Allogeneic stem cell transplantation for high-relapse-risk CEBPA mutant acute myeloid leukemia

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

This study is testing whether a stem cell transplant can help people with a specific type of acute myeloid leukemia that has a high chance of coming back after treatment.

Quick facts

• Study type: Observational
• Enrollment: 50 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Ruijin Hospital (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Shanghai, Shanghai Municipality)
• Trial ID: NCT06458257 on ClinicalTrials.gov

What this trial studies

This study evaluates the effectiveness of allogeneic hematopoietic stem cell transplantation in patients with newly diagnosed high-relapse-risk acute myeloid leukemia (AML) characterized by CEBPA mutations. Participants will undergo one cycle of induction chemotherapy followed by two cycles of consolidation before receiving the transplant. The study aims to assess disease-free survival, overall survival, and non-relapse mortality rates among these patients. High-relapse-risk is defined by specific molecular markers and minimal residual disease thresholds.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-65 with confirmed CEBPA mutant AML and high-risk molecular markers.

Not a fit: Patients with treatment-related AML or those outside the specified age range may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could significantly improve survival rates for patients with high-relapse-risk CEBPA mutant AML.

How similar studies have performed: Previous studies have shown promising results with allogeneic stem cell transplantation in similar high-risk AML populations, suggesting potential for success.

Eligibility criteria

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

Where this trial is running

Shanghai, Shanghai Municipality

• Ruijin Hospital Affiliated to Shanghai Jiaotong University School Of Medicine — Shanghai, Shanghai Municipality, China (Recruiting)

Conditions Acute Myeloid Leukemia; newly diagnosed; high-relapse-risk; CEBPA mutant