This is a prospective, single-center, randomized controlled, investigator-initiated clinical study. The purpose of this study is to evaluate the efficacy and safety of Romiplostim N01 for Injection in promoting platelet reconstitution after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Eligible participants will be randomly assigned in a 2:1 ratio to receive either Romiplostim N01 for Injection (experimental group) or the same treatment with the exception of Romiplostim N01 for Injection (control group).In the experimental group, Romiplostim N01 for Injection will be administered subcutaneously once weekly starting from Day 1 after allo-HSCT, with an initial dose of 3.0 μg/kg and dose titration based on platelet counts, up to a maximum of 10 μg/kg once weekly. Treatment will continue until platelet count exceeds 20×10⁹/L for 7 consecutive days without platelet transfusion, or until lack of efficacy after 4 weeks at 10 μg/kg, or at the investigator's discretion. Participants will enter a safety follow-up period for 28 days after treatment completion, with visits as frequently as weekly to collect adverse events, concomitant medications, and supportive care information. The primary efficacy endpoint is the time to platelet engraftment, defined as the first day of platelet count ≥20×10⁹/L for 7 consecutive days without platelet transfusion.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
87
Subcutaneous injection once weekly, initiated on Day +1 after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The starting dose is 3.0 μg/kg. The dose will be titrated according to peripheral platelet counts up to a maximum weekly dose of 10 μg/kg. Treatment will be discontinued when any of the following criteria are met: platelet count ≥20×10⁹/L sustained for 7 consecutive days without platelet transfusion; inadequate platelet response after 4 consecutive weeks at the maximum dose of 10 μg/kg; or discontinuation at the investigator's clinical discretion.
The First Affiliated Hospital of Henan University of Science and Technology
Luoyang, Henan, China
Platelet engraftment time
the first day of platelet count ≥20×10⁹/L sustained for 7 consecutive days in the absence of platelet transfusion
Time frame: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Neutrophil engraftment time
the first day when absolute neutrophil count (ANC) ≥0.5×10⁹/L sustained for 3 consecutive days in the absence of G-CSF support
Time frame: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Median time to achieve complete remission (CR)
Defined as the median time to achieve complete remission (CR), where complete remission is defined as platelet count ≥50×10⁹/L sustained for 7 consecutive days without platelet transfusion.
Time frame: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence of primary poor graft function (PGF)
Defined as the incidence of primary poor graft function, a condition occurring within 28 days after transplantation, characterized by full donor chimerism, absence of severe GVHD or disease relapse, successful myeloid and erythroid engraftment (absolute neutrophil count \>0.5×10⁹/L and hemoglobin \>70 g/L), with sustained platelet count ≤20×10⁹/L.
Time frame: Within 28 days after allo-HSCT
Incidence of secondary platelet recovery failure
Incidence of secondary platelet recovery failure (defined as a decline in platelet count to \<20,000/μL after achievement of initial platelet recovery, lasting for at least 7 days or requiring platelet transfusion within 7 days)
Time frame: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence of graft-versus-host disease
Acute GVHD is graded according to the Glucksberg grading criteria; chronic GVHD is diagnosed and graded based on the National Institutes of Health (NIH) consensus criteria and Chinese expert consensus.
Time frame: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence of primary graft failure
① For peripheral blood stem cells (PBS) or non-mobilized bone marrow cells: ANC \< 0.5×10⁹/L accompanied by pancytopenia on Day 30 after infusion. ② For umbilical cord blood (UCB): ANC \< 0.5×10⁹/L accompanied by pancytopenia on Day 42 after infusion.
Time frame: At Day 30 or Day 42 after cell infusion
Incidence of secondary graft failure
deterioration of hematopoietic function (involving hemoglobin and/or platelets and/or neutrophils) after achievement of hematopoietic reconstitution (including hemoglobin, platelets and/or neutrophils), requiring blood product transfusion or growth factor support.
Time frame: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Comparison of treatment-related costs between Romiplostim N01 group and control group
Comparison of treatment-related costs between the Romiplostim N01 group and the control group.
Time frame: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Platelet transfusion requirements during engraftment
Total number of platelet transfusions administered during the platelet engraftment period.
Time frame: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence and severity of adverse events (AEs) and adverse drug reactions (ADRs)
Adverse events and adverse drug reactions will be recorded and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time frame: From treatment initiation to 28 days after treatment completion
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