This is a multicenter, open-label, prospective one arm study to explore the efficacy and safety of Avatrombopag in non-severe aplastic anemia. Patients meeting the inclusion and exclusion criteria would be recruited. Treatment of Avatrombopag would be started with 20mg/day. The dosage would be increased by 20 mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L. The dosage could range from 20mg/week to 60mg/day. All patients would receive treatment for at least 6 months except that the platelet \<20×10e9/L at the dosage of 60mg/day for 4 weeks or the platelet ≥200×10e9/L at the dosage of 20mg/week for 2 weeks. The hematological response rate and safety will be recorded and compared at every month after starting the study treatment. The patients would be followed up for at least 6 months.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
40
Avatrombopag 20mg/week to 60mg/day, starting at 20mg/day and increased by 20 mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L.
Peking Union Medical College Hospital
Beijing, China
ORR at 6 Months
Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) at 6 months
Time frame: 6 months
ORR at 3 Months
ORR will be calculated after 3 months of treatment by measuring platelet, reticulocyte, neutrophil and transfusion independence.
Time frame: 3 months
Percentage of patients with clonal evolution at 6 months
Percentage of patients with clonal evolution would be evaluated by bone marrow biopsy at 6 months follow up.
Time frame: 6 months
percentage of side effects at 6 months
percentage of side effects would be recorded during the study and be calculated according to CTCAE 5.0 at 6 months
Time frame: 6 months
Total volume of Platelet Transfusions
Total volume of Platelet Transfusions
Time frame: 1 month
Total volume of Red Blood Cells Transfusions
Total volume of Red Blood Cells Transfusions
Time frame: 1 month
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