Faculty, Staff and Student Publications

Language

English

Publication Date

5-18-2026

Journal

BMC Neurology

DOI

10.1186/s12883-026-04921-6

PMID

42151856

Abstract

Objectives: To report early neurodevelopmental outcomes in a child with Tuberous Sclerosis Complex (TSC) treated with everolimus and vigabatrin.

Methods: The authors report a five-year-old girl with Tuberous Sclerosis Complex who was initiated on vigabatrin at seven weeks old to treat infantile spasms and on everolimus at two months of age to treat a subependymal giant cell astrocytoma. Neuropsychological assessments were conducted in this patient at 12, 24, and 36 months using the Bayley-III and Vineland-II.

Results: On the Bayley-III, the patient had a high average cognitive composite score at 12 months and a high average language composite score at 36 months. Bayley-III assessments at 12, 24, and 36 months, yielded average cognitive, language, and motor composite scores. On the Vineland-II at 12 months, her standard score fell in the moderately low range on daily living skills and overall adaptive behavior. Vineland-II assessments at 12, 24, and 36 months yielded average range standard scores in the areas of communication, socialization, daily living skills, overall adaptive behavior, and motor skills.

Discussion: This case highlights early neurodevelopmental outcomes in one TSC child treated with everolimus and vigabatrin in infancy. Continued assessment and longitudinal follow-up is required to understand the broader implications of early treatment with mTOR inhibitors and vigabatrin in TSC patients in childhood.

Keywords

Bayley-III, Everolimus, Neurodevelopment, Neuropsychological Testing, Tuberous Sclerosis Complex (TSC), Vigabatrin, Vineland-II, mTOR inhibitor

Published Open-Access

yes

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