Fenfluramine could be an important new treatment option for patients with Dravet syndrome, say the authors of a study published in the Lancet.
In the double-blind trial, 119 children and young adults (2-18 years) with Dravet syndrome were randomised to receive add-on treatment of either fenfluramine 0.2 mg/kg/day (F2; n=39), fenfluramine 0.7 mg/kg/day (F7; n=40) or placebo (n=40).
During treatment, the median reduction in seizure frequency was 74.9 per cent in the F7 group, 42.3 per cent in the F2 group and 19.2 per cent with placebo.
The F7 group showed a 62.3 per cent greater reduction in mean monthly convulsive seizure frequency (MCSF) compared with placebo (P<.0001 and f2 showed a per cent reduction vs placebo>
Adverse events were reported in 26 (65%) patients in the placebo group, 37 (95%) in the F2 group and 38 (95%) in the F7 group.
The authors said echocardiographic examinations revealed valve function within the normal physiological range in all patients during the trial and no signs of pulmonary arterial hypertension.
However, they added that conclusions about the cardiovascular safety of fenfluramine are limited by the short treatment and observation period of 14 weeks.