Safety and Efficacy of Low-dose Sirolimus to Kaposiform Hemangioendothelioma:A Prospective, Randomized Open Trial
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 92
- 试验地点
- 1
- 主要终点
- response to treatment
研究概览
简要总结
to evaluate the safety and efficacy of Low-dose sirolimus in Kaposiform Hemangioendothelioma in Chinese children by a prospective, randomized open trial.
详细描述
The clinically commonly used dose of sirolimus for Kaposiform Hemangioendothelioma is 0.8 mg/m2 administered twice daily, and the blood concentration can be maintained at 10-15 ng/ml according to the pharmacokinetic formula.Related research reports that maintaining low blood concentration of sirolimus is effective in the treatment of certain vascular malformations and hemangioma, and complications are less. In the clinical practice, we found that the blood concentration was maintained at 7-10 ng/ml, and the patients still achieved good results and the chance of infections decreased. Therefore, this clinical trial was designed.In this trial, two different dosing regimens with corresponding blood concentration were designed to compare the safety and efficacy.In the high concentration group, the sirolimus dosage was adjusted monthly to achieve trough levels between 10 and 15 ng/mL(excluding 10 ng/ml), and it is still used at 0.8 mg/m2 administered twice daily.The low concentration group is 7-10 ng/ml (including 10 ng/ml), and the initial use of sirolimus is 0.7mg/m2 administered twice daily.The dose was adjusted according to the formula after two weeks.The follow-up and evaluation were performed according to a strictly established follow-up schedule after taking the drug.
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
- None
入排标准
- 年龄范围
- — 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Kaposiform Hemangioendotheliomas with or without Kasabach-Merritt Phenomenon.
- •0 - 12 years of age at the time of study entry.
- •Male or female.
- •Consent of parents (or the person having parental authority in families): Signed and dated written informed consent.
排除标准
- •with hematological diseases.
- •with other solid tumors.
- •with hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney dysfunction, and cardiopulmonary insufficiency.
- •with tuberculosis,cytomegalovirus and Epstein-Barr virus infection before the treatment.
研究组 & 干预措施
high blood concentration group
The blood concentration is maintained at 10-15ng/ml (not including 10ng/ml).
干预措施: Sirolimus(0.8mg/m2) (Drug)
low blood concentration group
The blood concentration is maintained at 7-10ng/ml (including 10ng/ml).
干预措施: Sirolimus(0.7mg/m2) (Drug)
结局指标
主要结局
response to treatment
时间窗: 1 year after taking the drug
it is a variable outcome, including complete responses, partial response and no response. The definitions are : Complete Response： 1. platelets counts is greater than 100×10\^9/L. 2. significant volume reduction is greater than 80%. 3. Fibrinogen levels at 2-4g/L. 4. The surface skin of the tumor is lighter or the tumor is softer significantly. Partial Response: 1. platelets counts is greater than 40×10\^9/L. 2. significant volume reduction is greater than 50%. 3. Fibrinogen levels at less than 50% reduction from baseline. 4. The surface skin of the tumor and palpation of the tumor have no change or less change. No Response: 1. platelets counts is less than 40×10\^9/L. 2. significant volume reduction is less than 50% or the tumor is bigger. 3. Fibrinogen levels at grater then 50% reduction from baseline. 4. The surface skin of the tumor is darker or the tumor is harder.
次要结局
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0(1 year after taking the drug)