Retrospective Multicenter Chart Review Study of Adjunctive Cannabidiol for Seizures Associated with Lennox-Gastaut Syndrome, Dravet Syndrome and Tuberous Sclerosis Complex

被引:0
作者
Strzelczyk, Adam [1 ]
Klotz, Kerstin Alexandra [2 ,3 ]
Mayer, Thomas [4 ]
von Podewils, Felix [5 ]
Knake, Susanne [6 ]
Kurlemann, Gerhard [7 ]
Herold, Luise [5 ]
Immisch, Ilka [6 ]
Buhleier, Elisa [1 ]
Rosenow, Felix [1 ]
Schubert-Bast, Susanne [1 ,8 ]
机构
[1] Goethe Univ Frankfurt, Univ Med Frankfurt, Epilepsy Ctr Frankfurt Rhine Main, Dept Neurol, Theodor Stern Kai 7, D-60596 Frankfurt, Germany
[2] Univ Hosp Bonn, Dept Pediat Neurol, Bonn, Germany
[3] Univ Freiburg, Ctr Pediat, Dept Neuropediat & Muscle Disorders, Med Ctr,Fac Med, Freiburg, Germany
[4] Epilepsy Ctr Kleinwachau, Radeberg, Germany
[5] Univ Hosp Greifswald, Epilepsy Ctr, Dept Neurol, Greifswald, Germany
[6] Philipps Univ Marburg, Epilepsy Ctr Hessen, Dept Neurol, Marburg, Germany
[7] St Bonifatius Hosp, Lingen, Germany
[8] Goethe Univ Frankfurt, Univ Hosp Children & Adolescents, Univ Med Frankfurt, Dept Pediat Epileptol, Frankfurt, Germany
关键词
Cannabidiol; Clinical Global Impression of Change; Developmental and epileptic encephalopathies; Dravet syndrome; Epilepsy; Lennox-Gastaut syndrome; Responder rate; Tuberous sclerosis complex;
D O I
10.1007/s40120-025-00788-w
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Introduction Effectiveness and tolerability of plant-derived highly purified cannabidiol (CBD) in patients with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC)-associated epilepsy in clinical practice in Germany were evaluated. Methods This multicenter, retrospective, chart review study analyzed patients with LGS, DS, or TSC-associated epilepsy receiving >= 1 dose of adjunctive CBD (Epidyolex (R) 100 mg/mL oral solution). Treatment characteristics, seizure outcomes, physician-rated Clinical Global Impression of Change (CGI-C), treatment retention rates, and adverse events (AEs) were analyzed <= 12 months. Results Among 202 patients identified (159 LGS; 34 DS; 9 TSC), median (interquartile range; range) age was 18.0 (7.9-32.0; 0.3-72.0) years, and median (range) number of prior and concomitant antiseizure medications was 6 (1-24) and 3 (1-7), respectively. Median target CBD dose was 11.1 mg/kg/day (17.6, 15.2, and 9.9 mg/kg/day in the < 6, 6-17, and >= 18 years subgroups, respectively). Responder rates (>= 50% seizure reduction) for total seizures at 3 (n = 194) and 12 (n = 168) months were 43.3% (37.0-50.0% across ages) and 44.0% (37.0-52.5% across ages), respectively, and for generalized tonic-clonic seizures 54.3% (n = 94) (50.0-66.7% across ages) and 47.7% (n = 88) (37.8-66.7% across ages), respectively. Median (range) number of seizure days per month significantly decreased from 30 (0.3-30) to 18 (0-30) in the 3 months before the last 3 months of CBD treatment (p < 0.001). Any improvement in CGI-C was observed in 62% of patients. Of those with available data at 3 and 12 months, 89.6% and 67.1% remained on CBD, respectively. Retention was similar across age groups. AEs reported in >= 5% of patients were sedation and diarrhea. Conclusions In patients with LGS, DS, or TSC-associated epilepsy, adjunctive CBD was associated with a reduction in seizure frequency across age groups. CBD demonstrated tolerability consistent with its known profile, and 67% of patients remained on treatment at 12 months.
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