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 the reduced dose of cyclophosphamide (hypo-CASH) for severe aplastic anemia.
Subject has a diagnosis of naïve severe or very severe aplastic anemia
Male or female age ≥ 12 years
Unwilling or unable to receive allogeneic hematopoietic stem cell transplantation.
ECOG performance status ≤2
Previously received immunosuppressive therapy > 4 weeks
Previously treated with TPO-RA > 4 weeks
Have an allergy or intolerance to anti-lymphocyte globulin, cyclosporine, herombopag or cyclophosphamide.
Uncontrolled fungal, bacterial, or viral infection. Hepatitis-associated aplastic anemia is allowed; hepatitis B or C infection is permitted unless it causes severe liver failure.
Single group
Reduced dose of cyclophosphamide combined with standard immunosuppressive therapy
DrugSevere 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. Cyclophosphamide (20mg/kg) will be administered on days 15-16.
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).
Superior response rate
Percentage of patients with superior response, including CR, VGPR and GPR.
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).
Incidence of the adverse event
Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event
Time to achieve robust superior response
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