The Combination of Tacrolimus and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia: A Randomized, Controlled, Open-label Trial
试验速览
- 阶段
- 2 期
- 入组人数
- 120
- 试验地点
- 1
- 主要终点
- Sustained response
研究概览
简要总结
Randomized, open-label, multicenter study to compare the efficacy and safety of combination of tacrolimus and danazol versus danazol for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
详细描述
The investigators are undertaking a randomized controlled trial of 120 adults with steroid-resistant/relapse ITP in China. Patients were randomized to tacrolimus plus danazol and danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 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
- •Primary immune thrombocytopenia (ITP);
- •18 years older;
- •Platelet count of less than 30×10^9/L at enrollment;
- •Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation;
- •Patients who were receiving other maintenance regimens, primarily corticosteroids, ciclosporin, or mycophenolate mofetil, were also eligible if the dose of treatment had been stable in the past month and the dose was expected to be stable after enrolment and remained unchanged at least for the first 4 weeks of study until initial response was assessed, unless severe adverse events were suspected.
排除标准
- •Secondary ITP (e.g., patients with HIV, HBV, HCV, Helicobacter pylori infection or SLE);
- •Congestive heart failure, severe arrhythmia;
- •Nursing or pregnant women;
- •ALT or AST levels ≥ 3× the upper limit of the normal threshold;
- •Creatinine or serum bilirubin levels ≥ 1.5× the upper limit;
- •Active or previous malignancy ;
- •Patients who had received danazol treatment or did not respond to danazol;
- •Patients unable to have routine blood tests because of reasons such as insufficient time.
研究组 & 干预措施
tacrolimus and Danazol
Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.
干预措施: TAC (Drug)
tacrolimus and Danazol
Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.
干预措施: Danazol (Drug)
Danazol
Danazol is given at 200mg bid for 12 weeks.
干预措施: Danazol (Drug)
结局指标
主要结局
Sustained response
时间窗: 6 months
Sustained response was defines as the maintenance of platelet count ≥ 30 x 10\^9/L, and at least 2-fold increase of the baseline count, and the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
次要结局
- Response(6 months)
- Complete response(6 months)
- Time to response(6 months)
- Duration of response(6 months)
- Adverse events(6 months)
研究者
Xiao Hui Zhang
Vice president of Peking Univeristy Institute of Hematology
Peking University People's Hospital