Medicine and Health Sciences Commons^™

Measurable Residual Disease And Fusion Partner Independently Predict Survival And Relapse Risk In Childhood Kmt2a-Rearranged Acute Myeloid Leukemia: A Study By The International Berlin-Frankfurt-Münster Study Group, Romy E. Van Weelderen, Kim Klein, Christine J. Harrison, Yilin Jiang, Jonas Abrahamsson, Nira Arad-Cohen, Emmanuelle Bart-Delabesse, Barbara Buldini, Barbara De Moerloose, Michael N. Dworzak, Sarah Elitzur, José M. Fernández Navarro, Robert B. Gerbing, Bianca F. Goemans, Hester A. De Groot-Kruseman, Erin M. Guest, Shau-Yin Ha, Henrik Hasle, Charikleia Kelaidi, Hélène Lapillonne, Guy Leverger, Franco Locatelli, Riccardo Masetti, Takako Miyamura, Ulrika Norén-Nyström, Sophia Polychronopoulou, Mareike Rasche, Jeffrey E. Rubnitz, Jan Stary, Anne Tierens, Daisuke Tomizawa, C Michel Zwaan, Gertjan J L Kaspers

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Purpose: A previous study by the International Berlin-Frankfurt-Münster Study Group (I-BFM-SG) on childhood KMT2A-rearranged (KMT2A-r) AML demonstrated the prognostic value of the fusion partner. This I-BFM-SG study investigated the value of flow cytometry-based measurable residual disease (flow-MRD) and evaluated the benefit of allogeneic stem-cell transplantation (allo-SCT) in first complete remission (CR1) in this disease.

Methods: A total of 1,130 children with KMT2A-r AML, diagnosed between January 2005 and December 2016, were assigned to high-risk (n = 402; 35.6%) or non-high-risk (n = 728; 64.4%) fusion partner-based groups. Flow-MRD levels at both end of induction 1 (EOI1) …

Long Noncoding Rna Expression Independently Predicts Outcome In Pediatric Acute Myeloid Leukemia., Jason E. Farrar, Jenny L. Smith, Megan Othus, Benjamin J. Huang, Yi-Cheng Wang, Rhonda Ries, Tiffany Hylkema, Era L. Pogosova-Agadjanyan, Sneha Challa, Amanda Leonti, Timothy I. Shaw, Timothy J. Triche, Alan S. Gamis, Richard Aplenc, E Anders Kolb, Xiaotu Ma, Derek L. Stirewalt, Todd A. Alonzo, Soheil Meshinchi

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Purpose: Optimized strategies for risk classification are essential to tailor therapy for patients with biologically distinctive disease. Risk classification in pediatric acute myeloid leukemia (pAML) relies on detection of translocations and gene mutations. Long noncoding RNA (lncRNA) transcripts have been shown to associate with and mediate malignant phenotypes in acute myeloid leukemia (AML) but have not been comprehensively evaluated in pAML.

Methods: To identify lncRNA transcripts associated with outcomes, we evaluated the annotated lncRNA landscape by transcript sequencing of 1,298 pediatric and 96 adult AML specimens. Upregulated lncRNAs identified in the pAML training set were used to establish a regularized …

Irf7 And Unc93b1 Variants In An Infant With Recurrent Herpes Simplex Virus Infection., Megan H. Tucker, Wei Yu, Heather Menden, Sheng Xia, Carl F. Schreck, Margaret Gibson, Daniel A. Louiselle, T Pastinen, Nikita Raje, Venkatesh Sampath

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Neonatal herpes simplex virus (HSV) infection is a devastating disease with substantial morbidity and mortality. The genetic basis of susceptibility to HSV in neonates remains undefined. We evaluated a male infant with neonatal skin/eye/mouth (SEM) HSV-1 disease, who had complete recovery after acyclovir but developed HSV-1 encephalitis at 1 year of age. An immune workup showed an anergic PBMC cytokine response to TLR3 stimulation but no other TLRs. Exome sequencing identified rare missense variants in IFN-regulatory factor 7 (IRF7) and UNC-93 homolog B1 (UNC93B1). PBMC single-cell RNA-Seq done during childhood revealed decreased expression of several innate immune genes and a …

Recruitment And Reach In A School-Based Pediatric Obesity Intervention Trial In Rural Areas., Bethany Forseth, Brittany Lancaster, Megan Olalde, Christie A. Befort, Rebecca E. Swinburne Romine, Meredith Dreyer, Kelsey Dean, Eve-Lynn Nelson, Ann M. Davis

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INTRODUCTION: The purpose of this study is to evaluate two recruitment strategies on schools and participant participation rates and representativeness (reach) within a pediatric obesity treatment trial tailored for families who live in rural areas.

METHODS: Recruitment of schools was evaluated based on their progress toward enrolling participants. Recruitment and reach of participants were evaluated using (1) participation rates and (2) representativeness of demographics and weight status of participants compared to eligible participants (who did not consent and enroll) and all students (regardless of eligibility). School recruitment, as well as participant recruitment and reach, were evaluated across recruitment methods comparing …

X-Linked Variations In Shroom4 Are Implicated In Congenital Anomalies Of The Urinary Tract And The Anorectal, Cardiovascular And Central Nervous Systems, Caroline M. Kolvenbach, Tim Felger, Luca Schierbaum, Isabelle Thiffault, T Pastinen, Maria Szczepańska, Marcin Zaniew, Piotr Adamczyk, Allan Bayat, Öznur Yilmaz, Tobias T. Lindenberg, Holger Thiele, Friedhelm Hildebrandt, Katrin Hinderhofer, Ute Moog, Alina C Hilger, Bonnie Sullivan, Lauren E. Bartik, Piotr Gnyś, Phillip Grote, Benjamin Odermatt, Heiko M. Reutter, Gabriel C. Dworschak

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Background: SHROOM4 is thought to play an important role in cytoskeletal modification and development of the early nervous system. Previously, single-nucleotide variants (SNVs) or copy number variations (CNVs) in SHROOM4 have been associated with the neurodevelopmental disorder Stocco dos Santos syndrome, but not with congenital anomalies of the urinary tract and the visceral or the cardiovascular system.

Methods: Here, exome sequencing and CNV analyses besides expression studies in zebrafish and mouse and knockdown (KD) experiments using a splice blocking morpholino in zebrafish were performed to study the role of SHROOM4 during embryonic development.

Results: In this study, we identified putative …

Ehealth Technology In Cardiac Exercise Therapeutics For Pediatric Patients With Congenital And Acquired Heart Conditions: A Summary Of Evidence And Future Directions., David A. White, Aimee M. Layton, Tracy Curran, Naomi Gauthier, William B. Orr, Kendra Ward, Meg Vernon, Matthew N. Martinez, Malloree C. Rice, Katherine Hansen, Megan Prusi, Jesse E. Hansen

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Many children and adolescents with congenital and acquired heart disease (CHD) are physically inactive and participate in an insufficient amount of moderate-to-vigorous intensity exercise. Although physical activity (PA) and exercise interventions are effective at improving short- and long-term physiological and psychosocial outcomes in youth with CHD, several barriers including resource limitations, financial costs, and knowledge inhibit widespread implementation and dissemination of these beneficial programs. New and developing eHealth, mHealth, and remote monitoring technologies offer a potentially transformative and cost-effective solution to increase access to PA and exercise programs for youth with CHD, yet little has been written on this topic. …

Climate Change Imperils Pediatric Health: Child Advocacy Through Fossil Fuel Divestment., Sandra H. Jee, Elizabeth Friedman, Ruth A. Etzel, Vi T. Nguyen, Todd L. Sack, Kathi J. Kemper

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Climate change poses an existential threat to children's health. Divestment of ownership stakes in fossil fuel companies is one tool available to pediatricians to address climate change. Pediatricians are trusted messengers regarding children's health and therefore bear a unique responsibility to advocate for climate and health policies that affect children. Among the impacts of climate change on pediatric patients are allergic rhinitis and asthma; heat-related illnesses; premature birth; injuries from severe storms and fires; vector-borne diseases; and mental illnesses. Children are disproportionately affected as well by climate-related displacement of populations, drought, water shortages, and famine. The human-generated burning of fossil …

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 …

Is It Too Complex? A Survey Of Pediatric Residency Program's Educational Approach For The Care Of Children With Medical Complexity., Kira Sieplinga, Christopher Kruger, Emily J. Goodwin

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BACKGROUND: Although Entrustable Professional Activities (EPAs) regarding pediatric training in care for children with medical complexity (CMC) exist, it is unknown what US pediatric training programs provide for education related to care of CMC and whether educators perceive that pediatric residents are prepared to care for CMC upon graduation.

METHODS: From June, 2021 through March 2022, we surveyed US pediatric residency program delegates about practice settings, current educational offerings, perception of resident preparedness regarding care of CMC, and likelihood to implement CMC education in the future.

RESULTS: Response rate was 29% (56 /195). A third of responding programs (34%, n …

State Of The Art Bowel Management For Pediatric Colorectal Problems: Anorectal Malformations., Elizaveta Bokova, Wendy Jo Svetanoff, Joseph Lopez, Marc A. Levitt, Rebecca M. Rentea

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Up to 79% of patients with anorectal malformations (ARMs) experience constipation and/or soiling after a primary posterior sagittal anoplasty (PSARP) and are referred to a bowel management program. We aim to report the recent updates in evaluating and managing these patients as part of the manuscript series on the current bowel management protocols for patients with colorectal diseases (ARMs, Hirschsprung disease, functional constipation, and spinal anomalies). The unique anatomic features of ARM patients, such as maldeveloped sphincter complex, impaired anal sensation, and associated spine and sacrum anomalies, indicate their bowel management plan. The evaluation includes an examination under anesthesia and …

Real-World Experience Of Pediatric Patients Treated With Peanut (Arachis Hypogaea) Allergen Powder-Dnfp., Jay M. Portnoy, Jodi Shroba, Stephen Tilles, Hela Romdhani, Sarah M. Donelson, Dominick Latremouille-Viau, Rebecca Bungay, Kathleen Chen, William Mccann

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BACKGROUND: Peanut (Arachis hypogaea) allergen powder-dnfp (PTAH) is the first oral immunotherapy indicated for children aged 4 to 17 years with peanut allergy. There are limited real-world data on patients treated with PTAH.

OBJECTIVE: To characterize pediatric patients treated with PTAH and associated treatment patterns in US clinical practice.

METHODS: US-based physicians with allergy and immunology training treating patients with peanut allergy aged 4 to 17 years with PTAH were recruited from an existing physician panel and completed an online case report form (October to December 2021) with data abstracted from patient medical charts. Physician practice circumstances, patient characteristics, and …

Rationale And Design Of Integrating A Parents First Obesity Intervention With A Pediatric Weight Management Intervention For Rural Families - Evaluating The Ripple Effect., Ann M. Davis, Christie A. Befort, Brittany D. Lancaster, Chris Tuck, Barbara J. Polivka, Jordan A. Carlson, Kandace Fleming, Rebecca Swinburne Romine, Kelsey Dean, Megan Murray

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Rural families are disproportionately affected by obesity. Obesity often runs in families and is impacted by hereditary components, the shared home environment, and parent modeling/child observational learning. Moreover, parent changes in weight predict child changes in weight. Thus, targeting the family unit has the potential to enhance outcomes for adults and children simultaneously. Additionally, engaging rural nurses in medical clinics and schools may be important in determining whether rural telehealth programs are successfully implemented and sustained. This paper describes the rationale and design of a randomized control trial (RCT) evaluating the effectiveness of an integrated adult- and child-focused obesity treatment …

Electronic Monitoring Of Mom's Schedule (Emomstm): Recruitment Of Pregnant Populations With Elevated Bmi In A Feasibility Randomized Controlled Trial, Lisette T. Jacobson, Michael Wolfe, Rosey Zackula, Hayrettin Okut, Faith E. Hampton, David A. Grainger, Adrianne K. Griebel-Thompson, Kai Ling Kong, Christie Befort

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Underrepresentation of pregnant populations in randomized controlled trials of lifestyle change interventions is concerning due to high attrition and providers' limited clinical time. The purpose of this evaluative study was to assess intervention uptake of pregnant individuals enrolled in a three-arm feasibility randomized controlled trial, electronic Monitoring Of Mom's Schedule (eMOMSTM), examining lifestyle changes and lactation support alone, and in combination. Measures included: (1) participation and completion rates, and characteristics of intervention completers versus other eligible participants; and (2) provider experiences with screening and enrolling pregnant participants. Pregnant people with a pre-pregnancy body mass index ≥ 25 and < 35 kg/m2 were enrolled into the eMOMSTM trial between September 2019 - December 2020. Of the 44 consented participants, 35 were randomized, at a participation rate of 35%, and 26 completed the intervention, resulting in a completion rate of 74%. Intervention completers were slightly older and entered the study earlier in pregnancy compared to non-completers. Completers were more likely to be first-time mothers, resided in urban areas, had higher educational attainment, and were slightly more racially and ethnically diverse. A majority of providers reported willingness to participate, believed the study aligned with their organization's mission, and were satisfied with using iPads for screening. Lessons learned to guide recruitment success include use of: (1) designated research staff in combination with physician support; and (2) user-friendly technology to help mitigate time burden on physicians and their staff. Future work should focus on successful strategies to recruit/retain pregnant populations in clinical trials.

Use, Durability, And Risks For Discontinuation Of Initial And Subsequent Biologics In A Large Pediatric-Onset Ibd Cohort., Jess L. Kaplan, Chunyan Liu, Eileen C. King, Julie A. Bass, Ashish S. Patel, Jeanne Tung, Shiran Chen, Trevor Lissoos, Ninfa Candela, Shehzad Saeed, Richard B. Colletti, Improvecarenow Network

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BACKGROUND: Biologic medications are recommended for treatment of moderately-to-severely active Crohn disease (CD) or ulcerative colitis (UC) in children. However, many patients require sequential biologic treatment because of nonresponse or loss of response to the initial biologic.

METHODS: We analyzed pediatric inflammatory bowel disease (IBD) data from the ImproveCareNow Network registry between May 2006 and September 2016, including time to biologic initiation, choice of first subsequent biologics, biologic durability, and reasons for discontinuation.

RESULTS: Of 17,649 patients with IBD [CD: 12,410 (70%); UC: 5239 (30%)], 7585 (43%) were treated with a biologic agent before age 18 (CD: 50%; UC: 25%). …

Trends In The Representation Of Women In The American Society Of Pediatric Nephrology Program At The Pediatric Academic Society Annual Meetings 2012-2021., Bahar Barani Najafabadi, David T. Selewski, Danielle E. Soranno, Darcy K. Weidemann

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INTRODUCTION: Women are under-represented in virtually all fields of academic medicine. Even in pediatrics, a field that historically attracts a workforce with a majority of women physicians, substantial gender disparities persist in leadership positions. However, previous studies of gender representation in various academic settings are limited to small studies or aggregate pediatric subspecialties, thereby omitting important granularity within each subspecialty. No prior studies have investigated potential gender disparities in pediatric nephrology. The aim of this study is to determine the representation of women physicians in leadership and speaking roles in the annual American Society of Pediatric Nephrology (ASPN) meeting.

METHODS: …

Neurocomputational Mechanisms Of Food And Physical Activity Decision-Making In Male Adolescents., Seung-Lark Lim, Amanda S. Bruce, Robin P. Shook

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We examined the neurocomputational mechanisms in which male adolescents make food and physical activity decisions and how those processes are influenced by body weight and physical activity levels. After physical activity and dietary assessments, thirty-eight males ages 14-18 completed the behavioral rating and fMRI decision tasks for food and physical activity items. The food and physical activity self-control decisions were significantly correlated with each other. In both, taste- or enjoyment-oriented processes were negatively associated with successful self-control decisions, while health-oriented processes were positively associated. The correlation between taste/enjoyment and healthy attribute ratings predicted actual laboratory food intake and physical activities …

Identification Of Small Extracellular Vesicle Protein Biomarkers For Pediatric Ewing Sarcoma., Soumya M. Turaga, Mihaela E. Sardiu, Vikalp Vishwakarma, Amrita Mitra, Leonidas E. Bantis, Rashna Madan, Michael L. Merchant, Jon B. Klein, Glenson Samuel, Andrew K. Godwin

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Ewing Sarcoma (EWS) is the second most common osseous malignancy in children and young adults after osteosarcoma, while it is the fifth common osseous malignancy within adult age population. The clinical presentation of EWS is quite often non-specific, with the most common symptoms at presentation consisting of pain, swelling or general discomfort. The dearth of clinically relevant diagnostic or predictive biomarkers continues to remain a pressing clinical challenge. Identification of tumor specific biomarkers can lend towards an early diagnosis, expedited initiation of therapy, monitoring of therapeutic response, and early detection of recurrence of disease. We carried-out a complex analysis of …

Perinucleolar Compartment (Pnc) Prevalence As An Independent Prognostic Factor In Pediatric Ewing Sarcoma: A Multi-Institutional Study., Elizabeth Gonzalez, Atif A. Ahmed, Laura Mccarthy, Katherine Chastain, Sahibu Sultan M Habeebu, Marta Zapata-Tarres, Rocio Cardenas-Cardos, Liliana Velasco-Hidalgo, Celso Corcuera-Delgado, Rodolfo Rodriguez-Jurado, Lilia García-Rodríguez, Alejandro Parrales, Tomoo Iwakuma, Midhat S. Farooqi, Brian R. Lee, Scott J. Weir, Terrie Flatt

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The perinucleolar compartment (PNC) is a small nuclear body that plays important role in tumorigenesis. PNC prevalence correlates with poor prognosis and cancer metastasis. Its expression in pediatric Ewing sarcoma (EWS) has not previously been documented. In this study, we analyzed 40 EWS tumor cases from Caucasian and Hispanic patients for PNC prevalence by immunohistochemical detection of polypyrimidine tract binding protein and correlated the prevalence with dysregulated microRNA profiles. EWS cases showed staining ranging from 0 to 100%, which were categorized as diffuse (≥77%, n = 9, high PNC) or not diffuse (p= 0.017) and in patients who relapsed with …

Multicentre Study Protocol Comparing Standard Nrp To Developed Educational Modules For Resuscitation Of Neonates In The Delivery Room With Congenital Heart Disease (Learn-Chd)., Philip Levy, Alyssa R. Thomas, Brenda Hiu Yan Law, Chloe Joynt, Ruby Gupta, Summer Elshenawy, Danielle Reed, Leeann R. Pavlek, Jennifer Shepherd, Sharada Gowda, Beth Ann Johnson, Molly Ball, Noorjahan Ali

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INTRODUCTION: Infants born with critical congenital heart defects (CCHDs) have unique transitional pathophysiology that often requires special resuscitation and management considerations in the delivery room (DR). While much is known about neonatal resuscitation of infants with CCHDs, current neonatal resuscitation guidelines such as the neonatal resuscitation programme (NRP) do not include algorithm modifications or education specific to CCHDs. The implementation of CCHD specific neonatal resuscitation education is further hampered by the large number of healthcare providers (HCPs) that need to be reached. Online learning modules (eLearning) may provide a solution but have not been designed or tested for this specific …

Assessment Of Psychosocial And Neonatal Risk Factors For Trajectories Of Behavioral Dysregulation Among Young Children From 18 To 72 Months Of Age., Julie A. Hofheimer, Monica Mcgrath, Rashelle Musci, Guojing Wu, Sarah Polk, Courtney K. Blackwell, Annemarie Stroustrup, Robert D. Annett, Judy Aschner, Brian S. Carter, Jennifer Check, Elisabeth Conradt, Lisa A. Croen, Anne L. Dunlop, Amy J. Elliott, Andrew Law, Leslie D. Leve, Jenae M. Neiderhiser, T Michael O'Shea, Amy L. Salisbury, Sheela Sathyanarayana, Rachana Singh, Lynne M. Smith, Andréa Aguiar, Jyoti Angal, Hannah Carliner, Cindy Mcevoy, Steven J. Ondersma, Barry Lester, Program Collaborators For Environmental Influences On Child Health Outcomes

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IMPORTANCE: Emotional and behavioral dysregulation during early childhood are associated with severe psychiatric, behavioral, and cognitive disorders through adulthood. Identifying the earliest antecedents of persisting emotional and behavioral dysregulation can inform risk detection practices and targeted interventions to promote adaptive developmental trajectories among at-risk children.

OBJECTIVE: To characterize children's emotional and behavioral regulation trajectories and examine risk factors associated with persisting dysregulation across early childhood.

DESIGN, SETTING, AND PARTICIPANTS: This cohort study examined data from 20 United States cohorts participating in Environmental influences on Child Health Outcomes, which included 3934 mother-child pairs (singleton births) from 1990 to 2019. Statistical analysis …

Examining The Relationship Between Nighttime Glucose Values In Youth With Type 1 Diabetes And Parent Fear Of Nighttime Hypoglycemia, Alexandra D. Monzon, Ryan Mcdonough, Christopher C. Cushing, Mark A. Clements, Susana R. Patton

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Objective. Youth with type 1 diabetes mellitus (T1D) are at risk for experiencing nighttime hypoglycemia, and many parents report significant anxiety at night regarding glucose management. Limited data exist examining continuous nighttime glucose levels as a predictor of parent fear of nighttime hypoglycemia. The present study aimed to examine the relationship between parent fear of nighttime hypoglycemia and nighttime blood glucose levels as measured by continuous glucose monitors (CGMs). Methods. A sample of 136 parents/caregivers of youth with T1D completed a one-time survey and youth provided 14 days of CGM data. We conducted regression models with mean nighttime …

Necrotizing Enterocolitis In Premature Infants-A Defect In The Brakes? Evidence From Clinical And Animal Studies., Venkatesh Sampath, Maribel Martinez, Michael Caplan, Mark A. Underwood, Alain Cuna

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A key aspect of postnatal intestinal adaptation is the establishment of symbiotic relationships with co-evolved gut microbiota. Necrotizing enterocolitis (NEC) is the most severe disease arising from failure in postnatal gut adaptation in premature infants. Although pathological activation of intestinal Toll-like receptors (TLRs) is believed to underpin NEC pathogenesis, the mechanisms are incompletely understood. We postulate that unregulated aberrant TLR activation in NEC arises from a failure in intestinal-specific mechanisms that tamponade TLR signaling (the brakes). In this review, we discussed the human and animal studies that elucidate the developmental mechanisms inhibiting TLR signaling in the postnatal intestine (establishing the …

Pediatric Bowel Management Options And Organizational Aspects., Elizaveta Bokova, Wendy Jo Svetanoff, Marc Aaron Levitt, Rebecca M. Rentea

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A bowel management program (BMP) to treat fecal incontinence and severe constipation is utilized for patients with anorectal malformations, Hirschsprung disease, spinal anomalies, and functional constipation, decreasing the rate of emergency department visits, and hospital admissions. This review is part of a manuscript series and focuses on updates in the use of antegrade flushes for bowel management, as well as organizational aspects, collaborative approach, telemedicine, the importance of family education, and one-year outcomes of the bowel management program. Implementation of a multidisciplinary program involving physicians, nurses, advanced practice providers, coordinators, psychologists, and social workers leads to rapid center growth and …

Sexual Behaviors, Contraception Use And Barriers Among Adolescents And Young Adults In Rural Haiti., Abbey R. Masonbrink, Emily A. Hurley, Nikolaus Schuetz, Jonathan Rodean, Emily Rupe, Kemi Lewis, Marie Daphnée Boncoeur, Melissa K. Miller

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BACKGROUND: Adolescents and young adults (AYAs) in Haiti experience a high unintended pregnancy rate, in part due to unmet contraception needs. Little is known about AYA opinions of and experiences with contraception that may explain remaining gaps in coverage. We aimed to describe barriers and facilitators to contraception use among AYAs in Haiti.

METHODS: We conducted a cross-sectional survey and semi-structured qualitative interviews with a convenience sample of AYA females aged 14-24 in two rural communities in Haiti. The survey and semi-structured interviews assessed demographics, sexual health and pregnancy prevention behaviors and explored contraception opinions and experiences according to Theory …

Protocol And Biomarker Strategy For A Multi-Site Randomized Controlled Trial Examining Biological Mechanisms And Dosing Of Active Music Engagement In Children With Acute Lymphoblastic Leukemia And Lymphoma And Parents., Sheri L. Robb, Kristen A. Russ, Steven J. Holochwost, Kristin Stegenga, Susan M. Perkins, Seethal A. Jacob, Amanda K. Henley, Jessica A. Maclean

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BACKGROUND: Music therapy is a standard palliative care service in many pediatric and adult hospitals; however, most research has focused on the use of music to improve psychosocial dimensions of health, without considering biological dimensions. This study builds on prior work examining psychosocial mechanisms of action underlying an Active Music Engagement (AME) intervention, designed to help manage emotional distress and improve positive health outcomes in young children with cancer and parents (caregivers), by examining its effects on biomarkers of stress and immune function.

METHODS: This two-group randomized controlled trial (R01NR019190) is designed to examine biological mechanisms of effect and dose-response …

Three-Year Update Of Tisagenlecleucel In Pediatric And Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia In The Eliana Trial., Theodore W. Laetsch, Shannon L. Maude, Susana Rives, Hidefumi Hiramatsu, Henrique Bittencourt, Peter Bader, André Baruchel, Michael Boyer, Barbara De Moerloose, Muna Qayed, Jochen Buechner, Michael A. Pulsipher, Douglas Myers, Heather E. Stefanski, Paul L. Martin, Eneida Nemecek, Christina Peters, Gregory Yanik, Seong Lin Khaw, Kara L. Davis, Joerg Krueger, Adriana Balduzzi, Nicolas Boissel, Ranjan Tiwari, Darragh O'Donovan, Stephan A. Grupp

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In the primary analysis of the global phase II ELIANA trial (ClinicalTrials.gov identifier: NCT02435849), tisagenlecleucel provided an overall remission rate of 81% in pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (R/R B-ALL), with 59% of responders remaining relapse-free at 12 months. Here, we report an update on efficacy, safety, and patient-reported quality of life in 79 pediatric and young adult patients with R/R B-ALL following a median follow-up of 38.8 months. The overall remission rate was 82%. The median event-free survival was 24 months, and the median overall survival was not reached. Event-free …

Risk And Resilient Functioning Of Families Of Children With Cancer During The Covid-19 Pandemic., Renee Gilbert, Carolyn R Bates, Devanshi Khetawat, Meredith Dreyer Gillette, Rachel Moore

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Previous literature highlights the impact of COVID-19 on family functioning. Less is known about the impact of the pandemic on families of pediatric cancer patients. In order to determine universal and unique risk and resilience factors of these families during the pandemic, a qualitative analysis was conducted on families currently receiving cancer treatment at a Midwestern hospital. Results of the data analysis depict ways in which these families have been impacted by and have adapted to COVID-19. These findings suggest that families of pediatric cancer patients have unique experiences in the context of COVID-19, in addition to universal experiences outlined …

Community-Onset Bacterial Coinfection In Children Critically Ill With Severe Acute Respiratory Syndrome Coronavirus 2 Infection., Kristin L. Moffitt, Mari M. Nakamura, Cameron C. Young, Margaret M. Newhams, Natasha B. Halasa, J Nelson Reed, Julie C. Fitzgerald, Philip C. Spinella, Vijaya L. Soma, Tracie C. Walker, Laura L. Loftis, Aline B. Maddux, Michele Kong, Courtney M. Rowan, Charlotte V. Hobbs, Jennifer E. Schuster, Becky J. Riggs, Gwenn E. Mclaughlin, Kelly N. Michelson, Mark W. Hall, Christopher J. Babbitt, Natalie Z. Cvijanovich, Matt S. Zinter, Mia Maamari, Adam J. Schwarz, Aalok R. Singh, Heidi R. Flori, Shira J. Gertz, Mary A. Staat, John S. Giuliano, Saul R. Hymes, Katharine N. Clouser, John Mcguire, Christopher L. Carroll, Neal J. Thomas, Emily R. Levy, Adrienne G. Randolph

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BACKGROUND: Community-onset bacterial coinfection in adults hospitalized with coronavirus disease 2019 (COVID-19) is reportedly uncommon, though empiric antibiotic use has been high. However, data regarding empiric antibiotic use and bacterial coinfection in children with critical illness from COVID-19 are scarce.

METHODS: We evaluated children and adolescents agedadmission, we adjudicated whether patients had community-onset bacterial coinfection. We compared demographic and clinical characteristics of those who did and did not (1) receive antibiotics and (2) have bacterial coinfection early in admission. Using Poisson regression models, we assessed factors associated with these outcomes.

RESULTS: Of the 532 patients, 63.3% received empiric antibiotics, but …

Higher Doses Of Tisagenlecleucel Are Associated With Improved Outcomes: A Report From The Pediatric Real-World Car Consortium., Heather E. Stefanski, Anne Eaton, Christina Baggott, Jenna Rossoff, Michael R. Verneris, Snehit Prabhu, Holly L. Pacenta, Christine L. Phillips, Julie-An Talano, Amy Moskop, Steven P. Margossian, Douglas Myers, Nicole A. Karras, Patrick A. Brown, Muna Qayed, Michelle Hermiston, Prakash Satwani, M Christa Krupski, Amy K. Keating, Rachel Wilcox, Cara A. Rabik, Vanessa A. Fabrizio, Vasant Chinnabhandar, A Yasemin Goksenin, Kevin J. Curran, Crystal L. Mackall, Theodore W. Laetsch, Liora M. Schultz

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Remarkable complete response rates have been shown with tisagenlecleucel, a chimeric antigen receptor (CAR) T-cell therapy targeting CD19, in patients up to age 26 years with refractory/relapsed B-cell acute lymphoblastic leukemia; it is US Food and Drug Administration approved for this indication. Currently, patients receive a single dose of tisagenlecleucel across a wide dose range of 0.2 to 5.0 × 106 and 0.1 to 2.5 × 108 CAR T cells per kg for patients ≤50 and >50 kg, respectively. The effect of cell dose on survival and remission is not yet well established. Our primary goal was to determine if …

Impact Of Duodenal Pathology On Oral Drug Bioavailability And Disease Outcomes In Pediatric Crohn's Disease., Rebecca Casini, Carrie A. Vyhlidal, Julia M. Bracken, Ashley K. Sherman, Atif Ahmed, Vivekanand Singh, Veronica Williams, Valentina Shakhnovich

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Background: Crohn's disease with upper gastrointestinal tract involvement occurs more often in children than adults and has the potential to interfere with oral drug absorption. We aimed to compare disease outcomes in children receiving oral azathioprine for the treatment of Crohn's disease with (DP) and without (NDP) duodenal pathology at diagnosis.

Methods: Duodenal villous length, body mass index (BMI), and laboratory studies were compared in DP vs. NDP during the first year post-diagnosis, using parametric/nonparametric tests and regression analysis (SAS v9.4); the data are reported as the median (interquartile range) or the mean ± standard deviation. Thiopurine metabolite concentration (pmol/8 …