A Clinical Study on the Treatment of Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP) by Hormone Shock and Sirolimus Maintenance
试验速览
- 阶段
- 1 期
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- response to treatment
研究概览
简要总结
a phase I trial focusing on safety and efficacy of prednison shock plus sirolimus maintenance in treating Kaposiform hemangioendothelioma (KHE) with Kasabach-Merritt phenomenon (KMP)
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 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
prednison group
干预措施: prednison (Drug)
prednison+sirolimus group
干预措施: prednison and Sirolimus (Drug)
结局指标
主要结局
response to treatment
时间窗: 6 months after taking the drug
Complete Response： platelets counts is greater than 100×10\^9/L. significant volume reduction is greater than 80%. Fibrinogen levels at 2-4g/L. The surface skin of the tumor is lighter or the tumor is softer significantly. Partial Response: platelets counts is greater than 40×10\^9/L. significant volume reduction is greater than 50%. Fibrinogen levels at less than 50% reduction from baseline. The surface skin of the tumor and palpation of the tumor have no change or less change. No Response: platelets counts is less than 40×10\^9/L. significant volume reduction is less than 50% or the tumor is bigger. Fibrinogen levels at grater then 50% reduction from baseline. The surface skin of the tumor is darker or the tumor is harder.
次要结局
- side effect rate(6 months after taking the drug)