Tuberous Sclerosis Complex (TSC)–Associated Neuropsychiatric Disorder (TAND) Outcomes Following Add-on Cannabidiol (CBD) Treatment: 3-Month Analysis of Open-Label Phase 3b/4 Trial EpiCom
Agnies van Eeghen1, Sarah Wilson2, Douglas Smith3, Roy Strowd4, Jane Boggs5, Teresa Greco6, Joanne Stevens6, Lisa Moore-Ramdin6
1Emma Center for Personalized Medicine, Emma Children’s Hospital, Amsterdam University Medical Centers, 2Department of Pediatrics, Division of Child and Adolescent Neurology, McGovern Medical School, The University of Texas Health Science Center at Houston, 3Minnesota Epilepsy Group, 4Departments of Neurology and Internal Medicine, and the Translational Sciences Institute, Comprehensive Cancer Center, Wake Forest School Of Medicine, 5Wake Forest School of Medicine, 6Jazz Pharmaceuticals, Inc.
Objective:
To investigate behavioral and co-occurring outcomes after initiation of add-on CBD treatment in patients with TSC who experience seizures (NCT05864846).
Background:
Epidiolex® is a plant-derived highly purified pharmaceutical formulation of CBD approved for treatment of seizures associated with TSC; anecdotal evidence suggests neuropsychiatric improvements.
Design/Methods:
In this open-label study, participants received CBD (100 mg/mL oral solution) ≤25 mg/kg/day and standard of care (SOC) for 26 weeks, followed by CBD with SOC/SOC alone up to 52 weeks. Most problematic behavior (MPB) on TAND Self-Report Quantified Checklist (TAND-SQ) and Aberrant Behavior Checklist (ABC) were assessed at week 13, and Caregiver Global Impression of Severity (CareGI-S) and Clinician Global Impression of Severity (CGI-S) scales were assessed at weeks 4 and 13.
Results:
Of 24 participants enrolled, 19 started CBD (median [range] age 21 [5–42] years); ≥1 postbaseline assessment was available for 17. A median (range) of 3 (1–5) antiseizure medications were used. At baseline, MPB numerical rating scale (NRS) value was 8.8 (1.01), suggesting severe TAND problems. At week 13 (n=5), mean (95% CI) change from baseline in MPB NRS was −4.6 (−8.1, −1.1). Greatest changes were in dysregulated behavior (−3.5 [−6.5, −0.5]) and overactivity/impulsivity scores (−2.9 [−7.1, 1.3]) among 7 TAND-SQ clusters and in irritability (−12.2 [−22.3, −2.1]) and hyperactive noncompliance (−11.0 [−25.1, 3.1]) in ABC. Compared with baseline, smaller proportions of caregivers and clinicians rated behavioral problems as severe/very severe at weeks 4 and 13, respectively (severe: 41%, 7%, 0% [CareGI-S] and 38%, 7%, 0% [CGI-S]; very severe: 6%, 0%, 0% [for both]). Adverse events occurred in 12/19 participants (63%); most common were diarrhea (42%), vomiting, decreased appetite, and lethargy (11% each).
Conclusions:
After initiating CBD, improvements were seen on TAND-SQ and ABC subscales and in severity of psychiatric and behavioral problems reported by caregivers and clinicians.
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