Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies: a Randomized Controlled Trial
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 75
- 试验地点
- 1
- 主要终点
- Effective rate
研究概览
简要总结
In this study, we investigate the safety and efficacy of topical sirolimus in the treatment of superficial complicated vascular anomolies.
详细描述
Vascular anomaly is a kind of rare disease. According to histology, biological behavior and clinical manifestations, it can be divided into two categories: vascular tumor and vascular malformation.
mTOR inhibitors are proved with the properties of anti-proliferation and anti-angiogenesis. Therefore, they have been used in the treatment of vascular anomalies. Sirolimus, by its ability to prevent downstream protein synthesis and subsequent cell proliferation and angiogenesis, has become a novel and effective treatment. However, after the children reach complete response, there may still be skin manifestations that affect the appearance and cause psychological shadows. Therefore, intervention is required.
Studies have reported that topical sirolimus is effective in treating Kaposiform Hemangioendothelioma (KHE). It is absorbed through the skin, avoiding the first pass elimination effect of the liver. Fewer adverse reactions have been observed. In this study, we investigate the efficacy and safety of percutaneous administration of sirolimus in the treatment of superficial complicated vascular anomalies.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 0 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant clinically or pathologically diagnosed with KHE, TA or complicated superficial vascular anomolies involving lymphatic components.
- •The case is initial, with a relatively limited superficial lesion.
- •The participant has residual surface lesions after oral medication.
- •Participant with no use of other medication or surgical treatment
- •Participant with detailed medical records of the disease at the time of screening
- •Participant with signed and dated informed consent from the guardian(s)
排除标准
- •Participants with Kasabach-Merritt Phenomenon, with platelets <50×10 9 /L.
- •Participants with general disease such as hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney dysfunction, and cardiopulmonary insufficiency.
- •Participants with other hematological diseases or solid tumor.
- •Participants allergic to sirolimus or dressing.
研究组 & 干预措施
Topical use of sirolimus
Drop 5 ml of sirolimus oral solution and 5 g of dressing into the mixed bottle. Apply mixed gel of topical sirolimus to affected area. Use it twice a day for 6 months.
干预措施: Percutaneous sirolimus (Drug)
Oral use of sirolimus
Oral dose of sirolimus is calculated according to body surface area. Take it twice a day for 6 months. Maintain the blood concentration of sirolimus at 5-15ng/ml.
干预措施: Oral sirolimus (Drug)
结局指标
主要结局
Effective rate
时间窗: From admission to follow-up six months
Achauer BM et al. made the four-level standard as follows. Grade I: tumor size and skin lesion color regression ≤ 25%; grade II: tumor size and skin lesion color regression 25%-50%; grade III: tumor size and skin lesion color regression 50-75%; grade IV: tumor size and skin lesion color regression ≥75%. After 6 months of treatment, the pzrticipant will be evaluated. Grade I will be viewed as invalid. Grade II and grade III will be viewed as effective, and grade IV will be viewed as very effective. Those in grade Ⅱ, Ⅲ or Ⅳ will be calculated in effectiveness rate.
次要结局
- Changes of peak blood flow(From admission to follow-up six months)
- Changes of resistance coefficient(From admission to follow-up six months)
- Platelet count(From admission to follow-up six months)
- Incidence of adverse events(From admission to follow-up six months)