Background and importance Cannabidiol (CBD) is an orphan medicine recently approved in Europe for the treatment of Dravet (DS) and Lennox–Gastaut syndromes (LGS) in combination with clobazam, and for tuberous sclerosis. However, there is growing evidence that other types of refractory epilepsy could be treated with this drug.
Aim and objectives To evaluate the use of CBD in a paediatric hospital, as well as its effectiveness and safety.
Material and methods Observational, retrospective study carried out between January 2017 and September 2021, including all patients treated with CBD in our hospital.
Variables included Age, sex, weight, concomitant antiepileptic drugs (AEDs), length of treatment, initial and maintenance dose, reasons for discontinuation and adverse events (AEs) related to CBD. Efficacy was assessed following two criteria: reduction in number of seizures and opinion of caregivers.
Data were collected from electronic medical records and the pharmacy dispensing program.
Results Thirty-one patients were included: male 61.3% (n=19); median age 10 (2–16) years. Median weight 28 (14–80) kg median initial dose: 3 mg/kg/day (1–12). CBD was prescribed for LGS 61.3% (n=19), refractory epilepsy 13% (n=4), DS 6.5% (n=2), epileptic encephalopathy 6.5% (n=2), West syndrome 6.5% (n=2), Rett syndrome 3.1% (n=1) and tuberous sclerosis 3.1% (n=1). Median of concomitant AEDs was 3 (0–4). Twenty (64.5%) patients received CBD in combination with clobazam.
Two patients (6.5%) discontinued CBD in the first 2 weeks of treatment. Both presented a significant increase in number of seizures. Twenty-nine (93.5%) patients reached a maintenance dose of 15 mg/kg/day (5–44); the median length of treatment was 7 (3–69) months. Treatment was discontinued in 8 cases (25.8%) because the number of seizures was not reduced, and one also presented rash (3.4%). To date, 21 (67.7%) patients continue on CBD: in 14 (45.2%) cases, number of seizures was significantly reduced and caregiver’s appreciation of effectiveness was good; and 7 (22.5%) responded partially.
Most frequent AEs were: irritability 24.4% (n=7), diarrhoea 13.79% (n=4) and anorexia 10.34% (n=3). Other AEs described were: drooling 6.9% (n=2), somnolence (n=2); rash 3.4% (n=1), hepatobiliary disorders (n=1) and asthenia (n=1).
Conclusion and relevance CBD was prescribed in numerous indications due to the lack of therapeutic alternatives in some seizures-refractory patients. It has been an effective option in most of our patients and its security profile is consistent with clinical trials.
Conflict of interest No conflict of interest
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