Objective:

To quantify the incremental treatment burden of adverse events (AEs) among patients with Lennox-Gastaut syndrome (LGS) receiving antiseizure medication (ASM).

Background:

LGS is a severe form of treatment-resistant childhood-onset epilepsy. Use of multiple ASMs frequently results in AEs and drug–drug interactions.

Design/Methods:

Data for this retrospective cross-sectional analysis were derived from the real-world Adelphi LGS Disease Specific Programme (DSP^TM). Neurologists/pediatric neurologists provided information on treatment patterns, AEs (from a pre-specified list) per treatment regimen, non-seizure symptoms, and resource use. Data were stratified by number of AEs (0–1; 2; 3+) irrespective of number of ASMs prescribed and compared using bivariate testing.

Results:

Of 803 patients (mean age 14.7 years), 117 (15%) experienced 2 AEs and 139 (17%) 3+ AEs. Mean number of current ASMs per patient was similar across groups (0–1 AEs, 2.5; 2 AEs, 2.4; 3+ AEs, 2.6). Mean number of seizure-related injuries experienced (last 6 months) was significantly higher among patients with 3+ AEs (overall, 7.0; head injuries, 1.5) than with 0–1 (1.3 and 0.3, respectively) or 2 AEs (2.3 and 0.6) (p<0.0001). Non-seizure burden (last 4 weeks) was also higher among patients with 3+ AEs versus 0–1 or 2 AEs, especially the proportions of patients having difficulty with gait, dysphasia, and cognitive impairment (all p<0.05). The proportions of patients with ≥1 hospitalization event, ≥1 ER visits, and ≥1 outpatient visits over the last 12 months were highest among those with 3+ AEs (all p<0.0001). Proportions with very poor to somewhat poor quality of life (p=0.0006) were also highest among those with 3+ AEs.

Conclusions:

Despite similar ASM use across groups, patients with more AEs (versus fewer) experienced a substantial clinical burden and greater healthcare resource use suggesting a potentially more vulnerable population. There is a clear need for new LGS treatments with a better tolerability profile.