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This is a prospetive,Single-Center, Single-Arm, Phase IIB Clinical Study.This study aims to evaluate the efficacy and safety of dose-attenuated IST combined with Hetrombopag in elderly patients (≥65 years) with VSAA/SAA.
Design : Prospective, single-center, single-arm, open-label Phase IIB trial.
Intervention :
P-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag : 15 mg daily. Follow-Up : Weekly for 24 weeks, with bone marrow evaluation at 24 weeks.
Inclusion Criteria :
Confirmed diagnosis of VSAA/SAA. Age ≥65 years. Completion of all screening assessments. Ability to swallow oral medication. Signed informed consent (by patient or legal guardian if patient is incapacitated).
Exclusion Criteria :
Clonal cytogenetic abnormalities (excluding isolated -Y or +8). Prior treatment with ATG/high-dose cyclophosphamide. Prior cyclosporine/tacrolimus use >12 months. Prior TPO-RA therapy >3 months. Uncontrolled malignancies or conditions contraindicating ATG. Severe organ dysfunction (e.g., creatinine ≥177 μmol/L). Investigator judgment of unsuitability.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| A-IST | Experimental | Porcine-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3-5 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag: 15 mg daily. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Dose-attenuated IST and Hetrombopag | Drug | Porcine-ATG : 15 mg/kg/day IV ×5 days. Cyclosporine : 3-5 mg/kg/day (adjusted to trough 100-200 μg/L). Hetrombopag: 15 mg daily for 24weeks. |
| Measure | Description | Time Frame |
|---|---|---|
| Hematologic response rate at 24 weeks post-ATG treatment. | PR+CR Partial Response (PR) : Independence from transfusions without meeting CR. No Response (NR) : Failure to achieve PR. | 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Early mortality rate | Early mortality rate within 12weeks | 12 weeks |
| Complete Response | Complete Response (CR) : HGB ≥100 g/L, PLT ≥100×10⁹/L, ANC ≥1.0×10⁹/L. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jianping Li, MD | Contact | +8613820961539 | lijianping@ihcams.ac.cn | |
| Jingyu Zhao, MPH | Contact | +8613752253515 | zhaojingyu@ihcams.ac.cn |
| Name | Affiliation | Role |
|---|---|---|
| Jun Shi, PhD | Institute of Hematology & Blood Diseases Hospital, CAMS | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Red Blood Cell Diseases Center | Recruiting | Tianjin | Tianjin Municipality | China |
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| 24 weeks |
| ID | Term |
|---|---|
| C000614661 | hetrombopag |
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