To evaluate the efficacy and safety of Romiplostim N01 for platelet recovery after umbilical cord blood transplantation (UCBT) in patients with hematological malignancies.
Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) is an important means of curing a wide range of hematologic diseases, and good hematopoietic and immune reconstitution after transplantation is one of the most important factors determining the success of the transplantation.Persistent thrombocytopenia after Allo-HSCT is one of the common post-transplantation complications.This is an investigator-initiated, single-center, single-arm, Phase II clinical trial to evaluate the efficacy and safety of Romiplostim N01 for platelet recovery after UCBT in patients with hematologic tumors. The study was conducted in patients ≥18 years of age with a diagnosis of hematological neoplasia who underwent UCBT. Subjects will be screened and receive roprostin N01 from +1 to +28 days after UCBT after signing an informed consent form, and the study will focus on the cumulative PLT implantation rate at 28 days after transplantation as the primary efficacy measure. The study is planned to enroll 34 patients.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
34
Romiplostim N01 was administered subcutaneously once weekly from +1 day to +28 days after UCBT at a recommended starting dose of 5µg/Kg (maximum single dose not to exceed 250µg) for a total of 4 administrations, with the option of continuation of dosing/switching/discontinuing after +28 days, depending on the patient's platelet profile.
The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
Hefei, Anhui, China
RECRUITINGTime of platelet engraftment
Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10\^9/L.
Time frame: 28 days
The cumulative incidence of platelet engraftment at 28 days after transplantation
Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10\^9/L.
Time frame: 28 days
The cumulative incidence of platelet recovery rate at 28 days after transplantation
Platelet recovery was defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥50×10\^9/L.
Time frame: 28 days
Time of platelet recovery
The Median time of platelet count ≥20×10\^9/L, 50×10\^9/L, 100×10\^9/L without platelet transfusions
Time frame: 28 days
Total platelet transfusion during 4 weeks of treatment
Total platelet transfusion during 4 weeks of treatment
Time frame: 28 days
Cumulative implantation rate of the granulocyte lineage at 28 days post-transplantation
Absolute neutrophil count (ANC) ≥0.5×10\^9/L for 3 consecutive days
Time frame: 28 days
Megakaryocyte levels in bone marrow smears 4 weeks after treatment
Megakaryocyte levels in bone marrow smears 4 weeks after treatment
Time frame: 4 weeks
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