The burden of subependymal giant cell astrocytomas associated with tuberous sclerosis complex: Results of a patient and caregiver survey

Anne M. Skalicky, Anne M. Rentz, Zhimei Liu, James W. Wheless, Corey L. Pelletier, David W. Dunn, Michael D. Frost, Joanne Nakagawa, Matthew Magestro, Judith Prestifilippo, Chris Pashos

Research output: Contribution to journalArticle

7 Scopus citations

Abstract

Tuberous sclerosis complex is a genetic disorder characterized by benign tumor growth including lesions in the ventricular system of the brain known as subependymal giant cell astrocytomas. This analysis focuses on the clinical presentation, management, and associated burden of subependymal giant cell astrocytomas in patients with tuberous sclerosis complex in the United States. An institutional review board-approved web-based survey of tuberous sclerosis complex patients and caregivers collected information, and descriptive analyses were conducted on age-based subgroups. A total of 116 tuberous sclerosis complex-subependymal giant cell astrocytoma patients or caregivers responded (17% of the total tuberous sclerosis complex sample). Mean and median patient ages were 25.5 and 23.5 years. Besides subependymal giant cell astrocytomas, patients also experienced skin lesions (72%), seizures (65%), and cognitive concerns (60%). Forty-five percent reported having brain surgery (22% for subependymal giant cell astrocytoma). In the past year, 42% of patients were admitted at least once to the hospital whereas 39% went to the emergency department. Results demonstrate that tuberous sclerosis complex-subependymal giant cell astrocytoma is associated with significant clinical burden, resource utilization, and decreased well-being.

Original languageEnglish (US)
Pages (from-to)563-569
Number of pages7
JournalJournal of Child Neurology
Volume30
Issue number5
DOIs
StatePublished - Apr 27 2015

Keywords

  • health care utilization
  • health-related quality of life
  • subependymal giant cell astrocytomas
  • tuberous sclerosis complex

ASJC Scopus subject areas

  • Clinical Neurology
  • Pediatrics, Perinatology, and Child Health

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  • Cite this

    Skalicky, A. M., Rentz, A. M., Liu, Z., Wheless, J. W., Pelletier, C. L., Dunn, D. W., Frost, M. D., Nakagawa, J., Magestro, M., Prestifilippo, J., & Pashos, C. (2015). The burden of subependymal giant cell astrocytomas associated with tuberous sclerosis complex: Results of a patient and caregiver survey. Journal of Child Neurology, 30(5), 563-569. https://doi.org/10.1177/0883073814523318