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

NCT ID: NCT06851065

Last Updated: 2025-02-28

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Total Enrollment: 400 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2024-08-22
• Study Completion Date: 2025-06-30

Brief Summary

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

Conditions

Myelodysplastic Syndromes

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Study Groups

Participants receiving first-line luspatercept treatment

Luspatercept

Intervention Type: DRUG

As per product lable

Participants receiving first-line erythropoiesis-stimulating agents

Erythropoiesis-stimulating agents

Intervention Type: DRUG

As per product label

Interventions

Luspatercept

As per product lable

Intervention Type: DRUG

Erythropoiesis-stimulating agents

As per product label

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Bristol-Myers Squibb

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Bristol Myers Squibb

Role: STUDY_DIRECTOR

Bristol-Myers Squibb

Locations

RTI Health Solutions

Raleigh, North Carolina, United States

Countries

United States

Related Links

https://www.bms.com/researchers-and-partners/clinical-trials-and-research.html

BMS Clinical Trial Information

https://www.fda.gov/safety/recalls-market-withdrawals-safety-alerts

FDA Safety Alerts and Recalls

Other Identifiers

CA056-1121

Identifier Type: -

Identifier Source: org_study_id