Eltrombopag for Enhancing Platelet Engraftment in Pediatric Patients Undergoing Cord Blood Transplantation
试验速览
- 阶段
- 2 期
- 入组人数
- 20
- 试验地点
- 2
- 主要终点
- Platelet engraftment rate up to 50 days post transplantation, defined as the first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter.
研究概览
简要总结
Platelet recovery is significantly hampered following umbilical cord blood transplantation (UCB). Thus, after UCB transplantations, patients are platelet transfusion-dependent for prolong periods of time.
Eltrombopag is a thrombopoietin-receptor agonist that initiates thrombopoietin-receptor signaling and thereby induces proliferation and maturation of megakaryocytes.
We will evaluate the safety and efficacy of eltrombopag for enhancing platelet engraftment in pediatric patients undergoing cord blood transplantation.
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
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient ≥ 1 year and < 18 years old.
- •Patients receiving unmanipulated single or double UCB allogeneic grafts.
- •Malignant and non malignant indications for transplantation.
- •Myeloablative and reduced intensity conditioning regimens.
- •Patients must meet all other pre-transplantation criteria of the transplantation center including acceptable tests of heart, liver, kidney, and lung function (standard screening for transplantation per PI, and co-investigators).
- •Written informed must be obtained from the patient's guardian, and accompanying informed assent from the patient for children over 6 years old.
- •Able to comply with study protocol.
排除标准
- •Indications for transplantation
- •Patients with primary myelofibrosis.
- •French-American-British classification (FAB)M7 acute myeloid leukemia. Acute leukemia secondary to a myeloproliferative neoplasm.
- •Patients with persistent acute leukemia (>5% bone marrow blasts) at the time of transplantation.
- •Patients with prior thromboembolic event. Patients with previous catheter related thrombosis will be eligible if more than 3 months elapsed.
- •Hypersensitivity to eltrombopag.
- •Liver enzymes abnormalities:
- •Alanine aminotransferase (ALT) levels > 3 times the upper limit of normal (ULN) or serum bilirubin > 1.5 ULN (unless due to Gilbert's syndrome or hemolytic bilirubin).
研究组 & 干预措施
Eltrombopag
Pediatric patients undergoing allogeneic UCB transplantation will receive eltrombopag from day +1 until platelet count has exceeded 50,000/microliter for 14 consecutive days without platelet transfusion.
Starting doses are 100 mg/d for children >40 kg body weight (BW), 50 mg/d for children 20-40 kg BW, and 2 mg/kg/d for children <20 kg BW.
If unsupported platelet count has not reached the threshold of 20,000/microliter, doses will be escalated every 2 weeks up to maximal doses of 200 mg/d for children ≥ 40 kg BW, 150 mg/d for children 20-40 kg BW and 3.5 mg/kg for children <20 kg BW.
干预措施: eltrombopag (Drug)
结局指标
主要结局
Platelet engraftment rate up to 50 days post transplantation, defined as the first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter.
时间窗: 50 days
次要结局
- Time to partial platelet engraftment, defined as the first of 7 consecutive days of an unsupported platelet count of at least 20,000/microliter.(180 days)
- Time to sustained complete platelet engraftment defined as the first of 7 consecutive days of an unsupported platelet count of at least 50,000/microliter.(180 days)
- Complete platelet engraftment rates up to 50 days post transplantation defined as the first of 7 consecutive days of an unsupported platelet count of at least 50,000/microliter.(50 days)
- Time to neutrophil engraftment defined as the first of 3 consecutive days to achieve an absolute neutrophil count ≥ 500/microliter.(60 days)
研究者
moshe yeshurun
Head, BMT unit, Rabin Medical Center
Rabin Medical Center