Pediatrics Commons^™

Hematopoietic Stem Cell Transplantation Outcomes For High-Risk Aml: A Report From The Children's Oncology Group., Benjamin J. Huang, Lauren K. Meyer, Todd A. Alonzo, Yi-Cheng Wang, Adam J. Lamble, Rhonda E. Ries, Weijie Wang, Betsy Hirsch, Gordana Raca, Xiaotu Ma, Alan S. Gamis, Richard Aplenc, E Anders Kolb, Todd M. Cooper, Katherine Tarlock, Michael R. Loken, Soheil Meshinchi, Joseph H. Chewning, William G. Woods, John T. Horan

Manuscripts, Articles, Book Chapters and Other Papers

PURPOSE: Hematopoietic stem cell transplantation (HSCT) is used as consolidation for pediatric patients with high-risk AML in first complete remission (CR1). The definition of high-risk AML has evolved considerably over the past two decades with the successive identification of new unfavorable risk factors. We conducted a cross-study analysis to determine whether HSCT improves the outcomes of patients with contemporarily defined high-risk AML.

METHODS: We combined data from AAML0531 and AAML1031, the last two phase III clinical trials completed by the Children's Oncology Group (COG). These two trials established the prognostic importance of measurable residual disease (MRD) and several high-risk cryptic …

Parallel Use Of Low-Complexity Automated Nucleic Acid Amplification Tests And Lateral Flow Urine Lipoarabinomannan Assays To Detect Tuberculosis Disease In Adults And Adolescents Living With Hiv, Stephanie Bjerrum, Bada Yang, Johanna Åhsberg, Laura Olbrich, Mathias Weis Damkjær, Ruvandhi R Nathavitharana, Tobias Broger, Ioana Diana Olaru, Brittney Sweetser, Hayley Poore, Alia Razid, Alexander W Kay, Claudia M Denkinger, Ian Schiller, Nandini Dendukuri, Devan Jaganath, Andreas Lundh, Maunank Shah

Faculty, Staff and Students Publications

Background: Low-complexity automated nucleic acid amplification tests (LC-aNAATs) are molecular World Health Organization (WHO)-recommended rapid diagnostic tests (also known as mWRDs) widely used to diagnose tuberculosis disease. The lateral flow urine lipoarabinomannan assay (LF-LAM) is recommended by the WHO to assist in diagnosing tuberculosis disease amongst people with HIV. Previous systematic reviews have assessed the diagnostic accuracy of LC-aNAATs and LF-LAM used in isolation for the detection of tuberculosis, but in clinical practice the tests may be used in parallel (i.e. LC-aNAAT in combination with LF-LAM).

Objectives: To compare the diagnostic accuracy of the parallel use of LC-aNAAT on respiratory …

Real-World Outcomes For Young Adult Patients Receiving Cd19 Car T-Cell Therapy., Hannah Lust, Liora M. Schultz, Soyang Kwon, Gregory W. Roloff, Ibrahim Aldoss, Christina Baggott, Samuel John, Jenna E. Rossoff, Kevin O. Mcnerney, Vanessa A. Fabrizio, Julie-An Talano, Amy Moskop, Kevin J. Curran, Christine L. Phillips, Nicole Karras, Susanne H C Baumeister, Stacy L. Cooper, Michelle Hermiston, Prakash Satwani, Muna Qayed, Sunil S. Raikar, Margaret Macmillan, Erin Hall, Khanh Nguyen, Ryan D. Cassaday, Noam E. Kopmar, Vamsi K. Kota, John Mathews, Paul Shaughnessy, Marc S. Schwartz, Abdullah Ladha, George Yaghmour, Muthu V. Kumaran, Veronika Bachanova, Sean Tracy, Tamer Othman, Marlise R. Luskin, Evan C. Chen, Anjani S. Advani, Nikeshan Jeyakumar, Katharine Miller, Amy Zhang, Katherine C. Sutherland, Bijal D. Shah, Lori Muffly, Rawan Faramand

Manuscripts, Articles, Book Chapters and Other Papers

Tisagenlecleucel (tisa-cel) and brexucabtagene autoleucel (brexu-cel) are approved CD19 chimeric antigen receptor T-cell therapy (CAR T) products for young adults (YA) with relapsed/refractory B-cell acute lymphoblastic leukemia. A distinct analysis of YAs receiving commercial CD19 CAR T has not been reported. Using retrospective data from the Pediatric Real-World CAR T Consortium and the Real-World Outcomes of CAR T in Adult ALL collaboration, we describe the efficacy and safety of tisa-cel and brexu-cel in 70 YAs (18-26 years; tisa-cel, n = 50; brexu-cel, n = 20). Cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) were observed more frequently …

Adolescent Scoliosis In Autism Spectrum Disorder: Is It Idiopathic Or Syndromic??, Ravi Rajendra, Matthew B. Bratton, Claudia Leonardi, R. Carter Clement

School of Medicine Faculty Publications

Purpose: Minimal data exists on whether adolescents with both autism spectrum disorder (ASD) and scoliosis exhibit distinct curve types or require varying surgical management compared to neurotypical adolescents. Our study at a high-volume scoliosis center compares patterns of scoliosis in ASD patients with those of neurotypical patients. We hypothesized adolescents with ASD would present with atypical scoliosis curve characteristics compared to neurotypical adolescents. Methods: Using ICD-10 codes, we constructed an electronic database of adolescents aged 10 to 18 with diagnoses of both scoliosis and ASD. To avoid confounding with syndromic conditions, adolescents with a syndrome independently associated with scoliosis were …

Childhood Gastroesophageal Reflux Disease: A Comprehensive Review Of Disease, Diagnosis, And Therapeutic Management, Daniyal Raza, Farhan Mohiuddin, Muhammad Haris Khan, Maheen Fawad, Syed Musa Raza

School of Medicine Faculty Publications

Gastroesophageal reflux disease (GERD) affects both adults and children, although the symptoms differ significantly between these groups. While adults typically experience heartburn and regurgitation, children may present with more subtle signs, such as failure to thrive, chronic cough, wheezing, and Sandifer syndrome. Diagnosing GERD in children necessitates a multifaceted approach due to the diverse symptomatology and challenges in communication. Clinical assessment serves as the cornerstone of diagnosis, supported by tools like pH monitoring, esophageal impedance testing, and upper gastrointestinal endoscopy. Imaging studies, such as barium swallow, can also provide valuable insights into anatomical abnormalities and the extent of reflux. Treatment …

Oxygen Saturation Targeting In The Neonatal Intensive Care Unit, Faeq Almudares, Bheru Gandhi, Jonathan Davies, Xanthi Couroucli, Natalie Villafranco, Nidhy Paulose Varghese, Milenka Cuevas Guaman, Charleta Guillory, Binoy Shivanna

Faculty, Staff and Students Publications

Oxygen (O2) is vital for cellular development, function, proliferation, and repair, underscoring its critical role in organogenesis. Both hypoxia (reduced tissue O2) and hyperoxia (excess tissue O2), when prolonged, can trigger inflammation and oxidative stress, contributing to acute and long-term cardiopulmonary and neurodevelopmental morbidity. In sick neonates, immature defense mechanisms and coexisting morbidities demand nuanced and sometimes opposing strategies for O2 saturation targets and therapeutic titration. Most current neonatal O2 targeting guidelines are based on animal models or small clinical studies, resulting in recommendations with limited evidence. This narrative review aims to provide an updated overview of the physiological roles …

Emerging Therapeutic Potential Of Fisetin For Nephrotoxicity, Kidney Injury, And Nephropathy: A Systematic Review, Saeed Mohajeri, Alizamen Salehifard Jouneghani, Saeid Heidari-Soureshjani, Catherine M.T. Sherwin, Faramarz Beigi

Pediatrics Faculty Publications

INTRODUCTION/OBJECTIVE: Kidney diseases cause high morbidity and mortality worldwide. This study investigated the mechanistic effects of Fisetin (FIS) on nephrotoxicity, kidney injury, and nephropathy induced by drugs, toxic chemicals, diabetes, lupus, diet, ureteral obstruction, and ischemic situations.

METHODS: To identify pertinent articles published before Oct 1, 2024, a comprehensive electronic search was performed across several databases, including MEDLINE/PubMed, Embase, Cochrane Library, Web of Science, and Scopus. After establishing clear inclusion and exclusion criteria, studies that met the research objectives were selected. Data were extracted and analyzed, documenting study characteristics, methodologies, and biological mechanisms.

RESULTS: Antioxidant benefits were evident with increased …

Beyond The Surface: Mapping Dde's Metabolic Footprint On Adolescent Obesity, Zhenjiang Li, Shudi Pan, Brittney O Baumert, Jiawen Carmen Chen, Jesse A Goodrich, Hongxu Wang, Sarah Rock, Justin Ryder, Damaskini Valvi, Todd Jenkins, Stephanie Sisley, Xiangping Lin, Scott M Bartell, Thomas H Inge, Stavra Xanthakos, Brooklynn Mcneil, Anna R Robuck, Catherine E Mullins, Sandrah P Eckel, Rob S Mcconnell, Michele A La Merrill, Douglas I Walker, David V Conti, Lida Chatzi

Faculty, Staff and Students Publications

Background: Bariatric surgery is an intervention for severe obesity, leading to significant weight loss and metabolic improvements. However, the release of lipophilic chemicals accumulated in adipose tissue during weight loss presents a unique clinical challenge and research opportunity. Dichlorodiphenyldichloroethylene (DDE) is a persistent organic pollutant increasingly recognized as obesogen, while the biological mechanisms through which DDE influences body mass index (BMI) and waist circumference remain underexplored.

Objectives: We aimed to identify metabolic signatures mediating the association between DDE exposure and weight loss by plasma and adipose tissue metabolomics.

Methods: We conducted a longitudinal study involving 60 adolescents with severe obesity …

A Novel Cardiomyopathy Phenotype Linked To A Chd7 Missense Variant, In Young Park, Chih-Wei Hsu, Karim Bouazoune, Christina E Espindola, Madeline Hannah Mclaughlin Armond, Cristian Coarfa, Sandra L Grimm, James F Martin, Donna M Martin, Cheryl Lyn Walker

Center for Medical Ethics and Health Policy Staff Publications

Loss of function in the chromatin remodeler CHD7 causes CHARGE syndrome, characterized by variable penetrance and diverse abnormalities. However, establishing genotype-phenotype correlations has been challenging, as most CHD7 inactivating mutations are null alleles. Through CHD7 missense variant analysis at potential phosphorylation sites, we identified T730 (T720 in mice) as a critical residue associated with pathogenesis. Using a CHD7 T730 missense variant (Chd7T720A) and a frameshift null allele (Chd7fs) in a mouse model, we found that Chd7fs/fs mice were non-viable, while Chd7fs/+ mice exhibited haploinsufficiency-related circling behavior. Notably, Chd7fs/T720A mice died before postnatal …

The Use Of Biased Language In The Care Of Seriously Ill Children: A Pilot Study, Terri Major-Kincade, Erin Kim, Cameron A Price, Ankona Banerjee, Nikashia Franklin, Israel Castañeda Almendárez, Jaime Jump, Jared Rubenstein, Jill Ann Jarrell

Faculty, Staff and Students Publications

Background

Objective

Setting/Participants

Cytarabine Pharmacogenomics And Outcomes Among Children And Young Adults With Acute Myeloid Leukemia., Richard J. Marrero, Vivek M. Shastri, Deedra Nicolet, Krzysztof Mrózek, Christopher J. Walker, William G. Blum, Bayard L. Powell, Jonathan E. Kolitz, Joseph O. Moore, Geoffrey L. Uy, Wendy Stock, Andrew J. Carroll, John C. Byrd, Richard Aplenc, Todd M. Cooper, Alan S. Gamis, Huiyun Wu, Stanley Pounds, Yi-Cheng Wang, Todd A. Alonzo, Soheil Meshinchi, Ann-Kathrin Eisfeld, Edward A. Kolb, Jatinder K. Lamba

Manuscripts, Articles, Book Chapters and Other Papers

IMPORTANCE: Therapeutic responses in acute myeloid leukemia (AML) demonstrate considerable variability both across and within established risk stratifications and age groups. Moreover, significant racial disparities persist, with Black patients experiencing inferior survival outcomes compared with their White counterparts.

OBJECTIVE: To validate the association of the previously reported 10 single nucleotide variant (SNV)-based ara-C pharmacogenomics score (ACS10) with survival outcomes in a large cohort of pediatric AML patients; to evaluate whether ACS10 remains relevant in an adolescent and young adult (AYA) population of patients with AML treated with similar intensive induction chemotherapy protocols; and to assess the association of ACS10 with …

Intraosseous Vs Intravenous Access For Epinephrine In Pediatric Out-Of-Hospital Cardiac Arrest, Masashi Okubo, Sho Komukai, Junichi Izawa, Sunhee Chung, Cameron Dezfulian, Francis X Guyette, Joshua R Lupton, Christian Martin-Gill, Sylvia Owusu-Ansah, Sriram Ramgopal, Clifton W Callaway

Faculty, Staff and Students Publications

Importance: While epinephrine is commonly administered in children with out-of-hospital cardiac arrest (OHCA) via an intraosseous (IO) or intravenous (IV) route, the optimal route of epinephrine delivery is unclear.

Objective: To evaluate the association between the route of epinephrine administration (IO or IV) and patient outcomes after pediatric OHCA.

Design, setting, and participants: Retrospective cohort study of pediatric patients (aged < 18 years) with nontraumatic OHCA treated by emergency medical services who received prehospital epinephrine either via an IO or IV route. Patients were included in the Resuscitation Outcomes Consortium Epidemiologic Registry, a prospective OHCA registry at 10 sites in the US and Canada from April 2011 to June 2015. Data analysis was performed from May 2024 to April 2025.

Exposure: Epinephrine administration route: IO or IV route.

Main outcomes and measures: The primary outcome was survival to hospital discharge. The secondary outcome was return of spontaneous circulation (ROSC) before hospital arrival. Propensity scores were calculated and inverse probability of …

A Case Of Early Pediatric-Onset Multiple Sclerosis, Mellanie De Guzman, Marcy Vandament

Posters

Pediatric-onset multiple sclerosis (POMS) is a central nervous system neurodegenerative disease impacting individuals who experience symptom onset prior to the age of 18-years-old.17 Children and adolescents diagnosed with POMS prior to 16-years-old are considered as early POMS cases. 1,6,10,11, 18 The current case study presents an 11-year-old boy, “Patient A,” who was diagnosed with POMS at 10-years-old. Common neurocognitive weaknesses and behavioral concerns seen in this patient population include deficits in language (receptive, expressive, verbal fluency), memory, attention, executive functions, processing speed, visual-motor/fine-motor coordination/dexterity, depression, anxiety, and fatigue.2-5,15,20 Results from testing demonstrated mild weaknesses in abstract reasoning and fine motor …

Mechanisms Of Interventions Targeting Modifiable Factors For Dementia Risk Reduction, Anna Matton, Ruth Stephen, Makrina Daniilidou, Mariagnese Barbera, Vilma Alanko, Marcel Ballin, Jamie Ford, Katri Hemiö, Jenni Lehtisalo, Chinedu Momoh

Brain and Mind Institute

The global prevalence of dementia is increasing. With no widely available and accessible treatments to halt or reverse the progression of dementia, exploring preventative strategies is critical. Lifestyle-based interventions show promise in preventing or delaying dementia onset. However, understanding the complex and multifactorial mechanisms underlying dementia, and how interventions target these pathways, is essential for developing personalized and effective strategies. In this review, we examined the current evidence of the mediating pathways in dementia risk reduction. We focused on mechanisms investigated in single-domain interventions on physical exercise, cognitive training, diet, metabolic/cardiovascular or psycho-social risk factors in line with those combined …

Impact Of Cessation Of Caffeine Citrate Therapy On Intermittent Hypoxemia Patterns Among Preterm Infants Born Before 34 Weeks, Peter Kipkurui Mashep, Roseline Ochieng, Jesse Coleman, Mary Waiyego, Amy Sarah Ginsburg, Dorothy Chomba, Morris Ogero Ondieki, Grace Irimu, William Macharia, Mark Ansermino

Paediatrics and Child Health, East Africa

Introduction: Intermittent hypoxemia (IH) is defined as oxygen saturation (SpO₂) drop ≥5% from the baseline (set at 90 s preceding the event) to a level less than 90% lasting for ≥5 s. Caffeine citrate, the standard of care for apnea of prematurity, reduces IH events. IH contributes to both short and long-term adverse neurologic outcomes. Standard patient monitors cannot detect IH events due to long averaging times.

Objective: Describe change in patterns of IH events in preterms < 34 weeks before and after cessation of caffeine citrate and factors associated.

Methods: Interrupted time series study design. Data was collected from 1 December 2022 to 30 June 2023. MASIMO RAD-G oximeter was …

22q11.2 Enhanced Recovery After Surgery Pathway (Eras), Children's Mercy Kansas City

Clinical Pathways

No abstract provided.

Small And Sick Newborn Care: Changes In Service Readiness Scoring Between Baseline And 2023 For 65 Neonatal Units Implementing With Nest360 In Kenya, Malawi, Nigeria, And Tanzania, Rebecca E. Penzias, Morris Ondieki Ogero, Robert Tillya, Irabi Kassim, Olabisi Dosunmu, Opeyemi Odedere, Hannah Mwaniki, Vincent Ochieng, Dolphine Mochache, William Macharia

Paediatrics and Child Health, East Africa

Health Facility Assessments (HFAs) are important for measuring and tracking service readiness for small and sick newborn care (SSNC). NEST360 Alliance aims to reduce neonatal mortality in four countries (Kenya, Malawi, Nigeria, Tanzania). NEST360 and UNICEF facilitated HFA tool design with ministries of health in four African countries and developed two complimentary approaches to summarize readiness. Using the NEST360/UNICEF HFA tool, we collected data, developed two service readiness scoring approaches for SSNC (standards-based scoring by adapted World Health Organization (WHO) health system building blocks (HSBBs) and assessing service readiness across the health system, and level-2+ scoring by WHO clinical interventions), …

Prenatal Substance Exposure And Positive Developmental Delay Screening Among Patients In Foster Care, Lauren Malthaner, Jill Mcleigh, Gregory Knell, Katelyn Jetelina, Folefac Atem, Sarah Messiah

Behavioral Health/Developmental Delay

Prenatal substance exposure (PSE) is a known risk factor for negative birth outcomes and long-term health outcomes like neurodevelopmental problems. Children in foster care have increased exposure to PSE and higher proportions of developmental delay compared with the general population. It is unclear whether differences still exist among developmental delay screening among children in foster care with and without PSE.

For questions about this study or others, please email [email protected]

childrens.com/fostercare

Supplementation With Ready-To-Use Therapeutic Food Has No Effect On Adverse Outcomes Among Undernourished Children Aged 6–59 Months With Severe Pneumonia, Damalie Nalwanga, Elisa Giallongo, Victor Musiime, Sarah Kiguli, Peter Olupot-Olupot, Florence Alaroker, Robert Opoka, Abner Tagoola, Mainga Hamaluba, Christabel Mogaka

Paediatrics and Child Health, East Africa

Objectives: To investigate the effect of supplementation with ready-to-use therapeutic food (RUTF) on adverse outcomes among undernourished children aged 6–59 months with severe pneumonia.

Methods: This secondary analysis of the COAST-Nutrition (ISRCTN10829073) included children hospitalized for severe pneumonia in Uganda and Kenya. Undernutrition was defined as having either a weight-for-age z score, height-for-age z score, or weight-for-height/length z score below the median of the WHO reference population (< 0) or mid-upper arm circumference (MUAC) below 13.5 cm. Participants were randomized to receive 1 sachet of RUTF daily for 8 weeks in addition to the usual diet (intervention) or usual diet alone (control). The primary composite outcome for adverse events was any one of mortality, re-admission, or deterioration of nutritional status by day 90 of follow-up.

Results: Of 846 main trial participants, 741 (88%) met the inclusion criteria (intervention: 374 versus control: 367). Of 687 (93%) participants in whom the primary outcome was assessed, 370 …

Down Syndrome In Africa: Challenges, Opportunities, And Future Directions, Eimear Mcglinchey, Salina Tewolde, Amanda He, Juan Fortea, Lingani Mbakile-Mahlanza, Ayda Tefera, Emmanuel Epenge, Jason Hassenstab, Rachel Maina, Zul Merali

Brain and Mind Institute

INTRODUCTION: As life expectancy improves for people with Down syndrome (DS) in Africa, the risk of developing DS-associated Alzheimer’s disease (DSAD) will rise. There is a pressing need to plan for this emerging challenge, particularly in the context of existing health and social disparities.

METHODS: This work emerged from a pan-African collaboration, including discussions at the Brain Ageing and Dementia in Low- and Middle-Income Countries conference held in Nairobi in 2024, where stakeholders identified regional priorities for DS and dementia care.

RESULTS: Limited epidemiological, cognitive, biomarker data, delayed diagnoses, and gaps in specialized services may impact access …

Scoring Large Muscle Movements In Pediatric Sleep Studies: An Educational Module For Sleep Technologists, Shauna Michelle Vandoren, David G. Ingram

Posters

Introduction Restless Sleep Disorder (RSD) has recently been recognized as a distinct sleep disorder, with Large Muscle Movement (LMM) scoring being a crucial component of its diagnostic criteria. This project aimed to develop and implement an online educational module to enhance the knowledge and skills of sleep technologists to accurately score LMMs. Methods We constructed an online learning module that included training on the identification and scoring of LMMs in pediatric sleep studies. The module consisted of a 10-question pre-assessment, the educational content (which explained the background of RSD diagnosis, the role of LMMs in diagnostic criteria, and current scoring …

When Macroglossia Is Not Beckwith-Wiedemann: A Case Of An Infant With Congenital Central Hypoventilation Syndrome, Shauna Michelle Vandoren, Samira Naime

Posters

Introduction Congenital central hypoventilation syndrome (CCHS) is a rare genetic disorder caused by the mutation of the paired-like homeobox 2B (PHOX2B) gene that leads to alveolar hypoventilation during sleep and at times while awake. The majority of patients with CCHS have polyalanine repeat mutations (PARM) whereas 10% have non-PARM (NPARM). Of those with NPARM, less than 1% are caused by whole gene deletion. NPARM is associated with more severe phenotypes; however, some may have variable penetrance causing variable presentation within families. Report of case(s) Our patient is a 4-month-old infant born at 32 weeks in an outside hospital and transferred …

Twenty-Five Years Of Palivizumab: A Global Historic Review Of Its Impact On The Burden Of Respiratory Syncytial Virus Disease In Children, Xavier Carbonell-Estrany, Eric A F Simões, Louis Bont, Paolo Manzoni, Heather J Zar, Anne Greenough, Octavio Ramilo, Renato Stein, Barbara Law, Asuncion Mejias, Manuel Sanchez Luna, Paul A Checchia, Leonard Krilov, Marcello Lanari, Ron Dagan, Brigitte Fauroux, Bernhard Resch, Terho Heikkinen, Joseph B Domachowske, Joanne G Wildenbeest, Federico Martinon-Torres, Richard Thwaites, Merih Cetinkaya, Adel S Alharbi, Carlos E Rodriguez-Martinez, Daniel E Noyola, Asiah Kassim, Satoshi Kusuda, Ji-Man Kang, Barry Rodgers-Gray, Anna Platonova, Fungwe Jah, Bosco Paes

Faculty, Staff and Students Publications

Introduction: Respiratory syncytial virus (RSV) causes significant morbidity and mortality in young children. For 25 years, palivizumab has been the only effective pharmaceutical RSV preventive.

Areas covered: We summarize the development and a quarter-century of real-world evidence with palivizumab. We highlight its positive impact on the burden of RSV in high-risk children. Based on lessons learnt from its implementation, we suggest strategies for effective and equitable deployment of newer RSV preventives.

Expert opinion: Following failure of the formalin-inactivated RSV vaccine in 1967, RSV intravenous immunoglobulin was approved in 1996 after three decades' research. Subsequently, palivizumab emerged as the most effective …

Point-Of-Care Ultrasound For Guiding Pulmonary Artery Catheter Placement, Erik Su, Saul Flores

Faculty, Staff and Students Publications

No abstract provided.

Severe Gastrointestinal Manifestations In Childhood-Onset Lupus: A Single-Center Cohort And Review Of The Literature, James Orozco, Jessica Nguyen, Sara Mm Yasrebi, Marnie Blalock, Sara L Grisales, Craig L Jensen, Marietta M Deguzman

Faculty, Staff and Students Publications

Objective

Systemic lupus erythematosus (SLE) is a complex autoimmune condition with diverse manifestations. Childhood-onset SLE (cSLE) presents more severely than adult-onset disease with a higher incidence of severe neurologic or renal manifestations and lower estimated survival rates. There is limited literature on the range of severe gastrointestinal manifestations at presentation in cSLE, and this lack of information results in underappreciation of these potentially life-threatening complications and little guidance regarding management of these patients.

Methods

We reviewed cases of patients with cSLE and severe GI manifestations at presentation diagnosed at our institution and additionally provide a comprehensive review of existing cases …

Are Education, Socioeconomic Status, And Household Size Dependent Variables Influencing The Developmental Origins Of Inflammatory Bowel Disease?, Richard Kellermayer

Faculty, Staff and Students Publications

No abstract provided.

Design Of The Type 1 Diabetes And Life (T1dal) Pilot And Feasibility Study: A Brief Telehealth Intervention Targeting Health-Related Quality Of Life Across Clinical Settings, Marisa E Hilliard, Wendy Levy, Ruth S Weinstock, Korey K Hood, Paula M Trief, Daniel J Desalvo, Yuliana Rojas, Kyrah Holland, Aika K Schneider-Utaka, Selma A Alamarie, Lynn Agostini, Se-Kang Kim, Maartje De Wit, Meghan E Mcgrady, Laurel H Messer, Barbara J Anderson

Faculty, Staff and Students Publications

Background: Type 1 diabetes (T1D) management is demanding and can impact quality of life among persons with diabetes (PWDs) and their family members. Behavioral intervention research has largely focused on adolescents, and previous interventions that have benefitted quality of life have limited potential for implementation in routine care. This trial is piloting a brief behavioral intervention that targets health-related quality of life (HRQOL), which can be implemented with PWDs of all ages in a range of clinical care settings. The aims are to (1) evaluate intervention feasibility and acceptability, (2) explore pre-post change in psychosocial and clinical outcomes, and (3) …

Sci – U: Validation Of An Updated Self-Care Inventory For Contemporary Diabetes Management In Adolescents With Type 1 Diabetes, Sarah S Jaser, Tamim A Hamdan, Marisa E Hilliard, Joyce P Yi-Frazier, Randi Streisand, Faisal S Malik, Daniel J Desalvo, Maeve B O'Donnell, Annette M La Greca

Faculty, Staff and Students Publications

Aims: The Self-Care Inventory is a widely used measure to assess diabetes self-management behaviors. We sought to adapt and streamline the measure to reflect advances in diabetes management, including increased use of continuous glucose monitor and automated insulin delivery systems.

Methods: Through an expert-driven, iterative process, the updated Self-Care Inventory (SCI-U) was created by modifying items to reflect modern diabetes management and reducing the measure to 8 items. The measure was administered to 369 adolescents with type 1 diabetes in 4 regions of the United States, at baseline of two separate intervention trials (mean age = 15.5 ± 1.5, 56 …

Mitral Annular Disjunction Is Associated With Ventricular Arrhythmias In Children With Marfan Syndrome, Carlos A Sanchez Parra, Ruthie M Vaughan, Alejandra Iturralde Chavez, Christina Y Miyake, Santiago O Valdes, Ricardo H Pignatelli, Anitha Parthiban, Shaine A Morris, Tam T Doan

Faculty, Staff and Students Publications

No abstract provided.

Plasma Microbial Cell-Free Dna Sequencing For The Detection Of Kingella Kingae Pediatric Spinal Infections, Asim A Ahmed, Sarah Y Park, Matthew J Smollin, Martin S Lindner, Adriana Sarmiento Clemente, Ana Del Valle Penella, P Marcelo Laufer, Carolina Sanchez-Vegas, Manuel R Cotilla, Marian E Melish, Connie Trieu, Nazia A Kabani, Joshua Cooper, Audrey R Lloyd, David W Kimberlin, John C Arnold, Alejandro Jordan-Villegas, Lori E Patterson, Catherine E Foster, Pablo Yagupsky

Faculty, Staff and Students Publications

Background: Diagnosis of Kingella kingae skeletal system infections is made challenging by the microbe's fastidious nature. Detection and quantification of circulating microbial cell-free DNA (mcfDNA) in plasma by the Karius Test, a commercial metagenomic sequencing test, may offer promise in diagnosing pediatric spinal infections caused by difficult-to-culture organisms such as K. kingae .

Methods: Plasma mcfDNA sequencing detections of K. kingae from April 2018 to December 2020 were reviewed to identify pediatric (age < 18 years) patients. Medical charts of those with spinal infections were reviewed, and mcfDNA sequencing diagnostic performance was compared with usual care tests (ie, cultures, polymerase chain reaction).

Results: Ten children with K. kingae spinal infections were identified across 7 institutions. The median age was 16.5 months (range 11-23 months). All case-patients had vertebral osteomyelitis …