Long-term open-label study of adjunctive topiramate in infants with refractory partial-onset seizures

Area of Science:

• Pediatric Neurology
• Clinical Pharmacology
• Epileptology

Background:

• Refractory partial-onset seizures pose significant challenges in infants.
• Limited long-term safety data exists for antiepileptic drug treatments in this age group.

Purpose of the Study:

• To evaluate the long-term safety and tolerability of adjunctive topiramate in infants (<2 years) with refractory partial-onset seizures.
• To assess growth parameters and seizure frequency during up to one year of treatment.

Main Methods:

• Pooled data from two studies (Phase 1 and Phase 3) involving 284 infants.
• Open-label extension with topiramate titration up to 60 mg/kg/d.
• Adverse events, growth parameters (Z scores), and seizure rates were monitored.

Main Results:

• Most common adverse events (≥30%): fever (52%), respiratory tract infections (51%), anorexia (35%), acidosis (31%).
• Mean changes in Z scores for body weight, length, and head circumference were -0.82, -0.45, and -0.36, respectively.
• Tolerability was consistent with previous studies in infants.

Conclusions:

• Adjunctive topiramate demonstrates acceptable long-term safety and tolerability in infants with refractory partial-onset seizures.
• Observed growth parameter changes and adverse events align with known topiramate profiles.
• Topiramate can be considered as a treatment option for this pediatric population.