Epilepsy & behavior : E&B · 2026
Efficacy and safety of fenfluramine in Dravet syndrome; A monocentric, observational study focused on initial dose, blood concentration, and reduction of concomitant medications.
СтатьяНаблюдательное исследованиеНазвание и аннотация: Перевод пока не подготовлен
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Iguchi Akihiro, Yamaguchi Tokito, Tsuyusaki Yu, Otani Hideyuki, Mizutani Satoshi, Miyashita Mitsuhiro, Yabe Manami, Imai Katsumi
Аннотация
OBJECTIVE: To evaluate the optimal initial dose, efficacy, and adverse events of fenfluramine (FFA) in the treatment of intractable epilepsy in Dravet syndrome (DS) in Japanese patients.
METHODS: This single-center, observational study included Dravet patients treated with FFA. Information retrospectively collected from medical records included age, sex, concomitant antiseizure medications (including stiripentol [STP]), the initial and maximum dose of FFA, differences in the effects of FFA with and without STP, convulsive seizure frequency, adverse events, and FFA blood concentration.
RESULTS: Of the 27 patients, 13 (48.1%) were male, and the median age was 14.9 years. After the initiation of FFA, 59.3% and 25.9% of patients were 50% and 90% responders, respectively. Of the sixteen 50% responders, 13 (81.3%) had either discontinued a concomitant antiseizure medication or were in the process of reducing the dose. In 88.9% of patients, the initial dose of FFA was less than 0.2 mg/kg/day, and ≥ 50% seizure reduction was achieved in 45.8% at less than 0.2 mg/kg/day. The effectiveness of FFA was not significantly different with or without STP. Among the ≥ 50% responders, cognitive and behavioral function improved in 9 (56.3%). The major adverse events were diarrhea (29.6%). No patients developed valvular heart disease or pulmonary artery hypertension. Among patients whose blood concentrations of FFA were measured, all 50% responders had < 80 ng/mL.
SIGNIFICANCE: FFA effectively reduced convulsive seizures in DS even at low concentrations. we recommend the initiation of low-dose FFA, which may allow dose reduction or discontinuation of concomitant antiseizure medications in FFA responders.
METHODS: This single-center, observational study included Dravet patients treated with FFA. Information retrospectively collected from medical records included age, sex, concomitant antiseizure medications (including stiripentol [STP]), the initial and maximum dose of FFA, differences in the effects of FFA with and without STP, convulsive seizure frequency, adverse events, and FFA blood concentration.
RESULTS: Of the 27 patients, 13 (48.1%) were male, and the median age was 14.9 years. After the initiation of FFA, 59.3% and 25.9% of patients were 50% and 90% responders, respectively. Of the sixteen 50% responders, 13 (81.3%) had either discontinued a concomitant antiseizure medication or were in the process of reducing the dose. In 88.9% of patients, the initial dose of FFA was less than 0.2 mg/kg/day, and ≥ 50% seizure reduction was achieved in 45.8% at less than 0.2 mg/kg/day. The effectiveness of FFA was not significantly different with or without STP. Among the ≥ 50% responders, cognitive and behavioral function improved in 9 (56.3%). The major adverse events were diarrhea (29.6%). No patients developed valvular heart disease or pulmonary artery hypertension. Among patients whose blood concentrations of FFA were measured, all 50% responders had < 80 ng/mL.
SIGNIFICANCE: FFA effectively reduced convulsive seizures in DS even at low concentrations. we recommend the initiation of low-dose FFA, which may allow dose reduction or discontinuation of concomitant antiseizure medications in FFA responders.