Efficacy and Safety of CsA+AVA in the Treatment of NSAA in the Elderly

• Status: Recruiting
• Trial end date: 2025-08-01
• Target enrollment: 0
• Participant gender: All

Summary

For elderly patients who cannot tolerate anti-thymocyte globulin (ATG) treatment, the addition of avatrombopag (AVA), which has a slight adverse reaction, can theoretically improve the hematological response rate in elderly patients with non-severe aplastic anemia (NSAA) without significantly increasing adverse reactions. Based on this, this study treated NSAA patients older than 60 with AVA combined with CsA to evaluate the hematological response rate and safety of AVA in the elderly who could not tolerate ATG therapy.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Peking Union Medical College Hospital
• Treatments: Cyclosporine; Cyclosporins

Criteria

Inclusion Criteria:

1. Elderly patients with well-defined NSAA anemia who meet the diagnostic criteria for aplastic anemia (AA) but do not meet the diagnostic criteria for severe aplastic anemia (SAA).

2. Age 60 years or older, male or female.

3. Able to swallow or administer orally.

4. Intolerant or refused anti-thymocyte globulin treatment

6. No previous treatment with cyclosporine, tacrolimus or hormones or treatment for less than 1 month.

7. No prior treatment with thrombopoietin (TPO) receptor agonists (including eltrombopag, herombopag, romiplostim, etc.) 8. Informed consent must be signed before the start of all specific research procedures. Considering the patient's condition, if the patient's signature is not conducive to the treatment of the disease, the informed consent shall be signed by the patient's immediate family.

Exclusion Criteria:

1. Known congenital AA (such as Fanconi anemia) and other causes of pancytopenia and hematological bone marrow disorders;

2. With paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%

3. With a history of hematopoietic stem cell transplantation.

4. History of thrombosis

5. Patients with underlying cancer who also have malignant tumors or are receiving immunosuppressive therapy.

6. Baseline creatine levels greater than twice of the upper limit of normal (ULN) and/or alanine aminotransferase (ALT) greater than 2.5 times of the ULN;

7. Serious heart, liver and kidney disease.

8. With uncontrolled bleeding and/or infection after standard treatment.

9. Participants considered unsuitable for inclusion by the researchers.