Quality of Life and Functional Outcomes for Ketogenic Dietary Treatment: Developing and Piloting a Proxy-Reported Measurement Tool for Children with Epilepsy

The aim of this research is therefore to develop a novel proxy-reported measurement tool to identify meaningful changes in quality of life, alertness, concentration and behaviour in children and young people treated with the ketogenic diet for the management of drug-resistant epilepsy. By improving the consistency and relevance of quality-of-life reporting, this tool has the potential to strengthen clinical decision-making and enhance the evidence base supporting ketogenic diet treatment.

The overarching aim of this research is to develop and evaluate a proxy-reported measurement tool for assessing quality of life, alertness, concentration, and behaviour in children with drug-resistant epilepsy following a ketogenic diet.
Principal Research Question
Can a proxy-reported measurement tool be developed to assess quality of life and functional outcomes for paediatric patients with drug-resistant epilepsy who follow a ketogenic diet, incorporating the caregiver and clinician perspectives while being clinically feasible, focusing on the constructs of quality of life, alertness and concentration, and behaviour?
Research Sub-Questions
a. What outcome measurement instruments are available to assess quality of life, alertness and concentration, and behaviour in paediatric populations with developmental delay, physical disability or neurodisability including epilepsy, cerebral palsy and learning disabilities?
b. How do caregivers and professionals define and understand the domains quality of life, alertness and concentration, and behaviour. What are the important aspects of these domains from their perspectives?
c. Does the draft proxy-reported quality of life measurement tool for patients with epilepsy following a ketogenic diet, represent the original constructs and are they relevant to the cohort according to the study advisory group?
d. Do healthcare professionals and caregivers identify any issues with understanding, grammar, or flow in the draft proxy-reported quality of life measurement tool for patients with epilepsy following a ketogenic diet?
e. How feasible is the implementation of a newly developed measurement tool for paediatric patients with epilepsy following a ketogenic diet in two specialist centres in the United Kingdom, with a focus on:
i. Tool implementation and integration into clinical practice
ii. Practical administration and user experience
iii. Representation and inclusivity of the target population
iv. Effectiveness in measuring outcomes.
f. How can statistical methods such as Classical Test Theory (CTT), Rasch measurement theory, and Item Response Theory (IRT) be applied to reduce the number of items in the newly developed measurement tool whilst maintaining psychometric integrity, clinical utility, and caregiver/ clinician acceptability?