Safety and Efficacy of Immunomodulator Drug Thalidomide in Multi-transfused Thalassemia Patients: A Single Center Experience From South Punjab, Pakistan
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 121
- 试验地点
- 1
- 主要终点
- Efficacy of treatment
研究概览
简要总结
This study aimed to determine the safety and efficacy of thalidomide in multi-transfused thalassemic children.
详细描述
Given the significant burden of transfusion dependency, an effective and safe inducer is needed that could improve quality of life and reduce the healthcare costs associated with frequent transfusions and iron chelation therapy. If this study could establish the safety of thalidomide in terms of teratogenicity along with its efficacy, it would be a great support to multi-transfused thalassemic children.
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 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children of both genders
- •Aged 2-5 years
- •With transfusion-dependent thalassemia (TDT)
- •Whose parents/guardians showed willingness to adhere to follow-up visits
排除标准
- •Children with uncontrolled infections
- •Significant hepatic or renal dysfunction
- •Malignancy
- •Known contraindications to thalidomide
研究组 & 干预措施
Thalidomide group
Thalidomide was administered at a dose of 1.5 mg/kg/day, and all patients continued standard iron chelation therapy and other supportive treatments as per institutional protocols.
干预措施: Thalidomide (Drug)
结局指标
主要结局
Efficacy of treatment
时间窗: 6 months
Efficacy was categorized as excellent response showing an increase in hemoglobin by 1-2 g/dL or freedom from blood transfusions, partial response as an increase in hemoglobin \<1 g/dL or a decrease in transfusion frequency, or no response if there was no increase in hemoglobin or an increase in transfusion requirement within three months.
Safety of treatment
时间窗: 6 months
Treatment was considered safe if no adverse events were documented based on clinical assessment and laboratory findings.
次要结局
未报告次要终点
研究者
Muhammad Aamir Latif
Research Consultant
RESnTEC, Institute of Research