The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment
研究概览
简要总结
This is a prospective, single-arm, open-label study. Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, including blood transfusion and iron removal, based on the clinician's judgment and practice. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.
详细描述
Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, being injected subcutaneously every 3 weeks ,by the dose of 1-1.25 mg/kg. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.
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
- 否
入选标准
- •≥18 years old
- •The patient was clearly diagnosed as transfusion-dependent β -thalassemia, the blood transfusion period was ≤60 days, and the red blood cell infusion volume was not less than 3-12U in the 12 weeks before enrollment, which could provide the red blood cell infusion volume record in the 12 weeks before enrollment
- •Patients who were treated with thalidomide before enrollment were required to stop the treatment for more than 4 weeks, and their hemoglobin decreased to below 90g/L. Blood transfusion was required, and records of blood transfusion within 12 weeks could be provided
- •Voluntarily participate in the study and sign the informed consent;
排除标准
- •(1) Pregnant or lactating women
- •persons known to be allergic to Luspatercept and/or Luspatercept excipients for injection
- •Severe liver dysfunction (liver enzyme (ALT or AST) ≥3 TIMES ULN)
- •Severe renal impairment (eGFR < 30 ml/min/1.73m3 or patients with end-stage renal disease)
- •Heart disease, heart failure classified as Class 3 or higher by the New York Heart Association (NYHA), or severe arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization.
- •The patient has uncontrolled hypertension. According to NCI CTCAE version 5.0, controlled hypertension in this protocol is considered to be ≤
- •Patients with a history of deep vein thrombosis or stroke within 24 weeks prior to enrollment
- •Erythropoietin, Luspatercept or hydroxyurea were used 8 weeks before enrollment
- •Any other significant medical condition, laboratory abnormality, or mental illness that the Investigator deems inappropriate for inclusion.
研究组 & 干预措施
treatment group
accept Luspatercept treatment
干预措施: Luspatercept Injectable Product (Drug)
结局指标
主要结局
The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment
时间窗: 24weeks
incidence of reported adverse events
A 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline
时间窗: 24 weeks
the incidence of 33% reduction in RBC transfusion
次要结局
- Changes in mean serum ferritin (SF) levels in the population from baseline(24 weeks)
- The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population(24 weeks)
- the incidence of 50% reduction in transfusion in any 12 consecutive weeks(24 weeks)
- changes in reticulocyte levels from baseline during treatment(24 weeks)
- Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24(24 weeks)
- Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*)(24 weeks)
- changes in fetal hemoglobin levels from baseline during treatment(24weeks)
研究者
Jianpei FANG
Professor
Sun Yat-sen University