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

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

Study Overview

• Status: Completed
• Conditions: Myelodysplastic Syndromes
• Intervention / Treatment: Drug: Luspatercept; Drug: Erythropoiesis-stimulating agents

• Study Type: Observational
• Enrollment (Actual): 418

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Raleigh, North Carolina, United States, 27709-2194
      • RTI Health Solutions

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 include adults in the United States with a clinician-confirmed diagnosis of primary or secondary lower-risk myelodysplastic syndromes (LR-MDS) who initiated first-line luspatercept treatment on or after LR-MDS diagnosis date between 28 August 2023 to 31 July 2024

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Participants receiving first-line luspatercept treatment	Drug: Luspatercept; As per product lable
Participants receiving first-line erythropoiesis-stimulating agents	Drug: Erythropoiesis-stimulating agents; As per product label

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Participant baseline clinical characteristics	Baseline
Participant baseline demographics	Baseline
Time from Lower Risk- myelodysplastic syndromes diagnosis to index treatment initiation	Baseline
Rationale for therapy selection	Baseline
Duration of index treatment	Up to 15 months
Treatment dose at treatment initiation and discontinuation	Up to 6 months
Treatment dose/dosing schedule changes, and treatment interruptions	Up to 12 months
Other supportive care therapies prescribed while on index treatment	Up to 15 months
Treatments prescribed post index treatment	Up to 15 months
Treatments for anemia management received after discontinuing the index treatment	Up to 15 months
Receipt of stem cell transplant at any time post index treatment	Up to 15 months
Participant red-blood cell (RBC) transfusion burden post index treatment	At 3-months, and up to 6 months
Hematologic improvement-erythroid (HI-E) response post index treatment	At 3-months, and up to 6 months
Progression to acute myeloid leukemia post index treatment	Up to 15 months
Progression to high-risk myelodysplastic syndromes per the International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) criteria	Up to 15 months
Participant adverse events during and post index treatment	Up to 15 months
Overall survival (OS)	At 3-, 6-, 12-, and up to 15-months
Healthcare resource utilization (HCRU) during index treatment	Up to 15 months

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): August 22, 2024
• Primary Completion (Actual): March 10, 2025
• Study Completion (Actual): March 10, 2025

Study Registration Dates

• First Submitted: February 24, 2025
• First Submitted That Met QC Criteria: February 24, 2025
• First Posted (Actual): February 28, 2025

Study Record Updates

• Last Update Posted (Actual): February 17, 2026
• Last Update Submitted That Met QC Criteria: February 13, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: Lower-risk myelodysplastic syndromes (LR-MDS)
• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Pharmacologic Actions; Chemical Actions and Uses; Therapeutic Uses; Hematologic Agents; Hematinics; luspatercept
• Other Study ID Numbers: CA056-1121

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
• product manufactured in and exported from the U.S.: Yes