基于治疗药物监测的西罗莫司在新生儿罕见病中的精准用药实践

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

关键词:  脉管畸形、结节性硬化症、先天性高胰岛素血症、新生儿、西罗莫司

Key words:  Vascular anomalies  Tuberous sclerosis complex  Congenital hyperinsulinism  Neonate  Sirolimus  Therapeutic drug monitoring (TDM)

摘要:

目的 探讨治疗药物监测下西罗莫司在新生儿罕见病个体化治疗中应用价值和实践策略。方法 回顾性总结我院确诊为复杂脉管畸形、结节性硬化症、先天性高胰岛素血症等罕见病并接受西罗莫司治疗的新生儿病例，临床药师基于治疗药物监测制定个体化给药方案，并实施严密的药学监护。结果 治疗药物监测引导下的西罗莫司个体化方案使所有患儿实现血药浓度达标，并伴随显著临床疗效，且患儿耐受良好。结论 西罗莫司为新生儿难治性疾病提供新选择，基于治疗药物监测的精准用药策略是确保其疗效和安全性的关键

Abstract:

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 (TSC), and congenital hyperinsulinism (CHI) who received sirolimus treatment in our hospital. 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.