PMID- 36862522
OWN - NLM
STAT- Publisher
LR  - 20240216
IS  - 2378-8763 (Electronic)
IS  - 2378-8763 (Linking)
DP  - 2023 Mar 1
TI  - Cannabidiol Add-On in Glycosylphosphatidylinositol-Related Drug-Resistant 
      Epilepsy.
LID - 10.1089/can.2022.0255 [doi]
AB  - Background: Glycosylphosphatidylinositol-anchored protein deficiencies (GPI-ADs) 
      are commonly associated with drug-resistant epilepsy (DRE). Cannabidiol (CBD) is 
      approved for the adjunctive treatment of seizures in Dravet/Lennox-Gastaut 
      Syndromes and Tuberous Sclerosis Complex. We report on the efficacy and safety of 
      CBD for the treatment of DRE in patients with genetically proven GPI-AD. Patients 
      and Methods: Patients received add-on treatment with purified GW-pharma CBD 
      (Epidyolex((R))). Efficacy endpoints were the percentage of patients with >/=50% 
      (responders) or >25<50% (partial responders) reduction in monthly seizures from 
      baseline and at 12 (M12) months of follow-up. Safety was evaluated through 
      adverse events (AEs) monitoring. Results: Six patients (5 males) were enrolled. 
      The median age at seizures onset was 5 months and the syndromic diagnosis was 
      early infantile developmental and epileptic encephalopathy in 4 patients and 
      focal non-lesional epilepsy or GEFS+ in one patient each. Five out of six (83%) 
      patients were responders at M12, while one was a partial responder. No severe AEs 
      were reported. Mean prescribed CBD dose was 17.85 mg/kg/day and median treatment 
      duration is currently 27 months. Conclusions: In summary, off-label treatment 
      with CBD was effective and safe in patients with DRE due to GPI-ADs.
FAU - Riva, Antonella
AU  - Riva A
AUID- ORCID: 0000-0001-9152-5571
AD  - Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal 
      and Child Health, University of Genoa, Genoa, Italy.
AD  - Pediatric Neurology and Muscular Diseases Unit, IRCCS Istituto "Giannina 
      Gaslini," Genoa, Italy.
FAU - D'Onofrio, Gianluca
AU  - D'Onofrio G
AD  - Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal 
      and Child Health, University of Genoa, Genoa, Italy.
AD  - Pediatric Neurology and Muscular Diseases Unit, IRCCS Istituto "Giannina 
      Gaslini," Genoa, Italy.
FAU - Pisati, Angelica
AU  - Pisati A
AD  - Pediatric Neurology and Muscular Diseases Unit, IRCCS Istituto "Giannina 
      Gaslini," Genoa, Italy.
FAU - Roberti, Roberta
AU  - Roberti R
AD  - Department of Science of Health, School of Medicine, University of Catanzaro, 
      Catanzaro, Italy.
FAU - Amadori, Elisabetta
AU  - Amadori E
AD  - Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal 
      and Child Health, University of Genoa, Genoa, Italy.
AD  - Pediatric Neurology and Muscular Diseases Unit, IRCCS Istituto "Giannina 
      Gaslini," Genoa, Italy.
FAU - Bosch, Friedrich
AU  - Bosch F
AD  - Children's Hospital, Furth, Germany.
FAU - de Souza, Carolina Fischinger Moura
AU  - de Souza CFM
AD  - Medical Genetics Service, HCPA, Porto Alegre, Brazil.
FAU - Thomas, Ashley
AU  - Thomas A
AD  - University of Alabama, Birmingham, Alabama, USA.
FAU - Russo, Emilio
AU  - Russo E
AD  - Department of Science of Health, School of Medicine, University of Catanzaro, 
      Catanzaro, Italy.
FAU - Striano, Pasquale
AU  - Striano P
AD  - Department of Neurosciences, Rehabilitation, Ophthalmology, Genetics, Maternal 
      and Child Health, University of Genoa, Genoa, Italy.
AD  - Pediatric Neurology and Muscular Diseases Unit, IRCCS Istituto "Giannina 
      Gaslini," Genoa, Italy.
FAU - Bayat, Allan
AU  - Bayat A
AD  - Department of Epilepsy Genetics and Personalized Medicine, Danish Epilepsy 
      Center, Dianalund, Denmark.
AD  - Institute for Regional Health Services, University of Southern Denmark, Odense, 
      Denmark.
LA  - eng
PT  - Journal Article
DEP - 20230301
PL  - United States
TA  - Cannabis Cannabinoid Res
JT  - Cannabis and cannabinoid research
JID - 101684827
SB  - IM
OTO - NOTNLM
OT  - GPI
OT  - PIG'opathy
OT  - cannabidiol
OT  - drug-resistant epilepsy
OT  - epileptic encephalopathy
EDAT- 2023/03/03 06:00
MHDA- 2023/03/03 06:00
CRDT- 2023/03/02 12:12
PHST- 2023/03/02 12:12 [entrez]
PHST- 2023/03/03 06:00 [pubmed]
PHST- 2023/03/03 06:00 [medline]
AID - 10.1089/can.2022.0255 [doi]
PST - aheadofprint
SO  - Cannabis Cannabinoid Res. 2023 Mar 1. doi: 10.1089/can.2022.0255.