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Epilepsy Behav. 2018 Sep;86:131-137. doi: 10.1016/j.yebeh.2018.05.013. Epub 2018 Jul 11.

Open-label use of highly purified CBD (Epidiolex®) in patients with CDKL5 deficiency disorder and Aicardi, Dup15q, and Doose syndromes.

Author information

1
New York University School of Medicine, NY, United States. Electronic address: od4@nyu.edu.
2
New York University School of Medicine, NY, United States. Electronic address: Chloe.verducci@nyumc.org.
3
Massachusetts General Hospital, MA, United States. Electronic address: ETHIELE@mgh.harvard.edu.
4
Lurie Children's Hospital of Chicago, IL, United States. Electronic address: LLaux@luriechildrens.org.
5
Nationwide Children's Hospital, OH, United States. Electronic address: Anup.Patel@nationwidechildrens.org.
6
Utah School of Medicine, UT, United States. Electronic address: Francis.Filloux@hsc.utah.edu.
7
UAB Epilepsy Center, 1719 6th Avenue South, CIRC 312, Birmingham, AL 35294, United States. Electronic address: jszaflarski@uabmc.edu.
8
Texas Children's Hospital, Houston, TX, United States; Baylor College of Medicine, Houston, TX, United States. Electronic address: awilfong@phoenixchildrens.com.
9
Neurology and Neuroscience, Baylor College of Medicine, TX, United States; Neurology Service, Texas Children's Hospital, United States; Professors of Child Neurology, United States. Electronic address: gdclark@texaschildrens.org.
10
Neurology and Pediatrics, Medical College of Georgia, Augusta University, GA, United States; Child Neurology, Medical College of Georgia, Augusta University, United States. Electronic address: YPARK@augusta.edu.
11
University of Rochester Medical Center, NY, United States. Electronic address: Laurie_Seltzer@URMC.Rochester.edu.
12
Neurology and Pediatrics, UAB Epilepsy Center, Birmingham, AL 35294, United States. Electronic address: ebebin@uabmc.edu.
13
PANDA Neurology (Pediatric and Adolescent NeuroDevelopmental Associates), Atlanta, GA, United States.
14
Consultants in Epilepsy & Neurology, 1499 West Hays Street, Boise, ID 83702, United States; Idaho Comprehensive Epilepsy Center, 1499 West Hays Street, Boise, ID 83702, United States.
15
New York University School of Medicine, NY, United States. Electronic address: Daniel.friedman@nyumc.org.

Abstract

OBJECTIVE:

We studied our collective open-label, compassionate use experience in using cannabidiol (CBD) to treat epilepsy in patients with CDKL5 deficiency disorder and Aicardi, Doose, and Dup15q syndromes.

METHODS:

We included patients aged 1-30 years with severe childhood-onset epilepsy who received CBD for ≥10 weeks as part of multiple investigator-initiated expanded access or state access programs for a compassionate prospective interventional study: CDKL5 deficiency disorder (n = 20), Aicardi syndrome (n = 19), Dup15q syndrome (n = 8), and Doose syndrome (n = 8). These patients were treated at 11 institutions from January 2014 to December 2016.

RESULTS:

The percent change in median convulsive seizure frequency for all patients taking CBD in the efficacy group decreased from baseline [n = 46] to week 12 (51.4% [n = 35], interquartile range (IQR): 9-85%) and week 48 (59.1% [n = 27], IQR: 14-86%). There was a significant difference between the percent changes in monthly convulsive seizure frequency during baseline and week 12, χ2(2) = 22.9, p = 0.00001, with no difference in seizure percent change between weeks 12 and 48. Of the 55 patients in the safety group, 15 (27%) withdrew from extended observation by week 144: 4 due to adverse effects, 9 due to lack of efficacy, 1 withdrew consent, and 1 was lost to follow-up.

SIGNIFICANCE:

This open-label drug trial provides class III evidence for the long-term safety and efficacy of CBD administration in patients with treatment-resistant epilepsy (TRE) associated with CDKL5 deficiency disorder and Aicardi, Dup15q, and Doose syndromes. Adjuvant therapy with CBD showed similar safety and efficacy for these four syndromes as reported in a diverse population of TRE etiologies. This study extended analysis of the prior report from 12 weeks to 48 weeks of efficacy data and suggested that placebo-controlled randomized trials should be conducted to formally assess the safety and efficacy of CBD in these epileptic encephalopathies.

KEYWORDS:

Aicardi syndrome; CDKL5 deficiency disorder; Cannabidiol; Doose syndrome; Dup15q syndrome

PMID:
30006259
DOI:
10.1016/j.yebeh.2018.05.013
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