Use of Oral Sirolimus in Paediatric Patients with Serious Vascular Anomalies: Case Report

Authors

Hospital Pharmacy Specialist, Hospital Pharmacy Department, Complexo Hospitalario Universitario, A Coruña, Spain.

Abstract

Background
Pediatric vascular anomalies comprise a heterogeneous group of clinical disorders of varying severity. These anomalies are divided into vascular tumors or vascular malformations. Different therapeutic strategies have been used depending on the pathology in question. In recent years, mammalian target of rapamycin (mTOR) inhibitors have been explored as a therapeutic option in patients with complex vascular anomalies that fail to respond to conventional therapies.


Case Report
Two patients affected by complicated vascular anomalies were treated with sirolimus. Case 1: fourteen-month-old male patient with primary congenital lymphedema in the right arm. Case 2: boy aged two years and eight months, diagnosed with a non-respectable cervical aposiform hemangioendothelioma (KHE). Both patients received sirolimus at doses of 0.8 mg/m2/12 h during 12 and 13 months respectively. Monitoring plasma sirolimus concentrations made it possible to safely deal with problems associated with the medication. Both patients experiment an important improvement of their pathology, maintained after suspending treatment. No adverse effects related to treatment with sirolimus were observed.


Conclusion
Oral sirolimus proved to be an effective strategy in the treatment of complicated vascular anomalies in our pediatric population.

Keywords