CORE OUTCOMES FOR REFRACTORY CHILDHOOD EPILEPSY TREATED WITH KETOGENIC DIET THERAPY: THE CORE-KDT STUDY

Abstract

Background: Ketogenic diet therapy can result in seizure and non-seizure related benefits for children with drug resistant epilepsy. However, clinical trials report a wide range of outcomes which makes evidence synthesis difficult and they 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 - a minimum standardised set of outcomes that should be measured and reported. (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 international Delphi survey of parents and professionals to prioritise outcomes of importance for inclusion in a core outcome set. This informed a stakeholder consensus meeting and consultation process which finalised the core outcome set.

Results: In total, 97 outcomes were identified; 90 from the scoping review and seven from parent interviews. These were rationalised to 77 by the study advisory group, then rated by 49 parents and 96 health professionals in round one of the Delphi. Participants suggested 12 new outcomes for inclusion in round two, completed by 66% (30 parents and 66 professionals). Twenty-two outcomes met criteria for inclusion. Twenty-seven undecided outcomes were discussed and scored in the consensus meeting (9 parents and 13 professionals); one further outcome reached consensus for inclusion. Following the consensus meeting and ratification, 14 outcomes across five domains were included in the core outcome set.

Conclusions: A core outcome set for childhood epilepsy treated with KD therapy has been developed, incorporating the views of international parents and professionals. Implementation in research and clinical settings will help to standardise outcome selection and reporting, facilitate data synthesis and ultimately enhance the relevance of outcomes to parents, researchers and health professionals.