PMID- 28202028
OWN - NLM
STAT- MEDLINE
DCOM- 20171130
LR  - 20190109
IS  - 1750-1172 (Electronic)
IS  - 1750-1172 (Linking)
VI  - 12
IP  - 1
DP  - 2017 Feb 15
TI  - Management of everolimus-associated adverse events in patients with tuberous 
      sclerosis complex: a practical guide.
PG  - 35
LID - 10.1186/s13023-017-0581-9 [doi]
LID - 35
AB  - 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.
FAU - Davies, Mark
AU  - Davies M
AD  - Department of Oncology, South West Wales Cancer Centre, Singleton Hospital, 
      Swansea, SA2 8QA, UK. David.Davies4@wales.nhs.uk.
FAU - Saxena, Anurag
AU  - Saxena A
AD  - Division of Cancer and Genetics, Cardiff University School of Medicine, Institute 
      of Medical Genetics, Cardiff, CF14 4XN, UK.
FAU - Kingswood, John C
AU  - Kingswood JC
AD  - Sussex Kidney Unit, Royal Sussex County Hospital, Eastern Road, Brighton, BN2 
      5BE, UK.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Review
DEP - 20170215
PL  - England
TA  - Orphanet J Rare Dis
JT  - Orphanet journal of rare diseases
JID - 101266602
RN  - 9HW64Q8G6G (Everolimus)
SB  - IM
MH  - Adult
MH  - Child
MH  - Everolimus/*adverse effects/*therapeutic use
MH  - Humans
MH  - Tuberous Sclerosis/*drug therapy
PMC - PMC5311836
OTO - NOTNLM
OT  - AML
OT  - Adverse events
OT  - Everolimus
OT  - Renal angiomyolipoma
OT  - SEGA
OT  - Subependymal giant astrocytoma
OT  - TSC
OT  - Tuberous sclerosis complex
EDAT- 2017/02/17 06:00
MHDA- 2017/12/01 06:00
PMCR- 2017/02/15
CRDT- 2017/02/17 06:00
PHST- 2016/11/17 00:00 [received]
PHST- 2017/01/31 00:00 [accepted]
PHST- 2017/02/17 06:00 [entrez]
PHST- 2017/02/17 06:00 [pubmed]
PHST- 2017/12/01 06:00 [medline]
PHST- 2017/02/15 00:00 [pmc-release]
AID - 10.1186/s13023-017-0581-9 [pii]
AID - 581 [pii]
AID - 10.1186/s13023-017-0581-9 [doi]
PST - epublish
SO  - Orphanet J Rare Dis. 2017 Feb 15;12(1):35. doi: 10.1186/s13023-017-0581-9.