Medical Specialties Commons^™

Metreleptin And Metformin Use In An Infant With Congenital Generalized Lipodystrophy Secondary To Agpat2 Mutation, Cintya Schweisberger

Research Days

Background: Congenital generalized lipodystrophy (CGL) is a rare inherited disease characterized by widespread loss of subcutaneous fat and severe metabolic abnormalities. Metreleptin, a synthetic analog of leptin, is a treatment modality that has been shown to decrease fasting triglycerides, fasting glucose, and HbA1c. Metformin use in infants has only been described in a few case reports of CGL and Donohue syndrome (insulin receptor mutation), and there is no established dosing for this age group. We report metreleptin, insulin, and metformin use in an infant with type 1 CGL who presented with marked hypertriglyceridemia, hypoleptinemia, hyperglycemia, and transaminitis.

Clinical Case: A …

Survival Following Relapse In Children With Acute Myeloid Leukemia: A Report From Aml-Bfm And Cog., Mareike Rasche, Martin Zimmermann, Emma Steidel, Todd Alonzo, Richard Aplenc, Jean-Pierre Bourquin, Heidrun Boztug, Todd Cooper, A S. Gamis, Robert B. Gerbing, Iveta Janotova, Jan-Henning Klusmann, Thomas Lehrnbecher, Nora Mühlegger, Nils V. Neuhoff, Naghmeh Niktoreh, Lucie Sramkova, Jan Stary, Katharina Waack, Christiane Walter, Ursula Creutzig, Michael Dworzak, Gertjan Kaspers, Edward Anders Kolb, Dirk Reinhardt

Manuscripts, Articles, Book Chapters and Other Papers

Post-relapse therapy remains critical for survival in children with acute myeloid leukemia (AML). We evaluated survival, response and prognostic variables following relapse in independent cooperative group studies conducted by COG and the population-based AML-BFM study group. BFM included 197 patients who relapsed after closure of the last I-BFM relapse trial until 2017, while COG included 852 patients who relapsed on the last Phase 3 trials (AAML0531, AAML1031). Overall survival at 5 years (OS) was 42 ± 4% (BFM) and 35 ± 2% (COG). Initial high-risk features (BFM 32 ± 6%, COG 26 ± 4%) and short time to relapse (BFM …

Neonatal Renal Failure In The Setting Of Anorectal Malformation: A Case Report And Literature Review., Wendy Jo Svetanoff, Asma Ahmed, Richard J. Hendrickson, Rebecca M. Rentea

Manuscripts, Articles, Book Chapters and Other Papers

Anorectal malformations (ARMs) can occur in isolation or in association with other anomalies, most commonly those of the genitourinary systems. Morbidity and mortality are highest among patients who develop end-stage renal disease (ESRD) either from severe congenital anomalies (dysplastic kidneys) or from repeated infections in those who have vesicoureteral reflux or persistent recto-urinary fistulas. We describe our management strategy for a patient born with an ARM and bilateral dysplastic kidneys to highlight the nuances and complex decision-making considerations required in taking care of this complex patient population. Our patient is a male twin born at 32 weeks' gestational age who …

Evaluation Of The Hypothesis That Viral Meningitis Is A Mimic Of Abusive Head Trauma, Danielle Horton

Research Days

Background: Viral meningitis (VM) has been proposed as an alternative cause of subdural hemorrhage (SDH) in young children diagnosed with abusive head trauma (AHT). It has been proposed that VM may mimic symptoms and clinical features of AHT, resulting in an incorrect diagnosis of abuse.

Objectives/Goal: We aim to evaluate the hypothesis that VM is a mimic of AHT by comparing the history of present illness (HPI) and initial clinical presentation of young children with proven VM to those with subdural hemorrhage and concomitant suspicious injuries (SDH + CSIs) and to those with SDH and no CSIs (SDH only). We …

Local Implementation And Standardization Of The Pediatric Severe Traumatic Brain Injury Guidelines, Michelle Brown

Research Days

Problem Statement/Question: Severe pediatric Traumatic Brain Injury (TBI) remains a significant cause of morbidity and mortality. Evidence-based guidelines leave gaps in how to best implement and deliver standardized care on the local hospital level.

Background/Project Intent (Aim Statement): We aim to improve clinical outcomes for severe TBI patients (Glasgow Coma Scale ≤8) with standardization of care according to updated evidence-based guidelines. We will target early guideline adherence (initial 72 hours of PICU admission) with a focus on 1) targeted temperature management (TTM40mmHg).

Methods (include PDSA cycles): The Children’s Mercy Hospital Pediatric ICU Severe TBI Guidelines were updated after the 3rd …

Simulation Based Clinical Systems Testing Of A Pediatric Ed To Improve Staff And Process Readiness For Pediatric Hypoglycemia, Kevin Meilak Md

Research Days

Background/Project Intent (Aim Statement): Hypoglycemia is the most common metabolic disorder in children in pediatric emergency department (ED) settings 1 . Children may present with nonspecific symptoms, or asymptomatically2 . So identification/treatment is very challenging. There is evidence in the adult literature that there is wide variability in the treatment of hypoglycemia3 . Currently Children’s Mercy does not have a standardized approach to the treatment of hypoglycemia not associated with diabetes mellitus. From observation in the clinical setting we believe there are gaps regarding the identification and treatment of hypoglycemia not associated with diabetes mellitus. Delayed recognition and under treatment …

Optimization Of Perioperative Prophylaxis In Penicillin-Allergic Labeled Patients, Katie A. Vandervelde

Research Days

Problem Statement/Question: A penicillin (PCN)-allergy label is life-long and has been shown to result in more costly, less effective, and broader-spectrum antibiotics being utilized for surgical prophylaxis. At CMH, 6.6% of surgical patients receiving perioperative antibiotics are labeled PCN-allergic. A common myth is that approximately 10% of patients with a PCN-allergy history will experience an allergic reaction if administered a cephalosporin, whereas recent observational studies have found crossreactivity rates between 0.17-0.7%. Most of these patients receive a cefazolin alternative unnecessarily. Currently, no process exists to identify and clarify PCN-allergic patients preoperatively. Data from November 2018-December 2019 found ~2.33 patients per …

Tnfaip3: Evolving Clinical Picture From Autoinflammatory Syndrome To Autoimmune Lymphoproliferative Syndrome, Esosa Adah Md

Research Days

Background: Autoimmune lymphoproliferative syndrome (ALPS) is a rare genetic disorder of the immune system that can affect children and adults. The most frequent presentation is benign lymphoproliferation limited to lymphoid organs such as the lymph nodes, liver, and spleen which result in splenomegaly and lymphadenopathy, two prominent features in ALPS patients. ALPS can lead to anemia, thrombocytopenia, and neutropenia which increase risks of infection and hemorrhage. Most cases are caused by either a germline or somatic mutation in the FAS, FASLG, or CASP10 genes that are involved in cell apoptosis. Tumor Necrosis Factor Alpha, Induced Protein 3 (TNFAIP3) is a …

A Case Of Metabolic Genetic Emergency, Elizabeth Loughman

Research Days

Background: Newborn screening (NBS) is one of the largest public health initiatives to improve infant morbidity and mortality, and this has been shown to detect and lead to prompt treatment of uncommon but rapidly fatal conditions. While NBS has changed outcomes of diseases, it is still imperative that prompt and early recognition of abnormal results and medical interventions remain the mainstay of care.

Objective: To describe a case of inborn error of metabolism in a 4-day old asymptomatic infant, where early recognition and treatment affected outcome.

Design/Methods: This case is a 4-day old female, former 36-week gestational age infant who …

Association Between Blood Pressure And Esa Dose In Pediatric Patients On Dialysis, Heather A. Morgans

Research Days

Background: Hypertension is a reported side effect of Erythropoiesis Stimulating Agents (ESAs), with a mechanism of action related to elevated hematocrit levels and direct vasopressor effects. Limited information exists on the relationship between ESA dosage and hypertension in children receiving maintenance dialysis.

Objectives/Goal: The primary aim of this study was to determine whether there is a significant association between ESA dose and blood pressure (BP) in pediatric patients on dialysis. The secondary aim was to determine covariates in relation to ESA dose.

Methods/Design: Data from the International Pediatric Dialysis Network (IPDN) database was used to retrospectively analyze the association between …

Setting A Threshold For Discharge Antibiotics In Children With Perforated Appendicitis: A Study Update, Kayla B. Briggs

Research Days

Background: To address concerns of antibiotic overutilization, normal white blood cell count (WBC) for age was previous l criteria for discontinuing antibiotics at discharge in perforated appendicitis. Retrospective review suggested a decrease in threshold for additional antibiotic administration to a discharge WBC(IAA).

Objectives/Goal: The purpose of our study is to determine if lowering the threshold for additional antibiotic administration decreased the rate of IAA after laparoscopic appendectomy for perforated appendicitis in children who were not discharged with additional antibiotics.

Methods/Design: Following IRB approval, prospective data was retrospectively reviewed. Children with perforated appendicitis identified during laparoscopic appendectomy at our institution between …

Standardized Point-Of-Care Ultrasound (Pocus) Set-Up For Cardiac Evaluation During Covid-19 Pandemic In A Pediatric Population., Anmol Goyal

Research Days

Background: The novel coronavirus SARS-CoV2 is known to cause significant cardiac dysfunction, effusion, thromboembolism and coronary dilation. Currently no pediatric specific guidelines exist from ACC or ASE for use of POCUS. Our objective was to identify ideal pediatric appropriate POCUS equipment and standardize its use.

Methods: A Phillips Lumify probe paired with Android-Samsung tablet and a Butterfly iQ probe paired with an iPad were considered. A workflow algorithm and cleaning instructions were created. If the POCUS scan answered the clinical question, then a detailed echo was deferred. If the POCUS scan revealed findings requiring comprehensive evaluation, a follow-up echo was …

C-Reactive Protein Values To Predict Sepsis-Induced Inflammatory Response In Premature Infants, Megan H. Tucker

Research Days

Background: C-reactive protein (CRP) is an inflammatory marker that has been recognized as a biomarker of the systemic inflammatory response in preterm neonates. We hypothesized that initial and peak CRP values would correlate with the degree of sepsis-induced acute lung injury (ALI) as measured by the pulmonary severity score (PSS).

Objectives/Goal: 1) Determine if confirmed (CF) sepsis events are associated with higher initial and peak CRP values than rule out (RO) sepsis events. 2) Investigate if initial and/or peak CRP correlates with severity of sepsis-induced ALI as measured by the PSS.

Methods/Design: In this retrospective case control study, we included …

Kernicterus Spectrum Disorders Diagnostic Toolkit: Validation Using Retrospective Chart Review, Vijaya Dasari

Research Days

Background: Kernicterus Spectrum Disorders (KSDs) result from hyperbilirubinemia-induced brain injury. While rare, KSDs can result in devastating neurologic sequelae affecting motor function and tone, causing dystonia and choreoathetosis, and/or auditory neuropathy spectrum disorders. Families of children with undiagnosed neurodevelopmental disorders and a history of neonatal hyperbilirubinemia often question this potential diagnosis. We developed a Toolkit (KSD-TK) questionnaire in an effort to provide a standardized method to predict the likelihood of KSDs.

Objectives/Goal: This study compares the accuracy of the KSD-TK to clinical diagnoses made by our Children’s Mercy Hospital Kernicterus Center of Excellence (KCOE). The primary objective of this study …

Bivalirudin And Thrombolytic Therapy: A Novel Successful Treatment Of Severe Aortic Arch Thrombosis In A Term Neonate, Amjad Taha

Research Days

SUMMARY An early term infant with uncomplicated perinatal history was found to have a large thrombus in the aortic arch after he failed regular newborn critical congenital heart defect (CCHD) screen. He responded well to Bivalirudin thrombolytic and tissue-Plasminogen activator (tPA) combination therapy, with a significant resolution of the thrombus. The infant tolerated hospital admission well with no significant complications. He was discharged home on daily aspirin at two weeks of life. To our knowledge, the combination therapy approach with Bivalirudin and tPA is the first one reported in the literature in the neonatal age group.

BACKGROUND Aortic arch thrombosis …

Functional Outcomes Of Infants With Chiari Ii Malformation With Tracheostomy And Home Ventilator Dependence, Abbigayle Carlson

Research Days

Background: Chiari II malformation is relatively common with an incidence of about 1 in 1000 live birth and is associated with reduced life expectancy. Characteristic features are beaked midbrain, downward displacement of the tonsils, and cerebellar vermis, and spinal myelomeningocele that can be associated with hydrocephalus needing ventriculoperitoneal shunt (VP shunt). Mortality is about 50-70% if not recognized early. Survivors have high morbidity and develop neurologic abnormalities and significant developmental delays. Long-term outcome is poorly understood.

Objective: Not much has been published that describes the outcome of infant with Chiari II malformation that received tracheostomy for long-term ventilator support. Our …

Diagnosis And Management Of Otitis Media With Effusion In Pediatric Urgent Care Clinics, Ashley Deschepper

Research Days

Background: Otitis media with effusion (OME)’s clinical presentation is often confused with acute otitis media (AOM) by providers. Despite OME guidelines recommending watchful waiting with no antibiotics, rates of antibiotic use remain elevated. In our pediatric urgent care clinics (UCCs), an estimated 50% of patients diagnosed with OME receive antibiotic prescriptions.

Objectives/Goal: To determine the provider diagnosis validity and the rates of antibiotics prescribed among pediatric OME patients evaluated in 3 UCCs within a pediatric healthcare system.

Methods/Design: We randomly selected 75% of encounters for children age 0-18 years who had a billing diagnosis of OME in 2019. Charts were …

Activation Of Aryl Hydrocarbon Receptor Signaling In Human Trophoblasts Alters Markers Of Growth And Differentiation, Asmaa Alsousi

Research Days

Background: It is estimated that 1.7% of pregnant women smoke during their pregnancy globally, with the highest levels observed in Europe at 8.1%, and lowest in Africa at 0.8. The association of maternal cigarette smoking with increased risk of poor birth outcomes such as preterm birth, congenital anomalies, and neonatal mortality is well-established. In addition, evidence suggests that intrauterine exposure to maternal smoking impacts the risk of developing diseases later in life; however, we still do not understand the exact mechanism(s) leading to these outcomes. Once components of cigarette smoke (CS) cross the placenta and enter the fetal compartment, several …

Evaluation Of A Symptom-Based Algorithm For Managing Battery Ingestions In Children, James Fraser

Research Days

Background/Objectives/Goal: Complications from battery ingestion in the pediatric population are becoming more severe due to the emergence of stronger and smaller batteries. We previously established a symptom-focused, evidence-based algorithm for battery ingestion and seek to evaluate this protocol to investigate its safety and potential cost benefit. (Figure 1)

Methods/Design: Following IRB approval, all radiographs performed for foreign body ingestion between 2017-2020 were reviewed. All button battery ingestions were included to evaluate an institutional symptom-based algorithm emphasizing observation over intervention.

Results: Review of 2,237 foreign bodies series demonstrated 44 button battery ingestions. Median age at ingestion was 3.8 years [2.5,5.2]. Locations …

Medical Neglect: Trends In Reporting Practices And Patient Characteristics, Danielle Horton

Research Days

Background: Medical neglect, an important but less commonly recognized subset of child maltreatment, has significant consequences on child health and well-being. Improved processes for defining, recognizing, and reporting medical neglect are needed, however there is limited published data on the subject. Insight into patient characteristics and current reporting practices will help identify areas for intervention to improve outcomes for children at risk for medical neglect.

Objectives/Goal: To describe characteristics of children identified for concern of medical neglect and trends in reporting of cases to child protective services (CPS).

Methods/Design: We performed a 1-year retrospective descriptive study of subjects < 18 years of age at a single, tertiary-care, academic pediatric institution for whom a Patient at Risk (PAR) assessment was completed exclusively for the concern of medical neglect. The PAR is a unique institutional process by which health professionals and social workers document concerns for child maltreatment. Medical records were reviewed for data regarding demographics, medical history, health care utilization and psychosocial risk factors. The role of the initiating provider and healthcare setting in which the PAR was recorded were also obtained.

Results: Of …

Phenotypic Characterization Of Jarid2-Related Intellectual Disability: A Case Series, Maxime Cadieux-Dion

Research Days

Background: In recent years, wide implementation of research and clinical next generation sequencing has led to an astonishing number of novel disease-gene assertions. Recently, loss of function variants in JARID2 were reported in 16 patients with a clinically distinct neurodevelopmental phenotype that consisted of neurodevelopmental delay, intellectual disability (ID), learning disability, autism and behavioral abnormalities. Dysmorphic features were seen in most patients and included high anterior hairline, deep-set eyes, full lips, broad forehead, bulbous nasal tip, or depressed nasal bridge. Cleft lip/palate was observed in only 1/16 patients. Most cases were de novo, with only one inherited case from an …

Educational Impact Of Supervising Fellow Visits In The Emergency Department, Grace E. Arends Md

Research Days

Problem Statement/Question: After meeting certain criteria, pediatric emergency medicine (PEM) fellows were previously permitted to independently manage and discharge patients. These encounters anecdotally fostered autonomy but missed educational opportunities. We have previously reported that fellow positive perception of education and autonomy was low (22% and 11%). Additionally, the absence of an attending’s attestation on these patient charts prevented the collection of a professional service fee, an average potential billed amount of $125.67 per unit of service or total of $620,307 in 2019.

Background/Project Intent (Aim Statement): After key stakeholder involvement, we recognized attending supervision for all fellow patients as an …

Smart Rounding: Development Of A Nurse-Driven Rounding Checklist As A Sustainable Intervention For Improved Care Communication, Charles I. Maloy

Research Days

Background/Project Intent: Use of checklists in the healthcare setting is increasingly common and is important to the development of high reliability and standard avenues for communication. At our tertiary care freestanding pediatric hospital, there was no widely used rounding checklist that discussed items important to quality care and patient safety outside of the intensive care unit. The development and implementation of medical checklists for the standard patient floor are inadequately described. We will develop and implement a rounding checklist with at least 80% daily checklist use sustained over 6 months. Team members will report improved multidisciplinary discussion of patient safety …

Educating Providers: Timely Post-Operative Pain Management In A Level Iv Neonatal Intensive Care Unit (Nicu), Jamesia Donato, Jamesia Donato

Research Days

Background/Project Intent (Aim Statement): NICU patients often encounter painful procedures and can have significant short and long-term consequences from inadequately treated pain. Ensuring adequate pharmacological and nonpharmacological pain management while minimizing associated risks is paramount. Our NICU is part of the Children’s Hospitals Neonatal Consortium “Erase Pain” collaborative to improve management of postop pain in neonates. SMART Aim: Increase the percentage of patients receiving acetaminophen within one hour after surgery to greater than 70% by July 2020.

Methods (include PDSA cycles): A multidisciplinary team developed pain treatment algorithms and postop order plans to standardize the approach to postop pain management. …

Improving The Approach To Fellowship Trainee Feedback, Katie A. Vandervelde

Research Days

Problem Statement/Question: Giving and receiving feedback are difficult but necessary skills in medical training. We identified a need for improved feedback based on decreased learner satisfaction on our annual ACGME survey. In an anonymous survey, 2018-19 infectious disease fellows reported receiving formal feedback from 52.4% of their inpatient clinical experiences with faculty.

Background/Project Intent (Aim Statement): Increase the occurrence of feedback to fellows by faculty during clinical service rotations from 52.4% to >90.0% in 6 months utilizing a standardized method.

Methods (include PDSA cycles): A Feedback Card (Figure 1), was developed by fellows and faculty. Both parties were instructed to …

Leveraging Collaboration In Pediatric Multidisciplinary Colorectal Care Using A Telehealth Platform, Joseph Lopez

Research Days

Purpose: Pediatric colorectal problems require complex multidisciplinary care. The COVID-19 health crisis has substantially shifted how this healthcare delivery can safely take place. We sought to track the characteristics of in-person (IP) and telehealth (TH) visits and subsequent satisfaction and utilization in this quality improvement project.

Methods: We present the multidisciplinary clinic experience from October 1, 2019 to August 31, 2020 in which patient volume, visit time length, patient satisfaction survey results, and frequencies of colorectal diagnoses seen before (October 1, 2019 through January 31, 2020) and during the present pandemic (February 1, 2020 through August 31, 2020). Microsoft Teams …

Does Access To Care Influence A Child’S Presentation With Appendicitis?, Neal Talukdar

Research Days

Background + Objectives/Goal: Racial demographic and health care coverage impact how and when patients seek care. Previous studies have documented that minority children present with higher rates of perforated appendicitis. We aimed to identify if racial and coverage disparities were associated with the timing and mechanism of appendicitis presentation.

Methods/Design: We performed a prospective study of children (≤ 18 years old) who underwent laparoscopic appendectomy from May 2018 - September 2020. Prior to discharge, the patient’s parent/guardian completed a survey focused on health care encounters within the past year. Surveys were collected using RedCap®; data were analyzed using STATA®.

Results: …

Pediatric Intensivists' Use Of Nudging In Care Conferences, Aliza Olive

Research Days

Background: Nudging is a behavioral economics term describing a form of choice architecture that affects behavior without eliminating options. Shared decisions with families are made frequently in the PICU during care conferences, where nudging likely occurs, but has not been studied.

Objectives/Goal: We aimed to identify how pediatric intensivists use nudging to guide decision making.

Methods/Design: We conducted a retrospective analysis of 70 PICU care conference transcripts using a validated coding scheme for choice architecture. Choice frames included: saliency, gain loss or mixed frame, yes/no, two options, multiple options, open ended, default, endowment, commission, omission, recommend, expert opinion, certainty, and …

Pulmonary Severity Score As A Measure Of Sepsis-Induced Acute Lung Injury In Preterm Infants, Megan H. Tucker

Research Days

Background: Sepsis is the most common indirect cause of acute lung injury (ALI) in adult and pediatric patients, yet no studies have described this phenomenon in preterm infants. We hypothesize that sepsis-induced ALI occurs in neonates and increases the risk of bronchopulmonary dysplasia (BPD) in preterm infants.

Objectives/Goal: 1) To investigate whether late onset sepsis (LOS) and other systemic inflammatory diseases are temporally correlated with ALI as quantified by pulmonary severity score (PSS) trajectory. 2) To determine the sepsis subtypes associated with the greatest severity of ALI.

Methods/Design: We included infants < 31 weeks gestational age and < 1500 grams with LOS in this retrospective case control study. The PSS was calculated at 72, 48, and 24 hours (hr) prior to, at the time of, and 24, 48, 72, and 168hr after sepsis diagnosis. We further defined rule out (RO) sepsis (blood culture negative; antibiotics continued only 48-72hr), blood culture positive (Cx+) sepsis, necrotizing enterocolitis (NEC), urinary tract infection (UTI), and culture negative (Cx-) clinical sepsis (blood culture negative; antibiotics continued > 6 days; specific lab criteria met). PSS trajectories for …

Tricuspid Atresia With Totally Anomalous Pulmonary Venous Connection: An Uncommon Finding, Anmol Goyal

Research Days

Background: Tricuspid atresia comprises 3-4% of all congenital heart disease (CHD). Anatomic findings are no true connection between the right atrium (RA) and right ventricle (RV) with concomitant findings of a ventricular septal defect (VSD), right ventricular hypoplasia, abnormalities of right ventricular outflow tract, pulmonary valve and pulmonary arteries. Common associated lesions have been described including transposition of great vessels, hypoplasia of aortic arch, double outlet ventricle or a common arterial trunk. Total anomalous pulmonary venous connection (TAPVC) is relatively rare and we present a case in addition to the six previously described cases in literature.

Case: A 33-year-old woman …