of self-reported PROMs for health status (HS) and quality of life (QoL) in the long-term follow-up of children with foregut anomalies. We evaluated data of PROMs for HS and/or QoL among eight-year-olds born with congenital diaphragmatic hernia (CDH), esophageal atresia (EA), or congenital lung malformations (CLM), collected within the infrastructure of a multidisciplinary, longitudinal follow-up program. Clinical outcomes were categorized into different outcome domains, and their relationships with self-reported HS and QoL were assessed through multivariable linear regression analyses. A total of 220 children completed HS and/or QoL self-reports. In children with CDH and EA, lower cognition was significantly associated with lower self-reported HS. Due to the low number of cases, multivariable linear regression analysis was not possible in children with CLM. HS, QoL, and clinical outcomes represent different aspects of a child’s wellbeing and should be measured simultaneously to facilitate a more holistic approach to clinical decision making.

Highlights

Survival among children with congenital anomalies has greatly improved in recent decades
15 children (3%) had syndromes with severe intellectual disability, 101 children with congenital lung malformations (CLM) had no surgery within 28 days after birth, and 1 child with congenital diaphragmatic hernia (CDH) had an incidental finding of CLM, and was only analyzed in the CDH group, leaving 350 eligible children
Self-perceived health status (HS) and quality of life (QoL) could not be evaluated in these children. In this cohort of eight-year-old children born with foregut anomalies, we evaluated the

Summary

Introduction

Survival among children with congenital anomalies has greatly improved in recent decades. The numbers of children and adults living with a chronic condition are steadily increasing, causing significant financial strain on healthcare systems [1,2,3]. To optimize economic sustainability while improving health, emphasis must be placed on value-based healthcare (VBHC) [4]. This can be achieved by focusing on the impact of disease-related morbidities on a patient’s self-perceived health status (HS) and quality of life (QoL) [4,5,6]. It remains unclear how PROMs relate to clinical outcomes such as health, morbidity, and daily functioning in children with chronic conditions [5]

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