Effects Of Chronic Rapamycin Treatment On Plasma Lipids And Fatty Acid Oxidation In Children With Tuberous Sclerosis Complex

Objective:Todetermine changes of plasma lipidsand fatty acid oxidation after chronic rapamycin treatmentinchildren with tuberous sclerosiscomplexand to elucidate theeffects ofrapamycin on lipid metabolism.Method:Collect the clinical data of 52 children diagnosed with TSC from pediatric department of PLA general hospital from Aug.2014 to Dec.2015. According to the inclusion and exclusion standards, there were 40 cases effective. Analyzethe role of rapamycin in lipid metabolism from the following two respects.Firstly,observechanges of plasma lipids in children with tuberous sclerosis complex after rapamycin treatment. Total serum cholesterol,triglycerides,low density lipoprotein cholesterol and high density lipoprotein cholesterolwere evaluatedbefore and 3months and 12months after rapamycin treatment.Additionally, compare the four detection index withcontrol groups, whose gender and age matched with the cases of 3 months and 12months after rapamycinrespectively.Secondly,discuss the role of rapamycin in fatty acid oxidation. The content of carnitine were evaluatedbefore and 3months and 12months after rapamycin treatment.41 healthy controls were assessed at baseline.Result:There were 21 males and 19 females in the 40 children. The median age for rapamycin treatment was 29.5 months (12.8-60.5 months).The total serum cholesterol and low density lipoprotein cholesterol significantly increased at 3months after rapamycin treatment (p<0.01), and increased in comparison with the controls(p<0.05), and stabilized thereafter.The high density lipoprotein cholesterol significantly increased at 12 months after rapamycin treatment(p<0.05). However, triglycerides had no statistically significant change.The amounts of carnitinewere pronouncedly higher in children with TSC in comparison with healthy controls. Themedium acylcarnitinesincreased the most obviously.After rapamycin treatment, the content offree carnitine and acylcarnitines were decreasedand the downward trend was more apparent with the treatment prolonged.The medium acylcarnitines was the most obvious as well.The content of free carnitine was below the normal value at 12 months after rapamycin treatment.The value of free carnitine reduced variously among different age groups. The group of 1-3 ages was the most obvious.Conclusion:1) The total serum cholesterol, low density lipoprotein cholesterol and high density lipoprotein cholesterol significantly increaseafter rapamycin treatment. However, triglycerides have no statistically significant change.2) Rapamycin facilitatesfatty acid oxidation.3) The value of free carnitine reduced variously among different age groups. The group of 1-3 ages was the most obvious.