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Pediatric blood & cancer · Jul 2016
Case ReportsMammalian Target of Rapamycin Inhibitor Induced Complete Remission of a Recurrent Subependymal Giant Cell Astrocytoma in a Patient Without Features of Tuberous Sclerosis Complex.
- Deepika Appalla, Andres Depalma, and Stanley Calderwood.
- Division of Pediatric Hematology and Oncology, Department of Paediatrics, The Children's Hospital at Saint Peter's University Hospital, New Brunswick, New Jersey.
- Pediatr Blood Cancer. 2016 Jul 1; 63 (7): 1276-8.
AbstractThe majority of patients with subependymal giant cell astrocytoma (SEGA) have tuberous sclerosis complex (TSC). In such patients, the mammalian target of rapamycin (mTOR) inhibitor everolimus has been shown to induce responses. Isolated SEGA have been reported in patients without clinical or genetic features of TSC. The treatment of these patients with everolimus has not previously been reported. We treated a patient with a recurrent isolated SEGA with an mTOR inhibitor. The patient tolerated therapy well and had a sustained complete remission. MTOR inhibitors may be useful for the treatment of isolated SEGA. Further study is warranted.© 2016 Wiley Periodicals, Inc.
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