The Efficacy of Allogeneic Hematopoietic Stem Cell Transplantation in Newly Diagnosed High-relapse-risk CEBPA Mutant Acute Myeloid Leukemia

For newly diagnosed high-relapse-risk CEBPA mutant acute myeloid leukemia patients, we aim to perform allogeneic hematopoietic stem cell transplantation after patients finished one cycle of induction and two cycles of consolidation. To access whether the therapeutic regimen is effective for high-relapse-risk CEBPA mutant acute myeloid leukemia, the disease-free-survival (DFS), overall survival (OS), non-relapse-mortality of patients is evaluated.

Study Overview

• Status: Recruiting
• Conditions: Acute Myeloid Leukemia
• Intervention / Treatment: Drug: Chemotherapy; Procedure: Allogeneic Hematopoietic Stem Cell Transplantation

Detailed Description

High-relapse-risk definition:

Participants with non-bZIP in-frame mutations, WT1 mutated status or CD7 negative status are considered of high-relapse-risk; The positive threshold for flow cytometry MRD was 0.0001%; The MRD threshold of molecular biology is the lowest value of the detection protocol of the center.

• Study Type: Observational
• Enrollment (Estimated): 50

Contacts and Locations

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 200025
      • Recruiting
      • Ruijin Hospital Affiliated to Shanghai Jiaotong University School of Medicine
      • Principal Investigator: Yang Shen, PhD
      • Contact: Jian Li, PhD; Phone Number: 02164370045; Email: lj12116@rjh.com.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Patients Diagnosed in Ruijin Hospital

Description

Inclusion Criteria:

1. Patients with confirmed CEBPA mutant AML. Diagnostic criteria include the presence of CEBPA mutant gene detected at the molecular level;
2. Patients with high-risk molecular markers or gene mutations, or complex karyotypes for disease recurrence, or flow cytometry/gene MRD positivity after two chemotherapy treatments; high-risk molecular markers or gene mutations include: CD7 positive, WT1 mutation, non-bzip-inframe CEBPA mutation; The positive threshold for flow cytometry MRD was 0.0001%; The MRD threshold of molecular biology is the lowest value of the detection protocol of the center.
3. Medical history and diagnosis of MICM, exclusion of MDS, transformation and treatment-related AML;
4. Age 18-65 years old (18 years old ≤Age< 65 years old);
5. Liver and kidney function: blood bilirubin ≤ 35 μmol/L, AST/ALT below 2 times the upper limit of normal, serum creatinine ≤ 150 μmol/L;
6. Normal cardiac function (EF≥50%, New York Cardiac Function Classification NYHA I/II);
7. Physical condition score 0-2 (ECOG score);
8. For patients with peripheral blood leukocytes < 50*109/L at the initial onset, no chemotherapy has been given except for hydroxyurea before the start of induction therapy;
9. For patients with peripheral blood leukocytes ≥ 50*109/L at the initial onset, cytarabine and hydroxyurea are allowed to be treated before the start of induction therapy;
10. Non-pregnant and lactating women;
11. For all women of childbearing age, a pregnancy test must be performed to measure hCG to rule out pregnancy;
12. Obtain informed consent signed by the patient or family member.

Exclusion Criteria:

1. MDS-converted AML, treatment-related AML; mixed cell leukemia; AML patients with central nervous system infiltrates and extramedullary lesions at the time of onset;
2. Relapse patients;
3. Allergies or contraindications to any of the drugs involved in the protocol;
4. Liver and kidney function are obviously abnormal, exceeding the enrollment criteria;
5. Cardiac disease: including echocardiogram EF <50%, cardiac insufficiency (New York cardiac function classification NYHA: III/IV), pericardial effusion (CTCAE score >2) within six months after acute myocardial infarction, ECG QTc >470ms;
6. Lung diseases: pulmonary edema, pleural effusion (CTCAE score >2);
7. Suffering from malignant tumors of other organs at the same time;
8. Active patients with HAV, HBV, HCV and tuberculosis, HIV-positive patients;
9. Concomitant other hematologic diseases (including coagulation abnormalities unrelated to leukemia);
10. Inability to understand or follow the study protocol;
11. Those who participate in other clinical studies at the same time; Presence of any other condition that would preclude the conduct of the study

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
HSCT-group; patients received allogeneic hematopoietic stem cell transplantation after consolidation treatment	Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Allogeneic Hematopoietic Stem Cell Transplantation
Chemo-group; patients received chemotherapy after consolidation treatment	Drug: Chemotherapy; Chemotherapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
disease-free-survival	disease-free-survival	from data of AML diagnosis until the data of AML relapse, assessed up to 3 years
The relation of CEBPA Mutant AML genetics subgroup with MRD negativity rate after chemotherapy and DFS after transplantation	The relation of CEBPA Mutant AML genetics subgroup with MRD negativity rate after chemotherapy and DFS after transplantation	from enrollment to study completion, a maximum of 3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
non-relapse-mortality	non-relapse-mortality	from enrollment to study completion, a maximum of 3 years
overall survival	overall survival	from enrollment to study completion, a maximum of 3 years
adverse events	adverse events	from enrollment to study completion, a maximum of 3 years

Collaborators and Investigators

• Sponsor: Ruijin Hospital

Study record dates

Study Major Dates

• Study Start (Actual): October 19, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2028

Study Registration Dates

• First Submitted: January 9, 2024
• First Submitted That Met QC Criteria: June 9, 2024
• First Posted (Actual): June 13, 2024

Study Record Updates

• Last Update Posted (Actual): July 29, 2025
• Last Update Submitted That Met QC Criteria: July 24, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Keywords: newly diagnosed; high-relapse-risk; CEBPA mutant
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Disease Attributes; Neoplasms by Histologic Type; Hematologic Diseases; Recurrence; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute
• Other Study ID Numbers: CEBPA-AML01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes