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Management of everolimus-associated adverse events in patients with tuberous sclerosis complex: a practical guide

Davies, Mark, Saxena, Anurag and Kingswood, John C. 2017. Management of everolimus-associated adverse events in patients with tuberous sclerosis complex: a practical guide. Orphanet Journal of Rare Diseases 12 (1) 10.1186/s13023-017-0581-9

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Tuberous sclerosis complex (TSC) is a genetic disorder characterised by highly variable comorbid dysfunction and subsequent morbidity. The mTOR inhibitor everolimus is indicated for the treatment of adult TSC patients with renal angiomyolipomas (AMLs) and for subependymal giant astrocytoma (SEGA) in both adults and children, based on data from the EXIST-1 and EXIST-2 trials. However, due to the historical predominance of everolimus in the oncology setting, some physicians who treat TSC patients may be unfamiliar with everolimus-associated adverse events (AEs) and appropriate management strategies. This article aims to serve as a resource for specialists including nephrologists, paediatricians, neurologists and geneticists who require practical guidance on the management of events such as non-infectious pneumonitis, rash, stomatitis, infections, and renal AEs. Additional consideration is given to drug interactions, hepatic impairment, fertility, and sexual maturation. Since patients with TSC receive clinical benefit from continued therapy, it is important that everolimus-related events are dealt with appropriately through strategies such as dose modification, interruption, the provision of supportive care, regular monitoring, and patient education.

Item Type: Article
Date Type: Published Online
Status: Published
Schools: Medicine
Uncontrolled Keywords: Everolimus, Adverse events, Tuberous sclerosis complex, TSC, Subependymal giant astrocytoma, SEGA, Renal angiomyolipoma, AML
Publisher: BioMed Central
ISSN: 1750-1172
Date of First Compliant Deposit: 29 August 2017
Date of Acceptance: 31 January 2017
Last Modified: 06 May 2023 08:59

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