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Vigabatrin therapy for infantile epileptic spasms syndrome with periventricular leukomalacia
Azusa Oba1, Ryuki Matsuura1, Haruhito Horita2
1Division of Neurology, Saitama Children's Medical Center, 1-2, Shin-toshin, Chuo-ku, Saitama, Japan; Division of Epilepsy Center, Saitama Children's Medical Center, 1-2, Shin-toshin, Chuo-ku, Saitama, Japan; Department of Pediatrics, The Jikei University School of Medicine, 3-25-8, Nishi-Shimbashi, Minato-ku, Tokyo, Japan.
Insights
Vigabatrin effectively treated 42.8% of infants with Infantile Epileptic Spasm Syndrome (IESS) and Periventricular Leukomalacia (PVL). The medication was well-tolerated, with mild adverse effects observed.
Area of Science:
- Pediatric Neurology
- Neurodevelopmental Disorders
- Epileptology
Background:
- Infantile Epileptic Spasm Syndrome (IESS) and Periventricular Leukomalacia (PVL) are significant causes of developmental regression and poor neurological outcomes in infants.
- Current treatments for IESS, such as adrenocorticotropin and vigabatrin, lack specific evaluation in patients with co-occurring PVL.
- This study addresses the unmet need to assess vigabatrin's efficacy and safety in this combined patient population.
Purpose of the Study:
- To investigate the efficacy of vigabatrin in treating infantile epileptic spasms in patients with co-existing periventricular leukomalacia.
- To evaluate the safety profile and adverse effects of vigabatrin in this specific pediatric cohort.
- To determine treatment response rates and the time to seizure cessation.
Main Methods:
- A retrospective analysis of pediatric patients diagnosed with both IESS and PVL who received vigabatrin treatment.
- Inclusion of ophthalmological examinations and electroretinography (ERG) before and after vigabatrin therapy.
- Definition of treatment response as complete cessation of epileptic spasms and all other seizures at 3 months post-initiation.
Main Results:
- Vigabatrin treatment led to complete cessation of epileptic spasms in 42.8% (3 out of 7) of patients within 3 months.
- The median time to seizure cessation was 5 days after vigabatrin initiation.
- Observed adverse effects included poor feeding (1 patient) and transient abnormal ERG findings (2 patients) that normalized upon dose adjustment or discontinuation.
Conclusions:
- Vigabatrin demonstrates efficacy in a significant portion (42.8%) of infants with combined IESS and PVL.
- The drug was generally well-tolerated, with only mild adverse effects noted in this population.
- Treatment-related electroretinography abnormalities were reversible, suggesting a manageable safety profile.
Background:
Infantile epileptic spasm syndrome (IESS) and periventricular leukomalacia (PVL) cause developmental regression and poor neurological outcomes. The primary treatments for IESS include adrenocorticotropin and vigabatrin; however, no study has yet evaluated the efficacy of vigabatrin for patients with both IESS and PVL. Therefore, we investigated the efficacy and safety of vigabatrin in patients with both disorders.
Methods:
We retrospectively enrolled patients with IESS and PVL who were treated with vigabatrin. We performed ophthalmological examinations and electroretinography (ERG) before and after vigabatrin. Treatment response was defined as complete cessation of epileptic spasms and no other seizures after 3 months of vigabatrin. We investigated the responses to vigabatrin, doses used, and adverse effects.
Results:
Seven patients (5 males) with IESS and PVL were treated with vigabatrin. The median age at vigabatrin initiation was 10 (range, 7-25) months. Three of the seven patients (42.8%) showed complete clinical cessation of epileptic spasms after 3 months of vigabatrin. The median duration between vigabatrin initiation and epileptic spasm cessation was 5 (range: 3-61) days. The adverse effects included poor feeding (n = 1) and abnormal ERG findings during vigabatrin therapy (n = 2). These ERG findings normalized after vigabatrin was reduced or stopped. No respiratory failure, increased secretion, or somnolence was observed.
Conclusion:
Vigabatrin was effective for treating 42.8% of our patients with both IESS and PVL. Vigabatrin was well tolerated, with only a mild effect observed in patients with IESS and PVL. Some treatment-related ERG abnormalities were normalized after vigabatrin was reduced or stopped.
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