Up-front Hematopoietic Stem Cell Transplantation in Patients With Acute Myeloid Leukemia Aged 65-75
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 19
- 试验地点
- 1
- 主要终点
- Rate of complete remission equal or higher than 60%
研究概览
简要总结
Patients with acute myeloid leukemia aged 65-75 have a very poor prognosis, irrespective of the treatment strategy, including demethylating agents or conventional chemotherapy. With these approaches, remission rates do not exceed 40%, and overall disease-free survival at 1 year is in the order of 15%. The hypothesis is that up-front allogeneic hematopoietic stem cell transplant will produce a complete remission rate of 60% on day +56-70, and disease-free survival at 1 year of 30%. This is a single arm phase II study of upfront allogeneic stem cell transplantation, for patients with acute myeloid leukemia aged 65-75: the primary endpoint is a complete remission rate on day +56-70. The secondary endpoint is a 1-year overall disease-free survival of 30%.
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
- 65 Years 至 75 Years（Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged 65-75
- •Patients with de novo or secondary acute myeloid leukemia (AML) - intermediate or high risk according to European LeukemiaNet (ELN) recommendations 2017
- •Untreated patients at diagnosis of acute myeloid leukemia - patients may have received treatment for high-risk myelodysplastic syndromes with hypomethylating agents (HMA). They should not have received a course of induction chemotherapy to be eligible for this study
- •Haploidentical family stem cell donor or other suitable donors available
- •Fit and unfit patients by geriatric scale assessment
- •Signed informed consent.
排除标准
- •Acute Myeloid Leukemia good risk according to European LeukemiaNet 2017
- •Positive serology for Human Immunodeficiency Virus.
- •Serious organ dysfunction: left ventricular ejection fraction < 40%, forced expiratory volume in one second (FEV1), forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLCO) <50% of predicted, Liver Function Tests > 5 x the upper limit of normal, or creatinine clearance <30 ml/min .
- •Life expectancy less than 30 days.
- •Frail patients by geriatric scale assessment
研究组 & 干预措施
Allogeneic hematopoietic stem cell transplantation
Day -6, -5 Thiotepa 5 mg/kg/day . Day -4 to -3 Busulfan i. v 3,2 mg/kg/day and fludarabine i.v. 50 mg/m2 /day Day -2 fludarabine i.v. 50 mg/m2 Day -1 Rest Day 0 Begin cyclosporine; Infusion of T cell replete bone marrow transplant Day 1 Begin mycophenolate mofetil Day 3 and 5 Cyclophosphamide 50 mg/kg IV and Mesna Day 6 G-colony stimulating factor
干预措施: Up-front allogeneic hematopoietic stem cell transplantation (HSCT) (Drug)
结局指标
主要结局
Rate of complete remission equal or higher than 60%
时间窗: From day 56 to day +70 post-transplant
As a primary outcome measure, the rate of complete remission in treated patients will be evaluated. Complete remission will be assessed from days +56 to days +70 after transplant. A complete remission rate equal to or higher than 60% is expected.
次要结局
未报告次要终点
研究者
BACIGALUPO ANDREA
Professor
Fondazione Policlinico Universitario Agostino Gemelli IRCCS