A Single-center Prospective Clinical Trial of the Efficacy and Safety of Ultra Small Dose Decitabine for the Lower Risk Myelodysplastic Syndrome Patients With Transfusion Dependent
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 50
- 试验地点
- 2
- 主要终点
- complete response
研究概览
简要总结
Myelodysplastic syndrome (MDS) is widely recognized as a clonal hematopoietic stem cell disorder. Decitabine has been approved for the treatment of all subtypes of myelodysplastic syndrome (MDS). However, the use of decitabine is often limited by its severe toxicity represented by myelosuppression even at relatively low doses. In lower-risk patients (including IPSS low and int-1 risk groups), treatment mainly aims at improving cytopenias, especially anemia. However, although several drugs may improve anemia, sometimes durably, most of lower risk MDS eventually require red blood cell (RBC) transfusions during their disease course. Long term RBC transfusions lead to iron overload mainly due to an increase in reticulo-endothelial iron recycling.Cardiac, liver and endocrine (diabetes mellitus) dysfunction due to iron overload and often leading to fatal outcome has been reported in heavily transfused lower risk MDS patients.
To date, the optimal regimen for decitabine treatment is not well established. In this study, we perform a prospective analysis to explore the decitabine schedule for the treatment of lower risk myelodysplastic syndrome patients with transfusion dependent.
详细描述
The investigators are undertaking a single-center, single-arm study of 50 lower risk myelodysplastic syndrome patients with transfusion dependent from Shandong University Qilu Hospital . All the participants are selected to receive ultra small dose decitabine treatment (given intravenously at a dose of 3.5mg/m2, qd x 5d, every four weeks for one cycle). A routine blood examination is performed twice every week. Bone marrow (BM) is examined with routine aspirate smear and G-banding analysis every 1-2 treatment courses to evaluate responses.Adverse events are also recorded throughout the study.
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
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diagnosis of MDS
- •The IPSS [17] score ≤ 1
- •patients with transfusion dependent
- •Adequate hepatic and renal function (aspartate aminotransferase [AST] ≤ 2.5 x upper normal limit, alanine aminotransferase [ALT] ≤ 2.5 x upper normal limit, bilirubin ≤ 1.5 x upper normal limit and creatinine < 2 x upper normal limit, Ccr > 60ml/min ).
排除标准
- •Decitabine and Arsenic trioxide allergy
- •Pregnancy and lactation
- •Cardiovascular disease
- •ECOG score > 2
- •HCV, HIV, HBsAg seropositive status
研究组 & 干预措施
Ultra small dose decitabine
Decitabine 3.5mg/m2，ivdrip，qd x 5d, every four weeks for one cycle. It will be given six cycles.
干预措施: decitabine (Drug)
结局指标
主要结局
complete response
时间窗: 30 days from the emrollment
Bone marrow blasts not more than 5%, absolute neutrophil count more than 1\*10\^9/L, HgB more than 100g/L, and platelet count more than 100\*10\^9/L.
次要结局
未报告次要终点
研究者
Ming Hou
Professor and Director
Shandong University