Pediatrics Commons^™

A Previously Healthy Infant With Lemierre Syndrome In The Emergency Department: Case Report, Adeola Adekunbi Kosoko, Omoyeni O Clement

Faculty, Staff and Student Publications

INTRODUCTION: Lemierre syndrome (LS) is a rare condition with a high mortality risk. It is well described in older children and young adults involving bacteremia, thrombophlebitis, and metastatic abscess commonly due to Fusobacterium infections. Young, pre-verbal children are also susceptible to LS; thus, careful attention must be given to their pattern of symptoms and history to identify this condition in the emergency department (ED).

CASE REPORT: A 12-month-old previously healthy boy with a recent diagnosis of acute otitis media and viral illness presented to the ED with a complaint of fever. Additional symptoms developed at the head and neck and …

Worldwide Exploration Of Renal Replacement Outcomes Collaborative In Kidney Disease (We-Rock), Shina Menon, Kelli A Krallman, Ayse A Arikan, Dana Y Fuhrman, Stephen M Gorga, Theresa Mottes, Nicholas Ollberding, Zaccaria Ricci, Natalja L Stanski, David T Selewski, Danielle E Soranno, Michael Zappitelli, Huaiyu Zang, Katja M Gist

Faculty, Staff and Students Publications

INTRODUCTION: Continuous renal replacement therapy (CRRT) is used for the symptomatic management of acute kidney injury (AKI) and fluid overload (FO). Contemporary reports on pediatric CRRT are small and single center in design. Large international studies evaluating CRRT practice and outcomes are lacking. Herein, we describe the design of a multinational collaborative.

METHODS: The Worldwide Exploration of Renal Replacement Outcomes Collaborative in Kidney Disease (WE-ROCK) is an international collaborative of pediatric specialists whose mission is to improve short- and long-term outcomes of children treated with CRRT. The aims of this multicenter retrospective study are to describe the epidemiology, liberation patterns, …

Two Pediatric Cases Within A Familial Cluster And Hereditary Diffuse Gastric Carcinoma: A Tale Of 2 Sisters, Lacey Falgout, Lawrence L. Gensler

School of Medicine Faculty Publications

Hereditary diffuse gastric cancer is a familial form of poorly differentiated signet ring cell carcinoma (SRCC) caused by a mutation in the CDH1/E-cadherin gene-mediating cell adhesion. CDH1 mutations are inherited in an autosomal dominant fashion and exhibit a high level of penetrance. SRCC diagnosis is exceedingly rare in the pediatric population. We report a case of SRCC in 2 sisters (10 and 15 years) and their father (41 years).

Screening For Food Insecurity In A Suburban Pediatric Urgent Care, Rachel Foutz

Dissertations

Problem: Screening for Social Determinants of Health (SDOH), including food insecurity (FI) should be done on a regular basis at a child’s pediatrician. Many children in the state of Missouri do not have a pediatrician or do not regularly see their pediatrician and are therefore never being screened for FI.

Methods: This observational descriptive quality improvement (QI) project utilized a prospective observational analysis to implement a voluntary FI screening tool in a pediatric only urgent care. The project sampled pediatric patients aged two days to 21 years of life. All participants were given local resources after completing the screening. The …

Persistent Increase In Serum Ferritin Levels Despite Converting To Permanent Vascular Access In Pediatric Hemodialysis Patients: Pediatric Nephrology Research Consortium Study., Ali Mirza Onder, Md Abu Yusuf Ansari, Fang Deng, Matthew M. Grinsell, Larry Patterson, Jennifer Jetton, Sahar Fathallah-Shaykh, Daniel Ranch, Diego Aviles, Lawrence Copelovitch, Eileen Ellis, Vimal Chadha, Ayah Elmaghrabi, Jen-Jar Lin, Lavjay Butani, Maha Haddad, Olivera Marsenic, Paul Brakeman, Raymond Quigley, H Stella Shin, Rouba Garro, Rupesh Raina, Craig B. Langman

Manuscripts, Articles, Book Chapters and Other Papers

Our objective was to examine serum ferritin trends after conversion to permanent vascular access (PVA) among children who started hemodialysis (HD) using tunneled cuffed catheters (TCC). Retrospective chart reviews were completed on 98 subjects from 20 pediatric HD centers. Serum ferritin levels were collected at the creation of PVA and for two years thereafter. There were 11 (11%) arteriovenous grafts (AVG) and 87 (89%) arteriovenous fistulae (AVF). Their mean TCC use was 10.4 ± 17.3 months. Serum ferritin at PVA creation was elevated at 562.64 ± 492.34 ng/mL, increased to 753.84 ± 561.54 ng/mL (p = < 0.001) in the first year and remained at 759.60 ± 528.11 ng/mL in the second year (p = 0.004). …

Persistent Increase In Serum Ferritin Levels Despite Converting To Permanent Vascular Access In Pediatric Hemodialysis Patients: Pediatric Nephrology Research Consortium Study, Ali Mirza Onder, Md Abu Yusuf Ansari, Fang Deng, Matthew M. Grinsell, Larry Patterson, Jennifer Jetton, Sahar Fathallah-Shaykh, Daniel Ranch, Diego Aviles, Lawrence Copelovitch, Eileen Ellis, Vimal Chadha, Ayah Elmaghrabi, Jen-Jar Lin, Lavjay Butani, Maha Haddad, Olivera Marsenic, Paul Brakeman, Raymond Quigley, H Stella Shin, Rouba Garro, Rupesh Raina, Craig B. Langman

School of Medicine Faculty Publications

Our objective was to examine serum ferritin trends after conversion to permanent vascular access (PVA) among children who started hemodialysis (HD) using tunneled cuffed catheters (TCC). Retrospective chart reviews were completed on 98 subjects from 20 pediatric HD centers. Serum ferritin levels were collected at the creation of PVA and for two years thereafter. There were 11 (11%) arteriovenous grafts (AVG) and 87 (89%) arteriovenous fistulae (AVF). Their mean TCC use was 10.4 ± 17.3 months. Serum ferritin at PVA creation was elevated at 562.64 ± 492.34 ng/mL, increased to 753.84 ± 561.54 ng/mL (p = < 0.001) in the first year and remained at 759.60 ± 528.11 ng/mL in the second year (p = 0.004). The serum ferritin levels did not show a statistically significant linear association with respective serum hematocrit values. In a multiple linear regression model, there were three predictors of serum ferritin during the first year of follow-up: steroid-resistant nephrotic syndrome as primary etiology (p = 0.035), being from a center that enrolled >10 cases (p = …

Neighborhood Disadvantage And Pediatric Inpatient Opioid Prescription Patterns, Ashley W. Kranjac, Dinko Kranjac, Zeev N. Kain, Louis Ehwerhemuepha, Candice Donaldson, Brooke N. Jenkins

Sociology Faculty Articles and Research

Background

To explore the role of children's residential environment on opioid prescribing patterns in a predominantly Latinx sample.

Methods

We connected geocoded data from electronic medical records in a diverse sample of pediatric patients to neighborhood environments constructed using latent profile modeling techniques. We then estimated a series of multilevel models to determine whether opioid prescribing patterns vary by residential context.

Results

A stepwise pattern exists between neighborhood disadvantage and pediatric opioid prescription patterns, such that higher levels of disadvantage associate with a greater likelihood of opioid prescription, independent of the patient's individual profile.

Conclusion

In a largely Latinx sample …

Promoting Ocd Wellness And Resilience (Power) Study: Rationale, Design, And Methods, Jessica D Leuchter, Minjee Kook, Daniel A Geller, Alyssa G Hertz, Jessica Garcia, Erika S Trent, Tracey Dibbs, Ogechi Onyeka, Wayne K Goodman, Andrew G Guzick, Andrew D Wiese, Amanda D Palo, Brent J Small, H Blair Simpson, Lauren K Havel, Sohail A Nibras, Kirti Saxena, Eric A Storch

Faculty, Staff and Students Publications

Obsessive-compulsive disorder (OCD) affects 1-2% of children and is associated with functional impairment and diminished quality of life. Several treatments are efficacious: cognitive behavioral therapy (CBT) with exposure and response prevention, serotonin reuptake inhibitor (SRI) monotherapy, and combined treatment (SRI + CBT). Expert clinician-informed practice parameters suggest that youth with mild to moderate OCD should be treated initially with CBT yet SRIs are frequently employed as the first-line intervention or in combination with psychotherapy in applied practice. Empirical data to guide SRI discontinuation in pediatric OCD are very limited. This study, Promoting OCD Wellness and Resiliency (POWER), aims to address …

Imaging Of Pediatric Cardiac Tumors: A Cog Diagnostic Imaging Committee/Spr Oncology Committee White Paper, Cara E Morin, Lindsay M Griffin, Rebecca S Beroukhim, Pablo Caro-Domínguez, Sherwin Chan, Jason N Johnson, Juan C Infante, Christopher Z Lam, Ladonna J Malone, Elizabeth R Tang, Michael D Taylor, James C Wilkinson, Prakash M Masand

Faculty, Staff and Students Publications

Cardiac tumors in children are rare and the majority are benign. The most common cardiac tumor in children is rhabdomyoma, usually associated with tuberous sclerosis complex. Other benign cardiac masses include fibromas, myxomas, hemangiomas, and teratomas. Primary malignant cardiac tumors are exceedingly rare, with the most common pathology being soft tissue sarcomas. This paper provides consensus-based imaging recommendations for the evaluation of patients with cardiac tumors at diagnosis and follow-up, including during and after therapy.

Diagnostic Role Of Fractional Exhaled Nitric Oxide In Pediatric Eosinophilic Esophagitis, Relationship With Gastric And Duodenal Eosinophils., Panamdeep Kaur, Rachel Chevalier, Craig A. Friesen, Jamie Ryan, Ashley K. Sherman, Stephanie Page

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Eosinophilic esophagitis (EoE) is an eosinophilic-predominant inflammation of the esophagus diagnosed by upper endoscopy and biopsies. A non-invasive and cost-effective alternative for management of EoE is being researched. Previous studies assessing utility of fractional exhaled nitric oxide (FeNO) in EoE were low powered. None investigated the contribution of eosinophilic inflammation of the stomach and duodenum to FeNO.

AIM: To assess the utility of FeNO as a non-invasive biomarker of esophageal eosinophilic inflammation for monitoring disease activity.

METHODS: Patients aged 6-21 years undergoing scheduled upper endoscopy with biopsy for suspected EoE were recruited in our observational study. Patients on steroids …

Contributions To Auditory System Conduction Velocity: Insights With Multi-Modal Neuroimaging And Machine Learning In Children With Asd And Xyy Syndrome, Jeffrey I. Berman, Luke Bloy, Lisa Blaskey, Carissa R. Jackel, Judith S. Miller, Judith Ross, J. Christopher Edgar, Thimothy P.L. Roberts

Department of Pediatrics Faculty Papers

Introduction: The M50 electrophysiological auditory evoked response time can be measured at the superior temporal gyrus with magnetoencephalography (MEG) and its latency is related to the conduction velocity of auditory input passing from ear to auditory cortex. In children with autism spectrum disorder (ASD) and certain genetic disorders such as XYY syndrome, the auditory M50 latency has been observed to be elongated (slowed).

Methods: The goal of this study is to use neuroimaging (diffusion MR and GABA MRS) measures to predict auditory conduction velocity in typically developing (TD) children and children with autism ASD and XYY syndrome.

Results: Non-linear TD …

Extracorporeal Membrane Oxygenation Characteristics And Outcomes In Children And Adolescents With Covid-19 Or Multisystem Inflammatory Syndrome Admitted To Us Icus, Melania M Bembea, Laura L Loftis, Ravi R Thiagarajan, Cameron C Young, Timothy P Mccadden, Margaret M Newhams, Suden Kucukak, Elizabeth H Mack, Julie C Fitzgerald, Courtney M Rowan, Aline B Maddux, Amanda R Kolmar, Katherine Irby, Sabrina Heidemann, Stephanie P Schwartz, Michele Kong, Hillary Crandall, Kevin M Havlin, Aalok R Singh, Jennifer E Schuster, Mark W Hall, Kari A Wellnitz, Mia Maamari, Mary G Gaspers, Ryan A Nofziger, Peter Paul C Lim, Ryan W Carroll, Alvaro Coronado Munoz, Tamara T Bradford, Melissa L Cullimore, Natasha B Halasa, Gwenn E Mclaughlin, Pia S Pannaraj, Natalie Z Cvijanovich, Matt S Zinter, Bria M Coates, Steven M Horwitz, Charlotte V Hobbs, Heda Dapul, Ana Lia Graciano, Andrew D Butler, Manish M Patel, Laura D Zambrano, Angela P Campbell, Adrienne G Randolph

Faculty, Staff and Students Publications

OBJECTIVES: Extracorporeal membrane oxygenation (ECMO) has been used successfully to support adults with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)-related cardiac or respiratory failure refractory to conventional therapies. Comprehensive reports of children and adolescents with SARS-CoV-2-related ECMO support for conditions, including multisystem inflammatory syndrome in children (MIS-C) and acute COVID-19, are needed.

DESIGN: Case series of patients from the Overcoming COVID-19 public health surveillance registry.

SETTING: Sixty-three hospitals in 32 U.S. states reporting to the registry between March 15, 2020, and December 31, 2021.

PATIENTS: Patients less than 21 years admitted to the ICU meeting Centers for Disease Control criteria …

Multi-Center Retrospective Study Of Children With Sickle Cell Disease Admitted To Pediatric Intensive Care Units In The United States, Nicholas A Ettinger, Danielle Guffey, Shaniqua J Anum, Titilope Fasipe, Julie Katkin, Saleh Bhar, Gladstone Airewele, Arun Saini, Venée N Tubman

Faculty, Staff and Students Publications

Data on outcomes and interventions for children with sickle cell disease (SCD) admitted to a pediatric intensive care units (PICU) are unknown. We provide the first comprehensive multi-center report on PICU interventions associated with death, the need for invasive respiratory support or stroke among critically ill children with SCD. We collected retrospective multi-center cohort data from January 1, 2012 to December 31, 2019 utilizing the Virtual Pediatric Systems, LLC database. We identified 3388 unique children with SCD, accounting for a total of 5264 PICU admissions from 138 PICUs. The overall mortality rate for the PICU admissions cohort was 1.8% (95/5264 …

Children's And Caregivers' Review Of A Guided Imagery Therapy Mobile App Designed To Treat Children With Functional Abdominal Pain Disorders: Leveraging A Mixed Methods Approach With User-Centered Design, John M Hollier, Tiantá A Strickland, C Michael Fordis, Miranda Al Van Tilburg, Robert J Shulman, Debbe Thompson

Faculty, Staff and Students Publications

BACKGROUND: Functional abdominal pain disorders (FAPDs) are highly prevalent and associated with substantial morbidity. Guided imagery therapy (GIT) is efficacious; however, barriers often impede patient access. Therefore, we developed a GIT mobile app as a novel delivery platform.

OBJECTIVE: Guided by user-centered design, this study captured the critiques of our GIT app from children with FAPDs and their caregivers.

METHODS: Children aged 7 to 12 years with Rome IV-defined FAPDs and their caregivers were enrolled. The participants completed a software evaluation, which assessed how well they executed specific app tasks: opening the app, logging in, initiating a session, setting the …

Symptom Management Care Pathway Adaptation Process And Specific Adaptation Decisions, Emily Vettese, Farha Sherani, Allison A. King, Lolie Yu, Catherine Aftandilian, Christina Baggott, Vibhuti Agarwal, Ramamoorthy Nagasubramanian, Kara M. Kelly, David R. Freyer, Etan Orgel, Scott M. Bradfield, Wade Kyono, Michael Roth, Lisa M. Klesges, Melissa Beauchemin, Allison Grimes, George Tomlinson, L. Lee Dupuis, Lillian Sung

School of Medicine Faculty Publications

BACKGROUND: There is substantial heterogeneity in symptom management provided to pediatric patients with cancer. The primary objective was to describe the adaptation process and specific adaptation decisions related to symptom management care pathways based on clinical practice guidelines. The secondary objective evaluated if institutional factors were associated with adaptation decisions. METHODS: Fourteen previously developed symptom management care pathway templates were reviewed by an institutional adaptation team composed of two clinicians at each of 10 institutions. They worked through each statement for all care pathway templates sequentially. The institutional adaptation team made the decision to adopt, adapt or reject each statement, …

Use Of Integrative, Complementary, And Alternative Medicine In Children With Epilepsy: A Global Scoping Review, Zahra Zhu, Daniela Dluzynski, Nouran Hammad, Deepika Pugalenthi, Sarah A. Walser, Rea Mittal, Debopam Samanta, Melanie L. Brown, Ali A. Asadi-Pooya, Angelina Kakooza-Mwesige, Alberto Spalice, Martina Capponi, Alain Lekoubou, Ashutosh Kumar, Sita Paudel, Paul R. Carney, Gayatra Mainali, Sunil Naik

Department of Neurology Faculty Papers

(1) Background: Epilepsy is one of the most common chronic neurological disorders in childhood. Complementary and alternative medicine (CAM) use is highly prevalent in patients with epilepsy. Despite CAM’s widespread and increasing popularity, its prevalence, forms, perceived benefits, and potential risks in pediatric epilepsy are rarely explored. (2) Methods: We performed a scoping review of the available literature on the use of CAM in pediatric epilepsy. (3) Results: Overall, global cross-sectional studies showed a variable degree of CAM usage among children with epilepsy, ranging from 13 to 44% in prevalence. Popular types of CAMs reported were supplements, cannabis products, aromatherapy, …

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), …

Association Of Qaca/B And Smr Carriage With Staphylococcus Aureus Survival Following Exposure To Antiseptics In An Ex Vivo Venous Catheter Disinfection Model, J Chase Mcneil, Lauren M Sommer, Jesus G Vallejo, Kristina G Hulten, Sheldon L Kaplan

Faculty, Staff and Students Publications

Many health care centers have reported an association between Staphylococcus aureus isolates bearing efflux pump genes and an elevated MIC/minimal bactericidal concentration (MBC) to chlorhexidine gluconate (CHG) and other antiseptics. The significance of these organisms is uncertain, given that their MIC/MBC is typically far lower than the CHG concentration in most commercial preparations. We sought to evaluate the relationship between carriage of the efflux pump genes qacA/B and smr in S. aureus and the efficacy of CHG-based antisepsis in a venous catheter disinfection model. S. aureus isolates with and without smr and/or qacA/B were utilized. The CHG MICs were determined. …

Pediatric Consultation-Liaison Psychology: Insights And Lessons Learned During The Covid-19 Pandemic, Nicole M Schneider, Dara M Steinberg, Andrea M Garcia, Jessy Guler, Emily Mudd, A Monica Agoston, Katherine N Schwartzkopf, Kristin A Kullgren, Laura Judd-Glossy

Faculty, Staff and Students Publications

COVID-19 has presented a variety of challenges to the provision of psychology services. In the first month of the pandemic, pediatric consultation-liaison (CL) psychologists reported significant changes in methodology of service delivery (Steinberg et al. in Clin Pract Pediatr Psychol 9:1, 2020). To better understand how and if these changes persisted, as well as other emerging trends, a follow-up study examined changes and challenges six months into the pandemic. An anonymous questionnaire assessed topics related to pediatric CL psychology including practice changes, perception of changes, and institutional support. The questionnaire was sent to the APA Society of Pediatric Society's special …

Prevalence And Predictors Of Using Antibiotics Without A Prescription In A Pediatric Population In The United States, Kiara Olmeda, Barbara W Trautner, Lindsey Laytner, Juanita Salinas, Stephanie Marton, Larissa Grigoryan

Faculty, Staff and Students Publications

Non-prescription antibiotic use (using antibiotics without clinical guidance) increases the risk of the development of antibiotic resistance, adverse drug reactions, and other potential patient harm. Few studies have explored non-prescription use in children in the U.S. From January 2021 to April 2022, a diverse sample of caregivers of children under 18 years were surveyed in English and Spanish at two safety net clinics in Texas. We assessed the prevalence of antibiotic use in children in the previous 12 months, storage of antimicrobials, and intended use of non-prescription antibiotics (professed intention for future non-prescription antibiotic use). We also measured sociodemographic factors, …

Heart Rate Variability (Hrv) Loss And Other Ekg Changes Prior To A Cardiopulmonary Event In Children Admitted To The Icu At A Tertiary Care Children’S Hospital, Sabrina R. Orta, Shreya Chandran, Utpal Bhalala

MEDI 9331 Scholarly Activities Clinical Years

Introduction: HRV and EKG changes prior to cardiopulmonary events (CPEs) occurring in children in intensive care unit (ICU) settings is not well documented. We assess if substantial losses of HRV and/or other EKG changes, if any, may be detectable prior to a CPE occurring in children admitted to the ICU.

Methods: A prospective observational cohort study was conducted in the ICU from January to February of 2023 at Driscoll Children’s Hospital. A “CPE” was defined as a change in the clinical status of a child warranting a cardiopulmonary intervention. Demographic and clinical data from each patient was collected, …

Repurposing Atovaquone As A Therapeutic Against Acute Myeloid Leukemia (Aml): Combination With Conventional Chemotherapy Is Feasible And Well Tolerated, Alexandra Mclean Stevens, Eric S Schafer, Minhua Li, Maci Terrell, Raushan Rashid, Hana Paek, Melanie B Bernhardt, Allison Weisnicht, Wesley T Smith, Noah J Keogh, Michelle C Alozie, Hailey H Oviedo, Alan K Gonzalez, Tamilini Ilangovan, Alicia Mangubat-Medina, Haopei Wang, Eunji Jo, Cara A Rabik, Claire Bocchini, Susan Hilsenbeck, Zachary T Ball, Todd M Cooper, Michele S Redell

Center for Medical Ethics and Health Policy Staff Publications

Survival of pediatric AML remains poor despite maximized myelosuppressive therapy. The pneumocystis jiroveci pneumonia (PJP)-treating medication atovaquone (AQ) suppresses oxidative phosphorylation (OXPHOS) and reduces AML burden in patient-derived xenograft (PDX) mouse models, making it an ideal concomitant AML therapy. Poor palatability and limited product formulations have historically limited routine use of AQ in pediatric AML patients. Patients with de novo AML were enrolled at two hospitals. Daily AQ at established PJP dosing was combined with standard AML therapy, based on the Medical Research Council backbone. AQ compliance, adverse events (AEs), ease of administration score (scale: 1 (very difficult)-5 (very easy)) …

Response Assessment In Pediatric Craniopharyngioma: Recommendations From The Response Assessment In Pediatric Neuro-Oncology (Rapno) Working Group, Lindsey M Hoffman, Camilo Jaimes, Kshitij Mankad, David M Mirsky, Benita Tamrazi, Christopher L Tinkle, Cassie Kline, Aparna Ramasubramanian, Fatema Malbari, Ross Mangum, Holly Lindsay, Vincent Horne, David J Daniels, Sameer Keole, David R Grosshans, Tina Young Poussaint, Roger Packer, Sergio Cavalheiro, Brigitte Bison, Todd C Hankinson, Hermann L Müller, Ute Bartels, Katherine E Warren, Murali Chintagumpala

Faculty, Staff and Student Publications

BACKGROUND: Craniopharyngioma is a histologically benign tumor of the suprasellar region for which survival is excellent but quality of life is often poor secondary to functional deficits from tumor and treatment. Standard therapy consists of maximal safe resection with or without radiation therapy. Few prospective trials have been performed, and response assessment has not been standardized.

METHODS: The Response Assessment in Pediatric Neuro-Oncology (RAPNO) committee devised consensus guidelines to assess craniopharyngioma response prospectively.

RESULTS: Magnetic resonance imaging is the recommended radiologic modality for baseline and follow-up assessments. Radiologic response is defined by 2-dimensional measurements of both solid and cystic tumor …

Antibiotic Indications And Appropriateness In The Pediatric Intensive Care Unit: A 10-Center Point Prevalence Study, Kathleen Chiotos, Jennifer Blumenthal, Juri Boguniewicz, Debra L Palazzi, Erika L Stalets, Jessica H Rubens, Pranita D Tamma, Stephanie S Cabler, Jason Newland, Hillary Crandall, Emily Berkman, Robert P Kavanagh, Hannah R Stinson, Jeffrey S Gerber

Faculty, Staff and Students Publications

BACKGROUND: Antibiotics are prescribed to most pediatric intensive care unit (PICU) patients, but data describing indications and appropriateness of antibiotic orders in this population are lacking.

METHODS: We performed a multicenter point prevalence study that included children admitted to 10 geographically diverse PICUs over 4 study days in 2019. Antibiotic orders were reviewed for indication, and appropriateness was assessed using a standardized rubric.

RESULTS: Of 1462 patients admitted to participating PICUs, 843 (58%) had at least 1 antibiotic order. A total of 1277 antibiotic orders were reviewed. Common indications were empiric therapy for suspected bacterial infections without sepsis or septic …

Genetic Variants In Arhgef6 Cause Congenital Anomalies Of The Kidneys And Urinary Tract In Humans, Mice, And Frogs, Verena Klämbt, Florian Buerger, Chunyan Wang, Thomas Naert, Karin Richter, Theresa Nauth, Anna-Carina Weiss, Tobias Sieckmann, Ethan Lai, Dervla M Connaughton, Steve Seltzsam, Nina Mann, Amar J Majmundar, Chen-Han W Wu, Ana C Onuchic-Whitford, Shirlee Shril, Sophia Schneider, Luca Schierbaum, Rufeng Dai, Mir Reza Bekheirnia, Marieke Joosten, Omer Shlomovitz, Asaf Vivante, Ehud Banne, Shrikant Mane, Richard P Lifton, Karin M Kirschner, Andreas Kispert, Georg Rosenberger, Klaus-Dieter Fischer, Soeren S Lienkamp, Mirjam M P Zegers, Friedhelm Hildebrandt

Faculty, Staff and Students Publications

Background: About 40 disease genes have been described to date for isolated CAKUT, the most common cause of childhood CKD. However, these genes account for only 20% of cases. ARHGEF6, a guanine nucleotide exchange factor that is implicated in biologic processes such as cell migration and focal adhesion, acts downstream of integrin-linked kinase (ILK) and parvin proteins. A genetic variant of ILK that causes murine renal agenesis abrogates the interaction of ILK with a murine focal adhesion protein encoded by Parva , leading to CAKUT in mice with this variant.

Methods: To identify novel genes that, when mutated, result in …

Lumasiran For Advanced Primary Hyperoxaluria Type 1: Phase 3 Illuminate-C Trial, Mini Michael, Jaap W Groothoff, Hadas Shasha-Lavsky, John C Lieske, Yaacov Frishberg, Eva Simkova, Anne-Laure Sellier-Leclerc, Arnaud Devresse, Fitsum Guebre-Egziabher, Sevcan A Bakkaloglu, Chebl Mourani, Rola Saqan, Richard Singer, Richard Willey, Bahru Habtemariam, John M Gansner, Ishir Bhan, Tracy Mcgregor, Daniella Magen

Faculty, Staff and Students Publications

Rationale & objective: Lumasiran reduces urinary and plasma oxalate (POx) in patients with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function. ILLUMINATE-C evaluates the efficacy, safety, pharmacokinetics, and pharmacodynamics of lumasiran in patients with PH1 and advanced kidney disease.

Study design: Phase 3, open-label, single-arm trial.

Setting & participants: Multinational study; enrolled patients with PH1 of all ages, estimated glomerular filtration rate ≤45 mL/min/1.73 m2 (if age ≥12 months) or increased serum creatinine level (if age < 12 months), and POx ≥20 μmol/L at screening, including patients with or without systemic oxalosis.

Intervention: Lumasiran administered subcutaneously; 3 monthly doses followed by monthly or quarterly weight-based dosing.

Outcome: Primary end point: percent change in POx …

Efficacy Of Eltrombopag With Immunosuppressive Therapy For Children With Acquired Aplastic Anemia, Yufei Zhao, Wenrui Yang, Xin Zhao, Xiangrong Hu, Jing Hu, Xu Liu, Jianping Li, Lei Ye, Youzhen Xiong, Yang Yang, Baohang Zhang, Xiaoxia Li, Xiawan Yang, Yimeng Shi, Guangxin Peng, Yuan Li, Huihui Fan, Kang Zhou, Liping Jing, Li Zhang, Fengkui Zhang

Faculty, Staff and Student Publications

BACKGROUND: Eltrombopag (EPAG), an oral thrombopoietin receptor agonist (TPO-RA), has been proven to improve the hematologic response without increasing toxic effects as a first-line therapy combined with standard immunosuppressive treatment (IST) in adults with severe aplastic anemia (SAA). Nevertheless, the clinical evidence on the efficacy of EPAG in children with acquired aplastic anemia is limited and controversial.

METHODS: We performed a single-center, retrospective study to analyze the clinical outcomes of fifteen patients aged ≤18 years with newly diagnosed acquired SAA who received first-line IST and EPAG (EPAG group) compared with those of forty-five patients who received IST alone (IST group) …

A Comparison Of Chief Complaints, Specific Diagnoses, And Demographics Of Pediatric Urgent Care Visits Before And During The Covid-19 Pandemic: A Retrospective Study, Zaharoula A. Viennas, Julie Martin, Benjamin Klick, Tammy Speerhas, Turaj Vazifedan, Danielle Millspaugh, Jennifer Ferris, Margret Bedle, Lauren Paluch, Theresa Guins

Ellmer School of Nursing Faculty Publications

There was an increased incidence of pediatric patients who presented with injuries caused by falls not related to sports or other recreational activities, as well as for animal bites, during the early pandemic period of April 2020. Education of parents and caregivers of young children is warranted to raise awareness of the even greater potential for falls and animal bites when children are confined at home for longer than typical periods of time, as occurred with the stay-at-home government orders during the initial period of the COVID-19 pandemic.

National Cross-Sectional Study On Cost Consciousness, Cost Accuracy, And National Medical Waste Reduction Initiative Knowledge Among Pediatric Hospitalists In The United States, Benjamin C Lee, Matt Hall, Ladan Agharokh, Andrew G Yu, Kavita Parikh, Samir S Shah

Faculty, Staff and Student Publications

BACKGROUND/OBJECTIVE: Despite initiatives to reduce waste and spending, there is a gap in physician knowledge regarding the cost of commonly ordered items. We examined the relationship between pediatric hospitalists' knowledge of national medical waste reduction initiatives, self-reported level of cost-consciousness (the degree in which cost affects practice), and cost accuracy (how close an estimate is to its hospital cost) at a national level.

METHODS: This cross-sectional study used a national, online survey sent to hospitalists at 49 children's hospitals to assess their knowledge of national medical waste reduction initiatives, self-reported cost consciousness, and cost estimates for commonly ordered laboratory studies, …

Chromosomal Microarray Analysis Supplements Exome Sequencing To Diagnose Children With Suspected Inborn Errors Of Immunity, Breanna J Beers, Morgan N Similuk, Rajarshi Ghosh, Bryce A Seifert, Leila Jamal, Michael Kamen, Michael R Setzer, Colleen Jodarski, Rylee Duncan, Devin Hunt, Madison Mixer, Wenjia Cao, Weimin Bi, Daniel Veltri, Eric Karlins, Lingwen Zhang, Zhiwen Li, Andrew J Oler, Kathleen Jevtich, Yunting Yu, Haley Hullfish, Bibiana Bielekova, Pamela Frischmeyer-Guerrerio, An Dang Do, Laryssa A Huryn, Kenneth N Olivier, Helen C Su, Jonathan J Lyons, Christa S Zerbe, V Koneti Rao, Michael D Keller, Alexandra F Freeman, Steven M Holland, Luis M Franco, Magdalena A Walkiewicz, Jia Yan

Faculty, Staff and Students Publications

PURPOSE: Though copy number variants (CNVs) have been suggested to play a significant role in inborn errors of immunity (IEI), the precise nature of this role remains largely unexplored. We sought to determine the diagnostic contribution of CNVs using genome-wide chromosomal microarray analysis (CMA) in children with IEI.

METHODS: We performed exome sequencing (ES) and CMA for 332 unrelated pediatric probands referred for evaluation of IEI. The analysis included primary, secondary, and incidental findings.

RESULTS: Of the 332 probands, 134 (40.4%) received molecular diagnoses. Of these, 116/134 (86.6%) were diagnosed by ES alone. An additional 15/134 (11.2%) were diagnosed by …