December 6–9, 2025, the 67th Annual Meeting of the American Society of Hematology (ASH) was held in Orlando, USA. As the world’s largest and most influential hematology conference, ASH annually brings together tens of thousands of experts to present the latest advances and breakthroughs in the field. At this year’s meeting, Prof. Rong Fu’s team from Tianjin Medical University General Hospital presented a series of important findings in aplastic anemia (AA), paroxysmal nocturnal hemoglobinuria (PNH), and myelodysplastic syndromes (MDS), while also addressing patients’ psychological health and rehabilitation—providing evidence for comprehensive clinical management.
Aplastic Anemia (AA)
01
Publication No.: 1425 Title: Ruxolitinib may treat aplastic anemia by reducing levels of macrophage pyroptosis First Author: Xintong Xu
Key Findings Experimental data indicate that ruxolitinib alleviates severe aplastic anemia (SAA) by reducing macrophage pyroptosis and improving the immune microenvironment. Both in vitro studies and mouse models showed significant suppression of pyroptosis-related factors and inflammatory responses. The effects may involve modulation of MHC II expression and the JAK/STAT pathway, influencing T-cell activation. Although further clinical validation and mechanistic studies are needed, this work offers a novel immunomodulatory approach for SAA.
02
Publication No.: 3195 Title: Efficacy and safety of romiplostim N01 combined with cyclosporine in refractory aplastic anemia: a multicenter phase II study First Author: Ting Wang
Key Findings Romiplostim N01 combined with cyclosporine demonstrated favorable efficacy and safety in refractory AA patients unresponsive to multiple TPO-RA plus IST regimens. Among patients completing 26 weeks of follow-up, the overall response rate reached 81.8%, with complete response increasing from 9.1% at week 13 to 18.2% at week 26. No treatment-related discontinuations due to adverse events were observed, supporting further exploration of this combination as a therapeutic option for refractory AA.
03
Publication No.: 4976 Title: Luspatercept combined with immunosuppressive therapy reduces inflammation in aplastic anemia by inhibiting monocyte pyroptosis First Author: Zining Wang
Key Findings This study shows that luspatercept exerts multi-target immunomodulatory effects in SAA by inhibiting the NF-κB/NLRP3 axis in monocytes. The treatment significantly reduced pyroptosis-related genes and pro-inflammatory cytokines in monocytes, while attenuating inflammatory cytokine secretion and cytotoxic activity of CD8⁺ T cells. These effects remodel the immune microenvironment, disrupt the inflammatory–pyroptotic vicious cycle, and ultimately promote hematopoietic recovery.
04
Publication No.: 4975 Title: The role and mechanism of exosomal aminopeptidase N (APN/CD13)-mediated CTL dysfunction in aplastic anemia First Author: Li Yan
Key Findings APN/CD13 is markedly overexpressed in SAA patients and may drive disease progression by promoting CTL proliferation and inflammatory cytokine production. Inhibition of CD13 reverses these effects, highlighting its therapeutic potential. This study identifies a novel candidate biomarker for SAA and provides new insights for personalized treatment strategies.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
01
Publication No.: 1112 Title: The role of neutrophil extracellular traps in thrombosis of paroxysmal nocturnal hemoglobinuria First Author: Mengting Che
Key Findings PNH patients exhibit significantly elevated levels of neutrophil extracellular traps (NETs) and platelet–neutrophil aggregates (PNAs), associated with heightened neutrophil sensitivity to PMA stimulation. NET burden correlates closely with thrombosis, suggesting that complement-triggered NETosis and platelet–neutrophil interactions synergistically amplify thrombus formation. NETs may serve as biomarkers for thrombotic risk and potential targets for combined complement/NET inhibition.
02
Publication No.: 4664 Title: Role of NK cells in immune escape in patients with classical paroxysmal nocturnal hemoglobinuria First Author: Chaomeng Wang
Key Findings The study reveals that activated normal NK cells contribute to immune escape in PNH. CD59⁺ NK cells display enhanced cytotoxicity and cytokine secretion, potentially facilitating clonal immune evasion, whereas CD59⁻ NK cells show functional impairment, reduced chemokine expression, and decreased proportions of mature NK cells. These alterations correlate with CD8⁺ T-cell proportions and disease severity, offering new insights into NK-cell dysfunction in PNH.
03
Publication No.: 1106 Title: Downregulation of TANK gene affects erythroid differentiation in PNH patients First Author: Liyan Li
Key Findings Single-cell sequencing revealed abnormal erythroid differentiation in PNH patients. During in vitro differentiation of patient-derived CD34⁺ cells to the CFU-E stage, TANK expression was significantly downregulated, suggesting a key regulatory role in erythropoiesis in PNH.
Myelodysplastic Syndromes (MDS)
01
Publication No.: 2059 Title: The USP22–NAP1L1 deubiquitination axis in the pathogenesis of myelodysplastic syndromes First Author: Lijuan Li
Key Findings USP22 stabilizes NAP1L1 via deubiquitination, maintaining its high expression in MDS CD34⁺ cells, thereby promoting proliferation, inhibiting apoptosis, and driving malignant clonal progression. Inhibition of the USP22–NAP1L1 axis reduces NAP1L1 stability and MDS cell viability, providing a mechanistically defined therapeutic strategy for MDS.
Other Research
01
Publication No.: 6423 Title: Construction of a mindfulness-based cancer rehabilitation training program for patients with malignant hematologic diseases based on the Liverpool mindfulness model Presenter: Jie Liu
Key Findings Based on the Liverpool mindfulness model, a structured mindfulness-based rehabilitation program for patients with hematologic malignancies was developed through systematic analysis of 17 studies, group discussions, qualitative interviews, and two rounds of Delphi expert consultation. The program demonstrates strong necessity, logical structure, scientific rigor, and clinical applicability, providing evidence-based support for psychological care in this patient population.
Expert Profile
Prof. Rong Fu Tianjin Medical University General Hospital
MD, Chief Physician, Second-Class Professor, PhD Supervisor Vice President, Tianjin Medical University General Hospital Director, Hematology Center Director, Tianjin Key Laboratory of Bone Marrow Failure and Malignant Hematopoietic Clonal Disorders Director, Tianjin Institute of Hematology Chair, Department of Hematology, Tianjin Medical University Director, Aplastic Anemia and Bone Marrow Failure Disease Center
Standing Committee Member, Chinese Society of Hematology (CSH) Head, Erythrocyte Disorders Group, CSH Standing Committee Member, Chinese Medical Doctor Association (Hematology Branch) Vice Chair, Physician Certification Committee, CMDA (Hematology) Chair, Red Cell Disease Committee, Beijing Association for Cancer Prevention and Treatment Deputy Head, China MDS/MPN Working Group, Chinese Anti-Cancer Association Vice Chair, Chinese Medical Association Hematology Institutions Branch Chair, Tianjin Society of Hematology
Editor-in-Chief, Journal of Clinical Laboratory Analysis (SCI) Deputy Editor-in-Chief, Chinese Journal of Hematology
Recipient of honors including Tianjin Distinguished Physician, Tianjin Distinguished Teaching Master, Tianjin Science Popularization Ambassador, and Tianjin Medical Talent Award.
