Caregiver measures for seizure control, efficacy, and tolerability of antiepileptic drugs for childhood epilepsy: Results of a preference survey

We sought to identify and quantify caregiver-defined characteristics of efficacy related to the perceived success of antiepileptic drug (AED) use. A 22-question survey was designed using physician input, focus groups, and clinical trial endpoints. Responses were pooled and analyzed with regard to seizure type and treatment, categorized as controlled (exposure to 1 AED), adjunctive (exposure to 2 AEDs), or refractory (exposure to ≥ 3 AEDs). Two hundred ninety-five surveys were completed: 109 (37%) controlled, 84 (28%) adjunctive, and 102 (35%) refractory. Seizure freedom and median seizure reduction > 90% maintained for > 1 year were reported as the most important indicators of medication efficacy by the majority of respondents. These measures were the same regardless of seizure type or treatment category. Our results demonstrate that current trial design may be inadequate to address the expectations of patients. Incorporating patient-defined AED efficacy measures may improve satisfaction and informed decision making regarding epilepsy treatment.

► Measures of antiepileptic drug efficacy considered important by consumers were identified and quantified. ► Caregivers identify seizure freedom and seizure reduction as the most important efficacy measures. ► Seizure freedom > 1 year and seizure reduction > 90% for more than 1 year are important markers of antiepileptic drug efficacy. ► Efficacy measures and quantification do not vary across seizures types or treatment resistance. ► Outcome measures such as reduction in seizure clustering are important in certain seizure types.