Objective To validate and evaluate the applicability and accuracy of the pooled cohort equation (PCE) model and the chronic kidney disease (CKD) add-on model in predicting the risk of atherosclerotic cardiovascular disease (ASCVD) in Chinese patients with CKD. Methods It was an external validation study of the predictive model based on the prospective multicenter cohort of the Chinese Cohort Study of Chronic Kidney Disease (C-STRIDE). This study included 3 039 CKD patients from the C-STRIDE of Peking University First Hospital. External validation was conducted for the PCE model and the CKD add-on model including renal disease indicators estimated glomerular filtration rate and urine albumin-to-creatinine ratio (ACR). The C-STRIDE cohort was a multi-center study involving 39 clinical centers across 28 cities in 22 provinces, with data collected from January 2012 to December 2017. Harrell's C-statistic was used to evaluate model discrimination, and calibration curves were used to assess the model calibration. Results This study included an external validation cohort of 3 039 CKD patients, with age of 50.0 (41.0, 61.0) years and 1 719 males (56.6%). The estimated glomerular filtration rate was 41.3 (27.1, 64.2) ml·min^-1·(1.73 m²)^-1. The ACR was 371.8 (81.7, 905.6) mg/g. Among the enrolled patients, 868 patients (28.6%) had a history of smoking, 1 607 patients (52.9%) were using antihypertensive drugs, and 620 patients (20.4%) had diabetes. The follow-up period was 4.7 (3.8, 5.0) years, and 135 patients (4.4%) experienced ASCVD events. The PCE model demonstrated good discrimination, with Harrell's C-statistics of 0.705 [95% confidence interval (CI) 0.645–0.761] in males and 0.738 (95% CI 0.673–0.798) in females. In contrast, the CKD add-on model showed weaker discrimination, with C-statistics of 0.557 (95% CI 0.504–0.621) in males and 0.551 (95% CI 0.492–0.630) in females. Compared with the PCE model, the C-statistic of the CKD add-on model were markedly lower by 0.148 (95% CI -0.224–-0.063) in males and 0.186 (95% CI -0.279–-0.084) in females. Calibration curves indicated that both models tended to overestimate 5-year ASCVD risk in Chinese CKD patients, particularly when the predicted risk probability exceeded 10%. Conclusions The PCE model exhibits good discriminatory performance in Chinese CKD patients and may have potential for clinical application. The CKD add-on model can not improve predictive accuracy and requires further validation before applied in Chinese clinical practice.

Objective To explore the correlation between micro-inflammatory status and urinary protein level as well as proteinuria remission in primary membranous nephropathy (PMN) patients with positive phospholipase A2 receptor (PLA2R) antibody, and evaluate its predictive value for prognosis in PMN patients. Methods This retrospective study included newly diagnosed PMN patients with positive PLA2R antibody and massive proteinuria at the Jiangsu Province Hospital from March 2020 to December 2023. Baseline demographic characteristics, laboratory parameters, and follow-up data were collected. Proteinuria remission was defined as complete remission or partial remission. Spearman correlation analysis was used to analyze the correlation between clinical parameters. Receiver-operating characteristic (ROC) curves were utilized to assess the predictive efficacy of micro-inflammatory status indicators systemic inflammatory response index (SIRI) and red blood cell distribution width-to-albumin ratio (RAR) for proteinuria remission. Cox regression analysis was performed to identify related factors of proteinuria remission in PMN patients, and Kaplan-Meier survival curves were used to compare proteinuria remission rates among PMN patients with different SIRI and RAR levels. Results A total of 109 PMN patients with massive proteinuria were enrolled in this study, with age of (52.46±13.47) years and 69 males (63.3%). Among the included patients, 74 patients (67.9%) achieved proteinuria remission, 20 patients (18.3%) had not achieved proteinuria remission, and 15 patients (13.8%) were lost to follow-up. Finally, 94 patients (86.2%) were included in the follow-up cohort. The non-proteinuria remission group exhibited significantly higher inflammatory markers, including SIRI (Z=-3.335, P=0.001), RAR (Z=-3.178, P=0.001), and C-reactive protein (Z=-3.239, P=0.001), than those in the proteinuria remission group. Correlation analysis revealed that SIRI was positively correlated with 24 h urinary protein quantity (r=0.305), urinary albumin-to-creatinine ratio (r=0.435), and chronic kidney disease stage (r=0.378), and negatively correlated with estimated glomerular filtration rate (r=-0.403) (all P<0.05). RAR was positively correlated with anti-PLA2R antibody titers (r=0.302) and urinary albumin-to-creatinine ratio (r=0.297), and negatively correlated with serum albumin (r=-0.954) (all P<0.05). ROC curve analysis demonstrated that SIRI [area under the curve (AUC)=0.744] and RAR (AUC=0.732) had good predictive ability for proteinuria remission, with the combined use improving the AUC to 0.751. The optimal cutoff values of SIRI and RAR for predicting remission were 0.953 (sensitivity 85.0%, specificity 55.4%) and 0.599 (sensitivity 85.0%, specificity 59.5%), respectively. Kaplan-Meier survival analysis showed high SIRI (>0.953, Log-rank test, χ2=4.267, P=0.039) and RAR (>0.599, Log-rank test, χ2=9.578, P=0.002) groups had lower proteinuria remission rates compared to the low SIRI (≤0.953) and RAR (≤0.599) groups. Multivariate Cox regression analysis identified high SIRI group (low SIRI group as a reference, HR=0.586, 95% CI 0.354–0.968) and high RAR group (low RAR group as a reference, HR=0.509, 95% CI 0.313–0.828) were independent factors correlated with proteinuria remission. Conclusions SIRI and RAR are independently associated with proteinuria remission in PLA2R antibody-positive PMN patients with massive proteinuria. SIRI and RAR may serve as potential tools for predicting proteinuria and remission status in PMN patients based on microinflammatory status.

Objective To investigate the association between platelet distribution width (PDW) and the prognosis of patients with non-dialysis chronic kidney disease (CKD). Methods It was a single center retrospective cohort study. The relevant information on CKD patients admitted to the Nephrology Department of Xuanwu Hospital, Capital Medical University from December 1, 2017 to December 1, 2020 were collected. The patients were divided into high PDW group and low PDW group by using the median PDW as the threshold, and followed up. The follow-up deadline was October 10, 2021, and the endpoint event was all-cause death or progression to end-stage renal disease. Kaplan-Meier survival analysis was used to plot survival curves, log-rank test was used to compare the differences in survival analysis results between groups, and Cox proportional hazards regression model was used to analyze the related factors of all-cause mortality in CKD patients. Results A total of 504 patients were ultimately included, with 253 patients in the high PDW group (PDW≥12.4%) and 251 patients in the low PDW group (PDW<12.4%). Among them, 69 patients (13.69%) died and 82 patients (16.27%) progressed to end-stage renal disease. Inter group comparisons revealed that serum creatinine [135.0 (82.5, 200.0) μmol/L vs. 181.0 (119.0, 249.0) μmol/L, Z=-4.865, P<0.001], blood urea nitrogen [9.23 (6.52, 12.17) mmol/L vs. 10.87 (7.57, 15.84) mmol/L, Z=-3.335, P=0.001], proportion of CKD 3 phase [50.6% (128/253) vs. 60.2% (151/251), χ2=4.666, P=0.031], fibrinogen [4.22 (3.49, 5.01) g/L vs. 4.56 (3.69, 5.62) g/L, Z=-2.969, P=0.003], C-reactive protein [3.90 (2.15, 8.60) mg/L vs. 6.90 (3.00, 11.10) mg/L, Z=-4.377, P<0.001], platelet count [(184.98±63.16) ×10? /L vs. (235.67±76.87) ×10? /L, t=-8.091, P<0.001], serum phosphorus [1.23 (1.05, 1.51) mmol/L vs. 1.36 (1.14, 1.69) mmol/L, Z=-3.742, P<0.001], and proportion of all-cause mortality [6.3% (16/253) vs. 21.1% (53/251), χ2=23.330, P<0.001] in the high PDW group were lower than those in the low PDW group, and the proportion of CKD 1 stage [18.2% (46/253) vs. 8.0% (20/251), χ2=11.550, P=0.001] and alanine aminotransferase [17.00 (12.00, 24.00) U/L vs. 13.00 (10.00, 21.00) U/L, Z=3.276, P=0.001] were higher than those in the low PDW group. Kaplan-Meier survival analysis showed that the all-cause mortality rate (Log-rank test, χ2=10.417, P=0.001), and the composite outcome survival rate (all-cause mortality and progression to end-stage renal disease) in the low PDW group were higher than those in the high PDW group (Log-rank test, χ2=7.027, P=0.008). Multivariate Cox regression analysis showed that age [hazard ratio (HR)=1.064, 95% confidence interval (CI) 1.038-1.090, P<0.001], combined diabetes (HR=2.221, 95% CI 1.314-3.756, P=0.003), serum albumin (HR=0.949, 95% CI 0.910-0.990, P=0.015), PDW<12.4% (taking PDW≥12.4% as a reference, HR=1.846, 95% CI 1.016-3.354, P=0.044), and blood phosphorus (HR=2.458, 95% CI 1.381-4.374, P=0.002) were independent correlated factors of all cause death in non-dialysis CKD patients. Conclusion PDW<12.4% is an independent factor correlated with all-cause mortality in non-dialysis CKD patients.

Objective To investigate the clinical characteristics and prognosis of pediatric patients with transplantation-associated thrombotic microangiopathy (TA?TMA) following allogeneic hematopoietic stem cell transplantation (alloHSCT), and to analyze related factors for the occurrence and poor prognosis of TA?TMA. Methods This was a single-center retrospective cohort study. Relevant data were collected from children who underwent alloHSCT at the Department of Hematology, Beijing Children's Hospital, Capital Medical University, from January 2015 to December 2023. Patients were divided into TA?TMA group and non-TA?TMA group based on the occurrence of TA?TMA. Patients were classified into high-risk TA?TMA group and non-high-risk TA?TMA group according to whether they met criteria for high-risk TA?TMA within 100 days after transplantation. High-risk TA?TMA was defined as fulfillment of the diagnostic criteria for TA?TMA in addition to at least two of the following three conditions: random urine protein-to- creatinine ratio ≥2 mg/mg; plasma soluble complement membrane attack complex level exceeding the upper limit of the normal reference range; and the presence of multiple organ dysfunction syndrome (MODS). The follow-up endpoint was December 31, 2023, or patient death. Cox regression models were used to analyze factors associated with the occurrence of TA?TMA and mortality in children after alloHSCT. The Kaplan-Meier method was used to calculate cumulative survival rates, and the log-rank test was used to compare differences of cumulative survival rates between groups. Results A total of 456 children were enrolled, including 37 patients (8.1%) in the TA?TMA group. The follow-up time was 12.0 (6.0, 24.0) months. Among children with TA-TMA, acute kidney injury occurred in 23 patients (62.2%), hypertension in 22 patients (59.5%), proteinuria in 21 patients (56.8%), and progression to chronic kidney disease in 1 patient (2.7%). Multivariate Cox regression analysis showed that among the complications occurring within 100 days after alloHSCT, hypertension [hazard ratio (HR)=11.081, 95% confidence interval (CI) 5.122-23.973, P=0.001], veno-occlusive disease/sinusoidal obstruction syndrome (HR=12.518, 95% CI 4.594-34.111, P=0.001) were independent factors correlated with the occurrence of TA?TMA after alloHSCT. MODS (HR=0.333, 95% CI 0.113-0.984, P=0.047) was an independent factor correlated with mortality in patients with TA?TMA. Survival analysis revealed that the 1-year cumulative survival rate for all patients was 92.1% (95% CI 89.4%-94.8%). The cumulative survival rate in the non-TA?TMA group was significantly higher than those in the TA?TMA group (Log-rank test, χ2=50.768, P<0.001). The cumulative survival rate in the non-high-risk TA?TMA group was higher than that in the high-risk TA?TMA group (Log-rank test, χ2=5.404, P=0.020). Among TA?TMA patients, massive proteinuria group had lower cumulative survival rate than non- massive proteinuria group (Log-rank test, χ2=7.571, P=0.006). MODS group had lower cumulative survival rate than non-MODS group (Log-rank test, χ2=13.613, P=0.001). Conclusions Renal impairment is common in pediatric patients with TA?TMA after alloHSCT, but the rate of progression to chronic kidney disease is relatively low. Among the complications occurring within 100 days after alloHSCT, hypertension and hepatic veno-occlusive disease/sinusoidal obstruction syndrome are independent factors associated with the occurrence of TA?TMA. The presence of MODS is an independent factor associated with mortality in patients with TA?TMA.

Objective To explore the efficacy, safety and related factors of the first single-dose rituximab (RTX) in treating children with steroid-dependent minimal-change nephrotic syndrome (SD?MCNS). Methods It was a retrospective cohort study. Clinical data of SD?MCNS children receiving a single-dose RTX for the first time from October 2011 to December 2022 at the Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine were collected and the efficacy and safety of the single-dose treatment were analyzed. Logistic regression analysis and Cox regression analysis were used to analyze the correlated factors of efficacy, and receiver- operating characteristic (ROC) curve was plotted. Kaplan-Meier method was applied for the survival analysis of disease-free status. Results A total of 116 SD?MCNS children received a single-dose RTX for the first time, with 82 males (70.7%). The age was 10.1 (7.3, 13.1) years old, and the disease duration since diagnosed as primary nephrotic syndrome to firstly using RTX was 60 (35, 88) months. The follow-up time was 48 (26, 72) months. The efficacy rate of the first single dose RTX in treating SD?MCNS was 94.0% (109/116), and 92 patients (79.3%) achieved complete remission and were able to discontinue steroids therapy. For children who did not discontinue steroids after RTX, the steroid-dependent dosage was significantly reduced compared to that before RTX [0.120 (0.002, 0.311) mg/kg vs. 0.092 (0.044, 0.213) mg/kg, t=2.366, P=0.020]. Children who achieved complete remission after RTX experienced the first relapse after 8.5 (5.0, 15.3) months. The relapse rate was 67.0% (77/115) at 1 year, 81.7% (94/115) at 2 years, 87.8% (101/115) at 3 years, and 90.4% (104/115) at 5 years. The correlated factors of the efficacy were 24-hour protein quantity before RTX with cut-off value of 42.24 mg/kg (Z=3.00, P=0.003), CD20/CD45 before RTX with cut-off value of 10.45% (Z=-2.84, P=0.004), and a history of cyclosporine A (CsA) use (Z=2.01, P=0.046). The correlated factors of relapse were female gender (Z=-2.86, P=0.004), age at RTX infusion (Z=-2.74, P=0.006), discontinuation of steroids after RTX (Z=9.66, P<0.001), and a history of tacrolimus (FK506) (Z=2.76, P=0.006). Adverse reactions occurred in 12 patients (10.3%) after the first single-dose RTX. Among them, 7 patients (6.0%) had acute infusion reactions, and 5 patients (4.3%) had long-term hypoglobulinemia. No severe adverse reactions occurred. Conclusions The first single-dose RTX in treating children with SD?MCNS is effective and safe, but the relapse rate is high. 24-hour protein quantity <42.24 mg/kg, CD20/CD45>10.45% before RTX, and a history of CsA use are the related factors of effectiveness of RTX treatment in SD?MCNS. Females, age <11.1 years, a history of FK506 use, and discontinuation of steroids after medication are associated with a higher relapse rate in SD?MCNS children treated with RTX.

This article reports the clinical features and management of a case of multi-system involvement caused by a pathogenic variant in the single?strand binding protein 1 (SSBP1) gene. The patient was a 13-year-old girl with onset in early childhood. Her clinical manifestations included progressive optic atrophy, growth retardation, tubulointerstitial nephritis (progressing to chronic kidney disease stage 5), high-frequency sensorineural hearing loss, skeletal lesion, and renal anemia. Genetic testing identified a heterozygous SSBP1 gene variant, c.320G>A (p.Arg107Gln), classified as pathogenic. Following conventional symptomatic treatment (including antihypertensive therapy, erythropoietin supplementation, and anti-infections), renal function continued to deteriorate rapidly. Heterozygous variants in the SSBP1 gene can lead to a multi-system disorder characterized by optic atrophy, progressive renal failure, sensorineural hearing loss, and skeletal abnormalities. The disease phenotype is diverse and insidious, making early diagnosis challenging. For patients presenting with similar multi-system manifestations, SSBP1 genetic testing should be considered to facilitate early definitive diagnosis and intervention.

Composite heterozygous mutations in the CEP290 gene typically result in syndromic disorders such as Meckel-Gruber syndrome, Joubert syndrome, or Senior-Loken syndrome. The paper reports an unusual case of a syndrome characterized by biallelic CEP290 variations, presenting with adolescent blindness and slowly progressive renal and hepatic failure. The whole exome sequencing and Sanger sequencing identified two CEP290 variants. One wasc.1593C>A (p.Tyr531*), a pathogenic variation, and the other was c.43C>T (p.Pro15Ser), a unexplained variation. CEP290 mutations can cause multi-system ciliopathies, and molecular genetic diagnosis of such cases can lead to precise diagnosis, screening of high-risk relatives, and appropriate management.