Early Vigabatrin Treatment Prevents Infantile Spasms but Fails to Prevent Seizures Overall in Children with TSC
Research By: Darcy Krueger, MD, PhD
Post Date: August 28, 2023 | Publish Date: Aug. 28, 2023
Neurology | Top Scientific Achievement
Tuberous sclerosis complex (TSC) is an uncommon genetic disorder caused by changes in two genes, TSC1 and TSC2. When these genes don’t work properly, they can lead to problems throughout the body.
Up to 90% of newborns with TSC develop epilepsy, often within their first year of life. For nearly two-thirds of these children, epilepsy becomes difficult to control with medication. Because TSC-related brain activity problems can start before visible seizures, researchers wondered if starting treatment before seizures begin might improve outcomes. Darcy Krueger, MD, PhD, Division of Neurology, and Martina Bebin, MPA, MD, University of Alabama at Birmingham, led the phase IIb PREVeNT trial, testing whether early treatment with the anti-seizure drug vigabatrin could improve outcomes.
The team randomly assigned 56 newborns with TSC to two groups: one began vigabatrin within the first six months, the other received standard care. After two years, those on early vigabatrin treatment were less likely to develop infantle spasms. However, no significant differences were observed for focal seizures or seizures overall. Children who received vigabatrin also did not have higher cognitive scores than those who received standard care.
“These findings suggest that while vigabatrin can help manage certain seizure types, starting it early does not change overall outcomes in children with TSC,” Krueger says.
Further research is needed to improve quality of life and cognitive health in children with TSC. Krueger and Bebin are leading a clinical trial using sirolimus to target TSC more directly at the molecular level. Sirolimus and similar medications have already been FDA-approved to treat several other clinical manifestations of TSC.
Co-authors include Jurriaan M. Peters, MD, PhD, from Boston Children’s Hospital and Brenda E. Porter, MD, PhD, from Stanford University. Research support came from the Cincinnati Children’s Epilepsy Research Program and the University of Alabama Biostatistics Core.
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| Original title: | Early Treatment with Vigabatrin Does Not Decrease Focal Seizures or Improve Cognition in Tuberous Sclerosis Complex: The PREVeNT Trial |
| Published in: | Annals of Neurology |
| Publish date: | Aug. 28, 2023 |
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