Cognitive and behavioral clinical outcome assessments in children with developmental and epileptic encephalopathies: Issues and instruments.
Systematic review identifies gaps in validated outcome measures for rare severe childhood epilepsies, directly informing design of future drug trials for these conditions.
This Epilepsia review systematically evaluates clinical outcome assessments for cognitive and behavioral domains in children with developmental and epileptic encephalopathies — a heterogeneous group of rare, severe pediatric epilepsies. The identified gaps in validated DEE-specific COAs are directly relevant for drug development in conditions like Dravet syndrome and CDKL5 deficiency disorder.
What the study was
- Study design
- Systematic review of outcome assessment instruments
- Population
- Children with developmental and epileptic encephalopathies (DEE)
- Category
- Other
- Maturity
- Exploratory
- Journal
- Epilepsia
Why it surfaced
DEE represents a high-unmet-need rare pediatric epilepsy group. Outcome instrument review is foundational for drug development trials; gaps in validated COAs are a known barrier to DEE drug approval. Epilepsia is the leading epilepsy journal.
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