Mapping epilepsy-specific patient-reported outcome measures for children to a proposed core outcome set for childhood epilepsy

Abstract

The objectives of the study were to (1) map questions in epilepsy-specific patient-reported outcome measures (PROMs) of children's health-related quality of life (HRQoL) to a proposed core outcome set (COS) for childhood epilepsy research and (2) gain insight into the acceptability of two leading candidate PROMs. Method: We identified 11 epilepsy-specific PROMs of children's HRQoL (17 questionnaire versions) in a previous systematic review. Each item from the PROMs was mapped to 38 discrete outcomes across 10 domains of the COS: seizures, sleep, social functioning, mental health, cognition, physical functioning, behavior, adverse events, family life, and global quality of life. We consulted with three children with epilepsy and six parents of children with epilepsy in Patient Public Involvement and Engagement (PPIE) work to gain an understanding of the acceptability of the two leading PROMs from our review of measurement properties: Quality of Life in Childhood Epilepsy (QOLCE-55) and Health-Related Quality of Life Measure for Children with Epilepsy (CHEQOL). Results: Social Functioning is covered by all PROMs except DISABKIDS and G-QOLCE and Mental Health is covered by all PROMs except G-QOLCE and Hague Restrictions in Childhood Epilepsy Scale (HARCES). Only two PROMs (Epilepsy and Learning Disability Quality of Life (ELDQOL) and Glasgow Epilepsy Outcome Scale (GEOS-YP)) have items that cover the Seizure domain. The QOLCE-55 includes items that cover the domains of Physical Functioning, Social Functioning, Behavior, Mental Health, and Cognition. The CHEQOL parent and child versions cover the same domains as QOLCE-55 except for Physical Functioning and Behavior, and the child version has one item that covers the discrete outcome of Overall Quality of Life and one item that covers the discrete outcome of Relationship with parents and siblings. The QOLCE-55 parent version was acceptable to the parents we consulted with, and CHEQOL parent and child versions were described as acceptable to our child and parent advisory panel members. Significance: Mapping items from existing epilepsy-specific PROMs for children is an important step in operationalizing our COS for childhood epilepsy research, alongside evaluation of their measurement properties. Two leading PROMS, QOLCE-55 and CHEQOL, cover a wide range of domains from our COS and would likely be used in conjunction with assessment tools selected for specific study objectives. The PPIE work provided practical insights into the administration and acceptability of candidate PROMs in appropriate context. We promote our COS as a framework for selecting outcomes and PROMs for future childhood epilepsy evaluative research.

Health-related quality of life in children with epilepsy: a concept analysis

“…Here, health status includes physical and mental health, as well as cognitive and behavioral functions that show problems, because diseases cause difficulties in an individual's daily life or role performance. In addition, individuals' unique inner resources such as self-esteem and self-efficacy are strongly related to HRQOL in children with epilepsy, and their presence plays an important buffer in attenuating the negative effects of factors that deteriorate HRQOL [3,25,[32][33][34][35][36][37][38][39].…”

ICF Linking and Cognitive Interviewing Are Complementary Methods for Optimizing Content Validity of Outcome Measures: An Integrated Methods Review

“…Although this paper provides insights into current content validation methods it is not a comprehensive of all methods that can be used. For example, quantitative methods like content validity indices (6,10,68,70,77,78) (a survey method), concept/content mapping (62), mapping to consensus core sets (79), theoretical models other than ICF, clarification of conceptual models/construct definitions (80), or qualitative methods other than cognitive interviewing (69) were not explored in this paper. Given the array of appropriate methods that could be used in content validation, a prescriptive approach to content validation might lead to narrow thinking.…”

Patient perspectives in pediatric neurology: a critical shift in the paradigm of outcome measurement

“…For this reason, researchers have used the Delphi technique to ask stakeholders, including families, to determine what issues they consider important enough to be included in PROMs. 18,20,42…”

“…To support comprehensive evidence‐based recommendations for selecting the most suitable PROM for a given purpose, the Consensus‐based Standards for the selection of Health Measurement INstruments (COSMIN) initiative developed methodological standards for studies by selecting a minimum core set of outcomes that should be measured and reported in all clinical trials of a specific condition or trial population 16 . Systematic reviews are available for many neurologically‐based conditions: muscular dystrophy, 48,49 autism spectrum disorder, 50 child disability, 51 epilepsy, 42,52 survivors of brain tumour and other acquired brain injuries, 53 and pediatric multiple sclerosis 54 . The Core Outcome Measures in Effectiveness Trials are seeking consensus on what is important and meaningful to measure and are available for antiepileptic therapy in pediatric epilepsy 55 .…”