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dc.creatorScheffer, I.E. (Ingrid E.)-
dc.creatorHalford, J.J. (Jonathan J.)-
dc.creatorMiller, I. (Ian)-
dc.creatorNabbout, R. (Rima)-
dc.creatorSanchez-Carpintero, R. (Rocío)-
dc.creatorShiloh-­Malawsky, Y. (Yael)-
dc.creatorWong, M. (Matthew)-
dc.creatorZolnowska, M. (Marta)-
dc.creatorChecketts, D. (Daniel)-
dc.creatorDunayevich, E. (Eduardo)-
dc.creatorDevinsky, O. (Orrin)-
dc.identifier.citationScheffer IE, Halford JJ, Miller I, Nabbout R, Sanchez-Carpintero R, ShilohMalawsky Y, et al. Add-on cannabidiol in patients with Dravet syndrome: Results of a long-term open-label extension trial. Epilepsia. 2021;62:2505– 2517.es_ES
dc.description.abstractObjective: Add-on cannabidiol (CBD) reduced seizures associated with Dravet syndrome (DS) in two randomized, double-blind, placebo-controlled trials: GWPCARE1 Part B (NCT02091375) and GWPCARE2 (NCT02224703). Patients whocompletedGWPCARE1 PartA(NCT02091206)orPartB,orGWPCARE2,were enrolled in a long-term open-label extension trial, GWPCARE5 (NCT02224573). We present an interim analysis of the safety, efficacy, and patient-reported outcomes from GWPCARE5. Methods: Patientsreceived a pharmaceutical formulation of highly purified CBD in oral solution (100 mg/ml), titrated from 2.5 to 20 mg/kg/day over a 2-week period, added to their existing medications. Based on response and tolerance, CBD could be reduced or increased to 30 mg/kg/day. Results: Of the 330 patients who completed the original randomized trials, 315 (95%) enrolled in this open-label extension. Median treatment duration was 444 days (range = 18–1535), with a mean modal dose of 22 mg/kg/day; patients received a median of three concomitant antiseizure medications. Adverse events (AEs)occurredin97%patients(mild,23%;moderate,50%;severe,25%).Commonly reported AEs were diarrhea (43%), pyrexia (39%), decreased appetite (31%), and somnolence (28%). Twenty-eight (9%) patients discontinued due to AEs. Sixtynine (22%) patients had liver transaminase elevations >3 × upper limit of normal; 84% were on concomitant valproic acid. In patients from GWPCARE1 Part B and GWPCARE2, the median reduction from baseline in monthly seizure frequency assessed in 12-week periods up to Week 156 was 45%–74% for convulsive seizures and 49%–84% for total seizures. Across all visit windows, ≥83% patients/caregivers completing a Subject/Caregiver Global Impression of Change scale reported improvement in overall condition. Significance: We show that long-term CBD treatment had an acceptable safety profile and led to sustained, clinically meaningful reductionsin seizure frequency in patients with treatment-resistant DS.es_ES
dc.description.sponsorshipThe authorsthank the patients and theirfamilies who took part in the trial, as well as the staff at the clinical research sites; and Alchemy Medical Writing for medical writing and editorial support, funded by Greenwich Biosciences. The views expressed are those of the authors. The trial was sponsored by GW Research.es_ES
dc.subjectAntiseizure medicationes_ES
dc.subjectChildhood onset epilepsyes_ES
dc.subjectConvulsive seizureses_ES
dc.subjectDravet syndromees_ES
dc.titleAdd-­on cannabidiol in patients with Dravet syndrome: Results of a long-­term open-­label extension triales_ES
dc.description.noteThis is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs Licensees_ES

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