This is a prospective, single-center, single-arm, phase 2 study. This study aims to evaluate the efficacy and safety of Anti-lymphocyte globulin plus herombopag in combination with moderate-dose cyclophosphamide for severe aplastic anemia.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
43
Severe aplastic anemia patients will receive a daily dose of Anti-lymphocyte globulin (25mg/kg) for the initial five days at the beginning of the treatment. Cyclosporine will be administered daily at a dosage of 3-5mg/kg. Herombopag will be administered daily at a dosage of 15mg starting from the first day of treatment and continuing for a duration of six months. The moderate-dose cyclophosphamide (20mg/kg) will be administered on days 29-30 and days 43-44.
Regenerative Medicine Center
Tianjin, Tianjin Municipality, China
Overall response rate
Percentage of patients with hematological response. Hematological response includes complete rate, near complete rate(CR), very good partial response(VGPR), good partial response(GPR) and partial response(PR).
Time frame: Within 3 months
Robust response
Percentage of patients with robust response, including CR, near CR, VGPR and GPR.
Time frame: Within 3 months
All-cause mortality
Time frame: Within 3 months
Time to achieve robust hematological response
Time frame: Within 6 months
Incidence of the adverse event
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Time frame: Within 6 months
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