The objective of this study, Consensus Outcomes and Readiness Evaluation in pediatric Autoimmune Encephalitis (CORE-AE), is to improve clinical trial readiness in pediatric AE by addressing a critical gap: absence of a consensus-based core outcome set (COS). We will conduct a modified Delphi process with a multidisciplinary panel including clinicians, clinical trialists, research coordinators, and caregiver representatives. Participants will prioritize outcome domains to develop a consensus-based COS. This study will generate a consensus-based COS to improve comparability across trials, which will form the foundation for a future CORE-AE Toolkit.
A review of previously registered COS identified COS development for acute encephalitis in children and for understanding parental perspectives for encephalitis in children. Our study differs from this given ours focuses solely on autoimmune encephalitis, which differs from infectious etiologies of encephalitis both in underlying mechanism, treatment, prognosis, and outcomes.
Melissa Wright, MD¹; Ka-Ho Wong²; Rebecca Delaney, PhD3; Jennie Hill, PhD3
¹ Division of Pediatric Neurology, Department of Pediatrics, University of Utah, Salt Lake City, UT
² Department of Neurology, University of Utah, Salt Lake City, UT
³ Division of Health System Innovation and Research, Department of Population Health Sciences, University of Utah, Salt Lake City, UT
Disease Category: Neurology
Disease Name: Encephalitis
Age Range: 0 - 18
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Consumers (caregivers)
- Methodologists
- Patient/ support group representatives
- Researchers
- Other
- COS for clinical trials or clinical research
- Delphi process
- Systematic review
The research team will conduct a structured literature review of outcome domains used in AE clinical trials and observational studies. Candidate domains will be refined with input from a Steering Committee to ensure clinical relevance, methodological rigor, and incorporation of caregiver perspectives, with attention to developmental appropriateness across pediatric age groups.
Outcome domains will be evaluated over 2-3 rounds using a rating scale and pre-defined consensus criteria. Domains meeting consensus criteria will be included in the final COS, grouped into clinically meaningful categories.