Every year, approximately 15,000 children in China are diagnosed with hematological diseases, with severe conditions such as leukemia, aplastic anemia, and hemophagocytic syndrome accounting for over 30%. Over the past two decades, China's diagnostic and treatment system for childhood hematological diseases has evolved from "scattered breakthroughs" to "systemic integration," forming a full-cycle ecological network covering screening and early warning, precision diagnosis and treatment, and rehabilitation support. Professor Sun Yuan, Director of the Hematology Oncology Center at Beijing Jingdu Children's Hospital, as one of the core architects of this ecological system, has propelled China's childhood hematological disease prevention and treatment landscape toward international leading standards by driving technological innovation and prioritizing patient well-being.
The cornerstone of China's diagnostic and treatment ecological system for childhood hematological diseases is a "three-level " (three-tiered) hierarchical network:
Grassroots Screening: Early warning for high-risk children is achieved through neonatal genetic screening (e.g., thalassemia gene testing) and regional blood routine big data analysis.
Regional Referral: National-level children's medical centers in Beijing, Shanghai, and other cities have established 72-hour green referral channels to rapidly connect complex cases to specialized treatment.
National Platform: Organizations such as the Children's Leukemia Cooperative Group (CCLG) led by the National Children's Medical Center have unified diagnostic and treatment pathways and molecular typing standards, increasing the 5-year survival rate for acute lymphoblastic leukemia from 60% to 85%.
Professor Sun Yuan's Systematic Contributions:
1. Networked Transplantation Centers: Established a trinity platform of "Hematology Oncology-Transplantation Medicine-Intensive Care" at Jingdu Children's Hospital, completing over 150 hematopoietic stem cell transplants annually, with hemophagocytic syndrome accounting for one-third of cases, making it the largest pediatric transplantation center in North China.
2. Standardized Guideline Development: As a core drafter of the Clinical Operation Guidelines for Umbilical Cord Blood Transplantation in Children, she clarified parameters such as umbilical cord blood cell dosage and conditioning regimens, reducing transplantation-related mortality to below 8%.
Once regarded as "medical waste," umbilical cord blood has been systematically studied by Professor Sun Yuan's team to reveal its unique value:
Autologous Transplantation Breakthrough: In 2009, the first autologous umbilical cord blood transplant for severe aplastic anemia in China was completed. A 10-year follow-up showed complete normalization of the patient's hematopoietic function.
Solid Tumor Combination Protocol: In 2016, the innovative "chemotherapy + autologous umbilical cord blood reinfusion" protocol for neuroblastoma increased the 3-year survival rate from 20% to 65%.
Family Resource Bank Construction: Promoted the establishment of China's largest pediatric umbilical cord blood bank, reducing sibling transplantation costs by 40% and the incidence of graft-vs-host disease (GVHD) to only 18%.
Addressing the dilemma that 80% of children lack fully matched donors, Professor Sun's team innovated technical pathways:
Stratified Conditioning: Low-risk children receive fludarabine + low-dose radiotherapy, while high-risk children are treated with cladribine combination, reducing transplantation-related mortality from 30% to 8% .
Precision T Cell Regulation: Through TCRαβ+/CD45RA+ cell depletion technology, the incidence of acute GVHD was reduced from 40-60% to 15%, reaching the highest international standards.
Professor Sun pioneered the establishment of a "molecular typing-dynamic monitoring-targeted intervention" system in China:
Individualized chemotherapy protocols based on fusion genes such as BCR-ABL and MLL rearrangement;
Next-generation sequencing to monitor minimal residual disease (MRD) after transplantation, reducing the recurrence rate by 35% ;
CD19 CAR-T sequential transplantation for refractory leukemia, achieving a 5-year disease-free survival rate of 58%.
Professor Sun's team introduced international palliative care concepts for children with untreatable conditions:
Developed a pain grading management protocol using fentanyl transdermal patches for cancer pain control;
Established the "Sunshine Little Blood Drop" online community, providing psychological support to over 20,000 families.
Medical Assistance Network: Launched the "Little Soldiers of Life" program in collaboration with the Soong Ching Ling Foundation, providing free transplants for over 300 impoverished families;
Medical Insurance Policy Promotion: Participated in formulating clinical pathways for diseases such as hemophagocytic syndrome and aplastic anemia, which were included in Hebei Province's special insurance coverage for childhood hematological diseases in 2025.
Professor Sun proposed the "1000-Day Post-Transplantation Management Model":
Immune Reconstruction Monitoring: Regular detection of T/B cell subsets and immunoglobulin levels starting 3 months post-surgery;
Endocrine Intervention: Early use of rhGH therapy for growth hormone deficiency to prevent short stature;
Social Function Rehabilitation: Collaborated with educational departments to establish "ward schools" to ensure academic continuity during treatment .
Professor Sun's team is developing two systems:
Transplantation Prognosis Model: Integrates HLA epitope matching and metabolomics data (e.g., kynurenine/tryptophan ratio) to predict GVHD risk;
Remote Quality Control Platform: Achieves over 95% accuracy in anemia classification diagnosis through AI analysis of blood smear images from grassroots hospitals.
As a member of the International Histiocyte Society, Professor Sun has promoted the establishment of transnational research networks:
Collaborated with St. Jude Children's Research Hospital on the Pediatric Leukemia Genome Project, identifying 7 East Asian-specific mutation sites;
Led the multicenter clinical trial "CURE-HLH" to explore the application of JAK inhibitor ruxolitinib in refractory hemophagocytic syndrome.
The core breakthrough in China's diagnostic and treatment system for childhood hematological diseases lies in breaking disciplinary barriers, integrating social resources, and spanning the entire lifecycle, evolving from "solitary combat" to "ecological collaboration." As a leader in this process, Professor Sun Yuan has not only rewritten treatment paradigms through technologies such as autologous umbilical cord blood transplantation and related haploidentical transplantation but also extended high-quality medical resources to grassroots levels through standardized guidelines, hierarchical networks, and humanistic care systems. As of 2025, her team has completed over 1,900 transplants with a long-term survival rate of 75%, at only one-third the cost of Western countries. This exemplifies a truth: the ultimate value of medicine lies not in the dazzle of technology but in enabling every child to embrace life with dignity. As Professor Sun Yuan stated in Chinese Medical Journal, "We are building not just a diagnostic and treatment system but a nation's commitment to the future."