Medicine and Health Sciences Commons^™

Incidence Of Initial Renal Replacement Therapy Over The Course Of Kidney Disease In Children., Derek K. Ng, Matthew B. Matheson, Bradley A. Warady, Susan R. Mendley, Susan L. Furth, Alvaro Muñoz

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The Chronic Kidney Disease in Children Study, a prospective cohort study with data collected from 2003 to 2018, provided the first opportunity to characterize the incidence of renal replacement therapy (RRT) initiation over the life course of pediatric kidney diseases. In the current analysis, parametric generalized gamma models were fitted and extrapolated for RRT overall and by specific treatment modality (dialysis or preemptive kidney transplant). Children were stratified by type of diagnosis: nonglomerular (mostly congenital; n = 650), glomerular-hemolytic uremic syndrome (HUS; n = 49), or glomerular-non-HUS (heterogeneous childhood onset; n = 216). Estimated durations of time to RRT after …

The Pediatrician's Role In Eliminating Racial And Ethnic Disparities In Sleep-Related Infant Deaths., Jeffrey D. Colvin, Rachel Y. Moon

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No abstract provided.

Acetaminophen Protein Adducts In Hospitalized Children Receiving Multiple Doses Of Acetaminophen., Sibo Jiang, Valvanera Vozmediano, Susan M. Abdel-Rahman, Stephan Schmidt, Laura P. James

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Previous reports have questioned the safety of multiple doses of acetaminophen administered to ill children. Acetaminophen protein adducts (adducts) are a biomarker of acetaminophen-induced liver injury and reflect the oxidative metabolism of acetaminophen, a known mechanism in acetaminophen toxicity. In this prospective observational study, we analyzed adduct concentrations in 1034 blood samples obtained from 181 hospitalized children (1 to 18 years inclusive) who received 2 or more doses of acetaminophen. Linear regression analysis showed that serum adduct concentrations increased as a function of the cumulative acetaminophen dose, which could be attributed, in part, to a long half-life of adducts (2.17 …

Comparison Of Echocardiographic Measurements To Invasive Measurements Of Diastolic Function In Infants With Single Ventricle Physiology: A Report From The Pediatric Heart Network Infant Single Ventricle Trial., Suma P. Goudar, Victor Zak, Andrew M. Atz, Karen Altmann, Steven D. Colan, Christine B. Falkensammer, Mark K. Friedberg, Michele Frommelt, Kevin D. Hill, Daphne T. Hsu, Jami C. Levine, Renee Margossian, Christopher R. Mart, Joshua Sticka, Peter Shrader, Girish S. Shirali, Pediatric Heart Network Investigators

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BACKGROUND: While echocardiographic parameters are used to quantify ventricular function in infants with single ventricle physiology, there are few data comparing these to invasive measurements. This study correlates echocardiographic measures of diastolic function with ventricular end-diastolic pressure in infants with single ventricle physiology prior to superior cavopulmonary anastomosis.

METHODS: Data from 173 patients enrolled in the Pediatric Heart Network Infant Single Ventricle enalapril trial were analysed. Those with mixed ventricular types (n = 17) and one outlier (end-diastolic pressure = 32 mmHg) were excluded from the analysis, leaving a total sample size of 155 patients. Echocardiographic measurements were correlated to …

Burden Of Disease In Pediatric Patients With Hypophosphatasia: Results From The Hpp Impact Patient Survey And The Hpp Outcomes Study Telephone Interview., Eric T. Rush, Scott Moseley, Anna Petryk

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BACKGROUND: Hypophosphatasia (HPP) is a rare, inherited, metabolic bone disease caused by deficient tissue-non-specific isoenzyme of alkaline phosphatase activity that manifests as a broad range of signs/symptoms, including bone mineralization defects and systemic complications. The burden of disease is poorly characterized, particularly in children. This study aimed to characterize the patient-reported burden of disease among children with HPP using two survey instruments: the HPP Impact Patient Survey (HIPS) and the HPP Outcomes Study Telephone interview (HOST).

METHODS: Between September 2009 and June 2011, pediatric patients (aged younger than 18 years) with HPP were recruited to participate in the study via …

Text Messaging For Disease Monitoring In Childhood Nephrotic Syndrome., Chia-Shi Wang, Jonathan P. Troost, Larry A. Greenbaum, Tarak Srivastava, Kimberly Reidy, Keisha Gibson, Howard Trachtman, John D. Piette, Christine B. Sethna, Kevin Meyers, Katherine M. Dell, Cheryl L. Tran, Suzanne Vento, Krishna Kallem, Emily Herreshoff, Sangeeta Hingorani, Kevin Lemley, Gia Oh, Elizabeth Brown, Jen-Jar Lin, Frederick Kaskel, Debbie S. Gipson

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Introduction: There is limited information on effective disease monitoring for prompt interventions in childhood nephrotic syndrome. We examined the feasibility and effectiveness of a novel text messaging system (SMS) for disease monitoring in a multicenter, prospective study.

Methods: A total of 127 patientsresults, symptoms, and medication adherence were sent to a designated caregiver (n = 116) or adolescent patient (n = 3). Participants responded by texting. Feasibility of SMS was assessed by SMS adoption, retention, and engagement, and concordance between participant-reported results and laboratory/clinician assessments. The number of disease relapses and time-to-remission data captured by SMS were compared …

Abcb1 Snp Predicts Outcome In Patients With Acute Myeloid Leukemia Treated With Gemtuzumab Ozogamicin: A Report From Children's Oncology Group Aaml0531 Trial., Roya Rafiee, Lata Chauhan, Todd A. Alonzo, Yi-Cheng Wang, Ahlam Elmasry, Michael R. Loken, Jessica Pollard, Richard Aplenc, Susana Raimondi, Betsy A. Hirsch, Irwin D. Bernstein, A S. Gamis, Soheil Meshinchi, Jatinder K. Lamba

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Gemtuzumab-ozogamicin (GO), a humanized-anti-CD33 antibody linked with the toxin-calicheamicin-γ is a reemerging and promising drug for AML. Calicheamicin a key element of GO, induces DNA-damage and cell-death once the linked CD33-antibody facilitates its uptake. Calicheamicin efflux by the drug-transporter PgP-1 have been implicated in GO response thus in this study, we evaluated impact of ABCB1-SNPs on GO response. Genomic-DNA samples from 942 patients randomized to receive standard therapy with or without addition of GO (COG-AAML0531) were genotyped for ABCB1-SNPs. Our most interesting results show that for rs1045642, patients with minor-T-allele (CT/TT) had better outcome as compared to patients with CC …

How To Conduct Clinical Trials In Children: A Tutorial., Valentina Shakhnovich, Christoph P. Hornik, Gregory L. Kearns, Jaylene Weigel, Susan M. Abdel-Rahman

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Despite a growing interest in, and commitment to, implementing pediatric clinical trials, approximately one in every five trials in children fails because of inappropriate study design, suboptimal experiment planning, or inadequate participant enrollment. This tutorial, presented from the perspectives of seasoned pediatric investigators, an experienced research coordinator, and an established pediatric clinical trials network, is designed to provide practical guidance for successfully implementing pediatric clinical trials at an academic center or another comparable institution.

Global Variation Of Nutritional Status In Children Undergoing Chronic Peritoneal Dialysis: A Longitudinal Study Of The International Pediatric Peritoneal Dialysis Network., Franz Schaefer, Laura Benner, Dagmara Borzych-Dużałka, Joshua Zaritsky, Hong Xu, Lesley Rees, Zenaida L Antonio, Erkin Serdaroglu, Nakysa Hooman, Hiren Patel, Lale Sever, Karel Vondrak, Joseph Flynn, Anabella Rébori, William Wong, Tuula Hölttä, Zeynep Yuruk Yildirim, Bruno Ranchin, Ryszard Grenda, Sara Testa, Dorota Drożdz, Attila J. Szabo, Loai Eid, Biswanath Basu, Renata Vitkevic, Cynthia Wong, Stephen J. Pottoore, Dominik Müller, Ruhan Dusunsel, Claudia Gonzalez Celedon, Marc Fila, Lisa Sartz, Anja Sander, Bradley A. Warady, International Pediatric Peritoneal Dialysis Network (Ippn) Registry

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While children approaching end-stage kidney disease (ESKD) are considered at risk of uremic anorexia and underweight they are also exposed to the global obesity epidemic. We sought to investigate the variation of nutritional status in children undergoing chronic peritoneal dialysis (CPD) around the globe. The distribution and course of body mass index (BMI) standard deviation score over time was examined prospectively in 1001 children and adolescents from 35 countries starting CPD who were followed in the International Pediatric PD Network (IPPN) Registry. The overall prevalence of underweight, and overweight/obesity at start of CPD was 8.9% and 19.7%, respectively. Underweight was …

Prevalence Of Bacteremia And Bacterial Meningitis In Febrile Neonates And Infants In The Second Month Of Life: A Systematic Review And Meta-Analysis., Eric A. Biondi, Brian R. Lee, Shawn L. Ralston, Jared M. Winikor, Justin F. Lynn, Angela Dixon, Russell Mcculloh

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Importance: Febrile neonates (persons in the first month of life) are believed to be at higher risk for bacteremia or bacterial meningitis than infants in their second month of life. However, the true prevalence is unclear.

Objective: To determine modern rates of bacteremia and bacterial meningitis in febrile neonates and infants in the second month of life presenting to an ambulatory setting.

Data Sources: A comprehensive, no-limit search was conducted in PubMed using previously published search terms in February 2015 and repeated in September 2016.

Study Selection: Abstracts and full texts were reviewed independently by several investigators. Studies were included …

Considerations For Implementing Precision Therapeutics For Children., Matthew J. Mclaughlin, Jonathan B. Wagner, Valentina Shakhnovich, Bruce Carleton, J Steven Leeder

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Improving the utilization of pharmacologic agents in the pediatric population yields significant, perhaps life-long, benefits. Genetic factors related to the disposition of a medication or an alteration at the target receptor site contributes to the observed variability of exposure and response between individuals. An additional source of this variability specific to the pediatric population is ontogeny, where age-specific changes during development may require dose adjustments to obtain the same levels of drug exposure and response. With significant improvements in characterizing both the ontogeny and genetic contributions of drug metabolizing enzymes, the time is right to begin placing more emphasis on …

Csf Inflammatory Markers Differ In Gram-Positive Versus Gram-Negative Shunt Infections., Gwenn L. Skar, David C. Synhorst, Matthew Beaver, Jessica N. Snowden

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Background: Cerebrospinal fluid (CSF) shunt placement is frequently complicated by bacterial infection. Shunt infection diagnosis relies on bacterial culture of CSF which can often produce false-negative results. Negative cultures present a conundrum for physicians as they are left to rely on other CSF indices, which can be unremarkable. New methods are needed to swiftly and accurately diagnose shunt infections. CSF chemokines and cytokines may prove useful as diagnostic biomarkers. The objective of this study was to evaluate the potential of systemic and CSF biomarkers for identification of CSF shunt infection.

Methods: We conducted a retrospective chart review of children with …

Insulin Pump Adherence Behaviors Do Not Correlate With Glycemic Variability Among Youth With Type 1 Diabetes (T1d)., Emily Paprocki, Vincent S. Staggs, Susan Patton, Mark A. Clements

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No abstract provided.