Efficacy and Safety of Avatrombopag for Platelet Recovery After Unrelated Cord Blood Transplantation in Patients With Bone Marrow Failure Disease: a Single-center, Single-arm, Phase II Trial
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Time of platelet engraftment
研究概览
简要总结
To evaluate the efficacy and safety of Avatrombopag for platelet recovery after unrelated cord blood transplantation (UCBT) in patients with bone marrow failure disease
详细描述
Persistent thrombocytopenia is a common complication after umbilical cord blood transplantation (UCBT), which necessitates platelet transfusion and leads to increased transplant-related mortality. This is a single-center, single-arm, phase II clinical trial aimed at evaluating the efficacy and safety of Avatrombopag for platelet recovery after UCBT in patients with bone marrow failure disease (BMFD). The study was conducted in patients diagnosed with BFMD and receiving UCBT. After the subjects signed and informed and qualified, they received Avatrombopag treatment from the first day to the 28th day after transplantation. In this study, the cumulative of platelet implantation rate at 28 days after transplantation was used as the main therapeutic index, and 40 patients were planned to be enrolled.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥12 years old, male or female;
- •Patients diagnosed with bone marrow failure disease including aplastic anemia (AA), Fanconi anemia (FA), paroxysmal nocturnal hemoglobinuria (PNH) and undergoing UCBT;
- •ECOG score ≤2;
- •Voluntary participation in this clinical trial, patients fully understand the trial content and sign the informed consent；
排除标准
- •Pregnant or lactating women；
- •Known allergy to Avatrombopag;
- •A history of severe thrombotic events or known risk factors for thrombosis or active thromboembolism requiring anticoagulation;
- •A history of platelet dysfunction or bleeding prone disease or severe bleeding (requiring more than 2 units of red blood cell infusion or a hematocrit drop of ≥10%) within 7 days prior to screening;
- •Chronic active hepatitis B and C;
- •Repeat or multiple transplantation or multiple organ transplantation;
- •HIV positive, EBV-DNA positive, CMV-DNA positive;
- •Have severe infection or complicated with serious heart, liver, lung, kidney, neurological or metabolic diseases;
- •A serious adverse event (Level 4 or 5) as defined by the Standard General Terminology for Adverse Events Version 4.0 occurred during the pre-treatment period;
- •Participants participate in another clinical study with any exploratory drug or device within 30 days prior to baseline visit; Observational studies are allowed;
- •Subjects with cognitive impairment or uncontrolled mental illness;
- •Subjects and/or authorized family members to refuse treatment with Avatrombopag;
- •Those who are judged by the researchers as not suitable for inclusion (such as accompanying medical history, which may affect the safety of the subjects or it is estimated that the treatment cannot be adhered to due to financial problems).
研究组 & 干预措施
Avatrombopag group
The duration of the study is 4 weeks, from +1 day to +28 days after UCBT, patients will be given Avatrombopag with a recommended dose of 40mg per day. After 28 days of administration, the patients may continue, change or discontinue medication based on the patients' platelet status.
干预措施: Avatrombopag (Drug)
结局指标
主要结局
Time of platelet engraftment
时间窗: 28 days
Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10\^9/L.
The cumulative incidence of platelet engraftment at 28 days after transplantation
时间窗: 28 days
Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10\^9/L.
次要结局
- erythroid reconstitution(28 days)
- Time of platelet ≥100×10^9/L.(100 days)
- The cumulative incidence of platelet recovery rate at 28 days after transplantation(28 days)
- Total platelet transfusion during 4 weeks of treatment(28 days)
- Megakaryocyte levels in bone marrow smears 4 weeks after treatment(4 weeks)
- neutrophil reconstitution(28 days)
- Time of platelet recovery(100 days)
研究者
Xiaoyu Zhu
Chief physician
Anhui Provincial Hospital