A Single-center, Single-arm, Prospective Clinical Study on the Efficacy and Safety of Cyclosporine Combined With Avatrombopag in the Treatment of Non-severe Aplastic Anemia in the Elderly
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- ORR at 6 Months
研究概览
简要总结
For elderly patients who cannot tolerate anti-thymocyte globulin (ATG) treatment, the addition of avatrombopag (AVA), which has a slight adverse reaction, can theoretically improve the hematological response rate in elderly patients with non-severe aplastic anemia (NSAA) without significantly increasing adverse reactions. Based on this, this study treated NSAA patients older than 60 with AVA combined with CsA to evaluate the hematological response rate and safety of AVA in the elderly who could not tolerate ATG therapy.
详细描述
Aplastic anemia (AA) can be divided into severe AA (SAA) and non-severe AA (NSAA), according to the severity of the disease. Anti-thymocyte globulin (ATG) in combination with CsA is the most typical combined immunosuppressive therapy regimen. For elderly patients who cannot tolerate anti-thymocyte globulin (ATG) treatment, the addition of avatrombopag, which has a slight adverse reaction, can theoretically improve the hematological response rate in elderly patients with NSAA without significantly increasing adverse reactions. Based on this, this study treated NSAA patients older than 60 with AVA combined with CsA to evaluate the hematological response rate and safety of AVA in the elderly who could not tolerate ATG therapy.
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
- 60 Years 至 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Elderly patients with well-defined NSAA anemia who meet the diagnostic criteria for aplastic anemia (AA) but do not meet the diagnostic criteria for severe aplastic anemia (SAA).
- •Age 60 years or older, male or female.
- •Able to swallow or administer orally.
- •Intolerant or refused anti-thymocyte globulin treatment
- •No previous treatment with cyclosporine, tacrolimus or hormones or treatment for less than 1 month.
- •No prior treatment with thrombopoietin (TPO) receptor agonists (including eltrombopag, herombopag, romiplostim, etc.)
- •Informed consent must be signed before the start of all specific research procedures. Considering the patient's condition, if the patient's signature is not conducive to the treatment of the disease, the informed consent shall be signed by the patient's immediate family.
排除标准
- •Known congenital AA (such as Fanconi anemia) and other causes of pancytopenia and hematological bone marrow disorders;
- •With paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%
- •With a history of hematopoietic stem cell transplantation.
- •History of thrombosis
- •Patients with underlying cancer who also have malignant tumors or are receiving immunosuppressive therapy.
- •Baseline creatine levels greater than twice of the upper limit of normal (ULN) and/or alanine aminotransferase (ALT) greater than 2.5 times of the ULN;
- •Serious heart, liver and kidney disease.
- •With uncontrolled bleeding and/or infection after standard treatment.
- •Participants considered unsuitable for inclusion by the researchers.
研究组 & 干预措施
treatment group
Cyclosporine: 3mg/kg/d 3-5mg/kg bid was administered to maintain the trough concentration at 100-200 μg/ml;
Avatrombopag: The dosage was 40~60mg orally, once a day, and the dosage was adjusted according to the subject's platelet count. The drug was administered for 24 weeks (6 months).
干预措施: Ciclosporin (Drug)
treatment group
Cyclosporine: 3mg/kg/d 3-5mg/kg bid was administered to maintain the trough concentration at 100-200 μg/ml;
Avatrombopag: The dosage was 40~60mg orally, once a day, and the dosage was adjusted according to the subject's platelet count. The drug was administered for 24 weeks (6 months).
干预措施: Avatrombopag (Drug)
结局指标
主要结局
ORR at 6 Months
时间窗: 6 months
Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR)
次要结局
- Percentage of patients with clonal evolution to myelodysplasia, PNH, acute leukemia(12 months)
- Relapse rate(6 months, last follow-up)
- ORR at 1, 2, 3 Months and at the end of follow-up(1, 2, 3 Months, last follow-up)
- Changes in Platelet in the Absence of Platelet Transfusion(6 months)
- Changes in Haemoglobin in the Absence of Red Blood Cells Transfusion(6 months)
研究者
Bing Han
Professor
Peking Union Medical College Hospital