Medical Specialties Commons^™

Insulin-Like Growth Factor-1 Supplementation Promotes Brain Maturation In Preterm Pigs, Line I Christiansen, Bo Holmqvist, Xiaoyu Pan, Kristine Holgersen, Sandy E H Lindholm, Nicole L Henriksen, Douglas G Burrin, David Ley, Thomas Thymann, Per Torp Sangild, Stanislava Pankratova

Faculty, Staff and Students Publications

Very preterm infants show low levels of insulin-like growth factor-1 (IGF-1), which is associated with postnatal growth restriction and poor neurologic outcomes. It remains unknown whether supplemental IGF-1 may stimulate neurodevelopment in preterm neonates. Using cesarean-delivered preterm pigs as a model of preterm infants, we investigated the effects of supplemental IGF-1 on motor function and on regional and cellular brain development. Pigs were treated with 2.25 mg/kg/d recombinant human IGF-1/IGF binding protein-3 complex from birth until day 5 or 9 before the collection of brain samples for quantitative immunohistochemistry (IHC), RNA sequencing, and quantitative PCR analyses. Brain protein synthesis was …

Admixture Mapping In African Americans Identifies New Risk Loci For Hcv-Related Cirrhosis, Hyun-Seok Kim, Priya B Shetty, Spiridon Tsavachidis, Jing Dong, Christopher I Amos, Hashem B El-Serag, Aaron P Thrift

Faculty, Staff and Students Publications

BACKGROUND & AIMS: Cirrhosis is the main predisposing condition for hepatocellular carcinoma. Host genetic risk factors have been reported for cirrhosis; however, whether there is a genetic contribution to racial disparities in cirrhosis requires further investigation.

METHODS: We used an affected-only mapping by admixture linkage disequilibrium analysis to characterize the genetic risk of cirrhosis in 227 African American patients with cirrhosis genotyped at 19,804 ancestry-informative marker single nucleotide polymorphisms. We additionally performed analyses stratified by hepatitis C virus (HCV) infection status. To replicate our findings, we conducted a case-control analysis in an external study population (452 cases and 196 controls). …

Clinical Implications Of Molecular Drug Resistance Testing For Mycobacterium Tuberculosis: A 2023 Tbnet/Resist-Tb Consensus Statement, José Domínguez, Martin J Boeree, Emmanuelle Cambau, Dumitru Chesov, Francesca Conradie, Vivian Cox, Keertan Dheda, Andrii Dudnyk, Maha R Farhat, Sebastien Gagneux, Martin P Grobusch, Matthias I Gröschel, Lorenzo Guglielmetti, Irina Kontsevaya, Berit Lange, Frank Van Leth, Christian Lienhardt, Anna M Mandalakas, Florian P Maurer, Matthias Merker, Paolo Miotto, Barbara Molina-Moya, Florence Morel, Stefan Niemann, Nicolas Veziris, Andrew Whitelaw, Charles R Horsburgh, Christoph Lange

Faculty, Staff and Students Publications

Drug-resistant tuberculosis is a substantial health-care concern worldwide. Despite culture-based methods being considered the gold standard for drug susceptibility testing, molecular methods provide rapid information about the Mycobacterium tuberculosis mutations associated with resistance to anti-tuberculosis drugs. This consensus document was developed on the basis of a comprehensive literature search, by the TBnet and RESIST-TB networks, about reporting standards for the clinical use of molecular drug susceptibility testing. Review and the search for evidence included hand-searching journals and searching electronic databases. The panel identified studies that linked mutations in genomic regions of M tuberculosis with treatment outcome data. Implementation of molecular …

Efficacy And Safety Of Lumasiran For Infants And Young Children With Primary Hyperoxaluria Type 1: 12-Month Analysis Of The Phase 3 Illuminate-B Trial, Wesley Hayes, David J Sas, Daniella Magen, Hadas Shasha-Lavsky, Mini Michael, Anne-Laure Sellier-Leclerc, Julien Hogan, Taylor Ngo, Marianne T Sweetser, John M Gansner, Tracy L Mcgregor, Yaacov Frishberg

Faculty, Staff and Students Publications

BACKGROUND: Primary hyperoxaluria type 1 (PH1) is a rare genetic disease that causes progressive kidney damage and systemic oxalosis due to hepatic overproduction of oxalate. Lumasiran demonstrated efficacy and safety in the 6-month primary analysis period of the phase 3, multinational, open-label, single-arm ILLUMINATE-B study of infants and children < 6 years old with PH1 (ClinicalTrials.gov: NCT03905694 (4/1/2019); EudraCT: 2018-004,014-17 (10/12/2018)). Outcomes in the ILLUMINATE-B extension period (EP) for patients who completed ≥ 12 months on study are reported here.

METHODS: Of the 18 patients enrolled in the 6-month primary analysis period, all entered the EP and completed ≥ 6 additional months of lumasiran treatment (median (range) duration of total exposure, 17.8 (12.7-20.5) months).

RESULTS: Lumasiran treatment was previously reported to reduce spot urinary oxalate:creatinine ratio by 72% at month 6, …

Fit24, A Digital Health Intervention To Reduce Type 2 Diabetes Risk Among Hispanic Youth: Protocol For A Feasibility Pilot Study, Erica G Soltero, Callie Lopez, Salma M Musaad, Teresia M O'Connor, Debbe Thompson

Faculty, Staff and Students Publications

BACKGROUND: Poor physical activity (PA) and sleep behaviors in Hispanic adolescents contributes to increased risk for type 2 diabetes. Commonly owned digital devices and services like smartphones and text-messaging are highly used among adolescents and are promising intervention tools for reaching this age group. Personal activity trackers assess activity and sleep, making them ideal tools for addressing these behaviors. We propose to examine the feasibility of a 12-week intervention that uses theoretically grounded text messages and a Fitbit device to improve PA and sleep among Hispanic adolescents with obesity, as compared to a wait-list control group with a Fitbit device …

Monogenic Early-Onset Lymphoproliferation And Autoimmunity: Natural History Of Stat3 Gain-Of-Function Syndrome, Jennifer W Leiding, Tiphanie P Vogel, Valentine G J Santarlas, Rahul Mhaskar, Madison R Smith, Alexandre Carisey, Alexander Vargas-Hernández, Manuel Silva-Carmona, Maximilian Heeg, Anne Rensing-Ehl, Bénédicte Neven, Jérôme Hadjadj, Sophie Hambleton, Timothy Ronan Leahy, Kornvalee Meesilpavikai, Charlotte Cunningham-Rundles, Cullen M Dutmer, Svetlana O Sharapova, Mervi Taskinen, Ignatius Chua, Rosie Hague, Christian Klemann, Larysa Kostyuchenko, Tomohiro Morio, Akaluck Thatayatikom, Ahmet Ozen, Anna Scherbina, Cindy S Bauer, Sarah E Flanagan, Eleonora Gambineri, Lisa Giovannini-Chami, Jennifer Heimall, Kathleen E Sullivan, Eric Allenspach, Neil Romberg, Sean G Deane, Benjamin T Prince, Melissa J Rose, John Bohnsack, Talal Mousallem, Rohith Jesudas, Maria Marluce Dos Santos Vilela, Michael O'Sullivan, Jana Pachlopnik Schmid, Štěpánka Průhová, Adam Klocperk, Matthew Rees, Helen Su, Sami Bahna, Safa Baris, Lisa M Bartnikas, Amy Chang Berger, Tracy A Briggs, Shannon Brothers, Vanessa Bundy, Alice Y Chan, Shanmuganathan Chandrakasan, Mette Christiansen, Theresa Cole, Matthew C Cook, Mukesh M Desai, Ute Fischer, David A Fulcher, Silvanna Gallo, Amelie Gauthier, Andrew R Gennery, José Gonçalo Marques, Frédéric Gottrand, Bodo Grimbacher, Eyal Grunebaum, Emma Haapaniemi, Sari Hämäläinen, Kaarina Heiskanen, Tarja Heiskanen-Kosma, Hal M Hoffman, Luis Ignacio Gonzalez-Granado, Anthony L Guerrerio, Leena Kainulainen, Ashish Kumar, Monica G Lawrence, Carina Levin, Timi Martelius, Olaf Neth, Peter Olbrich, Alejandro Palma, Niraj C Patel, Tamara Pozos, Kahn Preece, Saúl Oswaldo Lugo Reyes, Mark A Russell, Yael Schejter, Christine Seroogy, Jan Sinclair, Effie Skevofilax, Daniel Suan, Daniel Suez, Paul Szabolcs, Helena Velasco, Klaus Warnatz, Kelly Walkovich, Austen Worth, Stat3 Gof Working Group Members, Mikko R J Seppänen, Troy R Torgerson, Georgios Sogkas, Stephan Ehl, Stuart G Tangye, Megan A Cooper, Joshua D Milner, Lisa R Forbes Satter

Faculty, Staff and Students Publications

BACKGROUND: In 2014, germline signal transducer and activator of transcription (STAT) 3 gain-of-function (GOF) mutations were first described to cause a novel multisystem disease of early-onset lymphoproliferation and autoimmunity.

OBJECTIVE: This pivotal cohort study defines the scope, natural history, treatment, and overall survival of a large global cohort of patients with pathogenic STAT3 GOF variants.

METHODS: We identified 191 patients from 33 countries with 72 unique mutations. Inclusion criteria included symptoms of immune dysregulation and a biochemically confirmed germline heterozygous GOF variant in STAT3.

RESULTS: Overall survival was 88%, median age at onset of symptoms was 2.3 years, and median …

Therapeutic Plasma Exchange Is Associated With Improved Major Adverse Kidney Events In Children And Young Adults With Thrombocytopenia At The Time Of Continuous Kidney Replacement Therapy Initiation, Dana Y Fuhrman, Sameer Thadani, Claire Hanson, Joseph A Carcillo, John A Kellum, Hyun Jung Park, Liling Lu, Nahmah Kim-Campbell, Christopher M Horvat, Ayse Akcan Arikan

Faculty, Staff and Students Publications

Therapeutic plasma exchange (TPE) has been shown to improve organ dysfunction and survival in patients with thrombotic microangiopathy and thrombocytopenia associated with multiple organ failure. There are no known therapies for the prevention of major adverse kidney events after continuous kidney replacement therapy (CKRT). The primary objective of this study was to evaluate the effect of TPE on the rate of adverse kidney events in children and young adults with thrombocytopenia at the time of CKRT initiation.

DESIGN: Retrospective cohort.

SETTING: Two large quaternary care pediatric hospitals.

PATIENTS: All patients less than or equal to 26 years old who received …

Nfkb2 Haploinsufficiency Identified Via Screening For Ifn-Α2 Autoantibodies In Children And Adolescents Hospitalized With Sars-Cov-2-Related Complications, Aaron Bodansky, Sara E Vazquez, Janet Chou, Tanya Novak, Amer Al-Musa, Cameron Young, Margaret Newhams, Suden Kucukak, Laura D Zambrano, Anthea Mitchell, Chung-Yu Wang, Kristin Moffitt, Natasha B Halasa, Laura L Loftis, Stephanie P Schwartz, Tracie C Walker, Elizabeth H Mack, Julie C Fitzgerald, Shira J Gertz, Courtney M Rowan, Katherine Irby, Ronald C Sanders, Michele Kong, Jennifer E Schuster, Mary A Staat, Matt S Zinter, Natalie Z Cvijanovich, Keiko M Tarquinio, Bria M Coates, Heidi R Flori, Mary K Dahmer, Hillary Crandall, Melissa L Cullimore, Emily R Levy, Brandon Chatani, Ryan Nofziger, Overcoming Covid-19 Network Study Group Investigators, Raif S Geha, Joseph Derisi, Angela P Campbell, Mark Anderson, Adrienne G Randolph

Faculty, Staff and Students Publications

BACKGROUND: Autoantibodies against type I IFNs occur in approximately 10% of adults with life-threatening coronavirus disease 2019 (COVID-19). The frequency of anti-IFN autoantibodies in children with severe sequelae of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection is unknown.

OBJECTIVE: We quantified anti-type I IFN autoantibodies in a multicenter cohort of children with severe COVID-19, multisystem inflammatory syndrome in children (MIS-C), and mild SARS-CoV-2 infections.

METHODS: Circulating anti-IFN-α2 antibodies were measured by a radioligand binding assay. Whole-exome sequencing, RNA sequencing, and functional studies of peripheral blood mononuclear cells were used to study any patients with levels of anti-IFN-α2 autoantibodies exceeding …

Functional Effects Of Disease-Associated Variants Reveal That The S1-M1 Linker Of The Nmda Receptor Critically Controls Channel Opening, Lingling Xie, Miranda J Mcdaniel, Riley E Perszyk, Sukhan Kim, Gerarda Cappuccio, Kevin A Shapiro, Beatriz Muñoz-Cabello, Pedro A Sanchez-Lara, Katheryn Grand, Jing Zhang, Kelsey A Nocilla, Rehan Sheikh, Lluis Armengol, Roberta Romano, Tyler Mark Pierson, Hongjie Yuan, Scott J Myers, Stephen F Traynelis

Faculty, Staff and Students Publications

The short pre-M1 helix within the S1-M1 linker (also referred to as the pre-M1 linker) between the agonist-binding domain (ABD, S1) and the M1 transmembrane helix of the NMDA receptor (NMDAR) is devoid of missense variants within the healthy population but is a locus for de novo pathogenic variants associated with neurological disorders. Several de novo variants within this helix have been identified in patients presenting early in life with intellectual disability, developmental delay, and/or epilepsy. In this study, we evaluated functional properties for twenty variants within the pre-M1 linker in GRIN1, GRIN2A, and GRIN2B genes, including six novel missense …

Obesity Heterogeneity By Neighborhood Context In A Largely Latinx Sample, Ashley W. Kranjac, Dinko Kranjac, Zeev N. Kain, Louis Ehwerhemuepha, Brooke N. Jenkins

Sociology Faculty Articles and Research

Neighborhood socioeconomic context where Latinx children live may influence body weight status. Los Angeles County and Orange County of Southern California both are on the list of the top ten counties with the largest Latinx population in the USA. This heterogeneity allowed us to estimate differential impacts of neighborhood environment on children’s body mass index z-scores by race/ethnicity using novel methods and a rich data source. We geocoded pediatric electronic medical record data from a predominantly Latinx sample and characterized neighborhoods into unique residential contexts using latent profile modeling techniques. We estimated multilevel linear regression models that adjust for …

What To Look For In Relationships: Development, Inter-Rater Reliability, And Initial Validity Estimates For A Young Child-Caregiver Relationship Assessment, Joy D. Osofsky, Margaret A. Fields-Olivieri, Andrew L. Frazer, Rebecca A. Graham, Bethany H. Mccurdy, Carl F. Weems

School of Medicine Faculty Publications

Introduction: Observational assessments are important for understanding a range of behaviors and emotions in the young child-caregiver relationship. This paper provides initial data on a multidimensional assessment for professionals who work with young children and their caregivers, the What to Look for in Relationships (WLR). The WLR was designed to assist providers in evaluating strengths and areas for improvement in five areas of young child-caregiver relationship dimensions. This paper reports on the development, interrater reliability, initial convergent and discriminant validity, and incremental utility of the scales. Methods: Data were collected from caregiver-child dyads, who participated in a semi-structured observational caregiver-child …

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

Manuscripts, Articles, Book Chapters and Other Papers

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 …

Relationships Among Biochemical Measures In Children With Diabetic Ketoacidosis, Nicole S Glaser, Michael J Stoner, Maria Y Kwok, Kimberly S Quayle, Kathleen M Brown, Jeff E Schunk, Jennifer L Trainor, Julie K Mcmanemy, Leah Tzimenatos, Arleta Rewers, Lise E Nigrovic, Jonathan E Bennett, Sage R Myers, Mckenna Smith, T Charles Casper, Nathan Kuppermann

Faculty, Staff and Students Publications

OBJECTIVES: Investigating empirical relationships among laboratory measures in children with diabetic ketoacidosis (DKA) can provide insights into physiological alterations occurring during DKA. We determined whether alterations in laboratory measures during DKA conform to theoretical predictions.

METHODS: We used Pearson correlation statistics and linear regression to investigate correlations between blood glucose, electrolytes, pH and PCO2 at emergency department presentation in 1,681 pediatric DKA episodes. Among children with repeat DKA episodes, we also assessed correlations between laboratory measures at the first vs. second episode.

RESULTS: pH and bicarbonate levels were strongly correlated (r=0.64), however, pH and PCO2 were only loosely correlated (r=0.17). …

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

Manuscripts, Articles, Book Chapters and Other Papers

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

Manuscripts, Articles, Book Chapters and Other Papers

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 …

Venetoclax For Acute Myeloid Leukemia In Pediatric Patients: A Texas Medical Center Experience, Adriana Trabal, Amber Gibson, Jiasen He, David Mccall, Michael Roth, Cesar Nuñez, Miriam Garcia, Meredith Buzbee, Laurie Toepfer, Aram Bidikian, Naval Daver, Tapan Kadia, Nicholas J Short, Ghayas C Issa, Farhad Ravandi, Courtney D Dinardo, Guillermo Montalban Bravo, Sofia Garces, Andrea Marcogliese, Hana Paek, Zoann Dreyer, Julienne Brackett, Michele Redell, Joanna Yi, Guillermo Garcia-Manero, Marina Konopleva, Alexandra Stevens, Branko Cuglievan

Faculty, Staff and Student Publications

The BCL-2 inhibitor venetoclax improves survival for adult patients with acute myeloid leukemia (AML) in combination with lower-intensity therapies, but its benefit in pediatric patients with AML remains unclear. We retrospectively reviewed two Texas Medical Center institutions' experience with venetoclax in 43 pediatric patients with AML; median age 17 years (range, 0.6-21). This population was highly refractory; 44% of patients (n = 19) had ≥3 prior lines of therapy, 37% (n = 16) had received a prior bone marrow transplant, and 81% (n = 35) had unfavorable genetics KMT2A (n = 17), WT1 (n = 13), FLT3-ITD (n = 10), …

Soybean Gmsaul1, A Bona Fide U-Box E3 Ligase, Negatively Regulates Immunity Likely Through Repressing The Activation Of Gmmpk3, Jun-Mei Li, Mei-Yan Ye, Chaofeng Wang, Xiao-Han Ma, Ni-Ni Wu, Chen-Li Zhong, Yanjun Zhang, Ninghui Cheng, Paul A Nakata, Lirong Zeng, Jian-Zhong Liu

Faculty, Staff and Students Publications

E3 ubiquitin ligases play important roles in plant immunity, but their role in soybean has not been investigated previously. Here, we used Bean pod mottle virus (BPMV)-mediated virus-induced gene silencing (VIGS) to investigate the function of GmSAUL1 (Senescence-Associated E3 Ubiquitin Ligase 1) homologs in soybean. When two closely related SAUL1 homologs were silenced simultaneously, the soybean plants displayed autoimmune phenotypes, which were significantly alleviated by high temperature, suggesting that GmSAUL1a/1b might be guarded by an R protein. Interestingly, silencing GmSAUL1a/1b resulted in the decreased activation of GmMPK6, but increased activation of GmMPK3 in response to flg22, …

Impact Of Intravenous Fluid Administration On Cardiac Output And Oxygenation During Cardiopulmonary Resuscitation, Jennifer Lutz, Yosef Levenbrown, Md Jobayer Hossain, Anne Hesek, Kelly E. Massa, James P. Keith, Thomas H. Shaffer

Department of Pediatrics Faculty Papers

Background: The efect of intravenous fuid (IVF) administration during cardiopulmonary resuscitation (CPR) is an unexplored factor that may improve cardiac output (CO) during CPR. The aim of this study was to determine the efect of IVF administration on CO and oxygenation during CPR.

Methods: This experimental animal study was performed in a critical care animal laboratory. Twenty-two Landrace-Yorkshire female piglets weighing 27–37 kg were anesthetized, intubated, and placed on positive pressure ventilation. Irreversible cardiac arrest was induced with bupivacaine. CPR was performed with a LUCAS 3 mechanical compression device. Pigs were randomized into IVF or no-IVF groups. Pigs in the …

Does Positive Psychology Coaching Improve Trainee Well-Being? Evidence From A Longitudinal Professional Development Coaching Program In A Cohort Of Pediatric Trainees, Benjamin Nelson, Jacqueline T. Chu, Michael G. Healy, Yoon Soo Park, Kerri Palamara Md

Journal of Wellness

Introduction: Trainee burnout is common and evidence supporting the effectiveness of well-being interventions for this population is limited. We studied the effect of a longitudinal coaching program grounded in positive psychology on measures of pediatric trainee well-being.

Methods: Pediatric interns and fellows (n = 67) were enrolled in a positive psychology coaching program in 2017-2019. Pediatric faculty (n = 23) underwent training and were paired with trainees outside their field of interest. Trainees were surveyed at the beginning and end of the program to assess burnout and well-being, and key skills necessary to achieve well-being.

Results: Thirty-one …

Interleukins, Chemokines, And Tumor Necrosis Factor Superfamily Ligands In The Pathogenesis Of West Nile Virus Infection, Emna Benzarti, Kristy O Murray, Shannon E Ronca

Faculty, Staff and Students Publications

West Nile virus (WNV) is a mosquito-borne pathogen that can lead to encephalitis and death in susceptible hosts. Cytokines play a critical role in inflammation and immunity in response to WNV infection. Murine models provide evidence that some cytokines offer protection against acute WNV infection and assist with viral clearance, while others play a multifaceted role WNV neuropathogenesis and immune-mediated tissue damage. This article aims to provide an up-to-date review of cytokine expression patterns in human and experimental animal models of WNV infections. Here, we outline the interleukins, chemokines, and tumor necrosis factor superfamily ligands associated with WNV infection and …

Catalyzing Next-Generation Artificial Intelligence Through Neuroai, Anthony Zador, Sean Escola, Blake Richards, Bence Ölveczky, Yoshua Bengio, Kwabena Boahen, Matthew Botvinick, Dmitri Chklovskii, Anne Churchland, Claudia Clopath, James Dicarlo, Surya Ganguli, Jeff Hawkins, Konrad Körding, Alexei Koulakov, Yann Lecun, Timothy Lillicrap, Adam Marblestone, Bruno Olshausen, Alexandre Pouget, Cristina Savin, Terrence Sejnowski, Eero Simoncelli, Sara Solla, David Sussillo, Andreas S Tolias, Doris Tsao

Faculty, Staff and Students Publications

Neuroscience has long been an essential driver of progress in artificial intelligence (AI). We propose that to accelerate progress in AI, we must invest in fundamental research in NeuroAI. A core component of this is the embodied Turing test, which challenges AI animal models to interact with the sensorimotor world at skill levels akin to their living counterparts. The embodied Turing test shifts the focus from those capabilities like game playing and language that are especially well-developed or uniquely human to those capabilities - inherited from over 500 million years of evolution - that are shared with all animals. Building …

Inhibitors Of Histone Deacetylase And Mcl-1 Synergistically Reduce Proliferation In Malignant Melanoma, Mehrnoosh Ghafouri, Chester Gauss, Yue Xi Phd, William Azkoul Ii Md, Abby Knudsen, Jordan Zuckerman, Sharon K. Michelhaugh Phd, Sandeep Mittal Md, Andrew M. Fribley Phd

Medical Student Research Symposium

Melanoma is a skin cancer that arises in melanocytes; it is the fifth most common cancer in the United States with approximately 100,000 new cases per year. Current treatments for malignant melanoma are surgical excision, radiation therapy and systemic therapy; however, the five-year survival rate for patients with stage IV is 29.8%. There is an urgent unmet clinical need to investigate novel treatments for these patients. Panobinostat is an orally available histone deacetylase inhibitor used in several hematologic malignancies, but it was ineffective as a single agent against melanoma in Phase 1. To address the insufficiency of options for melanoma …

Residential Racial Segregation And Neighborhood Adversity: Associations With Hemoglobin A1c In Adolescents With Type 1 Diabetes, Zechariah Jean, April Carcone, Deborah Ellis

Medical Student Research Symposium

Black adolescents with Type 1 Diabetes (T1D) are at increased risk for disparities in health outcomes. More research is necessary on the effects of neighborhood characteristics such as residential racial segregation (RRS) and neighborhood adversity on health.

149 Black youth with T1D were recruited from seven pediatric diabetes clinics in Detroit and Chicago to participate in a clinical trial to improve diabetes management. RRS was calculated at the census block group level based on US census data using Location Quotients. LQs represented the ratio of patients to total population in the block group compared to the same ratio in the …

Assessing Accessibility And Equity Of Autism Spectrum Disorder Screening Tools, Kiernan S. Bloye, Joseph Aderemi Md, Roman Grossi, Tessa Hampton, Melissa Maye Phd

Medical Student Research Symposium

Introduction: Disparities to accessing autism spectrum disorder (ASD) screening tools have been demonstrated across race, ethnicity, socioeconomic status, and language. Additionally, there are concerns that existing screening tools are not as accurate for minoritized individuals. Thus, it is likely that level 1 screening tools must be adapted and developed for, and with, marginalized populations in the United States to promote increased levels of universal screening access and accuracy among minoritized populations.

Methods: Using the PRISMA scoping review checklist, an initial search produced 1880 unique records. Following title and abstract review, 35 articles met criteria for full manuscript review. Finally, 7 …

Trauma-Informed And Family-Centered Paediatric Resuscitation: Defining Domains And Practices, Nancy Kassam-Adams, Lucas Butler, Julia Price, Marcie Gawel, Leila Graham, Sage Myers, Marc Auerbach

Department of Pediatrics Faculty Papers

Aim

For paediatric patients and families, resuscitation can be an extremely stressful experience with significant medical and psychological consequences. Psychological sequelae may be reduced when healthcare teams apply patient- and family-centered care and trauma-informed care, yet there are few specific instructions for effective family-centered or trauma-informed behaviours that are observable and teachable. We aimed to develop a framework and tools to address this gap.

Methods

We reviewed relevant policy statements, guidelines, and research to define core domains of family-centered and trauma-informed care, and identified observable evidence-based practices in each domain. We refined this list of practices via review of provider/team …

Scurvy, Abnormal Mri, And Gelatinous Bone Marrow In An Adolescent With Avoidant Restrictive Food Intake Disorder, Ginny Claire Kim, Asha M Davidson, Rebecca M Beyda, Mona A Eissa

Faculty, Staff and Student Publications

BACKGROUND: Although medical literature describes pediatric scurvy as "rare", a growing number of case reports suggests otherwise. Patients often undergo costly and unnecessary workup due to unfamiliarity with the presentation of scurvy. This case report further supports the small yet growing literature documenting scurvy and its manifestations in patients with eating disorders.

CASE PRESENTATION: A 15-year-old female presented to the emergency department with bilateral knee and ankle swelling and pain in the setting of chronic lower limb rash and BMI of 16.3. For years, she had restricted her diet to carbohydrates. Exam showed perifollicular petechial hemorrhagic rash with corkscrew hairs, …

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

Manuscripts, Articles, Book Chapters and Other Papers

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 …

Minimalistic Approach To Enhanced Recovery After Pediatric Scoliosis Surgery, Scott A. Barnett, Bryant M. Song, Matthew Bauer, Matthew E. Nungesser, Claudia Leonardi, Michael J. Heffernan

School of Medicine Faculty Publications

Purpose: Prior studies of enhanced recovery protocols (ERP) have been conducted at large institutions with abundant resources. These results may not apply at institutions with less resources directed to quality improvement efforts. The purpose of this study was to assess the value of a minimalistic enhanced recovery protocol in reducing length of stay (LOS) following PSF for adolescent idiopathic scoliosis. We hypothesized that accelerated transition to oral pain medications and mobilization alone could shorten hospital length of stay in the absence of a formal multimodal pain regimen. Methods: AIS patients aged 10–18 who underwent PSF at a tertiary pediatric hospital …

Cognitive Sparing In Proton Versus Photon Radiotherapy For Pediatric Brain Tumor Is Associated With White Matter Integrity: An Exploratory Study, Lisa E Mash, Lisa S Kahalley, Kimberly P Raghubar, Naomi J Goodrich-Hunsaker, Tracy J Abildskov, Luz A De Leon, Marianne Macleod, Heather Stancel, Kelley Parsons, Brian Biekman, Nilesh K Desai, David R Grosshans, Arnold C Paulino, Zili D Chu, William E Whitehead, Mehmet Fatih Okcu, Murali Chintagumpala, Elisabeth A Wilde

Faculty, Staff and Student Publications

Radiotherapy for pediatric brain tumors is associated with reduced white matter structural integrity and neurocognitive decline. Superior cognitive outcomes have been reported following proton radiotherapy (PRT) compared to photon radiotherapy (XRT), presumably due to improved sparing of normal brain tissue. This exploratory study examined the relationship between white matter change and late cognitive effects in pediatric brain tumor survivors treated with XRT versus PRT. Pediatric brain tumor survivors treated with XRT (

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

Manuscripts, Articles, Book Chapters and Other Papers

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 …