Pediatrics Commons^™

Pharmacogenetic Testing In Patients With Autism Spectrum Disorder Evaluated In The Children’S Mercy Hospital Goldiloks© Clinic, Rachel Goodson

Research Days

Background: Autism Spectrum Disorder (ASD) affects 1 in 54 children in the United States. Children with ASD are more likely to be diagnosed with co-occurring mental health disorders. There is currently little research guiding medication choice and dosing in patients with ASD. Children with ASD are at increased risk for reduced clinical response and adverse reaction (including due to polypharmacy). Pharmacogenomics is an approach for medication decision making that leverages an individual’s genetic information and clinical presentation to make informed choices, but no studies have specifically investigated the outcomes of PGX for patients with ASD. Understanding the clinical and genetic …

Outcomes Of Infants With Severe Rop At Risk Of Treatment With Avastin Compared To Laser Surgery, Stephen Doxey

Research Days

Background: Retinopathy of prematurity (ROP) is a well-known complication occurring in the most premature infants leading to visual impairment and in the most severe cases associated with retinal detachment and childhood blindness. Factors contributing the development of ROP include chronic hypoxia and disordered vascular genesis primarily through the vascular endothelial growth factor (VEGF) signaling pathway. Infants at risk of developing ROP are screened with serial eye exam and treated with either laser surgery or intraocular injects of bevacizumab (Avastin) to prevent progression of ROP and preserve vision. Bevacizumab, an inhibitor of VEGF signaling, may have positive effects on preventing ROP …

Hyperglycemia Requiring Insulin Among Pediatric Patients Diagnosed With Hemophagocytic Lymphohistiocytosis, Cintya Schweisberger

Research Days

Background: Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening disorder marked by massive cytokine release due to macrophage and T-cell activation. Hallmarks of the diagnosis include fever, splenomegaly, cytopenias, hypertriglyceridemia, hypofibrinogemia, and elevations in ferritin and soluble IL-2 receptor. Given HLH is associated with critical illness, elevation in inflammatory markers, and treated with glucocorticoids, the development of hyperglycemia during its course is not unexpected. However, detailed descriptions of the severity of hyperglycemia and strategies in insulin management among HLH patients are lacking. We describe 10 years’ experience at a single tertiary pediatric health center with HLH patients who developed insulin dependent …

Long-Term Safety Of Growth Hormone Treatment In Childhood: Two Large Observational Studies: Nordinet Ios And Answer., Lars Sävendahl, Michel Polak, Philippe Backeljauw, Joanne C Blair, Bradley S Miller, Tilman R Rohrer, Anita Hokken-Koelega, Alberto Pietropoli, Nicky Kelepouris, Judith L. Ross

Department of Pediatrics Faculty Papers

CONTEXT: Growth hormone (GH) treatment has a generally good safety profile; however, concerns about increased mortality risk in adulthood have been raised.

OBJECTIVE: This work aims to assess the long-term safety of GH treatment in clinical practice.

METHODS: Data were collected from 676 clinics participating in 2 multicenter longitudinal observational studies: the NordiNet International Outcome Study (2006-2016, Europe) and ANSWER Program (2002-2016, USA). Pediatric patients treated with GH were classified into 3 risk groups based on diagnosis. Intervention consisted of daily GH treatment, and main outcome measures included incidence rates (events/1000 patient-years) of adverse drug reactions (ADRs), serious adverse events …

The Power Of Outpatient Botulinum Injections In Preventing Hirschsprung Associated Enterocolitis, Wendy Jo Svetanoff

Research Days

Background: Hirschsprung associated enterocolitis (HAEC) is a potentially life-threatening complication, where the child often presents with obstructive symptoms. Failure of the internal anal sphincter (IAS) to relax is thought to play a role in these symptoms. Botulinum toxin (BT) injections assist in temporarily relieving obstructive symptoms due to IAS achalasia in patients with Hirschsprung’s disease following surgical pull-through. The use of BT may play a role in preventing HAEC episodes related to IAS dysfunction.

Objectives/Goal: Our aim was to determine the effect of scheduled outpatient Botulinum injections on the development of HAEC.

Methods/Design: A retrospective review between July 2010 – …

Iv Amiodarone Vs Iv Sotalol Use In Postoperative Junctional Ectopic Tachycardia (Jet): A Randomized Study, Tanner Isaacson

Research Days

Background: Junctional ectopic tachycardia (JET) is one of the most common post-operative arrhythmias encountered following congenital heart surgery. For many, amiodarone is the pharmacologic treatment of choice. While effective, amiodarone has been associated with dose-related adverse effects. Sotalol has been approved for intravenous use in pediatric patients in the United States to treat both supraventricular and ventricular arrhythmias, although pediatric data remains limited. Sotalol has class III antiarrhythmic properties with some mild β-adrenergic blocking effects, similar to amiodarone. Data shows a lower rate of cardiovascular collapse and adverse events with sotalol in patients with congenital heart disease. At present, it …

Long-Term Outcomes And Satisfaction Rates After Costal Cartilage Resection For Slipping Rib Syndrome, James Fraser

Research Days

Background/Objectives/Goal: Slipping rib syndrome (SRS) is a challenging and underdiagnosed condition that has proven to be an elusive diagnosis due to an extensive differential with presenting symptoms that mimic a myriad of alternative diagnoses of both thoracic and abdominal pathology. The diagnosis of SRS is recognized in a patient with consistent, progressive, often debilitating unilateral or bilateral chest wall and/or upper abdominal pain with intermittent clicking or rib slipping. SRS frequently does not resolve with conservative management, including physical therapy, rest, and NSAIDs, as well as intercostal injections, and even opioid medications. Workup can be extensive including multiple subspecialty referrals …

Multimodal Pain Control In Common Neonatal Surgeries: Post-Operative Pain Protocol Reduces Opiate Exposure And Side Effects, Jamesia Donato

Research Days

Background: Opioids are the primary post-operative (post-op) analgesic in neonates. Side effects include hypotension, apnea and ileus. Previous studies show IV acetaminophen decreases opiate need in specific populations including cleft palate repair, cardiac surgery. The effect of IV acetaminophen as part of a post-op pain algorithm is not known in common neonatal surgical procedures.

Objectives/Goal: To determine if (a) a post-op pain algorithm including IV acetaminophen and (b) IV acetaminophen exposure reduces cumulative opioid requirements in neonates undergoing common surgeries in the NICU.

Methods/Design: This is a retrospective cohort study comparing cumulative post-operative opiate exposure between infants with common surgeries …

The Effect Of Covid-19 On Pediatric Orthopaedic Trauma Volume At A Level 1 Pediatric Trauma Center In The Midwestern United States, Kirsten Norrell

Research Days

Background: School and playground closures and the cancellation of extracurricular activities in the Kansas City region on March 17, 2020 due to the COVID-19 pandemic created an unprecedented situation for children and parents. Early reports in the literature on adult and pediatric trauma volume suggested a decrease in orthopaedic injuries during the pandemic. This is the first study to report how the pandemic affected orthopaedic trauma volume at a Level 1 pediatric trauma center during the entire five months since school and activity closures went into effect.

Objectives/Goal: The aim of this study was to compare the volume and type …

Pre-Hospital Traumatic Cardiopulmonary Arrest In Children At A Level 1 Pediatric Trauma Center, Kayla B. Briggs

Research Days

Background: The survival of traumatic cardiopulmonary arrest (TCA) requiring pre-hospital cardiopulmonary resuscitation (P-CPR) is abysmal across age groups.

Objectives/Goal: We aim to describe the patterns of injury, pre-hospital interventions, and outcomes of children suffering from TCA leading to P-CPR at our institution to support standardized guidelines addressing the termination of prolonged resuscitation attempts in children.

Methods/Design: Following IRB approval, retrospective review was conducted to identify children ages 0-17 years who suffered TCA leading to P-CPR at our institution between 5/1/2009–3/1/2020. For analysis, patients were stratified into those still undergoing CPR at arrival and those who attained pre-hospital return of spontaneous …

Improving Thromboprophylaxis In Hospitalized Pediatric Inflammatory Bowel Disease Patients- A Quality Improvement Project, Amy Issa, Panam Kaur

Research Days

Background/Project Intent (Aim Statement): Pediatric inflammatory bowel disease (IBD) patients in acute flare have 1.5 to 3-fold higher risk of thromboembolism compared to the general population. Anticoagulation therapy is recommended for pediatric IBD patients who have ≥ 1 risk factors for venous thromboembolic events. At Children’s Mercy Hospital (CMH), approximately 80 patients/year are admitted for acute flare, of which, roughly 30% receive appropriate thromboprophylaxis. Lack of standardization of thromboprophylaxis and an educational gap amongst providers are significant barriers. Our goal is to standardize and improve the rates of thromboprophylaxis in IBD patients admitted at CMH from 30% to 60% by …

Mini-Ace® Low-Profile Appendicostomy Button, Joseph Lopez

Research Days

Purpose: Malone antegrade continence enemas (MACE) provide a conduit in which the patient can achieve improved continence, be clean of stool, and gain independence in maintaining bowel function. The Mini-ACE® is a low-profile balloon button that is used to facilitate administration of antegrade enemas. We sought to describe our practice and short-term outcomes.

Methods: We present our experience using the Mini-ACE® at Children’s Mercy – Kansas City from April 2019 to September 2020. Patient demographics, colorectal diagnoses, tube sizes, flush regimens, patient experience and outcomes were examined.

Results: Thirty patients were included; 18 (60%) were male. The average age at …

Umbilical Access In Laparoscopic Surgery In Infants Less Than 3 Months: Single Institution Retrospective Review, James Fraser

Research Days

Background/Objectives/Goal: Umbilical access in laparoscopic surgery has been cited as a potential factor for increased complications in low-birth-weight infants and those less than three months old. Previous series noted a self-reported complication rate of 10.6% among 329 pediatric surgeons via anonymous survey, citing carbon dioxide (CO2) embolism as the most common complication. We report four-year outcomes with blunt transumbilical laparoscopic access to examine the safety of this technique.

Methods/Design: Following IRB approval, a retrospective database of patients less than three months of age who underwent laparoscopic pyloromyotomy or inguinal hernia repair from 2016-2019 at a tertiary care academic center was …

Pediatric Pulmonary Artery Aneurysms Causing Pulmonary Hemorrhage: A Case Of Transcatheter Intervention While On Va Ecmo Support, Igor Areinamo

Research Days

Background: The incidence of pulmonary artery aneurysm (PAA) in children remains unknown. There are few reports of a transcutaneous interventional approach to address this type of pathology. Management of PAAs while on VA ECMO has not been described in pediatrics.

Objectives/Goal:

Methods/Design:

Case: 9-year-old previously healthy male presented with MRSA associated ARDS and sepsis requiring urgent VA ECMO cannulation. Chest CT done on ECMO day 22 showed development of right sided PAAs. Patient had an episode of significant pulmonary hemorrhage. Angiogram demonstrated two fusiform right PAAs, one of which had ruptured and was bleeding into the lung parenchyma. Patient had …

Congenital Mitral Valve Regurgitation, The Dilemma Of Repair Vs Replacement., Bianca Cherestal

Research Days

Background: Congenital mitral regurgitation is a rare condition and can be challenging to manage when presenting in the neonatal period

Objectives/Goal: Two week old male presented with poor weight gain, murmur and cardiomegaly on chest X-ray. Echocardiogram showed moderate to severe mitral regurgitation (MR) and suprasystemic pulmonary hypertension (PHN) (fig 1 a, b). The mitral valve (MV) leaflets were thickened and tethered with failure of central coaptation. PHN was classified as WHO I and II (due to persistent PHN of newborn and MR respectively). Inhaled nitric oxide, Enalapril and Furosemide were initiated. Cardiac catheterization revealed PVRi of 8.9 WU x …

Online Echocardiography Modules For Pediatric Cardiology Fellows, Sarah Studyvin, Doaa Aly, Tyler Johnson, Laura Kuzava, Alison Samrany, Nitin Madan, Sanket Shah

Research Days

Background: Adequate echocardiographic (echo) education is integral to successful cardiology training. Online education is proven to be effective in other specialties, but pediatric echo training data are lacking.

Objectives/Goal: Our aim was to design and assess the role of a novel online module-based curriculum to enhance pediatric cardiology fellows’ echo education.

Methods/Design: Four interactive modules, focused on principles and interpretation of a normal echo, were delivered to fellows. Content included high quality 2D and 3D echo clips as well as interactive quizzes and could be accessed from a phone, tablet, or computer. All fellows completed pre- and post-tests, validated by …

The Impact Of Early Tracheostomy On Neurodevelopmental Outcome Of Infants With Severe Bronchopulmonary Dysplasia Exposed To Postnatal Steroids, Amjad Taha

Research Days

Background: BPD is associated with long-term neurodevelopmental impairment. Tracheostomy is performed in 5-12% of severe BPD for prolonged ventilation. There is evidence that chronic ventilation with tracheostomy in severe BPD may facilitate neurodevelopment and lead to improved outcome. However, there is no consensus on the optimal timing of tracheostomy. A large multicenter study of infants with tracheostomy performed at <120 days of life had better neurodevelopmental outcomes at 18-22 months of age. Use of steroids postnatally to ameliorate the severity of BPD had been controversial due to its negative effect on neurodevelopment. To date, no data has specifically evaluated the impact of early tracheostomy on neurodevelopmental outcome of infants with severe BPD who are exposed to postnatal steroids.

Objectives/Goal: To compare the cognitive, language and motor scores among 3 groups of severe BPD infants who received early vs late vs no tracheostomy. Secondly, evaluate if postnatal steroids had an additive negative effect on neurodevelopmental outcomes.

Methods/Design: IRB approved retrospective …

Efficacy Of Using Antibiotic Cocktail In Medically Refractory Colitis/Inflammatory Bowel Disease, David Aaron Simon

Research Days

Background: Antibiotic cocktail targeting intestinal bacteria may offer a promising approach in certain patients with medically refractory IBD. It may find a place in serving as a bridge to more effective long-term medical therapies and in some cases help in achieving clinical remission in patients with refractory disease as an adjunctive therapy. Its main attractions include efficacy in about 50% of medically refractory patients and a non-immunosuppressive adjunct in treatment of children who may otherwise suffer from complications of untreated disease or heavy immunosuppression. The main drawbacks are lack of understanding of true mechanism of action, potential development of drug …

Bar Removal Following Minimally Invasive Pectus Excavatum Repair – Does Removal At 2 Years Affect Recurrence Or Satisfaction Rates?, Wendy Jo Svetanoff

Research Days

Background: Some patients with pectus excavatum require bar removal despite having the bar in for less than three years after minimally invasive placement (MIRPE). It is unknown whether early removal is associated with a higher recurrence rate or lower cosmetic satisfaction.

Objectives/Goal: The aim of this study was to review post-operative outcomes, specifically recurrence rate, and patient satisfaction in patients who underwent bar removal prior to three years.

Methods/Design: A retrospective review was performed of patients who underwent MIRPE between October 2006 and June 2017 and had bar removal less than 3 years after repair. Demographics, reason for bar removal, …

Constrictive Pericarditis After Repair Of A Ruptured Sinus Of Valsalva, Sarah Studyvin, Laura Kuzava

Research Days

Background: Constrictive pericarditis is an uncommon complication of cardiac surgery. We report a patient who developed constrictive pericarditis after ruptured sinus of Valsalva (RSOV) repair.

Methods: A 23-year-old male presented with exertional dyspnea one year after RSOV repair. TTE showed a small, circumferential effusion with thickened pericardium, ventricular septal bounce, left atrial enlargement, diastolic hepatic flow reversal, and trivial mitral regurgitation without stenosis. He underwent cardiac catheterization, which revealed elevated filling pressures (RVEDP 16 mmHg, LVEDP 18 mmHg), RVEDP/RVSP ratio < 0.5, and a low cardiac index (1.65 L/min/m2). Cardiac MRI confirmed pericardial thickening with paradoxic septal motion, dilated pulmonary veins and retrograde flow in the SVC.

Results: Pericardiectomy of thickened and adherent pericardium was performed. The central venous pressure decreased from 23 to 7 mmHg and …

Prenatal Diagnosis Of An Uncommon Form Of A Hypoplastic Left Heart Syndrome Variant., Anmol Goyal

Research Days

Background: Mitral valve dysplasia syndrome (MVDS) is a rare form of congenital heart disease, similar to hypoplastic left heart syndrome (HLHS). Prenatal identification is important for counseling, delivery planning and postnatal management.

Case: A 39 year-old woman underwent fetal echocardiography at ~34 weeks gestation for evaluation of fetal cardiomegaly and hydrops revealing: biatrial enlargement, mild-to-moderate mitral and tricuspid valve insufficiency, echobright mitral valve apparatus, biventricular dilation, severe LV fibroelastosis (EFE) and systolic dysfunction, mild hypoplasia of aortic valve annulus and aortic arch, and a thick restrictive atrial septum (RAS) with left to right flow (Fig 1a-c).

Decision‐making: Although critical aortic …

The Role Of Fractionated Exhaled Nitric Oxide (Feno) In Eosinophilic Esophagitis And The Relationship With Downstream Eosinophils, Panamdeep Kaur

Research Days

Background: Eosinophilic Esophagitis (EoE) is a chronic immune mediated disease of the esophagus characterized by symptoms of esophageal dysfunction and eosinophilic-predominant inflammation of the esophagus. It can lead to feeding difficulties, failure to thrive, and esophageal stricturing. Currently, the gold standard for diagnosis and assessing response to therapy is upper endoscopy and histopathological analysis of biopsies. A noninvasive, cost-effective, and low risk alternative that can aid in the management of EoE is not currently available. Previous studies assessing correlation of fractionated exhaled nitric oxide (FeNO) with degree of esophageal eosinophilic inflammation were low powered but noted a trend for association. …

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 …

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 …

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 …

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 …