PMID- 27878332
OWN - NLM
STAT- MEDLINE
DCOM- 20170329
LR  - 20220331
IS  - 1432-1971 (Electronic)
IS  - 0172-0643 (Linking)
VI  - 38
IP  - 2
DP  - 2017 Feb
TI  - Accelerated Cardiac Rhabdomyoma Regression with Everolimus in Infants with 
      Tuberous Sclerosis Complex.
PG  - 394-400
LID - 10.1007/s00246-016-1528-y [doi]
AB  - Tuberous sclerosis complex is associated with benign tumors such as cardiac 
      rhabdomyomas (RHM) caused by the disinhibition of the mammalian target of 
      rapamycin (mTOR) protein. Recent reports on everolimus, an mTOR inhibitor, have 
      shown size reduction of RHM. We compared cases recently treated with everolimus 
      to historic controls whose first echocardiography was within first month of life. 
      The largest dimension of the largest RHM was reported as a percentage compared to 
      the earliest echocardiography study. Treatment of the four cases was started at a 
      median age of 6.5 days (range 2-20) with an initial enteral dose of 0.1 mg daily, 
      aiming at a therapeutic serum trough level of 5-15 ng/mL. Median duration of 
      everolimus treatment was 73 days (range 34-138). Compared to 10 historic 
      controls, everolimus-treated patients had 11.8 times faster RHM size regression 
      rate (slope -0.0285 vs. -0.0024; p < 0.001). The average time to 50% size 
      reduction was 1.13 +/- 0.33 month (range 0.66-1.4 months) with everolimus versus 
      72.9 +/- 53.03 months in controls (p = 0.026). Following treatment with everolimus, 
      one case was operated for congenital heart disease, without requirement of RHM 
      resection, two others had the massive left ventricle RHM shrink to 
      non-consequential size. The latter had a disappearance of RHM, but everolimus 
      therapy was maintained to prevent the regrowth of a significant cerebral tumor. 
      Everolimus is efficacious for size reduction of RHM during the neonatal period. 
      With limited safety data, this approach should be used with caution in selective 
      cases.
FAU - Aw, Fatou
AU  - Aw F
AD  - Division of Pediatric Cardiology, Department of Pediatrics, CHU Sainte-Justine, 
      University of Montreal, 3175, Cote Sainte-Catherine, Montreal, QC, H3T 1C5, 
      Canada.
AD  - Aristide le Dantec Hospital, Cheikh Anta Diop University, Dakar, Senegal.
FAU - Goyer, Isabelle
AU  - Goyer I
AD  - Division of Clinical Pharmacology, Department of Pharmacy, CHU Sainte-Justine, 
      University of Montreal, Montreal, QC, Canada.
FAU - Raboisson, Marie-Josee
AU  - Raboisson MJ
AD  - Division of Pediatric Cardiology, Department of Pediatrics, CHU Sainte-Justine, 
      University of Montreal, 3175, Cote Sainte-Catherine, Montreal, QC, H3T 1C5, 
      Canada.
FAU - Boutin, Christine
AU  - Boutin C
AD  - Division of Pediatric Cardiology, Department of Pediatrics, CHU Sainte-Justine, 
      University of Montreal, 3175, Cote Sainte-Catherine, Montreal, QC, H3T 1C5, 
      Canada.
FAU - Major, Philippe
AU  - Major P
AD  - Department of Neurological Sciences, CHU Sainte-Justine, University of Montreal, 
      Montreal, QC, Canada.
FAU - Dahdah, Nagib
AU  - Dahdah N
AUID- ORCID: 0000-0001-7715-3169
AD  - Division of Pediatric Cardiology, Department of Pediatrics, CHU Sainte-Justine, 
      University of Montreal, 3175, Cote Sainte-Catherine, Montreal, QC, H3T 1C5, 
      Canada. nagib.dahdah.hsj@ssss.gouv.qc.ca.
LA  - eng
PT  - Journal Article
DEP - 20161123
PL  - United States
TA  - Pediatr Cardiol
JT  - Pediatric cardiology
JID - 8003849
RN  - 0 (Antineoplastic Agents)
RN  - 9HW64Q8G6G (Everolimus)
SB  - IM
MH  - Antineoplastic Agents/*administration & dosage
MH  - Case-Control Studies
MH  - Echocardiography
MH  - Everolimus/*administration & dosage
MH  - Female
MH  - Heart Neoplasms/*drug therapy/pathology
MH  - Heart Ventricles/pathology
MH  - Humans
MH  - Infant, Newborn
MH  - Male
MH  - Regression Analysis
MH  - Rhabdomyoma/*drug therapy/pathology
MH  - Tuberous Sclerosis/*complications
OTO - NOTNLM
OT  - Cardiac tumor
OT  - Newborn
OT  - Rhabdomyoma
OT  - Tuberous sclerosis
EDAT- 2016/11/24 06:00
MHDA- 2017/03/31 06:00
CRDT- 2016/11/24 06:00
PHST- 2016/05/03 00:00 [received]
PHST- 2016/11/11 00:00 [accepted]
PHST- 2016/11/24 06:00 [pubmed]
PHST- 2017/03/31 06:00 [medline]
PHST- 2016/11/24 06:00 [entrez]
AID - 10.1007/s00246-016-1528-y [pii]
AID - 10.1007/s00246-016-1528-y [doi]
PST - ppublish
SO  - Pediatr Cardiol. 2017 Feb;38(2):394-400. doi: 10.1007/s00246-016-1528-y. Epub 
      2016 Nov 23.