The official implementation of the Regulations on Clinical Research and Translation of New Biomedical Technologies (State Council Decree No.818, hereinafter referred to as the "Regulations") marks a new stage of legal governance, standardization, and whole-chain regulation for emerging biomedical fields such as cell and gene therapy (CGT) in China, exerting a profound impact on the development of transfusion medicine as a discipline. Within the framework of the core regulatory systems established by the Regulations, and drawing upon the extensive experience of transfusion medicine in comprehensive quality control, cell processing and handling, ethical compliance, and clinical translation, this study systematically elucidates the intrinsic alignment between transfusion medicine and CGT with respect to technical workflows, quality control rationale, and management systems. It further clarifies the discipline-specific advantages of the department of transfusion medicine to proactively undertake CGT engagement, while analyzing current shortcomings and transformational bottlenecks. By leveraging the existing institutional framework, the department of transfusion medicine is well positioned to evolve into a core platform for whole-process quality control and a pivotal hub for in-hospital clinical translation of CGT. However, this transformation must concurrently address multiple challenges, including heightened compliance responsibilities, lagging technical infrastructure, a shortage of interdisciplinary talent, and increasingly stringent standards for clinical research and translation. The Regulations provide a legal basis and policy support for the department of transfusion medicine to transcend its traditional, singular function of blood safety assurance and establish a synergistic development model integrating blood safety and cell therapy. Therefore, the department of transfusion medicine should proactively align with regulatory requirements, refine whole-process compliance systems, upgrade technical platforms, and cultivate interdisciplinary professionals to achieve high-quality transformative development as a core supporting discipline for biotherapy.

Platelet-derived exosomes (PLT-Exos) are a subgroup of nanoscale extracellular vesicles released by platelets upon activatin. As key intercellular signaling mediators, they precisely regulate target cells through the delivery of functional molecules, exerting multiple biological effects that promote tissue repair. Accumulating evidence has demonstrated that PLT-Exos significantly facilitate the repair of various tissue injuries by modulating inflammatory responses, stimulating angiogenesis, activating key repair signaling pathways, and mediating stem cell homing. Furthermore, owing to their inherent targeting capacity and low immunogenicity, PLT-Exos have emerged as a promising platform for novel drug delivery carriers in tissue repair. This article systematically reviews the biological characteristics of PLT-Exos, their isolation and identification techniques, the underlying mechanisms of action, and recent advances in clinical application. It also addresses the current challenges in clinical translation, including standardized preparation, subpopulation heterogeneity, scalable production, and clinical implementation, aiming to provide a theoretical reference for both basic research and clinical translation of PLT-Exos in tissue repair.

Knee osteoarthritis (KOA) is a prevalent degenerative joint disease characterized by synovial inflammation, cartilage loss. Often manifesting as joint pain and limited mobility, it severely affects the quality of life of patients. Traditional treatment methods such as pharmacological injections and surgical interventions primarily aim to alleviate symptoms but have limited effects on cartilage repair. Human umbilical cord mesenchymal stem cells (hUC-MSCs), due to their anti-inflammatory and chondrogenic capabilities, is considered a new hope for the treatment of KOA. This article synthesizes the latest research findings from both domestic and international sources to discuss the theoretical basis for the clinical application of hUC-MSCs in treating KOA, clinical study design, and efficacy evaluation. It also addresses the challenges in the clinical application of hUC-MSCs and explores future directions, in the hope of providing feasible theoretical support for the treatment of KOA with hUC-MSCs.

[Objective] To explore the strategy of blood management in patients with massive transfusion in the frigid plateau region. [Methods] The treatment process of a patient with liver rupture in the frigid plateau region was analyzed, and the blood management strategy of the frigid plateau region was discussed in combination with the difficulties of blood transfusion and literature review. [Results] The preoperative complete blood count (CBC) test results of the patient were as follows: RBC 3.14×10¹²/L, Hb 10⁶ g/L, HCT 30.40%, PLT 115.00×10⁹/L; coagulation function: PT 18.9 s, FiB 1.31 g/L, DD > 6 μg/mL, FDP 25.86 μg/mL; ultrasound examination and imaging manifestations suggested liver contusion and laceration / intraparenchymal hematoma, splenic contusion and laceration, and massive blood accumulation in the abdominal cavity; it was estimated that the patient's blood loss was ≥ 2 000 mL, and massive blood transfusion was required during the operation; red blood cell components were timely transfused during the operation, and the blood component transfusion was guided according to the patient's CBC and coagulation function test results, providing strong support and guarantee for the successful treatment of the patient. The patient recovered well after the operation, and the CBC test results were as follows: RBC 4.32×10¹²/L, Hb 144 g/L, HCT 39.50%, PLT 329.00×10⁹/L; coagulation function: APTT 29.3 s, PT 12.1 s, FiB 2.728 g/L, DD>6 μg/mL, FDP 25.86 μg/mL. The patient was discharged after 20 days, and regular follow-up reexamination showed no abnormal results. [Conclusion] Individualized blood management strategy should comprehensively consider the patient’s clinical symptoms, the degree of hemoglobin decline, dynamic coagulation test results and existing treatment conditions. Efficient and reasonable patient blood management strategies can effectively improve the clinical outcomes of massive transfusion patients in the frigid plateau region.

Objective To evaluate the value of 68Ga-DOTATATE and 18F-FDG PET/CT imaging in staging and treatment decision for gastroenteropancreatic neuroendocrine neoplasms(GEP-NEN).Methods This retrospective analysis was conducted in 49 patients with GEP-NEN undergoing 18F-FDG and 68Ga-DOTATATE PET/CT imaging at our hospital from August,2020 to March,2023,including 34 newly diagnosed patients and 15 patients with recurrence or metastasis after treatment.GEP-NEN were classified into G1,G2,and G3 neuroendocrine tumors(NET)and neuroendocrine carcinomas(NEC)based on pathological typing.The detection efficiency were classified into 4 patterns based on the number of positive tumor lesions detected by the two tracers:68Ga-DOTATATE>18F-FDG(A);68Ga-DOTATATE=18F-FDG(B);68Ga-DOTATATE<18F-FDG(C);and complementation(D).The value of dual-modality imaging in staging and treatment decision were evaluated by visual analysis.Results In the 49 patients with GEP-NEN,68Ga-DOTATATE PET/CT was superior to 18F-FDG PET/CT for detecting systemic tumor lesions(P<0.001)and more sensitive for detecting primary/recurrent lesions,lymph node metastasis,liver metastasis,and bone metastasis(P<0.05),while 18F-FDG PET/CT had higher detection rates for lung metastasis and peritoneal metastasis(P<0.05).In terms of the detection efficiency,Pattern A was found in 46.9%(23/49)patients,Pattern B in 38.8%(19/49),Pattern C in 12.2%(6/49),and Pattern D in 2.0%(1/49).The complementary value of 18F-FDG PET/CT to 68Ga-DOTATATE PET/CT was 0%in G1 NET patients(0/13),8.3%in G2 NET patients(2/24),50%in G3 NET patients(3/6),and 33.3%in NEC patients(2/6).12.2%(6/49)of the patients had their staging confirmed or changed due to additional lesions detected by 18F-FDG PET/CT imaging,resulting subsequently in establishment or adjustment of their treatment plans.Conclusion 68Ga-DOTATATE PET/CT imaging should be the primary choice for GEP-NEN patients.Additional 18F-FDG PET/CT imaging can potentially improve precision of staging and treatment decision-making for G2,G3 and NEC patients but provides virtually no clinical benefits for G1 NET patients.

OBJECTIVE: To evaluate the differential expression of RNA in blood monocytes in patients with Juvenile idiopathic arthritis (JIA) treated with TNF antagonists (TNFi), and to explore the effect and mechanism of gene expression on the efficacy of JIA. METHODS: A total of 29 children with JIA treated with methotrexate (MTX) and TNFi in Guangzhou Women and Children's Medical Center of Guangzhou Medical University from April 2021 to November 2023 were enrolled. After 6 months, the children were divided into two groups according to the treatment effect, i.e., 13 cases in the ineffective group and 16 cases in the effective group, the peripheral blood of the children was collected, the blood mononuclear cells were isolated for transcriptome sequencing, the differentially expressed genes between the groups were analyzed, the signaling pathways and metabolic pathways related to the efficacy of TNFi were analyzed by GO and KEGG enrichment, and the mechanism related to the efficacy of TNFi was explored. This study was approved by Medical Ethics Committee of the Guangzhou Women and Children's Medical Center of Guangzhou Medical University (Ethics No.: 2023-330B00). RESULTS: There was a statistically significant difference in the gender and age distribution between the two groups of children (P < 0.05), while no statistically significant differences were observed in disease duration, rheumatoid antibody levels, or JIA subtypes (P > 0.05). After sequencing data quality control and comparison of reference genomes, a total of 18 523 protein-coding genes were identified in all children's samples. A total of 705 differentially expressed genes (DEGs) were identified between the effective group and the invalid group through differential analysis, of which 579 were up-regulated in the effective group and 126 in the inactive group. GO function and KEGG pathway enrichment analysis showed that DEG was significantly enriched in 55 GO entries and 32 KEGG metabolic pathways, which were mainly related to IL-1β production and regulation, cytokine production and regulation, cytokine-cytokine receptor interaction, immune response regulation, and Toll-like receptor signaling pathway. CONCLUSION DEG between the effective and ineffective groups of TNFi treatment may be involved in the biological processes such as cytokine production and regulation, cytokine-receptor interaction, and immune response regulation, which will be helpful to predict the efficacy and prognosis of TNFi treatment for JIA.
Humans ; Arthritis, Juvenile/blood* ; Female ; Male ; Child ; Methotrexate/therapeutic use* ; Child, Preschool ; Tumor Necrosis Factor-alpha/antagonists & inhibitors* ; Transcriptome ; Adolescent ; RNA/genetics* ; Signal Transduction ; Gene Expression Profiling

Objective:To evaluate the differential expression of RNA in blood monocytes in patients with Juvenile idiopathic arthritis (JIA) treated with TNF antagonists (TNFi), and to explore the effect and mechanism of gene expression on the efficacy of JIA.Methods:A total of 29 children with JIA treated with methotrexate (MTX) and TNFi in Guangzhou Women and Children′s Medical Center of Guangzhou Medical University from April 2021 to November 2023 were enrolled. After 6 months, the children were divided into two groups according to the treatment effect, 13 cases in the ineffective group and 16 cases in the effective group, the peripheral blood of the children was collected, the blood mononuclear cells were isolated for transcriptome sequencing, the differentially expressed genes between the groups were analyzed, the signaling pathways and metabolic pathways related to the efficacy of TNFi were analyzed by GO and KEGG enrichment, and the mechanism related to the efficacy of TNFi was explored. This study was approved by Medical Ethics Committee of the Guangzhou Women and Children′s Medical Center of Guangzhou Medical University (Ethics No.: 2023-330B00).Results:There was a statistically significant difference in the gender and age distribution between the two groups of children ( P<0.05), while no statistically significant differences were observed in disease duration, rheumatoid antibody levels, or JIA subtypes ( P> 0.05). After sequencing data quality control and comparison of reference genomes, a total of 18 523 protein-coding genes were identified in all children′s samples. A total of 705 differentially expressed genes (DEGs) were identified between the effective group and the invalid group through differential analysis, of which 579 were up-regulated in the effective group and 126 in the inactive group. GO function and KEGG pathway enrichment analysis showed that DEG was significantly enriched in 55 GO entries and 32 KEGG metabolic pathways, which were mainly related to IL-1β; production and regulation, cytokine production and regulation, cytokine-cytokine receptor interaction, immune response regulation, and Toll-like receptor signaling pathway. Conclusion:DEG between the effective and ineffective groups of TNFi treatment may be involved in the biological processes such as cytokine production and regulation, cytokine-receptor interaction, and immune response regulation, which will be helpful to predict the efficacy and prognosis of TNFi treatment for JIA.

Objective To investigate the value of fragmented QRS complex(fQRS)in the electro-cardiogram,heart rate variability(HRV)and LVEF in evaluating the prognosis in patients with dilated cardiomyopathy(DCM).Methods A retrospective analysis was conducted on 59 DCM pa-tients admitted in the Second Affiliated Hospital of Wannan Medical College from January 2020 to December 2023.According to the occurrence of MACE during 6-month follow-up period,they were classified into a poor prognosis group(26 cases)and a good prognosis group(33 cases).Clin-ical baseline data,positive rate of fQRS,HRV and LVEF were compared between the two groups.Time-domain measurements of HRV included standard deviation of normal NN intervals(SDNN),standard deviation of the averages of NN intervals in all 5 min segments of the entire recording(SDANN),mean of the standard deviation of NN intervals for all 5 min segments of the entire recording(SDNN index),root mean square of standard deviation of NN intervals(r-MSSD)and HRV triangular index.Spearman/Pearson correlation analysis was performed to analyze the correlation of prognosis of DCM with positive rate of fQRS,HRV and LVEF.ROC curve was drawn to analyze the efficiency of fQRS,HRV and LVEF in predicting the prognosis of DCM.Results The poor prognosis group exhibited significantly higher positive rate of fQRS and obvi-ously reduced SDNN,SDANN,SDNN index,r-MSSD,triangular index and LVEF when compared with the good prognosis group(P＜0.01).Correlation analysis suggested that poor prognosis of DCM was positively correlated with the positive rate of fQRS(P＜0.01),and negatively with SDNN,SDANN,SDNN index,r-MSSD,triangular index and LVEF(P＜0.05,P＜0.01).ROC curve analysis revealed that the AUC value of above indicators in turn in predicting the prognosis of DCM was 0.718,0.7 56,0.7 62,0.807,0.858,0.805 and 0.747,respectively,and the AUC value of their combination was 0.980(P＜0.01).Conclusion fQRS,HRV and LVEF have important cor-relation with poor prognosis of DCM patients.Their combination can be used as an effective mark-er for clinical evaluation and prediction of poor prognosis of DCM.

OBJECTIVES: To evaluate the value of ⁶⁸Ga-DOTATATE and ¹⁸F-FDG PET/CT imaging in staging and treatment decision for gastroenteropancreatic neuroendocrine neoplasms (GEP-NEN). METHODS: This retrospective analysis was conducted in 49 patients with GEP-NEN undergoing ¹⁸F-FDG and ⁶⁸Ga-DOTATATE PET/CT imaging at our hospital from August, 2020 to March, 2023, including 34 newly diagnosed patients and 15 patients with recurrence or metastasis after treatment. GEP-NEN were classified into G1, G2, and G3 neuroendocrine tumors (NET) and neuroendocrine carcinomas (NEC) based on pathological typing. The detection efficiency were classified into 4 patterns based on the number of positive tumor lesions detected by the two tracers: ⁶⁸Ga-DOTATATE>¹⁸F-FDG (A); ⁶⁸Ga-DOTATATE=¹⁸F-FDG (B); ⁶⁸Ga-DOTATATE<¹⁸F-FDG (C); and complementation (D). The value of dual-modality imaging in staging and treatment decision were evaluated by visual analysis. RESULTS: In the 49 patients with GEP-NEN, ⁶⁸Ga-DOTATATE PET/CT was superior to ¹⁸F-FDG PET/CT for detecting systemic tumor lesions (P<0.001) and more sensitive for detecting primary/recurrent lesions, lymph node metastasis, liver metastasis, and bone metastasis (P<0.05), while ¹⁸F-FDG PET/CT had higher detection rates for lung metastasis and peritoneal metastasis (P<0.05). In terms of the detection efficiency, Pattern A was found in 46.9% (23/49) patients, Pattern B in 38.8% (19/49), Pattern C in 12.2% (6/49), and Pattern D in 2.0% (1/49). The complementary value of ¹⁸F-FDG PET/CT to ⁶⁸Ga-DOTATATE PET/CT was 0% in G1 NET patients (0/13), 8.3% in G2 NET patients (2/24), 50% in G3 NET patients (3/6), and 33.3% in NEC patients (2/6). 12.2% (6/49) of the patients had their staging confirmed or changed due to additional lesions detected by ¹⁸F-FDG PET/CT imaging, resulting subsequently in establishment or adjustment of their treatment plans. CONCLUSIONS ⁶⁸Ga-DOTATATE PET/CT imaging should be the primary choice for GEP-NEN patients. Additional ¹⁸F-FDG PET/CT imaging can potentially improve precision of staging and treatment decision-making for G2, G3 and NEC patients but provides virtually no clinical benefits for G1 NET patients.
Humans ; Positron Emission Tomography Computed Tomography/methods* ; Neuroendocrine Tumors/therapy* ; Pancreatic Neoplasms/therapy* ; Retrospective Studies ; Organometallic Compounds ; Stomach Neoplasms/therapy* ; Neoplasm Staging ; Fluorodeoxyglucose F18 ; Intestinal Neoplasms/therapy* ; Female ; Male ; Middle Aged ; Aged ; Adult

Kleine-Levin Syndrome(KLS)is a rare neurological disease characterized by recurrent episodes of hypersomnia, hyperphagia, hypersexuality, and cognitive and behavioral abnormalities, with complete functional recovery between episodes. Its pathogenesis remains unclear. We report a 33-year-old female with a 17-year clinical course, characterized mainly by recurrent hypersomnia. During episodes, the patient exhibited prominent anorexia, irritability, derealization, and disorientation, followed by brief periods of excitement after episode resolution. Inter-episode periods were entirely normal. Long-term polysomnography monitoring was conducted for a total of 7 792 minutes. Unlike the typical hyperphagia commonly observed in KLS, this case was marked by prominent anorexia, underscoring the clinical heterogeneity of KLS.This report adds to the limited documentation of rare cases in China.