First-Line Luspatercept in Transfusion-Dependent Lower-Risk Myelodysplastic Neoplasms
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Luspatercept.
- Who it may be relevant to
- Registry conditions: Myelodysplastic Neoplasms. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Germany
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Prospective, Multicenter, Non-Interventional Study Assessing First-line Luspatercept in Anemic Patients With Very Low, Low, or Intermediate Risk Myelodysplastic Neoplasms Requiring Red Blood Cell Transfusions
Overview
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
- Drug Luspatercept
As per product label
Primary outcome measures
- Percentage of participants achieving red blood cell transfusion independence (RBC-TI) for at least 8 consecutive weeks [Time frame: Up to Week 24]
Secondary outcome measures (12)
- Percentage of participants achieving red blood cell transfusion independence for at least 12 consecutive weeks [Time frame: Up to Week 48]
- Percentage of participants achieving red blood cell transfusion independence for at least 16 consecutive weeks [Time frame: Up to Week 48]
- Mean change from baseline in hemoglobin concentration [Time frame: Day 1 through Week 24, Week 48, End of Treatment (up to 2-years), and End of Study (up to 2-years)]
- Percentage of participants with hemoglobin increase of at least 1.5 g/dl from baseline [Time frame: Day 1 through Week 24, Week 48, End of Treatment (up to 2-years), and End of Study (up to 2-years)]
- Percentage of participants achieving >50% reduction in transfusion burden compared with baseline [Time frame: 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. [Time frame: Up to 2-years]
- Time to red blood cell transfusion independence for at least 8 consecutive weeks [Time frame: 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. [Time frame: 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. [Time frame: 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. [Time frame: Up to 2-years]
- Percentage of participants achieving hematologic improvement-erythroid response according to International Working Group (IWG) 2006 criteria [Time frame: Up to Week 48]
- Number of participants with adverse events [Time frame: Up to 2-years]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Germany · 1 center
- Universitätsmedizin der Johannes Gutenberg-Univ. III. Med. Klinik Hämatologie/Onkologie — Mainz
Identifiers
NCT: NCT07752121 · CA056-1175