Thrombocytopenia
Conditions
Interventions
Treatment:Romiplostim N01: 500 µg administered subcutaneously once weekly
Sponsors
The Second Affiliated Hospital of Anhui Medical University
Eligibility
Sex/Gender
All
Age
18 Years to 75 Years
Inclusion criteria
Inclusion criteria: 1. Written informed consent prior to enrollment; 2. Age range: 18 to 75 years; 3. Diagnosis of MDS based on the 2016 WHO Classification of Myeloid Neoplasms; 4. Low- or intermediate-1-risk MDS as assessed by the International Prognostic Scoring System (IPSS) during screening; 5. Two consecutive platelet counts =30 x 10?/L within 1 week prior to enrollment, with at least 24 hours between measurements; 6. ECOG: 0-2; 7. Bone marrow biopsy and cytogenetic testing performed within 3 months after the first administration of the study product; 8. Reproductive-age participants agree to use reliable contraception throughout the study; 9. Patients deemed by the investigator to potentially benefit from the study.
Exclusion criteria
Exclusion criteria: 1. Presence of hematologic disorders other than MDS, such as primary immune thrombocytopenia, aplastic anemia, multiple myeloma, or leukemia. 2. IPSS intermediate-2 or high-risk classification. 3. Prior- treatment with thrombopoietin receptor agonists. 4. History of hematopoietic stem cell transplantation, leukemia, aplastic anemia, or non-MDS-related bone marrow stem cell disorders. 5. Active/uncontrolled conditions, including infections or cancer. 6. History of severe cardiovascular disease within 6 months before screening, such as congestive heart failure (NYHA Class III-IV), arrhythmias (e.g., atrial fibrillation), coronary artery stenting, angioplasty, or coronary artery bypass grafting. 7. History of arterial/venous thrombosis within 6 months before screening. 8. Known or suspected allergy/intolerance to romiplostim excipients. 9. HIV infection. 10. Pregnant or breastfeeding individuals. 11. Participation in other clinical trials involving investigational drugs or devices within 3 months before screening. 12. Significant health/safety risks or factors affecting efficacy assessment as determined by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of platelet response to treatment; | — |
Secondary
| Measure | Time frame |
|---|---|
| Incidence of clinically significant bleeding events;Time to reach peak platelet count;Duration of platelet treatment response;Neutrophil count;Number and total volume of platelet transfusions; | — |
Countries
China
Contacts
Public ContactZhimin Zhai
The Second Affiliated Hospital of Anhui Medical University
Outcome results
None listed