Myelodysplastic Neoplasms
Conditions
Keywords
Myelodysplastic Neoplasms, Myelodysplastic Syndromes, MDS, Lower-Risk MDS, RBC Transfusion Dependent, Anemia, Luspatercept, Reblozyl
Brief summary
This study will observe adults with lower-risk myelodysplastic neoplasms (MDS) who have anemia requiring regular red blood cell transfusions and who are prescribed first-line luspatercept as part of routine medical care. The study will follow participants for up to 2 years to understand how often treatment leads to periods without transfusions, changes in hemoglobin levels, health-related quality of life, and safety outcomes. Information on treatment use and outcomes in routine clinical practice in Germany will also be collected.
Interventions
DRUGLuspatercept
As per product label
Sponsors
Bristol-Myers Squibb
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Males and females ≥18 years of age at enrollment. * Documented diagnosis of myelodysplastic neoplasms according to World Health Organisation (WHO) 2022 or WHO 2016 classification meeting International Prognostic Scoring System-Revised (IPSS-R) criteria for very low-, low-, or intermediate-risk disease. * Documented red blood cell transfusion dependence of ≥2 units of red blood cells within the 8 weeks preceding Day 1 treatment initiation. * First-line treatment based on the approved luspatercept label and decision for treatment with luspatercept as assessed by the treating physician prior to study participation * Provision of written informed consent.
Exclusion criteria
* Contraindication according to the Reblozyl® (luspatercept) Summary of Product Characteristics (SmPC). * Parallel participation in an interventional clinical trial (except follow-up phase as specified in protocol). Patients who have completed their participation in an interventional clinical trial or who are not receiving any study drug anymore and who are only in the follow-up phase can be enrolled. For blinded studies, the study drug administered needs to be known at the time of enrolment. • Concurrent malignancy requiring treatment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percentage of participants achieving red blood cell transfusion independence (RBC-TI) for at least 8 consecutive weeks | Up to Week 24 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of participants achieving red blood cell transfusion independence for at least 12 consecutive weeks | Up to Week 48 | — |
| Percentage of participants achieving red blood cell transfusion independence for at least 16 consecutive weeks | Up to Week 48 | — |
| Mean change from baseline in hemoglobin concentration | Day 1 through Week 24, Week 48, End of Treatment (up to 2-years), and End of Study (up to 2-years) | Hemoglobin concentration (g/dL) measured during routine clinical practice. |
| Percentage of participants with hemoglobin increase of at least 1.5 g/dl from baseline | Day 1 through Week 24, Week 48, End of Treatment (up to 2-years), and End of Study (up to 2-years) | Hemoglobin concentration (g/dL) measured during routine clinical practice. |
| Percentage of participants achieving >50% reduction in transfusion burden compared with baseline | Day 1 through Week 24, Week 48, End of Treatment (up to 2-years), and End of Study (up to 2-years) | — |
| Time from first luspatercept administration to first on-treatment red blood cell transfusion. | Up to 2-years | — |
| Time to red blood cell transfusion independence for at least 8 consecutive weeks | Up to Week 24 | — |
| Duration of red blood cell transfusion independence (RBC-TI) among participants who achieve an RBC transfusion-free period lasting at least 56 consecutive days. | Up to 2-years | — |
| Duration of red blood cell transfusion independence (RBC-TI) among participants who achieve an RBC transfusion-free period lasting at least 84 consecutive days. | Up to 2-years | — |
| Duration of red blood cell transfusion independence (RBC-TI) among participants who achieve an RBC transfusion-free period lasting at least 112 consecutive days. | Up to 2-years | — |
| Percentage of participants achieving hematologic improvement-erythroid response according to International Working Group (IWG) 2006 criteria | Up to Week 48 | Hematologic improvement-erythroid (HI-E) response is defined as an increase in hemoglobin of at least 1.5 g/dL and/or a reduction of at least 4 red blood cell transfusions during an 8-week period compared with the 8 weeks before treatment, sustained over any consecutive 56-day period |
| Number of participants with adverse events | Up to 2-years | — |
| Change from baseline in health-related quality of life assessed by European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) | Day 1, Week 6, Week 12, Week 18, Week 24, Week 36, Week 48, and End of Treatment (up to 2-years) | — |
| Change from baseline in health-related quality of life assessed by Quality of Life in Myelodysplasia Scale (QUALMS) | Day 1, Week 6, Week 12, Week 18, Week 24, Week 36, Week 48, and End of Treatment (up to 2-years) | — |
Countries
Germany
Contacts
CONTACTBMS Clinical Trials Contact Center www.BMSClinicalTrials.com
CONTACTFirst line of the email MUST contain NCT # and Site #.
STUDY_DIRECTORBristol-Myers Squibb
Bristol-Myers Squibb
Outcome results
None listed