A Prospective, Randomized, Controlled Study of rhTPO in Combination With Herombopag + CsA vs Herombopag + CsA for the Treatment of Primary Transfusion-dependent NSAA
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 入组人数
- 90
- 试验地点
- 1
- 主要终点
- ORR at 6 Months
研究概览
简要总结
Aplastic anemia (AA) is a group of clinical syndromes. Treatment options are very limited. The results of a previous clinical study showed good efficacy and a high safety profile of herombopag in improving thrombocytopenia, but this result needs to be supported by more data.
In our study, patients who were willing to participate in this study and were diagnosed with transfusion-dependent non-heavy aplastic anemia were randomized to the rhTPO combined with herombopag + cyclosporine group and given rhTPO (at a dose of 1500 U by subcutaneous injection once daily for 7 d, 28 d for 3 courses) +Herombopag(10 mg/day for 3 months) + cyclosporine (3-5 mg/kg/d for 3 months). -5 mg/kg/d for at least 6 months) and herombopag + cyclosporine (10 mg/day for 3 months) + cyclosporine (3-5 mg/kg/d for at least 6 months) in the herombopag+ cyclosporine group to observe the efficacy and safety.
详细描述
Aplastic anemia (AA) is a group of clinical syndromes caused by a significant decrease in bone marrow hematopoietic tissue from different etiologies, resulting in hematopoietic failure. The prevalence of AA in China is 7.4 per 1 million. It peaks in the 15-25 and 60+ age groups and is more common in men than women. Treatment options are very limited. The results of a previous clinical study showed good efficacy and a high safety profile of herombopag in improving thrombocytopenia, but this result needs to be supported by more data.
In our study, patients who were willing to participate in this study and were diagnosed with transfusion-dependent non-heavy aplastic anemia were randomized to the rhTPO combined with herombopag + cyclosporine group and given rhTPO (at a dose of 1500 U by subcutaneous injection once daily for 7 d, 28 d for 3 courses) +Herombopag(10 mg/day for 3 months) + cyclosporine (3-5 mg/kg/d for 3 months). -5 mg/kg/d for at least 6 months) and Herombopag + cyclosporine (10 mg/day for 3 months) + cyclosporine (3-5 mg/kg/d for at least 6 months) in the herombopag+ cyclosporine group to observe the efficacy and safety.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Age ≥18 years old.
- •Clearly diagnosed untreated NSAA.
- •At least one of the following conditions was met at the time of enrollment: hemoglobin <90 g/L. Platelet <30×109/L, neutrophils <1.0×109/L.
- •Baseline liver and kidney function (ALT, AST, Cr) was less than 2 times the normal value.
- •No active infection; Not pregnant or breastfeeding.
- •Agree to sign the consent form.
- •The Eastern Cancer Collaboration Group (ECOG) score was 0-2.
排除标准
- •pancytopenia caused by other causes, such as myelodysplastic syndrome (MDS).
- •There is cytogenetic evidence of clonal hematologic bone marrow diseases (MDS, AML).
- •PNH clone ≥50%.
- •Had received hematopoietic stem cell transplantation (HSCT) before enrollment.
- •Immunosuppressive therapy such as ATG or cyclosporine use for more than 2 weeks.
- •Infection or bleeding that is not controlled by standard treatment.
- •Allergic to recombinant TPO or Hitrepopar.
- •Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.
- •Any concomitant malignancy or local basal cell carcinoma of the skin within 5 years.
- •Previous history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome) and current use of anticoagulants.
- •Women who are pregnant or nursing (lactation).
- •Have participated in other clinical trials within 3 months.
研究组 & 干预措施
Herombopag + CsA
Herombopag 10mg/ day, adjust the dose according to the blood image, the course of treatment is at least 3 months, CsA: 3-5 mg/kg/d, adjust the valley concentration 100-200ng/ml, at least 6 months, effective patients continue to use for 1 year, and then gradually reduce the dose.
干预措施: Herombopag + CsA (Drug)
rhTPO combined with Herombopag + CsA
Administer rhTPO (15000U, subcutaneously once daily for 7 days, once a month for 3 months),Herombopag 10mg/day, adjust dose according to blood picture for at least 3 months, CsA: 3-5 mg/kg/d, adjust trough concentration 100-200ng/ml for at least 6 months, continue for 1 year if effective, then gradually reduce dose
干预措施: rhTPO combined with Herombopag + CsA (Drug)
结局指标
主要结局
ORR at 6 Months
时间窗: Week 26
After 6 months of treatment, ORR was calculated by measuring platelets, hemoglobin, neutrophils, and transfusion independence
ORR at 3 Months
时间窗: Week 14
After 3 months of treatment, ORR was calculated by measuring platelets, hemoglobin, neutrophils, and transfusion independence
次要结局
- 6 months of drug safety(Week 26)
- 3 months of drug safety(Week 14)
研究者
Bing Han
Professor
Peking Union Medical College Hospital