Fenfluramine below the age of 2 years in Dravet Syndrome: what about safety and efficacy?

Dravet Syndrome (DS) is a rare developmental and epileptic encephalopathy. Infants with DS are especially vulnerable to the detrimental effects of prolonged and frequent seizures on the development. Fenfluramine (FFA) is approved for the treatment of DS in patients from the age of 2 years and above. This study aims to evaluate the safety and efficacy of FFA in patients with DS younger than 2 years of age. We analyzed safety, tolerability, seizure and neuropsychological outcome in a real-world setting. Developmental profile was investigated using Griffiths Mental Development Scales (GMDS). Five patients received FFA at a mean age of 14.9 months (9.6-18.6). Median follow-up was 13 months (IQR=12.9-24.4). All patients showed a good tolerance to FFA. No significant variation of BMI and echocardiographic issue were observed. Monthly median convulsive seizure frequency (MCSF) was 1.71 (IQR=1.56-3.27) at six-months baseline period and 0.92 (IQR=0.43-1.28) at last follow-up, with a median 54.43 (IQR=40.91-60.83) percentage reduction in MCSF. Two out of 5 patients had an improvement of GMDS performances sub-scales. Overall, the use of FFA below the age of 2 years in our small sample of patients was safe and represents a promising opportunity for seizure control and for protection of the neurodevelopmental outcome.

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