The Combination of Terbutaline and Danazol as the Treatment of Steroid-resistant/Relapse Immune Thrombocytopenia
试验速览
- 阶段
- 2 期
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Sustained response
研究概览
简要总结
Single-arm, open-label, single center study to assess the efficacy and safety of terbutaline plus danazol in patients with corticosteroid resistant/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. β2-AR agonist terbutaline modulates T cell differentiation and effector cell function.
A single center prospective study was performed in non-splenectomized ITP patients who were either resistant to a standard dose of corticosteroids or had relapsed. Patients were assigned to terbutaline plus danazol group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study, in order to assess the efficacy and safety of terbutaline plus danazol 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;
- •Subject has signed and dated written informed consent.
- •Fertile patients must use effective contraception during treatment and observational period
- •Negative pregnancy test.
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.
- •diagnosis with any of the following diseases: chronic hypertension, hyperthyroidism, diabetes, or seizure disorder.
研究组 & 干预措施
Terbutaline plus Danazol group
Terbutaline 2.5mg tid po plus danazol 200mg bid po for 12weeks
干预措施: Terbutaline (Drug)
Terbutaline plus Danazol group
Terbutaline 2.5mg tid po plus danazol 200mg bid po for 12weeks
干预措施: Danazol (Drug)
结局指标
主要结局
Sustained 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. Interim analysis was scheduled at 50% through recruitment.
次要结局
- complete remission(6 months)
- partial remission(6 months)
- duration of response(6 months)
- time to response(6 months)
- incidence of treatment-emergent adverse events(6 months)
研究者
Xiao Hui Zhang
Vice president of Peking Univeristy Institute of Hematology
Peking University People's Hospital