Outcomes from a Spanish Expanded Access Program on cannabidiol treatment in pediatric and adult patients with epilepsy

Villanueva, Vicente; García-Ron, Adrián; Smeyers, P; Arias, Eva; Soto, Victor; Garcı́a-Peñas, Juan José; González-Alguacil, Elena; Sayas, Débora; Serrano-Castro, Pedro J.; Garcés, Mercedes; Hampel, Kevin G.; Tomás, Miguel; Lara, Julian; Toledo, María de; Barceló, Ines; Aledo‐Serrano, Ángel; Gil‐Nagel, Antonio; Iacampo, Lucas; Falip, Mercé; Saiz-Díaz, Rosa Ana; Gómez‐Ibáñez, Asier; Sopelana, David; Sánchez-Larsen, Álvaro; López‐González, Francisco Javier

doi:10.1016/j.yebeh.2022.108958

Cited by 8 publications

(15 citation statements)

References 28 publications

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“…In a subsequent analysis of the same cohort, 22 which added 64 new patients, responder rate was confirmed to be around 68% [58-77%] at 12 months, consistent with our responder rate of 60% [45-70%] at 12 months. Furthermore, retention at 12 months for STP are consistent with other similar cohorts who reported a 53% 22 and were comparable to other drugs employed in the treatment of DREE such as valproate (78%), 23 lamotrigine (69%), 24 clobazam (67-51%), 24,25 and cannabidiol (61.4%), 26 and higher than others such as vigabatrin (55%), 27 brivaracetam (41%) 28 and topiramate (37%), 24 which may be explained by the good balanced profile of tolerability and efficacy in this cohort.…”

Section: Discussionsupporting

confidence: 88%

Efficacy and tolerability of add‐on stiripentol in real‐world clinical practice: An observational study in Dravet syndrome and non‐Dravet developmental and epileptic encephalopathies

Gil‐Nagel,

Aledo‐Serrano,

Beltrán‐Corbellini

et al. 2023

Epilepsia Open

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ObjectiveTo assess efficacy and tolerability of stiripentol (STP) as adjunctive treatment in Dravet syndrome and non‐Dravet refractory developmental and epileptic encephalopathies (DREEs).MethodsRetrospective observational study of all children and adults with DREE and prescribed adjunctive STP at Hospital Ruber Internacional from January 2000 to February 2023. Outcomes were retention rate; responder rate (proportion of patients with ≥50% reduction in total seizure frequency relative to baseline); seizure freedom rate; responder rate for status epilepticus; rate of adverse event and individual adverse events; reported at 3, 6, and 12 months, and at final visit. Seizure outcomes are reported overall, and for Dravet and non‐Dravet subgroups.ResultsA total of 82 patients, (55 Dravet syndrome and 27 non‐Dravet DREE) were included. Median age was 5 years (range 1–59 years), and median age of epilepsy onset was younger in the Dravet group (4.9 [3.6‐6] months) than non‐Dravet (17.9 [6‐42.3], p<0.001). Median follow‐up time STP was 24.1 months (2 years; range 0.3‐164 months), and was longer in the Dravet group (35.9 months; range 0.8‐164) than non‐Dravet (17 months range 0.3‐62.3, p<0.001). At 12 months, retention rate, responder rate and seizure free rate was 68.3% (56/82), 65% [48‐77%] and 18% [5.7‐29%], respectively. There were no statistically significant differences between groups on these seizure outcomes. Adverse events were reported in 46.3% of patients (38/82), without differences between groups.SignificanceIn this population of patients with epileptic and developmental encephalopathies, outcomes with adjunctive STP were similar in patients with non‐Dravet DREE to patients with Dravet syndrome.

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Section: Discussionsupporting

confidence: 88%

Efficacy and tolerability of add‐on stiripentol in real‐world clinical practice: An observational study in Dravet syndrome and non‐Dravet developmental and epileptic encephalopathies

Gil‐Nagel,

Aledo‐Serrano,

Beltrán‐Corbellini

et al. 2023

Epilepsia Open

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“…Long‐term effectiveness and tolerability have been confirmed in open‐label studies 7–9 . Finally, real‐world data information regarding the use of this medication has been reported in a few observational studies 10,11 …”

Section: Introductionmentioning

confidence: 83%

Prospective study of cenobamate on cognition, affectivity, and quality of life in focal epilepsy

Catalán‐Aguilar,

Hampel,

Cano‐López

et al. 2023

Epilepsia Open

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ObjectiveCenobamate is a recently approved antiseizure medication that proved to be safe and effective in randomized controlled trials. However, little is known about its impact on some areas frequently affected in epilepsy. For this reason, we explored the effects of cenobamate in cognitive performance, as well as on negative affectivity and quality of life in a sample of patients with drug‐resistant epilepsy.MethodsTwo prospective cohort studies were carried out. In Study 1, 32 patients (22 men and 10 women) underwent a baseline (T0) and a short‐term (T1) neuropsychological assessment after three months of cenobamate administration. In Study 2, 22 patients (16 men and 6 women) from the T1 sample also underwent a baseline and a follow‐up evaluation (T2) six months after T0.ResultsNo significant differences were found in cognitive variables, negative affectivity, and quality of life either in Study 1 or Study 2. Similarly, based on the reliable change index it was found that most patients showed no changes in these variables.SignificanceThese results suggest that cenobamate is a safe antiseizure medication in terms of cognition, negative affectivity, or quality of life since no adverse events have been found after three and six months of treatment.

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“…In a recently published real‐world retrospective study of 57 patients with highly active and extremely refractory focal epilepsy, 5% of subjects achieved seizure freedom, 42% showed remarkable seizure reduction of ≥75%, and an additional 28% reached seizure reduction of ≥50% with a median treatment duration of 11 months 10 . A further retrospective, multicenter, observational study involving 170 intractable patients reported a responder rate of 63% and a seizure freedom rate of 13% 11 . Similar to our patient cohort, these data probably reflect the spectrum of patients treated within specialized epilepsy centers.…”

Section: Discussionmentioning

confidence: 99%

“…Thus, in a cohort of 57 patients who received cenobamate for at least 3 months, 70% achieved a seizure reduction of ≥50% in the dose range of 100–300 mg/day in a dose‐dependent manner and 5% became seizure‐free taking cenobamate doses of >200 mg/day 10 . The second real‐world study explored efficacy of the drug in 170 patients, who received cenobamate during a mean time of 12 months, and revealed 63% responders with ≥50% reduction as well as 13% seizure‐free patients 11 . The study detected no statistical difference in improvement of seizure rates in patients who received cenobamate at a lower or higher dose.…”

Section: Introductionmentioning

confidence: 99%

Add‐on treatment with cenobamate is already effective at low doses in refractory focal epilepsy: A prospective observational study

Novitskaya,

Schütz,

Metternich

et al. 2024

Epilepsia

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Abstract:ObjectiveCenobamate, a novel ASM with a dual mechanism of action, has been shown in pivotal trials to significantly improve seizure control in treatment‐resistant focal epilepsy. We aimed to evaluate whether these promising results could be confirmed in a real‐world setting with a follow‐up period of up to 12 months.MethodsPatients from a tertiary epilepsy centre who received cenobamate add‐on between June 2021 to October 2023 were followed up prospectively at 3, 6 and 12 months after treatment initiation for assessment of seizure outcomes and treatment‐related adverse events.ResultsThe clinical cohort included 112 adult patients with 30% non‐lesional cases and a wide spectrum of epileptogenic lesions underlying refractory focal epilepsy. We observed a significant reduction in monthly seizure frequency of all seizure types already after 3 months of treatment at a median cenobamate dose of 100 mg/day. 46% of patients were responders with a ≥ 50% seizure reduction, 26% had a ≥ 75% seizure reduction, and 9% became seizure free. Among the 74 patients with available follow‐up of 12 months, the responder rates reached 55%, 35% and 19% for ≥ 50%, ≥ 75% and 100% of seizure reduction, respectively.After 3 months of treatment, 38% of patients reported adverse effects, mainly (84%) mild to moderate in intensity. Adjustment of comedication allowed successful management of adverse effects in 32% of patients. At a group level, there was no correlation between the cenobamate daily dose and the incidence of adverse effects.SignificanceWe found a clinically relevant response to cenobamate already at a low daily dose of 100 mg also in a patient cohort with a higher degree of drug resistance than in pivotal trials. Our prospectively collected data provide real‐world evidence for high efficacy and good tolerability of the drug although no standardized treatment protocol or comparison with a control group was applied.

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Outcomes from a Spanish Expanded Access Program on cannabidiol treatment in pediatric and adult patients with epilepsy

Cited by 8 publications

References 28 publications

Efficacy and tolerability of add‐on stiripentol in real‐world clinical practice: An observational study in Dravet syndrome and non‐Dravet developmental and epileptic encephalopathies

Efficacy and tolerability of add‐on stiripentol in real‐world clinical practice: An observational study in Dravet syndrome and non‐Dravet developmental and epileptic encephalopathies

Prospective study of cenobamate on cognition, affectivity, and quality of life in focal epilepsy

Add‐on treatment with cenobamate is already effective at low doses in refractory focal epilepsy: A prospective observational study

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