A Randomized Controlled Trial Comparing Three Sirolimus Discontinuation Strategies in Patients With Kaposiform Hemangioendothelioma
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 120
- 试验地点
- 1
- 主要终点
- Rebound growth rate of KHE
研究概览
简要总结
Sirolimus has demonstrated efficacy in the treatment of kaposiform hemangioendothelioma (KHE); however, a high rate of rebound growth following discontinuation has been reported, highlighting the clinical importance of establishing an appropriate withdrawal regimen.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •KHE patients who have received sirolimus treatment and met the criteria for drug discontinuation.
- •With evaluable clinical or imaging parameters to monitor disease activity or recurrence.
- •Patients or their guardians are able to understand the study and provide written informed consent.
- •Hematologic and hepatic/renal function meet the safety criteria for drug administration.
排除标准
- •Patients who are unable to comply with the follow-up or treatment schedule, potentially affecting the integrity of study data.
研究组 & 干预措施
Low-dose treatment group
Low-dose sirolimus treatment was administered for 6 months (with blood levels maintained at 2-5 ng/mL), followed by gradual tapering and discontinuation over 2 months.
干预措施: Sirolimus (Drug)
Intermittent treatment group
Sirolimus was administered using a 3-days-on/4-days-off weekly schedule for 6 months, followed by gradual tapering and discontinuation over 2 months.
干预措施: Sirolimus (Drug)
Direct discontinuation group
Sirolimus was gradually tapered and discontinued over a 2-month period.
干预措施: Sirolimus (Drug)
结局指标
主要结局
Rebound growth rate of KHE
时间窗: 12 months
The primary endpoint is the rebound growth rate of KHE during the follow-up period.
次要结局
- Incidence of disease sequelae(12 months)
- Incidence of adverse events throughout the study.(12 months)