Skuteczność i bezpieczeństwo sultiamu w leczeniu opornej na leki epilepsji u dzieci - badanie praktyczne
Real-world efficacy and safety profile of sulthiame treatment in drug-resistant childhood epilepsy
W skrócie
Badanie pokazuje, że lek o nazwie sultiaam jest skuteczny i bezpieczny w leczeniu epilepsji u dzieci, która nie reaguje na zwykłe leki przeciwpadaczkowe. Ponad 40% dzieci osiągnęło całkowitą swobodę od napadów, a lek był dobrze tolerowany bez poważnych skutków ubocznych. Najlepsze wyniki zaobserwowano u dzieci z napadami ogniskowanymi (pochodzącymi z jednego miejsca w mózgu), a leczenie było mniej skuteczne u pacjentów, którzy wcześniej nieudanie próbowali wiele innych leków.
Oryginalny abstract (angielski)
OBJECTIVE: Sulthiame (STM) is a potent carbonic anhydrase inhibitor primarily used in focal epilepsy syndromes; however, clinical data regarding its efficacy and safety in broader drug-resistant childhood epilepsy remain limited. This study aimed to evaluate the clinical efficacy, electroencephalographic response, and safety profile of STM in a cohort of pediatric patients with drug-resistant epilepsy. METHODS: A retrospective medical chart review was conducted for patients under 18 years of age treated with STM at a tertiary pediatric neurology clinic between 2011 and 2024. Data including demographic characteristics, etiology, seizure semiology, epilepsy syndromes, treatment duration, and dosage were analyzed. Electroencephalographic (EEG) findings were assessed using EEG recordings obtained within 1-3 months after initiation of sulthiame therapy. Seizure frequency outcomes were assessed by comparing baseline seizure frequency with seizure frequency at the final available clinical follow-up. RESULTS: Sixty-one patients (55.7% female; median seizure onset: 14 months) were included. A structural etiology was identified in 50.8% of patients, and 62.3% had a well-defined epilepsy syndrome. At the final available clinical follow-up, 41% (n = 25) of patients achieved complete seizure freedom, while 16.4% (n = 10) showed a ≥ 50% reduction in seizure frequency. Conversely, 42.6% demonstrated a < 50% reduction. A higher number of previously failed anti-seizure medications (ASMs) was significantly associated with a poorer treatment response (p = 0.014). EEG improvement was observed in 44.3% of the cohort. Notably, focal seizure semiology was associated with favorable EEG outcomes, whereas combined semiology was linked to electroencephalographic worsening (p = 0.026). STM was generally well-tolerated; adverse effects were reported in only nine patients, with treatment discontinuation required in only two cases (3.2%). CONCLUSIONS: Sulthiame is an effective and well-tolerated therapeutic option for children with drug-resistant epilepsy, particularly those with focal semiology. Our findings suggest that a higher burden of prior ASM treatment negatively impacts response rates, highlighting the potential benefit of considering STM earlier in the treatment algorithm for specific pediatric populations.