This single-center study aims to evaluate the early efficacy and safety of avatrombopag combined with immunosuppressive therapy (IST) in the first-line treatment of severe aplastic anemia (SAA).
This is a single center, single arm, phase II clinical study. Fifty-three patients will be enrolled. Treatment protocol is as follows: 1) Anti-human thymocyte porcine immunoglobulin (P-ATG 20mg/kg/d) or rabbit anti human thymocyte globulin (R-ATG 3.0mg/kg/d) was administered intravenously for 5 days; 2) Cyclosporine (CSA) is given at 3-5 mg/kg.d in divided doses for at least 6 months. The trough concentration is maintained at 150-250 ng/ml. 3) Avatrombopag is given orally at 60 mg once a day for patients with body weight ≥ 50 kg, and 40 mg orally once a day for patients with body weight\<50 kg, for a total of 12 weeks.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
53
Patients with body weight ≥50kg were given 60mg/day and patients with body weight \< 50kg were given 40mg/day for 12 weeks.
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, China
RECRUITINGTreatment response
Percentage of patients who achieves complete response(CR) at 12 weeks.
Time frame: From the start of study treatment (Day1) to end of week 12.
Treatment response
Percentage of patients achieving hematologic response (OR) at 12 weeks.
Time frame: From the start of study treatment (Day1) to end of week 12.
Treatment response
Percentage of patients achieving hematologic response and complete response (OR and CR) at 24 weeks.
Time frame: From the start of study treatment (Day1) to end of week 24.
Supportive treatment
The time of red blood cell or platelet recovery to transfusion independence.
Time frame: From the start study treatment (Day1) up to transfusion independence.
Incidence of Treatment-Emergent Adverse Events by CTCAE
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: From the start study treatment (Day1) up to week 12.
Dose-effect relationship
Correlation between avatrombopag's serum concentration with total and complete hematological response rate.
Time frame: From the start study treatment(Day1) up to week 24.
Change of CD34+ cell
Change of CD34+ cells' proportion in bone marrow before and after avatrombopag treatment at week 12 and 24.
Time frame: From the start study treatment(Day1) up to end of week 12 and 24.
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