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Objectives <p>To report early neurodevelopmental outcomes in a child with Tuberous Sclerosis Complex (TSC) treated with everolimus and vigabatrin.</p> Methods <p>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.</p> Results <p>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.</p> Discussion <p>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.</p>

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Early neurodevelopmental outcomes in a child with tuberous sclerosis complex treated with everolimus and vigabatrin

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年5月18日

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  • Sarah ML. Wilson,
  • Megan Samuels,
  • Luana Pimentel,
  • Shelley Varnado,
  • Claire Sartwell,
  • Rosleen Mansour,
  • Deborah Pearson,
  • Hope Northrup,
  • Mary Kay Koenig

摘要

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.