Abstract Details

Title Health Outcomes Stratified by Number of Adverse Events Experienced by Patients with Dravet Syndrome: Insights from a Global Real-world Study

Topic Epilepsy/Clinical Neurophysiology (EEG)

Presentation(s) P2 - Poster Session 2 (11:45 AM-12:45 PM)

Poster/Presentation
Number 1-005

Objective To examine clinical and economic outcomes among patients with Dravet syndrome (DS) receiving antiseizure medications (ASMs) and experiencing adverse events (AEs).

Background DS is a rare childhood-onset developmental epileptic encephalopathy. Polytherapy is common and multiple ASM use is frequently associated with increased AEs.

Design/Methods This retrospective cross-sectional analysis used physician-reported data from the multinational real-world Adelphi DS Disease Specific Programme (DSP^TM). Information on treatment patterns, AEs (from a pre-specified list) during each treatment regimen, non-seizure symptom severity (at the time of survey), and resource use were provided. Data were stratified by number of AEs (0–1; 2; 3+) and compared using bivariate testing.

Results Among 616 patients with DS (mean age 9.7 years, 59% [n=366] male), 63% (n=390) had 0–1 AE, 15% (n=95) 2 AEs, and 21% (n=131) 3+ AEs. Mean number of current ASMs were 2.3, 2.6, and 2.6, respectively (p=0.036); proportions prescribed an orphan drug specifically indicated for DS (stiripentol, cannabidiol, fenfluramine) were 42%, 40%, and 37%, respectively (p=0.582). Mean number of seizure-related injuries experienced (last 6 months) was significantly higher among patients with 3+ AEs (6.4) than among those with 0–1 (1.3) or 2 (1.9; p<0.0001). Considering non-seizure burden (last 4 weeks), the proportions of patients with difficulty speaking and cognitive impairment, and severity of impairment in communication and alertness were significantly higher among patients with 3+ AEs than fewer (all p<0.05). Proportions of patients with healthcare resource use over the last 12 months, including ≥1 hospitalizations (p<0.0001), ≥1 ER visits (p<0.0001), ≥1 outpatient visits (p=0.033), and AEs requiring hospital visit (p=0.0006) were also highest among those with 3+ AEs.

Conclusions Despite recent approval of newer medications for DS, the burden among patients with a higher incidence of AEs can be substantial. New treatment approaches that better manage AEs may improve health outcomes and reduce overall healthcare resource use.

Authors/Disclosures
Arturo I. Benitez, MD (Takeda) PRESENTER	Dr. Benitez has received personal compensation for serving as an employee of Takeda.
Drishti Shah, PhD (Takeda)	Dr. Shah has received personal compensation for serving as an employee of Takeda.
Jeffrey Andrews (Takeda Pharmaceuticals)	No disclosure on file
Jonathan de Courcy	No disclosure on file
Yasmin Taylor	Yasmin Taylor has received personal compensation for serving as an employee of Adelphi Real World.
Hannah Connolly (Adelphi Real World)	Hannah Connolly has nothing to disclose.
Sophie Lai (Adelphi Real World)	No disclosure on file
Vicente Villanueva	Vicente Villanueva has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Vicente Villanueva has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Angelini. Vicente Villanueva has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Xenon. Vicente Villanueva has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Rapport. Vicente Villanueva has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Vicente Villanueva has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Angelini. Vicente Villanueva has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for EISAI. Vicente Villanueva has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Paladin. Vicente Villanueva has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Jazz pharmaceutical.

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