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Use Of Clearguard Hd Caps In Pediatric Hemodialysis Patients., Amy E. Nau, Troy Richardson, Diana Cardwell, Jennifer Ehrlich, Jyothsna Gattineni, Melisha Hanna, Mahima Keswani, Emily Neibauer, Kelly Nitz, Raymond Quigley, Michelle Rheault, Rebekah Sims, Mayna Woo, Bradley A. Warady Jul 2024

Use Of Clearguard Hd Caps In Pediatric Hemodialysis Patients., Amy E. Nau, Troy Richardson, Diana Cardwell, Jennifer Ehrlich, Jyothsna Gattineni, Melisha Hanna, Mahima Keswani, Emily Neibauer, Kelly Nitz, Raymond Quigley, Michelle Rheault, Rebekah Sims, Mayna Woo, Bradley A. Warady

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Bloodstream infections (BSIs) are a leading cause of hospitalizations and mortality among patients receiving hemodialysis (HD) therapy, especially those with a central venous catheter (CVC) for dialysis access. The use of chlorhexidine impregnated catheter caps (ClearGuard) has been associated with a decrease in the rate of HD catheter-related BSIs (CA-BSIs) in adults; similar data have not been published for children.

METHODS: We compared CA-BSI data from participating centers within the Standardizing Care to Improve Outcomes in Pediatric Endstage Kidney Disease (SCOPE) collaborative based on the center's use of ClearGuard caps for patients with HD catheter access. Centers were characterized …


Characteristics And Treatment Of Acute Myeloid Neoplasms With Cutaneous Involvement In Infants Up To 6 Months Of Age: A Retrospective Study., Juliette Renaud, Bianca F. Goemans, Franco Locatelli, Martina Pigazzi, Shelagh Redmond, Claudia E. Kuehni, Alice Destaillats, Todd A. Alonzo, Robert B. Gerbing, Alan S. Gamis, Richard Aplenc, Raffaele Renella, Todd Cooper, Francesco Ceppi Jul 2024

Characteristics And Treatment Of Acute Myeloid Neoplasms With Cutaneous Involvement In Infants Up To 6 Months Of Age: A Retrospective Study., Juliette Renaud, Bianca F. Goemans, Franco Locatelli, Martina Pigazzi, Shelagh Redmond, Claudia E. Kuehni, Alice Destaillats, Todd A. Alonzo, Robert B. Gerbing, Alan S. Gamis, Richard Aplenc, Raffaele Renella, Todd Cooper, Francesco Ceppi

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Myeloid neoplasms account for 50% of cases of pediatric leukemias in infants. Approximately 25%-50% of patients with newborn leukemia have cutaneous extramedullary disease (EMD). In less than 10% of patients, aleukemic leukemia cutis or isolated extramedullary disease with cutaneous involvement (cEMD) occurs when skin lesions appear prior to bone marrow involvement and systemic symptoms. Interestingly, in acute myeloid leukemia with cutaneous EMD (AML-cEMD) and cEMD, spontaneous remissions have been reported.

METHOD: This is a multicentric retrospective cohort study aiming to describe characteristics, treatment, and outcome of infants with either cEMD or presence of cutaneous disease with involvement of the …


Delays To Antibiotics In The Emergency Department And Risk Of Mortality In Children With Sepsis., Roni D. Lane, Troy Richardson, Halden F. Scott, Raina M. Paul, Fran Balamuth, Matthew A. Eisenberg, Ruth Riggs, W Charles Huskins, Christopher M. Horvat, Grant E. Keeney, Leslie Hueschen, Justin M. Lockwood, Vishal Gunnala, Bryan P. Mckee, Nikhil Patankar, Venessa Lynn Pinto, Amanda M. Sebring, Matthew P. Sharron, Jennifer Treseler, Jennifer J. Wilkes, Jennifer K. Workman Jun 2024

Delays To Antibiotics In The Emergency Department And Risk Of Mortality In Children With Sepsis., Roni D. Lane, Troy Richardson, Halden F. Scott, Raina M. Paul, Fran Balamuth, Matthew A. Eisenberg, Ruth Riggs, W Charles Huskins, Christopher M. Horvat, Grant E. Keeney, Leslie Hueschen, Justin M. Lockwood, Vishal Gunnala, Bryan P. Mckee, Nikhil Patankar, Venessa Lynn Pinto, Amanda M. Sebring, Matthew P. Sharron, Jennifer Treseler, Jennifer J. Wilkes, Jennifer K. Workman

Manuscripts, Articles, Book Chapters and Other Papers

IMPORTANCE: Pediatric consensus guidelines recommend antibiotic administration within 1 hour for septic shock and within 3 hours for sepsis without shock. Limited studies exist identifying a specific time past which delays in antibiotic administration are associated with worse outcomes.

OBJECTIVE: To determine a time point for antibiotic administration that is associated with increased risk of mortality among pediatric patients with sepsis.

DESIGN, SETTING, AND PARTICIPANTS: This retrospective cohort study used data from 51 US children's hospitals in the Improving Pediatric Sepsis Outcomes collaborative. Participants included patients aged 29 days to less than 18 years with sepsis recognized within 1 hour …


Influence Of Eat, Sleep, And Console On Infants Pharmacologically Treated For Opioid Withdrawal: A Post Hoc Subgroup Analysis Of The Esc-Now Randomized Clinical Trial., Lori A. Devlin, Zhuopei Hu, Stephanie L. Merhar, Songthip T. Ounpraseuth, Alan E. Simon, Jeannette Y. Lee, Abhik Das, Margaret M. Crawford, Rachel G. Greenberg, P Brian Smith, Rosemary D. Higgins, Michele C. Walsh, Ward Rice, David A. Paul, Jessie R. Maxwell, Camille M. Fung, Tanner Wright, Julie Ross, Jennifer M. Mcallister, Moira Crowley, Sophie K. Shaikh, Lori Christ, Jaime Brown, Julie Riccio, Kara Wong Ramsey, Erica F. Braswell, Lauren Tucker, Karen Mcalmon, Krishna Dummula, Julie Weiner, Jessica R. White, Sarah Newman, Jessica N. Snowden, Leslie W. Young, Eunice Kennedy Shriver Nichd Neonatal Research Network And Nih Environmental Influences On Child Health Outcomes (Echo) Program Institutional Development Award States Pediatric Clinical Trials Network Jun 2024

Influence Of Eat, Sleep, And Console On Infants Pharmacologically Treated For Opioid Withdrawal: A Post Hoc Subgroup Analysis Of The Esc-Now Randomized Clinical Trial., Lori A. Devlin, Zhuopei Hu, Stephanie L. Merhar, Songthip T. Ounpraseuth, Alan E. Simon, Jeannette Y. Lee, Abhik Das, Margaret M. Crawford, Rachel G. Greenberg, P Brian Smith, Rosemary D. Higgins, Michele C. Walsh, Ward Rice, David A. Paul, Jessie R. Maxwell, Camille M. Fung, Tanner Wright, Julie Ross, Jennifer M. Mcallister, Moira Crowley, Sophie K. Shaikh, Lori Christ, Jaime Brown, Julie Riccio, Kara Wong Ramsey, Erica F. Braswell, Lauren Tucker, Karen Mcalmon, Krishna Dummula, Julie Weiner, Jessica R. White, Sarah Newman, Jessica N. Snowden, Leslie W. Young, Eunice Kennedy Shriver Nichd Neonatal Research Network And Nih Environmental Influences On Child Health Outcomes (Echo) Program Institutional Development Award States Pediatric Clinical Trials Network

Manuscripts, Articles, Book Chapters and Other Papers

IMPORTANCE: The function-based eat, sleep, console (ESC) care approach substantially reduces the proportion of infants who receive pharmacologic treatment for neonatal opioid withdrawal syndrome (NOWS). This reduction has led to concerns for increased postnatal opioid exposure in infants who receive pharmacologic treatment. However, the effect of the ESC care approach on hospital outcomes for infants pharmacologically treated for NOWS is currently unknown.

OBJECTIVE: To evaluate differences in opioid exposure and total length of hospital stay (LOS) for pharmacologically treated infants managed with the ESC care approach vs usual care with the Finnegan tool.

DESIGN, SETTING, AND PARTICIPANTS: This post hoc …


Improved Pediatric Icu Mortality Prediction For Respiratory Diseases: Machine Learning And Data Subdivision Insights, Johayra Prithula, Muhammad E H Chowdhury, Muhammad Salman Khan, Khalid Al-Ansari, Susu M Zughaier, Khandaker Reajul Islam, Abdulrahman Alqahtani May 2024

Improved Pediatric Icu Mortality Prediction For Respiratory Diseases: Machine Learning And Data Subdivision Insights, Johayra Prithula, Muhammad E H Chowdhury, Muhammad Salman Khan, Khalid Al-Ansari, Susu M Zughaier, Khandaker Reajul Islam, Abdulrahman Alqahtani

Journal Articles

The growing concern of pediatric mortality demands heightened preparedness in clinical settings, especially within intensive care units (ICUs). As respiratory-related admissions account for a substantial portion of pediatric illnesses, there is a pressing need to predict ICU mortality in these cases. This study based on data from 1188 patients, addresses this imperative using machine learning techniques and investigating different class balancing methods for pediatric ICU mortality prediction. This study employs the publicly accessible "Paediatric Intensive Care database" to train, validate, and test a machine learning model for predicting pediatric patient mortality. Features were ranked using three machine learning feature selection …


Seasonality, Clinical Characteristics, And Outcomes Of Respiratory Syncytial Virus Disease By Subtype Among Children Aged <5 Years: New Vaccine Surveillance Network, United States, 2016-2020., Ariana P. Toepfer, Justin Z. Amarin, Andrew J. Spieker, Laura S. Stewart, Mary Allen Staat, Elizabeth P. Schlaudecker, Geoffrey A. Weinberg, Peter G. Szilagyi, Janet A. Englund, Eileen J. Klein, Marian G. Michaels, John V. Williams, Rangaraj Selvarangan, Christopher J. Harrison, Joana Y. Lively, Pedro A. Piedra, Vasanthi Avadhanula, Brian Rha, James Chappell, Meredith Mcmorrow, Heidi Moline, Natasha B. Halasa May 2024

Seasonality, Clinical Characteristics, And Outcomes Of Respiratory Syncytial Virus Disease By Subtype Among Children Aged <5 Years: New Vaccine Surveillance Network, United States, 2016-2020., Ariana P. Toepfer, Justin Z. Amarin, Andrew J. Spieker, Laura S. Stewart, Mary Allen Staat, Elizabeth P. Schlaudecker, Geoffrey A. Weinberg, Peter G. Szilagyi, Janet A. Englund, Eileen J. Klein, Marian G. Michaels, John V. Williams, Rangaraj Selvarangan, Christopher J. Harrison, Joana Y. Lively, Pedro A. Piedra, Vasanthi Avadhanula, Brian Rha, James Chappell, Meredith Mcmorrow, Heidi Moline, Natasha B. Halasa

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Respiratory syncytial virus (RSV) is a leading cause of acute respiratory illnesses in children. RSV can be broadly categorized into 2 major subtypes: A and B. RSV subtypes have been known to cocirculate with variability in different regions of the world. Clinical associations with viral subtype have been studied among children with conflicting findings such that no conclusive relationships between RSV subtype and severity have been established.

METHODS: During 2016-2020, children aged/guardian interviews, chart reviews, and collection of midturbinate nasal plus/minus throat swabs for RSV (RSV-A, RSV-B, and untyped) using reverse transcription polymerase chain reaction.

RESULTS: Among 6398 RSV-positive …


Prognostic Impact Of Cooccurring Mutations In Flt3-Itd Pediatric Acute Myeloid Leukemia., Katherine Tarlock, Robert B. Gerbing, Rhonda E. Ries, Jenny L. Smith, Amanda Leonti, Benjamin J. Huang, Danielle Kirkey, Leila Robinson, Jack H. Peplinksi, Beverly Lange, Todd M. Cooper, Alan S. Gamis, E Anders Kolb, Richard Aplenc, Jessica A. Pollard, Todd A. Alonzo, Soheil Meshinchi May 2024

Prognostic Impact Of Cooccurring Mutations In Flt3-Itd Pediatric Acute Myeloid Leukemia., Katherine Tarlock, Robert B. Gerbing, Rhonda E. Ries, Jenny L. Smith, Amanda Leonti, Benjamin J. Huang, Danielle Kirkey, Leila Robinson, Jack H. Peplinksi, Beverly Lange, Todd M. Cooper, Alan S. Gamis, E Anders Kolb, Richard Aplenc, Jessica A. Pollard, Todd A. Alonzo, Soheil Meshinchi

Manuscripts, Articles, Book Chapters and Other Papers

We sought to define the cooccurring mutational profile of FLT3-ITD-positive (ITDpos) acute myeloid leukemia (AML) in pediatric and young adult patients and to define the prognostic impact of cooperating mutations. We identified 464 patients with FLT3-ITD mutations treated on Children's Oncology Group trials with available sequencing and outcome data. Overall survival, event-free survival (EFS), and relapse risk were determined according to the presence of cooccurring risk stratifying mutations. Among the cohort, 79% of patients had cooccurring alterations across 239 different genes that were altered through mutations or fusions. Evaluation of the prognostic impact of the cooccurring mutations demonstrated that patients …


Researching Covid To Enhance Recovery (Recover) Pediatric Study Protocol: Rationale, Objectives And Design., Rachel S. Gross, Tanayott Thaweethai, Erika B. Rosenzweig, James Chan, Lori B. Chibnik, Mine S. Cicek, Amy J. Elliott, Valerie J. Flaherman, Andrea S. Foulkes, Margot Gage Witvliet, Richard Gallagher, Maria Laura Gennaro, Terry L. Jernigan, Elizabeth W. Karlson, Stuart D. Katz, Patricia A. Kinser, Lawrence C. Kleinman, Michelle F. Lamendola-Essel, Joshua D. Milner, Sindhu Mohandas, Praveen C. Mudumbi, Jane W. Newburger, Kyung E. Rhee, Amy L. Salisbury, Jessica N. Snowden, Cheryl R. Stein, Melissa S. Stockwell, Kelan G. Tantisira, Moriah E. Thomason, Dongngan T. Truong, David Warburton, John C. Wood, Shifa Ahmed, Almary Akerlundh, Akram N. Alshawabkeh, Brett R. Anderson, Judy L. Aschner, Andrew M. Atz, Robin L. Aupperle, Fiona C. Baker, Venkataraman Balaraman, Dithi Banerjee, Deanna M. Barch, Arielle Baskin-Sommers, Sultana Bhuiyan, Marie-Abele C. Bind, Amanda L. Bogie, Tamara Bradford, Natalie C. Buchbinder, Elliott Bueler, Hülya Bükülmez, B J Casey, Linda Chang, Maryanne Chrisant, Duncan B. Clark, Rebecca G. Clifton, Katharine N. Clouser, Lesley Cottrell, Kelly Cowan, Viren D'Sa, Mirella Dapretto, Soham Dasgupta, Walter Dehority, Audrey Dionne, Kirsten B. Dummer, Matthew D. Elias, Shari Esquenazi-Karonika, Danielle N. Evans, E Vincent S. Faustino, Alexander G. Fiks, Daniel Forsha, John J. Foxe, Naomi P. Friedman, Greta Fry, Sunanda Gaur, Dylan G. Gee, Kevin M. Gray, Stephanie Handler, Ashraf S. Harahsheh, Keren Hasbani, Andrew C. Heath, Camden Hebson, Mary M. Heitzeg, Christina M. Hester, Sophia Hill, Laura Hobart-Porter, Travis K F Hong, Carol R. Horowitz, Daniel S. Hsia, Matthew Huentelman, Kathy D. Hummel, Katherine Irby, Joanna Jacobus, Vanessa L. Jacoby, Pei-Ni Jone, David C. Kaelber, Tyler J. Kasmarcak, Matthew J. Kluko, Jessica S. Kosut, Angela R. Laird, Jeremy Landeo-Gutierrez, Sean M. Lang, Christine L. Larson, Peter Paul C. Lim, Krista M. Lisdahl, Brian W. Mccrindle, Russell J. Mcculloh, Kimberly Mchugh, Alan L. Mendelsohn, Torri D. Metz, Julie Miller, Elizabeth C. Mitchell, Lerraughn M. Morgan, Eva M. Müller-Oehring, Erica R. Nahin, Michael C. Neale, Manette Ness-Cochinwala, Sheila M. Nolan, Carlos R. Oliveira, Onyekachukwu Osakwe, Matthew E. Oster, R Mark Payne, Michael A. Portman, Hengameh Raissy, Isabelle G. Randall, Suchitra Rao, Harrison T. Reeder, Johana M. Rosas, Mark W. Russell, Arash A. Sabati, Yamuna Sanil, Alice I. Sato, Michael S. Schechter, Rangaraj Selvarangan, S Kristen Sexson Tejtel, Divya Shakti, Kavita Sharma, Lindsay M. Squeglia, Shubika Srivastava, Michelle D. Stevenson, Jacqueline Szmuszkovicz, Maria M. Talavera-Barber, Ronald J. Teufel, Deepika Thacker, Felicia Trachtenberg, Mmekom M. Udosen, Megan R. Warner, Sara E. Watson, Alan Werzberger, Jordan C. Weyer, Marion J. Wood, H Shonna Yin, William T. Zempsky, Emily Zimmerman, Benard P. Dreyer, Recover-Pediatric Consortium May 2024

Researching Covid To Enhance Recovery (Recover) Pediatric Study Protocol: Rationale, Objectives And Design., Rachel S. Gross, Tanayott Thaweethai, Erika B. Rosenzweig, James Chan, Lori B. Chibnik, Mine S. Cicek, Amy J. Elliott, Valerie J. Flaherman, Andrea S. Foulkes, Margot Gage Witvliet, Richard Gallagher, Maria Laura Gennaro, Terry L. Jernigan, Elizabeth W. Karlson, Stuart D. Katz, Patricia A. Kinser, Lawrence C. Kleinman, Michelle F. Lamendola-Essel, Joshua D. Milner, Sindhu Mohandas, Praveen C. Mudumbi, Jane W. Newburger, Kyung E. Rhee, Amy L. Salisbury, Jessica N. Snowden, Cheryl R. Stein, Melissa S. Stockwell, Kelan G. Tantisira, Moriah E. Thomason, Dongngan T. Truong, David Warburton, John C. Wood, Shifa Ahmed, Almary Akerlundh, Akram N. Alshawabkeh, Brett R. Anderson, Judy L. Aschner, Andrew M. Atz, Robin L. Aupperle, Fiona C. Baker, Venkataraman Balaraman, Dithi Banerjee, Deanna M. Barch, Arielle Baskin-Sommers, Sultana Bhuiyan, Marie-Abele C. Bind, Amanda L. Bogie, Tamara Bradford, Natalie C. Buchbinder, Elliott Bueler, Hülya Bükülmez, B J Casey, Linda Chang, Maryanne Chrisant, Duncan B. Clark, Rebecca G. Clifton, Katharine N. Clouser, Lesley Cottrell, Kelly Cowan, Viren D'Sa, Mirella Dapretto, Soham Dasgupta, Walter Dehority, Audrey Dionne, Kirsten B. Dummer, Matthew D. Elias, Shari Esquenazi-Karonika, Danielle N. Evans, E Vincent S. Faustino, Alexander G. Fiks, Daniel Forsha, John J. Foxe, Naomi P. Friedman, Greta Fry, Sunanda Gaur, Dylan G. Gee, Kevin M. Gray, Stephanie Handler, Ashraf S. Harahsheh, Keren Hasbani, Andrew C. Heath, Camden Hebson, Mary M. Heitzeg, Christina M. Hester, Sophia Hill, Laura Hobart-Porter, Travis K F Hong, Carol R. Horowitz, Daniel S. Hsia, Matthew Huentelman, Kathy D. Hummel, Katherine Irby, Joanna Jacobus, Vanessa L. Jacoby, Pei-Ni Jone, David C. Kaelber, Tyler J. Kasmarcak, Matthew J. Kluko, Jessica S. Kosut, Angela R. Laird, Jeremy Landeo-Gutierrez, Sean M. Lang, Christine L. Larson, Peter Paul C. Lim, Krista M. Lisdahl, Brian W. Mccrindle, Russell J. Mcculloh, Kimberly Mchugh, Alan L. Mendelsohn, Torri D. Metz, Julie Miller, Elizabeth C. Mitchell, Lerraughn M. Morgan, Eva M. Müller-Oehring, Erica R. Nahin, Michael C. Neale, Manette Ness-Cochinwala, Sheila M. Nolan, Carlos R. Oliveira, Onyekachukwu Osakwe, Matthew E. Oster, R Mark Payne, Michael A. Portman, Hengameh Raissy, Isabelle G. Randall, Suchitra Rao, Harrison T. Reeder, Johana M. Rosas, Mark W. Russell, Arash A. Sabati, Yamuna Sanil, Alice I. Sato, Michael S. Schechter, Rangaraj Selvarangan, S Kristen Sexson Tejtel, Divya Shakti, Kavita Sharma, Lindsay M. Squeglia, Shubika Srivastava, Michelle D. Stevenson, Jacqueline Szmuszkovicz, Maria M. Talavera-Barber, Ronald J. Teufel, Deepika Thacker, Felicia Trachtenberg, Mmekom M. Udosen, Megan R. Warner, Sara E. Watson, Alan Werzberger, Jordan C. Weyer, Marion J. Wood, H Shonna Yin, William T. Zempsky, Emily Zimmerman, Benard P. Dreyer, Recover-Pediatric Consortium

Manuscripts, Articles, Book Chapters and Other Papers

IMPORTANCE: The prevalence, pathophysiology, and long-term outcomes of COVID-19 (post-acute sequelae of SARS-CoV-2 [PASC] or "Long COVID") in children and young adults remain unknown. Studies must address the urgent need to define PASC, its mechanisms, and potential treatment targets in children and young adults.

OBSERVATIONS: We describe the protocol for the Pediatric Observational Cohort Study of the NIH's REsearching COVID to Enhance Recovery (RECOVER) Initiative. RECOVER-Pediatrics is an observational meta-cohort study of caregiver-child pairs (birth through 17 years) and young adults (18 through 25 years), recruited from more than 100 sites across the US. This report focuses on two of …


Availability And Utilization Of Endoscopic Retrograde Cholangiopancreatography At Children's Hospitals., Sagar J. Pathak, Thomas M. Attard, Matthew Hall, Mustafa Arain, Melvin B. Heyman, Emily R. Perito May 2024

Availability And Utilization Of Endoscopic Retrograde Cholangiopancreatography At Children's Hospitals., Sagar J. Pathak, Thomas M. Attard, Matthew Hall, Mustafa Arain, Melvin B. Heyman, Emily R. Perito

Manuscripts, Articles, Book Chapters and Other Papers

OBJECTIVES: No study has explored whether availability of endoscopic retrograde cholangiopancreatography (ERCP) is adequate and equitable across US children's hospitals. We hypothesized that ERCP availability and utilization differs by geography and patient factors.

METHODS: Healthcare encounter data from 2009 to 2019 on children with pancreatic and biliary diseases from the Pediatric Health Information System were analyzed. ERCP availability was defined as treatment at a hospital that performed pediatric ERCP during the year of service.

RESULTS: From 2009 to 2019, 37,946 children (88,420 encounters) had a potential pancreatic or biliary indication for ERCP; 7066 ERCPs were performed. The commonest pancreatic diagnoses …


Approach For Defining Human Adenovirus Infection And Disease For Central Review Adjudication In Clinical Studies., Brian T. Fisher, Jesse Blumenstock, Craig L K Boge, Sydney Shuster, Alix E. Seif, Michael Green, Marian G. Michaels, Jessie L. Alexander, Monica I. Ardura, Tamara P. Miller, Diego R. Hijano, William J. Muller, Jennifer E. Schuster, Abby M. Green, Daniel E. Dulek, Adriana E. Kajon, Lara Danziger-Isakov May 2024

Approach For Defining Human Adenovirus Infection And Disease For Central Review Adjudication In Clinical Studies., Brian T. Fisher, Jesse Blumenstock, Craig L K Boge, Sydney Shuster, Alix E. Seif, Michael Green, Marian G. Michaels, Jessie L. Alexander, Monica I. Ardura, Tamara P. Miller, Diego R. Hijano, William J. Muller, Jennifer E. Schuster, Abby M. Green, Daniel E. Dulek, Adriana E. Kajon, Lara Danziger-Isakov

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Pediatric allogeneic hematopoietic cell transplant (allo-HCT) recipients are at risk for morbidity and mortality from human adenovirus (HAdV). HAdV can be detected in an asymptomatic state, referred to as infection or with signs or symptoms of illness, referred to as disease. Standardized case definitions are needed to distinguish infection from disease and allow for consistent reporting in both observational cohort studies and therapeutic clinical trials.

METHODS: A working group of experts in virology, transplant infectious disease, and HCT was assembled to develop HAdV infection and disease definitions with the degree of certainty (i.e., possible, probable, and proven). Definitions were …


Population Pharmacokinetic Analysis Of Atomoxetine And Its Metabolites In Children And Adolescents With Attention-Deficit/Hyperactivity Disorder., Shen Cheng, Mahmoud Al-Kofahi, J Steven Leeder, Jacob T. Brown May 2024

Population Pharmacokinetic Analysis Of Atomoxetine And Its Metabolites In Children And Adolescents With Attention-Deficit/Hyperactivity Disorder., Shen Cheng, Mahmoud Al-Kofahi, J Steven Leeder, Jacob T. Brown

Manuscripts, Articles, Book Chapters and Other Papers

Atomoxetine (ATX) is a non-stimulant used to treat attention-deficit/hyperactivity disorder (ADHD) and systemic exposure is highly variable due to polymorphic cytochrome P450 2D6 (CYP2D6) activity. The objective of this study was to characterize the time course of ATX and metabolites (4-hydroxyatomoxetine (4-OH); N-desmethylatomoxetine (NDA); and 2-carboxymethylatomoxetine (2-COOH)) exposure following oral ATX dosing in children with ADHD to support individualized dosing. A nonlinear mixed-effect modeling approach was used to analyze ATX, 4-OH, and NDA plasma and urine, and 2-COOH urine profiles obtained over 24-72 hours from children with ADHD (n = 23) following a single oral ATX dose. Demographics and CYP2D6 …


Prognostic Values Of Serum Lactate-To-Bicarbonate Ratio And Lactate For Predicting 28-Day In-Hospital Mortality In Children With Dengue Shock Syndrome, Thanh Nguyen Tat, Nhu Vo Hoang-Thien, Dat Nguyen Tat, Phuc Hoang Nguyen, Lien Thi Ho, Duong Hung Doan, Dung Tuan Phan, Yen Nguyen-Hoang Duong, Truc Huynh Nguyen, Tuyet Kim Nguyen, Ha Thi-Thu Dinh, Thuy Thi-Diem Dinh, Anh Thi-Mai Pham, Viet Do Chau, Tung Huu Trinh, Luan Vo Thanh Apr 2024

Prognostic Values Of Serum Lactate-To-Bicarbonate Ratio And Lactate For Predicting 28-Day In-Hospital Mortality In Children With Dengue Shock Syndrome, Thanh Nguyen Tat, Nhu Vo Hoang-Thien, Dat Nguyen Tat, Phuc Hoang Nguyen, Lien Thi Ho, Duong Hung Doan, Dung Tuan Phan, Yen Nguyen-Hoang Duong, Truc Huynh Nguyen, Tuyet Kim Nguyen, Ha Thi-Thu Dinh, Thuy Thi-Diem Dinh, Anh Thi-Mai Pham, Viet Do Chau, Tung Huu Trinh, Luan Vo Thanh

Journal Articles

This study aimed to assess the clinical utility of blood lactate-to-bicarbonate (L/B) ratio, as a prognostic factor for 28-day in-hospital mortality in children with dengue shock syndrome (DSS), admitted to the pediatric intensive care unit (PICU). This single-center retrospective study was conducted at a tertiary children hospital in southern Vietnam from 2013 to mid-2022. Prognostic models for DSS mortality were developed, using a predefined set of covariates in the first 24 hours of PICU admission. Area under the curves (AUCs), multivariable logistic and Least Absolute Shrinkage and Selection Operator (LASSO) regressions, bootstrapping and calibration slope were performed. A total of …


Structural Variants Involving Mllt10 Fusion Are Associated With Adverse Outcomes In Pediatric Acute Myeloid Leukemia., Oussama Abla, Rhonda E. Ries, Tim Triche, Robert B. Gerbing, Betsy Hirsch, Susana Raimondi, Todd Cooper, Jason E. Farrar, Nathaniel Buteyn, Lauren M. Harmon, Hong Wen, Aniruddha J. Deshpande, E Anders Kolb, Alan S. Gamis, Richard Aplenc, Todd Alonzo, Soheil Meshinchi Apr 2024

Structural Variants Involving Mllt10 Fusion Are Associated With Adverse Outcomes In Pediatric Acute Myeloid Leukemia., Oussama Abla, Rhonda E. Ries, Tim Triche, Robert B. Gerbing, Betsy Hirsch, Susana Raimondi, Todd Cooper, Jason E. Farrar, Nathaniel Buteyn, Lauren M. Harmon, Hong Wen, Aniruddha J. Deshpande, E Anders Kolb, Alan S. Gamis, Richard Aplenc, Todd Alonzo, Soheil Meshinchi

Manuscripts, Articles, Book Chapters and Other Papers

MLLT10 gene rearrangements with KMT2A occur in pediatric acute myeloid leukemia (AML) and confer poor prognosis, but the prognostic impact of MLLT10 in partnership with other genes is unknown. We conducted a retrospective study with 2080 children and young adults with AML registered on the Children's Oncology Group AAML0531 (NCT00372593) and AAML1031 trials (NCT01371981). Transcriptome profiling and/or karyotyping were performed to identify leukemia-associated fusions associated with prognosis. Collectively, 127 patients (6.1%) were identified with MLLT10 fusions: 104 (81.9%) with KMT2A::MLLT10, 13 (10.2%) with PICALM::MLLT10, and 10 (7.9%) X::MLLT10: (2 each of DDX3X and TEC), with 6 partners (DDX3Y, CEP164, SCN2B, …


Immunophenotypic Predictors Of Influenza Vaccine Immunogenicity In Pediatric Hematopoietic Cell Transplant Recipients., Justin Z. Amarin, Daniel E. Dulek, Joshua Simmons, Haya Hayek, James D. Chappell, Cindy Hager Nochowicz, Carrie L. Kitko, Jennifer E. Schuster, Flor M. Muñoz, Claire E. Bocchini, Elizabeth A. Moulton, Susan E. Coffin, Jason L. Freedman, Monica I. Ardura, Rachel L. Wattier, Gabriela Maron, Michael Grimley, Grant Paulsen, Lara Danziger-Isakov, Paul A. Carpenter, Janet A. Englund, Natasha B. Halasa, Andrew J. Spieker, Spyros A. Kalams Apr 2024

Immunophenotypic Predictors Of Influenza Vaccine Immunogenicity In Pediatric Hematopoietic Cell Transplant Recipients., Justin Z. Amarin, Daniel E. Dulek, Joshua Simmons, Haya Hayek, James D. Chappell, Cindy Hager Nochowicz, Carrie L. Kitko, Jennifer E. Schuster, Flor M. Muñoz, Claire E. Bocchini, Elizabeth A. Moulton, Susan E. Coffin, Jason L. Freedman, Monica I. Ardura, Rachel L. Wattier, Gabriela Maron, Michael Grimley, Grant Paulsen, Lara Danziger-Isakov, Paul A. Carpenter, Janet A. Englund, Natasha B. Halasa, Andrew J. Spieker, Spyros A. Kalams

Manuscripts, Articles, Book Chapters and Other Papers

Pediatric hematopoietic cell transplant (HCT) recipients exhibit poor serologic responses to influenza vaccination early after transplant. To facilitate the optimization of influenza vaccination timing, we sought to identify B- and T-cell subpopulations associated with influenza vaccine immunogenicity in this population. We used mass cytometry to phenotype peripheral blood mononuclear cells collected from pediatric HCT recipients enrolled in a multicenter influenza vaccine trial comparing high- and standard-dose formulations over 3 influenza seasons (2016-2019). We fit linear regression models to estimate relationships between immune cell subpopulation numbers before vaccination and prevaccination to postvaccination geometric mean fold rises in antigen-specific (A/H3N2, A/H1N1, and …


Durability Of Original Monovalent Mrna Vaccine Effectiveness Against Covid-19 Omicron-Associated Hospitalization In Children And Adolescents - United States, 2021-2023., Laura D. Zambrano, Margaret M. Newhams, Regina M. Simeone, Amanda B. Payne, Michael Wu, Amber O. Orzel-Lockwood, Natasha B. Halasa, Jemima M. Calixte, Pia S. Pannaraj, Kanokporn Mongkolrattanothai, Julie A Boom, Leila C Sahni, Satoshi Kamidani, Kathleen Chiotos, Melissa A Cameron, Aline B Maddux, Katherine Irby, Jennifer E. Schuster, Elizabeth H. Mack, Austin Biggs, Bria M. Coates, Kelly N. Michelson, Katherine E. Bline, Ryan A. Nofziger, Hillary Crandall, Charlotte V. Hobbs, Shira J. Gertz, Sabrina M. Heidemann, Tamara T. Bradford, Tracie C. Walker, Stephanie P. Schwartz, Mary Allen Staat, Samina S. Bhumbra, Janet R. Hume, Michele Kong, Melissa S. Stockwell, Thomas J. Connors, Melissa L. Cullimore, Heidi R. Flori, Emily R. Levy, Natalie Z. Cvijanovich, Matt S. Zinter, Mia Maamari, Cindy Bowens, Danielle M. Zerr, Judith A. Guzman-Cottrill, Ivan Gonzalez, Angela P. Campbell, Adrienne G. Randolph, Overcoming Covid-19 Investigators Apr 2024

Durability Of Original Monovalent Mrna Vaccine Effectiveness Against Covid-19 Omicron-Associated Hospitalization In Children And Adolescents - United States, 2021-2023., Laura D. Zambrano, Margaret M. Newhams, Regina M. Simeone, Amanda B. Payne, Michael Wu, Amber O. Orzel-Lockwood, Natasha B. Halasa, Jemima M. Calixte, Pia S. Pannaraj, Kanokporn Mongkolrattanothai, Julie A Boom, Leila C Sahni, Satoshi Kamidani, Kathleen Chiotos, Melissa A Cameron, Aline B Maddux, Katherine Irby, Jennifer E. Schuster, Elizabeth H. Mack, Austin Biggs, Bria M. Coates, Kelly N. Michelson, Katherine E. Bline, Ryan A. Nofziger, Hillary Crandall, Charlotte V. Hobbs, Shira J. Gertz, Sabrina M. Heidemann, Tamara T. Bradford, Tracie C. Walker, Stephanie P. Schwartz, Mary Allen Staat, Samina S. Bhumbra, Janet R. Hume, Michele Kong, Melissa S. Stockwell, Thomas J. Connors, Melissa L. Cullimore, Heidi R. Flori, Emily R. Levy, Natalie Z. Cvijanovich, Matt S. Zinter, Mia Maamari, Cindy Bowens, Danielle M. Zerr, Judith A. Guzman-Cottrill, Ivan Gonzalez, Angela P. Campbell, Adrienne G. Randolph, Overcoming Covid-19 Investigators

Manuscripts, Articles, Book Chapters and Other Papers

Pediatric COVID-19 vaccination is effective in preventing COVID-19-related hospitalization, but duration of protection of the original monovalent vaccine during SARS-CoV-2 Omicron predominance merits evaluation, particularly given low coverage with updated COVID-19 vaccines. During December 19, 2021-October 29, 2023, the Overcoming COVID-19 Network evaluated vaccine effectiveness (VE) of ≥2 original monovalent COVID-19 mRNA vaccine doses against COVID-19-related hospitalization and critical illness among U.S. children and adolescents aged 5-18 years, using a case-control design. Too few children and adolescents received bivalent or updated monovalent vaccines to separately evaluate their effectiveness. Most case-patients (persons with a positive SARS-CoV-2 test result) were unvaccinated, despite …


Multicenter Study Of Long-Term Outcomes And Quality Of Life In Phace Syndrome After Age 10., Mitchell Braun, Ilona J. Frieden, Dawn H. Siegel, Elizabeth George, Christopher P. Hess, Christine K. Fox, Sarah L. Chamlin, Beth A. Drolet, Denise Metry, Elena Pope, Julie Powell, Kristen Holland, Caden Ulschmid, Marilyn G. Liang, Kelly K. Barry, Tina Ho, Chantal Cotter, Eulalia Baselga, David Bosquez, Surabhi Neerendranath Jain, Jordan K. Bui, Irene Lara-Corrales, Tracy Funk, Alison Small, Wenelia Baghoomian, Albert C. Yan, James R. Treat, Griffin Stockton Hogrogian, Charles Huang, Anita Haggstrom, Mary List, Catherine C. Mccuaig, Victoria Barrio, Anthony J. Mancini, Leslie P. Lawley, Kerrie Grunnet-Satcher, Kimberly A. Horii, Brandon D. Newell, Amy J. Nopper, Maria C. Garzon, Margaret E. Scollan, Erin F. Mathes Apr 2024

Multicenter Study Of Long-Term Outcomes And Quality Of Life In Phace Syndrome After Age 10., Mitchell Braun, Ilona J. Frieden, Dawn H. Siegel, Elizabeth George, Christopher P. Hess, Christine K. Fox, Sarah L. Chamlin, Beth A. Drolet, Denise Metry, Elena Pope, Julie Powell, Kristen Holland, Caden Ulschmid, Marilyn G. Liang, Kelly K. Barry, Tina Ho, Chantal Cotter, Eulalia Baselga, David Bosquez, Surabhi Neerendranath Jain, Jordan K. Bui, Irene Lara-Corrales, Tracy Funk, Alison Small, Wenelia Baghoomian, Albert C. Yan, James R. Treat, Griffin Stockton Hogrogian, Charles Huang, Anita Haggstrom, Mary List, Catherine C. Mccuaig, Victoria Barrio, Anthony J. Mancini, Leslie P. Lawley, Kerrie Grunnet-Satcher, Kimberly A. Horii, Brandon D. Newell, Amy J. Nopper, Maria C. Garzon, Margaret E. Scollan, Erin F. Mathes

Manuscripts, Articles, Book Chapters and Other Papers

OBJECTIVE: To characterize long-term outcomes of PHACE syndrome.

STUDY DESIGN: Multicenter study with cross-sectional interviews and chart review of individuals with definite PHACE syndrome ≥10 years of age. Data from charts were collected across multiple PHACE-related topics. Data not available in charts were collected from patients directly. Likert scales were used to assess the impact of specific findings. Patient-Reported Outcomes Measurement Information System (PROMIS) scales were used to assess quality of life domains.

RESULTS: A total of 104/153 (68%) individuals contacted participated in the study at a median of 14 years of age (range 10-77 years). There were infantile hemangioma …


A Meta-Analysis On The Diagnostic Utility Of Ultrasound In Pediatric Distal Forearm Fractures, Amir Hassankhani, Melika Amoukhteh, Payam Jannatdoust, Parya Valizadeh, Delaram J Ghadimi, Pauravi S Vasavada, Jennifer H Johnston, Ali Gholamrezanezhad Apr 2024

A Meta-Analysis On The Diagnostic Utility Of Ultrasound In Pediatric Distal Forearm Fractures, Amir Hassankhani, Melika Amoukhteh, Payam Jannatdoust, Parya Valizadeh, Delaram J Ghadimi, Pauravi S Vasavada, Jennifer H Johnston, Ali Gholamrezanezhad

Journal Articles

Pediatric distal forearm fractures, comprising 30% of musculoskeletal injuries in children, are conventionally diagnosed using radiography. Ultrasound has emerged as a safer diagnostic tool, eliminating ionizing radiation, enabling bedside examinations with real-time imaging, and proving effective in non-hospital settings. The objective of this study is to evaluate the diagnostic efficacy of ultrasound for detecting distal forearm fractures in the pediatric population. A systematic review and meta-analysis were conducted through a comprehensive literature search in PubMed, Scopus, Web of Science, and Embase databases until October 1, 2023, following established guidelines. Eligible studies, reporting diagnostic accuracy measures of ultrasound in pediatric patients …


Accuracy Of Influenza Icd-10 Diagnosis Codes In Identifying Influenza Illness In Children., James W. Antoon, Tess Stopczynski, Justin Z. Amarin, Laura S. Stewart, Julie A. Boom, Leila C. Sahni, Marian G. Michaels, John V. Williams, Janet A. Englund, Eileen J. Klein, Mary A. Staat, Elizabeth P. Schlaudecker, Rangaraj Selvarangan, Jennifer E. Schuster, Geoffrey A. Weinberg, Peter G. Szilagyi, Ariana Perez, Heidi L. Moline, Andrew J. Spieker, Carlos G. Grijalva, Samantha M. Olson, Natasha B. Halasa Apr 2024

Accuracy Of Influenza Icd-10 Diagnosis Codes In Identifying Influenza Illness In Children., James W. Antoon, Tess Stopczynski, Justin Z. Amarin, Laura S. Stewart, Julie A. Boom, Leila C. Sahni, Marian G. Michaels, John V. Williams, Janet A. Englund, Eileen J. Klein, Mary A. Staat, Elizabeth P. Schlaudecker, Rangaraj Selvarangan, Jennifer E. Schuster, Geoffrey A. Weinberg, Peter G. Szilagyi, Ariana Perez, Heidi L. Moline, Andrew J. Spieker, Carlos G. Grijalva, Samantha M. Olson, Natasha B. Halasa

Manuscripts, Articles, Book Chapters and Other Papers

IMPORTANCE: Studies of influenza in children commonly rely on coded diagnoses, yet the ability of International Classification of Diseases, Ninth Revision codes to identify influenza in the emergency department (ED) and hospital is highly variable. The accuracy of newer International Statistical Classification of Diseases and Related Health Problems, Tenth Revision (ICD-10) codes to identify influenza in children is unknown.

OBJECTIVE: To determine the accuracy of ICD-10 influenza discharge diagnosis codes in the pediatric ED and inpatient settings.

DESIGN, SETTING, AND PARTICIPANTS: Children younger than 18 years presenting to the ED or inpatient settings with fever and/or respiratory symptoms at 7 …


Self-Regulation And Sensory Processing Education Program For Educators, Julianne Mendivil, Tina G. Bhakta, Angela Blackwell Apr 2024

Self-Regulation And Sensory Processing Education Program For Educators, Julianne Mendivil, Tina G. Bhakta, Angela Blackwell

Spring 2024 Virtual OTD Capstone Symposium

Children with low self-regulation skills may face many challenges in the classroom environment that can impact their performance in school-based activities. As teachers play a vital role in the development of young children, this educational program aims to improve teacher competence (knowledge, skills, and attitudes) in applying sensory processing and self-regulating concepts to the school environment.


Influence Of Novel Cyp2c-Haplotype On Proton Pump Inhibitor Pharmacokinetics In Children., Kathyrn Kyler, Andrea Gaedigk, Susan Abdel-Rahman, Vincent S. Staggs, Robin E. Pearce, Paul C. Toren, J Steven Leeder, Valentina Shakhnovich Apr 2024

Influence Of Novel Cyp2c-Haplotype On Proton Pump Inhibitor Pharmacokinetics In Children., Kathyrn Kyler, Andrea Gaedigk, Susan Abdel-Rahman, Vincent S. Staggs, Robin E. Pearce, Paul C. Toren, J Steven Leeder, Valentina Shakhnovich

Manuscripts, Articles, Book Chapters and Other Papers

In this brief report, we provide an analysis of the influence of a novel CYP2C haplotype (CYP2C:TG) on proton pump inhibitor (PPI) pharmacokinetics (PK) in children. The CYP2C:TG haplotype has been proposed to be associated with increased CYP2C19 activity. We sought to determine if this CYP2C:TG haplotype resulted in similar alterations in metabolism for proton pump inhibitors, which are primarily metabolized by CYP2C19. In a cohort of 41 children aged 6-21 participating in a PPI pharmacokinetic study, effects of the CYP2C:TG allele were assessed by fitting two linear regression models for each of the six PK outcomes assessed, the second …


Delayed Onset Of Neonatal Compartment Syndrome Associated With Compound Fetal Presentation, Nicholas Manini, Hayato Unno Apr 2024

Delayed Onset Of Neonatal Compartment Syndrome Associated With Compound Fetal Presentation, Nicholas Manini, Hayato Unno

Department of Medicine Faculty Papers

Neonatal compartment syndrome, although rare, has a classic presentation with sentinel skin findings and development of swelling, erythema, and tenderness of the affected extremity. Neonatal compartment syndrome requires prompt surgical intervention to preserve the affected limb and ensure its normal growth and development. Our patient was born at term via vaginal delivery complicated by a compound presentation involving the left upper extremity. No physical exam abnormalities were noted at birth, but she developed signs of neonatal compartment syndrome by 15 h of life. She was surgically treated at 22 h of life and recovered well. At one year of age, …


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

Manuscripts, Articles, Book Chapters and Other Papers

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 …


Pediatric Beta Blocker Therapy: A Comprehensive Review Of Development And Genetic Variation To Guide Precision-Based Therapy In Children, Adolescents, And Young Adults., Mollie Walton, Jonathan B. Wagner Mar 2024

Pediatric Beta Blocker Therapy: A Comprehensive Review Of Development And Genetic Variation To Guide Precision-Based Therapy In Children, Adolescents, And Young Adults., Mollie Walton, Jonathan B. Wagner

Manuscripts, Articles, Book Chapters and Other Papers

Beta adrenergic receptor antagonists, known as beta blockers, are one of the most prescribed medications in both pediatric and adult cardiology. Unfortunately, most of these agents utilized in the pediatric clinical setting are prescribed off-label. Despite regulatory efforts aimed at increasing pediatric drug labeling, a majority of pediatric cardiovascular drug agents continue to lack pediatric-specific data to inform precision dosing for children, adolescents, and young adults. Adding to this complexity is the contribution of development (ontogeny) and genetic variation towards the variability in drug disposition and response. In the absence of current prospective trials, the purpose of this comprehensive review …


Nf-Κb As An Inducible Regulator Of Inflammation In The Central Nervous System, Sudha Anilkumar, Elizabeth Wright-Jin Mar 2024

Nf-Κb As An Inducible Regulator Of Inflammation In The Central Nervous System, Sudha Anilkumar, Elizabeth Wright-Jin

Department of Medicine Faculty Papers

The NF-κB (nuclear factor K-light-chain-enhancer of activated B cells) transcription factor family is critical for modulating the immune proinflammatory response throughout the body. During the resting state, inactive NF-κB is sequestered by IκB in the cytoplasm. The proteasomal degradation of IκB activates NF-κB, mediating its translocation into the nucleus to act as a nuclear transcription factor in the upregulation of proinflammatory genes. Stimuli that initiate NF-κB activation are diverse but are canonically attributed to proinflammatory cytokines and chemokines. Downstream effects of NF-κB are cell type-specific and, in the majority of cases, result in the activation of pro-inflammatory cascades. Acting as …


Real-World Evidence On The Dosing And Safety Of C.E.R.A. In Pediatric Dialysis Patients: Findings From The International Pediatric Dialysis Network Registries., Laura Kohlhas, Milena Studer, Loes Rutten-Jacobs, Sylvie Meyer Reigner, Anja Sander, Hui-Kim Yap, Karel Vondrak, Paula A. Coccia, Francisco Cano, Claus Peter Schmitt, Bradley A. Warady, Franz Schaefer, Ipdn Collaborators Mar 2024

Real-World Evidence On The Dosing And Safety Of C.E.R.A. In Pediatric Dialysis Patients: Findings From The International Pediatric Dialysis Network Registries., Laura Kohlhas, Milena Studer, Loes Rutten-Jacobs, Sylvie Meyer Reigner, Anja Sander, Hui-Kim Yap, Karel Vondrak, Paula A. Coccia, Francisco Cano, Claus Peter Schmitt, Bradley A. Warady, Franz Schaefer, Ipdn Collaborators

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: This retrospective real-world study used data from two registries, International Pediatric Peritoneal Dialysis Network (IPPN) and International Pediatric Hemodialysis Network (IPHN), to characterize the efficacy and safety of continuous erythropoietin receptor activator (C.E.R.A.) in pediatric patients with chronic kidney disease (CKD) on peritoneal dialysis (PD) or hemodialysis (HD).

METHODS: IPPN and IPHN collect prospective data (baseline and every 6 months) from pediatric PD and HD centers worldwide. Demographics, clinical characteristics, dialysis information, treatment, laboratory parameters, number and causes of hospitalization events, and deaths were extracted for patients on C.E.R.A. treatment (IPPN: 2007-2021; IPHN: 2013-2021).

RESULTS: We analyzed 177 patients …


Associations Between Anemia And Fgf23 In The Ckid Study., Elizabeth Thomas, Alexandra M. Klomhaus, Marciana L. Laster, Susan L. Furth, Bradley A. Warady, Isidro B. Salusky, Mark R. Hanudel Mar 2024

Associations Between Anemia And Fgf23 In The Ckid Study., Elizabeth Thomas, Alexandra M. Klomhaus, Marciana L. Laster, Susan L. Furth, Bradley A. Warady, Isidro B. Salusky, Mark R. Hanudel

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Fibroblast growth factor 23 (FGF23) is a bone-derived hormone that plays a central role in chronic kidney disease-mineral bone disorder and is associated with CKD progression and cardiovascular morbidity. Factors related to CKD-associated anemia, including iron deficiency, can increase FGF23 production. This study aimed to assess whether anemia and/or iron deficiency are associated with increased circulating concentrations of FGF23 in the large, well-characterized Chronic Kidney Disease in Children (CKiD) study cohort.

METHODS: Hemoglobin concentrations, iron parameters, C-terminal (total) FGF23, intact FGF23, and relevant covariables were measured in cross-sectional analysis of CKiD study subjects.

RESULTS: In 493 pediatric patients with …


Racial And Ethnic Disparities In The Co-Occurrence Of Intellectual Disability And Autism: Impact Of Incorporating Measures Of Adaptive Functioning., Sarah M. Furnier, Ronald Gangnon, Julie L. Daniels, Susan Ellis Weismer, Cy Nadler, Karen Pazol, Nuri M. Reyes, Steven Rosenberg, Eric Rubenstein, Lisa D. Wiggins, Marshalyn Yeargin-Allsopp, Maureen S. Durkin Mar 2024

Racial And Ethnic Disparities In The Co-Occurrence Of Intellectual Disability And Autism: Impact Of Incorporating Measures Of Adaptive Functioning., Sarah M. Furnier, Ronald Gangnon, Julie L. Daniels, Susan Ellis Weismer, Cy Nadler, Karen Pazol, Nuri M. Reyes, Steven Rosenberg, Eric Rubenstein, Lisa D. Wiggins, Marshalyn Yeargin-Allsopp, Maureen S. Durkin

Manuscripts, Articles, Book Chapters and Other Papers

Intellectual disability (ID) commonly co-occurs in children with autism. Although diagnostic criteria for ID require impairments in both cognitive and adaptive functioning, most population-based estimates of the frequency of co-occurring ID in children with autism-including studies of racial and ethnic disparities in co-occurring autism and ID-base the definition of ID solely on cognitive scores. The goal of this analysis was to examine the effect of including both cognitive and adaptive behavior criteria on estimates of co-occurring ID in a well-characterized sample of 2- to 5-year-old children with autism. Participants included 3264 children with research or community diagnoses of autism enrolled …


Epigenome-Wide Association Study Identifies Neonatal Dna Methylation Associated With Two-Year Attention Problems In Children Born Very Preterm., Marie Camerota, Barry M. Lester, Francisco Xavier Castellanos, Brian S. Carter, Jennifer Check, Jennifer Helderman, Julie A. Hofheimer, Elisabeth C. Mcgowan, Charles R. Neal, Steven L. Pastyrnak, Lynne M. Smith, Thomas Michael O'Shea, Carmen J. Marsit, Todd M. Everson Feb 2024

Epigenome-Wide Association Study Identifies Neonatal Dna Methylation Associated With Two-Year Attention Problems In Children Born Very Preterm., Marie Camerota, Barry M. Lester, Francisco Xavier Castellanos, Brian S. Carter, Jennifer Check, Jennifer Helderman, Julie A. Hofheimer, Elisabeth C. Mcgowan, Charles R. Neal, Steven L. Pastyrnak, Lynne M. Smith, Thomas Michael O'Shea, Carmen J. Marsit, Todd M. Everson

Manuscripts, Articles, Book Chapters and Other Papers

Prior research has identified epigenetic predictors of attention problems in school-aged children but has not yet investigated these in young children, or children at elevated risk of attention problems due to preterm birth. The current study evaluated epigenome-wide associations between neonatal DNA methylation and attention problems at age 2 years in children born very preterm. Participants included 441 children from the Neonatal Neurobehavior and Outcomes in Very Preterm Infants (NOVI) Study, a multi-site study of infants born < 30 weeks gestational age. DNA methylation was measured from buccal swabs collected at NICU discharge using the Illumina MethylationEPIC Bead Array. Attention problems were assessed at 2 years of adjusted age using the attention problems subscale of the Child Behavior Checklist (CBCL). After adjustment for multiple testing, DNA methylation at 33 CpG sites was associated with child attention problems. Differentially methylated CpG sites were located in genes previously linked to physical and mental health, including several genes associated with ADHD in prior epigenome-wide and genome-wide association studies. Several CpG sites were located in genes previously linked to exposure to prenatal risk factors in the NOVI sample. Neonatal epigenetics measured at NICU discharge could be useful in identifying preterm children at risk for long-term attention problems and related psychiatric disorders, who could benefit from early prevention and intervention efforts.


Notes From The Field: Reemergence Of Mycoplasma Pneumoniae Infections In Children And Adolescents After The Covid-19 Pandemic, United States, 2018-2024., Chris Edens, Benjamin R. Clopper, Jourdan Devies, Alvaro Benitez, Erin R. Mckeever, Dylan Johns, Bernard Wolff, Rangaraj Selvarangan, Jennifer E. Schuster, Geoffrey A. Weinberg, Peter G. Szilagyi, Fatimah S. Dawood, Lakshmi Radhakrishnan, Christina Quigley, Leila C. Sahni, Natasha Halasa, Laura S. Stewart, Meredith L. Mcmorrow, Brett Whitaker, Danielle M. Zerr, Vasanthi Avadhanula, John V. Williams, Marian G. Michaels, Aaron Kite-Powell, Janet A. Englund, Mary Allen Staat, Kathleen Hartnett, Heidi L. Moline, Adam L. Cohen, Maureen Diaz Feb 2024

Notes From The Field: Reemergence Of Mycoplasma Pneumoniae Infections In Children And Adolescents After The Covid-19 Pandemic, United States, 2018-2024., Chris Edens, Benjamin R. Clopper, Jourdan Devies, Alvaro Benitez, Erin R. Mckeever, Dylan Johns, Bernard Wolff, Rangaraj Selvarangan, Jennifer E. Schuster, Geoffrey A. Weinberg, Peter G. Szilagyi, Fatimah S. Dawood, Lakshmi Radhakrishnan, Christina Quigley, Leila C. Sahni, Natasha Halasa, Laura S. Stewart, Meredith L. Mcmorrow, Brett Whitaker, Danielle M. Zerr, Vasanthi Avadhanula, John V. Williams, Marian G. Michaels, Aaron Kite-Powell, Janet A. Englund, Mary Allen Staat, Kathleen Hartnett, Heidi L. Moline, Adam L. Cohen, Maureen Diaz

Manuscripts, Articles, Book Chapters and Other Papers

No abstract provided.


Current Strategies For Increasing Knock-In Efficiency In Crispr/Cas9-Based Approaches, Andrés Felipe Leal, Angelica María Herreno-Pachón, Eliana Benincore-Flórez, Amali Karunathilaka, Shunji Tomatsu Feb 2024

Current Strategies For Increasing Knock-In Efficiency In Crispr/Cas9-Based Approaches, Andrés Felipe Leal, Angelica María Herreno-Pachón, Eliana Benincore-Flórez, Amali Karunathilaka, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Since its discovery in 2012, the clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein 9 (Cas9) system has supposed a promising panorama for developing novel and highly precise genome editing-based gene therapy (GT) alternatives, leading to overcoming the challenges associated with classical GT. Classical GT aims to deliver transgenes to the cells via their random integration in the genome or episomal persistence into the nucleus through lentivirus (LV) or adeno-associated virus (AAV), respectively. Although high transgene expression efficiency is achieved by using either LV or AAV, their nature can result in severe side effects in humans. For instance, …