Introduction. Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) are two serious epileptic syndromes with paediatric onset which are refractory to therapy and are associated with an important increase in mortality rates and comorbidities compared to the general population. These pathologies have a strong impact on the lives of patients and their families, because they undergo multiple pharmacological therapies (many of them without specific indication), with frequent changes due to poor efficacy and associated adverse effects. The specialists who care for these patients highlight unmet needs and the lack of specific, safe and effective treatments for better management of the syndrome. Development. A group of four neurologists specializing in epilepsy has met to review the scientific literature and evaluate the efficacy and safety of oral solution cannabidiol in the treatment of these syndromes, both in randomized clinical trials (CT) and in some observational studies. Conclusions. Cannabidiol is positioned as an innovative therapy that allows better control of epileptic seizures and comorbidities of DS and LGS, furthermore its efficacy and safety have been evaluated in more than 700 patients. In CTs, cannabidiol significantly reduced the percentage of convulsive seizures and drop seizures compared to placebo in patients with DS and LGS respectively, which could improve their quality of life and that of their family members. The most frequent adverse effects reported were somnolence and decreased appetite. Elevated liver aminotransferase levels were also reported, especially in patients given concomitant sodium valproate. This therapy may allow better control of the epileptic seizures associated with these syndromes.
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Great Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, EnglandGreat Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, England
Eltze, Christin
Alshehhi, Shaikha
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Great Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, EnglandGreat Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, England
Alshehhi, Shaikha
Al Ghfeli, Aisha
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Great Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, EnglandGreat Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, England
Al Ghfeli, Aisha
Vyas, Kishan
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Jazz Pharmaceut UK Ltd, 1 Cavendish Pl, London W1G 0QF, EnglandGreat Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, England
Vyas, Kishan
Saravanai-Prabu, Seeta
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Jazz Pharmaceut UK Ltd, 1 Cavendish Pl, London W1G 0QF, EnglandGreat Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, England
Saravanai-Prabu, Seeta
Gusto, Gaelle
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Certara, 71 Rue Miromesnil, F-75008 Paris, FranceGreat Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, England
Gusto, Gaelle
Khachatryan, Artak
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Certara, Acero Level 2,1 Concourse Way, Sheffield SN1 2BJ, EnglandGreat Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, England
Khachatryan, Artak
Martinez, Marta
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Certara, Av Gran Via 46, Bilbao 48011, Bizkaia, SpainGreat Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, England
Martinez, Marta
Desurkar, Archana
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Sheffield Childrens Hosp NHS Fdn Trust, Sheffield S10 2TH, EnglandGreat Ormond St Hosp Children NHS Fdn Trust, Great Ormond St, London WC1N 3JH, England