Medicine and Health Sciences Commons^™

Safety And Efficacy Of Voxelotor In Pediatric Patients With Sickle Cell Disease Aged 4 To 11 Years., Jeremie H. Estepp, Ram Kalpatthi, Gerald Woods, Sara Trompeter, Robert I. Liem, Kacie Sims, Adlette Inati, Baba P D Inusa, Andrew Campbell, Connie Piccone, Miguel R. Abboud, Kim Smith-Whitley, Sandra Dixon, Margaret Tonda, Carla Washington, Noelle M. Griffin, Clark Brown

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Sickle cell disease (SCD) is a devastating, multisystemic disorder that affects millions of people worldwide. The earliest clinical manifestations of SCD can affect infants as young as 6 months of age, and pediatric patients are at risk for acute and life-threatening complications. Early intervention with treatments that target the underlying pathophysiological mechanism of SCD, sickle hemoglobin (HbS) polymerization, are expected to slow disease progression and circumvent disease-associated morbidity and mortality.

PROCEDURE: The HOPE-KIDS 1 trial (NCT02850406) is an ongoing four-part, phase 2a, open-label, single- and multiple-dose study to evaluate the pharmacokinetics, efficacy, and safety of voxelotor-a first-in-class HbS polymerization …

Receptor Tyrosine Kinase (Rtk) Targeting In Pediatric High-Grade Glioma And Diffuse Midline Glioma: Pre-Clinical Models And Precision Medicine., Kallen Schwark, Dana Messinger, Jessica R. Cummings, Joshua Bradin, Abed Kawakibi, Clarissa M. Babila, Samantha Lyons, Sunjong Ji, Rodrigo T. Cartaxo, Seongbae Kong, Evan Cantor, Carl Koschmann, Viveka Nand Yadav

Manuscripts, Articles, Book Chapters and Other Papers

Pediatric high-grade glioma (pHGG), including both diffuse midline glioma (DMG) and non-midline tumors, continues to be one of the deadliest oncologic diagnoses (both henceforth referred to as "pHGG"). Targeted therapy options aimed at key oncogenic receptor tyrosine kinase (RTK) drivers using small-molecule RTK inhibitors has been extensively studied, but the absence of proper in vivo modeling that recapitulate pHGG biology has historically been a research challenge. Thankfully, there have been many recent advances in animal modeling, including Cre-inducible transgenic models, as well as intra-uterine electroporation (IUE) models, which closely recapitulate the salient features of human pHGG tumors. Over 20% of …

Characterization Of The Mucosally-Adherent Duodenal Microbiome In Children With And Without Crohn's Disease., Kenneth Schmidt, Janelle R. Noel-Macdonnell Phd, Carrie Vyhlidal, Daniel P. Heruth, Vivekanand Singh, Atif A. Ahmed, Taina Hudson, Veronica Williams, Valentina Shakhnovich

Manuscripts, Articles, Book Chapters and Other Papers

Manipulation of the microbiome is a rational treatment strategy for inflammatory bowel disease (IBD). Compared to the colon and terminal ileum (TI), understanding of the microbial composition in the duodenum is sparse. This gap in knowledge is especially significant for children with Crohn's disease (CD) because the prevalence of duodenal CD is higher in children than in adults. Our aim was to characterize the bacterial composition of the mucosally-adherent duodenal microbiome in children with and without CD as a first step toward development of targeted IBD treatment strategies at this disease location. Fresh-frozen mucosal biopsies were obtained from the duodenum …

Utility Of Av Nodal Characteristics In Identification Of Atrioventricular Nodal Reentrant Tachycardia And Risk Of Recurrence, Kimberly M. Decarr

Longitudinal Scholar's Project

Background: Catheter ablation of the slow atrioventricular (AV) nodal pathway is a safe and effective treatment for atrioventricular nodal reentrant tachycardia (AVNRT). While AVNRT occurs frequently in both children and adults, pediatric patients experience higher rates of AVNRT recurrence.

Objective: The purpose of this study was to characterize changes in AV nodal conduction properties following catheter ablation and correlate these properties with AVNRT recurrence in pediatric patients.

Methods: This was a retrospective review of patients aged(AVRT) at a single tertiary care center between January 01, 2010 and September 01, 2021. Demographics, pre- and post-ablation ECG intervals, and AV nodal conduction …

Infant Prostatic Rhabdomyosarcoma: A Diagnostic And Therapeutic Challenge, Sohail Dhanji, Leonardo D Borregales, Nicolette K Janzen, Guilherme Godoy, Maren Y Fuller, Jason K Au

Student and Faculty Publications

The following case report describes a case of prostatic rhabdomyosarcoma in a 6-month-old male who presented with urinary retention and constipation. MRI showed a prostatic mass that was displacing the rectum and bladder, leading to bladder outlet obstruction. A suprapubic tube was placed for urinary diversion and a transvesical approach was used for tissue diagnosis. Biopsy confirmed the diagnosis of prostatic rhabdomyosarcoma. Patient underwent chemotherapy regiment with VAC (vincristine, actinomycin D and cyclophosphamide) and subsequently ifosfamide and doxorubicin. Eventually, due to tumor progression, the patient underwent a radical cystoprostatectomy with pelvic lymph node dissection and ileal conduit.

Rural-Urban Trends In Congenital Heart Disease-Related Mortality In The United States, 1999 To 2019, Abdul Mannan Khan Minhas, Rachel A. Wyand, Robert W. Ariss, Salik Nazir, Vardhmaan Jain, Sadeer G. Al-Kindi, Michael D. Shapiro, William Campbell, Laurence Sperling, Salim S. Virani

Office of the Provost

No abstract provided.

Using Artificial Neural Networks To Predict Intra-Abdominal Abscess Risk Post-Appendectomy, Morouge M Alramadhan, Hassan S Al Khatib, James R Murphy, Kuojen Tsao, Michael L Chang

Student and Faculty Publications

OBJECTIVE: To determine if artificial neural networks (ANN) could predict the risk of intra-abdominal abscess (IAA) development post-appendectomy.

BACKGROUND: IAA formation occurs in 13.6% to 14.6% of appendicitis cases with "complicated" appendicitis as the most common cause of IAA. There remains inconsistency in describing the severity of appendicitis with variation in treatment with respect to perforated appendicitis.

METHODS: Two "reproducible" ANN with different architectures were developed on demographic, clinical, and surgical information from a retrospective surgical dataset of 1574 patients less than 19 years old classified as either negative (n = 1,328) or positive (n = 246) for IAA post-appendectomy …

Clinical Application And Feasibility Of Utilizing The Pedi-Cat To Assess Activity And Participation Among Children Receiving Physical Therapy Incorporating Hippotherapy, Susan Conroy, Trish Evans, Dana Butler-Moburg, Richard Beuttler, Janelle Robinson, Matt Huebert, Erin O. Mahoney, Marybeth Grant-Beuttler

Pharmacy Faculty Articles and Research

Background

Hippotherapy (HPOT) is a physical therapy (PT) treatment tool using equine movement to improve mobility for children with movement impairments. Although research suggests HPOT improves body structure and function, there is limited evidence regarding its impact on activity and participation outcomes in a clinical setting. The Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT) may be useful in HPOT settings to highlight changes in activity and participation.

Purpose

1) Evaluate the PEDI-CAT’s sensitivity to changes in activity and participation among children receiving PT using HPOT; 2) determine feasibility of administering the PEDI-CAT in a HPOT setting; and 3) …

Analyzing Relationships Between Economic And Neighborhood-Related Social Determinants Of Health And Intensive Care Unit Length Of Stay For Critically Ill Children With Medical Complexity Presenting With Severe Sepsis, Hunter Hamilton, Alina N West, Nariman Ammar, Lokesh Chinthala, Fatma Gunturkun, Tamekia Jones, Arash Shaban-Nejad, Samir H Shah

Student and Faculty Publications

OBJECTIVES: Of the Social Determinants of Health (SDoH), we evaluated socioeconomic and neighborhood-related factors which may affect children with medical complexity (CMC) admitted to a Pediatric Intensive Care Unit (PICU) in Shelby County, Tennessee with severe sepsis and their association with PICU length of stay (LOS). We hypothesized that census tract-level socioeconomic and neighborhood factors were associated with prolonged PICU LOS in CMC admitted with severe sepsis in the underserved community.

METHODS: This single-center retrospective observational study included CMC living in Shelby County, Tennessee admitted to the ICU with severe sepsis over an 18-month period. Severe sepsis CMC patients were …

Advantages Of Early Utilization Of Palliative Care In The Pediatric Icu: A Scoping Review, Shelby Noel Cain Bsn, Rn, Cpn, Dana Pabalate Zimmerman Msn, Rn, Charis Elizabeth Oji Msn, Aprn, Cpnp-Pc/Ac, Michelle Rickard Dnp, Cpnp-Ac

Doctor of Nursing Practice Projects

Abstract Purpose/Background Despite the increased use of palliative care in adults, only a tiny percentage of children with life-limiting illnesses or life-threatening conditions receive palliative care before dying. Palliative care has low utilization, and consults are often made late in a child’s hospitalization or diagnosis. Palliative care remains underutilized among hospitalized pediatric patients resulting in unnecessary treatments, costly and lengthy hospitalizations, and deaths occurring in the intensive care unit (ICU) rather than at home. The purpose of this scoping review is to evaluate current literature to determine the use and effect of pediatric palliative care, specifically, whether consultation of palliative …

Pain Management In Postoperative Pediatric Craniotomy Patients: A Scoping Review, Jessica M. Hiltenbrand Bsn, Rn, Sydney A. Scarbrough Bsn, Rn, Kaitlyn E. Shirley Bsn, Rn, Michelle Rickard Dnp, Cpnp-Ac

Doctor of Nursing Practice Projects

Purpose/Background The pediatric craniotomy procedure is a complex and painful procedure that requires adequate pain management and frequent neurological assessments. Generally, evidence for pediatric pain management is severely lacking in research, and evidence for pediatric craniotomy patients is lacking even further. This scoping review aims to collect, condense, and review the current data in order to fuel future studies to establish more data, protocols, and evidence-based recommendations for analgesia in the pediatric craniotomy patient population.

Methods The studies selected were peer-reviewed, published in a medical or nursing journal, included study ages 0-21 years, or provided evidence on pediatric analgesia. Sources …

Characteristics And Predictors Of Intensive Care Unit Admission In Pediatric Blunt Abdominal Trauma, Steven C Mehl, Megan E Cunningham, Christian J Streck, Rowland Pettit, Eunice Y Huang, Matthew T Santore, Kuojen Tsao, Richard A Falcone, Melvin S Dassinger, Jeffrey H Haynes, Robert T Russell, Bindi J Naik-Mathuria, Shawn D St Peter, David Mooney, Jeffrey Upperman, Martin L Blakely, Adam M Vogel

Student and Faculty Publications

BACKGROUND: Pediatric trauma patients sustaining blunt abdominal trauma (BAT) with intra-abdominal injury (IAI) are frequently admitted to the intensive care unit (ICU). This study was performed to identify predictors for ICU admission following BAT.

METHODS: Prospective study of children (< 16 years) who presented to 14 Level-One Pediatric Trauma Centers following BAT over a 1-year period. Patients were categorized as ICU or non-ICU patients. Data collected included vitals, physical exam findings, laboratory results, imaging, and traumatic injuries. A multivariable hierarchical logistic regression model was used to identify predictors of ICU admission. Predictive ability of the model was assessed via tenfold cross-validated area under the receiver operating characteristic curves (cvAUC).

RESULTS: Included were 2,182 children with 21% (n = 463) admitted to the ICU. On univariate analysis, ICU patients were associated with abnormal age-adjusted shock index, increased injury severity scores (ISS), lower Glasgow coma scores (GCS), traumatic brain injury (TBI), and severe solid organ injury (SOI). With multivariable logistic regression, factors associated with ICU admission were severe trauma (ISS > 15), …

Comprehensive Evaluation Of Left Ventricular Deformation Using Speckle Tracking Echocardiography In Normal Children: Comparison Of Three-Dimensional And Two-Dimensional Approaches., Doaa Aly, Nitin Madan, Laura Kuzava, Alison Samrany, Anitha Parthiban

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Three-dimensional (3D) speckle tracking echocardiography (STE) can overcome some of the inherent limitations of two-dimensional (2D) STE; however, clinical experience is lacking. We aimed to assess and compare the feasibility, agreement, and reproducibility of left ventricular (LV) global longitudinal (GLS), and regional strain by 3D vs 2D STE in normal children.

METHODS: Healthy pediatric subjects (n = 105, age mean = 11.2 ± 5.5 years) were prospectively enrolled. Three-dimensional and 2D LV GLS, as well as regional strain in 16 myocardial segments were quantified. Bland Altman analysis, intra- class correlation coefficients (ICC), percent error and linear regression were used …

Frequency Of Diabetes Team Contacts In Children And Adolescents Using Insulin Pumps, Funmbi T. Babalola, Michael R. Miller, Andrea R. Ens, Robert I. Stein, Patricia H. Gallego, Cheril L. Clarson

Paediatrics Publications

Background: The purpose of this study is to assess if a relationship exists between A1c within target (≤ 7.5%) and frequency of patient-initiated contact with diabetes team, in children with type 1 diabetes (T1DM) on an insulin pump. Additionally, to determine factors impacting frequency of contact. Methods: This was a retrospective study of children with T1DM on an insulin pump. Frequency of contact, type of contact, and A1c were collected. Study participants filled out a questionnaire at study entry. Results: One hundred and seventy-six participants were enrolled, with a mean age of 13 years. The median duration of T1DM was …

Real-World Evidence Of An Emollient Device For Atopic And Contact Dermatitis In Pediatric To Adult Patients - Data From A Post-Marketing Surveillance, Adelaide Ann Hebert

Faculty and Staff Publications

PURPOSE: Atopic and contact dermatitis have been attributed to skin barrier abnormalities, immune system dysfunction and inflammation leading to pruritus. As these factors may involve oxidative stress, emollient devices containing antioxidants furfuryl palmitate and tocopherol may help reduce itching and inflammation. In this study, a post-marketing questionnaire was carried out of 40 users of a novel emollient device containing furfuryl palmitate and tocopherol (Relizema™ cream), who purchased it from a pharmacy or health-care professional to ascertain the emollient device's action on itching, flushing and moisturizing.

PATIENTS AND METHODS: The post-marketing questionnaire, administered by trained pharmacy and health-care staff, collected data …

Preventive Training Program Feedback Complexity, Movement Control, And Performance In Youth Athletes, Hayley J. Root, Eleanor M. Beltz, Julie P. Burland, Jessica C. Martinez, R. Curt Bay, Lindsay J. Distefano

Rehabilitation Sciences Faculty Publications

Context: Preventive training programs (PTPs) reduce injury risk by improving movement control. Corrective feedback is important; however, many cues at once may be too complicated for athletes.

Objective: To compare movement control and long-jump (LJ) changes in youth athletes participating in a season-long PTP, with simplified feedback, traditional feedback, or a warmup of the coaches' choosing.

Design: Cluster-randomized controlled trial.

Setting: Soccer fields.

Patients or Other Participants: A total of 420 athletes (simplified feedback = 173, traditional feedback = 118, and control = 129; age = 11 ± 3 years).

Intervention(s): Teams were randomized into the simplified PTP, traditional PTP, …