Objective: Ketogenic diet therapy (KDT) can result in benefits (seizure-related and non-seizure-related) for children with drug-resistant epilepsy. However, clinical trials report a wide range of outcomes, making synthesis of evidence difficult, and do not adequately reflect parent views on important outcomes for their child. To address this, we established the first international parent, health professional, and researcher consensus to develop a core outcome set, guided by the Core Outcome Measures in Effectiveness Trials (COMET) Initiative (COMET registration #1116).
Methods: Ethical approval was granted (London-Surrey REC19/LO/1680). A scoping review and interviews with parents identified a comprehensive list of potentially important outcomes, followed by a two-round online Delphi survey of parents and health professionals to prioritize outcomes of importance for inclusion in a core outcome set. This informed a stakeholder consensus meeting and consultation process to finalize the core outcome set.
Results: In total, 97 outcomes were identified; 90 from the scoping review and seven from parent interviews. These were rationalized to 77 by the study advisory group, then rated in the first Delphi round by 49 parents and 96 health professionals, who suggested 12 new outcomes for rating in Round 2. Sixty-six percent of participants (30 parents and 66 professionals) completed Round 2, where 22 outcomes met criteria for inclusion. In the consensus meeting (nine parents and 13 professionals), 27 undecided outcomes were discussed and scored; one further outcome reached consensus for inclusion. After consultation and ratification, 14 outcomes across five domains were included in the core outcome set.
Significance: A core outcome set for childhood epilepsy treated with KDT has been developed, incorporating the views of international parents and professionals. Implementation in research and clinical settings will standardize outcome selection and reporting, facilitate data synthesis, and ultimately enhance the relevance of outcomes to parents, researchers, and health professionals.
Jennifer Carroll (principal investigator), Lecturer in Dietetics, School of Health Professions, Faculty of Health and Human Sciences, University of Plymouth, UK.
Professor Helen Cross, The Prince of Wales’s Chair of Childhood Epilepsy & Head of Developmental Neurosciences Programme, Clinical Neurosciences, UCL-Great Ormond Street-Institute of Child Health, University College London, UK.
Dr Avril Collinson, Associate Professor & Academic Lead Dietetics, School of Health Professions, Faculty of Health and Human Sciences, University of Plymouth, UK.
Professor Mary Hickson, Professor of Dietetics, School of Health Professions, Faculty of Health and Human Sciences, University of Plymouth, UK.
Emma Williams, Matthew's Friends Charity & Clinics, UK.
Val Aldridge, Matthew's Friends Charity & Clinics, UK.
Disease Category: Child health, Neurology
Disease Name: Epilepsy
Age Range: 1 - 18
Sex: Either
Nature of Intervention: Diet and nutrition
- Charities
- Clinical experts
- Consumers (caregivers)
- Families
- COS for clinical trials or clinical research
- COS for practice
- Consensus meeting
- Delphi process
- Interview
- Literature review
- Systematic review
NHS Health Research Authority ethical approval will be sought and informed consent from participants. This is a mixed methods study with four distinct phases.
Phase one: Systematic review of the literature to identify outcomes in studies of children with epilepsy treated with ketogenic diet therapy (KDT).
Phase two: Semi structured qualitative interviews with approximately 30 parents and carers of children with epilepsy treated with KDT.
Phase three: Define outcome domains based on the findings of the systematic review and qualitative interviews.
Phase four: 2 arm anonymous Delphi process whereby health care professionals (arm 1) and parents and carers (arm 2) rate the importance of the outcome domains. It is expected that two Delphi rounds will be undertaken. Finally a consensus meeting where both stakeholder groups are represented will be held and the core outcome set for childhood epilepsy treated with KDT agreed.