Perceived efficacy of cannabidiol-enriched cannabis extracts for treatment of pediatric epilepsy: A potential role for infantile spasms and Lennox-Gastaut syndrome.
There is a great need for safe and effective therapies for treatment of infantile spasms (IS) and Lennox-Gastaut syndrome (LGS). Based on anecdotal reports and limited experience in an open-label trial, cannabidiol (CBD) has received tremendous attention as a potential treatment for pediatric epilepsy, especially Dravet syndrome. However, there is scant evidence of specific utility for treatment of IS and LGS. We sought to document the experiences of children with IS and/or LGS who have been treated with CBD-enriched cannabis preparations. We conducted a brief online survey of parents who administered CBD-enriched cannabis preparations for the treatment of their children's epilepsy. We specifically recruited parents of children with IS and LGS and focused on perceived efficacy, dosage, and tolerability. Survey respondents included 117 parents of children with epilepsy (including 53 with IS or LGS) who had administered CBD products to their children. Perceived efficacy and tolerability were similar across etiologic subgroups. Eighty-five percent of all parents reported a reduction in seizure frequency, and 14% reported complete seizure freedom. Epilepsy was characterized as highly refractory with median latency from epilepsy onset to CBD initiation of five years, during which the patient's seizures failed to improve after a median of eight antiseizure medication trials. The median duration and the median dosage of CBD exposure were 6.8 months and 4.3mg/kg/day, respectively. Reported side effects were far less common during CBD exposure, with the exception of increased appetite (30%). A high proportion of respondents reported improvement in sleep (53%), alertness (71%), and mood (63%) during CBD therapy. Although this study suggests a potential role for CBD in the treatment of refractory childhood epilepsy including IS and LGS, it does not represent compelling evidence of efficacy or safety. From a methodological standpoint, this study is extraordinarily vulnerable to participation bias and limited by lack of blinded outcome ascertainment. Appropriately controlled clinical trials are essential to establish efficacy and safety.
Division of Pediatric Neurology, Mattel Children's Hospital at UCLA, David Geffen School of Medicine, Los Angeles, CA, USA. Electronic address: shussain@mednet.ucla.edu.
Shaun A
SA
Hussain
Division of Pediatric Neurology, Mattel Children's Hospital at UCLA, David Geffen School of Medicine, Los Angeles, CA, USA.
Raymond
R
Zhou
Division of Pediatric Neurology, Mattel Children's Hospital at UCLA, David Geffen School of Medicine, Los Angeles, CA, USA.
Catherine
C
Jacobson
Division of Pediatric Neurology, Mattel Children's Hospital at UCLA, David Geffen School of Medicine, Los Angeles, CA, USA.
Julius
J
Weng
Division of Pediatric Neurology, Mattel Children's Hospital at UCLA, David Geffen School of Medicine, Los Angeles, CA, USA.
Emily
E
Cheng
Division of Pediatric Neurology, Mattel Children's Hospital at UCLA, David Geffen School of Medicine, Los Angeles, CA, USA.
Johnson
J
Lay
Division of Pediatric Neurology, Mattel Children's Hospital at UCLA, David Geffen School of Medicine, Los Angeles, CA, USA.
Phoebe
P
Hung
Division of Pediatric Neurology, Mattel Children's Hospital at UCLA, David Geffen School of Medicine, Los Angeles, CA, USA.
Jason T
JT
Lerner
Division of Pediatric Neurology, Mattel Children's Hospital at UCLA, David Geffen School of Medicine, Los Angeles, CA, USA.
Raman
R
Sankar
D000293:Adolescent; D000339:Affect; D017668:Age of Onset; D000927:Anticonvulsants / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D001288:Attention; D002185:Cannabidiol / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D002188:Cannabis / Q000737:chemistry*; D002648:Child; D000069279:Drug Resistant Epilepsy / Q000188:drug therapy; D004827:Epilepsy / Q000188:drug therapy* / Q000523:psychology; D005260:Female; D019538:Health Care Surveys; D006801:Humans; D007223:Infant; D065768:Lennox Gastaut Syndrome / Q000150:complications / Q000188:drug therapy*; D008297:Male; D010936:Plant Extracts / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D012640:Seizures / Q000453:epidemiology; D012890:Sleep; D013036:Spasms, Infantile / Q000150:complications / Q000188:drug therapy*; D013577:Syndrome; D055815:Young Adult
D000927:Anticonvulsants; D010936:Plant Extracts; D002185:Cannabidiol
Cannabidiol; Cannabis; Infantile spasms; Lennox–Gastaut syndrome; Marijuana; Survey; West syndrome