Caregiver-reported non-seizure and seizure outcomes with cannabidiol and clobazam in patients aged ≥2 years with Lennox-Gastaut syndrome or Dravet syndrome: A subgroup analysis of the BECOME survey
M Scott Perry 1, Tracy Dixon-Salazar 2, Mary Anne Meskis 3, Sherry R Danese 4, Timothy B Saurer 5, Kishan Vyas 6, Anne T Berg 7
- 1Cook Children’s Medical Center, Fort Worth, TX, USA. Electronic address: Scott.Perry@cookchildrens.org.
- 2Lennox-Gastaut Syndrome Foundation, San Diego, CA, USA.
- 3Dravet Syndrome Foundation, Cherry Hill, NJ, USA.
- 4Outcomes Insights, Agoura Hills, CA, USA.
- 5Jazz Pharmaceuticals, Inc., Carlsbad, CA, USA.
- 6Jazz Pharmaceuticals, UK Ltd., London, UK.
- 7Decoding Developmental Epilepsies, WA D.C., USA.
Affiliationer
Purpose: This subgroup analysis of the BECOME (BEhavior, COgnition, and More with Epidiolex®) survey analyzed caregiver-reported seizure and non-seizure outcomes, including changes in caregiver burden, following cannabidiol (CBD) treatment in patients with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) aged ≥2 years taking concomitant clobazam, aligned with the European Medicines Agency indication.
Methods: US-based caregivers of patients with LGS/DS receiving CBD (Epidiolex®, 100 mg/mL oral solution) for ≥3 months rated patient outcome changes, comparing the previous month with pre-CBD initiation. Multiple-choice and rank-order questions with symmetrical 3-, 5-, or 7-point scales ranging from worsening to improvement were used.
Results: Patients (N = 243; 76 % LGS, 24 % DS; 52 % male; mean [range] age 15 [2-53] years) took a median of 14 mg/kg/day CBD and a median of four other antiseizure medications, including clobazam. Caregivers reported improvements in seizure frequency (87 %), severity (81 %), and weekly seizure-free days (net improvement across seizure types 68 %). Caregivers noted improvements in ≥1 question for non-seizure domains (net data across questions within each domain): alertness, cognition, and executive function (84 %); language and communication in non-verbal (81 %) and verbal (76 %) patients; emotional and social functioning (79 %); daily activities (56 %); physical functioning (44 %); and sleep (56 %). Additionally, 4-26 % of caregivers (net) reported worsening in ≥1 question in any domain. Overall, 94 % of caregivers planned to continue CBD treatment.
Conclusions: Consistent with the overall BECOME survey, most caregivers of patients with LGS/DS aged ≥2 years taking concomitant clobazam reported improvements in seizure and non-seizure outcomes since initiating CBD treatment.
