Treatment goals for pharmacoresistant epilepsy in childhood and youth – The parental perspective

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PURPOSE: Research on pharmacoresistant epilepsy including developmental and epileptic encephalopathies (DEE) predominantly focuses on seizure control and health-related quality of life (HRQoL), while explicit parental goals concerning treatment outcomes as well as perceived resources and support needs remain somewhat underrepresented. We aimed to systematically assess parental priorities, perceived resources, and support needs in families of children with therapy-resistant epilepsies. METHODS: We conducted an online survey (September-October 2025). Parents ranked 14 symptom domains according to personal relevance, rated opportunities to address these topics in different healthcare settings and evaluated support needs across medical and psychosocial domains. Descriptive and non-parametric statistical analyses were performed, including subgroup comparisons by age and Dravet/SCN1A status. RESULTS: A total of 224 questionnaires were analyzed (mean child age 12.0 ± 6.3 years; 34 different genetic etiologies, including 40 Dravet/SCN1A patients). Seizures were the top concern when it came to what most affects families' daily lives. However, cognitive development, sleep, and externalizing behavioral problems consistently followed, indicating a multidimensional goal structure beyond seizure control. Subgroup analyses revealed largely similar priorities. Parents reported sufficient time to address medical issues in specialized settings but expressed substantial unmet needs in psychological support for the child, themselves, and the family. Free-text responses highlighted deficits in access to therapies, educational inclusion, care coordination and psychosocial support. CONCLUSIONS: While seizures remain a central concern, families prioritize developmental, behavioral, sleep-related, and psychosocial domains nearly as strongly. Current care models and literature emphasize HRQoL measurement but rarely capture explicit parental goals. Systematic goal elicitation and genuine interdisciplinary, family-centered care are needed to alleviate the burden on families through appropriate treatment planning.

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