Pediatric Nephrology最新文献

{"title":"Comparison of GFR estimation equations using creatinine, cystatin C, and their combination in pediatric hematology-oncology: no single equation is superior across subgroups.","authors":"Katelyn J Phillips, Yilun Sun, Li Tang, Andrew Pappas, Shane J Cross, Jennifer L Pauley, John McCormick, Alejandro R Molinelli, John J Bissler, Anthony M Christensen, Clinton F Stewart","doi":"10.1007/s00467-025-06765-7","DOIUrl":"https://doi.org/10.1007/s00467-025-06765-7","url":null,"abstract":"<p><strong>Background: </strong>Accurate assessment of renal function is essential in treating pediatric patients dosed with nephrotoxic chemotherapy. The validity of the bedside Schwartz, 5-covariate St. Jude (5SJ), CKiD-CysC-U25, combined Cr-CysC-based CysPed, and the serum creatinine-BUN-cystatin C-based CKiD (CKiD Cr-CysC) equations were evaluated in pediatric hematology and oncology patients.</p><p><strong>Methods: </strong>A retrospective analysis was conducted comparing estimated glomerular filtration rate (eGFR) to measured GFR (mGFR) obtained from technetium- 99 m diethylenetriaminepentaacetic acid (^{99 m}Tc-DTPA) clearance between January 2016 and May 2022. The influence of corticosteroid use and inflammation in our patient population was evaluated for effect on serum cystatin C (CysC) concentrations and mGFR.</p><p><strong>Results: </strong>All equations agreed within 2 SD of the mean difference with mGFR, but the 5SJ equation had the smallest bias followed closely by the CysPed equation. Overall accuracy (P30) was assessed, and the 5SJ, CKiD Cr-CysC, CysPed, and CKiD-Cys-U25 exhibited comparable performance. In our patient population, we did not observe an effect of corticosteroids (cumulative dosage of > 0.5 mg/kg within the past 14 days) or the presence of inflammation (CRP > 1.2 mg/L) on cystatin C concentrations or mGFR.</p><p><strong>Conclusions: </strong>In our pediatric hematology and oncology patient population, no one estimating equation demonstrated superior accuracy and bias overall and in all subgroups. Neither corticosteroid use nor elevated CRP influenced serum CysC concentrations or eGFR.</p>","PeriodicalId":19735,"journal":{"name":"Pediatric Nephrology","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144036424","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The clinical characteristics of patients with congenital nephrotic syndrome secondary to NPHS1 mutation: Is nephrectomy still a therapeutic option for selected cases?","authors":"Yüksel Uğurlu, Bora Gülhan, İsmail Dursun, Hülya Nalçacıoğlu, Gülşah Kaya Aksoy, Nur Canpolat, Aysun Bayazıt, Zeynep Birsin Özçakar, Selcuk Yüksel, Gönül Parmaksız, Gülşah Özdemir, Eda Didem Kurt-Şükür, Ali Düzova, Mutlu Hayran, Fatih Ozaltin","doi":"10.1007/s00467-025-06774-6","DOIUrl":"https://doi.org/10.1007/s00467-025-06774-6","url":null,"abstract":"<p><strong>Background: </strong>Managing congenital nephrotic syndrome (CNS) remains a clinical challenge. While albumin infusions and nephrectomy have been long-standing treatments, a conservative approach is increasingly favored. This study aimed to compare clinical outcomes between nephrectomy (Nx) and non-Nx in patients with bi-allelic NPHS1 mutations.</p><p><strong>Methods: </strong>This retrospective cohort study included 29 pediatric CNS patients (15 female, 14 male) with confirmed NPHS1 mutations. Clinical parameters including albumin infusion requirements, infections, hospitalizations, growth, and survival rates were analyzed in the Nx and non-Nx groups.</p><p><strong>Results: </strong>The median age at the time CNS was diagnosed was 29 days (IQR: 11-62 days). In all, 24 patients (82.8%) had homozygous NPHS1 mutations and 5 (17.2%) had compound heterozygous NPHS1 mutations. None of the patients had Fin-major mutation (i.e., p. Leu41 Aspfs*50). Unilateral/bilateral nephrectomy was performed in 16 patients. At 12 months post-nephrectomy the number of albumin infusions required, infections, and hospitalizations decreased significantly in the Nx group, as compared to the pre-nephrectomy period (p = 0.001, p = 0.027, and p = 0.004, respectively). Among the 13 (44.8%) patients in the non-Nx group, at 12 months after CNS was diagnosed the number of serum albumin infusions required significantly decreased (p = 0.007); however, the number of infections and hospitalization did not differ significantly (p = 0.589 and p = 0.5, respectively). Receiver operating characteristic (ROC) analysis showed that requiring albumin infusions ≥ 14 days/month predicted the decision to perform nephrectomy with 68% accuracy (73% sensitivity and 62% specificity).</p><p><strong>Conclusions: </strong>Nephrectomy reduces albumin infusions, infections, and hospitalizations, suggesting it may be a beneficial treatment for selected CNS patients with NPHS1 mutations.</p>","PeriodicalId":19735,"journal":{"name":"Pediatric Nephrology","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144037106","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Association of COVID-19 vaccination with relapsed nephrotic syndrome and new onset nephrotic syndrome in children.","authors":"Dayna Mazza, Elizabeth Ward, Spandana Makeneni, Jarcy Zee, Benjamin Laskin, Michelle Denburg","doi":"10.1007/s00467-025-06778-2","DOIUrl":"https://doi.org/10.1007/s00467-025-06778-2","url":null,"abstract":"<p><strong>Background: </strong>Several case reports describe new onset or relapsed nephrotic syndrome (NS) after COVID-19 mRNA vaccination. However, there have been no systematic studies in children.</p><p><strong>Methods: </strong>In this single-center, retrospective cohort study, we used our electronic health record registry to identify patients with NS who received ≥ 1 dose of COVID-19 vaccine from 12/2020 to 12/2022. For each patient, we determined number of relapses in the 180 days pre- and 60 days post-vaccination. Conditional logistic regression was used to assess risk of relapse after vaccination. Linear regression was used to estimate the mean difference between individual-level post- and pre-vaccine relapse rates.</p><p><strong>Results: </strong>Ninety-five patients with relapsing NS were included (median age 12 years, 43% female). Their clinical phenotype was as follows: 33% infrequent relapsing, 52% frequently relapsing (FR)/steroid-dependent (SD)/secondarily steroid responsive (SSR), and 16% steroid-resistant. Twenty-five patients (26%) relapsed in the pre-vaccine period, 17 (18%) had ≥ 1 relapse post-vaccination, and 78 (82%) had no relapse documented after COVID-19 vaccination. There was no significant difference in the risk of relapse after versus before vaccination (odds ratio 0.43, p = 0.08), and no significant difference in relapse rates after versus before vaccination (mean difference 0.08 per 100 patient-days, p = 0.39), overall or by phenotype. Of post-vaccine relapses, 94% occurred among the FR/SD/SSR group. Five patients met criteria for new onset NS presenting ≤ 60 days after receipt of the COVID-19 vaccine.</p><p><strong>Conclusions: </strong>In a systematic pre/post comparison of individual-level relapse frequency, we found no significant difference in risk or rates of relapse after COVID-19 vaccination in children with NS.</p>","PeriodicalId":19735,"journal":{"name":"Pediatric Nephrology","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144030751","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"IPNA webinars: an effective teaching platform advancing patient care in pediatric nephrology worldwide.","authors":"Eugene Yu-Hin Chan, Sharon Teo, Michelle López, Abdullahi Mudi, Pankaj Hari, Olivia Boyer, John D Mahan, Dieter Haffner, Elena Levtchenko","doi":"10.1007/s00467-025-06775-5","DOIUrl":"https://doi.org/10.1007/s00467-025-06775-5","url":null,"abstract":"","PeriodicalId":19735,"journal":{"name":"Pediatric Nephrology","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144047432","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Remembering Professor Bahia H. Moustafa (1948-2024).","authors":"Neveen A Soliman, Ihab Sakr Shaheen","doi":"10.1007/s00467-025-06773-7","DOIUrl":"https://doi.org/10.1007/s00467-025-06773-7","url":null,"abstract":"","PeriodicalId":19735,"journal":{"name":"Pediatric Nephrology","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2025-04-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144030752","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Are we ACE-ing the early treatment of Alport syndrome?","authors":"Samantha J Williamson, Max Liebau, R Rachel Lennon","doi":"10.1007/s00467-025-06722-4","DOIUrl":"https://doi.org/10.1007/s00467-025-06722-4","url":null,"abstract":"","PeriodicalId":19735,"journal":{"name":"Pediatric Nephrology","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2025-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143973056","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Risks and benefits of ChatGPT in informing patients and families with rare kidney diseases: an explorative assessment by the European Rare Kidney Disease Reference Network (ERKNet).","authors":"Albertien M van Eerde, Ana Teixeira, Flavia Galletti, Michal Maternik, Valentina Capone, Rik Westland, Jaap Mulder, Jan Halbritter, Thomas Osterholt, Valentina Neukel, Lutz T Weber, Max C Liebau, Franz Schaefer, Stefan Kohl","doi":"10.1007/s00467-025-06746-w","DOIUrl":"https://doi.org/10.1007/s00467-025-06746-w","url":null,"abstract":"<p><strong>Background: </strong>Rare diseases affect fewer than 1 in 2000 individuals, but approximately 150 rare kidney diseases account for about 10% of the chronic kidney disease (CKD) population, impacting millions across Europe and globally. The scarcity of medical experts for these conditions results in an unmet need for accurate and helpful patient information. Large language models like ChatGPT may offer a technological solution to assist medical professionals in educating patients and improving doctor-patient communication. We hypothesized that ChatGPT could provide accurate responses to frequently asked basic questions from patients with rare kidney diseases.</p><p><strong>Methods: </strong>Medical professionals and members of European Patient Advocacy Groups (ePAGs) affiliated with the European Rare Kidney Disease Reference Network (ERKNet) simulated patient-ChatGPT interactions using a Microsoft forms questionnaire and ChatGPT 3.5 and 4.0. Participants selected any rare kidney disease for a structured conversation with ChatGPT 3.5 or 4.0. Responses were evaluated for accuracy and helpfulness.</p><p><strong>Results: </strong>Forty-six ERKNet experts and 12 ePAGs from 13 European countries participated in this study. ChatGPT provided scientifically accurate and helpful information on 28 randomly selected rare kidney diseases, including prognostic information and genetic testing guidance. Participants expressed neutral positions regarding ChatGPT's recommendations on alternative treatments, second opinions, and other information sources. While ChatGPT generally was perceived as helpful and empathetic, concerns about patient safety persisted.</p><p><strong>Conclusions: </strong>ChatGPT exhibited substantial potential in addressing patient inquiries regarding rare kidney diseases in a real-world context. While it demonstrated resilience against misinformation in this application, careful human oversight remains essential and indispensable.</p>","PeriodicalId":19735,"journal":{"name":"Pediatric Nephrology","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2025-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143986040","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Expanded discussion of kidney health monitoring for critically ill term and late preterm infants after acute kidney injury: a report from the Neonatal Kidney Health Consensus Workshop.","authors":"Kim T Vuong, Brianna M Liberio, Samantha R Schwartz, Shina Menon, Tahagod H Mohamed, Danielle E Soranno, Kara Short Johnson, Jennifer G Jetton, Kyle A Merrill, Mina Hanna, Michelle C Starr, David T Selewski, Heidi J Steflik","doi":"10.1007/s00467-025-06757-7","DOIUrl":"https://doi.org/10.1007/s00467-025-06757-7","url":null,"abstract":"<p><strong>Background: </strong>Acute kidney injury (AKI) is common in the neonatal intensive care unit (NICU) and is associated with increased morbidity and mortality. Mounting evidence suggests infants with AKI in the NICU have higher risks of long-term kidney dysfunction, such as chronic kidney disease. However, guidelines for outpatient kidney-focused follow-up practices are lacking.</p><p><strong>Methods: </strong>As part of the National Institutes of Health-sponsored Consensus Workshop to Address Kidney Health in Neonatal Intensive Care Unit Graduates, a multidisciplinary workgroup within the US performed an in-depth review of the medical literature on term and late preterm (i.e. ≥ 34 weeks gestation) neonates admitted to the NICU with AKI to inform consensus recommendations for outpatient kidney health monitoring for high-risk and at-risk infants.</p><p><strong>Results: </strong>In this modified Delphi consensus statement, the workgroup developed three consensus recommendations and identified priority research gaps and opportunities for future study. Specific recommendations include completing a NICU discharge kidney health evaluation followed by a comprehensive kidney health assessment six months after discharge for high-risk infants and at two years of age for high-risk and at-risk infants.</p><p><strong>Conclusions: </strong>Critically ill term and late preterm infants with AKI have an increased risk of long-term kidney dysfunction and merit evaluation at NICU discharge with subsequent comprehensive kidney health assessments based on risk factors. Current research gaps and opportunities for improved care include identifying optimal pre-discharge planning approaches, examining the impacts of different etiologies and severity of AKI on long-term kidney and overall health, exploring modification to current AKI diagnosis standards, and development of high-yield educational tools for families and providers.</p>","PeriodicalId":19735,"journal":{"name":"Pediatric Nephrology","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144008616","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Etiology and outcomes of primary renal tubular acidosis.","authors":"Sukanya Priyadarshini, Aditi Sinha, Manisha Jana, Radhika Tandon, Kapil Sikka, Vijay Prakash Mathur, Girish Chandra Bhatt, Menka Yadav, Jitendra Kumar Meena, Priyanka Khandelwal, Pankaj Hari, Arvind Bagga","doi":"10.1007/s00467-025-06747-9","DOIUrl":"https://doi.org/10.1007/s00467-025-06747-9","url":null,"abstract":"<p><strong>Background: </strong>This study investigates the etiology, outcomes, and genotype-phenotype correlations in patients with renal tubular acidosis (RTA) at a tertiary care center in New Delhi.</p><p><strong>Methods: </strong>This cross-sectional study included children and young adults with RTA who underwent clinical, biochemical, radiological and/or genetic evaluations between July 2020 and December 2024. We report clinical phenotype, anthropometry, metabolic control and progression to chronic kidney disease (CKD) in relation to genotype of distal RTA.</p><p><strong>Results: </strong>Of 135 patients enrolled, 69 had distal RTA. The yield of genetic testing was 72% in distal RTA and 88.7% in Fanconi syndrome. Variants in SLC4A1 (42.4%) and ATP6V1B1 (28.8%) were the most common etiologies of distal RTA. Compared to other etiologies, patients with SLC4A1 variants were older at symptom onset (P = 0.008). Hematological abnormalities were more frequent in patients with biallelic compared to heterozygous SLC4A1 variants (50% vs. 12.5%; P = 0.18). Nephrocalcinosis and metabolic control were similarly prevalent across genetic categories of distal RTA. Sensorineural hearing loss was more common with ATP6V1B1 than with ATP6V0A4 variants (61.5% vs. 22.2%, P = 0.099) and did not vary by metabolic control. At median follow-up of 5-years, 74.1% of patients with distal RTA had short stature, 74.6% had poor metabolic control and 2.9% had progressed to CKD G3-5.</p><p><strong>Conclusions: </strong>This study outlines the genetic etiology and phenotype of distal RTA in south Asia. Over short-term follow-up, poor metabolic control and severe stunting were common, while CKD was uncommon.</p>","PeriodicalId":19735,"journal":{"name":"Pediatric Nephrology","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2025-04-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144019450","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Donor-derived cell-free DNA testing in pediatric kidney transplant recipients: indications and clinical utility.","authors":"Jayanthi Chandar, Vaka Sigurjonsdottir, Marissa Defreitas, Tara Gavcovich, Mingming Zhou, Renata Glehn-Ponsirenas, George Burke","doi":"10.1007/s00467-025-06770-w","DOIUrl":"https://doi.org/10.1007/s00467-025-06770-w","url":null,"abstract":"<p><strong>Background: </strong>We describe our single-center experience in performing donor-derived cell-free DNA (dd-cfDNA) testing for a clinical indication in pediatric kidney transplant recipients.</p><p><strong>Methods: </strong>Dd-cfDNA was done for increase in creatinine, appearance of de novo anti-HLA antibodies (dnHLAab) and for a clinical indication. We compared clinical characteristics of patients with dd-cfDNA > 1 with those with dd-cfDNA ≤ 1 and also compared dd-cfDNA in patients with no biopsy proven rejection (BPAR) or dnHLAab with those with BPAR, and those with dnHLAab and no BPAR.</p><p><strong>Results: </strong>Chart review was performed in 106 patients with a mean age of 11.0 ± 5.5 years. When compared with 62 patients with dd-cfDNA ≤ 1, 59.0% (26/44) of patients with dd-cfDNA > 1 had BPAR (OR 13.5: 95%CI 4.6,38; p < 0.0001), and 88.1% (37/44) had dnHLAab (OR 60.3 95%CI 17.2,192.2; p < 0.0001). Patients with DQ and DR dnHLAab (OR 115.2: 95%CI 24.8, 509.5; p < 0.0001) and those with donor-specific antibodies (DSAs) (OR 50.8: 95%CI 13.0, 168.7; p < 0.0001) were likely to have dd-cfDNA > 1. A repeated measures linear mixed effect model revealed a significant difference in dd-cfDNA between those with no antibodies or BPAR (p < 0.0001) and patients with BPAR and dnHLAab, with or without DSA. At the end of the follow-up period, eGFR was 72 mL/min/1.73 m² in those without BPAR or dnHLAab and was significantly different from those with BPAR (eGFR 51 mL/min/1.73 m² (p < 0.0001).</p><p><strong>Conclusions: </strong>Elevated dd-cfDNA is strongly associated with BPAR, class II dnHLAab and DSAs. Conversely, low values are observed in immunoquiescent states. Dd-cfDNA can be a useful tool for non-invasive clinical decision-making.</p>","PeriodicalId":19735,"journal":{"name":"Pediatric Nephrology","volume":" ","pages":""},"PeriodicalIF":2.6,"publicationDate":"2025-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144036451","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}