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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
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Overall cohort of participants with transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS) |
| |
| Cohort 2 | Participants that initiated first-line treatment with erythropoiesis-stimulating agents (ESAs) |
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| Cohort 3 | Participants that initiated first-line treatment with luspatercept |
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| Cohort 4 | Participants that initiated first-line treatment with hypomethylating agents (HMAs) |
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| Cohort 5 | Participants that initiated first-line treatment with lenalidomide |
| |
| Cohort 6 |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Erythropoiesis-stimulating agents (ESAs) | Drug | According to the product label |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS) | Up to 5-years | |
| Prevalence of transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS) | Up to 5-years |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of participants receiving each first-line treatment category | Treatment categories include:
| Up to 5-years |
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Inclusion Criteria:
Exclusion Criteria:
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The study population will consist of adult participants diagnosed with transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS) identified in Spain using the BIG-PAC® database
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| Name | Affiliation | Role |
|---|---|---|
| Bristol-Myers Squibb | Bristol-Myers Squibb | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Local Institution - 0001 | Madrid | 28001 | Spain |
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| Label | URL |
|---|---|
| BMS Clinical Trial Information | View source |
| FDA Safety Alerts and Recalls | View source |
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Participants that receive conservative management (red blood cell transfusions without disease modifying therapy)
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| Cohort 7 | Participants with low red-blood cell (RBC) transfusion dependance (<4 RBC per 8 weeks) |
|
| Cohort 8 | Participants with moderate red-blood cell (RBC) transfusion dependance (4-5 RBC per 8 weeks) |
|
| Cohort 9 | Participants with high red-blood cell (RBC) transfusion dependance (≥6 RBC per 8 weeks) |
|
| Cohort 10 | Participants aged <75 years |
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| Cohort 11 | Participants aged ≥75 years |
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| Luspatercept | Biological | According to the product label |
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| Lenalidomide | Drug | According to the product label |
|
| Hypomethylating agents (HMAs) | Drug | According to the product label |
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| Reb blood cell transfusion | Biological | According to the product label |
|
| Proportion of participants by treatment category at each line of therapy |
Treatment category received at each line of therapy (first line, second line, third line and beyond), categorized as erythropoiesis-stimulating agents (ESA), luspatercept, hypomethylating agents (HMA), lenalidomide, or conservative management (red blood cell transfusions without disease-modifying therapy). Treatment sequences will be derived from retrospective medical chart abstraction of documented treatment start and stop dates. |
| 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 | Healthcare resource utilization events, including primary care visits, specialist visits, outpatient visits all-cause hospitalizations, transfusion-related hospitalizations, general laboratory tests, number of prescriptions related to transfusion-dependent lower-risk myelodysplastic syndromes, red blood cell transfusions, use of concomitant medicines related to other comorbidities as documented in participant medical records. | 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 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 serious cardiovascular events requiring emergency room visit or hospitalization | Cardiovascular events include: will include acute myocardial infarction or acute coronary syndrome, heart failure decompensation, clinically significant arrhythmias (including atrial fibrillation/flutter and ventricular arrhythmia), stroke or transient ischemic attack, and venous thromboembolism. | 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 |
| Number of comorbidities | Baseline |
| Smoking status | Baseline |
| ID | Term |
|---|---|
| D006397 | Hematinics |
| C000621232 | luspatercept |
| D000077269 | Lenalidomide |
| ID | Term |
|---|---|
| D006401 | Hematologic Agents |
| D045506 | Therapeutic Uses |
| D020228 | Pharmacologic Actions |
| D020164 | Chemical Actions and Uses |
| D010797 | Phthalimides |
| D010795 | Phthalic Acids |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D010881 | Piperidones |
| D010880 | Piperidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D054833 | Isoindoles |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
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