Pediatrics Commons^™

Prospective Analysis Of Liver Stiffness Measurement By Vibration Controlled Transient Elastography As A Predictor Of Outcomes In Biliary Atresia, Jean P Molleston, Nathan P Goodrich, Wen Ye, Daniel H Leung, Ronald J Sokol, Benjamin L Shneider, Children Force Outcomes Working Group

Faculty, Staff and Students Publications

No abstract provided.

American Cochlear Implant Alliance Position Statement On Newborn Congenital Cytomegalovirus Screening, Megan Honor Pesch, Kevin David Brown, Amy L Birath, Gail J Demmler-Harrison, Cailtin Sapp, Anne Morgan Selleck, Alex D Sweeney

Faculty, Staff and Students Publications

It is estimated that 1 in every 200 US newborns has congenital cytomegalovirus (cCMV). Delayed identification of cCMV in newborns precludes timely intervention to mitigate sequelae of the infection such as hearing loss and other neurological complications. Newborn testing for cCMV enables appropriate diagnosis and intervention by multidisciplinary teams to properly manage the immediate sequelae of cCMV, avoid unnecessary additional testing that can result from delayed diagnosis, and monitor for future complications. It is the position of the American Cochlear Implant Alliance, the National CMV Foundation, and the American Academy of Otolaryngology-Head and Neck Surgery that universal newborn cCMV screening …

Sudden Cardiac Death Associated With Fatty Liver Disease, Jonathan Vo, Thien T T T Truyen, Audrey Uy-Evanado, Arayik Sargsyan, Harpriya Chugh, Christopher Young, Sean Hurst, Christina Y Miyake, Kyndaron Reinier, Sumeet S Chugh

Faculty, Staff and Students Publications

BACKGROUND: Fatty liver disease or steatotic liver disease (SLD) affects 25% of the global population and has been associated with heart disease. However, there is a lack of postmortem studies in the context of sudden cardiac death (SCD).

OBJECTIVES: To investigate the relationship between SLD and SCD.

METHODS: A post-mortem case-case study was conducted in victims of SCD from an ongoing community-based study in Southern California (Ventura, CA, 2015-2023). Diagnosis of SLD was determined from post-mortem liver histopathology reports. For each patient, demographic variables, laboratory values, and presence of co-morbidities were ascertained from medical records and were compared between patients …

Management Of Children With Food Allergies By Allergists In The United States, Aikaterini Anagnostou, Matthew Greenhawt, Jay A Lieberman, Christina E Ciaccio, Sayantani B Sindher, Blaine Creasy, Katherine Baran, Sachin Gupta, Anna Nowak-Wegrzyn

Faculty, Staff and Students Publications

BACKGROUND: Management of patients with food allergies is complex, especially in cases of patients with multiple and potentially severe food allergies. Although international guidelines exist for food allergy management, the role of the allergist in the decision-making process is key.

OBJECTIVE: Our aim was to investigate the management patterns and educational needs of practicing allergists treating patients with food allergies.

METHODS: An online survey was e-mailed to United States-based practicing allergists (N = 2833) in November-December 2021. The allergists were screened for managing 1 or more patients (including ≥25% pediatric patients) with food allergies per month. The allergists responded to …

Effectiveness Of The Primary Bacillus Calmette-Guérin Vaccine Against The Risk Of Mycobacterium Tuberculosis Infection And Tuberculosis Disease: A Meta-Analysis Of Individual Participant Data, Puck T Pelzer, Logan Stuck, Leonardo Martinez, Alexandra S Richards, Carlos Acuña-Villaorduña, Naomi E Aronson, Maryline Bonnet, Anna C Carvalho, Pei-Chun Chan, Li-Min Huang, Chi-Tai Fang, Gavin Churchyard, Helena Del Corral-Londoño, Manjula Datta, Marcos A Espinal, Katherine Fielding, Andrew J Fiore-Gartland, Alberto Garcia-Basteiro, Willem Hanekom, Mark Hatherill, Phillip C Hill, Helena Huerga, Edward C Jones-López, Afranio Kritski, Anna M Mandalakas, Punam Mangtani, Eduardo Martins Netto, Harriet Mayanja, Rufaida Mazahir, Megan Murray, Molebogeng Rangaka, Thomas Scriba, Jitendra Singh, Sarman Singh, Catherine M Stein, Johan Vekemans, Lilly M Verhagen, Julian A Villalba, Anne Wajja, Basilea Watson, Richard G White, Frank G J Cobelens

Faculty, Staff and Students Publications

Background: Tuberculosis vaccine trials using disease as the primary endpoint are large, time consuming, and expensive. An earlier immunological measure of the protection against disease would accelerate tuberculosis vaccine development. We aimed to assess whether the effectiveness of the Bacillus Calmette-Guérin (BCG) vaccine for prevention of Mycobacterium tuberculosis infection was consistent with that for prevention of tuberculosis disease.

Methods: We conducted an individual participant data (IPD) meta-analysis on experimental and observational longitudinal studies before April 6, 2018, identified through systematic reviews, known to us through expert knowledge in the field, reporting on BCG vaccination status, M tuberculosis infection test (QuantiFERON …

Diagnostic Performance Of Contrast-Enhanced Ultrasound In Traumatic Solid Organ Injuries In Children: A Systematic Review And Meta-Analysis, Payam Jannatdoust, Parya Valizadeh, Amir Hassankhani, Melika Amoukhteh, Delaram J Ghadimi, Mahsa Heidari-Foroozan, Paniz Sabeghi, Paniz Adli, Jennifer H Johnston, Pauravi S Vasavada, Ali Gholamrezanezhad

Faculty, Staff and Student Publications

Background: Blunt abdominal trauma (BAT) is a significant contributor to pediatric mortality, often causing liver and spleen injuries. Contrast-enhanced computed tomography (CT), the gold standard for diagnosing solid organ injury, poses radiation risks to children. Contrast-enhanced ultrasound (CEUS) may be a promising alternative imaging modality.

Objectives: To evaluate the diagnostic utility of CEUS for detecting solid organ injuries following BAT in the pediatric population.

Methods: A systematic review and meta-analysis were conducted through a thorough literature search in PubMed, Scopus, Web of Science, and Embase databases up to October 1, 2023. Diagnostic accuracy metrics were aggregated using a bivariate model, …

A Guide To Diagnosis And Management Of Hypertrichosis, Rahib K. Islam, Victoria T. Tong, Shari R. Lipner

School of Medicine Faculty Publications

Hypertrichosis is a phenotype characterized by excessive hair growth in areas not typically prone to dense hair coverage. It may be congenital or acquired, and has significant clinical and psychosocial implications, often leading to psychological distress and social stigmatization. Diagnostic approaches emphasize the importance of thorough clinical evaluation and differentiation from hirsutism. Treatment options include topical and systemic treatments, as well as mechanical methods and laser therapies. Special considerations are given to pediatric patients regarding the psychosocial aspects of the phenotype. This review aims to provide physicians and researchers with a thorough understanding of hypertrichosis to facilitate better diagnosis, treatment, …

Routine Overnight Assessments In Stable Pediatric Surgery Patients: A Critical Reconsideration, Morgan Brown, Rebecca Moreci, Hannah Megison, Elizabeth Long, Michael Maurer, Benjamin Bienvenue, Denise M. Danos, James H. Wood

School of Medicine Faculty Publications

Background: Hospitalized patients are subject to overnight vital sign (OVS) monitoring which leads to subsequent sleep disturbance and contributes to adverse outcomes and negative hospital experiences. Studies in pediatric populations have shown that routine OVS checks infrequently detect significant events. We hypothesized that OVS monitoring in pediatric surgery patients rarely detects abnormalities resulting in meaningful interventions. Materials and methods: We performed a retrospective chart review of patients ≥ 5 years old admitted to the pediatric surgery service at a stand-alone Children's Hospital from 2019 to 2021. ICU patients were excluded from analysis. Overnight vital signs were defined as those recorded …

Interventions To Prevent And Manage Infections In Pregnancy, Rahima Yasin, Li Jiang, Jai K. Das, Zulfiqar Ahmed Bhutta

Institute for Global Health and Development

Background: Care interventions aimed at preventing and treating maternal infections during the gestational period are of paramount importance. Timely immunizations, screening strategies, and management of maternal infections reduce the risk of complications for the developing fetus and play a pivotal role in improving neonatal outcomes.
Summary: We aim to summarize evidence for a total of thirteen interventions, pertaining to the prevention and treatment of maternal infections during the antenatal period, from Every Newborn Series published in The Lancet 2014. We identified the most recent systematic reviews, extracted data from each review, and conducted a sub-group meta-analysis for low-income countries and …

Sex-Specific Astrocyte Regulation Of Spinal Motor Circuits By Nkx61, Navish A Bosquez Huerta, Zhung-Fu Lee, Eun-Ah Christine Song, Junsung Woo, Yi-Ting Cheng, Debosmita Sardar, Ozlem Sert, Ehson Maleki, Kwanha Yu, Ekin Su Akdemir, Kaitlyn Sanchez, Juyeon Jo, Matthew N Rasband, Hyun Kyoung Lee, Akdes Serin Harmanci, Benjamin Deneen

Faculty, Staff and Students Publications

Astrocytes exhibit diverse cellular and molecular properties across the central nervous system (CNS). Recent studies identified region-specific transcription factors (TF) that oversee these diverse properties; how sex differences intersect with region-specific transcriptional programs to regulate astrocyte function is unknown. Here, we show that the TF Nkx6.1 is specifically expressed in ventral astrocytes of the spinal cord and that its deletion results in sex-specific effects on astrocyte morphology. Astrocytes from males exhibit enhanced morphological complexity, accompanied by increased motor function and cholinergic synapses. In contrast, female astrocytes exhibit reduced complexity and no changes in motor function. Mechanistically, we found that Nkx6.1 …

The Adiponectin-Pparγ Axis In Hepatic Stellate Cells Regulates Liver Fibrosis, Shangang Zhao, Qingzhang Zhu, Wang-Hsin Lee, Jan-Bernd Funcke, Zhuzhen Zhang, May-Yun Wang, Qian Lin, Bianca Field, Xue-Nan Sun, Guannan Li, Mbolle Ekane, Toshiharu Onodera, Na Li, Yi Zhu, Christine M Kusminski, Terry D Hinds, Philipp E Scherer

Faculty, Staff and Students Publications

Hepatic stellate cells (HSCs) are key drivers of local fibrosis. Adiponectin, conventionally thought of as an adipokine, is also expressed in quiescent HSCs. However, the impact of its local expression on the progression of liver fibrosis remains unclear. We recently generated a transgenic mouse line (Lrat-rtTA) that expresses the doxycycline-responsive transcriptional activator rtTA under the control of the HSC-specific lecithin retinol acyltransferase (Lrat) promoter, which enables us to specifically and inducibly overexpress or eliminate genes in these cells. The inducible elimination of HSCs protects mice from methionine/choline-deficient (MCD) diet-induced liver fibrosis, confirming their causal involvement in fibrosis development. We generated …

Virginia Academy Of Sleep Medicine Position Statement On School Start Times In Virginia, Sean Hesselbacher, Mariana Szklo-Coxe, Sachin Relia, Jonathan Gellman, Nicole Mcintyre

Eastern Virginia Medical Journal

The VASM endorses the 2017 AASM position statement that middle and high schools should begin no earlier than 8:30 a.m. The existing and updated scientific literature repeatedly identifies the detrimental impact of early high school start times, and the benefits of delaying start times, on the safety and well-being of students in Virginia. The adverse effects of early school start times on Virginia’s students have been demonstrated, with regards to academic performance, mental health, and driving risks. Data published since the AASM position paper highlight these dangers further. Importantly, recent studies also demonstrate some of the benefits that can come …

Social, Racial, And Economic Disparities Affecting Outcomes Of Hypertensive Adolescents, Kanya Singhapakdi, Amelia Haydel, Marla Johnston, Shengping Yang, Tamara Bradford, Dedrick Moulton, Thomas R. Kimball

School of Medicine Faculty Publications

Essential hypertension is one of the most common conditions managed in pediatric cardiology and can result in lasting deleterious effects on the cardiovascular system. Pediatric hypertension is so prevalent in the United States that it is often referred to as a public health challenge. Social determinants of health (SDH) are the cultural, economic, educational, healthcare accessibility, and political influences in the environment in which an individual is born or lives, all of which can affect that individual's overall health. This study investigated the impact of social determinants such as rurality, food insecurity, transportation challenges, minority status, income, and race on …

Ethnic Disparities In Hba1c And Hypoglycemia Among Youth With Type 1 Diabetes: Beyond Access To Technology, Social Deprivation And Mean Blood Glucose, J. Pemberton, Zhide Fang, Stuart Chalew, S. Uday

School of Public Health Faculty Publications

Introduction The UK national pediatric diabetes audit reports higher HbA1c for children and young people (CYP) with type 1 diabetes (T1D) of Black ethnicity compared with White counterparts. This is presumably related to higher mean blood glucose (MBG) due to lower socioeconomic status (SES) and less access to technology. We aimed to determine if HbA1c ethnic disparity persists after accounting for the above variables. Research design and methods A retrospective analysis of participants who received structured education in continuous glucose monitoring (CGM) use was conducted at a tertiary center. HbA1c was paired with glucose metrics from 90-day CGM data. The …

A Randomized, Placebo-Controlled, Cross-Over Trial Of Ketamine In Rett Syndrome, Kathleen Campbell, Jeffrey L Neul, David N Lieberman, Elizabeth Berry-Kravis, Tim A Benke, Cary Fu, Alan Percy, Bernhard Suter, David Morris, Randall L Carpenter, Eric D Marsh, Jana Von Hehn

Faculty, Staff and Students Publications

BACKGROUND: Preclinical studies and anecdotal case reports support the potential therapeutic benefit of low-dose oral ketamine as a treatment of clinical symptoms in Rett syndrome (RTT); however, no controlled studies have been conducted in RTT to evaluate safety, tolerability and efficacy.

DESIGN: This was a sequentially initiated, dose-escalating cohort, placebo-controlled, double blind, randomized sequence, cross-over study of oral ketamine in 6-12-year-old girls with RTT to evaluate short-term safety and tolerability and explore efficacy.

METHODS: Participants were randomized to either five days treatment with oral ketamine or matched placebo, followed by a nine-day wash-out period and then crossed-over to the opposite …

Are Maternal Adverse Childhood Experiences (Aces) And Resilience Associated With Breastfeeding Rates And Neonatal Growth?, Lily Zheng, Krystal Hunter, Hsiao-Wei M. Banks, Leslie Abraham, Alla Kushnir

Cooper Rowan Medical Journal

Background: Adverse childhood experiences (ACEs) have shown to have a negative impact on health outcomes. There is evidence of how negative childhood events adversely affect self-esteem in adulthood. Not only do ACEs have an impact on one's own life, but maternal ACEs have shown to have consequences on infants’ development as well, indicating a negative intergenerational impact.

Objectives: To analyze the relationship between maternal ACEs on infant outcomes, breastfeeding adherence, and maternal self-perception

Design/Methods: Between 2020 and 2023, 120 mother-infant pairs from a New Jersey urban hospital were approached between their 2nd trimester and the 2-month well …

Using Community Engagement To Create A Telecoaching Intervention To Improve Self-Management In Adolescents And Young Adults With Cystic Fibrosis: Qualitative Study., Christina L. Duncan, Emily F. Muther, Jennifer J. Lindwall, Kristine Durkin, Elizabeth Ruvalcaba, Eliza Williamson, Corrine Ahrabi-Nejad, Evelyn Bord, Angela Green, Megan L. Harrison, Deepika Polineni

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Adolescents and young adults (AYA) with cystic fibrosis (CF) are at risk for deviating from their daily treatment regimen due to significant time burden, complicated daily therapies, and life stressors. Developing patient-centric, effective, engaging, and practical behavioral interventions is vital to help sustain therapeutically meaningful self-management.

OBJECTIVE: This study aimed to devise and refine a patient-centered telecoaching intervention to foster self-management in AYA with CF using a combination of intervention development approaches, including an evidence- and theory-based approach (ie, applying existing theories and research evidence for behavior change) and a target population-centered approach (ie, intervention refinement based on the …

Management And Outcome Of Invasive Clindamycin-Resistant Mrsa Community-Associated Infections In Children, Amanda E Macias, Grant Stimes, Sheldon L Kaplan, Jesus G Vallejo, Kristina G Hulten, J Chase Mcneil

Faculty, Staff and Students Publications

Background: Clindamycin resistance among community-associated methicillin-resistant Staphylococcus aureus (CA-MRSA) complicates the management of a challenging infection. Little data exist to guide clinicians in the management of invasive clindamycin-resistant CA-MRSA infections in children and studies using oral regimens such as trimethoprim-sulfamethoxazole (TMP-SMX) and linezolid for treatment of these infections are limited. We sought to reevaluate antibiotic management among invasive CA-MRSA at a tertiary children’s hospital.

Methods: Cases of invasive clindamycin-resistant MRSA infections in children were identified through an ongoing S. aureus surveillance study. Eligible cases were those occurring in otherwise healthy children from 2011–2021. Medical records were reviewed.

Results: Thirty-four subjects …

Resurgence Of Pediatric Mycoplasma Pneumoniae Infections In Southeast Texas, November 2023-June 2024, Margaret Taylor Danner, Heather Colvin Binns, Khanh Nguyen, Coreen Johnson, Jim Dunn, Denver Niles, Diana K Nguyen

Faculty, Staff and Students Publications

We conducted a retrospective review of children tested for Mycoplasma pneumoniae from January 1, 2020 to June 30, 2024. M. pneumoniae polymerase chain reaction positivity increased starting in November 2023, peaking at 18.3% in June 2024. During the resurgence, children with laboratory-confirmed infection often had severe respiratory disease or extrapulmonary manifestations.

Prevalence Of Flat Foot Among School-Age Girls And Its Relationship With Body Mass Index And Activity Level, Hisham Mohamed Hussein, Ehab Kamel, Walid Mohamed Kamel, Ibrahim Metwally Dewir, Ahmed Abdelmoniem Ibrahim

Internet Journal of Allied Health Sciences and Practice

Background: The foot and ankle are important structures in the skeletal system of the human body. Deviation in normal structure of the foot,such as in flat foot deformity, can lead to many health problems. Our study aimed to explore the prevalence of flexible flat foot (FFF) among school-age female students in the Ha’il region, Saudi Arabia, and to assess the association between the body mass index (BMI), activity level, and FFF in the selected samples. Materials and Methods: This cross-sectional study included 543 female students between 7 and 12 years of age. Data regarding age, height, weight, level …

Examining The Impact Of An Education Intervention On Parents' Perceptions Of Sport Specialization, Lauren Schlacht Butler, Justin S. Disanti, Joseph J. Janosky, Jane S. Chung, James F. Russomano, Amanda M. Black, Shelly Fetchen Dicesaro, Eric Post, Jonathan A. Santana, Troy M. Smurawa, Tamara Valovich Mcleod

Internet Journal of Allied Health Sciences and Practice

PURPOSE: Several organizations have developed educational materials to provide stakeholders with information about the health risks of early sport specialization (ESS), though the impact of these efforts is not yet known. Therefore, the purpose of this study was to describe and assess the effect of an educational intervention on parental knowledge and perceptions of ESS. We hypothesized that a video-based educational intervention would increase parental knowledge and change perceptions of ESS. METHODS: Eighty-three parents of youth athletes completed a web-based survey. Participants were randomized into an intervention (ESS video) or control (concussion video) group. Each group completed a pre-and post-intervention …

Congenital Rickets, Melissa Intriago

Mako: NSU Undergraduate Student Journal

No abstract provided.

Sex Differences In Response To Diet Enriched With Glutathione Precursors In The Aging Heart, Aude Angelini, Grecia Garcia Marquez, Anna Malovannaya, Marta L Fiorotto, Alexander Saltzman, Antrix Jain, Joann Trial, George E Taffet, Katarzyna A Cieslik

Faculty, Staff and Students Publications

Common features of the aging heart are dysregulated metabolism, inflammation, and fibrosis. Elevated oxidative stress is another hallmark of cardiac aging that can exacerbate each of these conditions. We hypothesize that by increasing natural antioxidant levels (glutathione), we will improve cardiac function. Twenty-one-month-old mice were fed glycine and N-acetyl cysteine (GlyNAC; glutathione precursors)-supplemented or control diets for 12 weeks. Heart function was monitored longitudinally, and the exercise performance was determined at the end of the study. We found that the GlyNAC diet was beneficial for old male but not old female mice, leading to an increase of Ndufb8 expression (a …

Synesis As A Framework To Enable Safety Interventions In Complex Healthcare Environments, Aarti Bavare, Tiffany Wrenn, Anne Lam, Jamie Cargill, Lauren Salinas, Itode Idowu, Asma Razavi, Venessa Lynn Pinto, Eric Williams

Faculty, Staff and Students Publications

BACKGROUND: Despite wide adoption in the healthcare of safety event report (SER) systems, there is a paucity of unified structures for prompt analysis and action while retaining reporter confidentiality. We used a synesis framework to change siloed reviews of safety reports to a comprehensive appraisal of quality, safety, productivity and reliability to facilitate interventions.

METHODS: After a needs assessment survey, we launched serial plan-do-study-act cycles to (1) enhance teams' ability to access SERs, (2) facilitate regular multidisciplinary review of SERs to identify actionable opportunities, (3) allocate action priority using failure mode and effects analysis, and (4) launch actions and summarise …

Dna Methylation Profile In Buffy Coat Identifies Methylation Differences Between Cirrhosis With And Without Hepatocellular Carcinoma, Hyeyeun Lim, Hashem B El-Serag, Michelle Luster, Megan L Grove, Jinyoung Byun, Yuri Jung, Younghun Han, Eric Boerwinkle, Christopher I Amos, Aaron P Thrift

Faculty, Staff and Students Publications

Background/Objectives: Cirrhosis is the precursor to most cases of hepatocellular carcinoma (HCC). Understanding the mechanisms leading to the transition from cirrhosis to HCC and identifying key biomarkers is crucial to developing effective screening strategies and reducing HCC-related mortality. DNA methylation is associated with gene inactivation and plays an important role in physiological and pathological processes; however, its role in cirrhosis progression to HCC is unknown.

Methods: We performed genome-wide DNA methylation profiling using Illumina Infinium MethylationEPI BeadChip in pre-diagnostic samples from 22 cirrhosis patients who subsequently developed HCC and 22 cirrhosis patients who remained HCC-free during an average 4-year follow-up. …

Pre-Emptive Treatment For Cytomegalovirus Viraemia To Prevent Cytomegalovirus Disease In Solid Organ Transplant Recipients, Robin Wm Vernooij, Mini Michael, Julia Mt Colombijn, Daniel S Owers, Angela C Webster, Giovanni Fm Strippoli, Elisabeth M Hodson

Faculty, Staff and Students Publications

Background: Cytomegalovirus (CMV) is a significant cause of morbidity and death in solid organ transplant recipients. Pre-emptive treatment of patients with CMV viraemia using antiviral agents has been suggested as an alternative to routine prophylaxis to prevent CMV disease. This is an update of a Cochrane review first published in 2006 and updated in 2013.

Objectives: To determine the benefits and harms of pre-emptive treatment of CMV viraemia to prevent CMV disease and death (any cause) and the indirect effects of CMV infection (acute rejection, graft loss, opportunistic infections) in solid organ transplant recipients.

Search methods: The Cochrane Kidney and …

Identification And Characterization Of A Novel Pavlovskyella (Acari: Argasidae) From Chile, Parasite Of The Culpeo Fox (Lycalopex Culpaeus), Sebastián Muñoz-Leal, Alexander R Kneubehl, Karen Ardiles, Catalina Parragué-Migone, Ignacio Troncoso-Toro, Pablo Oyarzún-Ruiz, Job E Lopez

Faculty, Staff and Students Publications

Soft ticks (Argasidae) of the subgenus Pavlovskyella Pospelova-Shtrom are worldwide distributed parasites of medical importance. However, the systematics of the subgenus are currently under debate because genetic data shows that the group is paraphyletic. Meanwhile, species of Pavlovskyella continue to be discovered. In this study a novel species of the subgenus is described from specimens collected on a fox in central Chile. The larva of this new species differentiates from other Pavlovskyella spp. by having the following combination of characters: subpyriform dorsal plate; 15 pairs of setae, 7 anterolateral, 3 central, and 5 posterolateral, and hypostome with denticles in the …

Hyperleukocytosis In A Neuroblastoma Patient After Treatment With Natural Killer T Cells Expressing A Gd2-Specific Chimeric Antigen Receptor And Il-15, Gengwen Tian, Amy N Courtney, Hangjin Yu, Saleh Bhar, Xin Xu, Gabriel A Barragán, Claudia Martinez Amador, Nisha Ghatwai, Michael S Wood, Deborah Schady, Antonino Montalbano, Shantan Reddy, Aoife M Roche, David De La Cerda, Donald Williams Parsons, Erica J Di Pierro, Frederic D Bushman, Andras Heczey, Leonid S Metelitsa

Faculty, Staff and Students Publications

The ability of immune cells to expand numerically after infusion distinguishes adoptive immunotherapies from traditional drugs, providing unique therapeutic advantages as well as the potential for unmanageable toxicities. Here, we describe a case of lethal hyperleukocytosis in a patient with neuroblastoma treated on phase 1 clinical trial (NCT03294954) with autologous natural killer T cells (NKTs) expressing a GD2-specific chimeric antigen receptor and cytokine interleukin 15 (GD2-CAR.15). This patient was the first to be treated on dose level (DL) 5 and the first patient whose product was restimulated with K562-derived artificial antigen-presenting cells (aAPCs) instead of autologous peripheral blood mononuclear cells …

Attributes Of Pediatric Music Therapists: A Qualitative Analysis Of Parent Descriptions., Elizabeth Harman, Kristin Stegenga, Sheri L. Robb

Manuscripts, Articles, Book Chapters and Other Papers

Therapist attributes are known to contribute to positive therapeutic outcomes and are important to effective training and clinical practice. Although well researched in psychology and play therapy, few studies have directly explored music therapist attributes. To explore and understand these descriptions, we conducted a secondary analysis of parent interview data from a multisite trial investigating a music therapist-delivered intervention for young children with cancer and their parents. We used qualitative descriptive analysis to identify music therapists' attributes as described by parents who participated in the intervention. Our inductive analysis of 28 interviews revealed 135 descriptors of music therapists. We discovered …

Advancing Monogenic Diabetes Research And Clinical Care By Creating A Data Commons: The Precision Diabetes Consortium (Predict), Michael E Mccullough, Lisa R Letourneau-Freiberg, Rochelle N Naylor, Siri Atma W Greeley, David T Broome, Mustafa Tosur, Raymond J Kreienkamp, Erin Cobry, Neda Rasouli, Toni I Pollin, Miriam S Udler, Liana K Billings, Cyrus Desouza, Carmella Evans-Molina, Suzi Birz, Brian Furner, Michael Watkins, Kaitlyn Ott, Samuel L Volchenboum, Louis H Philipson

Faculty, Staff and Students Publications

Monogenic diabetes mellitus (MDM) is a group of relatively rare disorders caused by pathogenic variants in key genes that result in hyperglycemia. Lack of identified cases, along with absent data standards, and limited collaboration across institutions have hindered research progress. To address this, the UChicago Monogenic Diabetes Registry (UCMDMR) and UChicago Data for the Common Good (D4CG) created a national consortium of MDM research institutions called the PREcision DIabetes ConsorTium (PREDICT). Following the D4CG model, PREDICT has successfully established a multicenter MDM data commons. PREDICT has created a consensus data dictionary that will be utilized to address critical gaps in …