Open, Prospective Study of CBD Use in Dravet Syndrome, Lennox-gastaut Syndrome, Tuberous Sclerosis and Other Drug-resistant Epilepsies
Abstract number :
2.384
Submission category :
7. Anti-seizure Medications / 7C. Cohort Studies
Year :
2024
Submission ID :
938
Source :
www.aesnet.org
Presentation date :
12/8/2024 12:00:00 AM
Published date :
Authors :
Presenting Author: Rima Nabbout, MD, PhD – Member of European Reference Network EpiCARE, Reference Centre for Rare Epilepsies, Necker Enfants Malades Hospital, APHP, U 1163 Institut Imagine, Université Paris Cité
Nicole Chemaly, MD, PhD – APHP, Necker Enfants Malades Hospital, member of the European Reference Center EpiCARE, Institut Imagine INSERM 1163, Université Paris Cité, Paris, France
Emma Losito, MD – APHP, Necker Enfants Malades Hospital, member of the European Reference Center EpiCARE, Institut Imagine INSERM 1163, Université Paris Cité, Paris, France
Anna Kaminska, MD, PhD – APHP, Necker Enfants Malades Hospital, member of the European Reference Center EpiCARE, Université Paris Cité, Paris, France
Coste Zeitoun Delphine, MD – APHP, Necker Enfants Malades Hospital, member of the European Reference Center EpiCARE, Université Paris Cité, Paris, France
isabelle Desguerre, MD, PhD – APHP, Necker Enfants Malades Hospital, member of the European Reference Center EpiCARE, Université Paris Cité, Paris, France
Marie Hully, MD – APHP, Necker Enfants Malades Hospital, member of the European Reference Center EpiCARE, Université Paris Cité, Paris, France
Mathieu Kuchenbuch, MD, PHD – APHP, Necker Enfants Malades Hospital, member of the European Reference Center EpiCARE, Université Paris Cité, Paris, France
Rationale: In the EU, CBD is approved for use in combination with clobazam for Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS), and as an add-on treatment for tuberous sclerosis complex (TSC) in patients aged 2 and older. We aimed to evaluate the retention rate of CBD in a pediatric population with drug-resistant epilepsies using a family-structured survey to assess efficacy and safety.
Methods: Methods: This prospective open study is based on caregivers reported-outcome measures through a questionnaire used at month 1 2 and 6 to introduction of CBD. This questionnaire addressed the impact on seizures, their frequency, duration and severity and on major non-seizure outcomes as sleep, communication, behavior, appetite, awakening, motor skills and language using a 7-points Likert scale (from -3: major aggravation to 3: major improvement).
We included pediatric patients receiving CBD as adjunctive therapy for drug-resistant epilepsies between 2019 and 2021. To explore factors associated with the retention rate, we used Cox models with the factor presence or absence of clobazam comedication, age and category of epilepsy syndromes.
Results: We included 103 individuals, including 52 males with a mean age of 11.2 years (interquartile range: 7.1-15.4 years). In 50% of case, CBD was used in the label (47.9% DS, 31.3% LGS and 20.8% TSC) and 53.4% off label (n=55/103). One month after the start of treatment, caregivers reported a predominant improvement in seizures (frequency and duration) and overall impression. Between month 1 and month 2, there was an improvement in overall impression as well as in communication and alertness. These non-seizure-related improvements continued between month 2 and month 6. Discontinuation rates of CBD were 3%, 10%, 18%, 34%, and 45% at 1, 2, 6, 12, and 18 months, respectively. Off-label use (p=0.005, OR= 2.8) and age at introduction (p=0.06, OR=1.1) were positively associated with retention, whereas adjunctive therapy with Clobazam did not show a significant effect (p=0.99).
Conclusions: In this population, including those with market access syndrome, CBD demonstrates a good retention rate at 18 months. Beyond this study consolidate the use of CBD in clinical practice for pediatric drug epilepsies with good efficacy and tolerability.
Funding: This research received no specific grant from any funding agency in the public, commercial, or not-for-profit sectors
Anti-seizure Medications