1.Efficacy and Safety of Acupuncture Combined with Levodopa in the Treatment of Parkinson's Disease: A Randomized Controlled Trial
Jingyun ZHU ; Xiyan GAO ; Linlin WANG ; Zhixin REN ; Guiling WANG ; Jing GUO ; Yanrong WU
Journal of Traditional Chinese Medicine 2025;66(14):1456-1462
ObjectiveTo evaluate the clinical efficacy and safety of acupuncture combined with levodopa in the treatment of Parkinson's disease(PD). MethodsA total of 60 patients with PD were enrolled and randomly assigned to test group or control group, with 30 patients in each group. The control group received levodopa only, starting at 100 mg per dose, three times daily, with gradual increases not exceeding a maximum daily dose of 800 mg. The test group received acupuncture three times per week in addition to levodopa. Both groups were treated for 12 weeks. Assessments were conducted before treatment, after 6 and 12 weeks treatment, using the Unified Parkinson's Disease Rating Scale(UPDRS), Wearing-Off Questionnaire-9(WOQ-9), Montreal Cognitive Assessment(MoCA), Mini-Mental State Examination(MMSE), Depression Rating Scale(DRS), Hamilton Depression Scale(HAMD), Hamilton Anxiety Scale(HAMA), PD Questionnaire-39(PDQ-39), and Pittsburgh Sleep Quality Index(PSQI). Repeated measures ANOVA was utilized to evaluate the effects of time, group, and their interaction on each index. Spearman correlation analysis was conducted to examine the relationships between combined treatment and outcome scores. Adverse events in both groups were recorded throughout the study. ResultsBoth groups showed significant improvements after 6 and 12 weeks treatment, with decreases in UPDRS total score, WOQ-9 total score, DRS score, HAMD score, HAMA score, PDQ-39 score, and PSQI score, and increases in MoCA and MMSE scores(P<0.05). Compared with the control group, the test group demonstrated significantly greater improvements in all the above indicators after 6 and 12 weeks (P<0.05). Repeated measures ANOVA showed significant time main effects, group main effects, and their interaction across all outcome measures(P<0.01). Spearman correlation analysis revealed that combined therapy was significantly negatively correlated with UPDRS, WOQ-9, DRS, HAMD, HAMA, PDQ-39, and PSQI scores, while positively correlated with MoCA and MMSE scores after 12 weeks of treatment(P<0.05). Both groups did not experience any serious adverse events and did not affect treatment. ConclusionAcupuncture combined with levodopa is more effective than levodopa alone in improving motor function, non-motor symptoms, cognitive function, depression and anxiety, quality of life, and sleep quality in patients with PD, with good safety.
2.Recent advance in clinical characteristics and gene therapy of spinal muscular atrophy
Han ZHANG ; Zhixin WU ; Xueping ZHU
Chinese Journal of Neuromedicine 2025;24(3):293-298
Spinal muscular atrophy (SMA), a hereditary neuromuscular disease, may lead to progressive muscle weakness and atrophy in the proximal limbs resulting from decreased survival motor neuron (SMN) protein expression caused by SMN1 gene mutation. Currently, drugs clinically used for SMA gene therapy include nusinersen, sofosbuvir, and risdiplam, whic have limited scopes of application. Some drugs need to be used for a long time, and none of them can achieve a complete cure. Intrathecal injection of supplemented SMN1 gene has entered clinical trials, showing good curative effect. At the same time, basic researches indicate that CRISPR/Cas9 gene editing technology shows good prospect in correcting the mutant genes in SMA. This article reviews the clinical characteristics of SMA and research progress of gene therapy, aiming to provide a new perspective for diagnosis and treatment of SMA.
3.Risk factors for complications in neonates with early-onset group B Streptococcus sepsis
Qiuping SHEN ; Haifeng GENG ; Wenqiang SUN ; Zhixin WU ; Xueping ZHU
Chinese Journal of Perinatal Medicine 2025;28(5):381-388
Objective:To identify the risk factors and their predictive value for complications in neonates with early-onset group B streptococcus (GBS) sepsis. Methods:This case-control study retrospectively analyzed 96 neonates with early-onset GBS sepsis (age of onset<7 days) admitted to Children's Hospital of Soochow University between January 1, 2007, and December 31, 2022. Patients were categorized into complication ( n=36) and non-complication ( n=60) groups. Receiver operating characteristic (ROC) curves determined optimal cutoff values of Pediatric Sequential Organ Failure Assessment (pSOFA) and Pediatric Logistic Organ Dysfunction Score 2 (PELOD-2) for predicting complications in the neonates with early-onset GBS sepsis. Independent t-tests, Mann-Whitney U tests, Chi-square tests and Fishe exact tests were used for group comparison of general information, clinical manifestations, auxiliary examinations, and treatment during hospitalization. Multivariate logistic regression identified independent risk factors, and ROC curves evaluated their predictive performance for complications in the neonates with early-onset GBS sepsis. Results:ROC analysis identified pSOFA>4.5 scores and PELOD-2>5.5 scores as optimal thresholds for complication prediction in neonates with early-onset GBS sepsis. (1) The complication group exhibited higher rates of preterm birth [30.6% (11/36) vs. 5.0% (3/60), χ2=11.80], maternal clinical chorioamnionitis [25.0% (9/36) vs. 5.0% (3/60), χ2=6.50], prolonged rupture of membranes≥18 h [22.2% (8/36) vs. 5.0% (3/60), χ2=4.99], invasive mechanical ventilation [36.1% (13/36) vs. 13.3% (8/60), χ2=6.83], fever [22.2% (8/36) vs. 3.3% (2/60), χ2=6.70], lethargy [77.8% (28/36) vs. 51.7% (31/60), χ2=6.48], mottled skin as the initial clinical manifestation [38.9% (14/36) vs. 20.0% (12/60), χ2=4.07], leukopenia [44.4% (16/36) vs. 18.3% (11/60), χ2=7.59], hypoalbuminemia [27.8% (10/36) vs. 3.3% (2/60), χ2=10.16], pSOFA>4.5 [83.3% (30/36) vs. 35.0% (21/60), χ2=21.11], PELOD-2>5.5 [50.0% (18/36) vs. 5.0% (3/60), χ2=26.66], and dual-positive blood and cerebrospinal fluid cultures [25.0% (9/36) vs. 0.0% (0/60), Fisher exact test] compared to the non-complication group (all P<0.05). Serum creatinine [(88.4±17.7) vs. (61.9±17.7) μmol/L, t=-6.02], urea nitrogen [(3.7±0.4) vs. (3.4±0.6) mmol/L, t=-3.18], and lactate [(7.5±3.4) vs. (5.8±2.2) mmol/L, t=-2.80] were elevated, while fibrinogen [(2.2±1.1) vs. (2.7±1.0) g/L, t=2.03], pH (7.3±0.2 vs. 7.4±0.1, t=2.04), and albumin [(28.2±3.9) vs. (31.9±4.2) g/L, t=4.32] were reduced in the complication group (all P<0.05). (2) Multivariate analysis identified preterm birth ( OR=6.642, 95% CI: 1.210-36.473), along with hypoalbuminemia ( OR=8.202, 95% CI: 1.184-56.811), pSOFA>4.5 scores ( OR=5.284, 95% CI: 1.573-17.749), and PELOD-2>5.5 scores ( OR=8.464, 95% CI: 1.922-37.279) assessed on admission day 1 as independent risk factors (all P<0.05). The area under the curve for predicting complications in early-onset GBS sepsis neonates was 0.628 (95% CI: 0.523-0.724) for preterm birth, and 0.622 (95% CI: 0.517-0.719), 0.742 (95% CI: 0.642-0.826), and 0.725 (95% CI: 0.624-0.811) for hypoalbuminemia, pSOFA>4.5 scores, and PELOD-2>5.5 scores assessed on admission day 1, respectively. The combined predictive model integrating all four risk factors achieved the highest area under the curve of 0.868 (95% CI: 0.784-0.929). Conclusion:Preterm birth as well as hypoalbuminemia, pSOFA>4.5 scores, and PELOD-2>5.5 scores at admission are critical risk factors for complications in early-onset GBS sepsis, warranting heightened clinical vigilance.
4.Impact of postoperative complications on adverse outcomes following curative-intent resection for gallbladder cancer: a national multicenter real-world study
Zhipeng LIU ; Cheng CHEN ; Jie BAI ; Yan JIANG ; Dong ZHANG ; Wei GUO ; Zhixin WANG ; Xiang LAN ; Yufu YE ; Zhaoping WU ; Jinxue ZHOU ; Shuo JIN ; Yi ZHU ; Wei CHEN ; Dalong YIN ; Yao CHENG ; Haisu DAI ; Lei ZHANG ; Zhiyu CHEN
Chinese Journal of Digestive Surgery 2025;24(7):874-881
Objective:To investigate the impact of postoperative complications on adverse outcomes following curative-intent resection for gallbladder cancer (GBC).Methods:The multi-center real-world study was conducted. The clinicopathological data of 629 patients with GBC, who were admitted to 14 medical centers including The First Affiliated Hospital of Army Medical University from the national multicenter database of Biliary Surgery Group of Elite Group of Chinese Journal of Digestive Surgery, from April 2020 to April 2024 were collected. There were 225 males and 404 females, aged (64±10)years. Patients underwent open curative-intent resection for GBC. Observation indicators: (1)surgery, postoperative complica-tions and adverse outcomes; (2) analysis of risk factors affecting postoperative adverse outcomes in patients and population attributable fraction (PAF). Missing data in predictor variables were addressed using multiple imputation with chained equations, while cases with missing outcome variables were addressed using the "multiple imputation then deletion (MID)" strategy. The severity of multicollinearity among independent variables was assessed using the variance inflation factor (VIF) test. Multivariable possion regression models with log link and robust error variance were construc-ted incorporating restricted cubic splines (3 knots) to address nonlinear relationships in continuous variables, calculating adjusted relative risk ( RR) with corresponding 95% confidence interval ( CI). Adjusted PAF was calculated for each imputed dataset using the AF package of R software, with subsequent pooling performed according to Rubin's rules. Results:(1) Surgery, postoperative complications and adverse outcomes. All 629 patients underwent curative-intent resection for GBC, of which 143 cases had postoperative complications, including 68 cases of intra-abdominal ascites, 39 cases of pulmonary infection, 21 cases of bile leakage, 12 cases of intra-abdominal hemorrhage, 11 cases of liver failure, 10 cases of pan-creatic fistula, 10 cases of wound infection, 10 cases of gastroparesis, 7 cases of cholangitis, 7 cases of sepsis. The same patient could have more than one kind of complication. Of 629 patients, there were 19 cases of postoperative 90-day death and 11 cases of missing data, 42 cases with post-operative 90-day reoperation and 7 cases with missing data, 44 cases with postoperative 90-day readmission and 3 cases with missing data, 155 cases with prolonged postoperative hospital stay and 3 cases with missing data. (2) Analysis of risk factors affecting the postoperative adverse outcomes in patients and PAF. Results of multivariate analysis showed that pulmonary infection and liver failure were independent risk factors for postoperative 90-day mortality ( RR=3.74, 12.15, 95% CI as 1.18-11.83, 1.98-74.48, P<0.05). Pulmonary infection demons-trated the highest PAF as 4.61% (95% CI as 3.94%-5.28%, P<0.05). Intra-abdominal ascites, pulmonary infection, bile leakage, and intra-abdominal hemorrhage were independent risk factors for post-operative 90-day reoperation ( RR=4.80, 3.62, 3.46, 4.99, 95% CI as 2.49-9.26, 1.42-9.21, 1.34-8.92, 1.55-16.06, P<0.05). Intra-abdominal ascites demonstrated the highest PAF as 8.65% (95% CI as 8.22%-9.08%, P<0.05). Intra-abdominal ascites, bile leakage, and liver failure were independent risk factors for postoperative 90-day readmission ( RR=6.20, 3.33, 14.33, 95% CI as 3.21-11.95, 1.33-8.35, 3.72-55.28, P<0.05). Intra-abdominal ascites demonstrated the highest PAF as 9.11% (95% CI as 8.85%-9.37%, P<0.05). Intra-abdominal ascites, pulmonary infection, bile leakage, liver failure, and wound infection were independent risk factors for prolonged postoperative hospital stay ( RR=2.29, 2.21, 2.26, 2.14, 3.35, 95% CI as 1.63-3.23, 1.41-3.46, 1.32-3.86, 1.11-4.13, 1.70-6.60, P<0.05). Intra-abdominal ascites demonstrated the highest PAF as 6.03% (95% CI as 5.71%-6.35%, P<0.05). Conclusion:Pulmonary infection is the most significant risk factor for postoperative 90-day mortality after curative-intent resection for GBC, while intra-abdominal ascites is the most significant risk factor for postoperative 90-day reoperation, postoperative 90-day readmission, and prolonged postoperative hospital stay.
5.Factors associated with QTc interval prolongation in long-term hospitalized patients with schizophrenia
Jian YANG ; Yi XU ; Zhixin ZHU
Chinese Journal of Primary Medicine and Pharmacy 2025;32(5):646-651
Objective:To investigate the factors associated with prolonged QTc interval in long-term hospitalized patients with schizophrenia, and to develop and validate a predictive model.Methods:A cross-sectional study was conducted involving 215 long-term hospitalized patients with schizophrenia at Xianyang Hospital, Yan'an University from January to December 2023. The incidence of prolonged QTc intervals among the patients was analyzed. Univariate and multivariate analyses of the factors associated with prolonged QTc intervals were performed. A mathematical model was developed to predict prolonged QTc intervals in long-term hospitalized patients with schizophrenia, and the predictive performance of the model was evaluated.Results:Among the 215 patients, 42 exhibited prolonged QTc intervals, with an incidence rate of 19.53%. Univariate analysis revealed statistically significant differences between the groups with and without prolonged QTc in terms of age, gender, body mass index, categories of antipsychotic medication used, fasting blood glucose, and potassium levels ( t = -5.66, χ2 = 29.03, t = -2.01, χ2 = 10.96, t = -5.78, t = 5.92, all P < 0.05). Multivariate logistic regression analysis indicated that age, gender, fasting blood glucose, and potassium levels were independent factors affecting QTc interval prolongation ( OR = 1.097, 10.221, 2.449, 0.014, all P < 0.05). The goodness-of-fit of the logistic regression model was validated to be satisfactory (Hosmer-Lemeshow χ2 = 14.56, P > 0.05). A risk nomogram model based on the variables from the multivariate analysis had a C-index of 0.713. Receiver operating characteristic curve analysis was performed using the independent influential factors and their P values from the logistic regression model to predict the probability of QTc interval prolongation. The area under the curve values were 0.762, 0.725, 0.730, 0.792, and 0.920, respectively. Conclusions:The incidence of prolonged QTc interval is relatively high in long-term hospitalized patients with schizophrenia. Female gender, older age, hyperglycemia, and hypokalemia are all associated with prolonged QTc intervals. The mathematical model developed based on these factors demonstrates good predictive performance for QTc interval prolongation.
6.IDH3A Inhibits Cardiomyocyte Hypertrophy via Elevating α-Ketoglutarate Level
Huayan WU ; Yihong WEN ; Hengli ZHAO ; Yuan GAO ; Chuanmeng ZHOU ; Ya WANG ; Jiening ZHU ; Zhixin SHAN
Journal of Sun Yat-sen University(Medical Sciences) 2025;46(2):275-283
[Objective]To investigate the regulatory effect and potential mechanisms of isocitrate dehydrogenase 3A(IDH3A)on cardiomyocyte hypertrophy.[Methods]The expression of IDH3A in the myocardium of healthy volunteers(n=10)and patients with heart failure(HF)(n=10),and in the myocardium of mice subjected to transverse aortic constriction(TAC)surgery and sham operation,as well as in phenylephrine(PE)-induced neonatal rat ventricular cardiomyocytes(NRVCs),was assessed by real-time quantitative polymerase chain reaction(RT-qPCR)and Western blot assay.The effect of adenovirus-mediated overexpression of IDH3A on the expression of hypertrophy-related genes in PE-induced NRVCs was also evaluated.The effect of IDH3A on NRVCs area was examined by phalloidin staining assay.A mutant of IDH3A with abolished enzymatic activity,IDH3A_D208A,was generated through site-directed mutagenesis.The impact of this IDH3A mutant on the hypertrophic phenotype,ATP and ROS levels in NRVCs was evaluated to investigate whether the regulatory role of IDH3A in cardiomyocyte hypertrophy was dependent on its enzymatic activity.The effect of exogenous α-ketoglutaric acid(AKG)on cardiomyocyte hypertrophy was also detected by Western blot and phalloidin staining assay,respectively.[Results]IDH3A was significantly decreased in the myocardium of HF patients,in the myocardium of TAC-operated mice,and in PE-induced NRVCs(P=0.005 2,P=0.026 6,P=0.041 3 and P=0.006 6,respectively).Overexpression of IDH3A markedly suppressed the expression of hypertrophy-related genes and the increase of cell size of PE-induced NRVCs(P<0.000 1,P=0.000 1 and P=0.000 2,respectively).The ATP and ROS analysis indicated that IDH3A inhibited the increases of ATP and ROS levels in PE-induced NRVCs(P=0.001 2 and P<0.000 1,respectively),whereas the enzymatically inactive IDH3A mutant lacked this effect.Exogenous AKG provision could,but overexpression of IDH3A mutant failed to suppress PE-induced NRVCs hypertrophy.[Conclusion]IDH3A inhibits cardiomyocyte hypertrophy via elevating AKG level,providing scientific evidence for study on IDH3A-based treatment of cardiac hypertrophy.
7.Impact of postoperative complications on adverse outcomes following curative-intent resection for gallbladder cancer: a national multicenter real-world study
Zhipeng LIU ; Cheng CHEN ; Jie BAI ; Yan JIANG ; Dong ZHANG ; Wei GUO ; Zhixin WANG ; Xiang LAN ; Yufu YE ; Zhaoping WU ; Jinxue ZHOU ; Shuo JIN ; Yi ZHU ; Wei CHEN ; Dalong YIN ; Yao CHENG ; Haisu DAI ; Lei ZHANG ; Zhiyu CHEN
Chinese Journal of Digestive Surgery 2025;24(7):874-881
Objective:To investigate the impact of postoperative complications on adverse outcomes following curative-intent resection for gallbladder cancer (GBC).Methods:The multi-center real-world study was conducted. The clinicopathological data of 629 patients with GBC, who were admitted to 14 medical centers including The First Affiliated Hospital of Army Medical University from the national multicenter database of Biliary Surgery Group of Elite Group of Chinese Journal of Digestive Surgery, from April 2020 to April 2024 were collected. There were 225 males and 404 females, aged (64±10)years. Patients underwent open curative-intent resection for GBC. Observation indicators: (1)surgery, postoperative complica-tions and adverse outcomes; (2) analysis of risk factors affecting postoperative adverse outcomes in patients and population attributable fraction (PAF). Missing data in predictor variables were addressed using multiple imputation with chained equations, while cases with missing outcome variables were addressed using the "multiple imputation then deletion (MID)" strategy. The severity of multicollinearity among independent variables was assessed using the variance inflation factor (VIF) test. Multivariable possion regression models with log link and robust error variance were construc-ted incorporating restricted cubic splines (3 knots) to address nonlinear relationships in continuous variables, calculating adjusted relative risk ( RR) with corresponding 95% confidence interval ( CI). Adjusted PAF was calculated for each imputed dataset using the AF package of R software, with subsequent pooling performed according to Rubin's rules. Results:(1) Surgery, postoperative complications and adverse outcomes. All 629 patients underwent curative-intent resection for GBC, of which 143 cases had postoperative complications, including 68 cases of intra-abdominal ascites, 39 cases of pulmonary infection, 21 cases of bile leakage, 12 cases of intra-abdominal hemorrhage, 11 cases of liver failure, 10 cases of pan-creatic fistula, 10 cases of wound infection, 10 cases of gastroparesis, 7 cases of cholangitis, 7 cases of sepsis. The same patient could have more than one kind of complication. Of 629 patients, there were 19 cases of postoperative 90-day death and 11 cases of missing data, 42 cases with post-operative 90-day reoperation and 7 cases with missing data, 44 cases with postoperative 90-day readmission and 3 cases with missing data, 155 cases with prolonged postoperative hospital stay and 3 cases with missing data. (2) Analysis of risk factors affecting the postoperative adverse outcomes in patients and PAF. Results of multivariate analysis showed that pulmonary infection and liver failure were independent risk factors for postoperative 90-day mortality ( RR=3.74, 12.15, 95% CI as 1.18-11.83, 1.98-74.48, P<0.05). Pulmonary infection demons-trated the highest PAF as 4.61% (95% CI as 3.94%-5.28%, P<0.05). Intra-abdominal ascites, pulmonary infection, bile leakage, and intra-abdominal hemorrhage were independent risk factors for post-operative 90-day reoperation ( RR=4.80, 3.62, 3.46, 4.99, 95% CI as 2.49-9.26, 1.42-9.21, 1.34-8.92, 1.55-16.06, P<0.05). Intra-abdominal ascites demonstrated the highest PAF as 8.65% (95% CI as 8.22%-9.08%, P<0.05). Intra-abdominal ascites, bile leakage, and liver failure were independent risk factors for postoperative 90-day readmission ( RR=6.20, 3.33, 14.33, 95% CI as 3.21-11.95, 1.33-8.35, 3.72-55.28, P<0.05). Intra-abdominal ascites demonstrated the highest PAF as 9.11% (95% CI as 8.85%-9.37%, P<0.05). Intra-abdominal ascites, pulmonary infection, bile leakage, liver failure, and wound infection were independent risk factors for prolonged postoperative hospital stay ( RR=2.29, 2.21, 2.26, 2.14, 3.35, 95% CI as 1.63-3.23, 1.41-3.46, 1.32-3.86, 1.11-4.13, 1.70-6.60, P<0.05). Intra-abdominal ascites demonstrated the highest PAF as 6.03% (95% CI as 5.71%-6.35%, P<0.05). Conclusion:Pulmonary infection is the most significant risk factor for postoperative 90-day mortality after curative-intent resection for GBC, while intra-abdominal ascites is the most significant risk factor for postoperative 90-day reoperation, postoperative 90-day readmission, and prolonged postoperative hospital stay.
8.Factors associated with QTc interval prolongation in long-term hospitalized patients with schizophrenia
Jian YANG ; Yi XU ; Zhixin ZHU
Chinese Journal of Primary Medicine and Pharmacy 2025;32(5):646-651
Objective:To investigate the factors associated with prolonged QTc interval in long-term hospitalized patients with schizophrenia, and to develop and validate a predictive model.Methods:A cross-sectional study was conducted involving 215 long-term hospitalized patients with schizophrenia at Xianyang Hospital, Yan'an University from January to December 2023. The incidence of prolonged QTc intervals among the patients was analyzed. Univariate and multivariate analyses of the factors associated with prolonged QTc intervals were performed. A mathematical model was developed to predict prolonged QTc intervals in long-term hospitalized patients with schizophrenia, and the predictive performance of the model was evaluated.Results:Among the 215 patients, 42 exhibited prolonged QTc intervals, with an incidence rate of 19.53%. Univariate analysis revealed statistically significant differences between the groups with and without prolonged QTc in terms of age, gender, body mass index, categories of antipsychotic medication used, fasting blood glucose, and potassium levels ( t = -5.66, χ2 = 29.03, t = -2.01, χ2 = 10.96, t = -5.78, t = 5.92, all P < 0.05). Multivariate logistic regression analysis indicated that age, gender, fasting blood glucose, and potassium levels were independent factors affecting QTc interval prolongation ( OR = 1.097, 10.221, 2.449, 0.014, all P < 0.05). The goodness-of-fit of the logistic regression model was validated to be satisfactory (Hosmer-Lemeshow χ2 = 14.56, P > 0.05). A risk nomogram model based on the variables from the multivariate analysis had a C-index of 0.713. Receiver operating characteristic curve analysis was performed using the independent influential factors and their P values from the logistic regression model to predict the probability of QTc interval prolongation. The area under the curve values were 0.762, 0.725, 0.730, 0.792, and 0.920, respectively. Conclusions:The incidence of prolonged QTc interval is relatively high in long-term hospitalized patients with schizophrenia. Female gender, older age, hyperglycemia, and hypokalemia are all associated with prolonged QTc intervals. The mathematical model developed based on these factors demonstrates good predictive performance for QTc interval prolongation.
9.Recent advance in clinical characteristics and gene therapy of spinal muscular atrophy
Han ZHANG ; Zhixin WU ; Xueping ZHU
Chinese Journal of Neuromedicine 2025;24(3):293-298
Spinal muscular atrophy (SMA), a hereditary neuromuscular disease, may lead to progressive muscle weakness and atrophy in the proximal limbs resulting from decreased survival motor neuron (SMN) protein expression caused by SMN1 gene mutation. Currently, drugs clinically used for SMA gene therapy include nusinersen, sofosbuvir, and risdiplam, whic have limited scopes of application. Some drugs need to be used for a long time, and none of them can achieve a complete cure. Intrathecal injection of supplemented SMN1 gene has entered clinical trials, showing good curative effect. At the same time, basic researches indicate that CRISPR/Cas9 gene editing technology shows good prospect in correcting the mutant genes in SMA. This article reviews the clinical characteristics of SMA and research progress of gene therapy, aiming to provide a new perspective for diagnosis and treatment of SMA.
10.Risk factors for complications in neonates with early-onset group B Streptococcus sepsis
Qiuping SHEN ; Haifeng GENG ; Wenqiang SUN ; Zhixin WU ; Xueping ZHU
Chinese Journal of Perinatal Medicine 2025;28(5):381-388
Objective:To identify the risk factors and their predictive value for complications in neonates with early-onset group B streptococcus (GBS) sepsis. Methods:This case-control study retrospectively analyzed 96 neonates with early-onset GBS sepsis (age of onset<7 days) admitted to Children's Hospital of Soochow University between January 1, 2007, and December 31, 2022. Patients were categorized into complication ( n=36) and non-complication ( n=60) groups. Receiver operating characteristic (ROC) curves determined optimal cutoff values of Pediatric Sequential Organ Failure Assessment (pSOFA) and Pediatric Logistic Organ Dysfunction Score 2 (PELOD-2) for predicting complications in the neonates with early-onset GBS sepsis. Independent t-tests, Mann-Whitney U tests, Chi-square tests and Fishe exact tests were used for group comparison of general information, clinical manifestations, auxiliary examinations, and treatment during hospitalization. Multivariate logistic regression identified independent risk factors, and ROC curves evaluated their predictive performance for complications in the neonates with early-onset GBS sepsis. Results:ROC analysis identified pSOFA>4.5 scores and PELOD-2>5.5 scores as optimal thresholds for complication prediction in neonates with early-onset GBS sepsis. (1) The complication group exhibited higher rates of preterm birth [30.6% (11/36) vs. 5.0% (3/60), χ2=11.80], maternal clinical chorioamnionitis [25.0% (9/36) vs. 5.0% (3/60), χ2=6.50], prolonged rupture of membranes≥18 h [22.2% (8/36) vs. 5.0% (3/60), χ2=4.99], invasive mechanical ventilation [36.1% (13/36) vs. 13.3% (8/60), χ2=6.83], fever [22.2% (8/36) vs. 3.3% (2/60), χ2=6.70], lethargy [77.8% (28/36) vs. 51.7% (31/60), χ2=6.48], mottled skin as the initial clinical manifestation [38.9% (14/36) vs. 20.0% (12/60), χ2=4.07], leukopenia [44.4% (16/36) vs. 18.3% (11/60), χ2=7.59], hypoalbuminemia [27.8% (10/36) vs. 3.3% (2/60), χ2=10.16], pSOFA>4.5 [83.3% (30/36) vs. 35.0% (21/60), χ2=21.11], PELOD-2>5.5 [50.0% (18/36) vs. 5.0% (3/60), χ2=26.66], and dual-positive blood and cerebrospinal fluid cultures [25.0% (9/36) vs. 0.0% (0/60), Fisher exact test] compared to the non-complication group (all P<0.05). Serum creatinine [(88.4±17.7) vs. (61.9±17.7) μmol/L, t=-6.02], urea nitrogen [(3.7±0.4) vs. (3.4±0.6) mmol/L, t=-3.18], and lactate [(7.5±3.4) vs. (5.8±2.2) mmol/L, t=-2.80] were elevated, while fibrinogen [(2.2±1.1) vs. (2.7±1.0) g/L, t=2.03], pH (7.3±0.2 vs. 7.4±0.1, t=2.04), and albumin [(28.2±3.9) vs. (31.9±4.2) g/L, t=4.32] were reduced in the complication group (all P<0.05). (2) Multivariate analysis identified preterm birth ( OR=6.642, 95% CI: 1.210-36.473), along with hypoalbuminemia ( OR=8.202, 95% CI: 1.184-56.811), pSOFA>4.5 scores ( OR=5.284, 95% CI: 1.573-17.749), and PELOD-2>5.5 scores ( OR=8.464, 95% CI: 1.922-37.279) assessed on admission day 1 as independent risk factors (all P<0.05). The area under the curve for predicting complications in early-onset GBS sepsis neonates was 0.628 (95% CI: 0.523-0.724) for preterm birth, and 0.622 (95% CI: 0.517-0.719), 0.742 (95% CI: 0.642-0.826), and 0.725 (95% CI: 0.624-0.811) for hypoalbuminemia, pSOFA>4.5 scores, and PELOD-2>5.5 scores assessed on admission day 1, respectively. The combined predictive model integrating all four risk factors achieved the highest area under the curve of 0.868 (95% CI: 0.784-0.929). Conclusion:Preterm birth as well as hypoalbuminemia, pSOFA>4.5 scores, and PELOD-2>5.5 scores at admission are critical risk factors for complications in early-onset GBS sepsis, warranting heightened clinical vigilance.

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