The Combination of High-dose Dexamethasone and Tacrolimus Versus High-dose Dexamethasone as the First-Line Treatment of Newly-diagnosed Immune Thrombocytopenia: A Randomized, Controlled, Multicenter, 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（Durable） response
研究概览
简要总结
Randomized, open-label, multicenter study to compare the efficacy and safety of Combination of High-dose Dexamethasone and Tacrolimus versus High-dose Dexamethasone 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 100 adults with ITP in China. Patients were randomized to tacrolimuis plus high-dose dexamethasone and high-dose dexamethasone 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
- •Confirmed newly-diagnosed, treatment-naive ITP;
- •Platelet counts <30×109/L ;
- •Platelet counts < 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above);
- •Willing and able to sign written informed consent.
排除标准
- •Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;
- •Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ;
- •Current HIV infection or hepatitis B virus or hepatitis C virus infections;
- •Active infection;
- •Severe medical condition (lung, hepatic or renal disorder) other than chronic ITP. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);
- •Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period; a history of clinically significant adverse reactions to previous corticosteroid therapy
- •Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;
- •Patients who are deemed unsuitable for the study by the investigator.
研究组 & 干预措施
HD-DXM
Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone is repeated in the case of lack of response by day 14) .
干预措施: Dexamethasone (Drug)
TAC and HD-DXM
Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone is repeated in the case of lack of response by day 14).
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: Dexamethasone (Drug)
TAC and HD-DXM
Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone is repeated in the case of lack of response by day 14).
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: Tacrolimus (Drug)
结局指标
主要结局
Sustained（Durable） response
时间窗: 6 months
The maintenance of platelet count ≥ 30 x 10\^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
次要结局
- Complete response (CR)(Day 14)
- Loss of response(6 months)
- Response (R)(Day 14)
- Duration of response (DOR)(6 months)
- Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale(From the start of study treatment (Day 1) up to the end of week 24.)
- Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ)(Time Frame: From the start of study treatment (Day 1) up to the end of week 24.)
- Time to response(6 months)
- Functional Assessment of Chronic Illness Therapy fatigue subscale (FACIT-F)(Time Frame: From the start of study treatment (Day 1) up to the end of week 24.)
- Number of Participants with side effects of the drugs(Time Frame: From the start of study treatment (Day 1) up to the end of week 24.)
研究者
Xiao Hui Zhang
Vice President of Peking University Institute of Hematology
Peking University People's Hospital