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.
This is a prospective, single-center, randomized, open-label, parallel-controlled Phase II clinical trial. The primary objective is to provide a preliminary efficacy estimate and evaluate the safety of romiplostim compared to recombinant human thrombopoietin (rhTPO) for promoting platelet engraftment following allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with myelodysplastic syndrome (MDS) or aplastic anemia (AA). Study Design and Population: The study will enroll approximately 74 patients to achieve 66 evaluable subjects, randomized in a 1:1 ratio to the romiplostim group or the rhTPO group. Randomization will be stratified by the underlying disease (MDS vs. AA). Eligible patients are aged 18-65 years, diagnosed with MDS or severe/very severe AA (SAA/VSAA), and are candidates for allo-HSCT from a matched sibling, haploidentical, or unrelated donor (including cord blood). Key inclusion criteria require a platelet count \<20×10⁹/L with transfusion dependence between day +4 and day +10 post-transplant. Major exclusion criteria include uncontrolled active infection, significant history of thrombosis, active transplant-associated thrombotic microangiopathy (TA-TMA), pre-transplant bone marrow fibrosis ≥ MF-2 grade, and known allergy to the study drugs. Interventions: Experimental Group (n≈33): Romiplostim administered subcutaneously at a starting dose of 5.0 µg/kg once weekly, beginning on transplant day +4. Subsequent doses (from day +18) will be adjusted weekly based on platelet counts. Active Control Group (n≈33): rhTPO administered subcutaneously at a fixed dose of 15000 U once daily, beginning on transplant day +4. Treatment for both groups continues until the platelet count is ≥50×10⁹/L without transfusion support for 7 consecutive days, until day +60 post-transplant, or for a maximum of 8 weeks, whichever occurs first. All patients will receive standardized transplant supportive care. Endpoints: Primary Endpoints: Platelet engraftment time, defined as the first of three consecutive days with a platelet count ≥20×10⁹/L without platelet transfusion in the preceding 7 days. Incidence of ≥ Grade 3 adverse events (AEs) within 100 days post-transplant, with specific focus on graft-versus-host disease (GVHD) and thrombotic events. Secondary Endpoints: Platelet engraftment rate at day 60 (≥50×10⁹/L), total platelet transfusion units from day 0 to 60, overall survival (OS), progression-free survival (PFS), and non-relapse mortality (NRM). Exploratory Endpoints: Immune reconstitution (lymphocyte subsets), megakaryocyte-related biomarkers, and a pharmacoeconomic evaluation. Oversight and Safety: An Independent Data Safety Monitoring Committee (DSMB) will be established to periodically review accumulated safety data. Safety will be actively monitored through weekly clinical and laboratory assessments during the treatment period, with specific focus on thrombotic events, GVHD, and potential clonal evolution. The study will be conducted at the Department of Hematology, The First Affiliated Hospital of Soochow University, China, following approval by its institutional Ethics Committee.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
66
Romiplostim 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 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.
The First Affiliated Hospital of Soochow University, Department of Hematology
Suzhou, Jiangsu, China
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).
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.
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.
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.
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.
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.
Non-Relapse Mortality (NRM)
Cumulative incidence of death from causes other than disease relapse/progression.
Time frame: From date of transplant until death from non-relapse causes, assessed up to 2 years.
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