Real-world Insights On The Use Of Cannabidiol (CBD) In Children Aged <2 Years With LGS, DS, Or TSC: A Physician Survey

Introduction CBD (Epid[iy]olex® 100 mg/mL oral solution) is approved for the treatment of Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC) in patients (pts) aged >=2 years (EU & AUS) / >=1 year (USA). Most CBD treatment outcomes data are from paediatric pts. This study obtained insights on care and CBD treatment in adults with LGS, DS, or TSC. Methods An online survey was completed by physicians treating pts aged >=18 years. Results Seven physicians from Australia, Israel, Italy, and the USA were included in the analyses, representing 119 pts treated with CBD (LGS n=55, DS n=41, TSC n=23; median age 25 years). Major pt management challenges reported included use of multiple antiseizure medications (ASMs), suboptimal seizure control, risk of sudden unexpected death in epilepsy, poor pt cooperation, and non-seizure comorbidities. Median (range) CBD dosage was 12 (3–20) mg/kg/day (n=75). On reaching maintenance, other ASMs were withdrawn in 16% (n=12/75) of represented pts. After CBD treatment, physicians perceived in most pts an increase in seizure-free days and a decrease/no increase in seizure duration; decreases in pain/discomfort and anxiety/depression were also reported (Table). The reported TEAEs were somnolence, change in ALT/AST, diarrhoea, fatigue, and decreased appetite. Conclusion This physician survey highlights challenges in the treatment of adults with LGS, DS, TSC. CBD improved seizure/non-seizure outcomes. TEAEs were consistent with the known safety profile of CBD. Funding: Jazz Pharmaceuticals, Inc.

Kishan Vyas
Jazz Pharmaceuticals UK Ltd.
United Kingdom

Alexis Arzimanoglou
Barcelona Children’s Hospital Sant Joan de Déu, Universitat de Barcelona, and European Reference Network EpiCARE
Spain

Patrick Kwan
Monash University
Australia

Rima Nabbout
Necker Enfants Malades, Assistance Publique – Hôpitaux de Paris, Université Paris Cité, and European Reference Network EpiCARE
France

Pasquale Striano
University of Genova, and European Reference Network EpiCARE
Italy

Cameron Costello
Costello Medical
United Kingdom

Declan Summers
Costello Medical
United Kingdom

Sophie Schoeni
Costello Medical
United States

Charlotte Nortvedt
Jazz Pharmaceuticals UK Ltd.
United Kingdom

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Rima Nabbout
Necker Enfants Malades, Assistance Publique – Hôpitaux de Paris, Université Paris Cité, and European Reference Network EpiCARE
France
  • About the Author: Rima Nabbout