Long-Term Assessment of Thalidomide and Hydroxyurea Combination Therapy in β-Thalassemia Patients
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 603
- 试验地点
- 1
- 主要终点
- Change in laboratory levels
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. Participants were monitored for six months on Hydroxyurea alone and then the combination therapy of hydroxyurea and thalidomide was started. Findings of physical examination, vital signs, laboratory, and ultrasound findings were recorded at baseline, during, and end of the study.
The assessment of treatment outcomes was conducted at the 1-year, 2-year, and 3-year follow-up points during the combination therapy period, categorizing patients as either "good responders," "responders," or "non-responders."
详细描述
This study is conducted to evaluate the long-term efficacy and safety of the combination therapy of hydroxyurea and thalidomide in beta-thalassemia patients.
Monotherapy of Hydroxyurea was sustained at a daily dosage ranging from 10-20 mg/kg for a duration of 6 months, during which the treatment response was documented. After this initial 6-month period, thalidomide was introduced into the treatment regimen. Thalidomide was administered orally at bedtime, with an initial dosage of 2-5 mg/kg. An incremental dosing approach was employed for thalidomide, with individuals who exhibited an insufficient response to lower doses having their dosage increased to a maximum of 5 mg/kg. Additionally, aspirin was prescribed at a daily dosage of 2-4 mg/kg to mitigate the risk of thrombosis.
Blood transfusions were administered under specific conditions throughout the study. Transfusions were initiated if the Hb levels dropped below 7 g/dL or if patients exhibited symptoms or instability, regardless of their Hb levels. Patients who were undergoing iron chelation therapy (utilizing deferasirox, deferiprone, and/or deferoxamine) while on HU monotherapy continued this regimen throughout the combination therapy phase.
Throughout combination therapy, various assessments were conducted and documented, including blood transfusion events and comprehensive blood count evaluations, carried out at baseline, as well as during the 1-year, 2-year, and 3-year follow-up periods. Concurrently, safety parameters, such as urea and creatinine levels, liver function tests, and measurements of liver and spleen size, were consistently monitored and recorded.
Outcome:
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 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with clinical and genetic diagnoses of β-thalassemia major and intermedia
- •Patients who showed partial response or a decline in response to hydroxyurea
- •Patients who are not 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.
干预措施: Hydroxyurea and Thalidomide (Drug)
结局指标
主要结局
Change in laboratory levels
时间窗: 1-3 years on combination therapy
Mean changes in hemoglobin, platelets, leukocytes, urea, creatinine, and ferritin level from baseline
Change in the liver and spleen size
时间窗: 1-3 years on combination therapy
Mean changes in the liver and spleen size from baseline
Response at different time intervals
时间窗: 1-3 years on combination therapy
Frequency of good responder, responder, and non-responder.
次要结局
- XmnI polymorphism(1-3 years on combination therapy)