Efficacy and Safety of Autologous Peptide-induced Active Immunity in AML Maintenance Therapy

A Randomized, Controlled, Prospective Study on the Efficacy and Safety of Active Immunity Induced by Autologous Peptides for Maintenance Therapy in Acute Myeloid Leukemia (AML)

Acute myeloid leukemia (AML) is the most common acute leukemia in adults. While approximately 70% of patients achieve complete remission (CR) with induction chemotherapy, traditional consolidation therapy (predominantly high-dose cytarabine) has a persistently high recurrence rate - nearly 30% at 1 year for low-risk groups and 80% for high-risk groups - with a long-term survival rate <40%. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) improves survival but is limited by donor matching and patient tolerance, resulting in a transplantation rate <20%. Clinically, there is an urgent need for a well-tolerated, low hepatotoxic/nephrotoxic maintenance regimen effective for preventing recurrence.

Tumor immunotherapy is a major breakthrough, and neoantigen-based personalized vaccines are a key anti-recurrence direction due to their strong tumor specificity and ability to induce long-term immune memory. However, existing neoantigen vaccines rely on NGS sequencing and bioinformatics for epitope screening, suffering from long development cycles, high costs, proneness to missing cancer-causing mutations, and poor clinical feasibility, hindering widespread use. This study adopts a patented Sino-US innovative technology: in vitro induction of patients' own AML cells to obtain a complete set of tumor antigen peptides for personalized vaccine preparation, circumventing traditional bottlenecks to achieve "full antigen coverage" personalized active immunity.

This study has significant clinical and scientific value: (1) It is the first application of this patented technology in AML maintenance therapy, filling domestic and international research gaps and providing a novel treatment option; (2) Using a randomized controlled design, it compares the efficacy of immunotherapy administered during vs. after consolidation chemotherapy to identify the optimal treatment mode; (3) It screens reliable anti-leukemia immunity monitoring methods and time points, offering evidence-based support for efficacy evaluation and prognostic prediction; (4) It verifies the treatment's safety, laying a foundation for developing low-toxic, high-efficacy AML maintenance regimens, ultimately improving patients' long-term survival and advancing precision immunotherapy for AML.

Inclusion Criteria:

• Newly diagnosed with acute myeloid leukemia (AML) in accordance with the 2018 WHO Classification and Diagnostic Criteria for Acute Leukemias; received 1-2 courses of conventional chemotherapy, achieved remission, and are undergoing routine consolidation therapy.
• Aged 18 to 70 years.
• Receiving a maintenance therapy regimen without hormonal agents.
• Leukocyte and lymphocyte counts have basically returned to the normal range.
• Patients judged by the investigator to have an expected survival of at least 12 months after achieving remission.
• Patients who voluntarily participate in this study and sign the informed consent form.

Exclusion Criteria：

• Patients who still require hormonal maintenance therapy after achieving remission.
• Patients with a concomitant history of other malignant tumors or a history of uncontrolled malignant tumors.
• Having participated in other clinical trials within 1 month prior to screening.
• Complicated with uncontrolled cerebrovascular diseases, coagulation disorders, connective tissue diseases and other similar conditions.
• Having other uncontrolled diseases that the investigator deems unfit for enrollment.
• Patients with psychiatric disorders or those known/suspected to be unable to fully comply with the study protocol.
• Pregnant or lactating women.
• HIV-infected individuals.
• Other conditions that the investigator deems may prevent the subject from completing the study or pose a significant safety risk to the subject.

Withdrawal Criteria：

• Judged by the investigator to be in the best interest of the subject.
• Disease progression or initiation of other anti-leukemia therapy.
• The subject requests to withdraw from the study for any reason at any time.
• Lost to follow-up.
• Death.
• Occurrence of severe chemotherapy-induced toxic reactions, or chemotherapy delay of more than 4 weeks due to adverse reactions.
• Cardiac toxicity: Left Ventricular Ejection Fraction (LVEF) ≤ 50% or a decrease of > 10%; or QTc prolongation meeting the following criteria: ① QTc > 500 ms; ② QTc > 530 ms in patients with bundle branch block.
• Hepatic toxicity: Persistent elevation of alanine transaminase (ALT) and/or aspartate transaminase (AST) to more than 2 times the upper limit of normal (ULN), with no response to hepatoprotective treatment.