International forskning

Retrospective chart review study of use of cannabidiol (CBD) independent of concomitant clobazam use in patients with Lennox-Gastaut syndrome or Dravet syndrome

Rima Nabbout 1, Alexis Arzimanoglou 2, Stéphane Auvin 3, Patrick Berquin 4, Archana Desurkar 5, Douglas Fuller 6, Charlotte Nortvedt 7, Patrizia Pulitano 8, Anna Rosati 9, Victor Soto 10, Vicente Villanueva 11, J Helen Cross 12

  • 1Assistance Publique-Hôpitaux de Paris (APHP), Member of the European Reference Network (ERN) EpiCARE, Necker Enfants Malades University Hospital, Université Paris Cité, 149 Rue de Sèvres, 75015 Paris, France; INSERM U1163, Institut Imagine, 24 Bd du Montparnasse, 75015 Paris, France. Electronic address:
  • 2Paediatric Epilepsy Department, Coordinator of the ERN EpiCARE, University Hospitals of Lyon, 59 Bd Pinel, 60700 Lyon, France; Epilepsy Research Unit, Member of the ERN EpiCARE, Hospital San Juan de Déu, Passeig de Sant Joan de Déu, 2, 08950 Esplugues de Llobregat, Barcelona, Spain.
  • 3APHP, Service de Neurologie Pédiatrique, Member of the ERN EpiCARE, Hôpital Robert Debré, 48 Bd Sérurier, 75019 Paris, France; INSERM NeuroDiderot, Université Paris-Cité, 48 Bd Sérurier, 75019 Paris, France; Institut Universitaire de France (IUF), 1 Rue Descartes, Île-de-France, 75005 Paris, France.
  • 4Centre Hospitalier Universitaire (CHU) Amiens-Picardie Site Sud, Service de Neurologie Pédiatrique, 1 Rond Point du Pr Christian Cabrol, 80054 Amiens CEDEX, France.
  • 5Sheffield Children’s Hospital NHS Foundation Trust, Clarkson St, Broomhall, Sheffield S10 2TH, UK.
  • 6Jazz Pharmaceuticals, Inc., 3170 Porter Dr, Palo Alto, CA 94304, USA.
  • 7Jazz Pharmaceuticals, Inc., 1 Cavendish Place, London W1G 0QF, UK.
  • 8Department of Human Neurosciences, Policlinico Umberto I, Sapienza University of Rome, Viale Regina Elena, 324, 00161 Roma RM, Italy.
  • 9Neuroscience Department, Children’s Hospital Anna Meyer, University of Florence, Viale Gaetano Pieraccini, 24, 50139 Firenze FI, Italy.
  • 10Department of Neurology, Member of the ERN EpiCARE, Hospital Universitario Niño Jesus, Av. de Menéndez Pelayo, 65, 28009 Madrid, Spain.
  • 11Refractory Epilepsy Unit, Hospital Universitario y Politécnico La Fe, Avenida Fernando Abril Martorell, 106, 46026 València, Spain.
  • 12UCL, National Institute of Health and Social Care Research (NIHR) Great Ormond Street Biomedical Research Centre (BRC), Institute of Child Health, 30 Guilford St, London WC1N 1EH, UK.


Purpose: This retrospective chart review study (GWEP20052) evaluated plant-derived highly purified cannabidiol (CBD; Epidyolex®; 100 mg/mL oral solution) use without clobazam as add-on therapy in patients aged ≥2 years with Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS) enrolled in a European Early Access Program.

Methods: Data were extracted from patient charts covering a period starting 3 months before CBD treatment and concluding after 12 months of CBD treatment, or sooner if a patient discontinued CBD or started clobazam.

Results: Of 114 enrolled patients, data were available for 107 (92 LGS, 15 DS) who received CBD without clobazam for ≥3 months. Mean age: 14.5 (LGS) and 10.5 (DS) years; female: 44% (LGS) and 67% (DS). Mean time-averaged CBD dose: 13.54 (LGS) and 11.56 (DS) mg/kg/day. Median change from baseline in seizure frequency per 28 days over 3-month intervals varied from -6.2% to -20.9% for LGS and 0% to -16.7% for DS. Achievement of ≥50% reduction in drop (LGS) or convulsive (DS) seizures at 3 and 12 months: LGS, 19% (n = 69) and 30% (n = 53); DS, 21% (n = 14) and 13% (n = 8). Retention on CBD without clobazam (enrolled set): 94%, 80%, 69%, and 63% at 3, 6, 9, and 12 months. Adverse event (AE) incidence was 31%, most commonly somnolence, seizure, diarrhea, and decreased appetite. Two patients discontinued CBD owing to AEs, and four patients with LGS experienced elevated liver enzymes.

Conclusion: Results support favorable effectiveness and retention of CBD without concomitant clobazam for up to 12 months in clinical practice.

Keywords: Cannabidiol; Clobazam; Dravet syndrome; Lennox-Gastaut syndrome; Real-world evidence; Seizure.