title: Management of everolimus-associated adverse events in patients with tuberous sclerosis complex: a practical guide.
abstract: 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.
authors.affiliation: Department of Oncology, South West Wales Cancer Centre, Singleton Hospital, Swansea, SA2 8QA, UK. David.Davies4@wales.nhs.uk.
authors.forename: Mark
authors.initials: M
authors.lastname: Davies
authors.affiliation: Division of Cancer and Genetics, Cardiff University School of Medicine, Institute of Medical Genetics, Cardiff, CF14 4XN, UK.
authors.forename: Anurag
authors.initials: A
authors.lastname: Saxena
authors.affiliation: Sussex Kidney Unit, Royal Sussex County Hospital, Eastern Road, Brighton, BN2 5BE, UK.
authors.forename: John C
authors.initials: JC
authors.lastname: Kingswood
mesh_terms: D000328:Adult; D002648:Child; D000068338:Everolimus / Q000009:adverse effects* / Q000627:therapeutic use*; D006801:Humans; D014402:Tuberous Sclerosis / Q000188:drug therapy*
chemical_list: D000068338:Everolimus
keywords: AML; Adverse events; Everolimus; Renal angiomyolipoma; SEGA; Subependymal giant astrocytoma; TSC; Tuberous sclerosis complex