Beta-thalassemia, Myelodysplastic Syndromes
Conditions
Keywords
Lower-risk myelodysplastic syndromes (LR-MDS), Beta-thalassemia
Brief summary
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
Interventions
DRUGLuspatercept
According to the product label
Sponsors
Bristol-Myers Squibb
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Participant age | 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 | Baseline | As documented in participant medical charts |
| Participant comorbidities | 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
| Measure | Time frame | Description |
|---|---|---|
| Number of participants that achieve red blood cell (RBC) transfusion independence | Up to 12 months | 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. |
| 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 | — |
Countries
Germany
Contacts
STUDY_DIRECTORBristol Myers Squibb
Bristol-Myers Squibb
Outcome results
None listed