The Combination of ATRA and Eltrombopag as the Treatment Strategy for Glucocorticoid-Resistant/Relapsed ITP Based on the Stratification of the New Biomarker MSC-C5b-9: A Prospective, Randomized, Open-Label, Multicenter Clinical Trial
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 96
- 试验地点
- 5
- 主要终点
- Sustained Response Rate (SR) at 18 months
研究概览
简要总结
A Prospective, Randomized, Open-Label, Multicenter Clinical Trial study to compare the efficacy and safety of ATRA plus eltrombopag compared to eltrombopag monotherapy in the treatment of steroid-resistant/relapsed immune thrombocytopenia (ITP).
详细描述
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of patients with ITP in China. Patients were tested for MSCs, and they were divided into MSC-C5b-9+ group and MSC-C5b-9- group according to the test results, and the two groups were randomized to ATRA + eltrombopag and eltrombopag 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
- •1. Isolated thrombocytopenia (platelet count <30 × 109/L);
- •age > 18 years;
- •normal white blood cells and red blood cells on bone marrow examination;
- •increased number of megakaryocytes (bone marrow examination was performed in all patients except for myelofibrosis or other conditions that can cause thrombocytopenia disease);
- •the spleen was normal in size;
- •Eastern Cooperative Oncology Group status score (ECOG score) ≤ 2;
- •ineffective or relapsed after at least 1 course of full-dose full-course hormone therapy;
- •Failure of prior ITP therapy (eg, hormones, splenectomy, and cyclosporine) and at least 4 weeks from enrollment.
排除标准
- •1. Secondary ITP such as drug-related thrombocytopenia;
- •thrombocytopenia due to viral infection (HIV, hepatitis B virus, or hepatitis C virus);
- •severe cardiac, renal, hepatic, or respiratory insufficiency;
- •severe immunodeficiency;
- •pregnancy or lactation;
- •myelodysplasia or Myelofibrosis;
- •history of malignancy;
- •ongoing immunosuppressive therapy for other diseases;
- •patients previously treated with eltrombopag were excluded from this study.
研究组 & 干预措施
Eltrombopag
The initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The highest is 75mg/d, if the dose is higher than 200×109/L, the dose is reduced. When the dose is higher than 400×109/L, the drug is temporarily discontinued, and the drug is repeated according to the platelet count. The treatment course is 12 weeks.
干预措施: Eltrombopag (Drug)
ATRA and Eltrombopag
ATRA 10 mg, 2 times a day, orally; The initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The maximum dose is 75 mg/d, the dose is reduced if it is higher than 200×109/L, and the drug is temporarily discontinued when it is higher than 400×109/L, and the drug is repeated according to the platelet count. The treatment course is 12 weeks.
干预措施: all-trans retinoic acid (Drug)
ATRA and Eltrombopag
ATRA 10 mg, 2 times a day, orally; The initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The maximum dose is 75 mg/d, the dose is reduced if it is higher than 200×109/L, and the drug is temporarily discontinued when it is higher than 400×109/L, and the drug is repeated according to the platelet count. The treatment course is 12 weeks.
干预措施: Eltrombopag (Drug)
结局指标
主要结局
Sustained Response Rate (SR) at 18 months
时间窗: 18 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 18-month follow-up
次要结局
- Inefficiency (NR)(18 months)
- Recurrence rate (relapse)(18 months)
- Initial response(1 month)
- Complete response rate (CR)(18 months)
- Response rate (R)(18 months)
- Time to relapse (duration of efficacy)(18 months)
- Early response(1 week)
- Time to response (TTR)(18 months)
研究者
Xiao Hui Zhang
Vice president of Peking Univeristy Institute of Hematology
Peking University People's Hospital