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

NCT07465029 | Active Not Recruiting FDA Drug

• 1 other identifier: CA056-1172
• Study Type: observational
• Target: 1,300
• Locations: 1 country
• Sites: 1

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

Conditions

Lower-risk Myelodysplastic Syndromes (TD LR-MDS)

Keywords

Transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (23)

• 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

Study Arms (11)

Cohort 1

Overall cohort of participants with transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)

Drug: Erythropoiesis-stimulating agents (ESAs); Biological: Luspatercept; Drug: Lenalidomide; Drug: Hypomethylating agents (HMAs); Biological: Reb blood cell transfusion

Cohort 2

Participants that initiated first-line treatment with erythropoiesis-stimulating agents (ESAs)

Drug: Erythropoiesis-stimulating agents (ESAs)

Cohort 3

Participants that initiated first-line treatment with luspatercept

Biological: Luspatercept

Cohort 4

Participants that initiated first-line treatment with hypomethylating agents (HMAs)

Drug: Hypomethylating agents (HMAs)

Cohort 5

Participants that initiated first-line treatment with lenalidomide

Drug: Lenalidomide

Cohort 6

Participants that receive conservative management (red blood cell transfusions without disease modifying therapy)

Biological: Reb blood cell transfusion

Cohort 7

Participants with low red-blood cell (RBC) transfusion dependance (\<4 RBC per 8 weeks)

Biological: Reb blood cell transfusion

Cohort 8

Participants with moderate red-blood cell (RBC) transfusion dependance (4-5 RBC per 8 weeks)

Biological: Reb blood cell transfusion

Cohort 9

Participants with high red-blood cell (RBC) transfusion dependance (≥6 RBC per 8 weeks)

Biological: Reb blood cell transfusion

Cohort 10

Participants aged \<75 years

Drug: Erythropoiesis-stimulating agents (ESAs); Biological: Luspatercept; Drug: Lenalidomide; Drug: Hypomethylating agents (HMAs); Biological: Reb blood cell transfusion

Cohort 11

Participants aged ≥75 years

Drug: Erythropoiesis-stimulating agents (ESAs); Biological: Luspatercept; Drug: Lenalidomide; Drug: Hypomethylating agents (HMAs); Biological: Reb blood cell transfusion

Interventions

Erythropoiesis-stimulating agents (ESAs) DRUG

According to the product label

Cohort 1; Cohort 10; Cohort 11; Cohort 2

Luspatercept BIOLOGICAL

According to the product label

Cohort 1; Cohort 10; Cohort 11; Cohort 3

Lenalidomide DRUG

According to the product label

Cohort 1; Cohort 10; Cohort 11; Cohort 5

Hypomethylating agents (HMAs) DRUG

According to the product label

Cohort 1; Cohort 10; Cohort 11; Cohort 4

Reb blood cell transfusion BIOLOGICAL

According to the product label

Cohort 1; Cohort 10; Cohort 11; Cohort 6; Cohort 7; Cohort 8; Cohort 9

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (1)

Local Institution - 0001

Madrid, 28001, Spain

Location

Related Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

MeSH Terms

Interventions

Hematinics; luspatercept; Lenalidomide

Intervention Hierarchy (Ancestors)

Hematologic Agents; Therapeutic Uses; Pharmacologic Actions; Chemical Actions and Uses; Phthalimides; Phthalic Acids; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidones; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Isoindoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 24, 2026
• First Posted: March 11, 2026
• Study Start: February 2, 2026
• Primary Completion: April 23, 2026
• Study Completion (Estimated): November 27, 2026
• Last Updated: October 1, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)