1.Clinical characteristics and efficacy of glyceryl phenylbutyrate treatment in 20 pediatric patients with urea cycle disorder
Wenjuan QIU ; Chengkai SUN ; Yuan XIAO ; Xiaoyan HUANG ; Cui SONG ; Jin WU ; Haiyan WEI ; Liwen WU ; Dan YU ; Hongwei DU ; Chen LIU ; Xuefan GU
Chinese Journal of Pediatrics 2025;63(9):1005-1010
Objective:To investigate the clinical characteristics of urea cycle disorder (UCD), the efficacy and safety of glyceryl phenylbutyrate (GPB) therapy in pediatric patients with UCD.Methods:This study was a retrospective, single-arm, multicenter clinical study. The clinical data of 20 pediatric patients with UCD who received GPB treatment at 9 hospitals nationwide between December 2021 and August 2024 were collected. The clinical manifestations, laboratory results, and molecular genetic characteristics were analyzed, ammonia levels and other laboratory results were evaluated pre-post GPB therapy by paired t-tests or Wilcoxon tests. Results:Among the 20 pediatric patients with UCD, there were 8 males and 12 females, and the onset age was 2.8 (1.4, 5.7) years. The ammonia levels were 174 (125, 342) μmol/L at first onset. The symptoms included vomiting in 6 cases, drowsiness in 5 cases, epilepsy in 5 cases, developmental delay in 5 cases, psychiatric and behavioral abnormalities in 3 cases, and lethargy in 1 case, and 18 cases exhibited abnormal liver function. Twenty cases included 6 UCD subtypes, with 11 cases being ornithine transcarbamylase deficiency. A total of 27 variants were identified, 11 (41%) of which were novel. The age of patients who began GPB therapy was 4.0 (1.5, 6.6) years. Ten cases stopped GPB after 4.2 (3.4, 5.3) months, with 4 patients undergoing liver transplantation and 6 discontinuing for financial reasons. The remaining ten patients continued GPB therapy for 11.6 (8.6, 14.0) months. The duration of GPB treatment was 6.0 (4.2, 12.3) months, at the final visit, the levels of ammonia, platelets and aspartate aminotransferase were lower compared to those of pre-treatment (all P<0.05). The serum albumin level was higher than that of pre-treatment ( P=0.016). Two patients suffered only one episode of acute hyperammonaemia, with ammonia levels of 232 and 141 μmol/L, respectively. Nine cases experienced adverse effects potentially related to GPB, decreased appetite in 6 cases, vomiting in 3 cases, abnormal skin oil odor in 2 cases, somnolence, fatigue and diarrhea each in 1 case, with symptoms improved within 6 (3, 10) days. Conclusions:UCD primarily manifests with neurological and gastrointestinal symptoms, and early diagnosis of UCD could be achieved through the analysis of ammonia. GPB may effectively reduce ammonia levels in UCD pediatric patients, with favorable safety and tolerability.
2.Preliminary study of carotid artery elasticity and intima thickness changes as assessed by ultrahigh-frequency ultrasound in combination with RF-data based quantitative analysis on vessel stiffness and the influencing factors in patients with ankylosing spondylitis
Chengkai WU ; Jianjun YUAN ; Tianshu CHU ; Xijun ZHANG ; Fan GAO ; Ming WU ; Yanyan GUO ; Haohui ZHU
Chinese Journal of Ultrasonography 2025;34(3):194-202
Objective:To evaluate the changes of carotid intima thickness(CIT)and stiffness parameters in ankylosing spondylitis(AS)patients by 24 MHz ultra-high frequency ultrasound probe and RF-data based quantitative analysis on vessel stiffness(R-VQS),and to explore its influencing factors.Methods:Sixty patients with AS who underwent consultation at Henan Provincial People's Hospital from November 2023 to May 2024 were prospectively collected as AS group,54 healthy volunteers matched for sex,age,and body mass index were collected as control group. CIT were measured using a 24 MHz ultra-high frequency ultrasound probe,hardness coefficien(HC)and pulse wave velocity(PWV)were measured by R-VQS,the differences of the parameters between the two groups were compared. The changes of carotid artery elasticity and intima thickness and their influencing factors in patients with AS were explored by univariate and multivariate linear regression analysis.Results:①C-reactiveprotein,erythrocyte sedimentation rate(ESR),neutrophil count and neutrophil to lymphocyte ratio(NLR)were elevated in the AS group compared with the control group,and the differences were statistically significant(all P < 0.05). ②CIT,carotid intima-media thickness(CIMT),HC,PWV and epicardial adipose tissue thickness(EATT)were all increased in the AS group compared with the control group,and the differences were statistically significant(all P < 0.05). ③Multifactorial linear regression analysis showed that EATT,ESR were the main influencing factors of HC,PWV and CIT( β=2.192,0.031;1.792,0.002;0.097,0.001;all P<0.05). Conclusions:Carotid CIT,CIMT,HC,PWV and EATT are all higher in the AS patients,and ESR and EATT are the main influencing factors of HC,PWV,and CIT,suggesting that more attention should be paid to the detection of ESR and EATT in patients with AS when interventions are performed clinically.
3.Clinical characteristics and efficacy of glyceryl phenylbutyrate treatment in 20 pediatric patients with urea cycle disorder
Wenjuan QIU ; Chengkai SUN ; Yuan XIAO ; Xiaoyan HUANG ; Cui SONG ; Jin WU ; Haiyan WEI ; Liwen WU ; Dan YU ; Hongwei DU ; Chen LIU ; Xuefan GU
Chinese Journal of Pediatrics 2025;63(9):1005-1010
Objective:To investigate the clinical characteristics of urea cycle disorder (UCD), the efficacy and safety of glyceryl phenylbutyrate (GPB) therapy in pediatric patients with UCD.Methods:This study was a retrospective, single-arm, multicenter clinical study. The clinical data of 20 pediatric patients with UCD who received GPB treatment at 9 hospitals nationwide between December 2021 and August 2024 were collected. The clinical manifestations, laboratory results, and molecular genetic characteristics were analyzed, ammonia levels and other laboratory results were evaluated pre-post GPB therapy by paired t-tests or Wilcoxon tests. Results:Among the 20 pediatric patients with UCD, there were 8 males and 12 females, and the onset age was 2.8 (1.4, 5.7) years. The ammonia levels were 174 (125, 342) μmol/L at first onset. The symptoms included vomiting in 6 cases, drowsiness in 5 cases, epilepsy in 5 cases, developmental delay in 5 cases, psychiatric and behavioral abnormalities in 3 cases, and lethargy in 1 case, and 18 cases exhibited abnormal liver function. Twenty cases included 6 UCD subtypes, with 11 cases being ornithine transcarbamylase deficiency. A total of 27 variants were identified, 11 (41%) of which were novel. The age of patients who began GPB therapy was 4.0 (1.5, 6.6) years. Ten cases stopped GPB after 4.2 (3.4, 5.3) months, with 4 patients undergoing liver transplantation and 6 discontinuing for financial reasons. The remaining ten patients continued GPB therapy for 11.6 (8.6, 14.0) months. The duration of GPB treatment was 6.0 (4.2, 12.3) months, at the final visit, the levels of ammonia, platelets and aspartate aminotransferase were lower compared to those of pre-treatment (all P<0.05). The serum albumin level was higher than that of pre-treatment ( P=0.016). Two patients suffered only one episode of acute hyperammonaemia, with ammonia levels of 232 and 141 μmol/L, respectively. Nine cases experienced adverse effects potentially related to GPB, decreased appetite in 6 cases, vomiting in 3 cases, abnormal skin oil odor in 2 cases, somnolence, fatigue and diarrhea each in 1 case, with symptoms improved within 6 (3, 10) days. Conclusions:UCD primarily manifests with neurological and gastrointestinal symptoms, and early diagnosis of UCD could be achieved through the analysis of ammonia. GPB may effectively reduce ammonia levels in UCD pediatric patients, with favorable safety and tolerability.
4.Preliminary study of carotid artery elasticity and intima thickness changes as assessed by ultrahigh-frequency ultrasound in combination with RF-data based quantitative analysis on vessel stiffness and the influencing factors in patients with ankylosing spondylitis
Chengkai WU ; Jianjun YUAN ; Tianshu CHU ; Xijun ZHANG ; Fan GAO ; Ming WU ; Yanyan GUO ; Haohui ZHU
Chinese Journal of Ultrasonography 2025;34(3):194-202
Objective:To evaluate the changes of carotid intima thickness(CIT)and stiffness parameters in ankylosing spondylitis(AS)patients by 24 MHz ultra-high frequency ultrasound probe and RF-data based quantitative analysis on vessel stiffness(R-VQS),and to explore its influencing factors.Methods:Sixty patients with AS who underwent consultation at Henan Provincial People's Hospital from November 2023 to May 2024 were prospectively collected as AS group,54 healthy volunteers matched for sex,age,and body mass index were collected as control group. CIT were measured using a 24 MHz ultra-high frequency ultrasound probe,hardness coefficien(HC)and pulse wave velocity(PWV)were measured by R-VQS,the differences of the parameters between the two groups were compared. The changes of carotid artery elasticity and intima thickness and their influencing factors in patients with AS were explored by univariate and multivariate linear regression analysis.Results:①C-reactiveprotein,erythrocyte sedimentation rate(ESR),neutrophil count and neutrophil to lymphocyte ratio(NLR)were elevated in the AS group compared with the control group,and the differences were statistically significant(all P < 0.05). ②CIT,carotid intima-media thickness(CIMT),HC,PWV and epicardial adipose tissue thickness(EATT)were all increased in the AS group compared with the control group,and the differences were statistically significant(all P < 0.05). ③Multifactorial linear regression analysis showed that EATT,ESR were the main influencing factors of HC,PWV and CIT( β=2.192,0.031;1.792,0.002;0.097,0.001;all P<0.05). Conclusions:Carotid CIT,CIMT,HC,PWV and EATT are all higher in the AS patients,and ESR and EATT are the main influencing factors of HC,PWV,and CIT,suggesting that more attention should be paid to the detection of ESR and EATT in patients with AS when interventions are performed clinically.
5.Predictive value of preoperative GLR levels for postoperative tumor recurrence in liver transplant recipients with liver cancer
Xiaoya WU ; Chengkai YANG ; Qiucheng CAI ; Jianyong LIU ; Lizhi LYU ; Yi JIANG
Chinese Journal of Hepatology 2024;32(7):657-664
Objective:To investigate the predictive value of preoperative γ-glutamyl transferase/lymphocyte count ratio (GLR) levels for postoperative tumor recurrence in liver transplant recipients with liver cancer.Methods:The clinical data of 158 recipients who were diagnosed with hepatocellular carcinoma (hereinafter referred to as liver cancer) and received liver transplantation at the No. 900 Hospital of the Joint Logistics Support Force of the Chinese People's Liberation Army from January 2008 to October 2022 were retrospectively analyzed. X-tile software, the Kaplan-Meier method, univariate and multivariate Cox regression, and other statistical methods were performed. The predictive value of preoperative GLR levels for postoperative tumor recurrence in liver transplant recipients with liver cancer and the risk factors for tumor recurrence in liver cancer patients post-liver transplantation were analyzed.Results:The X-tile software analysis confirmed that 96.8 was the optimal cutoff value for the preoperative GLR level to predict recurrence. The grouping threshold for survival analysis using the GLR cutoff value was 96.8. The tumor recurrence rates at 1, 3, and 5 years after surgery in the low-level GLR group (90 cases) and the high-level GLR group were 19.3% vs. 44.2%, 31.8% vs. 60.0%, and 34.1% vs. 62.9% (68 cases), respectively, and the differences were statistically significant between the two groups ( P<0.05). The Kaplan-Meier survival curve analysis results showed that the overall postoperative survival rate and recurrence-free survival rate were significantly lower in the high-level GLR group than the low-level GLR group ( P<0.05). The univariate Cox analysis result showed that there were statistically significant differences in preoperative aspartate aminotransferase, alpha fetoprotein, surgery time, maximum diameter of a solitary tumor, presence or absence of microvascular invasion, presence or absence of portal vein tumor thrombus, and preoperative GLR levels between the two groups ( P<0.05). Multivariate Cox analysis results showed that preoperative alpha-fetoprotein ≥400 ng/ml, GLR≥96.8, and the maximum diameter of a solitary tumor ≥5.0 cm were independent risk factors for postoperative tumor recurrence in liver transplant recipients with liver cancer ( P<0.05). Conclusion:GLR levels have a certain predictive value for postoperative tumor recurrence in liver transplant recipients with liver cancer. Furthermore, the postoperative tumor recurrence rate is relatively high when the preoperative GLR level in liver transplant recipients with liver cancer is ≥96.8.
6.Research progress in dual immune regulation of neutrophils in periodontal inflammation
Chengkai WANG ; Hui WANG ; Bingqing WU ; Yi LIU ; Zhenhua LUO
Chinese Journal of Stomatology 2024;59(7):721-725
Periodontitis is a chronic inflammatory condition of the periodontal tissues triggered by bacterial biofilm, leading to manifestations such as gingival bleeding, tooth mobility, and eventual exfoliation. Neutrophils exhibit a dual role throughout the course of periodontitis, both in defense against pathogens and in potentially detrimental effects on periodontal tissues. This article elucidates the intricate mechanisms underlying the dual functions of neutrophils in periodontitis, including respiratory burst, neutrophil extracellular traps (NETs) formation, degranulation, and phagocytosis. By providing a comprehensive understanding of neutrophils involvement in periodontitis, this study aims to empower clinicians with insights into the pathogenesis of periodontitis, thereby fostering novel strategies for its prevention and treatment.
7.Predictive values of ALBI and EZ-ALBI scores for early survival of recipients with liver failure after liver transplantation
Chengkai YANG ; Jiamian XU ; Huaxiang WANG ; Lizhi LYU ; Yi JIANG ; Aiping WU
Organ Transplantation 2022;13(5):611-
Objective To evaluate the predictive values of albumin-bilirubin (ALBI) and easy albumin-bilirubin (EZ-ALBI) scores for early survival (postoperative 3 months) of recipients with liver failure after liver transplantation. Methods Clinical data of 137 recipients diagnosed with liver failure and underwent liver transplantation were retrospectively analyzed. The optimal cut-off values of preoperative ALBI, EZ-ALBI and MELD scores to predict early survival of recipients with liver failure after liver transplantation were determined by the area under the receiver operating characteristic (ROC) curve. The risk factors of early death of recipients with liver failure after liver transplantation were identified by univariate and multivariate Cox regression analyses. The effects of different ALBI and EZ-ALBI levels upon early prognosis of recipients with liver failure after liver transplantation were analyzed. Results The optimal cut-off values of ALBI, EZ-ALBI and MELD scores were 0.21, -19.83 and 24.36, and the AUC was 0.706, 0.697 and 0.686, respectively. Univariate Cox regression analysis showed that preoperative alanine aminotransferase(ALT)≥50 U/L, aspartate aminotransferase(AST)≥60 U/L, ALBI score≥0.21 and EZ-ALBI score≥-19.83 were the risk factors for early postoperative death of recipients with liver failure after liver transplantation (all

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