Phase 2 Study of Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
试验速览
- 阶段
- 2 期
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Change in baseline transfusion frequency with increase of pre-transfusion hemoglobin
研究概览
简要总结
The study hypothesis that treatment with Erythropoietin (EPO) combined with Human Erythropoietin (HUO) therapy will result in hematologic improvement in thalassemia intermedia patients.
Second is to determine whether any of the following correlate with improved hematologic response:
A decrease in hemolysis, as assayed by a decrease in LDH, compared to baseline levels,baseline Erythropoietin levels,baseline hemoglobin levels and baseline reticulocyte counts (or % circulating nucleated erythroblasts/100 WBCs).
Goal:
The aim is to assess the possibility of steady increase of hemoglobin levels in thalassemia intermedia patients by at least 1g/dl above baseline levels during therapy using Hydroxyurea and Erythropoietin, growth evaluation,quality of life (QoL) and decline transfusion requirements during study period. Also to report and compare adverse events with other published data regarding.
详细描述
To determine whether any of the following correlate with improved hematologic response:
A decrease in hemolysis, as assayed by a decrease in LDH, compared to baseline levels,baseline Erythropoietin levels,baseline hemoglobin level and baseline reticulocyte counts (or % circulating nucleated erythroblasts/100 WBCs).
To assess the possibility of steady increase of hemoglobin levels in thalassemia intermedia patients by at least 1g/dl above baseline levels during therapy using Hydroxyurea and Erythropoietin, growth evaluation , quality of life ( QoL ) and decline transfusion requirements during study period. Also to report and compare adverse events with other published data regarding.
THE following criteria are used when including the patient in the study:
Patients with thalassemia intermedia.Diagnosis based on genetic mutations, hemoglobin electrophoresis and characteristic clinical data at presentation. Patients requiring different transfusion requirements and not transfusion dependent.Patients having a baseline hemoglobin of less than or equal to 6-8g/dl.Patients with normal renal and liver function.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with thalassemia intermedia. Diagnosis based on genetic mutations, hemoglobin electrophoresis and characteristic clinical data at presentation.
- •Require different transfusion requirements and not transfusion dependent.
- •Have a baseline hemoglobin of less than or equal to 6-8g/dl.
- •Patients with normal renal and liver function.
排除标准
- •Evidence of active hepatitis (ALT > 5 times above ULN).
- •Evidence of renal impairment (serum creatinine > ULN).
- •Patients who are dependent on red blood cell transfusions.
研究组 & 干预措施
Hydroxyurea,blood transfusion
Hydroxyurea (Myers-Squibb, USA) was administered in dosages ranging from 15 up to 35 mg/kg/day orally over 7 days/week.
干预措施: hydroxyurea, blood transfusion (Drug)
Hydroxyurea, Epiao
- Hydroxyurea (Myers-Squibb, USA) was administered in dosages ranging from 15 up to 35 mg/kg/day orally over 7 days/week. Hydroxyurea toxicity was defined as a white cell count of less than 2500/μL or a platelet count of less than 100,000/μL, in which case the drug was discontinued. White cell count and platelet count were determined on a monthly basis. Side effects such as nausea, vomiting, diarrhea, rashes, and malaise, experienced during the first 6 h after taking the HU will be considered as clinical toxicity.
- Erythropiotien therapy (rHuEPO - Epiao) from 250 to 500 IU/kg rHuEPO subcutaneously three times a week.
干预措施: Hydroxyurea ,Epiao (Drug)
结局指标
主要结局
Change in baseline transfusion frequency with increase of pre-transfusion hemoglobin
时间窗: baseline and 6 month hemoglobin level and transfusion frequency
Decrease in baseline transfusion frequency with increase of pre-transfusion hemoglobin by calculation of transfusion index and mean hemoglobin level
次要结局
- Change in baseline quality of life assessment.(baseline and 6 month QOL questionaire)
研究者
Mohsen Saleh Elalfy
prof. Mohsen el alfy
Ain Shams University