Related Experiment Videos
Lamotrigine therapy in juvenile neuronal ceroid lipofuscinosis
L Aberg1, E Kirveskari, P Santavuori
1Department of Pediatric Neurology, Hospital for Children and Adolescents, University of Helsinki, Finland.
Insights
Lamotrigine (LTG) therapy shows promise for juvenile neuronal ceroid lipofuscinosis (JNCL) patients, improving seizures and well-being in most participants. This study suggests LTG is a valuable treatment option for JNCL.
Area of Science:
- Neurology
- Pediatric Neurology
- Pharmacology
Background:
- Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) is a rare genetic disorder.
- Epileptic seizures are a common and debilitating symptom in JNCL patients.
- Current treatment options for JNCL seizures and well-being are limited.
Purpose of the Study:
- To evaluate the efficacy of lamotrigine (LTG) in managing epileptic seizures in JNCL patients.
- To assess the impact of LTG therapy on the general well-being of individuals with JNCL.
- To determine the long-term effectiveness and tolerability of LTG in this patient population.
Main Methods:
- LTG was administered to 28 JNCL patients, with dosages adjusted to maintenance levels.
- Patients were categorized into four groups based on the indication for LTG initiation (add-on, first AED, epileptiform activity, VPA replacement).
- Efficacy and well-being were assessed after one year, with a mean follow-up of 2.8 years.
Main Results:
- A favorable response to LTG was observed in 23 out of 28 patients.
- Significant seizure reduction (≥50% frequency decrease) occurred in 10 patients, and severity decreased in nine.
- Improvements in general well-being were reported in 18 of the 28 patients.
Conclusions:
- Lamotrigine (LTG) demonstrates significant therapeutic potential in managing seizures associated with JNCL.
- LTG appears to be a valuable treatment option, improving both seizure control and overall well-being in JNCL patients.
- The study supports the continued use and further investigation of LTG for JNCL management.
Purpose:
To evaluate the effects of lamotrigine (LTG) therapy on epileptic seizures and general well-being in patients with juvenile neuronal ceroid lipofuscinosis (JNCL).
Methods:
LTG was initiated in 28 patients with JNCL. The mean age of the patients at the initiation of LTG was 13.7 years (range, 6.7-28.2 years). LTG was started at a dosage of 0.1-0.5 mg/kg/day and increased every 2 weeks until a maintenance dose of 1.25-15 mg/kg/day was reached. On the basis of the indication for LTG therapy, the patients could be divided into four groups. In the first group, LTG was initiated on an add-on basis; in the second group, LTG was started as the first antiepileptic drug (AED) because of seizures, and in the third group, despite no preceding seizures, because of epileptiform activity in the whole-night polysomnography; in the fourth group, LTG replaced valproate (VPA), which was discontinued because of adverse side effects. The efficacy was assessed after 1 year on LTG. The mean follow-up time was 2.8 years (range, 1.3-5.8).
Results:
LTG had a favorable effect in 23 of 28 patients. A decrease in frequency of seizures of > or =50% was observed in 10 and a decrease in severity of seizures in nine of the 22 patients who had preceding seizures. Increases in well-being were found in 18 of 28. During the follow-up, LTG was continued as monotherapy in 13 of 19 patients.
Conclusions:
In light of our experiences, LTG seems to be a valuable drug in JNCL.