Authors :
Dennis Fleysh, BS – Children's Hospital of Philadlephia
Samantha Hagopian, RN, BSN, MSN – Children's Hospital of Philadlephia
Jennie Minnick, BS – Children's Hospital of Philadlephia
Presenting Author: Eric Marsh, MD PhD – Children's Hospital of Philadelphia
Rationale: Cannabidiol has gone through multiple phase 3 trials to confirm efficacy and safety in the treatment of epilepsy in Dravet and Lennox Gastaut syndromes. The role of additional THC or other cannabinoids in controlling seizures or improving non seizure behaviors has been questioned since the medical marijuana (MM) boom in the middle 2000s. To address this issue, we partnered with a dispensary in the state of Pennsylvania to perform an observational study of families asking to try MM for treatment of epilepsy that includes a more complete cannabinoid profile, including THC.
Methods:
Patients were enrolled into a CHOP IRB approved observational study of medical marijuana as adjunctive therapy for pediatric epilepsy. Qualitative surveys (Aberrant Behavior Checklist, Clinical Global Impression of Change, Medical Marijuana Side Effect Questionnaire, Quality of Life Inventory-Disability, and a sleep scale) were completed to obtain non-seizure changes. The relationship between seizures response, side effects, and behavior changes were studied. Curaleaf dispensary developed 4 products that are high in CBD but have THC; these include a 15:1 and 30:1 oil-based tinctures and 15:1 and 30:1 nano water-based tinctures.
Results:
Forty patients have been enrolled and 28 patients have received product. Seven have been on a 15:1 oil-based, 5 on 30:1 oil-based, 7 on 15:1 nano water-based and 4 on 30:1 nano water-based tinctures. Five patients changed products. Seven patients have stopped due to lack of efficacy (25%) and 5 experienced intolerable side effects. Four subjects report being seizure-free (14%). The side effect profile has included increased hyperactivity, mood swings, and appetite, decreased sleeping, speech, and appetite, nausea, emesis, altered mental status, sleepiness, urine retention, diarrhea, worsened depression, fear, paranoia, and anxiety, and dizziness.
On the variety of outcome measures, we have found that the group had moderate sleep difficulties overall with both difficulties falling asleep and significant nighttime disruptions. Subjects also had abnormal behaviors measured on the Aberrant Behavior Checklist, but with improvements with treatment across all five behavioral domains. The greatest average improvements were observed in patients showing less Irritability (mean = -2.33, n = 18) and Hyperactivity/Noncompliance (mean = -2.14, n = 21).
Using the CGI questionnaire, patients were rated on a 7-point Likert scale. The average reported change was 0.75, indicating a modest overall improvement. Using the Medical Marijuana Acute Intoxication Questionnaire, we found an average decrease of 1.57 in “Yes” responses and an average increase of 6.83 in “No” responses between patients’ first and last visits (n = 23) suggesting a trend toward reduced self-reported acute intoxication symptoms over time.
Conclusions: This observational study of marijuana products has shown these to be safe and well tolerated, with some signs of efficacy. Future work will compare the results of this study with previous purified CBD studies to determine if there is any additional benefit of THC to purified CBD for epilepsy or other symptoms in pediatric patients.
Funding: Curaleaf and the State of Pennsylvania