N-acetyl-L-cysteine for Promoting Hematopoietic Recovery in Patients With Severe Aplastic Anemia (SAA) After Haploidentical Transplantation -- a Prospective Single-arm Clinical Study
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 30
- 主要终点
- The incidence of poor graft function (PGF), which was assessed at +2M post-HSCT.
研究概览
简要总结
This is a prospective single-arm clinical study to evaluate the role of NAC among patients with severe aplastic anemia (SAA) can promote hematopoietic recovery after haploidentical transplantation.
详细描述
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective treatment of severe aplastic anemia (SAA). However, poor hematopoietic reconstitution including poor graft function (PGF) and prolonged isolated thrombocytopenia (PT), remains a life-threatening complication after allo-HSCT. Especially with the increasing use of haploidentical allo-HSCT (haplo-HSCT) in the past ten years, PGF and PT have become growing obstacles contributing to high morbidity and mortality after allo-HSCT. A previous clinical prospective cohort study showed that NAC could improve the function of bone marrow endothelial progenitor cells and promote hematopoietic recovery among leukemia patients after haploidentical transplantation. Therefore, we hypothesized that the prophylactic administration of NAC could facilitate the recovery of hematopoietic capacity by improving the bone marrow microenvironment of patients with SAA after haploidentical 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
- 18 Years 至 50 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with SAA or vSAA
- •No severe organ injury
- •No uncontrolled active infections
- •Sign informed consent form, have the ability to comply with study and follow-up procedures
排除标准
- •Hypersensitivity to NAC or history of bronchial asthma
- •Life expectancy less than 30 days post-transplantation
- •Uncontrolled infections pre-transplantation
- •Cardiac dysfunction (particularly congestive heart failure, unstable coronary artery disease and serious cardiac ventricular arrhythmias requiring antiarrhythmic therapy)
- •Respiratory failure ( PaO2 ≤60mmHg)
- •Hepatic abnormalities (total bilirubin ≥2 times the upper limit of normal [ULN], alanine aminotransferase or aspartate aminotransferase ≥2 times the ULN)
- •Renal dysfunction (creatinine ≥1.5 times the ULN or creatinine clearance rate < 30 mL/min)
- •ECOG performance status ≥3
- •With any conditions not suitable for the trial (investigators' decision)
研究组 & 干预措施
NAC group
Aplastic anemia patients receiving haploidentical transplantation will be enrolled, and NAC (400mg tid) will be administered orally from day 14 before conditioning until day +60 post-HSCT. The initial dose of NAC was 400mg orally three times daily (TID).
干预措施: N Acetyl L Cysteine (Drug)
结局指标
主要结局
The incidence of poor graft function (PGF), which was assessed at +2M post-HSCT.
时间窗: Two months post-HSCT.
PGF was defined as the presence of 2 or 3 cytopenic counts (ANC≤0.5×109/L, platelet≤20×109/L, or hemoglobin≤70 g/L) for at least 3 consecutive days beyond day +28 post-HSCT with a transfusion requirement, related with hypoplastic-aplastic BM, in the presence of complete donor chimerism (CDC).
The incidence of prolonged thrombcytopenia (PT), which was assessed at +2M post-HSCT.
时间窗: Two months post-HSCT.
PT was defined as platelet count less than 20×109/L or a dependence on platelet transfusion with the engraftment of other cell lines(ANC\>0.5×109/L and hemoglobin\>70 g/L without transfusion support) beyond day +60 post-HSCT in the presence of complete donor chimerism (CDC).
次要结局
- The cumulative incidences of Thrombotic Microangiopathy (TMA).(Two months post-HSCT.)
- The cumulative incidences of graft versus host disease (GvHD).(Two months post-HSCT.)
- The cumulative incidences of transplantation related mortality (TRM).(Two months post-HSCT.)
- The cumulative incidences of overall survival (OS).(One year post-HSCT.)
- Adverse reactions(Two months post-HSCT.)
研究者
Xiao-Jun Huang
Professor
Peking University People's Hospital