Efficacy and Safety of Low Dose Thalidomide in Transfusion Dependent Thalassemia Patients of Pakistan
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 654
- 试验地点
- 1
- 主要终点
- Adverse Effects
研究概览
简要总结
Thalidomide is known to have hypnosedative, immuno-modulatory and anti-angiogenic effects. The drug is widely used in several neoplastic disorders (e.g. multiple myeloma and malignant melanoma), inflammatory conditions (e.g. Crohn's disease) and skin disorders (e.g. leprosy). Thalidomide has been successfully used in adult thalassemia patients. The current study explores its role in transfusion-dependent thalassemia patients.
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
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Transfusion dependent thalassemia patients refractory to Hydroxyurea
排除标准
- •Those with active systemic comorbidity, with past personal or family history of thrombophilia, recent fracture or recent major surgery
研究组 & 干预措施
Thalidomide in combination with hydroxyurea
All the study patients refractory to hydroxyurea will be given thalidomide at an average dose of 2mg/kg/day (range 1-4mg/kg/day). Participants will also be continued on hydroxyurea at a dose of 10-20 mg/kg/day.
干预措施: Thalidomide and Hydroxyurea (Drug)
结局指标
主要结局
Adverse Effects
时间窗: 6-24 months
Haemoglobin Level
时间窗: 6-24 months
mg/dL
Response Rate (Excellent, Good, Partial and No response)
时间窗: 6-24 months
次要结局
未报告次要终点
研究者
Muhammad Tariq Masood Khan
Consultant Haematologist; Research Associate
Blood Diseases Clinic