At the 2025 EBMT Annual Meeting, cord blood transplantation (UCBT) emerged as a transformative strategy in the treatment of pediatric hematologic malignancies. Hematopoietic stem cell transplantation (HSCT) remains a life-saving therapy, and UCBT is gaining attention for its unique clinical advantages. During the 51st Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2025) held in Florence, Italy, Hematology Frontier invited Prof. Fang Liu from the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, to share insights into the current role of transplantation in pediatric hematologic malignancies. She also highlighted her team’s latest findings in UCBT and discussed significant developments in the field of pediatric transplantation presented at the conference.

Hematology Frontier：As an expert in pediatric hematologic oncology, could you first outline the current applications and challenges of transplantation in children?

Prof. Fang Liu: Hematopoietic stem cell transplantation is a crucial therapeutic option for many pediatric diseases and has brought hope of a cure to numerous children. Take acute leukemia, for example: despite continual advancements in treatment, some cases remain refractory or relapsed. For these children, transplantation often offers the best hope for long-term survival.

In addition, children differ from adults in that they are affected by a number of unique conditions, such as congenital immunodeficiencies and inherited metabolic disorders. For example, bone marrow failure syndromes linked to germline predispositions are particularly reliant on HSCT as a curative approach.

While immunotherapy, targeted therapy, and CAR-T cell therapies have led to major breakthroughs in pediatric leukemia and lymphoma, we are increasingly focusing on patients with congenital or genetic defects. For these children, transplantation may be the only path to a cure. However, because of their underlying diseases, these children are often in poor general health, and many have associated organ dysfunctions that complicate the transplant process. They face increased risks such as immunodeficiency, susceptibility to infection, organ dysfunction, and poor tolerance to intensive conditioning regimens or chemotherapy. Therefore, it is vital to explore strategies to mitigate these risks and improve both transplant success rates and overall survival in this vulnerable population.

Hematology Frontier：EBMT is one of the most prestigious global platforms for academic exchange in hematology and transplantation. Your team presented several studies at this year’s meeting. Could you briefly walk us through your findings and their potential impact on improving pediatric transplantation?

Prof. Fang Liu：Our pediatric transplant team presented 11 posters at this year’s EBMT Annual Meeting. Compared to adult patients, cord blood is used more frequently in pediatric transplant settings due to its distinct benefits. For children, UCBT offers two major advantages: it is associated with lower relapse rates in malignant diseases and fewer chronic graft-versus-host disease (GVHD) events in the long term. This translates into better quality of life post-transplantation, which is especially critical for growing children.

Among the research we presented, one key study focused on pre-engraftment syndrome (PES) following UCBT (Poster B178). PES is a unique complication associated with cord blood transplantation. If not properly managed, severe PES can significantly impact transplant outcomes. To address this, we developed a predictive model specifically for PES in pediatric UCBT patients. The goal is to identify high-risk patients early and intervene in a timely manner, thereby improving overall transplant efficacy.

We also shared data on using UCBT to treat rare pediatric diseases, such as juvenile myelomonocytic leukemia (JMML, Poster P265) and blast-phase chronic myeloid leukemia (CML, Poster P272). Our preliminary findings show that even in these rare and challenging diseases, patients can achieve favorable long-term outcomes through cord blood transplantation, thanks to the evolving transplantation protocols and systems we’ve developed.

These advancements mark a significant step forward in refining and expanding the use of UCBT in pediatric populations, bringing renewed hope to children facing complex and life-threatening conditions.

Hematology Frontier：At this year’s EBMT Annual Meeting, were there any other breakthrough studies or advancements in the field of pediatric transplantation that particularly impressed you? How do you think these developments might influence clinical practice or shape future research?

Prof. Fang Liu:：As a pediatrician, I focus primarily on child-related research, especially studies that may impact long-term quality of life for pediatric patients—particularly those related to chronic graft-versus-host disease (GVHD). At this year’s conference, I was particularly struck by research from several groups exploring the use of mesenchymal stem cells and novel agents such as belumosudil. These studies offer promising new strategies and approaches for the treatment of chronic GVHD.

In addition, when treating refractory malignant diseases, we aim to reduce tumor burden as much as possible before transplantation and strive to bring the child into complete remission beforehand. This significantly improves the chances of a successful transplant and better survival outcomes. Currently, CAR-T cell therapy remains a highly active area of research in the treatment of malignancies. I am following these developments closely, and I am hopeful that they will provide more therapeutic options and renewed hope for our young patients.

Pediatric Hematology and Oncology Center, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

Dr. Fang Liu, MD, is Associate Chief Physician and Director of the Pediatric Transplant Unit at the Pediatric Hematology and Oncology Center. She led the center’s first-ever allogeneic hematopoietic stem cell transplantation (HSCT) procedure.

She currently serves as Deputy Chair of the Youth Committee of the Umbilical Cord Blood Clinical Application Subcommittee under the Chinese Maternal and Child Health Association, and Deputy Head of the Hematology Working Group within the Pediatric Intensive Care Subcommittee of the Chinese Medical Doctor Association.

Dr. Liu was a visiting scholar at the City of Hope National Medical Center in the United States. Her main research interests focus on pediatric immunotherapy and hematopoietic stem cell transplantation. She has successfully performed cord blood transplantation for several rare pediatric diseases, including congenital bone marrow failure syndromes, juvenile myelomonocytic leukemia (JMML), pyruvate kinase deficiency anemia, and hereditary ring sideroblastic anemia.

As a key contributor, Dr. Liu has received multiple honors, including the 11th Soong Ching Ling Pediatric Medicine Award and the 3rd Golden Snail Award for Rare Diseases. Her research has been published in both domestic and international journals such as Cell Transplantation, Journal of Hematology & Oncology, and American Journal of Hematology. She has also led or participated in several national and institutional research grants.