The study included adult patients ≥18 years old with SAA and TD-NSAA who had no HLA-matched sibling donors or were not suitable for first-line allogeneic hematopoietic stem cell transplantation due to age, complications, patient wishes, etc. After admission, they were treated with standard IST combined with rhTPO for at least 28 days. After discharge, the sequential treatment of EPAG-PFOS (with an initial dose of 150mg/d) was continued for 2 months (8 weeks), and the follow-up was conducted until the 6th month (24 weeks). It was compared with the historical cohort previously treated with IST combined with EPAG to explore the short-term efficacy and safety of this treatment regimen.
Age ≥18 years old, gender not limited;
Patients diagnosed with aplastic anemia (AA) through blood routine, bone marrow puncture, bone marrow biopsy and excluded examinations, and determined as SAA or TD-NSAA according to the Camitta criteria;
Those who have not received treatment with immunosuppressants (such as cyclosporine, tacrolimus, ATG, etc.);
The patient has no HLA-matched sibling donor or is not suitable for first-line allogeneic hematopoietic stem cell transplantation due to reasons such as age, complications, or the patient's wishes.
Pregnant or lactating patients;
Have a history of any arterial or venous thrombosis (including stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism) within half a year before enrollment and have at least two of the following risk factors: Hormone replacement therapy, oral contraceptives (including estrogen), smoking, diabetes, hypercholesterolemia, drug-controlled hypertension, hereditary coagulation disorders;
Patients with severe cardiovascular diseases (such as NYHA Cardiac Function Score grade III-IV) within half a year before enrollment, known arrhythmias that increase the risk of thromboembolism such as atrial fibrillation, after coronary stent implantation, angioplasty, and after coronary artery bypass grafting;
Combined with malignant tumors that seriously affect survival;
Parallel
EPAG-pfos
DrugThe initial dose of EPAG-pfos is 150mg/d orally. The dose is adjusted according to hematological indicators, with a maximum dose of 150mg/d until hematological remission is achieved
overall response rate
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