The Efficacy and Safety of Reduced-Intensity Conditioning With Fludarabine- Melphalan-Busulfan for Allogeneic Hematopoietic Stem Cell Transplantation in the Treatment of Myelodysplastic Syndromes
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 45
- 试验地点
- 1
- 主要终点
- 2-year relapse-free survival after transplantation
研究概览
简要总结
Allogeneic hematopoietic cell transplantation (allo-HCT) is the only potential cure for Myelodysplastic syndrome (MDS). Currently, the conditioning regimen for MDS allogeneic transplantation still follows AML, and there are fewer explorations in this field. In a large-scale retrospective analysis with 532 MDS undergoing allo-HCT, reduced intensity conditioning is resulted in improved overall survival (OS), reduced non-relapse mortality (NRM) , while sparing relapse.
Therefore, the investigators conduct a prospective, single-arm, multicentre study to evaluate the efficacy and safety of Fludarabine-Melphalan-Busulfan reduced-intensity conditioning in MDS patients.
详细描述
This study is a prospective, multicenterstudy designed to evaluate the efficacy and safety of evaluate the efficacy and safety of Fludarabine-Melphalan-Busulfan intermediate-intensity transplantation conditioning regimen for the treatment of MDS. . Patients with MDS who are eligible for enrolment were pretreated with a Fludarabine-Melphalan-Busulfan regimen (Fludarabine 30mg/m2d-6 to -2; melphalan 70mg/m2d-6; busulfan 3.2mg/kg d-4 to -3). The primary endpoint is 2-year relapse-free survival (RFS) after transplantation. Secondary endpoints includes 2-year post-transplant survival (OS) and cumulative recurrence rate at 2 years post-transplant.
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
- 14 Years 至 70 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 14-70 (including boundaries) and gender;
- •Diagnosis of MDS is confirmed on the basis of bone marrow cytomorphology, immunophenotyping and chromosomal and molecular biology tests;
- •IPSS-R intermediate-risk or higher-risk or IPSS-M intermediate-risk or higher-risk MDS; transfusion-dependent MDS;
- •ECOG score≤ 3 points;
- •Have appropriate organ function, and laboratory results within 7 days prior to the start of trial treatment need to meet the following criteria:
- •Aspartate aminotransferase (AST) ≤ 3 times ULN (upper limit of normal, ULN);
- •Alanine aminotransferase (ALT) ≤ 3x ULN;
- •Total serum bilirubin ≤ 1.5 times the upper limit of normal ULN unless the patient has documented Gilbert syndrome; patients with Gilbert-Meulengracht syndrome with bilirubin ≤ 3.0 times the upper limit of normal and direct bilirubin ≤ 1.5 times the upper limit of normal may be included;
- •Serum creatinine ≤ 1.5 times ULN or creatinine clearance ≥ 60 mL/min;
- •Coagulation function: International Normalised Ratio (INR) ≤ 1.5 x ULN, Activated Partial Thromboplastin Time (APTT) ≤ 1.5 x ULN;
- •Left ventricular ejection fraction (LVEF) ≥50%;
- •Voluntarily signing the informed consent, understanding and complying with the requirements of the study, good compliance, and cooperating with the follow-up visits.
排除标准
- •Allergies or contraindications to any of the drugs in the protocol;
- •Currently have clinically significant active cardiovascular disease such as uncontrolled arrhythmias, uncontrolled hypertension, congestive heart failure, any grade 3 or 4 heart disease as determined by the New York Heart Association (NYHA) functional class, or a history of myocardial infarction within the 6 months prior to screening;
- •Serious medical conditions that may limit the patient's participation in this trial (e.g., progressive infection, uncontrolled diabetes);
- •Active autoimmune diseases such as SLE, rheumatoid arthritis, etc;
- •Patients with neurological or psychiatric disorders;
- •The patient is pregnant or breastfeeding;
- •Those who are unable to understand or comply with the study protocol or are unable to sign the informed consent form.
- •Other conditions that, in the opinion of the investigator, make the patient otherwise unsuitable for participation in this study;
研究组 & 干预措施
FBM arm
Adult MDS patients eligible for allo-HCT will receive Fludarabine 30mg/m^2 d-6 to -2; melphalan 70mg/m^2 d-6; busulfan 3.2mg/kg d-4 to -3 as conditioning regimen.
干预措施: Fludarabine; melphalan; busulfan (Drug)
结局指标
主要结局
2-year relapse-free survival after transplantation
时间窗: through study completion, an average of 2 year
relapse
次要结局
- 2-year post-transplant survival(through study completion, an average of 2 year)
- Cumulative recurrence rate at 2 years post-transplant(through study completion, an average of 2 year)
研究者
Hu Xiaoxia
Principal Investigator
Ruijin Hospital