Open label trial with vigabatrin in children with intractable epilepsy
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1Department of Paediatrics, University of Hong Kong, Queen Mary Hospital, Hong Kong.
Insights
Vigabatrin (VGB) add-on therapy showed a 43% response rate in children with intractable epilepsy, indicating a potential treatment option. No specific patient factors predicted treatment success in this open-label study.
Area of Science:
- Pediatric Neurology
- Epileptology
- Clinical Pharmacology
Background:
- Intractable epilepsy in children presents significant treatment challenges.
- Exploring novel therapeutic strategies is crucial for improving seizure control.
Purpose of the Study:
- To evaluate the efficacy and safety of vigabatrin (VGB) as an add-on therapy for pediatric intractable epilepsy.
- To identify factors influencing treatment response to VGB in this population.
Main Methods:
- An open-label study involving 30 children with intractable epilepsy receiving VGB (40-80 mg/kg/day) alongside existing antiepileptic drugs.
- Treatment duration ranged from 2 to 24 months, with seizure frequency reduction as the primary outcome measure.
- Patient data included seizure types, epilepsy etiology, and neurological status.
Main Results:
- Vigabatrin add-on therapy resulted in a 43% response rate (50-75% seizure frequency reduction) in 13 out of 30 children.
- No significant correlation was found between treatment outcome and patient demographics, seizure characteristics, epilepsy type, etiology, or neuroimaging findings.
- The study included various seizure types such as generalized tonic-clonic, complex partial, and myoclonic seizures.
Conclusions:
- Vigabatrin demonstrates a moderate efficacy as an add-on treatment for intractable epilepsy in children.
- Treatment response to VGB was not associated with specific clinical or etiological factors in this cohort.
- Further research with larger, controlled trials is warranted to confirm these findings and optimize VGB use.
Abstract:
Thirty children (20 males, 10 females) with intractable epilepsy received vigabatrin (VGB) as an open label basis to preexisting antiepileptic drugs. The seizure types consisted of generalized tonic clonic seizure [10], complex partial seizure [8], myoclonic seizure [7], and mixed type with simple partial seizure, complex partial seizure and/or generalized seizure [5]. The cause of the epilepsy was cryptogenic in 16 and symptomatic in 12. The current dosage regime of anticonvulsants were maintained during the trial period. VGB at 40-80 mg/kg/day were titrated according to the clinical response for a period of 2-24 months. The result of treatment was categorized as 'responders' with 13 (43%) having 50-75% reduction of seizure frequency; and 'non-responders' which consisted of 17 children. There was no relationship between outcome of VBG add-on therapy and the sex, age of onset, type of seizure, type of epileptic syndrome, etiology, associated neurological abnormality, mental retardation or abnormal brain CT/MRI findings.