Clinical Registration Study of Haploidentical Stem Cell Transplantation for Elderly Patients With Acute Leukemia or Myelodysplastic Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 120
- 试验地点
- 1
- 主要终点
- 1-year LFS
研究概览
简要总结
This study is an open, prospective, single-center, single-arm study expanded based on the study of NCT03412409.The primary objective of this study is to evaluate the transplantation efficacy of the RIC conditioning regimen in elderly patients receiving the transplantation of haploidentical hematopoietic stem cells. Elderly patients are defined as ≥55 years of age; High comorbidity is defined as an HCT-CI score ≥3.Primary endpoint is 1-year LFS. Secondary endpoints are 1-year OS and 1-year TRM, Other indicators to be assessed in this study include acute GVHD, chronic GVHD, CMV activation, EBV activation, engraftment.
详细描述
Currently, the transplantation of allogeneic hematopoietic stem cells (allo-SCT) remains the main and often the only cure for neoplastic hematologic disorder or hematological malignancy. Although HLA-matched siblings are the preferred donor, not all patients have one; or it needs to wait so long to obtain it that the optimal time for transplantation is past. Haploidentical transplants as secondary donors are more readily available and may be considered in combination with the conditions of the donor and the patient and the experience of the transplant facility when a matched donor is unavailable. Currently, haploidentical transplantation has been commonly applied to young patients. On the other hand, TRM rate will be higher in elderly patients and patients with high comorbidity due to tolerability, thus reducing the ultimate benefits of patients.
The choice of conditioning regimen is influenced by multiple factors such as disease type, disease status, physical condition, and source of transplant donor. For patients over 55 years of age or with a comorbidity index ≥3, the RIC regimen may be considered to improve patient tolerability. RIC regimen can improve the tolerability of patients receive transplants, while regimens such as immune suppressants are also needed to control and improve the overall transplant efficiency. An in-depth study of RIC regimen will help improve the effects of transplantation and increase patient benefits.
This study is aim to evaluate the efficacy of RIC conditioning regimen for transplantation in elderly patients or patients with high comorbidity receiving the transplantation of haploidentical hematopoietic stem cells.
Drugs and dosage of conditioning regimen:
Ara-C(2g/m2/day,-10 days to -9 days),Bu(9.6mg/kg,-8 days to -6 days),Flu(30mg/m2/day,-6 days to -2 days),Cy(1,000mg/m2/day,-5 days to -4 days),semustine(250mg/m2,-3 days),ATG(10 mg/kg,-5 to -2 days).
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
- 55 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients ≥55 years of age and <70 years of age, with acute leukemia or MDS (with or without HCT-CI≥3);
- •Patients eligible for the transplantation of allogeneic hematopoietic stem cells;
- •Patients unavailable with HLA matched donors but available with haploidentical donors;
- •Voluntary participation in this study and signing the informed consent form.
排除标准
- •Patients with the severe infections;
- •Pregnant or lactating women or female and male subjects of childbearing age and their spouses who are unable to secure effective contraception during the dosing period and within 90 days after the end of the dosing period.
- •Patients who are not eligible for hematopoietic stem cell transplantation;
- •Patients who are enrolled in other clinical trials within 1 month;
- •Patients who may not be able to complete the study for other reasons, or may be considered by the investigator not suitable to participate in the study;
- •Patients unable to properly understand or refusing to accept the informed consent form.
结局指标
主要结局
1-year LFS
时间窗: Participants will be followed for an expected average of 1 years
defined as disease relapse, disease progression or death (whichever occurs first) within 1 year from the day the patient receives transplantation.
次要结局
- 1-year TRM(Participants will be followed for an expected average of 1 years)
- 1-year OS(Participants will be followed for an expected average of 1 years)
研究者
Xiao-Jun Huang
Principal Investigator
Peking University People's Hospital