PMID- 25719621
OWN - NLM
STAT- MEDLINE
DCOM- 20151103
LR  - 20211203
IS  - 1473-5741 (Electronic)
IS  - 0959-4973 (Linking)
VI  - 26
IP  - 4
DP  - 2015 Apr
TI  - Complications of mammalian target of rapamycin inhibitor anticancer treatment 
      among patients with tuberous sclerosis complex are common and occasionally 
      life-threatening.
PG  - 437-42
LID - 10.1097/CAD.0000000000000207 [doi]
AB  - The aim of this study was to evaluate the most common adverse events (AEs) linked 
      to everolimus therapy, a mammalian target of rapamycin (mTOR) inhibitor, in 
      children and adolescents with tuberous sclerosis complex (TSC) hospitalized in 
      one medical center. The study group included 18 patients with a diagnosis of 
      subependymal giant cell astrocytoma or renal angiomyolipoma related to TSC. The 
      median duration of therapy was 15 months. All clinical symptoms and laboratory 
      abnormalities including complete blood count, fasting lipid profile, glucose 
      level, and liver and kidney function tests were analyzed as potential AEs. The 
      most common AEs of everolimus therapy were laboratory abnormalities (100% of 
      patients) and infection complications (83 episodes in 15 patients). Infectious 
      episodes of pharyngitis (67%), diarrhea (44%), stomatitis (39%), and bronchitis 
      (39%) were the most common infections. They were mostly mild or moderate in 
      severity (grade 1-2). In two cases, life-threatening conditions related to mTOR 
      inhibitor treatment were encountered. The first was classified as grade 4 
      pleuropneumonia and Streptococcus pneumoniae sepsis, whereas the second was 
      classified as death related to AE (grade 5) Escherichia coli sepsis. The most 
      common laboratory abnormalities were hypercholesterolemia (13/18 patients - 72%) 
      and hypertriglyceridemia (12/18 patients - 66%). Neutropenia (12/18 patents - 
      66%) and anemia (8/18 patients - 44%) were the most common hematologic 
      toxicities. Everolimus treatment in TSC patients may lead to life-threatening 
      outcomes, including sepsis and death. Long-lasting effects of everolimus 
      treatment in the context of high incidences of different laboratory abnormalities 
      found in TSC patients are another subject that should be researched further.
FAU - Trelinska, Joanna
AU  - Trelinska J
AD  - aDepartment of Pediatrics, Oncology, Hematology and Diabetology, Medical 
      University of Lodz, Lodz bDepartment of Neurology & Epileptology and Pediatric 
      Rehabilitation, The Children's Memorial Health Institute, Warsaw, Poland.
FAU - Dachowska, Iwona
AU  - Dachowska I
FAU - Kotulska, Katarzyna
AU  - Kotulska K
FAU - Fendler, Wojciech
AU  - Fendler W
FAU - Jozwiak, Sergiusz
AU  - Jozwiak S
FAU - Mlynarski, Wojciech
AU  - Mlynarski W
LA  - eng
PT  - Case Reports
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PL  - England
TA  - Anticancer Drugs
JT  - Anti-cancer drugs
JID - 9100823
RN  - 0 (Antineoplastic Agents)
RN  - 9HW64Q8G6G (Everolimus)
RN  - EC 2.7.1.1 (MTOR protein, human)
RN  - EC 2.7.11.1 (TOR Serine-Threonine Kinases)
RN  - W36ZG6FT64 (Sirolimus)
SB  - IM
MH  - Adolescent
MH  - Adult
MH  - Angiomyolipoma/drug therapy/pathology
MH  - Antineoplastic Agents/*adverse effects
MH  - Child
MH  - Child, Preschool
MH  - Everolimus
MH  - Glioma, Subependymal/drug therapy/pathology
MH  - Humans
MH  - Infant
MH  - Male
MH  - Sirolimus/adverse effects/*analogs & derivatives
MH  - TOR Serine-Threonine Kinases/*antagonists & inhibitors
MH  - Tuberous Sclerosis/*drug therapy/pathology
MH  - Young Adult
EDAT- 2015/02/27 06:00
MHDA- 2015/11/04 06:00
CRDT- 2015/02/27 06:00
PHST- 2015/02/27 06:00 [entrez]
PHST- 2015/02/27 06:00 [pubmed]
PHST- 2015/11/04 06:00 [medline]
AID - 00001813-201504000-00008 [pii]
AID - 10.1097/CAD.0000000000000207 [doi]
PST - ppublish
SO  - Anticancer Drugs. 2015 Apr;26(4):437-42. doi: 10.1097/CAD.0000000000000207.