Teriflunomide and High-dose Dexamethasone vs High-dose Dexamethasone Alone as First-line Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia: A Prospective, Multicenter, Randomized Trial
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 132
- 试验地点
- 3
- 主要终点
- Sustained response
研究概览
简要总结
A multicenter, open-label, randomized study to report the efficacy and safety of teriflunomide plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with newly diagnosed primary immune thrombocytopenia (ITP).
详细描述
The investigators are undertaking a parallel-group, multicenter, randomized controlled trial of 132 adults with ITP in China. Patients were randomized to teriflunomide 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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients aged ≥18 years;
- •Newly diagnosed, treatment naïve ITP patients;
- •Patients with a platelet count <30 x10^9/L or a platelet count <50 x10^9/L with bleeding manifestations at the enrollment;
- •Willing and able to sign written informed consent.
排除标准
- •Secondary ITP such as drug-related thrombocytopenia, viral infection (HIV, hepatitis B virus, or hepatitis C virus);
- •Pre-existing acute or chronic liver disease, or ALT/AST greater than 2 times the upper limit of normal (ULN);
- •Severe cardiac, renal, hepatic, or respiratory insufficiency;
- •Severe immunodeficiency;
- •Pregnancy or lactation;
- •Active or a history of malignancy;
- •Active infection requiring systemic therapy;
- •Myelodysplastic syndrome, aplastic anemia, or myelofibrosis;
- •A known diagnosis of other autoimmune diseases;
- •Patients who are deemed unsuitable for the study by the investigator.
研究组 & 干预措施
Teriflunomide plus Dexamethasone
Oral Teriflunomide was given at a dose of 14 mg once daily for 24 weeks, and dexamethasone was given at a dose of 40mg orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4). Nonresponsive participants with platelets less than 30 x10^9/L or with active bleeding were allowed to repeat the 4-day course of dexamethasone treatment.
干预措施: Teriflunomide (Drug)
Teriflunomide plus Dexamethasone
Oral Teriflunomide was given at a dose of 14 mg once daily for 24 weeks, and dexamethasone was given at a dose of 40mg orally, once per day for 4 consecutive days (Days 1, 2, 3, and 4). Nonresponsive participants with platelets less than 30 x10^9/L or with active bleeding were allowed to repeat the 4-day course of dexamethasone treatment.
干预措施: Dexamethasone (Drug)
Dexamethasone
Dexamethasone was given at a dose of 40mg, orally once per day for 4 consecutive days (Days 1, 2, 3, and 4). Nonresponsive participants with platelets less than 30 x10^9/L or with active bleeding were allowed to repeat the 4-day course of dexamethasone treatment.
干预措施: Dexamethasone (Drug)
结局指标
主要结局
Sustained response
时间窗: Week 24
Sustained response was defined as a platelet count ≥ 30 x10\^9/L and at least a 2-fold increase of the baseline count in the absence of bleeding and rescue therapy for at least three of the four visits of the last 8 weeks of treatment.
次要结局
- Initial response(Week 4)
- Time to response(Week 24)
- Duration of response(Week 24)
- Bleeding events(Week 24)
- Adverse events(Week 24)
- Health-related quality of life (HRQoL)(Week 24)
研究者
Xiao Hui Zhang
Vice president of Peking Univeristy Institute of Hematology
Peking University People's Hospital