A Novel Conditioning Regimen for Haplo-HSCT in Older Patients With SAA
2026年7月1日 更新者:Xiao-Jun Huang、Peking University People's Hospital
A Novel Conditioning Regimen for Haploidentical Hematopoietic Stem Cell Transplant in Patients Aged ≥ 40 Years Old With Severe Aplastic Anemia: a Multicenter, Single-arm, Observational Clinical Trial
The goal of this prospective, multicenter, single arm observational study is to evaluate the efficacy and safety of the BFCA regimen in ≥ 40 years old SAA patients undergoing haplo-HSCT.
研究概览
研究类型
观察性的
注册 (估计的)
64
联系人和位置
本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。
学习联系方式
- 姓名:Tingting Han
- 电话号码:8601088326666
- 邮箱:htt1984.love@163.com
学习地点
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Beijing Municipality
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Beijing、Beijing Municipality、中国、100044
- 招聘中
- Peking Universtiy Peoples' Hospital
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接触:
- Tingting Han, Doctor
- 电话号码:86-01088424953
- 邮箱:htt1984.love@163.com
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-
参与标准
研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
- 成人
接受健康志愿者
不
取样方法
非概率样本
研究人群
Patients aged 40-60 years old who are diagnosed with severe aplastic anemia according to NCCN guidelines and who will receive haploidentical hematopoietic stem cell transplantation at the centers will be recruited.
描述
Inclusion Criteria:
- Severe aplastic anemia;
- Aged 40-60 years old;
- Weight 45Kg-100Kg;
- Eastern Cooperative Oncology Group (ECOG) score ≤3;
- No major organ injury (ECG ejection fraction >45%; bilirubin < 2 times the upper limit of normal value; AST and ALT < 3 times the upper limit of normal value; serum creatinine < 2 times the upper limit of normal value);
- No severe infection;
- Subjects voluntarily participated in this clinical trial and signed the informed consent.
Exclusion Criteria:
- With other hematologic diseases;
- Expected survival of less than 1 month;
- Previous autologous or allogeneic hematopoietic stem cell transplantation;
- Pregnant patients;
- Patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events;
- Other conditions that the investigator determines to be inappropriate for enrollment.
- Current or recent (<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection
- A history of symptomatic herpes zoster infection within 12 weeks prior to screening
- Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)
- Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB
- Exposure to a live vaccine within 12 weeks prior to enrollment or expected to receive a live vaccine during the study
- Clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled
- Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure
- A history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data
Any of the following specific abnormalities on screening laboratory tests:
- ALT or AST >2 x ULN, or total bilirubin ≥1.5 x ULN
- hemoglobin <9 g/dL, or total white blood cell (WBC) count <2,500/µL, or neutropenia (absolute neutrophil count <1,200/µL), or lymphopenia (lymphocyte count <750/µL)
- eGFR <50 mL/min/1.73 m2
学习计划
本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。
研究是如何设计的?
设计细节
队列和干预
团体/队列 |
干预/治疗 |
|---|---|
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苏州大学第一附属医院
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busulfan 0.8 mg/kg/6h (days -8 to -7), fludarabine 30mg/m2/day (days -6 to -2), cyclophosphamide 25 mg/kg/day (days -5 to -2) and antithymocyte globulin(ATG) 2.5 mg/kg/day (days -5 to -2).
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Peking unviversity Peoples' Hospital
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busulfan 0.8 mg/kg/6h (days -8 to -7), fludarabine 30mg/m2/day (days -6 to -2), cyclophosphamide 25 mg/kg/day (days -5 to -2) and antithymocyte globulin(ATG) 2.5 mg/kg/day (days -5 to -2).
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Guangzhou First People's Hospital,
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busulfan 0.8 mg/kg/6h (days -8 to -7), fludarabine 30mg/m2/day (days -6 to -2), cyclophosphamide 25 mg/kg/day (days -5 to -2) and antithymocyte globulin(ATG) 2.5 mg/kg/day (days -5 to -2).
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The First Affiliated Hospital of USTC
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busulfan 0.8 mg/kg/6h (days -8 to -7), fludarabine 30mg/m2/day (days -6 to -2), cyclophosphamide 25 mg/kg/day (days -5 to -2) and antithymocyte globulin(ATG) 2.5 mg/kg/day (days -5 to -2).
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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Faliure free survival (FFS)
大体时间:From enrollment to 2 years post-HSCT
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survival with a response to therapy after HSCT.
Death, graft faliue and relapse were considered as treatment failure.
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From enrollment to 2 years post-HSCT
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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混合嵌合现象的发生率
大体时间:HSCT 后 1 年
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混合嵌合被定义为存在5%-95%供体造血细胞。
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HSCT 后 1 年
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治疗方案相关毒性
大体时间:HSCT 后 100 天
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根据西雅图毒性标准(Bearman 等,1988)测量方案相关毒性(RTT)。
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HSCT 后 100 天
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骨髓和血小板植入
大体时间:HSCT 后 100 天
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骨髓和血小板植入被定义为国际标准。
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HSCT 后 100 天
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The probability of Overall survival
大体时间:From enrollment to 2 years post-HSCT
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Overall survival was defined as the time from transplantation to death from any cause or to the last follow-up
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From enrollment to 2 years post-HSCT
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The incidence of Transplantation related mortality
大体时间:2 years post HSCT
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Transplantation related mortality was defined as death without disease progression.
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2 years post HSCT
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The incidence of graft versus host disease(GVHD)
大体时间:100 days post HSCT for aGvHD and 2 years post HSCT for cGvHD
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The severity of acute and chronic GVHD was evaluated according to standard criteria.
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100 days post HSCT for aGvHD and 2 years post HSCT for cGvHD
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The incidence of Cytomegalovirus(CMV) and Epstein-Barr virus(EBV) reactivation
大体时间:180 days post HSCT
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The incidence of CMV and EBV reactivation was defined as CMV and EBV viremia.
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180 days post HSCT
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (估计的)
2026年6月1日
初级完成 (估计的)
2027年12月1日
研究完成 (估计的)
2029年12月30日
研究注册日期
首次提交
2026年5月8日
首先提交符合 QC 标准的
2026年5月8日
首次发布 (实际的)
2026年5月14日
研究记录更新
最后更新发布 (实际的)
2026年7月6日
上次提交的符合 QC 标准的更新
2026年7月1日
最后验证
2026年6月1日
更多信息
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