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| ID | Type | Description | Link |
|---|---|---|---|
| 09021420250003 | Other Grant/Funding Number | Suzhou University Affiliated First Hospital 'Yanzhen Lingfei' Program |
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| Name | Class |
|---|---|
| Nanfang Hospital, Southern Medical University | OTHER |
| Ruijin Hospital North Shanghai Jiao Tong University School of Medicine | UNKNOWN |
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The purpose of this study is to explore the efficacy and safety of venetoclax combined with azacitidine(VA) induction followed by bridging allogeneic hematopoietic stem cell transplantation (allo-HSCT) in the treatment of hypoplastic and secondary acute myeloid leukemia (AML).
Under the traditional intensive chemotherapy model, patients with hypoplastic or secondary acute myeloid leukemia (AML) often have poor bone marrow reserve and prolonged post-chemotherapy cytopenia, leading to high early mortality, low remission rates, and short median survival. To address this, the use of venetoclax combined with a hypomethylating agent (the VA regimen) as induction therapy significantly improves complete remission (CR) and overall survival (OS). Subsequent bridging to allogeneic hematopoietic stem cell transplantation (allo-HSCT) as consolidation further enhances OS and relapse-free survival (RFS). This combined strategy demonstrates a clear synergistic effect: the VA regimen effectively increases the transplantation rate, and for patients with relapsed/refractory (R/R) AML, a low-intensity regimen followed by bridging transplantation is non-inferior to traditional intensive chemotherapy, achieving a "1+1>2" therapeutic benefit. Therefore, the investigators conducted a prospective study to evaluate the efficacy and safety of VA regimen induction followed by allo-HSCT in patients with hypoplastic and secondary AML.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| A | Experimental | VA induction+Allo-HSCT consolidation |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Venetoclax (VEN) | Drug | Venetoclax was administered at 100 mg on day 1, 200 mg on day 2, and 400 mg on days 3-28 of cycle 1. |
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| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival (OS) | Time from the date of diagnosis to death due to any cause. | 2 year |
| Measure | Description | Time Frame |
|---|---|---|
| Complete Remission Rate(CRR) | Bone marrow blasts <5%, absence of extramedullary disease, no circulating blasts, peripheral blood neutrophil count ≥1.0×10⁹/L, platelet count ≥100×10⁹/L, independent of transfusion or growth factor support. | Day 28 of induction therapy and 56 days post-transplantation |
| Composite Complete Remission Rate (CCRR) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Zhen Shen, Doctor | Contact | 0512-67976802 | zhenshen96@sina.cn |
| Name | Affiliation | Role |
|---|---|---|
| Suning Chen, Doctor | The First Affiliated Hospital of Soochow University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The First Affiliated Hospital of Soochow University | Suzhou | Jiangsu | 215000 | China |
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| ID | Term |
|---|---|
| C579720 | venetoclax |
| D001374 | Azacitidine |
| ID | Term |
|---|---|
| D001372 | Aza Compounds |
| D009930 | Organic Chemicals |
| D003562 | Cytidine |
| D011741 | Pyrimidine Nucleosides |
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| Azacitidine (AZA) | Drug | Azacitidine was administered at 75 mg/m² on days 1-7 of cycle 1. |
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| Allo-HSCT | Procedure | All patients proceeded directly to allogeneic HSCT after cycle 1, regardless of remission. Myeloablative or intensified conditioning was preferred; reduced-intensity conditioning was allowed for intolerant patients. Donor source and transplant type were not restricted. |
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The proportion of patients achieving complete remission (CR), complete remission with incomplete hematologic recovery (CRi), or complete remission with partial hematologic recovery (CRh). |
| Day 28 of induction therapy and 56 days post-transplantation |
| Disease-free Survival (DFS) | Time from achieving disease remission to disease relapse or death from any cause. | 2 year |
| GVHD-free and Relapse-free Survival (GRFS) | Time from transplant date to the first occurrence of grade III-IV acute GVHD, chronic GVHD requiring systemic treatment, disease relapse, or death from any cause. | 2 year |
| Cumulative Incidence of Relapse (CIR) | Time from transplant date to disease relapse. | 2 year |
| Non-relapse Mortality (NRM) | Time from transplant date to death due to non-relapse/progression causes. | 2 year |
| D011743 |
| Pyrimidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D009705 | Nucleosides |
| D009706 | Nucleic Acids, Nucleotides, and Nucleosides |
| D012263 | Ribonucleosides |