Precision Medication Practice of Sirolimus Based on Therapeutic Drug Monitoring in Neonates with Rare Diseases

OBJECTIVE To explore the application value and practical strategy of individualized sirolimus therapy guided by therapeutic drug monitoring(TDM) in neonates with rare diseases.

METHODS A retrospective analysis was conducted on neonatal cases diagnosed with rare diseases such as complex vascular anomalies, tuberous sclerosis complex, and congenitalhyperinsulinism who received sirolimus treatment in The Second Affiliated Hospital and Yuying Children’s Hospital of Wenzhou Medical University. Clinical pharmacists developed individualized dosing regimens based on TDM and implemented rigorous pharmaceutical care.

RESULTS The TDM-guided individualized sirolimus regimens achieved target serum concentrations in all patients, which was accompanied by significant clinical efficacy, and the neonates tolerated the treatment well.

CONCLUSION Sirolimus provides a new therapeutic option for refractory diseases in neonates. A precision medication strategy based on TDM is crucial for ensuring its efficacy and safety.