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

Active, not recruiting

• Conditions: Myelodysplastic Syndromes
• Interventions: Drug: Luspatercept; Drug: Erythropoiesis-stimulating agents

• Registration Number: NCT06851065
• Lead Sponsor: Bristol-Myers Squibb

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

Detailed Description

Not available

Study Timeline

• Study Start: 2024-08-22
• Status Verified: 2025-02
• Study First Submitted: 2025-02-24
• Study First Submitted QC: 2025-02-24
• Last Update Submitted: 2025-02-24
• Study First Posted: 2025-02-28
• Last Update Posted: 2025-02-28
• Primary Completion: 2025-03-31
• Study Completion: 2025-06-30

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 400

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Participants receiving first-line luspatercept treatment	Luspatercept	-
Participants receiving first-line erythropoiesis-stimulating agents	Erythropoiesis-stimulating agents	-

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

RTI Health Solutions; 🇺🇸 Raleigh, North Carolina, United States