Enarodustat + CsA vs CsA in Newly Diagnosed TD-NSAA

Efficacy and Safety of Enarodustat Combined With Cyclosporine Versus Cyclosporine Monotherapy in the Treatment of Newly Diagnosed TD-NSAA

This study aimed to compare the efficacy and safety of enarodustat combined with cyclosporine versus cyclosporine monotherapy in the treatment of newly-diagnosed tansfusion-dependent non-severe aplastic anemia (TD-NSAA).

Inclusion Criteria:

1. Age ≥ 18 years old;
2. Diagnosed with aplastic anemia (AA) through routine blood tests, bone marrow puncture, bone marrow biopsy, and exclusion tests, and determined as transfusion-dependent non-severe aplastic anemia (TD NSAA) according to the Camitta criteria; Hemoglobin<90g/L;
3. Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT);
4. With baseline liver and kidney functions <2 ULN;
5. ECOG score ≤ 2;
6. Signed the informed consent;

Exclusion Criteria:

1. Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;
2. With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;
3. PNH clone ≥ 50%;
4. Received HSCT before enrollment;
5. Previously used immunosuppressive treatments such as ATG, CsA, TPO receptor agonists (TPO-RAs), roxadustat;
6. Allergic or intolerant to enarodustat or CsA;
7. Pregnant or lactating patients;
8. Severe bleeding or infection that cannot be controlled by standard treatment;
9. Complicated with malignant tumors;
10. Participated in other clinical trials within 3 months;
11. Patients considered not suitable to participate in this study by the investigator.