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 …

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) …

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 …

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 …

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, …