Comparison Between 5-azacytidine Treatment and 5-azacytidine Followed by Allogeneic Stem Cell Transplantation in Elderly Patients With Advanced MDS According to Donor Availability
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 191
- 试验地点
- 17
- 主要终点
- overall survival
研究概览
简要总结
5-azacytidine treatment prolongs survival in patients with myelodysplastic syndrome (MDS), but does not cure the disease. Allogeneic stem cell transplantation is a curative treatment option but is associated with a high risk treatment-related morbidity and mortality. In the current trial allogeneic stem cell transplantation will be compared to 5-azacytidine only treatment according to donor availability in elderly patients with MDS (55-70 years).
详细描述
5-azacytidine treatment prolongs survival in patients with myelodysplastic syndrome (MDS), but does not cure the disease. Allogeneic stem cell transplantation is a curative treatment option but is associated with a high risk treatment-related morbidity and mortality. Dose-reduced conditioning prior transplantation allows also treatment of elderly patients with MDS. In the current trial allogeneic stem cell transplantation will be compared to 5-azacytidine only treatment according to donor availability in elderly patients with MDS (55-70 years).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Factorial
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 55 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with proven de novo or therapy-related MDS / CMML (WBC <13 GPT/l)according to FAB and risk profile according to IPSS: intermediate II- risk or high-risk or intermediate I with high-risk cytogenetic (according to IPSS, taking into account that IPSS, however, was not validated for t- MDS), patients with secondary AML (according to WHO) and blasts ≤ 30 % (= RAEB-t according to FAB)
- •Previously untreated or maximal 1 cycle of 5-azacytidine (Vidaza®)
- •Male or Female; Age 55 - 70 years
- •Understand and voluntarily sign an informed consent form
- •ECOG performance status of ≤ 2 at study entry
- •Adequate renal and liver function: creatinine and bilirubin < 3 x the upper limit of normal
- •Sufficient cardiac function (ejection fraction > 30 %)
排除标准
- •Blasts > 30 % in bone marrow at time of diagnosis
- •Central nervous involvement
- •Severe irreversible renal, hepatic, pulmonary or cardiac disease, such as
- •Total bilirubin, SGPT or SGOT ≥ 3 times upper the normal level
- •Left ventricular ejection fraction < 30 %
- •Creatinine clearance < 30 ml/min
- •DLCO < 35 % and/or receiving supplementary continuous oxygen
- •Pregnant or breastfeeding female subject
- •Patients with a life-expectancy of less than six months because of another debilitating disease
- •Serious psychiatric or psychological disorders
- •Uncontrolled invasive fungal infection at time of registration
- •Known positive for HIV or acute infectious hepatitis, type A, B or C
- •Participation in another study with ongoing use of unlicensed investigational product from 28 days before study enrollment until the end of the study
研究组 & 干预措施
5-azacytidine treatment until progress
5-azacytidine until progress
干预措施: 5-azacytidine until progress (Procedure)
allogeneic stem cell transplantation
after 4 cycles 5-azacytidine and if donor available: allogeneic stem cell transplantation after reduced intensity conditioning
干预措施: allogeneic stem cell transplantation (Procedure)
结局指标
主要结局
overall survival
时间窗: three years
compare to overall survival of patients who receive after 4 cycles of 5-azacytidine either allogeneic stem cell transplantation or continuous 5-azacytidine if no compatible donor is available overall 230 patients
次要结局
- response(three years)
- event-free survival(three years)
- overall survival(three years)
- Treatment-related mortality(three years)
- impact of Comorbidity-index on outcome(three years)
- Evaluation of toxicity(three years)
- quality of life(three years)