Full metadata record
DC Field | Value | Language |
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dc.creator | Scheffer, I.E. (Ingrid E.) | - |
dc.creator | Halford, J.J. (Jonathan J.) | - |
dc.creator | Miller, I. (Ian) | - |
dc.creator | Nabbout, R. (Rima) | - |
dc.creator | Sanchez-Carpintero, R. (Rocío) | - |
dc.creator | Shiloh-Malawsky, Y. (Yael) | - |
dc.creator | Wong, M. (Matthew) | - |
dc.creator | Zolnowska, M. (Marta) | - |
dc.creator | Checketts, D. (Daniel) | - |
dc.creator | Dunayevich, E. (Eduardo) | - |
dc.creator | Devinsky, O. (Orrin) | - |
dc.date.accessioned | 2024-02-08T09:47:51Z | - |
dc.date.available | 2024-02-08T09:47:51Z | - |
dc.date.issued | 2021 | - |
dc.identifier.citation | Scheffer 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.identifier.issn | 0013-9580 | - |
dc.identifier.uri | https://hdl.handle.net/10171/68905 | - |
dc.description.abstract | Objective: 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.sponsorship | The 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.language.iso | eng | es_ES |
dc.publisher | Willey | es_ES |
dc.rights | info:eu-repo/semantics/openAccess | es_ES |
dc.subject | Antiseizure medication | es_ES |
dc.subject | Cannabinoid | es_ES |
dc.subject | Childhood onset epilepsy | es_ES |
dc.subject | Convulsive seizures | es_ES |
dc.subject | Dravet syndrome | es_ES |
dc.title | Add-on cannabidiol in patients with Dravet syndrome: Results of a long-term open-label extension trial | es_ES |
dc.type | info:eu-repo/semantics/article | es_ES |
dc.description.note | This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License | es_ES |
dc.identifier.doi | 10.1111/epi.17036 | - |
dc.identifier.pmid | 34406656 | - |
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