Luspatercept Treatment Patterns and Outcomes Among ESA-Naïve Patients With Lower-Risk MDS - A Retrospective Medical Record Review in the United States
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 418
- 试验地点
- 1
- 主要终点
- Participant baseline demographics
研究概览
简要总结
The purpose of this study is to understand real-world effectiveness of luspatercept treatment among erythropoiesis-stimulating agents -naïve patients with lower-risk- myelodysplastic syndromes in the United States
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
- •Had a documented diagnosis of primary or secondary myelodysplastic syndromes (MDS)
- •MDS diagnosis confirmed through bone marrow testing on (or 30 days prior to) MDS diagnosis date or within 1 year of MDS diagnosis date
- •Had a documented determination of Lower Risk (LR)-MDS as measured by International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) at or before index treatment (i.e., first-line luspatercept or first-line erythropoiesis-stimulating agents (ESA)) initiation
- •IPSS risk level: low, intermediate-1 (level-1 risk)
- •IPSS-R risk level: very low, low, intermediate
- •Received luspatercept as the first-line treatment for anemia any time from 28 August 2023 to 31 July 2024 (Cohort 1)
- •Receipt of combination therapy with ESAs and/or granulocyte colony-stimulating factors (G-CSFs) will be allowed
- •Received ESA as the first-line treatment for anemia any time from 28 August 2023 to 31 July 2024 (Cohort 2)
- •Was aged 18 years or older at the time of initial diagnosis of MDS
- •Known vital status (i.e., living, or deceased) at the time of record abstraction.
- •Records for patients who are dead or alive will be eligible
- •Complete medical record covering relevant past medical history, diagnosis of LR-MDS, treatment, laboratory assessments, red-blood cell (RBC) transfusions, and regular monitoring for LR-MDS, including any transfer record from other physicians/facilities (if applicable) is available to the abstracting physician for data abstraction
排除标准
- •Had a history of acute myeloid leukemia (AML) prior to MDS diagnosis
- •Received previous treatment with hypomethylating agents, disease-modifying agents (including lenalidomide), other immunosuppressants/immunomodulatory agents, or other MDS-directed chemotherapy
- •Received stem cell transplant prior to index treatment initiation
- •Participated in a clinical trial for the treatment of MDS before or while on index treatment (i.e., clinical trial participation after first-line luspatercept or ESA treatment discontinuation will be allowed)
- •Had evidence of other malignant neoplasms in the 12 months prior to diagnosis of MDS, except basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, carcinoma in situ of the breast, or incidental histologic finding of prostate cancer (stage T1a or T1b)
- •Patients for whom this information is not available (i.e., "unknown") will be included in the study
- •For Cohort 1 (i.e., first-line luspatercept treatment), receipt of combination therapy with hypomethylating agents, lenalidomide, other immunosuppressants/ immunomodulatory agents, or other MDS-directed chemotherapy
- •For Cohort 2 (i.e., first-line ESA treatment), receipt of combination therapy with hypomethylating agents, lenalidomide, luspatercept, other immunosuppressants/ immunomodulatory agents, or other MDS-directed chemotherapy
研究组 & 干预措施
Participants receiving first-line luspatercept treatment
干预措施: Luspatercept (Drug)
Participants receiving first-line erythropoiesis-stimulating agents
干预措施: Erythropoiesis-stimulating agents (Drug)
结局指标
主要结局
Participant baseline demographics
时间窗: Baseline
Participant baseline clinical characteristics
时间窗: Baseline
Time from Lower Risk- myelodysplastic syndromes diagnosis to index treatment initiation
时间窗: Baseline
Rationale for therapy selection
时间窗: Baseline
Duration of index treatment
时间窗: Up to 15 months
Treatment dose at treatment initiation and discontinuation
时间窗: Up to 6 months
Treatment dose/dosing schedule changes, and treatment interruptions
时间窗: Up to 12 months
Other supportive care therapies prescribed while on index treatment
时间窗: Up to 15 months
Treatments prescribed post index treatment
时间窗: Up to 15 months
Treatments for anemia management received after discontinuing the index treatment
时间窗: Up to 15 months
Receipt of stem cell transplant at any time post index treatment
时间窗: Up to 15 months
Participant red-blood cell (RBC) transfusion burden post index treatment
时间窗: At 3-months, and up to 6 months
Hematologic improvement-erythroid (HI-E) response post index treatment
时间窗: At 3-months, and up to 6 months
Progression to acute myeloid leukemia post index treatment
时间窗: Up to 15 months
Progression to high-risk myelodysplastic syndromes per the International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) criteria
时间窗: Up to 15 months
Participant adverse events during and post index treatment
时间窗: Up to 15 months
Overall survival (OS)
时间窗: At 3-, 6-, 12-, and up to 15-months
Healthcare resource utilization (HCRU) during index treatment
时间窗: Up to 15 months
次要结局
未报告次要终点