Efficacy and Safety of Combination of Hydroxyurea and Low-dose Thalidomide on Hemoglobin Synthesis in Thalassemia Patients
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 135
- 试验地点
- 1
- 主要终点
- Change in transfusion requirements
研究概览
简要总结
Objectives
Primary objective:
• To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients.
Secondary objective:
• To determine the change in liver and spleen size of beta-thalassemia patients on the combination therapy
A single-arm non-randomized trial to evaluate the efficacy and safety of combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients. It was a twelve months study. Participants were monitored for six months on Hydroxyurea alone and then the combination therapy of hydroxyurea and thalidomide for another six months. Findings of physical examination, vital signs, laboratory, and ultrasound findings were recorded at baseline, during and end of the study.
Sample Size and Population This study included 135 Beta-thalassemia patients.
详细描述
The purpose of the study was to evaluate the efficacy and safety of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients. The participants were evaluated on the basis of eligibility criteria, consent was obtained and baseline investigations were performed on the screening visit. Participants were continued on Hydroxyurea (10-20mg/kg/day) for the first six months and then for the next six months thalidomide (2-5 mg/kg/day) was added to the intervention. Aspirin was also added (2-4mg/kg/day).
Efficacy:
Maintenance or rise in Hemoglobin (Hb) levels and changes in transfusion frequency before and after the use of combination therapy was used to evaluate the efficacy of the combination therapy. Good responders were the patients who were on transfusion and went off-transfusion after the combination therapy or individuals who were already off transfusion and after combination therapy demonstrated an increase in Hb of at least 1gm/dl. Responders were those who remained on transfusion on combination therapy but displayed a 50% reduction in blood requirements. Whereas, non-responders, were individuals who were off-transfusion before combination therapy and had improvement in Hb of <1gm/dl, or those who remained on transfusion during combination therapy and did not experience transfusion reduction of at least 50%.
Safety:
Safety of the drug was evaluated on the basis of the following parameters and intervention was discontinued or put on hold if:
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
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with clinical and genetic diagnosis of β-thalassemia major and intermedia
- •Patients who showed partial response or a decline in response to hydroxyurea
- •Patients who are not the candidates for the bone marrow transplant procedure.
排除标准
- •Married Patients
- •Patients with comorbidities such as liver, cerebrovascular, cardiovascular, or kidney diseases
- •Patients allergic to the drug ingredients
- •Patients with mental disorders
- •Patients who are enrolled in other clinical trials
- •Patients with a history of venous or arterial thrombosis
研究组 & 干预措施
Combination of hydroxyurea and thalidomide
Hydroxyurea was continued at a dose of 10-20 mg/kg/day for 6 months and then thalidomide was added orally at a dose of 2-5mg/kg/day for 6 months.
干预措施: hydroxyurea and thalidomide (Drug)
结局指标
主要结局
Change in transfusion requirements
时间窗: 6 months on combination therapy
Change in transfusion needs (measured as blood volume in ml) per month from baseline
Change in Hemoglobin levels
时间窗: 6 months on combination therapy
Change in hemoglobin level of \>1g/dl from baseline
Change in the liver function test
时间窗: 6 months on combination therapy
Change in SGPT in U/l from baseline
Change in liver function test
时间窗: 6 months on combination therapy
Change in total, direct, and indirect bilirubin in mg/dl from baseline
Change in Kidney Function test
时间窗: 6 months on combination therapy
Change in urea and creatinine levels in mg/dl from baseline
次要结局
- Change in Spleen size(6 months on combination therapy)
- Change in liver size(6 months on combination therapy)