A Randomized Trial Testing Early vs Late Onset of EPO Alfa Treatment in Lower Risk MDS With Non RBC Transfusion Dependent Anemia and Without Del 5q
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 124
- 试验地点
- 76
- 主要终点
- Time to RBC transfusion dependence in non RBC transfusion dependent lower risk MDS patients with anemia with early (at inclusion of the patient) versus delayed onset,( at the threshold chosen for RBC transfusion) of EPO ALFA
研究概览
简要总结
This is an open-label, randomized, multicenter, phase III study
Patients with baseline Hb comprised between 9 and 10.5g/dl will be randomized to receive EPO Alfa 60000 UI/week for at least 12 weeks:
- Either at diagnosis Or
- at the Hb threshold chosen for RBC transfusions (must be < 9g/dl)
详细描述
in this trial we will compare the early introduction of EPO alfa to the delayed introduction in lower risk MDS with non RBC transfusion dependent anemia.
At enrollment patients will be randomised in the 2 arms (early and delayed start of EPO alfa).
Treatment Regimen Epoetin alfa 60000 UI/week for at least 12 weeks
- Early onset arm: early onset of EPO ALFA 60000 IU/week , at patient inclusion
- Delayed onset arm: late introduction of EPO ALFA 60000 IU/week, whenever the patient reaches the level chosen RBC transfusions (based on age, comorbidities, anticipated tolerance of anemia).
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 18 years
- •MDS according to WHO 2016 criteria, with low or int 1 classical IPSS
- •Non-RBC transfusion dependent anemia
- •Hb level between 9 and 10.5g/dl (at the center's lab)
- •Hb level should be at least 1g/dl higher than the Hb threshold chosen to start RBC transfusions based on age, comorbidities and predicted clinical tolerance of anemia (this transfusion threshold should be chosen between 8 and 9g/dl)
- •Serum EPO level <500U/l
- •No other cause of anemia (including iron deficiency, vitamin B12 or B9 deficiency, hemolysis, hypothyroidism….)
- •Performance status <=2
排除标准
- •Higher risk MDS (IPSS intermediate-2 or high)
- •Baseline Hemoglobin level > 10.5 g/dl or <9g/dl
- •Transfusion threshold (based on age , comorbidities…) >9g/dl
- •Transfusion threshold less than 1 g/dl below baseline Hb level
- •RBC transfusion dependence. Patients may have received only one transfusion series for MDS prior to inclusion
- •CMML , if >10 % BM blasts or WBC>13.000/mm3
- •Uncontrolled hypertension
- •Uncontrolled cardiovascular disease including angina pectoris or cardiac failure
- •Renal failure: Creatinine clearance<40ml/min (using MDRD formula)
- •Pregnancy (positive bettaHCG) or nursing
研究组 & 干预措施
Early onset arm
Intervention: early onset of Eprex60000 IU/week , at patient inclusion
干预措施: EPREX (Drug)
Delayed onset arm
Intervention: late introduction of Eprex60000 IU/week, whenever the patient reaches the level chosen RBC transfusions (based on age, comorbidities, anticipated tolerance of anemia).
干预措施: EPREX (Drug)
结局指标
主要结局
Time to RBC transfusion dependence in non RBC transfusion dependent lower risk MDS patients with anemia with early (at inclusion of the patient) versus delayed onset,( at the threshold chosen for RBC transfusion) of EPO ALFA
时间窗: 12 weeks
RBC transfusion dependence will be defined by requirement of at least transfusions of 2 PRBC within an interval of less than 8 weeks, given for Hb \<8g/dl or \<9g/dl according to comorbidities and in the absence of other cause of anemia (bleeding, surgery…), taking into account only transfusions given at least 12 weeks after onset of treatment with EPO ALFA.
次要结局
- Erythroid response (according to IWG 2006 criteria)(12 weeks)
- response duration to EPO ALFA(4 years)
- Overall survival(4 years)