PMID- 36615165
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20230111
IS  - 2077-0383 (Print)
IS  - 2077-0383 (Electronic)
IS  - 2077-0383 (Linking)
VI  - 12
IP  - 1
DP  - 2023 Jan 3
TI  - Safety of Sirolimus in Patients with Tuberous Sclerosis Complex under Two Years 
      of Age-A Bicenter Retrospective Study.
LID - 10.3390/jcm12010365 [doi]
LID - 365
AB  - BACKGROUND: mTOR inhibitors are a novel pharmacotherapy recommended for 
      subependymal giant astrocytomas, refractory epilepsy, and the treatment of the 
      other clinical manifestations of tuberous sclerosis complex (TSC). Clinical 
      trials on everolimus proved it to be effective and safe in children. Despite its 
      common use in clinical practice, the research on sirolimus is limited. This study 
      is the first to determine and assess the severity of the adverse effects (AEs) of 
      sirolimus administered to children with TSC under two years of age. METHODS: We 
      performed a bicenter retrospective data analysis of medical records of 
      individuals with TSC who initiated therapy with sirolimus under the age of two. 
      RESULTS: Twenty-one patients were included in the study. At least one AE was 
      reported in all participants. The most prevalent AEs were anemia, thrombocytosis, 
      and hyperlipidemia. Infections and mouth ulcerations, often reported in the 
      studies on older patients, were infrequent and of mild or moderate grade. 
      CONCLUSIONS: Adverse effects associated with sirolimus use in infants and young 
      children with TSC are frequent yet not life- or health-threatening. Further 
      multicenter prospective clinical trials should determine the long-term safety of 
      sirolimus.
FAU - Smialek, Dominika
AU  - Smialek D
AUID- ORCID: 0000-0003-0776-193X
AD  - Department of Pediatric Neurology, Medical University of Warsaw, 02-091 Warsaw, 
      Poland.
FAU - Jozwiak, Sergiusz
AU  - Jozwiak S
AUID- ORCID: 0000-0003-3350-6326
AD  - Research Department, The Children's Memorial Health Institute, 04-736 Warsaw, 
      Poland.
FAU - Kotulska, Katarzyna
AU  - Kotulska K
AD  - Department of Neurology and Epileptology, The Children's Memorial Health 
      Institute, 04-736 Warsaw, Poland.
LA  - eng
GR  - 2019/ABM/01/00034/Medical Research Agency, Poland/
GR  - 2020/ABM/01/00054/Medical Research Agency, Poland/
PT  - Journal Article
DEP - 20230103
PL  - Switzerland
TA  - J Clin Med
JT  - Journal of clinical medicine
JID - 101606588
PMC - PMC9821318
OTO - NOTNLM
OT  - adverse effect
OT  - infant
OT  - mTOR inhibitor
OT  - safety
OT  - sirolimus
OT  - tuberous sclerosis complex
COIS- The authors declare no conflict of interest.
EDAT- 2023/01/09 06:00
MHDA- 2023/01/09 06:01
PMCR- 2023/01/03
CRDT- 2023/01/08 01:32
PHST- 2022/11/18 00:00 [received]
PHST- 2022/12/29 00:00 [revised]
PHST- 2022/12/30 00:00 [accepted]
PHST- 2023/01/08 01:32 [entrez]
PHST- 2023/01/09 06:00 [pubmed]
PHST- 2023/01/09 06:01 [medline]
PHST- 2023/01/03 00:00 [pmc-release]
AID - jcm12010365 [pii]
AID - jcm-12-00365 [pii]
AID - 10.3390/jcm12010365 [doi]
PST - epublish
SO  - J Clin Med. 2023 Jan 3;12(1):365. doi: 10.3390/jcm12010365.