transfusion dependent non-severe aplastic anemia
Conditions
Interventions
Test Group:Hetrombopag + Cyclosporine + Androgens
Sponsors
West China Hospital of Sichuan University
Eligibility
Sex/Gender
All
Age
18 Years to 75 Years
Inclusion criteria
Inclusion criteria: 1. Understand the research procedures and methods, voluntarily participate in this trial, and provide written informed consent; 2. At the time of signing the informed consent form, the subject must be between 18 and 75 years of age, regardless of gender; 3. Diagnosed with TD-NSAA, based on the following criteria: Meets the Camitta NSAA criteria, requiring at least one component transfusion every 8 weeks on average (transfusion indications: HGB = 4 months; 4. Unsuitable for or unable to receive treatment with anti-lymphocyte globulin (ALG)/anti-thymocyte globulin (ATG) or hematopoietic stem cell transplantation (HSCT); 5. Adequate function of vital organs.
Exclusion criteria
Exclusion criteria: 1. Meeting the diagnostic criteria for very severe/severe aplastic anemia (V/SAA): SAA: a) Bone marrow cellularity = 45 mg/kg/day), or alemtuzumab. Cumulative use of CsA, androgens, or TPO-RA agents for > 2 weeks before enrollment; or use for = 50%, OR presence of a hemolytic PNH clone, OR evidence of cytogenetic abnormalities. 5. Previous history of hematopoietic stem cell transplantation. 6. Uncontrolled infection or bleeding despite standard treatment. 7. Active HIV, HCV, or HBV infection. 8. Liver cirrhosis or portal hypertension. 9. Severe liver dysfunction: ALT or AST > 3 times the upper limit of normal (ULN), or total bilirubin > 1.5 × ULN after treatment. 10. Severe renal dysfunction: Creatinine clearance 1.5 × ULN. 11. Pancytopenia caused by other underlying systemic diseases. 12. Uncontrolled hypertension, severe cardiac arrhythmia, or NYHA Class III/IV congestive heart failure. 13. History of arterial or venous thrombosis within 12 months prior to enrollment. 14. History of radiotherapy or chemotherapy for malignant solid tumors. 15. Known or suspected contraindications/hypersensitivity to Hetrombopag, Cyclosporine (CsA), or androgens. 16. Pregnancy or lactation; women of childbearing potential or male subjects with partners of childbearing potential unwilling to use effective contraception during the treatment period and for 28 days after the last dose. 17. Any other condition considered by the investigator as unsuitable for participation in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Overall hematologic response rate at 24 weeks of treatment; | — |
Secondary
| Measure | Time frame |
|---|---|
| Overall hematologic response rate at 12 weeks of treatment;Hematologic complete response (CR) and partial response (PR) rates at 24 weeks;Proportion of patients free from red blood cell (RBC) or platelet (PLT) transfusion dependency at 24 weeks of treatment;Time to first hematologic response (CR or PR) within 24 weeks of treatment;Proportion of patients with clonal evolution during treatment;Occurrence of adverse event (AE): including type, grade and incidence of AE; | — |
Countries
China
Contacts
Public ContactHong Chang
West China Hospital of Sichuan University
Outcome results
None listed