Efficacy and Safety of Avatrombopag Combined With IST for the Treatment of HAAA and SAA With Abnormal Liver Function

• Status: Recruiting
• Trial end date: 2024-06-28
• Target enrollment: 0
• Participant gender: All

Summary

This is a multicenter, single-arm clinical study. The objective was to evaluate the efficacy and safety of Avatrombopag combined with IST in very/sever aplastic anemia patients with abnormal liver function or HAAA patients treated for the first time. The design was: Patients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. Cyclosporine 3 mg/kg orally in two divided doses, with cyclosporine trough concentrations maintained at 200-250 ng/ml for 3 months to achieve maximum efficacy, and Avatrombopag, which was administered in the dose of 40 mg orally once daily for a total of 12 weeks. Thirty-nine patients are expected to be enrolled in this study. Evaluation endpoint: complete response rate at 12 weeks of treatment.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital
• Treatments: Cyclosporine; Cyclosporins

Criteria

Inclusion Criteria:

1. patients with V/SAA with a definite diagnosis.

2. age between 18-70 years, male or female.

3. Subjects must complete all screening assessments as outlined in the trial protocol.

4. Able to swallow or administer the drug orally.

5. No prior application of TPO receptor agonists (including Thrombopoietin, Eltrombopag, Hetrombopag, etc.) or application of TPO receptor agonists for treatment with ≤ 5 total doses and ≤ 7 days of TPO receptor agonist drugs such as Eltrombopag, Hetrombopag, etc.

6. Diagnosis as HAAA or abnormal liver function. ALT and AST more than 1.5 times of upper limit.

7. Informed consent must be signed prior to the start of all specific study procedures, in consideration of the patient's condition, or by a member of the patient's immediate family if the patient's signature is not conducive to the treatment of the condition.

Exclusion Criteria:

1. Known diagnosis of congenital hematopoietic failure disorders (e.g. Fanconi anemia) and other causes of allogeneic cytopenias and bone marrow hypoproliferative disorders (e.g. hemolytic PNH, hypoproliferative MDS/AML, autoantibody-mediated allogeneic cytopenias, etc.);

2. Patients with uncontrolled bleeding and/or infection despite standard treatment.

3. Patients with previous history of hematopoietic stem cell transplantation; previous history of thrombosis.

4. Patients with concurrent malignancy or potential cancer on immunosuppressive therapy.

5. Those who are considered unsuitable for enrollment by the investigator.