A Study of Incidence, Treatment Patterns, and Outcomes in Transfusion-dependent Lower-risk Myelodysplastic Syndromes in Spain

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

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

Study Overview

• Status: Active, not recruiting
• Conditions: Lower-risk Myelodysplastic Syndromes (TD LR-MDS)
• Intervention / Treatment: Drug: Erythropoiesis-stimulating agents (ESAs); Biological: Luspatercept; Drug: Lenalidomide; Drug: Hypomethylating agents (HMAs); Biological: Reb blood cell transfusion

• Study Type: Observational
• Enrollment (Estimated): 1300

Contacts and Locations

Study Locations

• Spain
  • Madrid, Spain, 28001
    • Local Institution - 0001

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

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

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

11

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cohort 1; Overall cohort of participants with transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)	Drug: Erythropoiesis-stimulating agents (ESAs); According to the product label; Biological: Luspatercept; According to the product label; Drug: Lenalidomide; According to the product label; Drug: Hypomethylating agents (HMAs); According to the product label; Biological: Reb blood cell transfusion; According to the product label
Cohort 2; Participants that initiated first-line treatment with erythropoiesis-stimulating agents (ESAs)	Drug: Erythropoiesis-stimulating agents (ESAs); According to the product label
Cohort 3; Participants that initiated first-line treatment with luspatercept	Biological: Luspatercept; According to the product label
Cohort 4; Participants that initiated first-line treatment with hypomethylating agents (HMAs)	Drug: Hypomethylating agents (HMAs); According to the product label
Cohort 5; Participants that initiated first-line treatment with lenalidomide	Drug: Lenalidomide; According to the product label
Cohort 6; Participants that receive conservative management (red blood cell transfusions without disease modifying therapy)	Biological: Reb blood cell transfusion; According to the product label
Cohort 7; Participants with low red-blood cell (RBC) transfusion dependance (<4 RBC per 8 weeks)	Biological: Reb blood cell transfusion; According to the product label
Cohort 8; Participants with moderate red-blood cell (RBC) transfusion dependance (4-5 RBC per 8 weeks)	Biological: Reb blood cell transfusion; According to the product label
Cohort 9; Participants with high red-blood cell (RBC) transfusion dependance (≥6 RBC per 8 weeks)	Biological: Reb blood cell transfusion; According to the product label
Cohort 10; Participants aged <75 years	Drug: Erythropoiesis-stimulating agents (ESAs); According to the product label; Biological: Luspatercept; According to the product label; Drug: Lenalidomide; According to the product label; Drug: Hypomethylating agents (HMAs); According to the product label; Biological: Reb blood cell transfusion; According to the product label
Cohort 11; Participants aged ≥75 years	Drug: Erythropoiesis-stimulating agents (ESAs); According to the product label; Biological: Luspatercept; According to the product label; Drug: Lenalidomide; According to the product label; Drug: Hypomethylating agents (HMAs); According to the product label; Biological: Reb blood cell transfusion; According to the product label

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of comorbidities		Baseline
Participant age		Baseline
Participant sex		Baseline
Proportion of participants receiving each first-line treatment category	Treatment categories include: Erythropoiesis-stimulating agents (ESA); Luspateracept; Hypomethylating agents (HMAs); Lenalidomide; Conservative management (red-blood cell (RBC) transfusions without disease modifying therapy)	Up to 5-years
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
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
Smoking status		Baseline

Collaborators and Investigators

• Sponsor: Bristol-Myers Squibb
• Investigators: Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

Helpful Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

Study record dates

Study Major Dates

• Study Start (Actual): February 2, 2026
• Primary Completion (Estimated): March 31, 2026
• Study Completion (Estimated): May 31, 2026

Study Registration Dates

• First Submitted: February 24, 2026
• First Submitted That Met QC Criteria: March 9, 2026
• First Posted (Actual): March 11, 2026

Study Record Updates

• Last Update Posted (Actual): March 11, 2026
• Last Update Submitted That Met QC Criteria: March 9, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: Transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)
• Additional Relevant MeSH Terms: Organic Chemicals; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Pharmacologic Actions; Chemical Actions and Uses; Therapeutic Uses; Carboxylic Acids; Piperidines; Phthalimides; Phthalic Acids; Acids, Carbocyclic; Piperidones; Isoindoles; Hematologic Agents; Lenalidomide; Hematinics; luspatercept
• Other Study ID Numbers: CA056-1172

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No