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

Chinese Academy of Medical Sciences & Peking Union Medical College

Status and phase: Enrolling; Phase 2

Conditions: Aplastic Anemia

Treatments: Drug: Avatrombopag; Drug: Ciclosporin

Study type: Interventional

Funder types: Other

Identifiers

NCT06004752

Avatrombopag-2

Details and patient eligibility

About

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.

Full description

Aplastic anemia (AA) can be divided into severe AA (SAA) and non-severe AA (NSAA), according to the severity of the disease. Anti-thymocyte globulin (ATG) in combination with CsA is the most typical combined immunosuppressive therapy regimen. For elderly patients who cannot tolerate anti-thymocyte globulin (ATG) treatment, the addition of avatrombopag, which has a slight adverse reaction, can theoretically improve the hematological response rate in elderly patients with 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.

Enrollment: 30 estimated patients

Sex: All

Ages: 60 to 90 years old

Volunteers: No Healthy Volunteers

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

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

6. 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

treatment group

Experimental group

Description:

Cyclosporine: 3mg/kg/d 3-5mg/kg bid was administered to maintain the trough concentration at 100-200 μg/ml; Avatrombopag: The dosage was 40\~60mg orally, once a day, and the dosage was adjusted according to the subject's platelet count. The drug was administered for 24 weeks (6 months).

Treatment:

Drug: Avatrombopag

Drug: Ciclosporin