A Multicenter Randomized Trial of First Line Treatment for Newly Diagnosed Immune Thrombocytopenia: Standard Steroid Treatment Versus Combined Steroid and Cyclosporin
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 253
- 试验地点
- 1
- 主要终点
- Treatment failure
研究概览
简要总结
Randomized, open-label, multicenter study to compare the efficacy and safety of cyclosporin plus standard steroid compared to standard steroid monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).
详细描述
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 253 adults with ITP in China. Patients were randomized to cyclosporin plus standard steroid compared to standard steroid 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
- Single (Outcomes Assessor)
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
- •Newly-diagnosed, treatment-naive primary ITP;
- •Platelet counts <30×10^9/L;
- •Platelet counts < 50×10^9/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
- •Willing and able to sign written informed consent.
排除标准
- •Pregnant or lactating women;
- •Secondary ITP (have a known diagnosis of connective tissue diseases, malignancy, active infection, HIV infections or hepatitis B virus or hepatitis C virus infections);
- •Received first-line and second-line ITP specific treatments (e.g., steroids, intravenous immunoglobulin, TPO-RAs, rhTPO, rituximab, etc);
- •Received drugs affecting the platelet counts within 6 months before the screening visit (e.g., chemotherapy, anticoagulants, etc);
- •Severe medical condition (lung, heart, hepatic or renal disorder);
- •Patients who are deemed unsuitable for the study by the investigator.
研究组 & 干预措施
Standard steroid
Standard regimen for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.
干预措施: Prednisone (Drug)
Cyclosporin plus Steroid
Cyclosporin: started orally 1 mg/kg/d in two divided doses for 1 week, increased to 1.5 mg/kg/d for 1week, further increased to 2.5 mg/kg/d and then continued for 24 weeks. After 26 weeks of cyclosporin treatment, the dose for patients who achieved complete response was reduced by 25 mg/d every 2 weeks to ensure continuing the lowest dose that achieved the targeted serum level of cyclosporin and a safe platelet count.
Standard regimen of steroid for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.
干预措施: Cyclosporine Oral Product (Drug)
Cyclosporin plus Steroid
Cyclosporin: started orally 1 mg/kg/d in two divided doses for 1 week, increased to 1.5 mg/kg/d for 1week, further increased to 2.5 mg/kg/d and then continued for 24 weeks. After 26 weeks of cyclosporin treatment, the dose for patients who achieved complete response was reduced by 25 mg/d every 2 weeks to ensure continuing the lowest dose that achieved the targeted serum level of cyclosporin and a safe platelet count.
Standard regimen of steroid for a total of 10 weeks: 1 mg per kilogram of body weight for 2 weeks followed by 40 mg daily for 2 weeks, 20 mg daily for 2 weeks, 10 mg daily for 2 weeks, 5 mg daily for 1 week and 5 mg every other day for the final week.
干预措施: Prednisone (Drug)
结局指标
主要结局
Treatment failure
时间窗: From date of randomization until 2 years or the end of follow-up
Nonresponse or loss of response for those who had achieved overall response (assessed in a time-to-event analysis). Overall response was defined as platelet count ≥ 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.
次要结局
- Time to response(From date of randomization until 2 years or the end of follow-up)
- Duration of response(From date of randomization until 2 years or the end of follow-up)
- Remission(at 12-month follow-up)
- Number of patients with side effects(From date of randomization until 2 years or the end of follow-up)
- Number of patients with bleeding(From date of randomization until 2 years or the end of follow-up)
- Sustained response(From date of randomization until 2 years or the end of follow-up)
- Overall response (OR)(From date of randomization until 2 years or the end of follow-up)
- Complete response (CR)(From date of randomization until 2 years or the end of follow-up)
- Rescue therapy(From date of randomization until 2 years or the end of follow-up)
- Associated factors of treatment failure, OR, SR and remission(From date of randomization until 2 years or the end of follow-up)
研究者
Xiao Hui Zhang
Vice president of Peking Univeristy Institute of Hematology
Peking University People's Hospital