Hypercholesterolemia in child on mTOR inhibitor

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rbanks47's picture
Hypercholesterolemia in child on mTOR inhibitor

We have been consulted to see a 22 m/o infant who has an unresectable hepatoblastoma with multiple lung metastases.  The child is enrolled in an IND protocol using vincristine, irinotecan, and temsirolimus. Prior to induction, the total cholesterol was 345 mg/dl, with HDL-C 38 mg/dl, LDL-C direct 241 mg/dl, and TG 109 mg/dl.  One week after starting protocol, TC 420 mg/dl, HDL-C 20 mg/dl, TG 207 mg/dl, and LDL-C direct 368 mg/dl.  Presumably, this is the response to the mTOR inhibiting effect of temsirolimus.  The protocol, which is a last ditch effort in this child, indicates that  therapy be discontinued  if hypercholesterolemia persists when treated with a statin. 


Has anyone run into a similar issue in a child this young, and if so, is there a prefered statin for hypercholesterolemia induced by mTOR inhibition, which evidently decreases hepatic LDL-R expression and decreases SR-B1 expression?  What would the dose be?  Is anyone aware of a weight- or BSA-based dosing for statins in this age?


Thank you.


Richard A. Banks MD
Pediatric Endocrinology

Arnold Palmer Pediatric Subspecialty Group

Orlando, FL

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