Placebo response in patients with Dravet syndrome: Post-hoc analysis of two clinical trials - Université de Paris - Faculté de Santé Access content directly
Journal Articles Epilepsy & Behavior Year : 2024

Placebo response in patients with Dravet syndrome: Post-hoc analysis of two clinical trials

Orrin Devinsky
  • Function : Author
Kerry Hyland
  • Function : Author
Rachael Loftus
  • Function : Author
Charlotte Nortvedt
  • Function : Author
  • PersonId : 1384061

Abstract

Objective Dravet syndrome is a rare, early childhood-onset epileptic and developmental encephalopathy. Responses to placebo in clinical trials for epilepsy therapies range widely, but factors influencing placebo response remain poorly understood. This study explored placebo response and its effects on safety, efficacy, and quality of life outcomes in patients with Dravet syndrome. Methods We performed exploratory post-hoc analyses of pooled data from placebo-treated patients from the GWPCARE 1B and GWPCARE 2 randomized controlled phase III trials, comparing cannabidiol and matched placebo in 2–18 year old Dravet syndrome patients. All patients had ≥4 convulsive seizures during a baseline period of 4 weeks. Results 124 Dravet syndrome-treated patients were included in the analysis (2–5 years: n = 35; 6–12 years: n = 52; 13–18 years: n = 37). Convulsive seizures were experienced by all placebo group patients at all timepoints, with decreased median convulsive seizure frequency during the treatment period versus baseline; the number of convulsive seizure-free days was similar to baseline. Convulsive seizure frequency had a nominally significant positive correlation with age and a nominally significant negative correlation with body mass index. Most placebo-treated patients experienced a treatment-emergent adverse event; however, most resolved quickly, and serious adverse events were infrequent. Placebo treatment had very little effect on reported Caregiver Global Impression of Change outcomes versus baseline. Interpretation Placebo had little impact on convulsive seizure-free days and Caregiver Global Impression of Change versus baseline, suggesting that these metrics may help differentiate placebo and active treatment effects in future studies. However, future research should further assess placebo responses to confirm these results.
Fichier principal
Vignette du fichier
PIIS1525505024001860.pdf (438.15 Ko) Télécharger le fichier
Origin Publisher files allowed on an open archive
licence

Dates and versions

hal-04581736 , version 1 (21-05-2024)

Licence

Identifiers

Cite

Orrin Devinsky, Kerry Hyland, Rachael Loftus, Charlotte Nortvedt, Rima Nabbout. Placebo response in patients with Dravet syndrome: Post-hoc analysis of two clinical trials. Epilepsy & Behavior, 2024, 156, pp.109805. ⟨10.1016/j.yebeh.2024.109805⟩. ⟨hal-04581736⟩

Collections

INSERM UP-SANTE
0 View
0 Download

Altmetric

Share

Gmail Mastodon Facebook X LinkedIn More