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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| treatment group | Experimental | 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). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ciclosporin | Drug | Cyclosporine was taken orally at 3-5mg/kg bid was administered to maintain the trough concentration at 100-200 μg/ml |
|
| Measure | Description | Time Frame |
|---|---|---|
| ORR at 6 Months | Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Changes in Haemoglobin in the Absence of Red Blood Cells Transfusion | The change in hematology values ( haemoglobin) were evaluated | 6 months |
| Changes in Platelet in the Absence of Platelet Transfusion |
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Inclusion Criteria:
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:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Bing Han | Contact | +861069151235 | hanbing_li@sina.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking union medical college hospital | Recruiting | Beijing | China |
individual participant data would be accepted upon request
always
email request
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| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
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| ID | Term |
|---|---|
| D016572 | Cyclosporine |
| C533238 | avatrombopag |
| ID | Term |
|---|---|
| D003524 | Cyclosporins |
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
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| Avatrombopag | Drug | Avatrombopag was administrated at 40 mg/d and increased by 20 mg/d during the biweekly follow-up if platelet was not 20 × 109/L higher than baseline. AVA dose was gradually reduced if PLT ≥ 150 × 109/L. The maximum dose was 60 mg/d, while the minimum was 20 mg/week. |
|
The change in hematology values (platelet) were evaluated
| 6 months |
| Percentage of patients with clonal evolution to myelodysplasia, PNH, acute leukemia | Clonal evolution to myelodysplasia is defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Evolution to leukemia is defined as greater than 20% peripheral blood and/or marrow blasts. Evolution to paroxysmal nocturnal hemoglobinuria (PNH) is defined as a clone at baseline < 10% that rose to greater than 50% on study. | 12 months |
| ORR at 1, 2, 3 Months and at the end of follow-up | Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) | 1, 2, 3 Months, last follow-up |
| Relapse rate | Relapse was defined as a substantial or progressive decline in at least one blood lineage counts of the responders that required the reinitiation or augmentation of AVA. | 6 months, last follow-up |
| D001855 | Bone Marrow Diseases |
| D010455 |
| Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |