Combining IST and TPO-RA for treating Aplastic Anemia

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

Phase 4 Interventional Peking Union Medical College Hospital · NCT06009965

This study is testing a new combination treatment for patients with Aplastic Anemia who can’t have a transplant to see if it helps them feel better.

Quick facts

PhasePhase 4
Study typeInterventional
Enrollment210 (estimated)
Ages18 Years to 75 Years
SexAll
SponsorPeking Union Medical College Hospital Academic / other
Locations1 site (Beijing)
Trial IDNCT06009965 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the efficacy of a combination treatment involving anti-thymocyte globulin (ATG), cyclosporine A (CsA), and Herombopag for treatment-naive patients with Aplastic Anemia (AA) who are not candidates for transplantation. Patients with severe AA (SAA) will receive ATG+CsA+Herombopag, while those with non-severe AA (NSAA) will receive CsA+Herombopag. The study aims to monitor treatment responses, taper medications, and develop a predictive model for relapse based on patient indicators. The trial is conducted at Peking Union Medical College Hospital in Beijing, China.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-75 with a confirmed diagnosis of Aplastic Anemia who are not eligible for hematopoietic stem cell transplantation.

Not a fit: Patients with congenital Aplastic Anemia or those who have received prior hematopoietic stem cell transplantation will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve outcomes for patients with Aplastic Anemia by providing an effective non-transplant therapy.

How similar studies have performed: Previous studies have shown promising results with similar treatment combinations in Aplastic Anemia, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria
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

Where this trial is running

Beijing

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Aplastic AnemiaCSADrug TherapyCsAATGTPO-RA
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.