Real World Evidence

Dr. Nicola Specchio et al., Italy
Prof. Adam Strzelczyk et al., Germany
Efficacy and safety of Fenfluramine hydrochloride for the treatment of seizures in Dravet syndrome:  A real-world study1
Dr. Nicola Specchio, Italy
Study design1
Eligible patients1
Population1

All patients carried a SCN1A genetic variant


†The most frequent concomitant AEDs were valproate, clobazam and stiripentol. 31 (59.6%) patients were concomitantly treated with stiripentol.


ASM, anti-seizure medication; IQR, interquartile range; N/A, not applicable; SD, standard deviation; SCN1A, Sodium Voltage-Gated Channel Alpha Subunit 1.

Efficacy results1

Change in the frequency of convulsive seizures per 28 days from baseline to the last follow-up visit (titration plus maintenance period)1

Primary Efficacy Endpoint1


Monthly median convulsive seizure frequency was 6.0 (IQR 4–14.0) at baseline and 1.9 (IQR 0.5–4.5) at last follow-up visit, with a median reduction in convulsive seizure frequency of 77.4% (IQR 43.6-94.4) during the titration plus maintenance period compared with the baseline period 



Data on seizure frequency were available for 45 patients (86.5%); the remaining 7 patients were excluded from the efficacy analysis due to incomplete data collection.

B, baseline; IQR, interquartile range; T + M, titration plus maintenance; The median follow-up was 9.0 months.


Proportion of patients who had ≥25, ≥50, ≥75, or 100% reduction in convulsive seizures from baseline1

Other Efficacy Outcomes1


Data on seizure frequency were available for 45 patients (86.5%); the remaining 7 patients were excluded from the efficacy analysis due to incomplete data collection.

Mean add-on fenfluramine dose of 0.46 mg/kg/day; The median follow-up was 9.0 months.

Safety1

Overview of adverse events

AE, adverse event; SAE, serious adverse event; The median follow-up was 9.0 months; Mean dose of fenfluramine: 0.46 mg/kg/d (range ± SD = 0.2-0.7 ± 0.16).

Summary1

Study limitations: This study is limited by the small sample size, the relatively short median follow-up, the open-label design, and lack of a control group.

AE, adverse event

View references

1. Specchio N et al. Epilepsia 2020;61(11):2405-14.

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