An Observational Study on the Promotion of Platelet Recovery by Hetrombopag 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
- 30
- 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 investigate the long-term effects of Hetrombopag in promoting platelet engraftment during haploidentical hematopoietic stem cell transplantation (HSCT) in children with thalassemia, with a specific focus on a 28-day time window post-transplantation. The core question to be addressed is: Is Hetrombopag safe and effective for platelet engraftment in children with thalassemia undergoing haploidentical HSCT within a 28-day post-transplant period? Subjects who received Hetrombopag as part of routine care for haploidentical HSCT in children with thalassemia will be required to complete a 28-day online survey on platelet engraftment outcomes.
详细描述
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains a critical and often the sole curative modality for diverse hematological malignancies and disorders. Post-transplant thrombocytopenia (platelet count <20×10⁹/L) significantly compromises long-term patient survival, with an incidence of 5-20% in allo-HSCT recipients, thereby escalating treatment morbidity and costs. Current research on platelet engraftment promotion in pediatric thalassemia patients undergoing allo-HSCT is limited. Repeated platelet transfusions are associated with substantial complications, including transfusion reactions, platelet alloimmunization, and transfusion-transmitted viral infections. Eltrombopag, a thrombopoietin receptor agonist (TPO-RA), carries a black-box warning for hepatotoxicity, with a real-world incidence of 11.8%. Transplant recipients frequently experience diarrhea, which impairs the absorption of oral thrombopoietic agents, while daily subcutaneous injections exacerbate pediatric patient discomfort and reduce treatment adherence. In contrast, long-acting TPO-RAs administered once post-transplant have demonstrated favorable tolerability and promising efficacy in thalassemia transplant pediatric populations. To date, clinical data on Hetrombopag use for platelet recovery in haploidentical HSCT for pediatric thalassemia are lacking. Therefore, this observational study aims to evaluate the efficacy and safety of Hetrombopag in facilitating platelet engraftment during haploidentical HSCT in children with thalassemia.
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
- •Diagnosed with severe thalassemia via thalassemia gene testing, transfusion history, and complete blood count (CBC).
- •Pediatric patients aged 2-17 years.
- •Consented to haploidentical transplantation and evaluated by the transplant team as having no transplant contraindications.
排除标准
- •Presence of a fully matched donor with refusal of haploidentical transplantation.
- •Donor or recipient with transaminase levels >2× the upper limit of normal (ULN).
- •Positive hepatitis B DNA test result.
- •Active infection at the time of enrollment.
- •Donor-specific antibodies (DSA) >5,000 and unable to decrease below 3,000 after antibody therapy.
- •Presence of transplant contraindications as assessed by the transplant team.
研究组 & 干预措施
Experimental group
In allogeneic hematopoietic stem cell transplantation, hetrombopag is initiated at 3 µg/kg subcutaneously on day +6 post-transplant. The dose is increased by 2 µg/kg weekly up to a maximum of 10 µg/kg. Treatment is discontinued when platelet counts rise to 100×10⁹/L. If platelet counts remain ≤20×10⁹/L on day +20, hetrombopag is combined with eltrombopag 25 mg orally once daily. Fresh apheresis platelet suspensions (1 therapeutic dose, containing >2.5×10¹¹ platelets) are administered when platelet counts are ≤20×10⁹/L or when counts are between 21-50×10⁹/L with active bleeding. If engraftment has not occurred by day +28 post-transplant, re-transplantation is required, and hetrombopag is considered ineffective.
干预措施: Hetrombopag (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
Required dosage of platelet suspension
次要结局
- Survival rate(From enrollment to 28 days after transplantation)
- Transplant-related mortality rate(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)
- Relapse-free survival rate(From enrollment to 28 days after transplantation)
- The rate of adverse drug reactions(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)