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

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.

Parent-Reported Perceived Cognitive Functioning Identifies Cognitive Problems in Children Who Survived Neonatal Critical Illness

“…Parents’ perception appears to be more consistent with objectively measured cognitive functions. The absence of associations between pediatric self-reports and clinical outcomes has been described before [ 14 ]. We believe that early recognition of neurocognitive problems is important, even when these problems do not seem to affect functioning in everyday life, since school-aged children cannot yet see the potential impact.…”

“…A longitudinal follow-up program for children with congenital anomalies and/or those treated with neonatal ECMO is being run in our tertiary level hospital since 1999. The program includes standardized screening of physical health, motor function, cognition, executive functioning, and quality of life at set ages [ 9 , 11 , 12 , 13 , 14 ]. In previous studies, neuropsychological assessments (NPA) revealed that children born with EA, CDH, and those treated with neonatal ECMO have problems in attention, memory, and executive functioning [ 3 , 4 , 5 , 6 , 7 , 8 , 9 ].…”

Variability in the Reporting of Baseline Characteristics, Treatment, and Outcomes in Esophageal Atresia Publications: A Systematic Review

“…The third remarkable trend is that only 18 studies described quality-oflife outcomes through patient-reported outcome measures (PROMs), and the applied instrument varied across the studies. 10,[12][13][14][17][18][19][20][21][22][23][24][25][26][27] The most used PROM was the generic, child-specific PedsQL, which, however, was mentioned in only 8/209 papers (4%). This scarce use of PROMs is surprising, as there is a general movement to patient-centered care and PROM research across the medical world.…”

Psychometric Performance of a Condition-Specific Quality-of-Life Instrument for Dutch Children Born with Esophageal Atresia

“…Given the strong correlation of condition-specific scores with generic PedsQL scores, the added value of implementing the EA-QOL© questionnaires in clinical practice should first be established. A possible approach is to correlate scores to clinical outcomes, like has been carried out for the PedsQL and DUX-25 [ 48 ]. A next step could be to combine the internationally obtained validation results into an IRT model, using the original EA-QOL© items available before item reduction [ 16 ], with the addition of topics brought up during cognitive debriefing in multiple countries.…”

“…Furthermore, the parental educational level was higher than in the general population. Although this is a common finding in the EA population [ 12 , 48 ] and in psychometric evaluation in general [ 49 ], it should be taken into account that this could lead to bias. Moreover, the online study set-up and some statistical assumptions differed from earlier EA-QOL© validation studies.…”