Abstract | BACKGROUND: METHODS AND PATIENTS: RESULTS: The short-term efficacy and safety of everolimus in patients with tuberous sclerosis complex has been demonstrated in placebo-controlled trials, and open-label extension studies are ongoing to monitor long-term effects, including safety. Examples of regrowth following discontinuation of mTOR inhibitors suggest that everolimus needs to be given indefinitely to maintain suppression of subependymal giant cell astrocytoma and other tuberous sclerosis complex-associated disease manifestations. No additional safety concerns have been reported to date with long-term administration of everolimus, but published long-term data (>1 year treatment) are currently limited to a small open-label trial and case reports for this relatively rare condition. CONCLUSIONS: From the limited data available, long-term administration of everolimus appears feasible with few safety concerns beyond those associated with short-term use. Further investigation is needed to determine the long-term efficacy and safety of everolimus in patients with tuberous sclerosis complex.
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Authors | Lily H Tran, Mary L Zupanc |
Journal | Pediatric neurology
(Pediatr Neurol)
Vol. 53
Issue 1
Pg. 23-30
(Jul 2015)
ISSN: 1873-5150 [Electronic] United States |
PMID | 26092412
(Publication Type: Journal Article, Review)
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Copyright | Copyright © 2015 Elsevier Inc. All rights reserved. |
Chemical References |
- Antineoplastic Agents
- Everolimus
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Topics |
- Antineoplastic Agents
(administration & dosage, adverse effects)
- Child
- Clinical Trials as Topic
- Everolimus
(administration & dosage, adverse effects)
- Humans
- Tuberous Sclerosis
(drug therapy, genetics, metabolism)
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