Dose-attenuated IST and Hetrombopag in the Treatment of Elderly (≥65 Years) Patients With Very Severe/Severe Aplastic Anemia: A Single-Center, Single-Arm, Phase IIB Clinical Study on Efficacy and Safety
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 65
- 试验地点
- 1
- 主要终点
- Hematologic response rate at 24 weeks post-ATG treatment.
研究概览
简要总结
This is a prospetive,Single-Center, Single-Arm, Phase IIB Clinical Study.This study aims to evaluate the efficacy and safety of dose-attenuated IST combined with Hetrombopag in elderly patients (≥65 years) with VSAA/SAA.
详细描述
Design : Prospective, single-center, single-arm, open-label Phase IIB trial.
Intervention :
P-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag : 15 mg daily. Follow-Up : Weekly for 24 weeks, with bone marrow evaluation at 24 weeks.
Inclusion Criteria :
Confirmed diagnosis of VSAA/SAA. Age ≥65 years. Completion of all screening assessments. Ability to swallow oral medication. Signed informed consent (by patient or legal guardian if patient is incapacitated).
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
- 65 Years 至 —（Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of VSAA/SAA.
- •Age ≥65 years.
- •Completion of all screening assessments.
- •Must be able to swallow tablets.
- •Signed informed consent (by patient or legal guardian if patient is incapacitated).
排除标准
- •Clonal cytogenetic abnormalities (excluding isolated -Y or +8).
- •Prior treatment with ATG/high-dose cyclophosphamide.
- •Prior cyclosporine/tacrolimus use >12 months.
- •Prior TPO-RA therapy >3 months.
- •Uncontrolled malignancies or conditions contraindicating ATG.
- •Severe organ dysfunction (e.g., creatinine ≥177 μmol/L).
- •Investigator judgment of unsuitability.
研究组 & 干预措施
A-IST
Porcine-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3-5 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag: 15 mg daily.
干预措施: Dose-attenuated IST and Hetrombopag (Drug)
结局指标
主要结局
Hematologic response rate at 24 weeks post-ATG treatment.
时间窗: 24 weeks
PR+CR Partial Response (PR) : Independence from transfusions without meeting CR. No Response (NR) : Failure to achieve PR.
次要结局
- Early mortality rate(12 weeks)
- Complete Response(24 weeks)