卢非酰胺治疗LGS综合症效果怎么样？

The effectiveness of rufinamide as an adjunctive treatment for seizures associated with Lennox-Gastaut syndrome (LGS) in adult and pediatric patients 4 years of age and older was determined in a multicenter, double-blind, placebo-controlled, randomized, parallel-group study (N = 138 people). Male and female patients (aged 4 to 30 years) were included if they were diagnosed with inadequately controlled seizures related to LGS (including atypical absence seizures and fall attacks) and were receiving 1 to 3 concomitant stable-dose AEDs (antiepileptic drugs). Each patient must have had at least 90 seizures in the month before study entry.

After completing a 4-week baseline phase of stable treatment, patients were randomly assigned to add either or placebo to their ongoing treatment during a 12-week double-blind phase. The double-blind phase consists of two cycles: a titration period (1 to 2 weeks) and a maintenance period (10 weeks). During titration, the dose is increased to a target dose of approximately 45 mg/kg per day (3200 mg in adults >70 kg) administered twice daily.

Dose reductions during titration are allowed if tolerability issues are encountered. The titration remains stable during the maintenance period. 88% of rufinamide-treated patients achieved target dose. The majority of these patients reached target dose within 7 days, and the remaining patients reached target dose within 14 days.

Trial results showed that the median percentage change in total seizure frequency per 28 days was -11.7 for placebo and -32.7 for rufinamide; the median percentage change in tonic and atonic seizure frequency per 28 days was 1.4 for placebo and -42.5 for rufinamide; and the improvement in epilepsy severity grade from the global assessment was 30.6 for placebo and 53.4 for rufinamide. It can be seen that rufinamide has a significant effect in treating epilepsy, which is LGS syndrome.

Rufinamide is suitable for the adjuvant treatment of epileptic seizures related to Lennox-Gastaut syndrome (LGS) in pediatric patients and adults aged 1 year and above. However, rufinamide is contraindicated in patients with familial short QT syndrome. It is recommended that patients take medication under the guidance of a doctor and receive symptomatic treatment.

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