Lower-risk Myelodysplastic Syndromes (TD LR-MDS)
Conditions
Keywords
Transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)
Brief summary
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
Interventions
DRUGErythropoiesis-stimulating agents (ESAs)
According to the product label
BIOLOGICALLuspatercept
According to the product label
DRUGLenalidomide
According to the product label
DRUGHypomethylating agents (HMAs)
According to the product label
BIOLOGICALReb blood cell transfusion
According to the product label
Sponsors
Bristol-Myers Squibb
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Serious adverse events (SAEs) | Up to 5-years | — |
| Proportion of participants receiving each first-line treatment category | Up to 5-years | Treatment categories include: * Erythropoiesis-stimulating agents (ESA) * Luspateracept * Hypomethylating agents (HMAs) * Lenalidomide * Conservative management (red-blood cell (RBC) transfusions without disease modifying therapy) |
| Proportion of participants by treatment category at each line of therapy | Up to 5-years | 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. |
| 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 | 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. |
| 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 | 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. |
| 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 | — |
| Number of participants that experience loss of response/secondary failure after an initial hematologic response | Up to 5-years | — |
| Smoking status | Baseline | — |
| Cause of death | Up to 5-years | — |
Countries
Spain
Contacts
STUDY_DIRECTORBristol-Myers Squibb
Bristol-Myers Squibb
Outcome results
None listed