Observational Retrospective Study on Incidence, First-line Treatment Patterns, and Clinical Outcomes in Transfusion-dependent Lower-risk Myelodysplastic Syndromes in Spain Using the BIG-PAC® Database
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 1,300
- 试验地点
- 1
- 主要终点
- Incidence of transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)
研究概览
简要总结
The purpose of this study is to understand the incidence of transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS) and describing real-world first-line treatment patterns, healthcare resource utilization, and associated clinical outcomes in adult patients with TD LR-MDS in Spain
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
- •Age ≥18 years at the time of lower-risk myelodysplastic syndromes (LR-MDS) diagnosis.
- •Documented diagnosis of LR-MDS identified through International Classification of Diseases (ICD) 9 codes recorded in medical history. In addition, recorded diagnosis of MDS with an explicitly documented International Prognostic Scoring System (IPSS) category of low or intermediate-1 and/or revised IPSS category of very low or low at or around the index date.
- •Evidence of transfusion dependence, defined as receiving ≥2 red blood cell (RBC) units within an 8-week interval, occurring within the selection window (January 1, 2021, to May 31, 2025, or the latest date ensuring detectable follow-up).
- •Active participants in BIG-PAC®, defined as ≥1 claim of any kind within 12 months prior to or on the index date (baseline period).
- •A minimum of 6 months of follow-up data available after the index date, unless the patient dies earlier
排除标准
- •Diagnosis of high-risk MDS (HR-MDS) or another hematologic malignancy (e.g., acute myeloid leukemia) before the index date.
- •Documented transformation to acute myeloid leukemia (AML) or HR-MDS occurring before initiation of first-line treatment.
- •Participation in interventional clinical trials during the period of first-line treatment.
- •Presence of anemia secondary to non-MDS-related causes, such as nutritional deficiencies, advanced chronic kidney disease, or active bleeding, when such conditions preclude accurate attribution of transfusion dependence to MDS.
- •Lack of sufficient clinical history, defined as <12 months of observable data before the index date.
- •Have missing key variables, e.g., age or sex.
- •Incomplete or inconsistent clinical information that prevents reliable evaluation of key study variables, including transfusion dependence status, treatment patterns, or outcomes.
研究组 & 干预措施
Cohort 10
Participants aged <75 years
干预措施: Luspatercept (Biological)
Cohort 10
Participants aged <75 years
干预措施: Hypomethylating agents (HMAs) (Drug)
Cohort 10
Participants aged <75 years
干预措施: Reb blood cell transfusion (Biological)
Cohort 11
Participants aged ≥75 years
干预措施: Erythropoiesis-stimulating agents (ESAs) (Drug)
Cohort 11
Participants aged ≥75 years
干预措施: Luspatercept (Biological)
Cohort 11
Participants aged ≥75 years
干预措施: Lenalidomide (Drug)
Cohort 11
Participants aged ≥75 years
干预措施: Hypomethylating agents (HMAs) (Drug)
Cohort 11
Participants aged ≥75 years
干预措施: Reb blood cell transfusion (Biological)
Cohort 4
Participants that initiated first-line treatment with hypomethylating agents (HMAs)
干预措施: Hypomethylating agents (HMAs) (Drug)
Cohort 5
Participants that initiated first-line treatment with lenalidomide
干预措施: Lenalidomide (Drug)
Cohort 3
Participants that initiated first-line treatment with luspatercept
干预措施: Luspatercept (Biological)
Cohort 1
Overall cohort of participants with transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)
干预措施: Erythropoiesis-stimulating agents (ESAs) (Drug)
Cohort 1
Overall cohort of participants with transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)
干预措施: Luspatercept (Biological)
Cohort 1
Overall cohort of participants with transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)
干预措施: Lenalidomide (Drug)
Cohort 1
Overall cohort of participants with transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)
干预措施: Hypomethylating agents (HMAs) (Drug)
Cohort 1
Overall cohort of participants with transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)
干预措施: Reb blood cell transfusion (Biological)
Cohort 2
Participants that initiated first-line treatment with erythropoiesis-stimulating agents (ESAs)
干预措施: Erythropoiesis-stimulating agents (ESAs) (Drug)
Cohort 6
Participants that receive conservative management (red blood cell transfusions without disease modifying therapy)
干预措施: Reb blood cell transfusion (Biological)
Cohort 7
Participants with low red-blood cell (RBC) transfusion dependance (<4 RBC per 8 weeks)
干预措施: Reb blood cell transfusion (Biological)
Cohort 8
Participants with moderate red-blood cell (RBC) transfusion dependance (4-5 RBC per 8 weeks)
干预措施: Reb blood cell transfusion (Biological)
Cohort 9
Participants with high red-blood cell (RBC) transfusion dependance (≥6 RBC per 8 weeks)
干预措施: Reb blood cell transfusion (Biological)
Cohort 10
Participants aged <75 years
干预措施: Lenalidomide (Drug)
Cohort 10
Participants aged <75 years
干预措施: Erythropoiesis-stimulating agents (ESAs) (Drug)
结局指标
主要结局
Incidence of transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)
时间窗: Up to 5-years
Prevalence of transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)
时间窗: Up to 5-years
次要结局
- Number of participants that experience loss of response/secondary failure after an initial hematologic response(Up to 5-years)
- Cause of death(Up to 5-years)
- Serious adverse events (SAEs)(Up to 5-years)
- Number of comorbidities(Baseline)
- Smoking status(Baseline)
- Proportion of participants receiving each first-line treatment category(Up to 5-years)
- Proportion of participants by treatment category at each line of therapy(Up to 5-years)
- Treatment duration (time from initiation to discontinuation) of first-line treatment(Up to 5-years)
- Defined as number of RBC units received per participant per 8-weeks(Up to 5-years)
- Number and Rate of Healthcare Resource Utilization Events(Up to 5-years)
- Number of participants that achieve hematologic improvement-erythroid (HI-E)(Up to 5-years)
- Number of participants that achieve red-blood cell (RBC) transfusion independence (TI)(Up to 5-years)
- Number of participants that experience a change in transfusion burden (change in number of red-blood cell units received per 8-weeks)(Up to 5-years)
- Number of participants that progress to higher-risk myelodysplastic syndromes (MDS)(Up to 5-years)
- Number of participants that progress to acute myeloid leukemia (AML)(Up to 5-years)
- Number of participants that do not respond to first-line treatment(Up to 5-years)
- Number of serious cardiovascular events requiring emergency room visit or hospitalization(Up to 5-years)
- Participant age(Baseline)
- Participant sex(Baseline)
- Year of first-line treatment initiation(Baseline)
- Participant Body mass index (BMI)(Baseline)
- Participant Charlson Comorbidity Index (CCI) score(Baseline)
- Participant Charlson Comorbidity Index (CCI) individual comorbidities(Baseline)