Monday, 21 July 2025

AED DBD

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Cannabidiol (CBD), a non‑intoxicating cannabinoid compound, has emerged as a scientifically supported treatment option for certain severe and treatment-resistant childhood epilepsy syndromes. Here's what current evidence and regulatory guidance show:


✅ 1. FDA‑Approved Medication: Epidiolex / Epidyolex

  • Epidiolex (under Epidiolex in the US and Epidyolex in the EU/UK) is a purified plant‑derived CBD oral solution approved to treat seizures associated with Dravet syndrome, Lennox‑Gastaut syndrome, and tuberous sclerosis complex in children aged one or two years and older (Wikipedia).

  • First approved by the FDA in June 2018 (Dravet and Lennox–Gastaut) and later extended in July 2020 to include tuberous sclerosis complex; EU approval followed in September 2019 (TIME, Wikipedia, Wikipedia).


📊 2. Clinical Trial Evidence (GWPCARE Series)

Dravet Syndrome

  • In pivotal randomized, double‑blind trials (ages 2–18):

    • ≈ 38–48% reduction in convulsive seizures vs. ≈ 13–27% with placebo.

    • ≥ 50% reduction responder rate: 43–46% vs. placebo 27% (PubMed, PubMed).

    • 62% of caregivers rated overall improvement vs. 34% in placebo group (PMC).

Lennox‑Gastaut Syndrome

  • In patients aged 2–55:

    • Drop seizures reduced by ≈ 30–44% vs. ≈ 17–22% with placebo.

    • Total seizure reduction ≈ 37–42% vs. placebo ≈ 18–19% (Wiley Online Library).

Sustained Effects

  • Long‑term follow‑up (GWPCARE5) showed seizure reductions maintained at 48–60% for drop seizures and 48–57% for total seizures over ~1 year (Wiley Online Library).


⚠️ 3. Safety and Side Effects

  • Common adverse effects include somnolence, decreased appetite, diarrhea, vomiting, and elevated liver enzymes (ALT/AST) (New England Journal of Medicine).

  • Liver enzyme elevations occurred notably when CBD was combined with valproate (≈9% of treated cases), most resolved with dose adjustment or cannabidiol discontinuation (New England Journal of Medicine, American Academy of Neurology).

  • Because of interactions with clobazam and other drugs (via CYP2C19/CYP3A4 enzymes), careful monitoring is essential—especially in polytherapy regimes (PubMed).


📋 4. Systematic Reviews & Meta-Analysis

  • A 2022 meta-analysis (1,034 patients) confirmed adjunctive CBD leads to:

    • ≈ 33% average reduction in seizure frequency

    • ≈ 20% increase in ≥50% responder rate

    • But also ≈ 12% greater risk of adverse events and a 3‑fold rise in elevated liver enzymes (≥3× normal) compared to placebo (PubMed).

  • A separate pediatric-specific review found CBD effective for Dravet and LGS, though other cannabinoids (e.g. THC-containing) lacked consistent benefit and increased psychiatric risks (PMC).


💬 5. Caregiver & Real‑World Reports

  • Qualitative studies and observational data highlight improvements in quality of life, mood, alertness, communication, and cognition, even beyond seizure reductions, as reported by caregivers of children using CBD-based treatments (PMC).

  • Anecdotal accounts (e.g., Charlotte’s Web story) sparked early interest, but most evidence now comes from standardized pharmaceutical formulations rather than artisanal products (Wikipedia).


🧭 6. Guidelines & Practical Recommendations

  • Organizations like NICE (UK) and NHS England endorse CBD (with clobazam) as third-line adjunctive therapy for Dravet and LGS only if standard treatments fail, with early review after ~6 months; discontinue if seizures haven’t dropped by ≥30% (Wiley Online Library).

  • In the UK, access remains limited—many families rely on private prescribing (often expensive and restricted) due to lack of pediatric prescribers and rigid NHS frameworks (Reddit).


⚙️ Summary Table

Syndrome Seizure Reduction Responder Rate (≥50%) Common Side Effects
Dravet syndrome ~38–48% ~43–46% Somnolence, diarrhea, appetite loss, elevated liver enzymes
Lennox–Gastaut syndrome ~30–44% (drop seizures); ~37–42% (total) ~44% vs ~24% placebo Similar profile: sedation, GI upset, liver enzyme rise

🧠 Considerations & Current Scope

  • CBD (Epidiolex/Epidyolex) is only approved for Dravet, Lennox‑Gastaut, and tuberous sclerosis‑related epilepsy.

  • Evidence in other childhood epilepsy types is limited or emerging—use outside these indications is considered off-label and should be managed cautiously.

  • THC-containing or unregulated “CBD oils” lack adequate evidence; may carry psychiatric or developmental risks, especially in children.


✅ Bottom Line

  • Purified CBD, as Epidiolex/Epidyolex, offers a clinically validated option for children with Dravet syndrome, Lennox‑Gastaut syndrome, or tuberous sclerosis complex whose seizures are resistant to standard therapies.

  • Benefits include meaningful seizure reduction and improved caregiver-assessed quality of life.

  • However, side effects—especially liver enzyme elevations and drug interactions—demand close medical supervision.

  • Access in the UK and NHS remains limited, with many families relying on private (and costly) routes.


If you're looking for info on using CBD off-label in other epilepsy types, managing drug interactions, or navigating access in your region—I’d be glad to help explore further.

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