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

NCT06851065 | Completed FDA Drug

• 1 other identifier: CA056-1121
• Study Type: observational
• Target: 418
• Locations: 1 country
• Sites: 1

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

Keywords

Lower-risk myelodysplastic syndromes (LR-MDS)

Outcome Measures

Primary Outcomes (18)

• Participant baseline demographics

  Baseline

• Participant baseline clinical characteristics

  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

Study Arms (2)

Participants receiving first-line luspatercept treatment

Drug: Luspatercept

Participants receiving first-line erythropoiesis-stimulating agents

Drug: Erythropoiesis-stimulating agents

Interventions

Luspatercept DRUG

As per product lable

Participants receiving first-line luspatercept treatment

Erythropoiesis-stimulating agents DRUG

As per product label

Participants receiving first-line erythropoiesis-stimulating agents

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 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

You may qualify if:

• 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
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (1)

RTI Health Solutions

Raleigh, North Carolina, 27709-2194, United States

Location

Related Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

luspatercept; Hematinics

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Hematologic Agents; Therapeutic Uses; Pharmacologic Actions; Chemical Actions and Uses

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, 2025
• First Posted: February 28, 2025
• Study Start: August 22, 2024
• Primary Completion: March 10, 2025
• Study Completion: March 10, 2025
• Last Updated: February 17, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)