Pharmacogenetic Study in Patients Received Iron Chelating Agent
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- Genetic polymorphism associated with side effects of deferasirox
研究概览
简要总结
To investigate effect of genetic variations on the toxicities and find optimal target population, the investigators planned to analyze the genetic polymorphisms of UDP-glucuronosyltransferase.
详细描述
Transfusion-associated iron overload induces systemic toxicity. Recently, deferasirox, a convenient long acting oral agent, has been introduced in clinical practice with promising efficacy. However, some patients experience drug-related toxicities and cannot tolerate it. To investigate effect of genetic variations on the toxicities and find optimal target population, we planned to analyze the genetic polymorphisms of UDP-glucuronosyltransferase 1A (UGT1A) subfamily, multi-drug resistance-associated protein 2 (MRP2) and breast cancer resistance protein (BCRP) among pediatric patients received deferasirox.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients who received deferasirox because of transfusion associated iron overload (Transfusion associated iron overload was defined as ferritin ≥ 1,000 ng/mL in patients who needed over 8 units of RBC transfusions per a year).
- •Patients with written informed consents
排除标准
- •Patients or parents refusal
结局指标
主要结局
Genetic polymorphism associated with side effects of deferasirox
时间窗: up to 1 year
Genetic polymorphism associated with side effects of deferasirox - Side effects: Increased AST or ALT \> 5 x ULN or increased bilirubin \> 3 x ULN which was thought to be caused by deferasirox Serum creatinine level increase \> 50% above the baseline value. * Biospecimen Retention: Samples With DNA * Candidate genes exhibit polymorphisms and encodes proteins that are involved in the pharmacokinetics and pharmacodynamics of deferasirox. Candidate genes : MRP2, BCRP, UGT1A subfamily
次要结局
未报告次要终点
研究者
Hyoung Jin Kang
MD. Ph D., Professor
Seoul National University Hospital