Hematopoietic stem cell transplantation (HSCT) remains a curative treatment for numerous hematologic disorders. Despite its life-saving potential, many clinical challenges persist, particularly concerning patient selection, transplant timing, and long-term management. To enhance academic exchange and practical insights in the field of transplantation, Hematology Frontier launched the special feature "Transplantation Pathways Spotlight" during the 51st Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2025). This column brings together leading experts from China and abroad to discuss full-cycle transplant care, from pre-transplant assessment through to long-term recovery, aiming to establish an integrated and forward-looking framework for clinical transplantation.

During the Annual Meeting, the editorial team invited the transplant group led by Prof. Erlie Jiang at the Stem Cell Transplantation Center of the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences to share their latest research. In this five-part series, we begin by highlighting the team’s important findings on transplant timing and patient selection, as presented in abstract OS7-02. These insights offer valuable clinical reference for the transplant and hematology communities.

HSCT plays a vital role in curing hematologic diseases such as leukemia, myelodysplastic syndromes (MDS), and severe aplastic anemia (SAA). Its success depends on a range of interconnected elements, including assessment of transplant indication, optimal timing, donor selection, comprehensive pre-transplant evaluation of both recipient and donor, tailored conditioning regimens, prevention and management of graft-versus-host disease (GVHD), infection control, relapse surveillance and intervention, and long-term follow-up and rehabilitation. These factors work together to ensure safety, therapeutic efficacy, and improved quality of life for patients following transplantation.

Dr. Mingyang Wang: Acute myeloid leukemia (AML) with the CBFB-MYH11 fusion gene is a genetically defined subtype commonly classified as low-risk, given its high sensitivity to chemotherapy. However, clinical data reveal that long-term survival in patients treated with chemotherapy alone remains below 50 percent. During treatment, some patients exhibit poor MRD (measurable residual disease) clearance, molecular relapse, or even morphologic relapse. These challenges make it critical to accurately identify the ideal timing for HSCT to improve patient outcomes.

Yet, the criteria for defining suboptimal MRD response and determining transplant timing have not been clearly established, posing an unresolved challenge in clinical practice. To address this, our center partnered with the Leukemia Center to conduct a retrospective analysis focused on the role of CBFB-MYH11 fusion transcript ratios in post-chemotherapy prognosis and therapeutic guidance.

The study included 188 eligible patients, of whom 137 received chemotherapy alone and 51 received chemotherapy followed by allogeneic HSCT. Findings revealed a significant positive correlation between the fusion transcript ratios after the second and third chemotherapy cycles. Specifically, in patients with a ratio greater than 0.5 percent after the second cycle, over 80 percent still had ratios above 0.2 percent after the third cycle. When the ratio exceeded 1 percent after the second cycle, more than 90 percent continued to show elevated levels above 0.2 percent in the third cycle (see Figure 1).

These results suggest that CBFB-MYH11 transcript dynamics after the second chemotherapy cycle can serve as an early indicator for disease persistence. Such data can help stratify patient prognosis and support timely decision-making regarding transplantation, especially in low-risk AML where molecular residual disease remains detectable.

Based on these findings, we identified 1% as the optimal cut-off value for the CBFB-MYH11 fusion transcript ratio after the second cycle of chemotherapy to distinguish differences in disease-free survival (DFS) and overall survival (OS). Among patients with a transcript ratio greater than 1%, allogeneic hematopoietic stem cell transplantation (allo-HSCT) significantly improved 3-year DFS [91.7% (95% CI: 81.3–100.0) vs. 47.8% (95% CI: 33.5–68.4), P=0.0081], and also enhanced 3-year OS in a landmark analysis [91.7% (95% CI: 81.3–100.0) vs. 72.9% (95% CI: 59.2–89.8), P=0.042] (see Figure 2).

We are honored to share these findings with colleagues around the world and hope that this research will contribute to ongoing efforts in refining transplant timing and improving outcomes for patients with low-risk AML. We look forward to further studies that continue to address this important clinical question.

Coming Up Next in the Transplantation Pathways Spotlight with Prof. Erlie Jiang’s Team

In the next installment of the Transplantation Pathways Spotlight, Dr. Rongli Zhang and Dr. Chen Liang from Prof. Erlie Jiang’s team will present two cutting-edge studies focused on conditioning regimen optimization for hematopoietic stem cell transplantation (HSCT).

The first study evaluates the effectiveness of total body irradiation (TBI) versus non-TBI myeloablative chemotherapy-based regimens in patients with B-cell acute lymphoblastic leukemia (B-ALL) undergoing HSCT.

The second study explores the efficacy of incorporating liposomal mitoxantrone hydrochloride into standard conditioning regimens—which include cladribine, cytarabine, busulfan, and cyclophosphamide—in patients with relapsed/refractory high-risk acute myeloid leukemia (AML).

Stay tuned for the next edition, where we continue to explore the clinical impact of these innovative strategies in the field of transplantation!

Director, Stem Cell Transplantation Center Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

Erlie Jiang, MD, is Professor of Hematology, Chief Physician, and Doctoral Supervisor at the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences. He currently serves as Director of the Hematopoietic Stem Cell Transplantation Committee of the China Primary Health Care Foundation, and Chair of the Leukemia Committee of the Chinese Society of Clinical Oncology (CSCO).

He is also Head of the Leukemia Autologous Transplantation Working Group within the China Hematology Specialty Alliance and serves as Deputy Chair of the Hematopoietic Stem Cell Application Group of the Hematology Branch of the Chinese Medical Association. Additionally, Prof. Jiang is an Executive Member of the Hematologic Oncology Committee of the Chinese Anti-Cancer Association and Deputy Chair of its Hematopoietic Stem Cell Transplantation and Cellular Therapy Group. He also serves as Deputy Chair of the CSCO Working Group on Autologous HSCT.

At the regional level, Prof. Jiang is Executive Council Member of the Tianjin Anti-Cancer Association and Vice President of the Tianjin Society of Hematology and Regenerative Medicine.

He is a member of the editorial boards of several prominent journals, including Chinese Journal of Hematology and Leukemia & Lymphoma. Prof. Jiang is recognized as a leading expert in the field of hematopoietic stem cell transplantation in China.

Stem Cell Transplantation Center Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

Dr. Mingyang Wang is an Attending Physician at the Stem Cell Transplantation Center of the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences. He holds a doctoral degree, with research focused on optimization of hematopoietic stem cell transplantation, as well as prediction and management of transplant-related complications.

Dr. Wang has contributed to over 20 SCI-indexed publications, with a cumulative impact factor of over 40 points as first author.