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Investigating The Use Of Finerenone In Children With Chronic Kidney Disease And Proteinuria: Design Of The Fiona And Open-Label Extension Studies., Franz Schaefer, Giovanni Montini, Hee Gyung Kang, Johan Vande Walle, Joshua Zaritsky, Michiel F. Schreuder, Mieczyslaw Litwin, Andrea Scalise, Helen Scott, James Potts, Pablo Iveli, Stefanie Breitenstein, Bradley A. Warady Mar 2024

Investigating The Use Of Finerenone In Children With Chronic Kidney Disease And Proteinuria: Design Of The Fiona And Open-Label Extension Studies., Franz Schaefer, Giovanni Montini, Hee Gyung Kang, Johan Vande Walle, Joshua Zaritsky, Michiel F. Schreuder, Mieczyslaw Litwin, Andrea Scalise, Helen Scott, James Potts, Pablo Iveli, Stefanie Breitenstein, Bradley A. Warady

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INTRODUCTION: Proteinuria is a modifiable risk factor for chronic kidney disease (CKD) progression in children. Finerenone, a selective, non-steroidal, mineralocorticoid receptor antagonist (MRA) has been approved to treat adults with CKD associated with type 2 diabetes mellitus (T2DM) following results from the phase III clinical trials FIDELIO-DKD (NCT02540993) and FIGARO-DKD (NCT02545049). In a pre-specified pooled analysis of both studies (N = 13,026), finerenone was shown to have an acceptable safety profile and was efficacious in decreasing the risk of adverse kidney and cardiovascular outcomes and of proteinuria.

OBJECTIVE: FIONA and the associated open-label extension (OLE) study aim to demonstrate that …


Infant Appendicitis: A Case Presentation Of Appendicitis In A Nine-Month-Old Infant With Respiratory Syncytial Virus (Rsv) And Otitis Media And Review Of Literature., Leslie Hueschen, April Mcneill-Johnson Mar 2024

Infant Appendicitis: A Case Presentation Of Appendicitis In A Nine-Month-Old Infant With Respiratory Syncytial Virus (Rsv) And Otitis Media And Review Of Literature., Leslie Hueschen, April Mcneill-Johnson

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Appendicitis in children < 5 years old is uncommon and even less common in children < 1 year old. Symptoms of appendicitis can be non-specific and mimic other common pediatric diseases, causing delays in diagnosis. Without a timely diagnosis, young children with appendicitis are at risk of developing worsening disease, intra-abdominal abscess, perforation, and bacteremia. We submit a case of a nine-month-old male infant presenting with fever, vomiting, and irritability seen the day prior and treated for otitis media, who was ultimately diagnosed with appendicitis with concomitant viral infection (respiratory syncytial virus and human rhinovirus/enterovirus) and treated with intravenous antibiotics and laparoscopic appendectomy. This case illustrates how easy it is to misdiagnose infant appendicitis due to factors including normal developmental speech barriers, nonspecific presentations, and overlap of symptoms with many other common childhood illnesses, along with a challenging abdominal examination. Delay in diagnosis leads to increased rates of perforation and complications. Providers should trust abnormal physical examination findings, especially abdominal guarding against palpation, and keep a wide differential diagnosis in order to diagnose appendicitis in young children.


Atrial Appendage Accessory Pathway Ablation: Tips For Recognition And Approach To Ablation, Nikola Dragisic, Emily R. Backes, Lindsey Malloy-Walton, Philip M. Chang Dec 2023

Atrial Appendage Accessory Pathway Ablation: Tips For Recognition And Approach To Ablation, Nikola Dragisic, Emily R. Backes, Lindsey Malloy-Walton, Philip M. Chang

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No abstract provided.


Persistent Increase In Serum Ferritin Levels Despite Converting To Permanent Vascular Access In Pediatric Hemodialysis Patients: Pediatric Nephrology Research Consortium Study., Ali Mirza Onder, Md Abu Yusuf Ansari, Fang Deng, Matthew M. Grinsell, Larry Patterson, Jennifer Jetton, Sahar Fathallah-Shaykh, Daniel Ranch, Diego Aviles, Lawrence Copelovitch, Eileen Ellis, Vimal Chadha, Ayah Elmaghrabi, Jen-Jar Lin, Lavjay Butani, Maha Haddad, Olivera Marsenic, Paul Brakeman, Raymond Quigley, H Stella Shin, Rouba Garro, Rupesh Raina, Craig B. Langman Jun 2023

Persistent Increase In Serum Ferritin Levels Despite Converting To Permanent Vascular Access In Pediatric Hemodialysis Patients: Pediatric Nephrology Research Consortium Study., Ali Mirza Onder, Md Abu Yusuf Ansari, Fang Deng, Matthew M. Grinsell, Larry Patterson, Jennifer Jetton, Sahar Fathallah-Shaykh, Daniel Ranch, Diego Aviles, Lawrence Copelovitch, Eileen Ellis, Vimal Chadha, Ayah Elmaghrabi, Jen-Jar Lin, Lavjay Butani, Maha Haddad, Olivera Marsenic, Paul Brakeman, Raymond Quigley, H Stella Shin, Rouba Garro, Rupesh Raina, Craig B. Langman

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Our objective was to examine serum ferritin trends after conversion to permanent vascular access (PVA) among children who started hemodialysis (HD) using tunneled cuffed catheters (TCC). Retrospective chart reviews were completed on 98 subjects from 20 pediatric HD centers. Serum ferritin levels were collected at the creation of PVA and for two years thereafter. There were 11 (11%) arteriovenous grafts (AVG) and 87 (89%) arteriovenous fistulae (AVF). Their mean TCC use was 10.4 ± 17.3 months. Serum ferritin at PVA creation was elevated at 562.64 ± 492.34 ng/mL, increased to 753.84 ± 561.54 ng/mL (p = < 0.001) in the first year and remained at 759.60 ± 528.11 ng/mL in the second year (p = 0.004). …


Diagnostic Role Of Fractional Exhaled Nitric Oxide In Pediatric Eosinophilic Esophagitis, Relationship With Gastric And Duodenal Eosinophils., Panamdeep Kaur, Rachel Chevalier, Craig A. Friesen, Jamie Ryan, Ashley K. Sherman, Stephanie Page May 2023

Diagnostic Role Of Fractional Exhaled Nitric Oxide In Pediatric Eosinophilic Esophagitis, Relationship With Gastric And Duodenal Eosinophils., Panamdeep Kaur, Rachel Chevalier, Craig A. Friesen, Jamie Ryan, Ashley K. Sherman, Stephanie Page

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BACKGROUND: Eosinophilic esophagitis (EoE) is an eosinophilic-predominant inflammation of the esophagus diagnosed by upper endoscopy and biopsies. A non-invasive and cost-effective alternative for management of EoE is being researched. Previous studies assessing utility of fractional exhaled nitric oxide (FeNO) in EoE were low powered. None investigated the contribution of eosinophilic inflammation of the stomach and duodenum to FeNO.

AIM: To assess the utility of FeNO as a non-invasive biomarker of esophageal eosinophilic inflammation for monitoring disease activity.

METHODS: Patients aged 6-21 years undergoing scheduled upper endoscopy with biopsy for suspected EoE were recruited in our observational study. Patients on steroids …


Safety And Efficacy Of Voxelotor In Pediatric Patients With Sickle Cell Disease Aged 4 To 11 Years., Jeremie H. Estepp, Ram Kalpatthi, Gerald Woods, Sara Trompeter, Robert I. Liem, Kacie Sims, Adlette Inati, Baba P D Inusa, Andrew Campbell, Connie Piccone, Miguel R. Abboud, Kim Smith-Whitley, Sandra Dixon, Margaret Tonda, Carla Washington, Noelle M. Griffin, Clark Brown Aug 2022

Safety And Efficacy Of Voxelotor In Pediatric Patients With Sickle Cell Disease Aged 4 To 11 Years., Jeremie H. Estepp, Ram Kalpatthi, Gerald Woods, Sara Trompeter, Robert I. Liem, Kacie Sims, Adlette Inati, Baba P D Inusa, Andrew Campbell, Connie Piccone, Miguel R. Abboud, Kim Smith-Whitley, Sandra Dixon, Margaret Tonda, Carla Washington, Noelle M. Griffin, Clark Brown

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BACKGROUND: Sickle cell disease (SCD) is a devastating, multisystemic disorder that affects millions of people worldwide. The earliest clinical manifestations of SCD can affect infants as young as 6 months of age, and pediatric patients are at risk for acute and life-threatening complications. Early intervention with treatments that target the underlying pathophysiological mechanism of SCD, sickle hemoglobin (HbS) polymerization, are expected to slow disease progression and circumvent disease-associated morbidity and mortality.

PROCEDURE: The HOPE-KIDS 1 trial (NCT02850406) is an ongoing four-part, phase 2a, open-label, single- and multiple-dose study to evaluate the pharmacokinetics, efficacy, and safety of voxelotor-a first-in-class HbS polymerization …


Receptor Tyrosine Kinase (Rtk) Targeting In Pediatric High-Grade Glioma And Diffuse Midline Glioma: Pre-Clinical Models And Precision Medicine., Kallen Schwark, Dana Messinger, Jessica R. Cummings, Joshua Bradin, Abed Kawakibi, Clarissa M. Babila, Samantha Lyons, Sunjong Ji, Rodrigo T. Cartaxo, Seongbae Kong, Evan Cantor, Carl Koschmann, Viveka Nand Yadav Aug 2022

Receptor Tyrosine Kinase (Rtk) Targeting In Pediatric High-Grade Glioma And Diffuse Midline Glioma: Pre-Clinical Models And Precision Medicine., Kallen Schwark, Dana Messinger, Jessica R. Cummings, Joshua Bradin, Abed Kawakibi, Clarissa M. Babila, Samantha Lyons, Sunjong Ji, Rodrigo T. Cartaxo, Seongbae Kong, Evan Cantor, Carl Koschmann, Viveka Nand Yadav

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Pediatric high-grade glioma (pHGG), including both diffuse midline glioma (DMG) and non-midline tumors, continues to be one of the deadliest oncologic diagnoses (both henceforth referred to as "pHGG"). Targeted therapy options aimed at key oncogenic receptor tyrosine kinase (RTK) drivers using small-molecule RTK inhibitors has been extensively studied, but the absence of proper in vivo modeling that recapitulate pHGG biology has historically been a research challenge. Thankfully, there have been many recent advances in animal modeling, including Cre-inducible transgenic models, as well as intra-uterine electroporation (IUE) models, which closely recapitulate the salient features of human pHGG tumors. Over 20% of …


Characterization Of The Mucosally-Adherent Duodenal Microbiome In Children With And Without Crohn's Disease., Kenneth Schmidt, Janelle R. Noel-Macdonnell Phd, Carrie Vyhlidal, Daniel P. Heruth, Vivekanand Singh, Atif A. Ahmed, Taina Hudson, Veronica Williams, Valentina Shakhnovich Jul 2022

Characterization Of The Mucosally-Adherent Duodenal Microbiome In Children With And Without Crohn's Disease., Kenneth Schmidt, Janelle R. Noel-Macdonnell Phd, Carrie Vyhlidal, Daniel P. Heruth, Vivekanand Singh, Atif A. Ahmed, Taina Hudson, Veronica Williams, Valentina Shakhnovich

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Manipulation of the microbiome is a rational treatment strategy for inflammatory bowel disease (IBD). Compared to the colon and terminal ileum (TI), understanding of the microbial composition in the duodenum is sparse. This gap in knowledge is especially significant for children with Crohn's disease (CD) because the prevalence of duodenal CD is higher in children than in adults. Our aim was to characterize the bacterial composition of the mucosally-adherent duodenal microbiome in children with and without CD as a first step toward development of targeted IBD treatment strategies at this disease location. Fresh-frozen mucosal biopsies were obtained from the duodenum …


Comprehensive Evaluation Of Left Ventricular Deformation Using Speckle Tracking Echocardiography In Normal Children: Comparison Of Three-Dimensional And Two-Dimensional Approaches., Doaa Aly, Nitin Madan, Laura Kuzava, Alison Samrany, Anitha Parthiban Jan 2022

Comprehensive Evaluation Of Left Ventricular Deformation Using Speckle Tracking Echocardiography In Normal Children: Comparison Of Three-Dimensional And Two-Dimensional Approaches., Doaa Aly, Nitin Madan, Laura Kuzava, Alison Samrany, Anitha Parthiban

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BACKGROUND: Three-dimensional (3D) speckle tracking echocardiography (STE) can overcome some of the inherent limitations of two-dimensional (2D) STE; however, clinical experience is lacking. We aimed to assess and compare the feasibility, agreement, and reproducibility of left ventricular (LV) global longitudinal (GLS), and regional strain by 3D vs 2D STE in normal children.

METHODS: Healthy pediatric subjects (n = 105, age mean = 11.2 ± 5.5 years) were prospectively enrolled. Three-dimensional and 2D LV GLS, as well as regional strain in 16 myocardial segments were quantified. Bland Altman analysis, intra- class correlation coefficients (ICC), percent error and linear regression were used …


Adding Family Digital Supports To Classroom-Based Physical Activity Interventions To Target In-And Out-Of-School Activity: An Evaluation Of The Stay Active Intervention Duringthe Covid-19 Pandemic, Bethany Forseth Hanson, Adrian Ortega, Paul R. Hibbing, Mallory Moon, Chelsea Steel, Mehar Singh, Avinash Kollu, Bryce Miller, Maurice Miller, Vincent S. Staggs, Hannah Calvert, Ann M. Davis, Jordan A. Carlson Nov 2021

Adding Family Digital Supports To Classroom-Based Physical Activity Interventions To Target In-And Out-Of-School Activity: An Evaluation Of The Stay Active Intervention Duringthe Covid-19 Pandemic, Bethany Forseth Hanson, Adrian Ortega, Paul R. Hibbing, Mallory Moon, Chelsea Steel, Mehar Singh, Avinash Kollu, Bryce Miller, Maurice Miller, Vincent S. Staggs, Hannah Calvert, Ann M. Davis, Jordan A. Carlson

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Introduction: Interventions targeting multiple levels of influence and settings may have a greater impact on children’s total daily physical activity than those targeting a single setting. This study evaluated the addition of family supports to a classroom-based physical activity intervention.

Methods: 6 schools were randomized to a remotely delivered classroom-based physical activity only (CBPA) or classroom-based physical activity plus family (CBPA+) arm. The latter added behavior change tools (e.g., self-regulation) delivered via text messages and newsletters to caregiver/child dyads. The intervention lasted for 7 weeks with a 1-week baseline and 1-week post-intervention assessment (9 weeks total). Children who enrolled in …


Mortality In Children Treated With Maintenance Peritoneal Dialysis: Findings From The International Pediatric Peritoneal Dialysis Network Registry., Sophie Ploos Van Amstel, Marlies Noordzij, Dagmara Borzych-Duzalka, Nicholas C. Chesnaye, Hong Xu, Lesley Rees, Il-Soo Ha, Zenaida L. Antonio, Nakysa Hooman, William Wong, Karel Vondrak, Yok Chin Yap, Hiren Patel, Maria Szczepanska, Sara Testa, Monica Galanti, Jameela A. Kari, Charlotte Samaille, Sevcan A. Bakkaloglu, Wai-Ming Lai, Luisa Fernanda Rojas, Mabel Sandoval Diaz, Biswanath Basu, Alicia Neu, Bradley A. Warady, Kitty J. Jager, Franz Schaefer Sep 2021

Mortality In Children Treated With Maintenance Peritoneal Dialysis: Findings From The International Pediatric Peritoneal Dialysis Network Registry., Sophie Ploos Van Amstel, Marlies Noordzij, Dagmara Borzych-Duzalka, Nicholas C. Chesnaye, Hong Xu, Lesley Rees, Il-Soo Ha, Zenaida L. Antonio, Nakysa Hooman, William Wong, Karel Vondrak, Yok Chin Yap, Hiren Patel, Maria Szczepanska, Sara Testa, Monica Galanti, Jameela A. Kari, Charlotte Samaille, Sevcan A. Bakkaloglu, Wai-Ming Lai, Luisa Fernanda Rojas, Mabel Sandoval Diaz, Biswanath Basu, Alicia Neu, Bradley A. Warady, Kitty J. Jager, Franz Schaefer

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RATIONALE & OBJECTIVE: Research on pediatric kidney replacement therapy (KRT) has primarily focused on Europe and North America. In this study, we describe the mortality risk of children treated with maintenance peritoneal dialysis (MPD) in different parts of the world and characterize the associated demographic and macroeconomic factors.

STUDY DESIGN: Prospective cohort study.

SETTING & PARTICIPANTS: Patients younger than 19 years at inclusion into the International Pediatric Peritoneal Dialysis Network registry, who initiated MPD between 1996 and 2017.

EXPOSURE: Region as primary exposure (Asia, Western Europe, Eastern Europe, Latin America, North America, and Oceania). Other demographic, clinical, and macroeconomic (4 …


Ventricular Fibrillation Due To A Likely Pathogenic Sos1 Variant: An Unrecognized Etiology Of Infantile Sudden Death?, Christopher Follansbee, Lindsey Malloy-Walton Aug 2021

Ventricular Fibrillation Due To A Likely Pathogenic Sos1 Variant: An Unrecognized Etiology Of Infantile Sudden Death?, Christopher Follansbee, Lindsey Malloy-Walton

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We present the case of a female infant presenting after a ventricular fibrillation arrest found to have ectopic atrial tachycardia (EAT). Evaluation revealed a likely pathogenic variant in SOS1 not previously reported in affected individuals. SOS1 variants are associated with Noonan syndrome, which belongs to a family of related genetic syndromes affecting the RAS/MAPK signaling pathway. To date, this is the first case reported of a ventricular fibrillation arrest in a patient with a RASopathy-related variant prior to development of the typically associated structural cardiac phenotype and may represent a previously unrecognized etiology of sudden death during infancy.


Potential Role Of Methotrexate Polyglutamates In Therapeutic Drug Monitoring For Pediatric Inflammatory Bowel Disease., Ryan Morrow, Ryan Funk, Mara Becker, Ashley K. Sherman, Leon Van Haandel, Taina Hudson, Rebecca Casini, Valentina Shakhnovich May 2021

Potential Role Of Methotrexate Polyglutamates In Therapeutic Drug Monitoring For Pediatric Inflammatory Bowel Disease., Ryan Morrow, Ryan Funk, Mara Becker, Ashley K. Sherman, Leon Van Haandel, Taina Hudson, Rebecca Casini, Valentina Shakhnovich

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Inside cells, the immunomodulator methotrexate (MTX) undergoes the addition of glutamates to form methotrexate polyglutamates (MTX-Glu)-promising biomarkers of systemic exposure and treatment response to MTX in rheumatology. MTX-Glu are underexplored in Inflammatory Bowel Disease (IBD), with no data in pediatrics. In this cross-sectional secondary analysis, we assessed the relationships between MTX-Glu and MTX dose and treatment response in pediatric IBD. Twenty-one children with IBD, receiving maintenance therapy with infliximab (IFX) and MTX, had MTX-Glu1-6 concentrations and IFX troughs/antibodies measured and disease activity assessed for comparison in remission vs. active IBD using non-parametric tests, with associations explored using Spearman's correlation (ρ) …


Survival Following Relapse In Children With Acute Myeloid Leukemia: A Report From Aml-Bfm And Cog., Mareike Rasche, Martin Zimmermann, Emma Steidel, Todd Alonzo, Richard Aplenc, Jean-Pierre Bourquin, Heidrun Boztug, Todd Cooper, A S. Gamis, Robert B. Gerbing, Iveta Janotova, Jan-Henning Klusmann, Thomas Lehrnbecher, Nora Mühlegger, Nils V. Neuhoff, Naghmeh Niktoreh, Lucie Sramkova, Jan Stary, Katharina Waack, Christiane Walter, Ursula Creutzig, Michael Dworzak, Gertjan Kaspers, Edward Anders Kolb, Dirk Reinhardt May 2021

Survival Following Relapse In Children With Acute Myeloid Leukemia: A Report From Aml-Bfm And Cog., Mareike Rasche, Martin Zimmermann, Emma Steidel, Todd Alonzo, Richard Aplenc, Jean-Pierre Bourquin, Heidrun Boztug, Todd Cooper, A S. Gamis, Robert B. Gerbing, Iveta Janotova, Jan-Henning Klusmann, Thomas Lehrnbecher, Nora Mühlegger, Nils V. Neuhoff, Naghmeh Niktoreh, Lucie Sramkova, Jan Stary, Katharina Waack, Christiane Walter, Ursula Creutzig, Michael Dworzak, Gertjan Kaspers, Edward Anders Kolb, Dirk Reinhardt

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Post-relapse therapy remains critical for survival in children with acute myeloid leukemia (AML). We evaluated survival, response and prognostic variables following relapse in independent cooperative group studies conducted by COG and the population-based AML-BFM study group. BFM included 197 patients who relapsed after closure of the last I-BFM relapse trial until 2017, while COG included 852 patients who relapsed on the last Phase 3 trials (AAML0531, AAML1031). Overall survival at 5 years (OS) was 42 ± 4% (BFM) and 35 ± 2% (COG). Initial high-risk features (BFM 32 ± 6%, COG 26 ± 4%) and short time to relapse (BFM …


Efficacy Of Weight Reduction On Pediatric Nonalcoholic Fatty Liver Disease: Opportunities To Improve Treatment Outcomes Through Pharmacotherapy., Chance S. Friesen, Chelsea Hosey-Cojocari, Sherwin S. Chan, Iván L. Csanaky, Jonathan B. Wagner, Brooke Sweeney, Alec Friesen, Jason D. Fraser, Valentina Shakhnovich Apr 2021

Efficacy Of Weight Reduction On Pediatric Nonalcoholic Fatty Liver Disease: Opportunities To Improve Treatment Outcomes Through Pharmacotherapy., Chance S. Friesen, Chelsea Hosey-Cojocari, Sherwin S. Chan, Iván L. Csanaky, Jonathan B. Wagner, Brooke Sweeney, Alec Friesen, Jason D. Fraser, Valentina Shakhnovich

Manuscripts, Articles, Book Chapters and Other Papers

Obesity is the single greatest risk factor for nonalcoholic fatty liver disease (NAFLD). Without intervention, most pediatric patients with NAFLD continue to gain excessive weight, making early, effective weight loss intervention key for disease treatment and prevention of NAFLD progression. Unfortunately, outside of a closely monitored research setting, which is not representative of the real world, lifestyle modification success for weight loss in children is low. Bariatric surgery, though effective, is invasive and can worsen NAFLD postoperatively. Thus, there is an evolving and underutilized role for pharmacotherapy in children, both for weight reduction and NAFLD management. In this perspective article, …


Early Impact Of The Covid-19 Pandemic On Congenital Heart Surgery Programs Across The World: Assessment By A Global Multi-Societal Consortium., Eleftherios M. Protopapas, Mauro Lo Rito, Vladimiro L. Vida, George E. Sarris, Christo I. Tchervenkov, Bohdan J. Maruszewski, Zdzislaw Tobota, Bistra Zheleva, Hao Zhang, Jeffery P. Jacobs, Joseph A. Dearani, Elizabeth H. Stephens, James S. Tweddell, Nestor F. Sandoval, Emile A. Bacha, Erle H. Austin, Kisaburo Sakamoto, Sachin Talwar, Hiromi Kurosawa, Zohair Y Al Halees, Marcello B. Jatene, Krishna S. Iyer, Cheul Lee, Rajesh Sharma, Yasutaka Hirata, Frank Edwin, Jorge L. Cervantes, James O'Brien, James D. St Louis, James K. Kirklin, Covid-19 International Congenital Heart Surgery Taskforce Nov 2020

Early Impact Of The Covid-19 Pandemic On Congenital Heart Surgery Programs Across The World: Assessment By A Global Multi-Societal Consortium., Eleftherios M. Protopapas, Mauro Lo Rito, Vladimiro L. Vida, George E. Sarris, Christo I. Tchervenkov, Bohdan J. Maruszewski, Zdzislaw Tobota, Bistra Zheleva, Hao Zhang, Jeffery P. Jacobs, Joseph A. Dearani, Elizabeth H. Stephens, James S. Tweddell, Nestor F. Sandoval, Emile A. Bacha, Erle H. Austin, Kisaburo Sakamoto, Sachin Talwar, Hiromi Kurosawa, Zohair Y Al Halees, Marcello B. Jatene, Krishna S. Iyer, Cheul Lee, Rajesh Sharma, Yasutaka Hirata, Frank Edwin, Jorge L. Cervantes, James O'Brien, James D. St Louis, James K. Kirklin, Covid-19 International Congenital Heart Surgery Taskforce

Manuscripts, Articles, Book Chapters and Other Papers

The coronavirus disease 2019 (COVID-19) pandemic currently gripping the globe is impacting the entire health care system with rapidly escalating morbidities and mortality. Although the infectious risk to the pediatric population appears low, the effects on children with congenital heart disease (CHD) remain poorly understood. The closure of congenital heart surgery programs worldwide to address the growing number of infected individuals could have an unintended impact on future health for COVID-19-negative patients with CHD. Pediatric and congenital heart surgeons, given their small numbers and close relationships, are uniquely positioned to collectively assess the impact of the pandemic on surgical practice …


Cinacalcet Studies In Pediatric Subjects With Secondary Hyperparathyroidism Receiving Dialysis., Bradley A. Warady, Eric Ng, Laura Bloss, May Mo, Franz Schaefer, Justine Bacchetta Sep 2020

Cinacalcet Studies In Pediatric Subjects With Secondary Hyperparathyroidism Receiving Dialysis., Bradley A. Warady, Eric Ng, Laura Bloss, May Mo, Franz Schaefer, Justine Bacchetta

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BACKGROUND: Secondary hyperparathyroidism (sHPT), a complication of chronic kidney disease (CKD) characterized by persistently elevated parathyroid hormone (PTH), alterations in calcium-phosphorus homeostasis, and vitamin D metabolism, affects 50% of children receiving dialysis. A significant proportion of these children develop CKD-mineral and bone disorder (CKD-MBD), associated with an increased risk of fractures and vascular calcification. The standard of care for sHPT in children includes vitamin D sterols, calcium supplementation, and phosphate binders. Several agents are approved for sHPT treatment in adults undergoing dialysis, including vitamin D analogs and calcimimetics, with limited information on their safety and efficacy in children. The calcimimetic …


Association Between Chronic Kidney Disease-Mineral Bone Disease (Ckd-Mbd) And Cognition In Children: Chronic Kidney Disease In Children (Ckid) Study., Jennifer S. Yokoyama, Mina Matsuda-Abedini, Michelle R. Denburg, Juhi Kumar, Bradley A. Warady, Susan L. Furth, Stephen R. Hooper, Anthony A. Portale, Farzana Perwad May 2020

Association Between Chronic Kidney Disease-Mineral Bone Disease (Ckd-Mbd) And Cognition In Children: Chronic Kidney Disease In Children (Ckid) Study., Jennifer S. Yokoyama, Mina Matsuda-Abedini, Michelle R. Denburg, Juhi Kumar, Bradley A. Warady, Susan L. Furth, Stephen R. Hooper, Anthony A. Portale, Farzana Perwad

Manuscripts, Articles, Book Chapters and Other Papers

Rationale & objective: Chronic kidney disease (CKD) in children is associated with cognitive dysfunction that affects school performance and quality of life. The relationship between CKD-mineral and bone disorder and cognitive function in children is unknown.

Study design: Observational study.

Participants: 702 children enrolled in the Chronic Kidney Disease in Children (CKiD) Study.

Predictors: Plasma fibroblast growth factor 23 (FGF-23), parathyroid hormone (PTH), calcium, phosphorus, 25 hydroxyvitamin D (25[OH]D), and 1,25 dihydroxyvitamin D (1,25[OH]2D).

Outcomes: Neurocognitive tests of intelligence, academic achievement, and executive functions.

Analytical approach: Linear regression models to analyze the cross-sectional associations between log2FGF-23, 25(OH)D, 1,25(OH)2D, PTH, calcium, …


Cortisol As An Acute Stress Biomarker In Young Hematopoietic Cell Transplant Patients/Caregivers: Active Music Engagement Protocol., Kristen A. Russ, Steven J. Holochwost, Susan M. Perkins, Kristin Stegenga, Seethal A. Jacob, David Delgado, Amanda K. Henley, Joan E. Haase, Sheri L. Robb May 2020

Cortisol As An Acute Stress Biomarker In Young Hematopoietic Cell Transplant Patients/Caregivers: Active Music Engagement Protocol., Kristen A. Russ, Steven J. Holochwost, Susan M. Perkins, Kristin Stegenga, Seethal A. Jacob, David Delgado, Amanda K. Henley, Joan E. Haase, Sheri L. Robb

Manuscripts, Articles, Book Chapters and Other Papers

Objective: Primary aims of the proposed protocol are to determine the feasibility/acceptability of the active music engagement intervention protocol during hematopoietic stem cell transplantation (HSCT) and clinical feasibility/acceptability of the biological sample collection schedule.

Design: The authors propose a single-case, alternating treatment design to compare levels of child and caregiver cortisol in blood and saliva collected on alternating days, when the dyad receives and does not receive AME sessions. Included are the scientific rationale for this design and detailed intervention and sample collection schedules based on transplant type.

Setting/Location: Pediatric inpatient HSCT unit.

Subjects: Eligible participants are dyads of children …


Comparison Of The Use Of Wireless Capsule Endoscopy With Magnetic Resonance Enterography In Children With Inflammatory Bowel Disease., Nadia Mazen Hijaz, Thomas M. Attard, Jennifer Colombo, Neil J. Mardis, Craig A. Friesen Jul 2019

Comparison Of The Use Of Wireless Capsule Endoscopy With Magnetic Resonance Enterography In Children With Inflammatory Bowel Disease., Nadia Mazen Hijaz, Thomas M. Attard, Jennifer Colombo, Neil J. Mardis, Craig A. Friesen

Manuscripts, Articles, Book Chapters and Other Papers

Background: Magnetic resonance enterography (MRE) and wireless capsule endoscopy (WCE) are equally accepted modalities for noninvasive screening of small bowel involvement (SBI) in children with Crohn's disease (CD) and indeterminate colitis (IC) albeit there is a paucity of data comparing the two and thereby guiding the clinician in selecting the ideal diagnostic approach. Therefore, the goal of this study is to provide additional evidence for capsule endoscopy role in the evaluation of established Crohn's disease exacerbation compared to MRE in relation to Pediatric Crohn's Disease Activity Index (PCDAI), and histological indices.

Aim: To prospectively compare the findings of MRE and …


Pediatric Dermatology Workforce In The United States., Brea Prindaville, Kimberly A. Horii, Elaine C. Siegfried, Heather Brandling-Bennett Jan 2019

Pediatric Dermatology Workforce In The United States., Brea Prindaville, Kimberly A. Horii, Elaine C. Siegfried, Heather Brandling-Bennett

Manuscripts, Articles, Book Chapters and Other Papers

Studies have suggested there is a shortage of pediatric dermatologists in the United States, but the workforce has not been well defined. The Society for Pediatric Dermatology (SPD) Workforce Committee sought to characterize the US pediatric dermatology workforce with a nine-question survey, sent to all 484 US SPD members in December 2016. The response rate was 30%. Most pediatric dermatologists were practicing in major metropolitan markets, seeing an average of 80 patients a week with an average 6-week wait time. These findings indicate that geographic maldistribution and long wait times for new patient appointments remain substantial hurdles for adequate access …


One-Day Oral Polyethylene Glycol Based Cleanout Is Effective For Pre-Colonoscopy Preparation In Children., Ashwath S. Kumar, Brooke L. Beutler, Thomas M. Attard Nov 2018

One-Day Oral Polyethylene Glycol Based Cleanout Is Effective For Pre-Colonoscopy Preparation In Children., Ashwath S. Kumar, Brooke L. Beutler, Thomas M. Attard

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: The adequacy of pre-procedure preparation is the principal determinant of the quality of colonoscopy in pediatric as in adult patients. There is a lack of consensus, among providers on a standard pre-procedure regimen. Professional society guidelines include the use of Polyethylene glycol (PEG). Herein we report on the provider-assessed adequacy of a one day, age-categorized dosing, PEG based cleanout regimen in children undergoing colonoscopy in a tertiary institution.

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METHODS: The standard bowel preparation regime at our institution includes an age dependent minimum PEG dosing regimen in addition to clear liquids the day prior to the procedure. We retrospectively …


Pediatric Headache Clinic Model: Implementation Of Integrative Therapies In Practice., Anna E. Esparham, Anne Herbert, Emily Pierzchalski, Catherine Tran, Jennifer J. Dilts, Madeline Boorigie, Tammie Wingert, Mark Connelly, Jennifer Bickel Jun 2018

Pediatric Headache Clinic Model: Implementation Of Integrative Therapies In Practice., Anna E. Esparham, Anne Herbert, Emily Pierzchalski, Catherine Tran, Jennifer J. Dilts, Madeline Boorigie, Tammie Wingert, Mark Connelly, Jennifer Bickel

Manuscripts, Articles, Book Chapters and Other Papers

The demand for integrative medicine has risen in recent years as research has demonstrated the efficacy of such treatments. The public has also become more conscientious of the potential limitations of conventional treatment alone. Because primary headache syndromes are often the culmination of genetics, lifestyle, stress, trauma, and environmental factors, they are best treated with therapies that are equally multifaceted. The Children’s Mercy Hospital, Kansas City, Missouri Headache Clinic has successfully incorporated integrative therapies including nutraceuticals, acupuncture, aromatherapy, biofeedback, relaxation training, hypnosis, psychology services, and lifestyle recommendations for headache management. This paper provides a detailed review of the implementation of …


Echocardiographic Detection Of Increased Ventricular Diastolic Stiffness In Pediatric Heart Transplant Recipients: A Pilot Study., Shahryar M. Chowdhury, Ryan J. Butts, Anthony M. Hlavacek, Carolyn L. Taylor, Karen S. Chessa, Varsha M. Bandisode, Girish S. Shirali, Arni Nutting, G Hamilton Baker Mar 2018

Echocardiographic Detection Of Increased Ventricular Diastolic Stiffness In Pediatric Heart Transplant Recipients: A Pilot Study., Shahryar M. Chowdhury, Ryan J. Butts, Anthony M. Hlavacek, Carolyn L. Taylor, Karen S. Chessa, Varsha M. Bandisode, Girish S. Shirali, Arni Nutting, G Hamilton Baker

Manuscripts, Articles, Book Chapters and Other Papers

Background: Pediatric heart transplant recipients are at risk for increased left ventricular (LV) diastolic stiffness. However, the noninvasive evaluation of LV stiffness has remained elusive in this population. The objective of this study was to compare novel echocardiographic measures of LV diastolic stiffness versus gold-standard measures derived from pressure-volume loop (PVL) analysis in pediatric heart transplant recipients.

Methods: Patients undergoing left heart catheterization were prospectively enrolled. PVLs were obtained via conductance. The end-diastolic pressure-volume relationship was obtained via balloon occlusion. The stiffness constant, β, was calculated. Echocardiographic measures of diastolic function were derived from spectral and tissue Doppler and two-dimensional …


Characteristics Of Pediatric Antimicrobial Stewardship Programs: Current Status Of The Sharing Antimicrobial Reports For Pediatric Stewardship (Sharps) Collaborative, Chrstopher Mcpherson, Brian R. Lee, Cindy Terrill, Adam L. Hersh, Jeffrey S. Gerber, Matthew P. Kronman, Jason G. Newland Jan 2018

Characteristics Of Pediatric Antimicrobial Stewardship Programs: Current Status Of The Sharing Antimicrobial Reports For Pediatric Stewardship (Sharps) Collaborative, Chrstopher Mcpherson, Brian R. Lee, Cindy Terrill, Adam L. Hersh, Jeffrey S. Gerber, Matthew P. Kronman, Jason G. Newland

Manuscripts, Articles, Book Chapters and Other Papers

In response to the growing epidemic of antibiotic-resistant bacterial infections, antimicrobial stewardship programs (ASP) have been rapidly implemented in the United States (US). This study examines the prevalence of the Centers for Disease Control and Prevention’s (CDC) seven core elements of a successful ASP within a large subset of US Children’s Hospitals. In 2016, a survey was conducted of 52 pediatric hospitals assessing the presence of the seven core elements: leadership commitment, accountability, drug expertise, action, tracking, reporting, and education. Forty-nine hospitals (94%) had established ASPs and 41 hospitals (79%) included all seven core elements. Physician accountability (87%) and a …


A Nationwide Study On The Prevalence Of Functional Gastrointestinal Disorders In School-Children., Miguel Saps, Jairo Enrique Moreno-Gomez, Carmen Rossy Ramírez-Hernández, John M. Rosen, C A. Velasco-Benitez Nov 2017

A Nationwide Study On The Prevalence Of Functional Gastrointestinal Disorders In School-Children., Miguel Saps, Jairo Enrique Moreno-Gomez, Carmen Rossy Ramírez-Hernández, John M. Rosen, C A. Velasco-Benitez

Manuscripts, Articles, Book Chapters and Other Papers

Introduction: Functional gastrointestinal disorders (FGIDs) are highly prevalent worldwide and are thought to result from the interplay of multiple factors that can vary from region to region. Nationwide studies can help understand the regional epidemiology and the pathogenesis of FGIDs. The objectives of this study were to determine the prevalence of FGIDs in school-children of Colombia and assess associated factors.

Methods: A cross-sectional study was carried out at twelve private and public schools in ten cities distributed through the four main geographic regions of Colombia. School-children completed a validated questionnaire to assess functional gastrointestinal disorders according to Rome III criteria. …


Yap And The Hippo Pathway In Pediatric Cancer., Atif Ahmed, Abdalla D. Mohamed, Melissa Gener, Weijie Li, Eugenio Taboada Jan 2017

Yap And The Hippo Pathway In Pediatric Cancer., Atif Ahmed, Abdalla D. Mohamed, Melissa Gener, Weijie Li, Eugenio Taboada

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The Hippo pathway is an important signaling pathway that controls cell proliferation and apoptosis. It is evolutionarily conserved in mammals and is stimulated by cell-cell contact, inhibiting cell proliferation in response to increased cell density. During early embryonic development, the Hippo signaling pathway regulates organ development and size, and its functions result in the coordinated balance between proliferation, apoptosis, and differentiation. Its principal effectors, YAP and TAZ, regulate signaling by the embryonic stem cells and determine cell fate and histogenesis. Dysfunction of this pathway contributes to cancer development in adults and children. Emerging studies have shed light on the upregulation …


A 9-Year-Old Boy With A Chest Mass And Eosinophilia., Laura E. Norton, Sean N. Curtis, Jennifer L. Goldman Dec 2016

A 9-Year-Old Boy With A Chest Mass And Eosinophilia., Laura E. Norton, Sean N. Curtis, Jennifer L. Goldman

Manuscripts, Articles, Book Chapters and Other Papers

No abstract provided.


Association Between Prolonged Neutropenia And Reduced Relapse Risk In Pediatric Aml: A Report From The Children's Oncology Group., Lillian Sung, Richard Aplenc, Todd A. Alonzo, Robert B. Gerbing, Yi-Cheng Wang, Soheil Meshinchi, A S. Gamis Nov 2016

Association Between Prolonged Neutropenia And Reduced Relapse Risk In Pediatric Aml: A Report From The Children's Oncology Group., Lillian Sung, Richard Aplenc, Todd A. Alonzo, Robert B. Gerbing, Yi-Cheng Wang, Soheil Meshinchi, A S. Gamis

Manuscripts, Articles, Book Chapters and Other Papers

Objective was to describe the relationship between the number of sterile site infections and duration of neutropenia during the first four cycles of chemotherapy and the risk of recurrence and overall survival in children with newly diagnosed acute myeloid leukemia (AML). AAML0531 was a Children's Oncology Group randomized phase 3 clinical trial that included 1022 children with de novo AML. For this analysis, we focused on non-Down syndrome favorable and standard risk patients who completed at least 4 cycles of chemotherapy without recurrence or withdrawal during protocol therapy. Those receiving hematopoietic stem cell transplantation in first remission were excluded. Five …


Association Of Blood Pressure Variability And Neurocognition In Children With Chronic Kidney Disease., Marc B. Lande, Susan R. Mendley, Matthew B. Matheson, Shlomo Shinnar, Arlene C. Gerson, Joshua A. Samuels, Bradley A. Warady, Susan L. Furth, Stephen R. Hooper Nov 2016

Association Of Blood Pressure Variability And Neurocognition In Children With Chronic Kidney Disease., Marc B. Lande, Susan R. Mendley, Matthew B. Matheson, Shlomo Shinnar, Arlene C. Gerson, Joshua A. Samuels, Bradley A. Warady, Susan L. Furth, Stephen R. Hooper

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Children with chronic kidney disease (CKD) and hypertension have increased blood pressure variability (BPV), which has been associated with lower neurocognitive test scores in adults. Children with CKD are at risk for decreased neurocognitive function. Our objective was to determine whether children with CKD and increased BPV had worse performance on neurocognitive testing compared with children with CKD and lower BPV.

METHODS: This was a cross-sectional and longitudinal analysis of the relation between BPV and neurocognitive test performance in children ≥6 years enrolled in the Chronic Kidney Disease in Children (CKiD) study. Visit-to-visit BPV was assessed by the standard …