The Study of Rituximab in the Treatment of Severe Aplastic Anemia With Platelet Transfusion Refractoriness
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- The response and complete remission rate with Rituximab protocol.
研究概览
简要总结
Due to long-term dependence on platelet transfusion, some severe aplastic anemia (SAA) patients suffer platelet transfusion refractoriness (PTR). Unlike immune thrombocytopenia (ITP), glucocorticoids and human immunoglobulin (IVIg) are generally ineffective for PTR. Due to the lack of effective intervention methods, patients with PTR suffer increased platelet transfusions, bleeding events and treatment costs, prolonged hospital stays, and decreased survival rate. SAA with PTR has become a challenge for physicians. The experiment aims to explore the efficacy of rituximab in the treatment of SAA with PTR, and establish a new effective, safe treatment method with relatively low treatment cost.
详细描述
During the treatment period, Rituximab is administered at a dose of 100mg per week, a total of 4 times.
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Initial diagnosed SAA with PTR
- •Age>18 years old, regardless of gender
- •Initial diagnosed SAA with PTR
- •Age>18 years old, regardless of gender
排除标准
- •Allergy to rituximab
- •Severe active infection
- •Hypogammaglobulinemia
- •Pregnant and lactating women
- •Heart failure (NYHA classification IV)
- •Individuals with epilepsy, dementia, and other mental disorders that require medication treatment who cannot understand or follow the research protocol
- •Chronic infections or other chronic diseases that may be risk to the experiment
- •The researchers believe that it is not suitable for participants
研究组 & 干预措施
rituximab group
Rituximab combined with cyclosporine
干预措施: Rituximab (Drug)
结局指标
主要结局
The response and complete remission rate with Rituximab protocol.
时间窗: 6 months
Response will be evaluated at each clinic visit. Complete response (CR) was defined as achieving all three peripheral blood count criteria: (1) Hb level up to the normal range; (2) ANC≥1.5×109/L; (3) PLT≥100×109/L. Partial response (PR) was defined as transfusion independent, no longer meeting criteria for severe disease. Persistence of transfusion requirement or death was evidence of no response (NR).
次要结局
- Relapse rate(12 months and 60 months)
- Sustained response (SR)(12 months and 60 months)
- Survival(60 months)
- Clonal evolution to myelodysplasia and acute leukemia.(60 months)