Editor’s Note: Severe aplastic anemia (SAA) has a sudden onset, severe symptoms, and progresses rapidly. With deepening understanding and continuous breakthroughs in research, hematopoietic stem cell transplantation (HSCT) has been applied in this field, significantly improving clinical efficacy and prognosis for patients. At the recent 19th National Conference on Red Blood Cell Disorders (Anemia) held in China, Professor Shunqing Wang from The First People’s Hospital of Guangzhou shared insights into the diagnosis and treatment challenges of SAA, advancements in hematopoietic stem cell transplantation therapy, and highlights from the conference.
Oncology Frontier – Hematology Frontier: The First People’s Hospital of Guangzhou ranks among the top in the country for HSCT. Could you please provide an overview of the current status and challenges in the diagnosis and treatment of aplastic anemia (AA) in China based on clinical practice?
Professor Shunqing Wang: Currently, the treatment of AA mainly includes two approaches: allogeneic hematopoietic stem cell transplantation (allo-HSCT) and immunosuppressive therapy (IST). IST has made significant progress, evolving from classic antithymocyte globulin (ATG) combined with cyclosporine to the current three-drug therapy combined with thrombopoietin receptor agonists (TPO-RAs), with improvements in overall response rate (ORR), complete response rate (CR), and onset time.
Allo-HSCT for treating SAA has also progressed rapidly. In addition to the classic matched sibling donor HSCT (MSD-HSCT), alternative donor HSCT has also developed rapidly, including matched unrelated donor HSCT (MUD-HSCT) and haploidentical donor HSCT (HID-HSCT). The widely used Beijing protocol for HID-HSCT has been globally adopted, showing comparable efficacy to MSD-HSCT, significantly improving overall survival (OS), failure-free survival (FFS), and quality of life for patients. Therefore, HID-HSCT is recommended as a first-line treatment for SAA by consensus among Chinese HSCT experts.
MUD-HSCT requires not only transplantation technology but also the identification of suitable stem cell donors in bone marrow registries. Currently, the China Bone Marrow Bank provides a rapid donor search channel, greatly increasing the possibility and accessibility of first-line MUD-HSCT treatment. The First People’s Hospital of Guangzhou has extensive experience in this field. MUD-HSCT shows similar efficacy to matched sibling donor transplantation and has advantages over IST, such as FFS. Overall, MUD-HSCT has many advantages in first-line treatment for adults, providing clinicians with new insights.
Oncology Frontier – Hematology Frontier: Breakthroughs continue to be made in the treatment of AA transplantation in China. What do you think are the urgent issues to be addressed currently? Do you have any recommendations for selecting treatment drugs?
Professor Shunqing Wang: Currently, IST or allo-HSCT each have their own advantages and drawbacks. IST has relatively good safety, but its efficacy, especially the CR rate, is low, leading to incomplete cure for some patients. Allo-HSCT can completely cure severe AA, with a higher cure rate, but it carries relatively high transplantation risks, including graft-versus-host disease (GVHD) and infection. Reducing GVHD after allo-HSCT, improving the efficacy of IST, and selecting suitable patients for IST or allo-HSCT are current research hotspots and challenges.
Additionally, the quality of life of patients after AA treatment is also a key concern. If IST can only achieve partial remission, especially for children or young patients, it may affect their future education, work, and family life. Therefore, we usually adopt a more proactive treatment strategy for these young patients. Adding new drugs such as TPO-RAs during IST or allo-HSCT to accelerate platelet recovery and improve efficacy is also being actively explored. Many institutions are also actively exploring umbilical cord blood transplantation, and the protocol at Anhui Provincial Hospital has shown promising initial results. For young women, consideration of ovarian function and fertility-related issues is necessary to truly improve the quality of life of patients after transplantation.
Oncology Frontier – Hematology Frontier: As an attendee of this conference, could you share some highlights from the event?
Professor Shunqing Wang: There were many highlights at this conference: Firstly, there was a large number of attendees and enthusiastic discussions. Secondly, a special emphasis was placed on basic research, with many domestic experts in red blood cell disease basic research invited to participate, promoting full communication between clinicians and basic experts. Another highlight of this conference was the youth forum, where young doctors showed greater innovation and enthusiasm, bringing forth many new perspectives and discussions. Additionally, this year’s conference included a sub-session on paroxysmal nocturnal hemoglobinuria (PNH). Price reductions and inclusion in medical insurance have made drugs more accessible, and new drugs (such as complement inhibitors) continue to emerge. Therefore, experts at the conference discussed advances in PNH diagnosis and treatment, comprehensive patient management, standardized diagnosis and treatment, and the promotion of the construction of domestic PNH centers of excellence, continuously improving the quality of life of patients.
Expert Profile
Professor Shunqing Wang
– Director of Internal Medicine and Hematology at the First People’s Hospital of Guangzhou
– Member of the Hematology Physician Branch of the Chinese Medical Association
– Member of the Red Blood Cell Disease Group of the Hematology Branch of the Chinese Medical Association
– Deputy Director of the Hematology Branch of the Guangdong Medical Association
– Deputy Director of the Hematology Physician Branch of the Guangdong Medical Association
– Chairman of the Hematology Branch of the Guangzhou Medical Association
– Director of the Hematology Disease Medical Quality Control Center of Guangzhou
– Member of the Hematologic Rehabilitation Professional Committee of the Chinese Rehabilitation Medicine Association
– Member of the Hematology Branch of the Chinese Geriatrics Society
– Member of the Hematologic Disease Special Committee of the Cross-Strait Medical Exchange Association
– Editorial Board Member of Blood (Chinese Edition) and “Thrombosis and Hemostasis”
