A Study to Evaluate Luspatercept Utilization in Patients With Lower Risk Myelodysplastic Syndromes or Beta-thalassemia

Real World Use of Luspatercept in Lower Risk Myelodysplastic Syndromes and Beta-thalassemia Patients

The purpose of this study is to understand the patient profile, utilized treatments, treatment patterns, resulting outcomes, and associated costs relating to luspatercept treatment in patients with lower-risk myelodysplastic syndromes (LR-MDS) or beta-thalassemia in the real-world setting

Study Overview

• Status: Terminated
• Conditions: Myelodysplastic Syndromes; Beta-thalassemia
• Intervention / Treatment: Drug: Luspatercept

• Study Type: Observational
• Enrollment (Actual): 30

Contacts and Locations

Study Locations

• Germany
  • Berlin, Germany
    • ZEG Berlin, Center for Epidemiology and Health Research

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 adult patients diagnosed with myelodysplastic syndromes or beta-thalassemia and treated with luspatercept between January 1, 2020, to June 30, 2022, identified from a German Statutory Health Insurance database

Description

Inclusion Criteria:

• Participants treated with luspatercept. The date of first treatment with luspatercept will be assigned as the index date.
• At least one Lower-risk myelodysplastic syndromes (LR-MDS) or beta-thalassemia diagnosis in inpatient or at least two MDS or beta-thalassemia diagnosis in the outpatient setting at least 30 days apart prior to or at index date.
• 18+ years old at index date.
• Participants with a minimum of 12-months observable pre-treatment baseline/look-back prior to index date.
• Participants with a minimum of 6-months of follow-up time from the index date unless the patient is lost due to death

Exclusion Criteria:

• None

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Cohort 1; Participants diagnosed myelodysplastic syndromes receiving luspatercept treatment	Drug: Luspatercept; According to the product label
Cohort 2; Participants diagnosed beta-thalassemia receiving luspatercept treatment	Drug: Luspatercept; According to the product label

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Participant age		Baseline
Participant comorbidities		Baseline
Participant gender		Baseline
Year of first luspatercept prescription		Baseline
Number of participants by Ring Sideroblast (RS) status (positive/negative/unknown) as measured by bone marrow aspiration	As documented in participant medical charts	Baseline
Length of follow-up from start of luspatercept treatment initiation until the earliest of the end of continuous enrolment, death, or end of data availability		Baseline

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants that achieve red blood cell (RBC) transfusion independence	Number of participants in Cohort 1 achieving RBC transfusion independence, defined as no RBC transfusions during any consecutive 56-day (8-week) or 84-day (12-week) period within the first 168 days (24 weeks) after the index date, as assessed from transfusion records and medical chart review.	Up to 12 months
Total number of RBC units transfused per participant, as assessed from transfusion records and medical chart review, from baseline up to 6 months		Baseline and up to 6 months
Time to acute myeloid leukemia transformation		Up to 6 months
Time to treatment discontinuation		Up to 6 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): November 16, 2023
• Primary Completion (Actual): May 1, 2025
• Study Completion (Actual): May 1, 2025

Study Registration Dates

• First Submitted: October 10, 2025
• First Submitted That Met QC Criteria: June 26, 2026
• First Posted (Actual): July 2, 2026

Study Record Updates

• Last Update Posted (Actual): July 2, 2026
• Last Update Submitted That Met QC Criteria: June 26, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: Beta-thalassemia; Lower-risk myelodysplastic syndromes (LR-MDS)
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Bone Marrow Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Thalassemia; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; beta-Thalassemia; luspatercept
• Other Study ID Numbers: CA056-1082

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.: No