bluuebunny/crossref_metadata_2025_split huggingface dataset:
MO1004IMBALANCE IN URINARY PROTEOGLYCANS AND THE INSULIN GROWTH FACTOR (IGF) AXIS IN CHILDREN WITH NEPHROLITHIASIS*
Authors: Kovacevic Larisa , Kovacevic Natalija , Lakshmanan Yegappan 
Published: May 2021

Abstract

Background and Aims
In vitro experiments have shown that exposure of renal epithelial cells to calcium oxalate crystals leads to synthesis of proteins involved in extracellular matrix (ECM) production. Cell proliferation and accumulation of ECM proteins have been reported in animal models and kidney biopsies from patients with stones. Based on the role of ECM in the Randall plaque formation, we aimed to identify and quantify ECM proteins in the urine of children with nephrolithiasis (RS) using a proteomic approach.

Method
Prospective, controlled, pilot study of pooled urine from RS (N=30, 24 females, mean age 12.95±4.03 years) versus age- and gender-matched healthy controls (HC), using liquid chromatography-mass spectrometry (LC-MS/MS). Patient inclusion criteria consisted of age 5-18 years, history of clinically (typical renal colic) and radiographically (ultrasound or CT) proven renal stone, at least two 24-hour satisfactory urine collections (urinary creatinine more than 15 mg/kg/day), absence of hematuria or pyuria, and normal renal function. Children who were not toilet trained, or those with bladder stones, nephrocalcinosis, neuropathic bladder, major congenital bladder abnormality, active urinary tract infection, presence of blood in urine, chronic kidney disease, previous major reconstructive bladder surgery requiring catheterization, and significant cardiac, pulmonary, gastro-intestinal, and neurological problems were excluded. Relative protein abundance was estimated using spectral counting. The criteria for protein selection were: 1) patient/control abundance ratios of &gt;5 or &lt;0.2 as a threshold to be well above observed technical variations in MS experiments; and 2) ≤0.05 p-value for the Fisher’s Exact Test. Results were confirmed by ELISA testing. Statistical analyses were conducted with IBM SPSS® version 20.

Results
We found 36 (15.7%) ECM proteins out of 229 proteins that met the above criteria. Significant differences between RS and HC were found among two proteoglycans and four insulin growth factor (IGF) proteins (Table). Significant increase in the urinary excretion of IGFBP4 in RS (37.00 ± 37.68 ng/mg creatinine ) versus HC (19.04 ± 20.32 ng/mg creatinine) was confirmed by ELISA (p=0.049). Statistically significant correlation was found between urinary IGFBP4 concentration and 24-hour urinary calcium excretion (p&lt;0.001).

Conclusion
Alteration in proteoglycans and the IGF axis appears to have a significant role in the mechanism of nephrolithiasis, likely by modulating ECM biosynthesis. Further understanding of their roles in nephrolithiasis may aid in generation of novel therapeutic approaches.
MO1013RETROSPECTIVE ANALYSIS OF THROMBOTIC COMPLICATIONS IN SHIGA-TOXIN ASSOCIATED HEMOLYTIC UREMIC SYNDROME IN CHILDREN
Authors: Emirova Khadizha , Tolstova Evgeniya , Orlova Olga , Muzurov Alexandr , Mstislavskaya Sofya , Pankratenko Tatiana , Abaseeva Tatiana , Generalova Galina 
Published: May 2021

Abstract

Background and Aims
Shiga-toxin associated hemolytic-uremic syndrome (STEC-HUS) is a thrombotic microangiopathy (TMA) followed by intestinal infection caused by shiga-toxin-producing E. coli. Endothelium damage of the microvasculature leads to the formation of platelet-fibrin thrombi and occlusion of small vessels. The aim of our study was a retrospective analysis of thrombotic complications in children with STEC-HUS.

Method
We retrospectively analyzed the case histories of patients in the dialysis department of St. Vladimir's Children's City Clinical Hospital from 2009 to 2019. The study included patients with STEC-HUS with the development of thrombotic complications.

Results
Among 410 patients with STEC-HUS, 16 patients with thrombotic complications were identified. Equally boys and girls, mean age 3.5 ± 2.4 g. Respiratory support was required in 6 cases. The duration of anuria was 9.9 +/- 6.2 days, in 2 cases only oliguria was noted. In all patients blood transfusion, fresh frozen plasma were needed, antibiotics were used. In all cases, continuous venovenous hemodiafiltration (CVVHDF) was performed with 2-way catheters, usually in the femoral vein. Thrombotic complications in all cases were associated with therapy. In 11 children, thrombosis was localized in the femoral, iliac veins and vena cava inferior. Clinical manifestations in the form of limb edema, cyanosis were noted in 4 patients, in other cases the diagnosis was made by ultrasound. 3 patients with thrombosis of the branches of the central retinal vein, vena cava superior, mesenteric thrombosis. 1 patient was diagnosed with dissecting femoral vein aneurysm. In 3 cases, CVVGDF had to be stopped due to catheter thrombosis. Before the current thrombosis, these children were not prescribed anicoagulants. Coagulation tests indices indicated hypercoagulability and were characterized by higher thrombin time, increased levels of D-dimer, and increased fibrinolysis time.

Conclusion
Large vessel thrombosis in children with STEC-HUS is a rare complication. The provoking factor for the implementation of thrombotic events in all cases was femoral vein catheterization. When managing patients with STEC-HUS monitoring of coagulation status is needed for the purpose of timely administration of anticoagulants.
MO1014PROSPECTIVE STUDY OF SAFETY AND EFFECTIVENESS OF OFATUMUMAB AS A TREATMENT IN REFRACTORY NEPHROTIC SYNDROME (CORTICODEPENDENT AND CORTICORRESISTANT)
Authors: Hernández Zúñiga Lina Catherine , Vinuesa Jaca Ana , Arango Sancho Pedro , Calzada Baños Yolanda , Codina Sampera Elena , López Baez Víctor , Madrid Aris Álvaro 
Published: May 2021

Abstract

Background and Aims
Ofatumumab (OFA) is an anti-CD-20 monoclonal antibody useful in nephrotic syndrome refractory to conventional treatments and rituximab (RTX). Our objective is to evaluate the response and safety in patients with nephrotic syndrome (NS) treated with Ofatumumab.

Method
A prospective descriptive study of 2 years duration (2017-2019) in children with NS refractory to first-line therapies who received treatment with anti-CD20 monoclonal antibodies. To do this, we divided the cohort into 3 groups: Patients with corticodependent NS (CDNS) without response or with adverse effects associated with first-line treatment that preclude its use (Group 1); Corticosteroid-resistant SN (CRNS) (Group 2) and SN with post-transplant recurrence (Group 3). In them, the results of safety and remission rate were evaluated.

Results
Thirty-three patients (21 with SNCD, 11 with SNCR, and 1 with SN recurrence in transplantation) were included and administered anti-CD20. The male / female ratio was 2: 1 and the mean age at diagnosis was 5.2 years. 100% of the children (33) received RTX and 18.2% (6) OFA. The RTX achieved complete remission in 87.9% (29) and 48.3% of these did not present new relapses after 70 months of follow-up. 100% of Group 1 presented complete remission after RTX, although 52.4% (11) presented at least 1 relapse after 22.9 months (mean 2.5 relapses). In group 2, 72.72% (8) complete remission and 27.2% (3) partial, with persistent proteinuria. 36.4% (4) presented relapse after 17 months of treatment (mean 1 relapse). Of the 6 who received OFA, 83.3% presented complete remission (1 SNCR and 4 SNCD) and 1 patient (SNCD) presented relapse at 24 months (mean follow-up 1 year). The other case, a 13-year-old girl with recurrence of focal segmental glomerulosclerosis (FSGS) in kidney transplantation, presented partial remission after one year of treatment in association with immunoadsorption sessions. Regarding safety, adverse reactions occurred in 6% (2): allergic reaction with 2nd dose of RTX and cytokine release syndrome with 1st dose of OFA.

Conclusion
Ofatumumab in our series has proven to be an effective and safe drug in difficult-to-manage NS, achieving complete remission in 5 patients who had not previously responded to Rituximab
MO1015EARLY URINARY SYSTEM COMPLICATIONS IN CHILDREN WITH HEMATOPOETIC STEM CELL TRANSPLANTATION
Authors: Gurbanov Anar , Gülhan Bora , Kuşkonmaz Barış , Okur Fatma Visal , Uçkan Çetinkaya Duygu , Ozdemir Gulsah , Taş Nesrin , Çelegen Kübra , Ozaltin Fatih , Duzova Ali , Topaloglu Rezan 
Published: May 2021

Abstract

Background and Aims
Urinary system complications after hematopoietic stem cell transplantation (HSCT) cause severe morbidity and mortality. The aim of the study is to investigate the incidence and risk factors of urinary system complications in patients who had HSCT during their childhood.

Method
Patients who had HSCT between January 2010-2019 with a minimum follow-up period of 6 months were included in the study. Data regarding urinary system complications were collected from the medical records of the patients. pRIFLE and KDIGO classification systems were used for the definition of acute kidney injury (AKI).

Results
167 patients (108 males and 59 females) with allogeneic (n=165) and autologous (n=2) HSCT were investigated for renal complications. In cohort, HSCT was performed in 41 patients (%24.6) secondary to malignant diseases and in 126 patients secondary to non-malignant diseases. Hemorrhagic cystitis (HC) developed in 28 patients (16.8%) after HSCT. The mean age of the patients with and without HC was 14.6±5.6 years and 10.5±6 years, respectively (p=0.044). Among patients with HC, 17 had concomitant viral infection. Presence of viral infection, gender, disease group, history conditioning regime, total body irradiation, acute graft-versus-host disease and veno-occlusive disease (VOD) did not have any effect on the development of HC in logistic regression analysis. In cohort, 126 patients (75.4%) developed AKI according to KDIGO classification (stage 1; 55 patients, stage 2; 36 patients, stage 3; 35 patients). The mean period of development of AKI after HSCT was 34±22 days. The risk of AKI (according to KDIGO) was higher in patients who had HSCT secondary to malignant diseases and/or who developed viral infections after HSCT (p=0.034 and p=0.013, respectively). Among patients, 71 patients (%42.5) developed AKI according to pRIFLE classification. The risk of AKI (according to pRIFLE) was higher in patients who had HSCT secondary to malignant diseases, who developed viral infections and/or who developed VOD during follow-up (p=0.043, p=0.001 and p=0.006, respectively). One patient developed thrombotic microangiopathy after HSCT. None of the patients had nephrotic syndrome during follow-up period.

Conclusion
Patients who had HSCT secondary to malignant disease, viral infections and/or VOD had higher risk of AKI and should be closely monitored.
MO1017PSYCHOSOCIAL REPERCUSSIONS AND QUALITY OF LIFE OF CHILDREN UNDERGOING HEMODIALYSIS IN A DEVELOPING COUNTRY
Authors: Naouaoui Salma , Ahtitich Kamal , Chettati Meriem , Fadili Wafaa , Laouad Inass 
Published: May 2021

Abstract

Background and Aims
Despite major advances in the provision of renal replacement therapy, children and adolescents on dialysis frequently have compromised daily life activities and a worse quality of life compared with healthy peers. While these aspects have been studied extensively in affluent countries, data from developing regions are scant. The aim of our study was assessing the psychosocial repercussions and the quality of life (QoL) of children with end stage renal disease (ESRD) undergoing hemodialysis in a Moroccan tertiary care hospital and comparing them with healthy controls.

Methods
We performed a cross-sectional investigation, at Mohammed VI university hospital of Marrakesh, using parent/child reports of generic module for QoL assessment: PedsQLTM Inventory version 4 for both cases and controls. Disease-specific module: PedsQLTM ESRD version 3 was used for ESRD cases. Twenty-two children on maintenance hemodialysis and 50 controls were enrolled in our study.

Results
The mean age of the ESRD cases was 11.6 ± 4.7 years (range 4– 18 years) with female predominance (58%). Controls were age- and sex-matched. A statistically significant difference was observed between ESRD cases and controls regarding all aspects of QoL. The total QOL mean score was 51.2 ± 13.6 and 82.8 ± 11.2 among cases and controls, respectively. All individual QoL domains were significantly worse in ESRD cases.

Conclusion
Our findings showed that the quality of life of children undergoing hemodialysis may be dramatically affected especially in limited-resources countries. And this raises the need to introduce quality of life assessment as a part of pediatric ESRD management.
MO1018IS LOW NORMAL BLOOD PRESSURE LEVEL IN PATIENTS WITH X-LINKED ALPORT SYNDROME OUR GOAL?
Authors: Aksenova Marina , Tutelman Konstantin , Konkova Natalia 
Published: May 2021

Abstract

Background and Aims
The mean blood pressure &lt;50th percentile for age, gender and height is recommended in children with chronic kidney disease, especially in case of proteinuria. The aim was to define the influence of low normal mean blood pressure level on the progression of renal disease in children with X-linked Alport syndrome (XLAS).

Method
mean day blood pressure normalized for sex, age, height (MBP), urine protein (mg/m2/day) and eGFR were assessed in retrospective single center study.

Results
The children with XLAS (n=69, 47M, age 9.49±4.18 years, eGFR=109±17.36 ml/min/1,73m2) were divided into 3 groups according the MBP level during follow up (4[3;5] years): (1) MBP&lt;50‰ (n=27, 13М), (2) MBP 50-90‰ (n=33, 25M), (3) MBP≥90‰ (9М). The decline of eGFR (ml/min/1,73m2/year) was higher in male (-3[-0.8;-4.7] vs 0.2[-0.4;2]; χ2= 21.15587, p = 0.0067), in children with MBP ≥90‰ (0.65[-2.65;2] vs -2.3[-6;2.3] vs -3[-7.5;-1.8] in 1st, 2nd and 3 groups, respectively; р1,3=0.0091) and in patients with proteinuria (-2.5[-7;0.5] vs 0.2[-4;3]; p=0.0018). The risk of achieving eGFR&lt;60 ml/min/1,73m2 was higher in pts of 3 group (OR=5.33, 95% CI 1.75;16.22) especially in the presence of proteinuria (OR=7.08, 95% CI 2.46;20.39). The multivariate analysis of predictive factors for eGFR reduction showed that the proteinuria (χ2=12.554, p=0.0019), the male gender (χ2=7.107, p=0.0077) and not MBP (χ2=5.4, p=0.07) were independent risk factors for eGFR decline.

Conclusion
The male gender and proteinuria are independent risk factors for renal disease progression in children with XLSA. We assumed that the goal of treatment, at least in XLAS’ patients with normal blood pressure, is to decrease proteinuria.
MO1019ASSOCIATION OF VEGF ANF TGF1 GENES POLYMORPHISMS WITH SERUM CONCENTRATION OF GENE PRODUCTS IN CHILDREN WITH LUPUS NEPHRITIS
Authors: Kazyra Ina , Sukalo Alexandr , Bialkevich Hanna , Krylova-Alefirenko Anna , Nikitchenko Natallia , Goncharova Roza 
Published: May 2021

Abstract

Background and Aims
Previously it was shown that significantly elevated serum concentration of vascular-endothelial growth factor (VEGF) and transforming growth factor β (TGF-β1) is observed in Lupus Nephritis (LN) patients, especially in those with disease exacerbation. In this study we investigated the association of selected single nucleotide polymorphisms (SNP) in TGF-β1 and VEGF genes with serum concentration of the gene product in children with LN.

Method
We enrolled 22 children with LN (m:f 2/20) aged 10 to 17 years. Blood samples were taken twice in every child – in remission and during exacerbation of the disease. Serum concentration of VEGF and TGF-β1 was measured using R&D Systems Quantikine ELISA “Human TGF-β1” and “VEGF” kits. DNA was analyzed by polymerase chain reaction for identification of selected SNPs – VEGF rs699947 (A/C), rs2010963 (G/C), TGF-β1 rs1800469 (G/A).

Results
We didn’t find any association of serum concentration of TGF-β1 with rs1800469 polymorphism in individuals with LN in exacerbation and in remission either (p&gt;0,05).
Concentration of VEGF was associated with genotypes of VEGF gene. It was significantly higher in LN patients harboring AA genotype for rs699947 comparing with those with CC+CA group of genotypes (ie carrying at least one minor allele A). This association was observed in exacerbation period (p&lt;0,001), but not in remission (p&gt;0,05).
We compared major genotype GG and group of genotypes GC+CC (ie patients with at least one minor allele C) of VEGF gene (rs2010963). It was found that statistically significant elevation of gene product was found in individuals with group of genotypes GC+CC in exacerbation period (p=0,036). In remission concentration of the gene product didn't differ in patients with GG and GC+CC genotypes (p=0,97).
VEGF gene genotypes rs699947 and rs2010963 are in high linkage disequilibrium (LD). Having analyzed combined genotypes we found that association with the gene product was the same as in rs699947 alone analysis. This may suggest functional importance of this SNP. Rs2010963 more likely to show association with the gene product due to linkage with rs699947.

Conclusion
There is an association between VEGF gene genotypes (rs699947 and rs2010963) and gene product serum concentration in LN pediatric patients in exacerbation period. Among two investigated loci of VEGF gene rs699947 might be considered more relevant risk marker of VEGF disfunction. No statistically significant association was found in our study for TGF-β1 gene genotypes (rs1800469) and its' product serum concentration.
MO1022CENTRAL VENOUS CATHETERS FIRST - THE ACHILLES’ HEEL IN PEDIATRIC HEMODIALYSIS VASCULAR ACCESS
Authors: Domingos Ana , Teixeira Ana , Matos Paula , Rocha Liliana , Almeida Paulo , Costa Teresa , Faria Maria do Sameiro , Mota Maria Conceição 
Published: May 2021

Abstract

Background and Aims
Management of children with end-stage kidney disease (ESKD) requiring dialysis is always challenging and particular concerns exists regarding vascular access. International reports through the years are not satisfactory, with a high rate of central venous catheter (CVC), when “Fistula First” is the goal in Hemodialysis (HD). Suggested reasons for this phenomenon include, for example, age-related anatomical limitations, shorter awaiting times for or “scheduled” transplantation, pain and anxiety related to punctures or lack of pediatric/surgical expertise. Here we present the results of the last 13 years of experience in pediatric HD vascular access from a reference center in northern Portugal.

Method
A retrospective descriptive study of patients admitted to our pediatric HD Unit between January 2007 and December 2020. Clinical data was collected from medical records.

Results
40 patients were enrolled, mainly boys (n=22, 55%), mean age at admission 10.9±5.3 years (1-17 years), 63% weighing more than 30 kg (n =25) and 15% less than 15kg (n=6). More than half were incident patients starting on HD (n=22, 55%), 42.5% were transferred from peritoneal dialysis (PD) and one patient had a previous kidney transplant (KT). Regarding CKD etiology, 52.5% (n=21) were mainly due to congenital anomalies of the kidney and urinary tract; chronic glomerulonephritis was responsible for 20% of cases (n=8). Most patients initiated HD with a CVC (n=35, 87.5%), including two patients with an arteriovenous fistula (AVF) who required a temporary CVC until fistula maturation. At the end of follow-up, about 45% of the patients ended up with an AVF (comparing to 17.5% at the beginning) and, not unexpectedly, 88.9% (n=16) of them weighed more than 30kg and only one child less than 15 kg. The mean duration of dialysis was 1.2±1.8 years (1 month-8 years); 24 patients were submitted to KT (60%), 6 transferred to PD (15%) and only 4 remain on HD. Average waiting times for KT are quite longer in patients with AFV (1.7±2 years), in comparison to CVC (0.4±0.3 years).

Conclusion
CVC is by far the most used access in incident patients starting on HD. In our center it seems justified by the anatomical limitations of younger and smaller patients, as wells as expected shorter awaiting time for KT. Infectious and mechanical complications must be weighed when using CVC, mainly considering the probable need for different renal replacing techniques over the years. Although our data is quite similar to several international reports and other center experiences, we still aim for the best, with an expected improvement of these results within the coming years. For now, a notable increase in AVF placement at the end of the follow-up is already an encouraging fact.
MO1005ADPEDKD: A GLOBAL ONLINE PLATFORM TO EXPLORE THE CHILDHOOD PHENOTYPE OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE*
Authors: Dachy Angélique , De Rechter Stéphanie , Guay-Woodford Lisa , Mallett Andrew John , Harris Tess , Bockenhauer Detlef , Schaefer Franz , Liebau Max , Mekahli Djalila 
Published: May 2021

Abstract

Background and Aims
Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the 4th common cause of renal replacement therapy worldwide. As the disorder has been historically considered an adult-onset disease, there is a lack of longitudinal data from large pediatric cohorts. However, evidence is growing that first manifestations of ADPKD may be detected in childhood and children represent a specific target population for future treatment, allowing a better chance of preserving long term kidney function. To better define the pediatric spectrum of the disease, a global multicenter observational study on childhood-diagnosed ADPKD was launched in 2017.

Method
The ADPedKD registry is a worldwide web-based database, including both retrospective and prospective longitudinal data from young ADPKD patients (≤19 years). Australia, North-America and the United Kingdom joined the initiative with their source databases, namely the KidGen Collaborative (KidGen), NIH-funded Hepato-Renal Fibrocystic Disease (HRFD) and National Registry of Rare Kidney Diseases (RaDaR). Under informed consent, de-identified patient data, including genetics, radiological and laboratory findings, treatments and follow-up were enrolled in the database accessible via https://www.ADPedKd.org/.

Results
1019 ADPKD children (from 89 centers and 33 countries) are enrolled in the registry of which 167 patients from RaDaR, 17 from KidGen, 11 from HRFD and 824 from ADPedKD (401 male/ 423 female) with a mean (± SD) age at diagnosis of 6.3 ± 5.2 years. 81 children (9.8%) were diagnosed prenatally at a mean gestational age of 26.8 ± 7.8 weeks. Reasons for initial visit were: family screening in 325 (39.4%), postnatal incidental finding in 223 (27.0%), presenting features (such as hematuria, hypertension, urinary tract infections and flank or back pain) in 150 (18.2%) or unknown/not available in 126 (15.3%). Genetic testing was performed in 42.8% of the population, with the following results: PKD1 mutation (85.4%), PKD2 mutation (11.7%) and others (6.0%).

Conclusion
The ADPedKD registry is a unique source of clinical observational data that will provide deep phenotyping of children with ADPKD and will allow to define unified diagnostic, treatment and follow-up recommendations.