Table I. Efficacy criteria for cannabidiol treatment.
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Efficacy criteriaa, listed in descending order of relevance
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% of reduction in drop seizures or seizures with motor a component (convulsions)
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It is the most important criteria, since convulsions or seizures with a motor component and drop seizures have a high impact on the quality of life of patients
The efficacy goal of the reduction in seizures depends on the baseline situation of each patient
In some cases (e.g. LGS patients with a high number of nocturnal seizures or drop seizures), a reduction of up to 50-70% in total seizures could be considered an excellent result even if total control of said seizures were not achieved.
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Reduction in generalised tonic-clonic seizures and drop seizures
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These are the most life- threatening seizures and the ones that have the highest impact on the quality of life of patients
Generalised tonic-clonic seizures can lead to status epilepticus and can be associated with a higher risk of sudden unexpected death in epilepsy (SUDEP) in DS patients
Controlling drop seizures is essential in LGS patients. The morbidity and mortality rates for trauma is very high and this gives rise to anxiety in patients’ family members and caregivers
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Reduction or improvement in the severity of other types of seizures
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Reduction in other types of seizures (atypical absences, minor motor seizures, or myoclonic seizures) and night tonic seizures:
- LGS: reduction of ≥ 30% in atypical absences
- DS: reduction of ≥ 30% in partial complex seizures
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Changes in seizures semiology and reduction in rescue medication
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- Duration of the seizures: achieve a reduction in their duration (e.g.: they used to last 5 minutes and now they last 30 seconds)
- Onset: it should be slower. It is important when it comes to drop seizures, since caregivers should be aware of possible falls so that injuries due to trauma can be reduced.
- Intensity
It is essential to reduce the ‘rescue medication’ (mainly benzodiazepines) administered by patients’ caregivers in order to avoid status epilepticus. Many AEs are reduced. The collection of this information weekly by caregivers is very relevant and helpful for the physicians when it comes to optimising the treatment
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Increase in seizure-free days
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The increase in seizure-free days is a parameter highly valued by caregivers. More seizure-free days allow them to carry out more activities, reassure the confidence of the patients’ families, and improve their QoL
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Cognitive improvement: improvement in alert or attention, which involves an improvement in the QoL
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Improvement in QoL: recovery of school days and daily living activities. Reinforcement of family confidence
QoL scales needed to assess these changes (for example CAVE, LQOL scales)
It is difficult to assess whether the patient is alert or more participative, by using scales because there are not specific scales for DS or LGS. The reports of the educational centre caregivers are very helpful
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Reduction in the number of concomitant ASMs or their doses
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DS or LGS patients are generally polymedicated
If the dose or the number of concomitant ASMs got to be reduced, treatment-related AEs would be reduced
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AE: adverse event; ASMs: antiseizure medications; CAVE: quality of life scale in childhood epilepsy; CBD: cannabidiol; DS: Dravet syndrome; LGS: Lennox-Gastaut syndrome; QoL: quality-of-life. a They can be easily quantified/identified by physicians and/or caregivers.
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