Pharmacokinetic Study of Lipo-PGE1 for Prevention of VOD After HSCT
试验速览
- 阶段
- 2 期
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- pharmacokinetics of lipo-PGE1 (Lipo-PGE1 level (B, P1, P2, O1, O2))
研究概览
简要总结
Veno-occlusive disease (VOD) after hematopoietic stem cell transplantation (HSCT) remains the major complication. VOD occurs in 11-31% of pediatric HSCT and the mortality reaches up to 50%.
Prostaglandin E1 (PGE1) have been reported to prevent and relieve the severity of VOD by Gluckman et al.. Lipo-PGE1 is a transporter of PGE1, which is superior in concentrating PGE1 and acts for prolonged time. Because of the prolonged effective time, lipo-PGE1 acts comparable effects by 1/4~1/8 of PGE1 dose. Empirically, pediatric HSCT centers adopt lipo-PGE1 in dose of 1 mcg/kg/day (0.042 mcg/kg/hr), which is 1/7 of the dose recommended by Gluckman, et al. This prospective study will investigate the concentration of lipo-PGE1 with preventive lipo-PGE1 with empirical dose (1 mcg/kg/day).
详细描述
Primary Objective: To evaluate the pharmacokinetics of lipo-PGE1. Secondary objective: To evaluate the relation between lipo-PGE1 concentration and VOD occurrence, severity.
Inclusion criteria
- Patients undergoing HSCT with high risk of VOD who cannot use low dose heparin.
- high risk of VOD : previous radiotherapy, liver function abnormality, children, conditioning regimen including busulfan or total body irradiation
- contraindication of heparin : low platelet count, bleeding tendency, allergy
- Patients (or one of parents if patients age < 19) should sign informed consent.
Exclusion criteria
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients undergoing HSCT with high risk of VOD who cannot use low dose heparin.
- •high risk of VOD : previous radiotherapy, liver function abnormality, children, conditioning regimen including busulfan or total body irradiation
- •contraindication of heparin : low platelet count, bleeding tendency, allergy
- •Patients (or one of parents if patients age < 19) should sign informed consent.
排除标准
- •Patient with heart failure.
- •Patient with bleeding (intracranial hemorrhage, gastrointestinal tract bleeding, hemoptysis).
- •History of hypersensitivity reaction as shock to lipo-PGE
- •Psychiatric disorder that would preclude compliance.
- •If the clinician decides that there is a condition improper for the clinical study.
研究组 & 干预措施
lipoprostaglandin E1 treatment arm
- lipoprostaglandin E1 1mcg/kg/day, continuous infusion
- lipoprostaglandin E1 1.5mcg/kg/day, continuous infusion (for patients with elevating total bilirubin, hepatomegaly, right upper quadrant abdominal pain, unexplained weight gain)
干预措施: lipoprostaglandin E1 (Drug)
结局指标
主要结局
pharmacokinetics of lipo-PGE1 (Lipo-PGE1 level (B, P1, P2, O1, O2))
时间窗: baseline (pre-dose), 1 to 72 hours from starting continuous infusion of lipo-PGE1
Lipo-PGE1 drug level of time points (B, P1, P2, O1, O2)
次要结局
- Incidence of VOD after transplantation(until 3 months after transplantation)