Editor's Note：The 5th Annual Meeting of Chinese Alliance for Societies of Hematology (2025 CASH) was held in Tianjin from January 3 to 5, featuring the theme of “Comprehensive Hematology, Comprehensive Health, and Comprehensive Well-being.” The event invited top hematology experts from China and abroad to discuss the latest advancements and future trends in the field.

Hematology Frontier had the privilege of interviewing Dr. Suning Chen from The First Affiliated Hospital of Soochow University, who shared insights into the significant advances in acute myeloid leukemia (AML) research in 2024. This discussion highlighted how these new therapeutic strategies might reshape clinical practice and bring renewed hope to AML patients in China.

Significant Advances in AML Treatment

Professor Chen noted that at the 2024 American Society of Hematology (ASH) Annual Meeting, groundbreaking research results were showcased, with the Department of Hematology at the First Affiliated Hospital of Soochow University presenting 31 studies in oral and poster sessions. Among the five oral presentations selected, two originated from Professor Chen’s team.

One study compared the efficacy of venetoclax combined with decitabine (VEN-DEC) versus a regimen of idarubicin (12 mg/m²) combined with cytarabine (IA-12) as induction therapy for fit younger AML patients (Abstract 971). The results demonstrated comparable overall efficacy between VEN-DEC and IA-12 regimens. However, the VEN-DEC group showed higher response rates in patients aged over 40 and in those with intermediate- and high-risk genetic profiles. In the RUNX1::RUNX1T1 subgroup, IA-12 was more effective (88% vs. 44%). Importantly, the VEN-DEC group had significantly improved safety during the first induction cycle, with reduced transfusion needs and infection risks compared to the IA-12 group. These findings suggest that venetoclax combined with hypomethylating agents could become a new standard induction therapy for fit younger AML patients, moving beyond the traditional “7+3” regimen. Future approaches are likely to integrate venetoclax with chemotherapy and targeted therapies.

The second study focused on avapritinib for core-binding factor AML (CBF-AML) patients with KIT mutations (Abstract 222). For patients who had completed at least one consolidation therapy but still tested positive for molecular fusion genes, avapritinib induced fusion gene negativity or at least a one-log reduction in approximately 60% to 70% of patients within one cycle. This suggests that avapritinib could become a crucial component of induction and consolidation therapy for KIT-mutant AML patients.

Global Breakthroughs and Future Directions in AML

Professor Chen highlighted that targeted therapies were the most significant breakthrough in AML research in 2024, with Menin inhibitors being a prominent example. In November 2024, the FDA approved the first Menin inhibitor for clinical use, developed by Syndax Pharmaceuticals. Previous studies had already demonstrated the remarkable efficacy of the Menin inhibitor Revumenib, with findings published in Nature. At the 2024 EHA and ASH meetings, additional clinical trial data on Menin inhibitors, including BN104 from China’s YH Pharma, underscored the potential of Menin inhibitors as a major advancement in AML treatment.

The Department of Hematology at the First Affiliated Hospital of Soochow University has also been actively conducting clinical trials on Menin inhibitors, including a registration study and an investigator-initiated trial. Menin inhibitors hold significant promise for patients with KMT2A and NUP98 rearrangements, as well as those with HOXA gene overexpression. However, it is clear that single-agent therapies are unlikely to cure all patients or achieve long-term survival in most cases. Thus, the future of AML treatment will lie in integrating Menin inhibitors with conventional chemotherapy, allogeneic hematopoietic stem cell transplantation, and other targeted therapies. Such combined strategies are expected to bring greater clinical benefits to patients and drive progress in hematology.

Future Plans for Advancing AML Treatment in China

As Vice Chair of the 12th Hematology Committee of the Chinese Medical Association, Professor Chen outlined her plans for advancing AML treatment in China.

She emphasized the importance of improving diagnostic capabilities, particularly in molecular diagnostics for hematological diseases, which has been her primary focus. The goal is to promote innovation in molecular diagnostics and integrative diagnostic technologies in China, ensuring faster, more comprehensive, and precise diagnostic information for patients throughout their treatment journey.

Her team has developed a groundbreaking model for acute leukemia diagnosis, capable of delivering sequencing reports within 72 hours. This enables clinicians to accurately identify key genetic mutations and fusion genes before induction therapy, facilitating precise risk stratification and targeted treatment planning. Looking forward, Professor Chen is committed to promoting this highly efficient diagnostic technology to benefit more hematologists and patients nationwide.

In the treatment domain, the development of small-molecule targeted therapies and immunotherapies is advancing rapidly. Venetoclax, enasidenib, and ivosidenib have already transformed the AML treatment landscape, and Menin inhibitors are expected to bring further innovation. In acute B-cell lymphoblastic leukemia, new immunotherapies such as CAR-T cells and blinatumomab have significantly improved outcomes. Third-generation tyrosine kinase inhibitors (TKIs) and STAMP inhibitors may offer new treatment options for Philadelphia chromosome-positive ALL. Additionally, CD7 CAR-T therapy for T-cell ALL has shown promising early results.

Professor Chen’s second major focus is on integrating these emerging therapies with traditional treatments, such as chemotherapy and allogeneic transplantation. This comprehensive approach aims to reduce treatment burdens, increase cure rates, and improve long-term survival for AML patients.

About Dr. Suning Chen

Dr. Suning Chen is a Ph.D. holder and chief physician at the First Affiliated Hospital of Soochow University.

She serves as:

• Chair of the Hematology Branch of the Jiangsu Medical Association
• Deputy Director of the Jiangsu Institute of Hematology
• Member of the Hematology Committee of the Chinese Medical Association
• Standing Committee Member of the Experimental Hematology Branch of the Chinese Pathophysiology Society
• Vice Chair of the Hematological Rehabilitation Professional Committee of the Chinese Rehabilitation Medicine Association

Professor Chen specializes in acute leukemia research and has contributed to numerous studies on targeted therapies and immunotherapy.