Medicine and Health Sciences Commons^™

Clarification Of Adverse Drug Reactions By A Pharmacovigilance Team Results In Increased Antibiotic Re-Prescribing At A Freestanding United States Children's Hospital., Keith Feldman, Sarah Suppes, Jennifer Goldman

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Documentation of adverse drug reactions (ADRs) is a key factor in guiding future prescribing. However, incomplete documentation is common and often fails to distinguish implicated drugs as true allergies. This in turn leads to unnecessary avoidance of implicated drug classes and may result in sub-optimal prescribing. Pharmacovigilance (PV) programs utilize a systematic approach to clarify ADR documentation and are known to improve patient safety. Yet it remains unclear if PV alters prescribing. Or, if the existence of the ADR documentation itself continues to prompt avoidance of implicated drugs. To address this, our work presents a retrospective cohort study assessing if …

Postprandial Glucose Variability Following Typical Meals In Youth Living With Type 1 Diabetes., Susana R. Patton, Simon Bergford, Jennifer L. Sherr, Robin L. Gal, Peter Calhoun, Mark A. Clements, Michael C. Riddell, Corby K. Martin

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We explored the association between macronutrient intake and postprandial glucose variability in a large sample of youth living with T1D and consuming free-living meals. In the Type 1 Diabetes Exercise Initiative Pediatric (T1DEXIP) Study, youth took photographs before and after their meals on 3 days during a 10 day observation period. We used the remote food photograph method to obtain the macronutrient content of youth's meals. We also collected physical activity, continuous glucose monitoring, and insulin use data. We measured glycemic variability using standard deviation (SD) and coefficient of variation (CV) of glucose for up to 3 h after meals. …

Distinct Reproductive Phenotypes Segregate With Differences In Body Weight In Adolescent Polycystic Ovary Syndrome., Angie Chen-Patterson, Angelina Bernier, Tania S. Burgert, Vanessa Davis, Tazeena Khan, David Geller, Emily Paprocki, Rachana Shah, Selma F. Witchel, Camila Pereira-Eshraghi, Aviva B. Sopher, Melanie G. Cree, Laura C. Torchen

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INTRODUCTION: Polycystic ovary syndrome (PCOS) is a heterogenous clinical syndrome defined by hyperandrogenism and irregular menses. In adult women with PCOS, discrete metabolic and reproductive subgroups have been identified. We hypothesize that distinct phenotypes can be distinguished between adolescent girls who are lean (LN-G) and girls with obesity (OB-G) at the time of PCOS diagnosis.

METHODS: Data were extracted from the CALICO multisite PCOS database. Clinical data collected at the time of diagnosis were available in 354 patients (81% with obesity) from 7 academic centers. Patients with body mass index (BMI) < 85th percentile for age and sex were characterized as lean (LN-G) and those with BMI percentile ≥ 95th percentile as obese (OB-G). We compared metabolic and reproductive phenotypes in LN-G and OB-G.

RESULTS: Reproductive phenotypes differed between the groups, with LN-G …

Lessons Learnt In The First Year Of An Australian Pediatric Cardio Oncology Clinic., Claudia Toro, Ben Felmingham, Mangesh Jhadav, David S. Celermajer, Andre La Gerche, John O'Sullivan, Sanjeev Kumar, Marion K. Mateos, Joy M. Fulbright, Dinisha Govender, Lane Collier, Michael Cheung, David D. Eisenstat, Peter W. Lange, Julian Ayer, David A. Elliott, Rachel Conyers

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BACKGROUND: Modern oncological therapies together with chemotherapy and radiotherapy have broadened the agents that can cause cardiac sequelae, which can manifest for pediatric oncology patients while on active treatment. Recommendations for high-risk patients who should be monitored in a pediatric cardio-oncology clinic have previously been developed by expert Delphi consensus by our group. In 2022 we opened our first multidisciplinary pediatric cardio-oncology clinic adhering to these recommendations in surveillance and management.

OBJECTIVES: Our pediatric cardio-oncology clinic aimed to: (i) Document cardiovascular toxicities observed within a pediatric cardio-oncology clinic and. (ii) Evaluate the applicability of the Australian and New Zealand Pediatric …

Effects Of A Preventive Mental Health Curriculum Embedded Into A Scholarly Gaming Course On Adolescent Self-Esteem: Prospective Matched Pairs Experiment., Christopher Jenson, Sharon Fitzgerald Wolff, Libby Milkovich

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BACKGROUND: Positive self-esteem predicts happiness and well-being and serves as a protective factor for favorable mental health. Scholarly gaming within the school setting may serve as a channel to deliver a mental health curriculum designed to improve self-esteem.

OBJECTIVE: This study aims to evaluate the impact of a scholarly gaming curriculum with and without an embedded preventive mental health curriculum, Mental Health Moments (MHM), on adolescents' self-esteem.

METHODS: The scholarly gaming curriculum and MHM were developed by 3 educators and a school-based health intervention expert. The scholarly gaming curriculum aligned with academic guidelines from the International Society for Technology Education, …

Association Between Remote Monitoring And Interstage Morbidity And Death In Patients With Single-Ventricle Heart Disease Across Socioeconomic Groups., Bianca Cherestal, Lori A. Erickson, Janelle R. Noel-Macdonnell Phd, Girish S. Shirali, Hayley S. Hancock, Doaa Aly, Matthew Files, Sarah Clauss, Natalie Jayaram

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BACKGROUND: Despite improvements in survival over time, the mortality rate for infants with single-ventricle heart disease remains high. Infants of low socioeconomic status (SES) are particularly vulnerable. We sought to determine whether use of a novel remote monitoring program, the Cardiac High Acuity Monitoring Program, mitigates differences in outcomes by SES.

METHODS AND RESULTS: Within the Cardiac High Acuity Monitoring Program, we identified 610 infants across 11 centers from 2014 to 2021. All enrolled families had access to a mobile application allowing for near-instantaneous transfer of patient information to the care team. Patients were divided into SES tertiles on the …

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.

Rare Tumors: Opportunities And Challenges From The Children's Oncology Group Perspective., Kris Ann P. Schultz, Murali Chintagumpala, Jin Piao, Kenneth S. Chen, Rachana Shah, Robyn D. Gartrell, Emily Christison-Lagay, Farzana Pashnakar, Jesse L. Berry, Allison F. O'Neill, Lauren M. Vasta, Ashley Flynn, Sarah G. Mitchell, Brittani Kn Seynnaeve, Jeremy Rosenblum, Samara L. Potter, Junne Kamihara, Carlos Rodriguez-Galindo, Douglas S. Hawkins, Theodore W. Laetsch

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While all childhood cancers are rare, tumors that are particularly infrequent or underrepresented within pediatrics are studied under the umbrella of the Children's Oncology Group Rare Tumor committee, divided into the Retinoblastoma and Infrequent Tumor subcommittees. The Infrequent Tumor subcommittee has traditionally included an emphasis on globally rare tumors such as adrenocortical carcinoma, nasopharyngeal carcinoma, or those tumors that are rare in young children, despite being common in adolescents and young adults, such as colorectal carcinoma, thyroid carcinoma, and melanoma. Pleuropulmonary blastoma, gonadal stromal tumors, pancreatic tumors including pancreatoblastoma, gastrointestinal stromal tumor, nonmelanoma skin cancers, neuroendocrine tumors, and desmoplastic small …

Obesity And Outcomes Of Kawasaki Disease And Covid-19-Related Multisystem Inflammatory Syndrome In Children., Michael Khoury, Ashraf S. Harahsheh, Geetha Raghuveer, Nagib Dahdah, Simon Lee, Marianna Fabi, Elif Seda Selamet Tierney, Michael A. Portman, Nadine F. Choueiter, Matthew Elias, Deepika Thacker, Frédéric Dallaire, William B. Orr, Tyler H. Harris, Kambiz Norozi, Dongngan T. Truong, Manaswitha Khare, Jacqueline R. Szmuszkovicz, Joseph J. Pagano, Cedric Manlhiot, Pedrom Farid, Brian W. Mccrindle, International Kawasaki Disease Registry

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IMPORTANCE: Obesity may affect the clinical course of Kawasaki disease (KD) in children and multisystem inflammatory syndrome in children (MIS-C) associated with COVID-19.

OBJECTIVE: To compare the prevalence of obesity and associations with clinical outcomes in patients with KD or MIS-C.

DESIGN, SETTING, AND PARTICIPANTS: In this cohort study, analysis of International Kawasaki Disease Registry (IKDR) data on contemporaneous patients was conducted between January 1, 2020, and July 31, 2022 (42 sites, 8 countries). Patients with MIS-C (defined by Centers for Disease Control and Prevention criteria) and patients with KD (defined by American Heart Association criteria) were included. Patients with …

The Modified Clinical Progression Scale For Pediatric Patients: Evaluation As A Severity Metric And Outcome Measure In Severe Acute Viral Respiratory Illness., Shannon B. Leland, Steven J. Staffa, Margaret M. Newhams, Robinder G. Khemani, John C. Marshall, Cameron C. Young, Aline B. Maddux, Mark W. Hall, Scott L. Weiss, Adam J. Schwarz, Bria M. Coates, Ronald C. Sanders, Michele Kong, Neal J. Thomas, Ryan A. Nofziger, Melissa L. Cullimore, Natasha B. Halasa, Laura L. Loftis, Natalie Z. Cvijanovich, Jennifer E. Schuster, Heidi Flori, Shira J. Gertz, Janet R. Hume, Samantha M. Olson, Manish M. Patel, David Zurakowski, Adrienne G. Randolph, Pediatric Acute Lung And Sepsis Investigator’S Network Pediatric Intensive Care Influenza Study Group (Palisi Picflu) Investigators And Overcoming Covid-19 Investigators

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OBJECTIVES: To develop, evaluate, and explore the use of a pediatric ordinal score as a potential clinical trial outcome metric in children hospitalized with acute hypoxic respiratory failure caused by viral respiratory infections.

DESIGN: We modified the World Health Organization Clinical Progression Scale for pediatric patients (CPS-Ped) and assigned CPS-Ped at admission, days 2-4, 7, and 14. We identified predictors of clinical improvement (day 14 CPS-Ped ≤ 2 or a three-point decrease) using competing risks regression and compared clinical improvement to hospital length of stay (LOS) and ventilator-free days. We estimated sample sizes (80% power) to detect a 15% clinical …

Epigenetic Age Acceleration, Neonatal Morbidities, And Neurobehavioral Profiles In Infants Born Very Preterm., Uriel Paniagua, Barry M. Lester, Carmen J Marsit, Marie Camerota, Brian S. Carter, Jennifer F. Check, Jennifer Helderman, Julie A. Hofheimer, Elisabeth C. Mcgowan, Charles R. Neal, Steven L. Pastyrnak, Lynne M. Smith, Sheri A. Dellagrotta, Lynne M. Dansereau, T Michael O'Shea, Todd M. Everson

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Epigenetic age acceleration is a risk factor for chronic diseases of ageing and may reflect aspects of biological ageing. However, few studies have examined epigenetic ageing during the early neonatal period in preterm infants, who are at heightened risk of developmental problems. We examined relationships between neonatal age acceleration, neonatal morbidities, and neurobehavioral domains among very preterm (gestation) infants to characterize whether infants with early morbidities or different neurobehavioral characteristics had accelerated or decelerated epigenetic ageing. This study uses data from the Neonatal Neurobehavior and Outcomes in Very Preterm Infants (NOVI) study, restricted to infants with data on variables assessed …

Mediators And Moderators Of Active Music Engagement To Reduce Traumatic Stress Symptoms And Improve Well-Being In Parents Of Young Children With Cancer., Sheri L. Robb, Kristin Stegenga, Susan M. Perkins, Timothy E. Stump, Karen M. Moody, Amanda K. Henley, Jessica Maclean, Seethal A. Jacob, David Delgado, Paul R. Haut

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OBJECTIVE: This trial examined the effects of proximal/distal mediators and moderators of an Active Music Engagement (AME) intervention on young child/parent distress, quality of life, and family function outcomes.

METHODS: Child/parent dyads (n = 125) were randomized to AME or Audio-storybooks attention control condition. Each group received 3 sessions with a credentialed music therapist for 3 consecutive days with data collection at baseline, post-intervention (T2), and 30-days later (T3). Potential proximal mediators included within session child and parent engagement. Potential distal mediators included changes in perceived family normalcy, parent self-efficacy, and independent use of play materials. Potential moderators included parent/child …

Leukocyte Cell-Derived Chemotaxin 2 Correlates With Pediatric Non-Alcoholic Fatty Liver Disease., Diego Paine-Cabrera, Lisa Harvey, Dakota R. Robarts, Michele T. Pritchard, John Thyfault, Steven A. Weinman, Udayan Apte, Voytek Slowik

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Non-alcoholic fatty liver disease (NAFLD), newly renamed metabolic dysfunction-associated liver disease (MASLD), is a leading cause of liver disease in children and adults. There is a paucity of data surrounding potential biomarkers and therapeutic targets, especially in pediatric NAFLD. Leukocyte cell-derived chemotaxin 2 (LECT2) is a chemokine associated with both liver disease and skeletal muscle insulin resistance. Our aim was to determine associations between LECT2 and common clinical findings of NAFLD in pediatric patients. Enzyme-linked immunosorbent assay (ELISA) was used to measure serum LECT2 concentrations in children (aged 2-17 years) with and without NAFLD. LECT2 concentrations were then correlated to …

"My Gut Feeling Is…": An Ethnographic Study Exploring Interprofessional Communication About Children And Adolescents With Chronic Musculoskeletal Pain In Paediatric Rheumatology., Rebecca R. Lee, Janet E. Mcdonagh, Tim Rapley, Albert Farre, Mark Connelly, Tonya M. Palermo, Karine Toupin-April, Emily Wakefield, Sarah Peters, Lis Cordingley

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Interprofessional communication about inflammatory and non-inflammatory musculoskeletal conditions is an important component of assessment and management in paediatric rheumatology. Chronic pain is a feature of some of these conditions which likely influences the extent and type of communication about pain. Research investigating interprofessional communication about paediatric pain is limited but has found that communication is inclusive of the biopsychosocial context of children/adolescents as well as their families. The aim of this ethnographic study was to explore interprofessional communication about children and adolescents with chronic musculoskeletal pain in paediatric rheumatology. We observed forty-five healthcare professionals recruited from 3 UK paediatric rheumatology …

Hold Off That Olanzapine! The Development Of Neuroleptic Malignant Syndrome In A Dehydrated Pediatric Patient., Maryam Tariq, Danielle N. Wentzel, Joshua B. Beggs

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No abstract provided.

Sars-Cov-2 Epidemiology And Covid-19 Mrna Vaccine Effectiveness Among Infants And Children Aged 6 Months-4 Years - New Vaccine Surveillance Network, United States, July 2022-September 2023., Ayzsa Tannis, Janet A. Englund, Ariana Perez, Elizabeth J. Harker, Mary Allen Staat, Elizabeth P. Schlaudecker, Natasha B. Halasa, Laura S. Stewart, John V. Williams, Marian G. Michaels, Rangaraj Selvarangan, Jennifer E. Schuster, Leila C. Sahni, Julie A. Boom, Geoffrey A. Weinberg, Peter G. Szilagyi, Benjamin R. Clopper, Yingtao Zhou, Meredith L. Mcmorrow, Eileen J. Klein, Heidi L. Moline

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SARS-CoV-2 infection in young children is often mild or asymptomatic; however, some children are at risk for severe disease. Data describing the protective effectiveness of COVID-19 mRNA vaccines against COVID-19-associated emergency department (ED) visits and hospitalization in this population are limited. Data from the New Vaccine Surveillance Network, a prospective population-based surveillance system, were used to estimate vaccine effectiveness using a test-negative, case-control design and describe the epidemiology of SARS-CoV-2 in infants and children aged 6 months-4 years during July 1, 2022-September 30, 2023. Among 7,434 children included, 5% received a positive SARS-CoV-2 test result, and 95% received a negative …

Relationship Between The Covid-19 Pandemic And Structural Inequalities Within The Pediatric Trauma Population., Christina Georgeades, Amelia T. Collings, Manzur Farazi, Carisa Bergner, Mary E. Fallat, Peter C. Minneci, K Elizabeth Speck, Kyle J. Van Arendonk, Katherine J. Deans, Richard A. Falcone, David S. Foley, Jason D. Fraser, Samir K. Gadepalli, Martin S. Keller, Meera Kotagal, Matthew P. Landman, Charles M. Leys, Troy A. Markel, Nathan S. Rubalcava, Shawn D. St Peter, Thomas T. Sato, Katherine T. Flynn-O'Brien, Midwest Pediatric Surgery Consortium (Mwpsc)

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BACKGROUND: The COVID-19 pandemic disrupted social, political, and economic life across the world, shining a light on the vulnerability of many communities. The objective of this study was to assess injury patterns before and after implementation of stay-at-home orders (SHOs) between White children and children of color and across varying levels of vulnerability based upon children's home residence.

METHODS: A multi-institutional retrospective study was conducted evaluating patients < 18 years with traumatic injuries. A "Control" cohort from an averaged March-September 2016-2019 time period was compared to patients injured after SHO initiation-September 2020 ("COVID" cohort). Interactions between race/ethnicity or social vulnerability index (SVI), a marker of neighborhood vulnerability and socioeconomic status, and the COVID-19 timeframe with regard to the outcomes of interest were assessed using likelihood ratio Chi-square tests. Differences in injury intent, type, and mechanism were then stratified and explored by race/ethnicity and SVI separately.

RESULTS: A total of 47,385 patients met study inclusion. Significant interactions existed between race/ethnicity and the COVID-19 SHO period for intent (p < 0.001) and mechanism of injury (p < 0.001). There was also significant interaction between SVI and the COVID-19 SHO period for mechanism of injury (p = 0.01). Children of color experienced a significant increase in intentional (COVID 16.4% vs. Control 13.7%, p = 0.03) and firearm (COVID 9.0% vs. Control 5.2%, p < 0.001) injuries, but no change was seen among White children. Children from the most vulnerable neighborhoods suffered an increase in firearm injuries (COVID 11.1% vs. Control 6.1%, p = 0.001) with children from the least vulnerable neighborhoods having no change. All-terrain vehicle (ATV) and bicycle crashes increased for children of color (COVID 2.0% vs. Control 1.1%, p = 0.04 for ATV; COVID 6.7% vs. Control 4.8%, p = 0.02 for bicycle) and White children (COVID 9.6% vs. Control 6.2%, p < 0.001 for ATV; COVID 8.8% vs. Control 5.8%, p < 0.001 for bicycle).

CONCLUSIONS: In contrast to White children and children from neighborhoods of …

Barriers To The Diagnosis, Care, And Management Of Pediatric Patients With Ehlers-Danlos Syndrome In The United States: A Qualitative Analysis., William R. Black, Lora L. Black, Jordan T. Jones

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Objectives: Ehlers-Danlos Syndromes (EDS) are a family of heritable connective tissue diseases. Primary practitioners are capable of diagnosing and managing EDS; however, few are knowledgeable and comfortable enough to see patients with EDS, resulting in delays in diagnosis and care. This study explores the barriers physicians experience with diagnosing, managing, and caring for patients with EDS, and potential resolutions to those barriers. Methods: As part of a larger online study, providers (n = 107) in the United States were asked to specify "What information would improve (their) comfort" in diagnosing, caring for, and managing EDS via open-ended questions. Results …

Mediation Of The Association Between Social Environmental Characteristics Of Family Childcare Home And Weight Status In Children By Diet Quality., Qianxia Jiang, Patricia Markham Risica, Alison Tovar, Kristen Cooksey Stowers, Marlene B. Schwartz, Caitlin Lombardi, Kim M. Gans

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BACKGROUND: The food and beverages served in family childcare homes (FCCHs) may play an important role in the development of childhood overweight and obesity. This analysis examines whether children's diet quality mediates the relationship between foods and beverages served in FCCHs and preschool-aged children's weight status.

METHODS: Trained and certified staff conducted observations for two days in each FCCH, using the Environment and Policy Assessment and Observation (EPAO) measure to determine the foods and beverages served to children (N = 370) in FCCHs (N = 120). They also used the Dietary Observation in Child Care (DOCC) protocol to assess children's …

Pharmacokinetics And Safety Of A Single Dose Of Telavancin In Pediatric Subjects 2-17 Years Of Age., John S. Bradley, Jennifer Goldman, Laura P. James, Byron Kaelin, Breanne H Y Gibson, Antonio Arrieta

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Antimicrobial resistance increases infection morbidity in both adults and children, necessitating the development of new therapeutic options. Telavancin, an antibiotic approved in the United States for certain bacterial infections in adults, has not been examined in pediatric patients. The objectives of this study were to evaluate the short-term safety and pharmacokinetics (PK) of a single intravenous infusion of telavancin in pediatric patients. Single-dose safety and PK of 10 mg/kg telavancin was investigated in pediatric subjects >12 months to ≤17 years of age with known or suspected bacterial infection. Plasma was collected up to 24-h post-infusion and analyzed for concentrations of …

Appropriateness Of Antibiotic Prescribing Varies By Clinical Services At United States Children's Hospitals., Devin T. Diggs, Alison C. Tribble, Rebecca G. Same, Jason G. Newland, Brian R. Lee, Sharing Antimicrobial Reports For Pediatric Stewardship (Sharps) Collaborative

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OBJECTIVE: To describe patterns of inappropriate antibiotic prescribing at US children's hospitals and how these patterns vary by clinical service.

DESIGN: Serial, cross-sectional study using quarterly surveys.

SETTING: Surveys were completed in quarter 1 2019-quarter 3 2020 across 28 children's hospitals in the United States.

PARTICIPANTS: Patients at children's hospitals with ≥1 antibiotic order at 8:00 a.m. on institution-selected quarterly survey days.

METHODS: Antimicrobial stewardship physicians and pharmacists collected data on antibiotic orders and evaluated appropriateness of prescribing. The primary outcome was percentage of inappropriate antibiotics, stratified by clinical service and antibiotic class. Secondary outcomes included reasons for inappropriate use …

Clinical Efficacy Of Onc201 In H3k27m-Mutant Diffuse Midline Gliomas Is Driven By Disruption Of Integrated Metabolic And Epigenetic Pathways., Sriram Venneti, Abed Rahman Kawakibi, Sunjong Ji, Sebastian M. Waszak, Stefan R. Sweha, Mateus Mota, Matthew Pun, Akash Deogharkar, Chan Chung, Rohinton S. Tarapore, Samuel Ramage, Andrew Chi, Patrick Y. Wen, Isabel Arrillaga-Romany, Tracy T. Batchelor, Nicholas A. Butowski, Ashley Sumrall, Nicole Shonka, Rebecca A. Harrison, John De Groot, Minesh Mehta, Matthew D. Hall, Doured Daghistani, Timothy F. Cloughesy, Benjamin M. Ellingson, Kevin Beccaria, Pascale Varlet, Michelle M. Kim, Yoshie Umemura, Hugh Garton, Andrea Franson, Jonathan Schwartz, Rajan Jain, Maureen Kachman, Heidi Baum, Charles F. Burant, Sophie L. Mottl, Rodrigo T. Cartaxo, Vishal John, Dana Messinger, Tingting Qin, Erik Peterson, Peter Sajjakulnukit, Karthik Ravi, Alyssa Waugh, Dustin Walling, Yujie Ding, Ziyun Xia, Anna Schwendeman, Debra Hawes, Fusheng Yang, Alexander R. Judkins, Daniel Wahl, Costas A. Lyssiotis, Daniel De La Nava, Marta M. Alonso, Augustine Eze, Jasper Spitzer, Susanne V. Schmidt, Ryan J. Duchatel, Matthew D. Dun, Jason E. Cain, Li Jiang, Sylwia A. Stopka, Gerard Baquer, Michael S. Regan, Mariella G. Filbin, Nathalie Y R Agar, Lili Zhao, Chandan Kumar-Sinha, Rajen Mody, Arul Chinnaiyan, Ryo Kurokawa, Drew Pratt, Viveka Nand Yadav, Jacques Grill, Cassie Kline, Sabine Mueller, Adam Resnick, Javad Nazarian, Joshua E. Allen, Yazmin Odia, Sharon L. Gardner, Carl Koschmann

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UNLABELLED: Patients with H3K27M-mutant diffuse midline glioma (DMG) have no proven effective therapies. ONC201 has recently demonstrated efficacy in these patients, but the mechanism behind this finding remains unknown. We assessed clinical outcomes, tumor sequencing, and tissue/cerebrospinal fluid (CSF) correlate samples from patients treated in two completed multisite clinical studies. Patients treated with ONC201 following initial radiation but prior to recurrence demonstrated a median overall survival of 21.7 months, whereas those treated after recurrence had a median overall survival of 9.3 months. Radiographic response was associated with increased expression of key tricarboxylic acid cycle-related genes in baseline tumor sequencing. ONC201 …

Risk Factors For Health Impairments In Children After Hospitalization For Acute Covid-19 Or Mis-C., Aline B. Maddux, Cameron C. Young, Suden Kucukak, Laura D. Zambrano, Margaret M. Newhams, Caitlin K. Rollins, Natasha B. Halasa, Shira J. Gertz, Elizabeth H. Mack, Stephanie Schwartz, Michele Kong, Laura L. Loftis, Katherine Irby, Courtney M. Rowan, Keiko M. Tarquinio, Matt S. Zinter, Hillary Crandall, Natalie Z. Cvijanovich, Jennifer E. Schuster, Julie C. Fitzgerald, Mary A. Staat, Charlotte V. Hobbs, Ryan A. Nofziger, Steven Shein, Heidi Flori, Melissa L. Cullimore, Brandon M. Chatani, Emily R. Levy, Katri V. Typpo, Janet R. Hume, Angela P. Campbell, Adrienne G. Randolph, Overcoming Covid-19 Investigators

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OBJECTIVE: To identify risk factors for persistent impairments after pediatric hospitalization for acute coronavirus disease 2019 (COVID-19) or multisystem inflammatory syndrome in children (MIS-C) during the SARS-CoV-2 pandemic.

METHODS: Across 25 U.S. Overcoming COVID-19Network hospitals, we conducted a prospective cohort study of patients

RESULTS: Of 232 children with acute COVID-19, 71 (30.6%) had persistent symptoms and 50 (21.6%) had activity impairments at follow-up; for MIS-C (n = 241), 56 (23.2%) had persistent symptoms and 58 (24.1%) had activity impairments. In adjusted analyses of patients with acute COVID-19, receipt of mechanical ventilation was associated with persistent symptoms [aRR 1.83 …

Small Intestinal Polyp Burden In Pediatric Peutz-Jeghers Syndrome Assessed Through Capsule Endoscopy: A Longitudinal Study., Jeremy Stewart, Nathan R. Fleishman, Vincent S. Staggs, Mike Thomson, Nicole Stoecklein, Caitlin E. Lawson, Michael P. Washburn, Shahid Umar, Thomas M. Attard

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The management of pediatric Peutz-Jeghers Syndrome (PJS) focuses on the prevention of intussusception complicating small intestinal (SI) polyposis. This hinges on the accurate appraisal of the polyp burden to tailor therapeutic interventions. Video Capsule Endoscopy (VCE) is an established tool to study SI polyps in children, but an in-depth characterization of polyp burden in this population is lacking. Methods: We performed a retrospective longitudinal cross-sectional analysis of VCE studies in pediatric PJS patients at our institution (CMKC) from 2010 to 2020. Demographic, clinical, and VCE findings reported by three reviewers in tandem were accrued. Polyp burden variables were modeled as …

Characteristics And Prognostic Impact Of Idh Mutations In Aml: A Cog, Swog, And Ecog Analysis., Sara Zarnegar-Lumley, Todd A. Alonzo, Robert B. Gerbing, Megan Othus, Zhuoxin Sun, Rhonda E. Ries, Jim Wang, Amanda Leonti, Matthew A. Kutny, Fabiana Ostronoff, Jerald P. Radich, Frederick R. Appelbaum, Era L. Pogosova-Agadjanyan, Kristen O'Dwyer, Martin S. Tallman, Mark Litzow, Ehab Atallah, Todd M. Cooper, Richard A. Aplenc, Omar Abdel-Wahab, Alan S. Gamis, Selina Luger, Harry Erba, Ross Levine, E Anders Kolb, Derek L. Stirewalt, Soheil Meshinchi, Katherine Tarlock

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Somatic mutations in isocitrate dehydrogenase (IDH) genes occur frequently in adult acute myeloid leukemia (AML) and less commonly in pediatric AML. The objective of this study was to describe the prevalence, mutational profile, and prognostic significance of IDH mutations in AML across age. Our cohort included 3141 patients aged betweenChildren's Cancer Group/Children's Oncology Group (n = 1872), Southwest Oncology Group (n = 359), Eastern Cooperative Oncology Group (n = 397) trials, and in Beat AML (n = 333) and The Cancer Genome Atlas (n = 180) genomic characterization cohorts. We retrospectively analyzed patients in 4 age groups (age range, n): …

Inducing Mitotic Catastrophe As A Therapeutic Approach To Improve Outcomes In Ewing Sarcoma., Soumya M. Turaga, Vikalp Vishwakarma, Stacey L. Hembruff, Benjamin K. Gibbs, Priya Sabu, Rajni V. Puri, Harsh B. Pathak, Glenson Samuel, Andrew K. Godwin

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Ewing sarcoma (EWS) is an aggressive pediatric malignancy of the bone and soft tissues in need of novel therapeutic options. To identify potential therapeutic targets, we focused on essential biological pathways that are upregulated by EWS-FLI1, the primary oncogenic driver of EWS, including mitotic proteins such as Aurora kinase A (AURKA) and kinesin family member 15 (KIF15) and its binding partner, targeting protein for Xklp2 (TPX2). KIF15/TPX2 cooperates with KIF11, a key mitotic kinesin essential for mitotic spindle orientation. Given the lack of clinical-grade KIF15/TPX2 inhibitors, we chose to target KIF11 (using SB-743921) in combination with AURKA (using VIC-1911) given …

Molecular Characterization Of Clostridium Perfringens Isolates From A Tertiary Children's Hospital In Guangzhou, China, Establishing An Association Between Bacterial Colonization And Food Allergies In Infants., Kun-Yi Huang, Bing-Shao Liang, Xiao-Yan Zhang, Huan Chen, Ni Ma, Jiao-Li Lan, Ding-You Li, Zhen-Wen Zhou, Min Yang

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BACKGROUND: Cow's milk protein allergy (CMPA) is one of the most common types of food allergy in infants. Faecal pathogen cultures showed that the positive rate of Clostridium perfringens was more than 30%, which was significantly higher than that for other bacteria. Therefore, it is speculated that Clostridium perfringens colonization may be one of the pathogenetic factors for CMPA in infants. We conducted a real-world evidence study. Infants aged 0-6 months with diarrhoea and mucoid and/or bloody stools were recruited from a large tertiary hospital in China. Faecal pathogen cultures for the detection of Clostridium perfringens were confirmed by flight …

3-Year Follow-Up Of A Prospective, Multicenter Study Of The Amplatzer Piccolo™ Occluder For Transcatheter Patent Ductus Arteriosus Closure In Children ≥ 700 Grams., Brian H. Morray, Shyam K. Sathanandam, Thomas Forbes, Matthew Gillespie, Darren Berman, Aimee K. Armstrong, Shabana Shahanavaz, Thomas Jones, Toby A. Rockefeller, Henri Justino, David Nykanen, Courtney Weiler, Dan Gutfinger, Evan M. Zahn

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OBJECTIVE: This study describes 3-year follow-up of 200 infants weighing ≥ 700 grams who underwent transcatheter patent ductus arteriosus (PDA) closure with the Amplatzer Piccolo™ Occluder.

STUDY DESIGN: Between June 2017 and February 2019, 200 children were enrolled in this U.S. study (NCT03055858). PDA closure, survival, and device- or procedure-related events were evaluated. A total of 156 of the available 182 patients (86%) completed the study.

RESULTS: The implant success rate was 95.5% (191/200). At 3 years, PDA closure was observed in 100% (33/33) of patients. Survival was >95% with 9 reported deaths. No deaths were adjudicated as device- or …

The Impact Of Pediatric Ckd On Educational And Employment Outcomes., Lyndsay A. Harshman, Ryan C. Ward, Matthew B. Matheson, Anne Dawson, Amy J. Kogon, Marc B. Lande, Stephen J. Molitor, Rebecca J. Johnson, Camille Wilson, Bradley A. Warady, Susan L. Furth, Stephen R. Hooper

Manuscripts, Articles, Book Chapters and Other Papers

KEY POINTS: This study evaluates educational and employment outcomes in patients with pediatric kidney disease and assesses predictors of educational attainment and employment in young adulthood. Despite high rates of high school graduation, nearly 20% of patients with CKD are unemployed or receiving disability at long-term follow-up.

BACKGROUND: Pediatric patients with CKD are at risk for neurocognitive deficits and academic underachievement. This population may be at risk for lower educational attainment and higher rates of unemployment; however, published data have focused on patients with advanced CKD and exist in isolation from assessment of neurocognition and kidney function.

METHODS: Data from …

Pharmacogenetic Testing In Patients With Autism Spectrum Disorder Evaluated In A Precision Medicine Clinic., Rachel Goodson, Jennifer Wagner, Tracy L. Sandritter, Vincent S. Staggs, Sarah E. Soden, Cy Nadler

Manuscripts, Articles, Book Chapters and Other Papers

OBJECTIVE: This study investigated outcomes of pharmacogenetic testing of youth with autism spectrum disorder (ASD) referred to a precision medicine clinic and explored associations between patient characteristics and pharmacogenomic testing results.

METHODS: Records for patients diagnosed with ASD and subsequently referred to a pediatric hospital's precision medicine clinic between July 1, 2010, and June 30, 2020, were reviewed. Pharmacogenetic testing results were abstracted focusing on CYP2D6 and CYP2C19. In addition, we compiled counts of patients' co-occurring diagnoses, histories of adverse drug reactions (ADRs), previously trialed ineffective medications, and previous psychiatric medication changes. Logistic regression models were fit to examine CYP2C19 …