Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorThe First Affiliated Hospital of Soochow University
About this trial
This study is for adults aged 18-65 with myelodysplastic syndrome (MDS) or severe aplastic anemia (AA) who are scheduled to receive a donor stem cell transplant (allogeneic hematopoietic stem cell transplant). After the transplant, it is critical for the body to start making its own blood cells again. A common and serious problem is a delay in the recovery of platelets (the cells that help stop bleeding), which increases the risk of bleeding, infection, and death.
This study aims to see if a new treatment can help platelets recover faster and more safely after transplant. We are comparing two drugs:
Romiplostim: A long-acting injection given just once a week. rhTPO (Recombinant Human Thrombopoietin): A standard injection given every day. Both drugs are designed to help the body make more platelets. The main question is whether the once-weekly romiplostim works as well or better than the daily rhTPO, and if it is safe.
About 66 patients will participate. By random chance (like flipping a coin), each participant will be assigned to receive either romiplostim or rhTPO. The treatment will start a few days after the transplant and continue until platelets recover to a safe level or for up to 8 weeks. Doctors will closely monitor all participants for 100 days to track platelet recovery, need for transfusions, side effects, and overall health.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age 18-65 years (inclusive).
Diagnosis of Myelodysplastic Syndrome (MDS) per WHO criteria, or Severe/Very Severe Aplastic Anemia (SAA/VSAA) per Camitta criteria, and deemed eligible for allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Planned to receive allo-HSCT from a matched sibling, haploidentical, or unrelated donor (including cord blood).
Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
Disqualifiers
Active, uncontrolled bacterial, fungal, or viral infection at the time of enrollment.
History of arterial thrombosis, or venous thromboembolism within the past 6 months (unless cured or stable for over 6 months).
Active transplant-associated thrombotic microangiopathy (TA-TMA).
Pre-transplant bone marrow biopsy showing fibrosis grade ≥ MF-2 (according to WHO criteria).
Trial design
Design model
Parallel
Treatments tested in this trial
Romiplostim
DrugRomiplostim is a thrombopoietin receptor agonist (TPO-RA) that stimulates platelet production. It is a fusion protein (peptibody) that binds to and activates the TPO receptor, promoting megakaryocyte proliferation and differentiation. In this study, it is administered as a subcutaneous injection once weekly.
Recombinant Human Thrombopoietin
DrugRecombinant human thrombopoietin is a cytokine that stimulates platelet production by binding to the TPO receptor on megakaryocytes. In this study, it is administered as a subcutaneous injection once daily.
Treatment groups
66 Participants
are divided into 2 treatment groupsGroup A: rhTPO (Recombinant Human Thrombopoietin)Active comparator 1 intervention
Group B: RomiplostimExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Rate of Platelet Engraftment by Day +60 (Platelets ≥50×10^9/L without transfusion for 7 consecutive days)
To preliminarily evaluate and compare the efficacy of romiplostim versus rhTPO in promoting platelet recovery after allo-HSCT. Engraftment is defined as achieving a platelet count ≥50×10\^9/L without transfusion support for 7 consecutive days.
Time frame
From the start of transplantation until Day 60 post-transplant (or until the engraftment criterion is met).2
Incidence of ≥ Grade 3 Adverse Events within 100 Days Post-Transplant (including GVHD and thrombotic events)
To assess and compare the safety profile between romiplostim and rhTPO, focusing on severe adverse events. Events of special interest include acute/chronic GVHD (graded per NIH criteria) and thrombotic events (e.g., TA-TMA, deep vein thrombosis).
Time frame
From the start of transplantation until Day 100 post-transplant.Secondary outcomes
1
60-Day Platelet Engraftment Rate
Proportion of participants achieving platelet engraftment (defined as platelet count ≥50×10⁹/L) by Day 60 post-transplant without platelet transfusion support.
Time frame
At Day 60 post-transplant.2
Platelet Transfusion Requirements (Days 0-60)
Total number of platelet transfusion units (including both prophylactic and therapeutic transfusions) administered from Day 0 (day of transplant) to Day 60 post-transplant.
Time frame
From Day 0 (day of transplant) to Day 60 post-transplant.3
Overall Survival (OS)
Time from the date of transplant until death from any cause. Participants alive at the end of follow-up will be censored.
Time frame
From date of transplant until death from any cause, assessed up to 2 years.4
Progression-Free Survival (PFS)
Time from the date of transplant until disease progression/relapse or death from any cause, whichever occurs first.
Time frame
From date of transplant until disease progression/relapse or death from any cause, assessed up to 2 years.Other outcomes
1
Immune reconstitution
Immune reconstitution (lymphocyte subsets, such as CD4+/CD8+ T cells, NK cells, B cell counts)
Time frame
At post-transplant day +30 and day +100 (±7 days)2
Megakaryocyte Count and Maturity in Bone Marrow
Assessment of megakaryocyte quantity and maturation stage in bone marrow post-transplant, as measured by flow cytometry for CD41+/CD61+ (total count) and CD41+/CD61+/CD36+ (maturity)
Time frame
At post-transplant day +14 (±3 days) and day +28 (±3 days)3
Serum Thrombopoietin (TPO) Level
Measurement of serum Thrombopoietin (TPO) concentration post-transplant.
Time frame
At post-transplant day +14 (±3 days) and day +28 (±3 days)4
From the start of transplantation (Day 0) until Day +100 post-transplant
Comparison of total direct medical costs between treatment groups, including costs of inpatient hospitalization and study drug administration (romiplostim or rhTPO). Costs will be calculated in Chinese Yuan (CNY) and reported as mean cost per participant.
Time frame
From the start of transplantation (Day 0) until Day +100 post-transplant.Sponsors and contacts
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Source ClinicalTrials.gov