Authors :
Presenting Author: Steven Wolf, MD – Boston Children's Health Physicians; Westchester Medical Center Health Network
Patricia McGoldrick, NP, MPA, MSN, FAES – Boston Children's Health Physicians; Westchester Medical Center Health Network
Syndi Seinfeld, DO – Joe DiMaggio Children’s Hospital
Katherine Labiner, MD – Child Neurology Consultants of Austin
Neel Parikh, MD – Johns Hopkins All Children's Hospital
Muhammad Zafar, MD – Duke University School of Medicine
Kenneth Crist, PharmD – Assertio Holdings Inc.
Howard Franklin, MD – Assertio Holdings Inc.
Rationale:
Lennox-Gastaut syndrome is a rare, severe childhood-onset epilepsy marked by frequent treatment-resistant seizures and cognitive impairment, affecting 2.9–28 patients (pts) per 100,000 individuals. Historically, clobazam treatment for seizures was available only as oral tablets or suspension. A new oral soluble film formulation is an alternative for pts with dysphagia—and may benefit pts with developmental delays, autism, or severe sensory processing conditions—who vomit medicines. This study compared the side effects of antiseizure medications (ASMs) and comorbidities in pts with underlying epilepsy treated with clobazam as oral soluble film or clobazam pill/suspension.
Methods:
Using a large database of ~66 million pts, we conducted a retrospective cohort study using routine care electronic health records collected at Stanford Health Care (2015–2024). Demographics, ASM use and associated symptoms, and comorbidities of pts with epilepsy taking clobazam oral soluble film or pill/suspension were extracted and described with summary statistics and propensity score–matched odds ratios (ORs).
Results:
Pts taking clobazam oral film (n=97) had a mean (SD) age of 23.1 (20.8) y; 55% were female. Pts taking clobazam pill/suspension (n=15,910) had a mean (SD) age of 26.7 (19.9) y; 50% were female.
The clobazam oral film group had higher use of cannabidiol (10%) than the pill/suspension group (3%). Additional ASMs in the oral film versus pill/suspension groups included levetiracetam (18% vs 22%, respectively) and zonisamide (6% vs 8%, respectively).
Regarding safety, when comparing pts treated with clobazam oral film versus pill/suspension, no statistically significant differences were observed in the incidence of constipation (8% vs 10%, respectively; odds ratio [OR]=1.23; P=1), fatigue (9% each; OR=1.58; P=0.74), pyrexia (9% vs 8%, respectively; OR=0.572; P=0.71), or somnolence (2% vs 4%, respectively; OR=1; P=1).
Additionally, lower prevalence of mood/neurobehavioral conditions (depression, anxiety, ADHD, conduct disorders) was observed in the film group versus pill/suspension group (13% vs 19%,respectively; P=0.015). In particular, the clobazam pill/suspension group had higher prevalence of ADHD than the oral film group (4% vs 1%, respectively; P=0.055); albeit a lower mean age in the oral film group may have affected the rate of diagnosis. Both groups had high prevalence of comorbid conditions, such as cerebral palsy (film: 14%; pill/suspension: 16%), hemiparaplegia (film: 7%; pill/suspension: 13%), and chronic pulmonary disease (film: 24%; pill/suspension: 16%).
Conclusions:
In this real-world study of pts with epilepsy, similar rates of ASM side effects such as constipation, fatigue, pyrexia, and somnolence, but lower prevalence of mood/neurobehavioral conditions, were observed among pts taking clobazam oral film compared with clobazam pill/suspension. Clobazam oral film can benefit pts with difficulty ingesting pills or liquid medication.
Funding:
Assertio Holdings, Inc.