Risk Stratification-directed Therapy for AML With t(8;21) /AML1-ETO+

Study Overview
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Brief Summary
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Acute myeloid leukemia with t(8;21) /AML1-ETO-positive (AE AML) is a heterogeneous disease entailing different prognoses. There were significant differences in the therapeutic effect between different subgroups of AE AML. Therefore, risk stratification-directed therapy is very necessary for AE AML.

Detailed Description
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Acute myeloid leukemia with t(8;21) /AML1-ETO-positive (AE AML) is a heterogeneous disease entailing different prognoses.There were significant differences in the therapeutic effect between different subgroups of AE AML. For example, patients with c-kit mutation had higher relapse rate and lower overall survival, compared with those without c-kit mutation. Therefore, risk stratification-directed therapy is very necessary for AE AML. The purpose of this study is to establish risk stratification-directed therapy for AE AML.

Official Title
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Risk Stratification-directed Therapy for Acute Myeloid Leukemia With t(8;21) /AML1-ETO-positive

Conditions
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Acute Myeloid Leukemia, Risk Stratification

Intervention / Treatment
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* Other: Consolidation with auto-HSCT or HLA-matched HSCT
* Other: allogeneic HSCT
* Other: Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: AE AML aged 14-70 No abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure) Expected survival time is more than 2 months Exclusion Criteria: Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure) Patients with any conditions not suitable for the trial (investigators' decision)

Ages Eligible for Study
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Minimum Age: 14 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Patients with newly diagnosed AML1/ETO-positive AML took risk-directed stratification therapy based on c-KIT and ASXL1 mutations and measurable residual disease (MRD). low risk (LR) group (KIT-ASXL1- with main molecular response (MMR)) was recommended to chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT). Intermediate risk (IR) group (KIT+/ASXL1+ with MMR) was suggested for auto-HSCT or HLA-matched HSCT. High risk (HR) group (KIT+ASXL1+ or without MMR) was treated with allogeneic (allo-) HSCT.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Low risk group<br>Patients with KIT-ASXL1- (non-mutation, NM) and acquiring main molecular response (MMR) after two cycles of consolidation. | Other: Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT)<br>* For CT, patients were treated with high dose cytarabine (HDAC), cytarabine at a dosage of 1-3 g/m2 q12 h ×6 doses, for 4-6 cycles. For auto-HSCT, patients were treated with 3 cycles of HDAC and then bridged to auto-HSCT.<br>|
| Experimental: Intermediate risk group<br>Patients with KIT+/ASXL1+ (single mutation, 1M) and acquiring MMR after two cycles of consolidation. | Other: Consolidation with auto-HSCT or HLA-matched HSCT<br>* For auto-HSCT, patients were treated with 3 cycles of HDAC and then bridged to auto-HSCT. For HLA-matched HSCT, patients were treated with 1-2 cycles of HDAC and then bridged to HLA-matched HSCT. HLA-matched donors were available in these patients.<br>|
| Experimental: High risk group<br>Patients with KIT+ASXL1+ (two mutations ,2M) or without acquiring MMR after two cycles of consolidation. | Other: allogeneic HSCT<br>* For allogeneic HSCT, patients were treated with 1-2 cycles of HDAC and then bridged to allogeneic HSCT, including HLA-matched and haploidentical transplantation.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| overall survival (OS) |  | 3 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| leukemia relapse rate |  | 3 year | 
| disease-free survival (DFS) |  | 3 year | 
| event Free Survival (EFS) |  | 3 year |