The Combination of Atorvastatin, Acetylcysteine and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
试验速览
- 阶段
- 1 期
- 入组人数
- 200
- 试验地点
- 1
- 主要终点
- the sustained platelet response at the 6-month follow-up
研究概览
简要总结
Single-arm, open-lable, multicentre study to compare the efficacy and safety of atorvastatin, acetylcysteine plus danazol with danazol monotherapy in patients with corticosteroidresistant/relapsed ITP.
详细描述
Immune thrombocytopenia (ITP) is a severe bleeding disorder. Approximately 2/3 of patients achieve remission from first-line therapies. However, the underlying mechanism of corticosteroid-resistant or relapsed ITP is not well understood; thus, treatment remains a great challenge. Atorvastatin was shown to enhance bone marrow endothelial cell function and N-acetylcysteine (NAC) was shown to inhibit PLT binding to endothelial cell.
A multicentre prospective study was performed in non-splenectomized ITP patients who were either resistant to a standard dose of corticosteroids or had relapsed. Patients were randomized to atorvastatin, acetylcysteine plus danazol with danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study, in order to compare the efficacy and safety of atorvastatin, acetylcysteine plus danazol with danazol monotherapy in patients with corticosteroid-resistant/relapsed ITP.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 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
- •ITP confirmed by excluding other supervened causes of thrombocytopenia;
- •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;
- •EPCs in bone marrow less than 0.02%
排除标准
- •Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus)
- •congestive heart failure
- •severe arrhythmia
- •nursing or pregnant women
- •aspartate aminotransferase and alanine transaminase levels ≥ 3× the upper limit of the normal threshold criteria
- •creatinine or serum bilirubin levels each 1.5 times or more than the normal range
- •active or previous malignancy
- •Unable to do blood routine test for the sake of time, distance, economic issues or other reasons.
研究组 & 干预措施
atorvastatin, acetylcysteine & danazol
atorvastatin 20mg qd po plus acetylcysteine 400mg tid po plus danazol 200mg bid po for 12 weeks
干预措施: atorvastatin (Drug)
atorvastatin, acetylcysteine & danazol
atorvastatin 20mg qd po plus acetylcysteine 400mg tid po plus danazol 200mg bid po for 12 weeks
干预措施: Acetylcysteine (Drug)
atorvastatin, acetylcysteine & danazol
atorvastatin 20mg qd po plus acetylcysteine 400mg tid po plus danazol 200mg bid po for 12 weeks
干预措施: Danazol (Drug)
结局指标
主要结局
the sustained platelet response at the 6-month follow-up
时间窗: From the start of study treatment (Day 1) up to the end of Month 6
The number of participants (responders) with platelet count \>=30x10\^9/L and at least a 2-fold increase in the baseline count (PR) or a platelet count \>=100x10\^9/L (CR) and the absence of bleeding, without rescue medication at 6-month follow-up.
次要结局
- overall response(From the start of study treatment (Day 1) up to the end of Month 6)
- time to response(From the start of study treatment (Day 1) up to the end of Year 2)
- duration of response(From the start of study treatment (Day 1) up to the end of Year 2)
- incidence of treatment-emergent adverse events(From the start of study treatment (Day 1) up to the end of Year 2)
研究者
Xiao-hui Zhang
Professor
Peking University People's Hospital