Objective: To probe into the blood concentration of sirolimus in children

and to investigate the correlation between blood concentration and related laboratory indicators

so as to provide more clinical evidence for the rational use of sirolimus. Methods: Fifty-four pediatric patients treated with sirolimus were enrolled

and the basic information

blood concentration of sirolimus (CSRL)

liver function and neutrophil (NEU) and other related information were recorded. Results: The blood concentration of sirolimus in children was (8.7±5.9) ng/mL

16.7% of the children with blood concentration of <4.0 ng/mL

62.9% of the children with blood concentration from 4.0 to 12.0 ng/mL

and 20.4 % of children with blood drug concentration >12.0 ng/mL. Sirolimus was used for tuberous sclerosis in 51.9% of children

with the lowest blood concentration of sirolimus 4.6 (3.9) ng/mL when compared with other diseases. In the treatment of tuberous sclerosis

60.7% of children were treated with antiepileptic drugs in combination

of which the proportion of drug combination of valproic acid was the highest (57.1%). Aspartate aminotransferase (AST) was positively correlated with CSRL (r=0.458

P<0.05). In children with blood concentration >12.0 ng/mL

there was a linear increase in the rate of abnormal AST

alanine aminotransferase (ALT) and NEU. Conclusion: When sirolimus is used in children

it is even more important to monitor the blood concentration

blood routine and liver function on a timely and regular basis to fully implement the individualized dosing to ensure clinical efficacy.