Establishment of an Efficacy and Relapse Prediction System for Immunosuppressants Combined With Thrombopoietin Receptor Agonists in the Treatment of Aplastic Anemia

SN-01

In this study, investigators intend to prospectively study treatment-naive AA patients (including SAA and NSAA) who are non-transplant candidates in northern China. Patients with SAA receive ATG+CsA+Herombopag, and patients with NSAA receive CsA+ Herombopag. Investigators explored possible indicators of participants' predictive efficacy and built predictive models. After the participants achieved response, they used a tapering regimen, observed relapse and clonal evolution, and developed a predictive model of relapse.

2023-01-20

2025-01-30

2025-01-31

Recruiting

Early phase I

210

Inclusion Criteria: 1. Age: 18-75 years old, gender is not limited 2. Definite diagnosis of AA 3. No HSCT indication or unconditional HSCT 4. SAA/VSAA patients were willing to accept ATG+CsA+TPO-RA treatment, NSAA was willing to accept CsA+TPO-RA treatment, and were willing to follow up regularly 5. Baseline serum transaminase, total bilirubin and serum creatinine were less than 1.5 times the normal value 6. Baseline liver and kidney function was less than 1.5 times the normal value 7. Eastern Cancer Cooperation Group (ECOG) score status 0-2 8. Agree to sign the consent form Exclusion Criteria: 1. Congenital AA 2. Cytogenetic evidence of clonal hematologic bone marrow disease 3. PNH clone ≥50% 4. Allergic to ATG, cyclosporine and hexapopal in the past 5. Uncontrolled infection or bleeding at enrollment 6. Received hematopoietic stem cell transplantation (HSCT) before enrollment 7. Any concomitant malignancy within 5 years, except for local skin basal cell carcinoma 8. A history of thromboembolic events, myocardial infarction, or stroke (including antiphospholipid antibody syndrome); Currently using anticoagulants 9. Pregnant or lactating women 10. Situations considered unsuitable for clinical research by other researchers

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE4'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 210, 'type': 'ESTIMATED'}}

2023-08-24