A Single-centre, Prospective, Open-label, Single-arm Study of Romiplostim for the Treatment of Refractory Transfusion-dependent Non-severe Aplastic Anaemia (NSAA)
试验速览
- 阶段
- 4 期
- 状态
- 尚未招募
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- CRR
研究概览
简要总结
Efficacy and safety of Romiplostim in the treatment of refractory transfusion-dependent NSAA.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Age ≥18 years, male or female.
- •Diagnosis consistent with refractory transfusion dependence NSAA defines refractory as patients who have failed to respond to at least 6 months of prior first-line treatment with adequate doses of cyclosporine (3-5 mg/kg) and who have been treated with an adequate dose of at least one povidone for 3 months. Definition of transfusion dependence: at least 1 component transfusion on average every 8 weeks and duration of transfusion dependence ≥ 4 months.
- •Satisfy at least one of the following conditions at the time of enrolment: haemoglobin <90 g/L, platelets <30×10^9/L, neutrophils <1.0×10^9/L.
- •Agree to sign the consent form.
- •An Eastern Cooperative Oncology Group (ECOG) score of 0-2.
排除标准
- •Other causes of whole blood cytopenia, such as myelodysplastic syndromes (MDS).
- •Presence of cytogenetic evidence of clonal haematological bone marrow disorders (MDS, AML).
- •PNH clones ≥50%.
- •Haematopoietic stem cell transplantation (HSCT) prior to enrolment.
- •Prior treatment with ATG.
- •Infection or haemorrhage uncontrolled by standard therapy.
- •Allergy to roprostin.
- •Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.
- •Any concomitant malignancy, localised basal cell carcinoma of the skin within 5 years.
- •Liver and renal function at baseline that is more than two times normal.
- •Active infection.
- •Past history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome) and current use of anticoagulants.
- •Pregnant or lactating (breastfeeding) women.
- •Participation in another clinical trial within 3 months.
研究组 & 干预措施
Romiplostim group
Romiplostim group
干预措施: Romiplostim (Drug)
结局指标
主要结局
CRR
时间窗: 6 months
CR was defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients who did not receive a transfusion.
Overall response rate (ORR)
时间窗: 6 months
Overall response rate (ORR) was defined as the ratio of complete response (CR) + partial response (PR).CR was defined as a haemoglobin level ≥120 g/L, neutrophil count \>1.5 × 10\^9/L and platelet count \>150 × 10\^9/L in patients who did not receive a transfusion.PR was defined as not being transfusion-dependent (if previously transfusion-dependent), or doubling or normalisation of at least one cell line or baseline increase, or initial ANC \<0.5 × 10\^9/L increased by at least 0.5 × 10\^9/L after treatment, or initial PLT \<20 × 10\^9/L increased by at least 20 × 10\^9/L after treatment.
次要结局
- safety events(6months)
研究者
Bing Han
chief physician
Peking Union Medical College Hospital