1.The Regulatory Effects and Mechanisms of Piezo1 Channel on Chondrocytes and Bone Metabolic Dysregulation in Osteoarthritis
Yan LI ; Tao LIU ; Yu-Biao GU ; Hui-Qing TIAN ; Lei ZHANG ; Bi-Hui BAI ; Zhi-Jun HE ; Wen CHEN ; Jin-Peng LI ; Fei LI
Progress in Biochemistry and Biophysics 2026;53(3):564-576
Osteoarthritis (OA), a highly prevalent degenerative joint disease worldwide, is defined by articular cartilage degradation, abnormal bone remodeling, and persistent chronic inflammation. It severely compromises patients’ quality of life, and currently, there is no radical cure. Abnormal mechanical stress is widely regarded as a core driver of OA pathogenesis, and the exploration of mechanical signal perception and transduction mechanisms has become crucial for deciphering OA’s pathophysiological processes. Piezo1, a key mechanosensitive cation channel belonging to the Piezo protein family, has recently gained significant attention due to its pivotal role in mediating cellular responses to mechanical stimuli in joint tissues. This review systematically examines Piezo1’s expression patterns, regulatory mechanisms, and pathological functions in OA, with a particular focus on its dual roles in modulating chondrocyte homeostasis and bone metabolism disorders, while also delving into the underlying molecular signaling pathways and potential therapeutic implications. Piezo1, consisting of approximately 2 500 amino acids and forming a unique trimeric propeller-like structure, is widely expressed in chondrocytes, osteocytes, mesenchymal stem cells, and synovial cells. It exhibits permeability to cations such as Ca2+, K+, and Na+, and directly responds to membrane tension changes induced by mechanical stimuli like fluid shear stress and mechanical overload. In OA patients and animal models, Piezo1 expression is significantly upregulated, especially in cartilage regions subjected to abnormal mechanical stress (e.g., human temporomandibular joint cartilage). This overexpression is closely associated with aggravated cartilage degeneration, increased chondrocyte apoptosis, accelerated cellular senescence, and intensified inflammatory responses. Mechanical overload and pro-inflammatory cytokines (e.g., IL-1β) are key inducers of Piezo1 upregulation: IL-1β activates the PI3K/AKT/mTOR signaling pathway to enhance Piezo1 expression, forming a pathogenic positive feedback loop that inhibits chondrocyte autophagy, promotes apoptosis, and further accelerates joint degeneration. Mechanistically, Piezo1 mediates OA progression through multiple interconnected pathways. When activated by mechanical stress, Piezo1 triggers excessive Ca2+ influx, leading to endoplasmic reticulum stress (ERS) and mitochondrial dysfunction, which directly induce chondrocyte apoptosis. This process involves the activation of downstream signaling cascades such as cGAS-STING and YAP-MMP13/ADAMTS5. YAP, a transcriptional regulator, upregulates the expression of matrix metalloproteinase 13 (MMP13) and aggrecanase (ADAMTS5), thereby accelerating cartilage matrix degradation. Additionally, Piezo1-driven Ca2+ overload promotes the accumulation of reactive oxygen species (ROS) and upregulates senescence markers (p16 and p21), accelerating chondrocyte senescence via the p38MAPK and NF-κB pathways. Senescent chondrocytes secrete senescence-associated secretory phenotype (SASP) factors (e.g., IL-6, IL-1β), further amplifying joint inflammation. In terms of bone metabolism, Piezo1 maintains joint homeostasis by promoting the differentiation of fibrocartilage stem cells into chondrocytes and balancing bone formation and resorption through regulating the FoxC1/YAP axis and RANKL/OPG ratio. Therapeutically, targeting Piezo1 shows promising potential. Preclinical studies have demonstrated that Piezo1 inhibitors (e.g., GsMTx4) can reduce joint damage and alleviate pain in OA mice. Simultaneously, siRNA-mediated co-silencing of Piezo1 and TRPV4 (another mechanosensitive channel) decreases intracellular Ca2+ concentration, inhibits chondrocyte apoptosis, and promotes cartilage repair. Conditional knockout of Piezo1 using Gdf5-Cre transgenic mice alleviates cartilage degeneration in post-traumatic OA models by downregulating MMP13 and ADAMTS5 expression. Despite existing challenges, such as off-target effects of inhibitors, inefficient local drug delivery, and interindividual genetic variability, strategies like developing selective Piezo1 antagonists, optimizing targeted nanocarriers, and combining Piezo1-targeted therapy with physical therapy provide viable avenues for clinical translation. The authors propose that Piezo1 serves as a critical therapeutic target for OA, and future research should focus on deciphering its context-dependent regulatory networks, developing tissue-specific intervention strategies, and validating their efficacy and safety in clinical trials to address the unmet medical needs of OA patients.
2.Comparison of Efficacy and Mechanism in Warming Yang and Dispersing Cold of Aconiti Radix Lateralis Praeparata Processed by ZHANG Zhongjing's Method and Pharmacopoeia Method
Mingjie JIAO ; Qian CHEN ; Shuyu YAN ; Yiyan SONG ; Jia ZHANG ; Fei LI
Chinese Journal of Experimental Traditional Medical Formulae 2026;32(2):207-217
ObjectiveTo investigate the therapeutic effects and mechanism of decoctions from four kinds of processed products of Aconiti Radix Lateralis Praeparata(ARLP) in deficiency-cold syndrome. MethodsA total of 36 SD rats were randomly divided into the control group, model group, Shengfupian(SFP) group, Paofuzi(PFZ) group, Heishunpian(HSP) group and Paofupian(PFP) group with 6 rats in each group. Except for the control group, rats in other groups were administered hydrocortisone sodium succinate via intramuscular injection to induce a cold deficiency syndrome model. After 14 consecutive days, each ARLP decoction pieces was administered via continuous gastric lavage at a dose of 12 g·kg-1·d-1 for 7 d, while the control and model groups received an equivalent volume of physiological saline. After the end of administration, body weight, spleen weight and thymus weight were measured for calculating the spleen and thymus indexes. Hematoxylin-eosin(HE) staining was used to observe the pathological morphology of adrenal tissue. The fully automatic biochemistry analyzer was used to measure the total cholesterol(TC), triglyceride(TG), lactic dehydrogenase(LDH) and lactate(LAC) levels in serum. Enzyme-linked immunosorbent assay(ELISA) was employed to measure the contents of 17-hydroxycorticosteroid(17-OHCS), cortisol(CORT), triiodothyronine(T3), thyroxine(T4), thyrotropin(TSH), immunoglobulin(Ig) M, IgG, cyclic adenosine monophosphate(cAMP) and cyclic guanosine monophosphate(cGMP). Western blot was used to measure the protein expression levels of protein kinase A(PKA), cAMP response element-binding protein(CREB), silent information regulator 1(Sirt1) and peroxisome proliferator-activated receptor γ coactivator-1α(PGC-1α). And high performance liquid chromatography(HPLC) was used to determine the content of major alkaloids, followed by Pearson correlation analysis with pharmacodynamic indicators. ResultsAfter modeling, compare with the control group, the model rats exhibited symptoms such as lethargy and loose stools, mild abnormalities were observed in adrenal tissue structure, and both spleen and thymus indices were significantly reduced(P<0.01). Thyroid, adrenal and immune system functions were suppressed, with decreased serum cAMP level and significantly elevated cGMP level(P<0.01). Compared with the model group, the adrenal injury by hydrocortisone sodium succinate were repaired and the spleen index were increased significantly in all four ARLP groups(P<0.05, P<0.01). The thymus index in SFP and PFZ groups were increased significantly(P<0.05). The contents of T3, TSH, 17-OHCS and CORT were increased significantly in SFP and PFZ groups(P<0.05). In addition, the content of IgG in SFP, PFZ and PFP groups were increased significantly(P<0.01), while the content of IgM in PFZ and HSP groups were also increased significantly(P<0.05). Regarding the cyclic nucleotide system, PFZ significantly elevated cAMP level while reducing cGMP level(P<0.05), exhibiting the most pronounced effect among the four decoction pieces. For energy metabolism indicators, PFZ significantly improved abnormal markers including TC, TG, LDH, and LAC(P<0.05). HSP showed marked improvement effects on TG, LDH, and LAC(P<0.05). Both PFZ and SFP significantly elevated the expression levels of PKA, CREB, Sirt1, and PGC-1α proteins(P<0.01). Additionally, the diester alkaloids in ARLP showed a strong positive correlation with TG, IgG, and CORT, a strong negative correlation with LAC, a moderate positive correlation with T4, and moderate negative correlations with cAMP and spleen index. Monomeric alkaloids showed strong positive correlations with TG and IgG, strong negative correlations with LAC, moderate positive correlations with CORT and T4, and moderate negative correlations with cAMP and spleen index. However, the content of water-soluble alkaloids showed strong positive correlations with TC, LDH, 17-OHCS, T3, TSH, and thymus index, moderate positive correlations with cAMP, CORT, T4, and spleen index, and moderate negative correlation with cGMP. ConclusionAmong different processed ARLP decoction pieces, PFZ processed according to ZHANG Zhongjing's method exhibits the most potent warming and cold-dispelling effects. Its pharmacological actions are mediated through regulating the thyroid, adrenal, immune, cyclic nucleotide systems, and material-energy metabolism pathways. Among these, water-soluble alkaloids show strong or moderate correlations with more indicators of deficiency-cold syndrome and exhibit the highest content in PFZ. Therefore, PFZ processed according to ZHANG Zhongjing's method may exert its warming and cold-dispelling effects through water-soluble alkaloids.
3.Study on accumulation of polysaccharide and steroid components in Polyporus umbellatus infected by Armillaria spp.
Ming-shu YANG ; Yi-fei YIN ; Juan CHEN ; Bing LI ; Meng-yan HOU ; Chun-yan LENG ; Yong-mei XING ; Shun-xing GUO
Acta Pharmaceutica Sinica 2025;60(1):232-238
In view of the few studies on the influence of
4.Xiaozhong Zhitong Mixture(消肿止痛合剂)Combined with Antibiotic Bone Cement in the Treatment of Diabetic Foot Ulcers with Damp-Heat Obstructing Syndrome:A Randomized Controlled Trial of 35 Patients
Xiaotao WEI ; Zhijun HE ; Tao LIU ; Zhenxing JIANG ; Fei LI ; Yan LI ; Jinpeng LI ; Wen CHEN ; Bihui BAI ; Xuan DONG ; Bo SUN
Journal of Traditional Chinese Medicine 2025;66(7):704-709
ObjectiveTo observe the clinical effectiveness and safety of Xiaozhong Zhitong Mixture (消肿止痛合剂) combined with antibiotic bone cement in the treatment of diabetic foot ulcer (DFU) with damp-heat obstructing syndrome. MethodsA total of 72 DFU patients with damp-heat obstructing syndrome were randomly assigned to treatment group (36 cases) and the control group (36 cases). Both groups received standard treatment and topical antibiotic bone cement for ulcer wounds, while the treatment group received oral Xiaozhong Zhitong Mixture (50 ml per time, three times daily) in additionally. Both groups underwent daily wound dressing changes for 21 consecutive days. Ulcer healing rate, serum levels of tumor necrosis factor-alpha (TNF-α), interleukin-1 beta (IL-1β), malondialdehyde (MDA), superoxide dismutase (SOD), C-reactive protein (CRP), and white blood cell (WBC) count were observed before and after treatment, and visual analog scale (VAS) scores for wound pain, traditional Chinese medicine (TCM) syndrome scores, and the DFU Healing Scale (DMIST scale) were also compared. Liver and kidney function were evaluated before and after treatment, and adverse events such as allergic reactions, worsening ulcer pain were recorded. ResultsTotally 35 patients in the treatment group and 33 in the control group were included in the final analysis. The ulcer healing rate in the treatment group was (87.93±9.34)%, significantly higher than (81.82±12.02)% in the control group (P = 0.035). Compared to pre-treatment levels, both groups showed significant reductions in serum CRP, WBC, MDA, IL-1β, and TNF-α levels, with an increase in SOD level (P<0.05). TCM syndrome scores, VAS, and DMIST scores also significantly decreased in both groups (P<0.05), with greater improvements in the treatment group (P<0.05). No significant adverse reactions were observed in either group during treatment. ConclusionXiaozhong Zhitong Mixture combined with antibiotic bone cement has significant advantages in promoting DFU healing, reducing inflammatory response, and alleviating oxidative stress in DFU patients with damp-heat obstructing syndrome, with good safety for DFU patients with damp-heat obstructing syndrome.
5.Influence of Shumian Capsules Combined with Quetiapine on Negative Symptoms,Sleep Quality and Neurotransmitters in Patients with Schizophrenia
Fei-yan CHEN ; Jian-yi XU ; Hong LIN
Progress in Modern Biomedicine 2025;25(18):2973-2980
Objective:This prospective study is conducted to explored the influence of Shumian Capsules combine with quetiapine on negative symptoms,sleep quality and neurotransmitters in patients with schizophrenia.Methods:50 patients with schizophrenia admitted to Fuzhou Gulou District Hospital of Fujian Province from April 2022 to May 2024 were retrospectively selected as the study group.According to the principle of 1∶1 matching cases,50 patients with schizophrenia in the same period were selected as the reference group.Reference group received quetiapine treatment,while study group was treated with Shumian capsules on the basis of the reference group.Negative symptom scale(SANS),positive and negative symptom scale(PANSS),Pittsburgh sleep quality index(PSQI),social-skills for psychiatric inpatients(SSPI),treatment emergent symptom scale(TESS),self-rating anxiety scale SAS),self-rating depression scale(SDS)score,neurotransmitter levels[γ-aminobutyric acid(GABA),brain-derived neurotrophic factor(BDNF),glutamic acid(Glu)and the occurrence of adverse reactions were compared between the two groups.Results:The scores of SANS,PANSS,PSQI,TESS,SAS,SDS and Glu levels decreased 4 weeks and 8 weeks after treatment in both groups,and study group were lower than those in reference group,SSPI score and GABA and BDNF levels increased,and study group were higher than those in the reference group(P<0.05).There was no significant difference in the incidence of adverse reactions between the two groups(P>0.05).Conclusion:Shumian Capsules combined with quetiapine can effectively improve negative symptoms,sleep quality and neurotransmitters in patients with schizophrenia,and have good safety.
6.Relationship between treatment time node of ST-segment elevation myocardial infarction and reperfusion microcirculation dysfunction and prognosis based on the establishment of chest pain center system
Fei FU ; Li-xia YANG ; Jun FAN ; Chang-zheng CHEN ; Yan-kun SHI
Chinese Journal of Interventional Cardiology 2025;33(7):376-382
Objective To investigate the relationship between ST-segment elevation myocardial infarction(STEMI)treatment time nodes and coronary microcirculation dysfunction(CMD)after primary percutaneous coronary intervention(PCI)and its prognosis,providing evidence for early identification of high-risk patients,further optimization of treatment system,shortening treatment time and improving prognosis.Methods This study selected 150 STEMI patients who received primary PCI at the 920th Hospital of the Joint Logistic Support Force of the People's Liberation Army of China from March 2023 to September 2023 as the CMD group after PCI(89 cases),with a microcirculation resistance index(caIMR)of≥40 U based on coronary angiography.caIMR<40 U was classified as the non-CMD group(61 cases).The clinical data,various examination indicators and imaging data of the two groups of patients were collected,and the occurrence of major adverse cardiovascular events(MACE)within half a year after direct PCI of the patients was followed up.The correlation between the two variables was analyzed using Spearman.Multivariate Logistic regression was used to analyze the relationship between each treatment time point and the occurrence of CMD after direct PCI.Using receiver operating characteristic(ROC)curve analysis to predict the occurrence of CMD after direct PCI.The survival curves between the two groups were plotted using the Kaplan-Meier method.Results The levels of symptom onset-to-balloon(S-to-B),symptom onset-to-first medical contact(S-to-FMC),first medical contact-to-balloon(FMC-to-B),symptom onset-to-door(S-to-D)and first medical contact-to-door(FMC-to-D)in the CMD group were significantly higher than those in the non-CMD group(all P<0.05).Correlation analysis showed that S-to-D,S-to-FMC,the time from FMC-to-B,and the time from FMC-to-D were all positively correlated with the time from S-to-B(r=0.996,P<0.001;r=0.937,P<0.001;r=0.431,P<0.001;r=0.441,P<0.001).Multivariate Logistic regression analysis indicated that the duration of S-to-D(OR 2.165,95%CI 1.655-2.830,P<0.001)was a significant influencing factor for CMD.Moreover,S-to-D has a relatively high predictive value for the occurrence of CMD after direct PCI.The area under the ROC curve is 0.898(95%CI 0.850-0.946,P<0.001),the optimal cut-off value is 231 min,the sensitivity is 82.0%,and the specificity is 88.5%.Kaplan-Meier curve analysis showed that the cumulative survival rate of MACE outcomes in the CMD group was significantly lower than that in the non-CMD group(Log-rank P=0.009).Conclusions Shortening the S-to-D time can reduce the occurrence of CMD after PCI,thereby reducing the incidence of MACE and improving prognosis.
7.Summary of experience in diagnosis and treatment of unexplained neonatal intestinal perforation
Weijun ZHENG ; Yifan FANG ; Dianming WU ; Lei YAN ; Fei CHEN ; Xiang WANG ; Yingjian CHEN ; Zhixiong LIN ; Mingkun LIU
Chinese Journal of Applied Clinical Pediatrics 2025;40(2):101-104
Objective:To summarize the experience of diagnosis and treatment of unexplained neonatal intestinal perforation.Methods:A case summary was conducted.Intestinal perforation children who showed unclear etiology during surgical exploration in Fujian Children′s Hospital from October 2017 to October 2023 were retrospectively analyzed.The perforation characteristics, pathological characteristics, etiological analysis and surgical methods were analyzed, and the diagnosis and treatment experience was summarized.Results:There were 21 neonates with unexplained intestinal perforation, including 15 boys and 6 girls.There were more full-term infants (12 cases) than preterm ones (9 cases), and more neonates with normal birth weight (12 cases) than those with low birth weight (9 cases).Intraoperative perforation was detected in the ileum in 13 cases and colon in 8 cases.The perforation diameter ranged between 0.5-2.0 cm.Single perforation was detected in 20 cases, and 1 case had two perforations.All patients underwent full-layer multipoint biopsy and enterostomy.Pathological reports after surgery showed that Hirschsprung disease was confirmed in 10 cases, and Hirschsprung disease allied disorders were diagnosed in 8 cases.The remaining 3 cases had normal ganglion cells, and 2 cases of them had spontaneous intestinal perforation, and 1 case had meconium ileus.After operation, 3 cases gave up treatment, 1 case died, and 17 patients were discharged from hospital at the first stage.Nine cases with Hirschsprung disease received radical surgery at the age of 3-6 months.Six out of 8 patients with Hirschsprung disease allied disorders underwent fistula closure surgery after accurate evaluation.Among the 6 children who received fistula closure surgery, 1 case underwent Santulli enterostomy because of inability to defecate and abdominal distension after surgery.Two of the 6 children undergoing fistula closure surgery were still being followed up.Two cases of spontaneous intestinal perforation and 1 case of meconium ileus underwent fistula closure surgery at the age of 3 months, and postoperative defecation was normal.Conclusions:Intestinal neuronal dysplasia, meconium ileus combined with intestinal perforation and spontaneous intestinal perforation should be considered in unexplained neonatal intestinal perforation.It is recommended that enterostomy and multipoint biopsy should be performed to clarify the pathology before further treatment in children with unexplained intestinal perforation.Santulli enterostomy is a feasible staging operation.
8.Transcriptomic Studies Reveal a New Mechanism by Which miR-3910 Promotes Neuroblastoma Progression
Jia-Jia LI ; Yan-Mei JIA ; Yu-Ling LIU ; Fei LIU ; Hui-Ru WANG ; Li-Rong CHEN
Chinese Journal of Biochemistry and Molecular Biology 2025;41(7):1019-1030
Neuroblastoma(NB),the most common type of extracranial solid tumor in children,is char-acterized by high malignancy and poor prognosis,warranting in-depth investigation.In recent years,mi-croRNAs(miRNAs)have emerged as crucial post-transcriptional regulators playing pivotal roles in tu-morigenesis and progression.Building upon this background,the present study specifically focuses on in-vestigating miR-3910's biological functions and underlying molecular regulatory mechanisms in the NB SH-SY5Y cell line.Through bioinformatics analysis and transcriptome sequencing,we identified potential key target molecules of miR-3910,thereby providing genetic targets for the precise diagnosis and effective treatment of NB.In this study,qRT-PCR was employed to measure miR-3910 expression levels in SH-SY5Y cells transfected with mimic negative control and miR-3910 mimic.Compared to the nc group,miR-3910 expression was significantly upregulated in the mimic group(P<0.01).The CCK-8 assay and scratch wound healing assay were used to quantitatively assess the impact of miR-3910 on cell prolif-eration and migration.Results showed that cell proliferation significantly increased at 48 h(P<0.05),and migration ability was markedly enhanced at 48 h(P<0.01).Flow cytometry was applied to deter-mine the effect of miR-3910 on cell cycle progression,revealing accelerated cell cycle progression,a re-duced proportion of G0/G,phase cells(P<0.01),and a significant increase in S-phase cells(P<0.05).Integrated bioinformatics analysis and high-throughput transcriptome sequencing predicted key molecular changes in SH-SY5Y cells following miR-3910 overexpression.Transcriptome sequencing and bioinformatics analysis identified six NB-related genes:EIF3CL(EIF3C),RNF103-CHMP3(VPS24),SULT1A4(SULT1A4),CORO7-PAM16(CORO7),H4C12(Histone H4),and TBC1D3(TBC1D3A/B/C)(aliases sourced from the GeneCards database).qRT-PCR and Western blotting(WB)results are consistency with sequencing results(P<0.01).In conclusion,miR-3910 overexpression significantly promotes SH-SY5Y cell proliferation,migration,and cell cycle progression,while uncovering a series of potential key target molecules.These findings provide new insights into the pathogenesis of NB and offer a theoretical foundation and potential intervention targets for molecular-targeted therapy in NB.
9.Clinical and prognostic analysis of nine cases of immune checkpoint inhibitor-related Stevens-Johnson syndrome/toxic epidermal necrolysis
Yongjun WAN ; Haijing YANG ; Qiao YAN ; Mei CHEN ; Fengyuan WANG ; Qianya SU ; Zhengbang DONG ; Fei WANG
Chinese Journal of Dermatology 2025;58(4):347-351
Objective:To summarize clinical characteristics and prognosis of immune checkpoint inhibitor (ICI) -related Stevens-Johnson syndrome (SJS) /toxic epidermal necrolysis (TEN) .Methods:A retrospective analysis was conducted on patients diagnosed with ICI-related SJS/TEN in Zhongda Hospital, Southeast University from January 2018 to October 2023. Data on clinical manifestations, laboratory examinations, treatment and prognosis of the patients were analyzed.Results:A total of 9 patients were diagnosed with ICI-related SJS/TEN, including 8 males and 1 female, with the onset age ranging from 58 to 77 (67.56 ± 7.33) years. ICI were applied to all the 9 patients before onset of SJS/TEN. The latent period was 6 - 261 d, and the median duration was 76 d. All the patients presented with erythema as initial lesions, 3 cases with target lesions, 6 with blisters, and 5 cases with a positive Nikolsky's sign. Oral mucosal damage occurred in 7 cases, eye damage occurred in 6 cases, and urogenital damage occurred in 6 cases. All the 9 cases were treated with systematic glucocorticoids, 7 cases with intravenous immunoglobulin and 7 cases with antibiotics. Eight cases recovered and 1 case died. Among 6 patients followed for an average of 6.92 months, none achieved complete or partial remission, 3 died and 3 experienced disease progression.Conclusions:ICI-related SJS/TEN occurred with a relatively long latent period, and all the cases presented with erythema initially, with mucosal damage present in most of the cases. Management required discontinuation of ICI, and most patients recovered with corticosteroids and intravenous immunoglobulin, however, the prognosis for the primary malignancy remained poor.
10.Impact of high-risk cytogenetic abnormalities on the efficacy of allogeneic hematopoietic stem cell transplantation with post-transplantation Cyclophosphamide in pediatric patients with acute myeloid leukemia
Fei LONG ; Zhi CHEN ; Li YANG ; Zhuo WANG ; Yan CHEN ; Yu DU ; Wenjie LU ; Ming SUN ; Shanshan QI ; Hao XIONG
Chinese Journal of Applied Clinical Pediatrics 2025;40(12):939-945
Objective:To explore the influence of initial high-risk cytogenetic abnormalities on the outcomes of children with acute myeloid leukemia (AML) after post-transplant Cyclophosphamide (PTCy)-based allogeneic hematopoietic stem cell transplantation (allo-HSCT).Methods:A retrospective cohort study.AML children who underwent PTCy-based allo-HSCT after the first complete remission at Wuhan Children′s Hospital, Tongji Medical College, Huazhong University of Science and Technology between April 2017 and April 2024 were enrolled.Patients were divided into intermediate-risk and high-risk groups based on their initial cytogenetic features.These patients were further divided into complex karyotype, 11q23 rearrangement, and other karyotype groups.Clinical characteristics and survival outcomes were compared among these groups.Measurement and count data were analyzed using Wilcoxon rank-sum/Kruskal-Wallis and χ2 tests, respectively.Survival and risk factor analyses were performed using Kaplan-Meier and Cox proportional hazards methods, respectively. Results:A total of 51 AML children who underwent allo-HSCT were included in this study.The median age at transplantation was 3.2 years and the median follow-up time was 4.6 years.There were 26 cases in the intermediate-risk group and 25 cases in the high-risk group; 8 cases in the complex karyotype group, 14 cases in the 11q23 rearrangement group, and 29 cases in the other karyotype groups.By the end of the follow-up on November 30, 2024, 11 patients relapsed, 8 patients died, and 13 patients developed grades Ⅱ-Ⅳ acute graft-versus-host disease (GVHD).The 3-year overall survival (OS), relapse-free survival (RFS), and grades Ⅱ-Ⅳ acute GVHD-free and relapse-free survival (GRFS) were 84.0% (95% CI: 74.4%-94.8%), 74.5% (95% CI: 63.4%-87.5%), and 58.8% (95% CI: 46.7%-74.0%), respectively.The 3-year OS of the high-risk group was significantly lower than that of the intermediate-risk group (71.8% vs.96.2%, P=0.022), while differences in 3-year RFS and GRFS between the 2 groups were not statistically significant (68.0% vs.80.8%, P=0.400; 52.0% vs.65.4%, P=0.420).The 3-year OS, RFS and GRFS of the complex karyotype group were significantly lower than those of 11q23 rearrangement and other karyotype groups (50.0% vs.85.7%, 93.1%, P=0.009; 37.5% vs.85.7%, 79.3%, P=0.022; 25.0% vs.64.3%, 65.5%, P=0.049).Multivariate analysis showed that a complex karyotype was an independent prognostic factor affecting 3-year OS and GRFS [OS: HR=6.79 (95% CI: 1.13-43.80), P=0.044; GRFS: HR=3.72(95% CI: 1.13-12.20), P=0.030]. Conclusions:High-risk cytogenetic features are significant predictors of survival outcomes in pediatric AML patients undergoing PTCy-based allo-HSCT.

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