Document Detail

Rapamycin as an alternative to surgical treatment of subependymal giant cell astrocytomas in a patient with tuberous sclerosis complex.
MedLine Citation:
PMID:  20887114     Owner:  NLM     Status:  MEDLINE    
Tuberous sclerosis complex (TSC) is associated with the potential development of benign hamartomas, including subependymal giant cell astrocytomas (SEGAs). Intracranial hypertension can be caused by SEGAs due to their propensity to block the foramen of Monro. The traditional management approach is to monitor SEGAs with periodic neuroimaging and to resect those that exhibit serial growth and/or cause clinical signs of intracranial hypertension. Recent observations suggest that rapamycin therapy may induce partial regression of SEGAs, therefore providing a potential alternative to resection. The authors present the case of an 8-year-old girl with bilateral SEGAs that led to progressive hydrocephaly and incipient signs of papilledema. Three months after initiating rapamycin therapy, the SEGAs exhibited significant reduction in size (82.6% on the left and 46.7% on the right), and the lesions remained stable 5 months later. Compared with previous case reports, similar or even greater antitumor efficacy was achieved with much lower trough levels of rapamycin (10–15 compared with 3.3–4.5 ng/ml, respectively). The authors discuss various aspects of rapamycin therapy and address unresolved issues that highlight the need for further prospective clinical trials.
Ala Birca; Claude Mercier; Philippe Major
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Publication Detail:
Type:  Case Reports; Journal Article    
Journal Detail:
Title:  Journal of neurosurgery. Pediatrics     Volume:  6     ISSN:  1933-0715     ISO Abbreviation:  J Neurosurg Pediatr     Publication Date:  2010 Oct 
Date Detail:
Created Date:  2010-10-04     Completed Date:  2010-10-25     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  101463759     Medline TA:  J Neurosurg Pediatr     Country:  United States    
Other Details:
Languages:  eng     Pagination:  381-4     Citation Subset:  IM    
Department of Pediatrics, Neurology Service, Centre Hospitalier Universitaire Sainte-Justine, Université de Montréal, Québec, Canada.
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MeSH Terms
Antibiotics, Antineoplastic / administration & dosage*
Astrocytoma / complications,  drug therapy*,  pathology
Brain Neoplasms / complications,  drug therapy*,  pathology
Magnetic Resonance Imaging
Neurosurgical Procedures
Sirolimus / administration & dosage*
Tuberous Sclerosis / complications*
Reg. No./Substance:
0/Antibiotics, Antineoplastic; 53123-88-9/Sirolimus

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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