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NCT Number: NCT07465029

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

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Local Institution - 0001

Madrid, 28001, Spain

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Erythropoiesis-stimulating agents (ESAs)

Drug

According to the product label

Luspatercept

Biological

According to the product label

Lenalidomide

Drug

According to the product label

Hypomethylating agents (HMAs)

Drug

According to the product label

Reb blood cell transfusion

Biological

According to the product label

Primary outcomes

  1. Incidence of transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)

    Time frame: Up to 5-years

  2. Prevalence of transfusion dependent lower-risk myelodysplastic syndromes (TD LR-MDS)

    Time frame: Up to 5-years

Secondary outcomes

  1. Proportion of participants receiving each first-line treatment category

    Time frame: 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)
  2. Proportion of participants by treatment category at each line of therapy

    Time frame: 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.

  3. Treatment duration (time from initiation to discontinuation) of first-line treatment

    Time frame: Up to 5-years

  4. Defined as number of RBC units received per participant per 8-weeks

    Time frame: Up to 5-years

  5. Number and Rate of Healthcare Resource Utilization Events

    Time frame: 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.

  6. Number of participants that achieve hematologic improvement-erythroid (HI-E)

    Time frame: Up to 5-years

  7. Number of participants that achieve red-blood cell (RBC) transfusion independence (TI)

    Time frame: Up to 5-years

  8. Number of participants that experience a change in transfusion burden (change in number of red-blood cell units received per 8-weeks)

    Time frame: Up to 5-years

  9. Number of participants that progress to higher-risk myelodysplastic syndromes (MDS)

    Time frame: Up to 5-years

  10. Number of participants that progress to acute myeloid leukemia (AML)

    Time frame: Up to 5-years

  11. Number of participants that do not respond to first-line treatment

    Time frame: Up to 5-years

  12. Number of participants that experience loss of response/secondary failure after an initial hematologic response

    Time frame: Up to 5-years

  13. Cause of death

    Time frame: Up to 5-years

  14. Serious adverse events (SAEs)

    Time frame: Up to 5-years

  15. Number of serious cardiovascular events requiring emergency room visit or hospitalization

    Time frame: 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.

  16. Participant age

    Time frame: Baseline

  17. Participant sex

    Time frame: Baseline

  18. Year of first-line treatment initiation

    Time frame: Baseline

  19. Participant Body mass index (BMI)

    Time frame: Baseline

  20. Participant Charlson Comorbidity Index (CCI) score

    Time frame: Baseline

  21. Participant Charlson Comorbidity Index (CCI) individual comorbidities

    Time frame: Baseline

  22. Number of comorbidities

    Time frame: Baseline

  23. Smoking status

    Time frame: Baseline

Sponsors and collaborators

Lead sponsor

Bristol-Myers Squibb

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2026
Study completion
2026
First posted
Mar 11, 2026
Registry last updated
Mar 11, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.