Health Outcomes Stratified by Number of Adverse Events Experienced by Patients with Dravet Syndrome: Insights from a Global Real-world Study
Drishti Shah1, J. Scott Andrews1, Arturo Benitez1, Jonathan DeCourcy2, Yasmin Taylor2, Hannah Connolly2, Sophie Lai2, Vicente Villanueva3
1Takeda Development Center Americas, Inc., 2Adelphi Real World, 3Hospital Universitario y Politécnico La Fe
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 (DSPTM). 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.