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

NCT02936089 | Completed DMC

• 1 other identifier: NFEC-2017-168
• Study Type: interventional
• Target: 207
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

Acute Myeloid Leukemia; Risk Stratification

Outcome Measures

Primary Outcomes (1)

• overall survival (OS)

  3 year

Secondary Outcomes (3)

• leukemia relapse rate

  3 year

• disease-free survival (DFS)

  3 year

• event Free Survival (EFS)

  3 year

Study Arms (3)

Low risk group

EXPERIMENTAL

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)

Intermediate risk group

EXPERIMENTAL

Patients with KIT+/ASXL1+ (single mutation, 1M) and acquiring MMR after two cycles of consolidation.

Other: Consolidation with auto-HSCT or HLA-matched HSCT

High risk group

EXPERIMENTAL

Patients with KIT+ASXL1+ (two mutations ,2M) or without acquiring MMR after two cycles of consolidation.

Other: allogeneic HSCT

Interventions

Consolidation with chemotherapy (CT) or autologous hematopoietic stem cell transplantation (auto-HSCT) OTHER

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.

Low risk group

Consolidation with auto-HSCT or HLA-matched HSCT OTHER

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.

Intermediate risk group

allogeneic HSCT OTHER

For allogeneic HSCT, patients were treated with 1-2 cycles of HDAC and then bridged to allogeneic HSCT, including HLA-matched and haploidentical transplantation.

High risk group

Eligibility Criteria

• Age: 14 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Nanfang Hospital, Southern Medical University: lead
• Zhujiang Hospital: collaborator

Related Publications (1)

• Xu D, Yang Y, Yin Z, Tu S, Nie D, Li Y, Huang Z, Sun Q, Huang C, Nie X, Yao Z, Shi P, Zhang Y, Jiang X, Liu Q, Yu G. Risk-directed therapy based on genetics and MRD improves the outcomes of AML1-ETO-positive AML patients, a multi-center prospective cohort study. Blood Cancer J. 2023 Nov 13;13(1):168. doi: 10.1038/s41408-023-00941-4. No abstract available.

  PMID: 37957175 DERIVED

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Drug Therapy

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Therapeutics

Study Officials

• Dan Xu

  Nanfang Hospital, Southern Medical University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: October 11, 2016
• First Posted: October 18, 2016
• Study Start: October 1, 2016
• Primary Completion: December 31, 2021
• Study Completion: December 31, 2022
• Last Updated: August 8, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: The data will be available after being published, for at least five years.
• Access Criteria: All collected IPD, all IPD that underlie results will be shared in a publication. For the detail, those who are interested in can contact the authors.