Abstracts

Rationale: Rarely have patients’ subjective experiences and preferences for outcome been used to evaluate treatment. Understanding patients’ and caregivers’ concept of antiepileptic drug (AED) efficacy and tolerability could impact the design of future clinical trials. We sought to understand how caregivers and patients determine efficacy and tolerability with regards to AED treatment of childhood epilepsy. Methods: A 22-question survey focused on measures of efficacy and tolerability was designed using physician input, patient-attended focus groups, and common clinical trial endpoint measures. Caregivers of children with epilepsy were administered the survey while attending routine Neurology Clinic visits at Emory University. Responses were pooled and frequencies for each question calculated. Results: Sixty-one surveys were completed with 60 (98.4%) patients taking at least one AED (mean 1.38 medications, range 0-4). Participants ranked seizure freedom and seizure reduction as the two most important goals of AED treatment. Forty-two participants felt their child’s seizures were controlled (median seizures/year =3, range 0-2920), yet only 7 (17%) reported seizure freedom. Eleven participants (18%) reported inadequate seizure control (median seizures/year=91, range 6-1095). Amongst this group, the mean reduction in seizure frequency necessary for respondents to consider a medication efficacious was 90% with 56% indicating this reduction should be maintained at least 1 year. Sixty seven percent of participants reported experiencing at least one adverse effect of medication, with somnolence most common (39%). Amongst 23 adverse effects, only those related to organ function, balance, and depression/behavior were regarded as “intolerable/very serious” by more than half of the participants. When asked what characteristics were most important in choosing an AED, 98% felt the medication’s ability to completely control seizures was most important. Physical side effects, impact on personal life/daily activity, and doctor recommendation were also regarded as important factors by over 70% of respondents. Conclusions: There is a discrepancy between data commonly available to physicians and results desired by caregivers when choosing AED treatments for their children. Most caregivers’ concept of seizure control does not require seizure freedom, though the majority are interested in seizure freedom as a primary efficacy measure. When reduction of seizure frequency is used as an efficacy measure, participants require a greater percent reduction maintained for a longer duration than that commonly reported in AED studies. While caregivers tolerate most adverse effects, alteration in organ function, psychological behavior, and balance are intolerable. Determining patients’ preferences for outcomes could improve the utility of clinical trial data for physicians and their patients.