Successful everolimus therapy for SEGA in pediatric patients with tuberous sclerosis complex

Página do item simplificado
dc.contributor.authorCappellano, A. M. [UNIFESP]
dc.contributor.authorSenerchia, A. A. [UNIFESP]
dc.contributor.authorAdolfo, F. [UNIFESP]
dc.contributor.authorPaiva, P. M. [UNIFESP]
dc.contributor.authorPinho, R. [UNIFESP]
dc.contributor.authorCovic, A. [UNIFESP]
dc.contributor.authorCavalheiro, S. [UNIFESP]
dc.contributor.authorSaba, N. [UNIFESP]
dc.contributor.institutionUniversidade Federal de São Paulo (UNIFESP)
dc.date.accessioned2016-01-24T14:34:46Z
dc.date.available2016-01-24T14:34:46Z
dc.date.issued2013-12-01
dc.description.abstractTuberous sclerosis complex (TSC) is associated with hamartomatous growths including subependymal giant cell astrocytomas (SEGAs). Although, SEGAs are slow-growing glioneuronal tumors, they represent a significant cause of morbidity and mortality due to the risk of sudden death from acute hydrocephalus. Neurosurgical resection has been the mainstay of therapy, since radiotherapy and chemotherapy were proved inefficient in those tumors. Recent studies support the use of everolimus for subependymal giant cell astrocytomas associated with tuberous sclerosis and suggest it might represent a disease-modifying treatment for other aspects of tuberous sclerosis.We describe the clinical and radiological progression of three pediatric patients with definitive diagnosis of TSC and SEGA, which have been treated with everolimus.Up to 34 % sustained SEGA decrease was observed in the three cases. All three patients have experienced seizure control and two of them have showed cognitive and behavioral improvement. Everolimus has been well tolerated by all. No severe adverse events have been observed to date.Everolimus offers significant promise in treating SEGAs. Studies are required to explore optimal therapy duration and management upon discontinuing therapy.en
dc.description.affiliationUniversidade Federal de São Paulo, GRAACC, Inst Pediat Oncol, BR-04023062 São Paulo, Brazil
dc.description.affiliationUnifespUniversidade Federal de São Paulo, GRAACC, Inst Pediat Oncol, BR-04023062 São Paulo, Brazil
dc.description.sourceWeb of Science
dc.description.sponsorshipNovartis Biociencias S.A.
dc.format.extent2301-2305
dc.identifierhttp://dx.doi.org/10.1007/s00381-013-2170-0
dc.identifier.citationChilds Nervous System. New York: Springer, v. 29, n. 12, p. 2301-2305, 2013.
dc.identifier.doi10.1007/s00381-013-2170-0
dc.identifier.issn0256-7040
dc.identifier.urihttp://repositorio.unifesp.br/handle/11600/37027
dc.identifier.wosWOS:000327894900024
dc.language.isoeng
dc.publisherSpringer
dc.relation.ispartofChilds Nervous System
dc.rightsinfo:eu-repo/semantics/restrictedAccess
dc.rights.licensehttp://www.springer.com/open+access/authors+rights?SGWID=0-176704-12-683201-0
dc.subjectTuberous sclerosis complexen
dc.subjectSubependymal giant cell astrocytomaen
dc.subjectmTORen
dc.subjectEverolimusen
dc.titleSuccessful everolimus therapy for SEGA in pediatric patients with tuberous sclerosis complexen
dc.typeinfo:eu-repo/semantics/article
Arquivos
Coleções
EPM - Artigos