Tacrolimus Treatment for Refractory Autoimmune Cytopenia
试验速览
- 阶段
- 4 期
- 入组人数
- 80
- 试验地点
- 1
- 主要终点
- Hemoglobin level
研究概览
简要总结
Autoimmune cytopenia, including autoimmune hemolytic anemia (AIHA), pure red cell aplasia (PRCA), Evans syndrome (ES), usually has good responses to steroids therapies as first line, but there is a considerable percentage of patients who relapse, become refractory or dependent on steroids to maintain an acceptable level of hemoglobin or platelets. The effects of the second line therapy are also not satisfactory and sometimes not available. The investigators aim to explore the efficacy and side-effect of tacrolimus for refractory autoimmune cytopenia.
详细描述
Tacrolimus binds FKBP12 with high affinity after entry into cytoplasm, suppresses calcineurin activity and prevents nuclear translocation of transcription factors such as NF-AT that are involved in IL-2 gene transcription. As a result, T-cell activation is inhibited with a subsequent reduction in the production of cytokines that include IL-2, TNF-α, IL-3, IL-4, IFN-γ, IL-6 and IL-10; B-cell activation, class-switching and immunoglobulin production are also attenuated. Reports on the use of tarcrolimus in the treatment of autoimmune cytopenia are anecdotal and describe therapy with a variety of drug dosages.
The purpose of this study is to evaluate the effect of tacrolimus on patients with refractory autoimmune cytopenia, the side-effects will be documented and plasma concentration of tacrolimus will be monitor.
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
- •Refractory autoimmune cytopenia, including autoimmune hemolytic anemia, pure red cell aplasia, Evans syndrome.
- •18-80 years old.
- •No response or intolerant to first and second line therapies.
- •ECOG Performance Status of 0-2
- •Written informed consent.
排除标准
- •Other diseases which might cause hematological abnormalities.
- •Response and well tolerate to first or second line therapy.
- •Patients who are under 18-year-old or over 80-year-old.
- •Pregnant or lactating.
- •Patients unwilling to or unable to comply with the protocol.
研究组 & 干预措施
Efficiency of tacrolimus on autoimmune cytopenia
A prospective research of the tacrolimus efficiency on refractory autoimmune cytopenia patients. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study.
Medication time should last at least 6 months.
干预措施: Tacrolimus (Drug)
结局指标
主要结局
Hemoglobin level
时间窗: 6 months
Hemoglobin level in g/L
Platelet count
时间窗: 6 months
Platelet count in /L
次要结局
- Hemoglobin level(2 years)
- Platelet count(2 years)