Efficacy and safety of oral sirolimus in the treatment of refractory vascular anomalies
10.3760/cma.j.cn114453-20200316-00159
- VernacularTitle:口服西罗莫司治疗难治性脉管性疾病的有效性与安全性
- Author:
Liangliang KONG
1
;
Tao HAN
;
Qingwen GAO
;
Jie CUI
;
Weimin SHEN
Author Information
1. 南京医科大学附属儿童医院烧伤整形外科 210008
- Keywords:
Sirolimus;
Hemangioendothelioma;
Lymphatic abnormalities;
Vascular malformation
- From:
Chinese Journal of Plastic Surgery
2020;36(5):487-493
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To investigate the efficacy and safety of oral sirolimus in the treatment of refractory vascular anomalies.Methods:From February 2017 to February 2018, 20 cases of vascular deformity with no obvious improvements after multiple therapies in our hospital were included. Among them, 5 have Kaposiform hemangioendothelioma (KHE) with Kasabach-Merritt phenomenon (KMP), 10 have lymphatic malformation, 3 have venous malformation and 2 have lymphatic venous malformation. A single course of oral sirolimus lasted for 3 months. Initial dose was 0.8 mg/m 2 once, oral administration twice per day, and subsequent dose was adjusted to maintain the concentration of blood drug at 10-15 ng/ml. Before and after taking sirolimus, the general information, tumor changes and the adverse reactions were gathered. Efficacy was evaluated at the end of the treatment course. Effective: tumor volume reduced by more than 50% and or platelets stabilized in the normal range. Partly effective: tumor volume reduced by 25%-50%. No effect: tumor volume reduced by less than 25% or no significant change. Results:All the patients were treated with sirolimus orally for 1-3 courses, among which 11 were effective, 6 were partially effective, and 3 were ineffective. They were followed up for more than 3 months after the end of the whole course and no tumor enlargement was observed. All the 5 cases with KHE and KMP had significantly reduced tumor size and the platelets were stable in the normal range, but 2 of them suffered from severe pneumonia, one of them eventually perished. Two of the remaining children had elevated liver enzymes and high fever. The lesions of 10 patients with lymphatic malformations were reduced, and 4 of them had mild liver dysfunction. Two with lymphatic venous malformations had shrunk in different degree, and 3 with venous malformations had no effect. Three of them showed significant relief of pain symptoms, and their pain score was significantly lower than that before oral administration (8.7±1.2 vs 1.3±1.2, P=0.001). Except for 1 case with venous malformation had oral ulcer. No obvious adverse reaction were observed in the remaining 11 patients. Conclusions:Oral sirolimus may be effective and safe in the treatment of refractory vascular anomalies, but it should be treated with caution in little infants.
