Annual Meeting Abstracts: View

  • (Abst. 2.372), 2014
  • POTENTIAL EFFICACY OF CANNABIDIOL FOR TREATMENT OF REFRACTORY INFANTILE SPASMS AND LENNOX GASTAUT SYNDROME
  • Authors: Raymond Zhou, Catherine Jacobson, Julius Weng, Emily Cheng, Johnson Lay, Phoebe Hung, Jason Lerner, Raman Sankar and Shaun Hussain
  • Content:

    Rationale: 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, and most notably Dravet syndrome. There is however 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. Methods: 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. Results: Survey respondents included 117 parents of children with epilepsy who had administered CBD products to their children and consented to share their experiences in our survey. We identified 53 children whose parents reported a diagnosis of IS and/or LGS. 92% of parents reported a reduction in seizures, and 13% reported complete seizure-freedom. 89% reported that improvement (or worsening) occurred within 2 weeks. The median age of epilepsy onset was 4.0 months (IQR 3.0-7.0), and the median number of failed medications prior to CBD was 8 (IQR 5-12). Among those with IS (n = 45), the percentage of patients who specifically failed hormonal therapy (prednisolone and/or ACTH) and vigabatrin before CBD use was 62% and 73%, respectively. Among the larger cohort (IS and/or LGS), 57% had failed the ketogenic diet and 15% had failed surgery (not including VNS) prior to CBD use. The median age at CBD initiation was 3.6 years (IQR 2.0-4.3); the youngest patient was 5 months old. The median duration of therapy was 6.9 months (IQR 4.3-9.3). 85% of respondents reported using preparations with a CBD:THC ratio of at least 15:1. Among a limited subset of respondents (n = 20) who reported both weight and CBD dosage (mg/day), the median weight-based CBD dosage was 4.0 mg/kg/day (IQR 2.6-7.2). 94% of parents continued to administer CBD at the time of survey completion. Parents were asked about the presence or absence of 16 potential treatment emergent adverse events (TEAEs) commonly associated with AEDs. Parents reported a median of 5 TEAEs (IQR 3 - 10) present before CBD exposure, and a median of 1 TEAE (IQR 0-2) during CBD therapy; 76% of parents reported successfully tapering at least 1 medication after beginning CBD therapy. A high proportion of respondents reported improvement in sleep (53%), alertness (77%), and mood (64%) during CBD therapy. Conclusions: Although this study suggests a potential role for CBD in the treatment of IS and LGS, it does not represent compelling evidence of efficacy or safety. From a methodological standpoint, this study is extraordinarily vulnerable to selection (participation) bias and placebo effect. Further study is clearly warranted and appropriately controlled clinical trials are essential to establish efficacy and safety.