Real World Use of Luspatercept in Lower Risk Myelodysplastic Syndromes and Beta-thalassemia Patients
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Participant age
研究概览
简要总结
The purpose of this study is to understand the patient profile, utilized treatments, treatment patterns, resulting outcomes, and associated costs relating to luspatercept treatment in patients with lower-risk myelodysplastic syndromes (LR-MDS) or beta-thalassemia in the real-world setting
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
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
- •Participants treated with luspatercept. The date of first treatment with luspatercept will be assigned as the index date.
- •At least one Lower-risk myelodysplastic syndromes (LR-MDS) or beta-thalassemia diagnosis in inpatient or at least two MDS or beta-thalassemia diagnosis in the outpatient setting at least 30 days apart prior to or at index date.
- •18+ years old at index date.
- •Participants with a minimum of 12-months observable pre-treatment baseline/look-back prior to index date.
- •Participants with a minimum of 6-months of follow-up time from the index date unless the patient is lost due to death
排除标准
- 未提供
研究组 & 干预措施
Cohort 2
Participants diagnosed beta-thalassemia receiving luspatercept treatment
干预措施: Luspatercept (Drug)
Cohort 1
Participants diagnosed myelodysplastic syndromes receiving luspatercept treatment
干预措施: Luspatercept (Drug)
结局指标
主要结局
Participant age
时间窗: Baseline
Participant gender
时间窗: Baseline
Year of first luspatercept prescription
时间窗: Baseline
Number of participants by Ring Sideroblast (RS) status (positive/negative/unknown) as measured by bone marrow aspiration
时间窗: Baseline
As documented in participant medical charts
Participant comorbidities
时间窗: Baseline
Length of follow-up from start of luspatercept treatment initiation until the earliest of the end of continuous enrolment, death, or end of data availability
时间窗: Baseline
次要结局
- Time to treatment discontinuation(Up to 6 months)
- Number of participants that achieve red blood cell (RBC) transfusion independence(Up to 12 months)
- Total number of RBC units transfused per participant, as assessed from transfusion records and medical chart review, from baseline up to 6 months(Baseline and up to 6 months)
- Time to acute myeloid leukemia transformation(Up to 6 months)