To evaluate the efficacy and safety of Avatrombopag for platelet recovery after unrelated cord blood transplantation (UCBT) in patients with bone marrow failure disease
Persistent thrombocytopenia is a common complication after umbilical cord blood transplantation (UCBT), which necessitates platelet transfusion and leads to increased transplant-related mortality. This is a single-center, single-arm, phase II clinical trial aimed at evaluating the efficacy and safety of Avatrombopag for platelet recovery after UCBT in patients with bone marrow failure disease (BMFD). The study was conducted in patients diagnosed with BFMD and receiving UCBT. After the subjects signed and informed and qualified, they received Avatrombopag treatment from the first day to the 28th day after transplantation. In this study, the cumulative of platelet implantation rate at 28 days after transplantation was used as the main therapeutic index, and 40 patients were planned to be enrolled.
Study Type
OBSERVATIONAL
Enrollment
40
Indications for drug reduction and withdrawal: excluding blood transfusion factors, the reduction will be 20mg/d when PLT≥50×10\^9/L; Drug administration will be stopped when PLT≥100×10\^9/L.
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
Total platelet transfusion during 4 weeks of treatment
Total platelet transfusion during 4 weeks of treatment
Time frame: 28 days
neutrophil reconstitution
neutrophil engraftment time was defined as the first of three consecutive days during which the neutrophil count was at least 0.5×10\^9/L.
Time frame: 28 days
erythroid reconstitution
RBC engraftment time was defined as the first day of achieving a reticulocyte count greater than 1% 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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Time of platelet recovery
Platelet count ≥50×10\^9/L for 3 consecutive days without platelet transfusions for 7 consecutive days
Time frame: 100 days
Time of platelet ≥100×10^9/L.
Platelet count ≥100×10\^9/L for 3 consecutive days without platelet transfusions for 7 consecutive days
Time frame: 100 days