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Completed

NCT Number: NCT06851065

A Study to Evaluate Luspatercept Treatment Patterns and Outcomes in Erythropoiesis-Stimulating Agents-Naïve Patients With Lower-Risk Myelodysplastic Syndromes in the United States

The purpose of this study is to understand real-world effectiveness of luspatercept treatment among erythropoiesis-stimulating agents -naïve patients with lower-risk- myelodysplastic syndromes in the United States

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Key information

Conditions

Myelodysplastic Syndromes Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

RTI Health Solutions

Raleigh, North Carolina, 27709-2194, United States

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Had a documented diagnosis of primary or secondary myelodysplastic syndromes (MDS)
  • MDS diagnosis confirmed through bone marrow testing on (or 30 days prior to) MDS diagnosis date or within 1 year of MDS diagnosis date
  • Had a documented determination of Lower Risk (LR)-MDS as measured by International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) at or before index treatment (i.e., first-line luspatercept or first-line erythropoiesis-stimulating agents (ESA)) initiation
  • IPSS risk level: low, intermediate-1 (level-1 risk)
  • IPSS-R risk level: very low, low, intermediate
  • Received luspatercept as the first-line treatment for anemia any time from 28 August 2023 to 31 July 2024 (Cohort 1)
  • Receipt of combination therapy with ESAs and/or granulocyte colony-stimulating factors (G-CSFs) will be allowed

OR

  • Received ESA as the first-line treatment for anemia any time from 28 August 2023 to 31 July 2024 (Cohort 2)
  • Was aged 18 years or older at the time of initial diagnosis of MDS
  • Known vital status (i.e., living, or deceased) at the time of record abstraction.
  • Records for patients who are dead or alive will be eligible
  • Complete medical record covering relevant past medical history, diagnosis of LR-MDS, treatment, laboratory assessments, red-blood cell (RBC) transfusions, and regular monitoring for LR-MDS, including any transfer record from other physicians/facilities (if applicable) is available to the abstracting physician for data abstraction

Exclusion criteria

  • Had a history of acute myeloid leukemia (AML) prior to MDS diagnosis
  • Received previous treatment with hypomethylating agents, disease-modifying agents (including lenalidomide), other immunosuppressants/immunomodulatory agents, or other MDS-directed chemotherapy
  • Received stem cell transplant prior to index treatment initiation
  • Participated in a clinical trial for the treatment of MDS before or while on index treatment (i.e., clinical trial participation after first-line luspatercept or ESA treatment discontinuation will be allowed)
  • Had evidence of other malignant neoplasms in the 12 months prior to diagnosis of MDS, except basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, carcinoma in situ of the breast, or incidental histologic finding of prostate cancer (stage T1a or T1b)
  • Patients for whom this information is not available (i.e., "unknown") will be included in the study
  • For Cohort 1 (i.e., first-line luspatercept treatment), receipt of combination therapy with hypomethylating agents, lenalidomide, other immunosuppressants/ immunomodulatory agents, or other MDS-directed chemotherapy
  • For Cohort 2 (i.e., first-line ESA treatment), receipt of combination therapy with hypomethylating agents, lenalidomide, luspatercept, other immunosuppressants/ immunomodulatory agents, or other MDS-directed chemotherapy

Treatment and study plan

Luspatercept

Drug

As per product lable

Erythropoiesis-stimulating agents

Drug

As per product label

Primary outcomes

  1. Participant baseline demographics

    Time frame: Baseline

  2. Participant baseline clinical characteristics

    Time frame: Baseline

  3. Time from Lower Risk- myelodysplastic syndromes diagnosis to index treatment initiation

    Time frame: Baseline

  4. Rationale for therapy selection

    Time frame: Baseline

  5. Duration of index treatment

    Time frame: Up to 15 months

  6. Treatment dose at treatment initiation and discontinuation

    Time frame: Up to 6 months

  7. Treatment dose/dosing schedule changes, and treatment interruptions

    Time frame: Up to 12 months

  8. Other supportive care therapies prescribed while on index treatment

    Time frame: Up to 15 months

  9. Treatments prescribed post index treatment

    Time frame: Up to 15 months

  10. Treatments for anemia management received after discontinuing the index treatment

    Time frame: Up to 15 months

  11. Receipt of stem cell transplant at any time post index treatment

    Time frame: Up to 15 months

  12. Participant red-blood cell (RBC) transfusion burden post index treatment

    Time frame: At 3-months, and up to 6 months

  13. Hematologic improvement-erythroid (HI-E) response post index treatment

    Time frame: At 3-months, and up to 6 months

  14. Progression to acute myeloid leukemia post index treatment

    Time frame: Up to 15 months

  15. Progression to high-risk myelodysplastic syndromes per the International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) criteria

    Time frame: Up to 15 months

  16. Participant adverse events during and post index treatment

    Time frame: Up to 15 months

  17. Overall survival (OS)

    Time frame: At 3-, 6-, 12-, and up to 15-months

  18. Healthcare resource utilization (HCRU) during index treatment

    Time frame: Up to 15 months

Sponsors and collaborators

Lead sponsor

Bristol-Myers Squibb

Industry

Registry information

Official study title

Luspatercept Treatment Patterns and Outcomes Among ESA-Naïve Patients With Lower-Risk MDS - A Retrospective Medical Record Review in the United States

Important dates

Study start
2024
Primary completion
2025
Study completion
2025
First posted
Feb 28, 2025
Registry last updated
Feb 17, 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.

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