An Observational Study on the Promotion of Platelet Recovery by Romiplostim During Haploidentical Hematopoietic Stem Cell Transplantation
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 16
- 试验地点
- 1
- 主要终点
- Platelet recovery time
研究概览
简要总结
The objective of this observational study is to explore the long-term effects of roprastine given to promote platelet implantation in hematopoietic stem cell hemicongruent transplantation in children with thalassemia. The main questions it aimed to answer is:
Is roprastine safe and effective for platelet implantation in children with thalassemia hemicongruent transplantation? Participants who have already received roprastine as part of routine medical care for hematopoietic stem cell hemicongruent transplantation in children with thalassemia will answer online survey questions about the effects of their platelet implantation within 8 weeks.
详细描述
Allogeneic hematopoietic stem cell transplantation is an important, if not the only, means of curing many diseases of the blood system. Thrombocytopenia after transplantation seriously affects the long-term survival rate of patients. allo - The incidence of thrombocytopenia in HSCT patients is 5-20% (< 20 x 10 ^ 9/L), increasing the risk and cost of treatment. There are currently few studies on the promotion of platelet growth in children with thalassemia. Repeated infusion of platelet suspension can lead to many adverse consequences, including blood transfusion reactions, platelet homeoimmune responses, and transfusion-associated virus infections. There is a black box warning of liver toxicity in eltropopa, and the incidence of real-world liver toxicity is 11.8%. Transplant patients are prone to diarrhea, which affects the absorption of oral platelet-raising drugs. Daily subcutaneous injection increases children's pain and poor tolerance. However, the long-acting platelet-raising drugs once after transplantation are well tolerated in children with thalassemia transplantation, and the efficacy is worth looking forward to. Up to now, there is a lack of relevant clinical data on the application of roprastine to promote platelet recovery in children with thalassemia hemiphase transplantation. Therefore, this study aimed to explore the observational study of roprastine to promote platelet implantation in children with thalassemia hemiphase transplantation, and to explore the efficacy and safety of the drug.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 年龄范围
- 2 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •After undergoing Mediterranean gene testing, reviewing the history of blood transfusions, and conducting a blood routine examination, the patient was diagnosed with severe thalassemia.
- •Children aged 2 - 17 years old.
- •Agree to the haploidentical transplantation, and the team has evaluated that there are no transplantation contraindications.
排除标准
- •There is a fully matched donor, and transplantation with a half - matched donor is not agreed.
- •For donors and recipients, the transaminase is more than twice the normal value.
- •Donor specific antibody Greater than 5000, and after antibody treatment, it should not be lower than
- •Positive for hepatitis B DNA.
- •There is an active infection.
- •After evaluation by the transplantation team, there are contraindications for transplantation.
研究组 & 干预措施
experimental group
After transplantation + 6 days, the subcutaneous injection of roprastine 3ug/kg was started, and the number of platelets in the machine-taken platelet suspension was increased by 2 µg/kg per week, and the maximum dose was 10 μg/kg. Discontinued until platelets rose to 100 × 109/L. If platelets ≤ 20 X 109/L were used for + 20 days, roprastine combined with atropopa 25mg was given orally once a day. Patients were given 1 therapeutic dose of fresh machine-taken platelet suspension when platelets were ≤ 20 X 109/L, or when there was active bleeding at 21~ 50 × 109/L. The number of platelets in the machine-taken platelet suspension per therapeutic dose was > 2.5 × 1011. If not implanted at + 28 days after transplantation, re-transplantation is required, and roprastine is considered ineffective.
干预措施: Romiplostim (Drug)
结局指标
主要结局
Platelet recovery time
时间窗: From enrollment to 28 days after transplantation
The time at nodes such as platelet \>20×10\^9/L, 50×10\^9/L and 100×10\^9/L
Platelet transfusion volume
时间窗: From enrollment to 28 days after transplantation
The required dosage of platelet suspension
次要结局
- Survival rate(From enrollment to 28 days after transplantation)
- Adverse drug reaction rate(From enrollment to 28 days after transplantation)
- Bleeding incidence rate(From enrollment to 28 days after transplantation)
- Thrombosis incidence rate(From enrollment to 28 days after transplantation)
- Recurrence - free survival rate(From enrollment to 28 days after transplantation)
- Transplant - related mortality(From enrollment to 28 days after transplantation)
- Major transplant-related complications(From enrollment to 28 days after transplantation)
- Cause of death(From enrollment to 28 days after transplantation)