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Therapy With Double-Bright Natural Killer Cell Infusions Increases Response Rates Among Frail Patients With Acute Myeloid Leukemia
 Therapy With Double-Bright Natural Killer Cell Infusions Increases Response Rates Among Frail Patients With Acute Myeloid Leukemia 969 533 Jessica Nye, PhD
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Illustration of NK Cells, T Cells, other immune cells floating across white background

A case series of 13 patients with relapsed/refractory acute myeloid leukemia (AML) finds that a novel approach using repeated infusions of cultured natural killer (NK) cells increases overall and complete response rates. NK cells are lymphocytes – white blood cell components of the innate immune response – that have anti-leukemic properties. Although efforts to incorporate…

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Repeated Injury to Stem Cells Could be a Significant Contributor to Chronic Lung Disease
 Repeated Injury to Stem Cells Could be a Significant Contributor to Chronic Lung Disease 1024 575 Abbie Roth
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Illustration of lungs on blue silhouette of upper chest on black background

A series of experiments shows that biological aging of tracheobronchial stem cells could drive the development of chronic lung disease. In a recent publication in STEM CELLS Translational Medicine (SCTM), researchers showed that repeated injury to epithelial cells in the airway caused biological aging of the stem cells located there. Tracheobronchial stem cells (TSCs) are…

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Treating Intrauterine Opioid Exposure in Infants Born Preterm
 Treating Intrauterine Opioid Exposure in Infants Born Preterm 1024 575 Deborah L. Ungerleider, MD, FAAP
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We need a protocol to evaluate and treat neonatal abstinence syndrome in preterm infants. Neonatal abstinence syndrome (NAS) occurs when infants who are exposed to opioids in utero develop withdrawal signs and symptoms after birth.1 According to past studies, NAS occurs in 55% to 94% of infants exposed to opioids, with an increasing incidence between…

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A Patient-Centered Medical Home for Pediatric Inflammatory Bowel Disease
 A Patient-Centered Medical Home for Pediatric Inflammatory Bowel Disease 1024 683 Mary Bates, PhD
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The Center for Pediatric and Adolescent IBD serves as a case study for comprehensive, multidisciplinary care. In a new paper published in Inflammatory Bowel Disease, researchers from Nationwide Children’s describe the multidisciplinary care model at the Center for Pediatric and Adolescent Inflammatory Bowel Disease as an example of a pediatric specialty medical home. The researchers…

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Standardizing Training in Pediatric Cardiac Critical Care
 Standardizing Training in Pediatric Cardiac Critical Care 150 150 Mary Bates, PhD
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In a new report, a taskforce of experts presented their recommendations to standardize training in pediatric cardiac critical care. The authors hope the report is a first step toward more oversight of training and certification in the field. The field of pediatric cardiac critical care is relatively young but growing quickly. Currently, there are 120…

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Validity of the WISC-V Intelligence Testing in Children With Autism Spectrum Disorder
 Validity of the WISC-V Intelligence Testing in Children With Autism Spectrum Disorder 1024 575 Mary Bates, PhD
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Evaluating thinking and reasoning abilities for children with autism using conventional intelligence tests can be done, but with some caution. The Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) is one of the most widely used intelligence tests for children. However, little is known about the validity of this test in children with autism spectrum…

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Codetection of Respiratory Bacteria in Infants With RSV Infection Associated With Worse Outcomes
 Codetection of Respiratory Bacteria in Infants With RSV Infection Associated With Worse Outcomes 1024 575 Lauren Dembeck
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Color photo of Black father holding infant on shoulder in front of nursery background with clouds on the wall

The interaction between the bacteria and respiratory syncytial virus (RSV) appears to be synergistic. Researchers are now working to understand the underlying biological mechanisms. Researchers at Nationwide Children’s Hospital have discovered that codetection of Haemophilus influenzae and Streptococcus pneumoniae in the upper respiratory tract of previously healthy infants with RSV infection was independently associated with…

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How Patient-Derived Stem Cells are Changing the Trajectory of Congenital Heart Disease Research
 How Patient-Derived Stem Cells are Changing the Trajectory of Congenital Heart Disease Research 1024 683 Katie Brind'Amour, PhD, MS, CHES
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Small blood samples — and the patient-specific, induced pluripotent stem cells (iPSCs) they enable — turn into valuable research material for understanding congenital cardiovascular disorders, especially when united with modern genome sequencing and editing, animal models and three dimensional tissue growth technologies. Patient-derived induced pluripotent stem cells (iPSCs) have a significant downside for studying most…

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Teens on the Road: How Technology, Policy and Parents Influence Driving Safety
 Teens on the Road: How Technology, Policy and Parents Influence Driving Safety 1024 633 Abbie Roth
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Traffic crashes are a leading cause of death in the United States. For teens, the stakes are especially high.

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Microsurgery for Acute Flaccid Myelitis: Personalized Approach Leads to Remarkable Recoveries
 Microsurgery for Acute Flaccid Myelitis: Personalized Approach Leads to Remarkable Recoveries 1024 440 Lauren Dembeck
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Children from around the world are coming to Nationwide Children’s for specialized care for AFM with lower extremity involvement. Dr. Amy Moore invented many of the procedures that are leading to remarkable recoveries for these patients. After an ordinary cold, most people continue with life as usual; however, in some rare cases, a previously healthy…

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Learning in Real Time to Overcome COVID-19 and MIS-C
 Learning in Real Time to Overcome COVID-19 and MIS-C 1024 491 Natalie Wilson
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Illustrations of clock faces at different times and angles

When multisystem inflammatory syndrome in children (MIS-C) emerged in May 2020, the new condition made headlines. Although rare, MIS-C can appear in kids about a month after they’ve recovered from infections with SARS-CoV-2, the virus that causes COVID-19 — even if they haven’t felt sick at all. “We don’t know why some children develop MIS-C,”…

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Lubiprostone Found to be Safe, as Effective as Placebo in Children With Constipation
 Lubiprostone Found to be Safe, as Effective as Placebo in Children With Constipation 1024 680 Mary Bates, PhD
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Close up color photo of little boy holding hands on his belly

The medication, FDA-approved for adults, may benefit older pediatric patients with less of a behavioral component to their constipation. Pediatric functional constipation, though common, remains challenging to treat. Lubiprostone is a medication approved by the U.S. Food and Drug Administration (FDA) for the treatment of constipation and irritable bowel syndrome with constipation in adults. In…

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Distinct Transcriptional Regulatory Domain Identified in Ewing Sarcoma Fusion Protein
 Distinct Transcriptional Regulatory Domain Identified in Ewing Sarcoma Fusion Protein 1024 1024 Lauren Dembeck
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Illustration of split DNA - Single strand ribonucleic acid

A better understanding of a newly defined region in the fusion protein that causes Ewing sarcoma may lead to novel approaches for therapeutic targeting. Ewing sarcoma is an aggressive pediatric bone cancer defined by the presence of a single genetic abnormality: a chromosomal translocation. The translocation splits two genes and joins them abnormally, creating a…

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Impulsivity, Not Inattention, Predicts Externalizing Disorders
 Impulsivity, Not Inattention, Predicts Externalizing Disorders 1024 683 Natalie Wilson
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Black and white image of five smiling kids/adolescents

Without early intervention, children with hyperactive-impulsive symptoms of attention-deficit/hyperactivity disorder may be vulnerable to developing other externalizing behavior disorders. Contributing to a small but growing body of litera­ture evaluating trait impulsivity theory, a team of researchers led by Mary A. Fristad, PhD, ABPP, director of Academic Affairs and Research Development in the Division of Child…

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Risk for Serious Complications From Vaccine-Preventable Infections After Hematopoietic Cell Transplant
 Risk for Serious Complications From Vaccine-Preventable Infections After Hematopoietic Cell Transplant 1024 683 Katie Brind'Amour, PhD, MS, CHES
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Clinician-scientists reveal the burden of vaccine-preventable infections among children post-transplant, when immunity is low and risk is high. When a hematopoietic cell transplant (HCT) recipient at Nationwide Children’s Hospital was diagnosed with a vaccine-preventable infection (VPI), treating clinicians decided to evaluate the burden of VPI in HCT patients at Nationwide Children’s and elsewhere. The team…

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How Policy Can Influence Distracted Driving
 How Policy Can Influence Distracted Driving 150 150 Laura Dattner
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To combat the risks of using a cellphone while driving, states have implemented a variety of laws. As of July 2021, 21 of 50 states have implemented comprehensive hands-free cellphone laws (i.e., comprehensive handheld cellphone bans), which prohibit almost all handheld cellphone use including texting, calling and using apps. In addition, three states and the…

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Kidney and Urinary Tract Protein Demonstrates Antimicrobial Activity
 Kidney and Urinary Tract Protein Demonstrates Antimicrobial Activity 1024 683 Lauren Dembeck
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A little-studied peptide found in the urinary tract is a potential biomarker for urinary tract infection risk and could be developed into a novel therapeutic against drug-resistant bacteria.  Researchers at Nationwide Children’s Hospital have demonstrated that an understudied protein expressed in the human kidney and bladder kills the bacteria that cause urinary tract infections (UTI).…

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Is Tracheostomy Tube Placement in Preemies Safe?
 Is Tracheostomy Tube Placement in Preemies Safe? 1024 575 Mary Bates, PhD
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Nurse caring for infant in Neonatal Intensive Care Unit (NICU)

The procedure is safe and effective in newborns requiring high ventilator pressures. Tracheostomy tube placement is a common procedure performed for premature infants requiring respiratory assistance. Compared to an endotracheal tube, placement of a tracheostomy tube is thought to allow the infant more opportunities for comfort, interaction, nutrition and growth. However, rates of tracheostomy tube…

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Long-Term Follow-up Study Shows Cannabidiol is Safe and Effective for Lennox-Gastaut Syndrome
 Long-Term Follow-up Study Shows Cannabidiol is Safe and Effective for Lennox-Gastaut Syndrome 695 473 Mary Bates, PhD
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Black and white environmental portrait of Dr. Anup Patel standing in the hospital in his lab coat with his hand in his pocket

Long-term treatment with highly purified plant-based CBD shows continued seizure reduction and no new side effects. The results of a long-term open-label extension trial of plant-derived, highly purified cannabidiol (CBD) show the treatment is effective and safe long term for patients with Lennox-Gastaut syndrome. The treatment had a similar safety profile as in the original…

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A Novel FUS/ETV4 Fusion and Other Rare FET/ETS Fusion Proteins Are Bona Fide Ewing Sarcoma
 A Novel FUS/ETV4 Fusion and Other Rare FET/ETS Fusion Proteins Are Bona Fide Ewing Sarcoma 1024 575 Jessica Nye, PhD
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Researchers conduct a molecular comparison between the established EWS/FLI translocation with rarer FET/ETS fusions, broadly supporting the inclusion of FET/ETS chimeric proteins in Ewing Sarcoma diagnosis and treatment.   Ewing sarcoma, most commonly caused by a chromosomal translocation of the amino-terminal domain of EWSR1 with the transcription factor FLI (EWS/FLI), presents as an aggressive pediatric…

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Identifying Early Urinary Biomarkers of Kidney Damage in Type 1 Diabetes
 Identifying Early Urinary Biomarkers of Kidney Damage in Type 1 Diabetes 1024 575 Mary Bates, PhD
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Markers of renal tubule injury and oxidative stress indicate diabetic nephropathy before microalbuminuria. Diabetic nephropathy is a common complication of type 1 diabetes. Traditionally, measurement of microalbuminuria is used to screen children with diabetes for diabetic nephropathy. However, evidence suggests that damage to the kidneys may begin soon after diagnosis, much earlier than microalbuminuria can…

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Probiotic Treatment Promotes Neurodevelopment After Experimental Necrotizing Enterocolitis
 Probiotic Treatment Promotes Neurodevelopment After Experimental Necrotizing Enterocolitis 777 640 Lauren Dembeck
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B&W image of Dr. Gail Besner posing for a photo in her lab coat

A single dose of a novel live probiotic therapeutic protects the rodent brain during early development and the effects persist into adolescence. Researchers at Nationwide Children’s Hospital have demonstrated that when given in a biofilm state, probiotic Lactobacillus reuteri prevents the detrimental neurodevelopmental sequalae of experimental necrotizing enterocolitis (NEC).1 The findings, published in Brain, Behavior…

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Specialized Treatment for Severe Bronchopulmonary Dysplasia Increases Survival
 Specialized Treatment for Severe Bronchopulmonary Dysplasia Increases Survival 150 150 Mary Bates, PhD
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Most infants with established BPD referred to Nationwide’s BPD Center improve and survive, despite the severity of their illness. In a new study, researchers analyzed the in-hospital outcomes for preterm infants with established bronchopulmonary dysplasia (BPD) referred to Nationwide Children’s Comprehensive Center for BPD. They found that despite the high degree of illness severity in…

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Postoperative Care for Benign Ovarian Neoplasms
 Postoperative Care for Benign Ovarian Neoplasms 150 150 Mary Bates, PhD
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Study suggests no benefit from routine imaging and supports symptomatic imaging for postoperative management of benign ovarian neoplasms. In a new study, researchers at Nationwide Children’s Hospital led a collaboration with eight children’s hospitals examining the follow-up care and subsequent outcomes of pediatric patients after surgery for a benign ovarian neoplasm to develop evidence-based recommendations…

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Administering NK Cells Post-Transplant Improves Relapse in Myeloid Malignancies
 Administering NK Cells Post-Transplant Improves Relapse in Myeloid Malignancies 150 150 Mary Bates, PhD
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A Phase I/II clinical trial demonstrates donor-derived NK cells are safe and effective at decreasing relapse and increasing survival in adults with myeloid malignancies. Haploidentical stem-cell transplantation can be a successful treatment for patients with myeloid malignancies, but relapse remains a common risk. Natural killer (NK) cells are a subset of lymphocytes capable of recognizing…

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Stent Implantations Are an Option for Small Patients
 Stent Implantations Are an Option for Small Patients 1024 670 Mary Bates, PhD
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Stents that can be dilated to adult size are safe and effective for the treatment of coarctation of the aorta in young patients. Studies have demonstrated stent implantation is more effective and safer than balloon angioplasty for the treatment of coarctation of the aorta in adult and older pediatric patients. In smaller patients, however, particularly…

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Two Novel Biomarkers for Urinary Tract Infection Diagnosis Identified
 Two Novel Biomarkers for Urinary Tract Infection Diagnosis Identified 1024 575 Mary Bates, PhD
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Study identifies two proteins that are elevated in children with UTIs compared to those with culture-negative pyuria. In a new study from Nationwide Children’s, researchers identified two novel biomarkers that could be developed to further fine tune the diagnosis of urinary tract infection (UTI) in children. UTIs are common, but challenging to diagnose, in children.…

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Unraveling the Role of Pioneer Factors in Childhood Cancer
 Unraveling the Role of Pioneer Factors in Childhood Cancer 150 150 Abbie Roth
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New study implicates PAX3-FOXO1, an oncogenic driver of rhabdomyosarcoma, as a chimeric pioneer factor. Transcription factors are proteins that read the DNA sequences and can direct the transcription of that sequence into mRNA for translation into a protein. Transcription can become deregulated in cancer. Normally, transcription factors read sequences of DNA that are described as…

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Cylinder Mitral Valve Construct is a Safe and Durable Alternative for Young Patients
 Cylinder Mitral Valve Construct is a Safe and Durable Alternative for Young Patients 1024 752 Mary Bates, PhD
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Top-down view of heart valves

The new technique could potentially reduce the number of lifetime surgeries required by these patients, researchers say. In a new study, researchers from Nationwide Children’s Hospital report that pediatric patients who underwent cylinder mitral valve construct, a new technique for replacing the mitral valve, had improved left ventricular function over time. In addition, there were…

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Robotic Surgery Is Here to Stay
 Robotic Surgery Is Here to Stay 150 150 Abbie Roth
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A recent publication describes increases in the use of robotic surgery in children’s hospitals across the country.  The use of robotic surgery in tertiary children’s hospitals has been on the rise since the 2000s. In a recent publication, researchers from Nationwide Children’s Hospital sought to characterize the use and growth in acceptance of robotic surgeries…

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Molecular Toolset: Real-Time Screening for SARS-CoV-2 Variants
 Molecular Toolset: Real-Time Screening for SARS-CoV-2 Variants 1024 683 Lauren Dembeck
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Tracking variants of concern or interest is critical to inform public health decisions and to ensure patients receive the best available COVID-19 treatments for SARS-CoV-2 infections. Researchers at Nationwide Children’s Hospital and The Ohio State University (OSU) have developed a set of mutation-specific tests with same-day reporting that can detect five variants of concern (VOCs)…

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EVALI and MIS-C: Overlapping Diagnoses
 EVALI and MIS-C: Overlapping Diagnoses 1024 575 Mary Bates, PhD
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Taking a thorough history can differentiate these similarly presenting conditions. In a new report, researchers from Nationwide Children’s Hospital describe six patients admitted to the hospital with concerns for MIS-C who were later diagnosed with EVALI. The researchers discuss the factors that led them to differentiate between the two disorders and arrive at the correct…

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The 5,000 Babies Project: Screening Newborns for Early Diagnosis of Developmental Delay
 The 5,000 Babies Project: Screening Newborns for Early Diagnosis of Developmental Delay 150 150 Lauren Dembeck
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Democratizing health care requires equal access to early identification of infant neurodevelopmental disorders.  Delayed diagnosis of disorders that involve developmental delays, such as cerebral palsy, can lead to lifelong disability. Yet not all families have access to health care facilities employing highly trained specialists who can test for these developmental delays. Infants from minority families…

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Improving Suicide Prevention by Examining Characteristics and Precipitating Circumstances Among Children Aged 5 to 11 Years
 Improving Suicide Prevention by Examining Characteristics and Precipitating Circumstances Among Children Aged 5 to 11 Years 150 150 Natalie Wilson
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Suicides among children aged 11 or younger are on the rise. In response to this trend and a call to action by the National Institute of Mental Health task force on child suicide research, researchers have identified common themes across coroner’s and police reports on suicide deaths in this age group to identify warning signs…

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Diabetes Distress Associated With Poor Glycemic Control in Adolescents With Diabetes
 Diabetes Distress Associated With Poor Glycemic Control in Adolescents With Diabetes 1024 683 Mary Bates, PhD
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Screening for diabetes distress, in addition to depression, could benefit adolescents with diabetes. Adolescents with type 1 diabetes are more likely to experience depression than their peers without diabetes, and symptoms of depression can directly impact adherence to diabetes care. Thus, both the American Diabetes Association and International Society for Pediatric and Adolescent Diabetes recommend…

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Lawnmower Injuries in Children Are Limb-Threatening and Avoidable
 Lawnmower Injuries in Children Are Limb-Threatening and Avoidable 1024 683 Ibrahim Khansa, MD
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During the warm months, lawnmowers are a ubiquitous sight in American lawns. Unfortunately, more than 9,000 children are injured by lawnmowers every year in the United States. In a recent study, published in the Journal of Plastic, Reconstructive and Aesthetic Surgery – Open, our group from the Department of Plastic and Reconstructive Surgery at Nationwide…

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Occurrence and Cost of Infections After Heart Transplant
 Occurrence and Cost of Infections After Heart Transplant 150 150 Mary Bates, PhD
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Researchers identify risk factors, long-term outcomes of vaccine-preventable infections following heart transplantation in pediatric patients. Recently, a study using the Pediatric Health Information System (PHIS) database found that one in six pediatric solid organ transplant recipients were hospitalized with respiratory syncytial virus (RSV) or vaccine-preventable illness in the first five years after transplant. Within this…

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Does Twinkle Artifact on Ultrasound Indicate a Kidney Stone in Pediatric Patients?
 Does Twinkle Artifact on Ultrasound Indicate a Kidney Stone in Pediatric Patients? 150 150 Abbie Roth
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The presence of twinkle artifact on ultrasound is a useful, though imperfect, tool for diagnosing kidney stones in children. For pediatric patients, ultrasound is the recommended imaging modality used to diagnose kidney stones. Several findings are used to identify stones on ultrasound, including the presence of an echogenic focus, posterior acoustic shadowing, or twinkle artifact.…

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Understanding the Social Neural Network
 Understanding the Social Neural Network 1024 575 Natalie Wilson
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Young boy turned sideways in front of a dark background, with dark lighting and an illustration of his brain appearing in front of the side of his head

Neuroimaging of participants with and without epilepsy allows researchers to explore the neural networks associated with social skills. Epilepsy is a chronic brain disease characterized by neural network dysfunction and seizures. According to the Centers for Disease Control (CDC), 470,000 children in the United States had active epilepsy in 2015. These children are more likely…

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Bronchopulmonary Dysplasia: Characterizing the Chronic Lung Disease of Prematurity
 Bronchopulmonary Dysplasia: Characterizing the Chronic Lung Disease of Prematurity 1024 575 Lauren Dembeck
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Nurse caring for infant in Neonatal Intensive Care Unit (NICU)

  Precise definitions and/or classifications of bronchopulmonary dysplasia are needed to optimize outcomes. Researchers at Nationwide Children’s Hospital are continuing to help refine the definition of severe bronchopulmonary dysplasia (sBPD). In a recent study, members of the BPD Collaborative demonstrated that preterm infants with sBPD who were on invasive mechanical ventilation at 36 weeks, a…

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Diuretic Therapy in Preterm Infants With Acute Kidney Injury Is Common: Should It Be?
 Diuretic Therapy in Preterm Infants With Acute Kidney Injury Is Common: Should It Be? 1024 575 Lauren Dembeck
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small baby with nose canula

Lack of efficacy and safety data on diuretic therapy in the critically ill infants is cause for concern. Researchers at Nationwide Children’s Hospital are beginning to fill in the gaps. Despite the common use of diuretic therapy in preterm infants with acute kidney injury, to date, no large studies have evaluated the patterns of prescription…

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Chronic Pediatric Pancreatitis and (No) Persistent Belly Pain
 Chronic Pediatric Pancreatitis and (No) Persistent Belly Pain 1024 575 Katie Brind'Amour, PhD, MS, CHES
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Underlying — even silent — chronic pancreatitis can be at play in children without previous acute pancreatitis episodes, including among children with little or no reported pain. Pancreatitis in children is rare (about 3-13 per 100,000 people per year), but diagnosis is increasing, at least in part due to increased awareness among physicians. The path…

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Understanding Black Youth Suicide: Steps Toward Prevention
 Understanding Black Youth Suicide: Steps Toward Prevention 1024 596 Abbie Roth
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To understand the how and why of rising rates of Black youth suicide, researchers call for a ‘Ground Zero’ approach. In December 2019, the Congressional Black Caucus released the statement “Ring the Alarm: The Crisis of Black Youth Suicide in America.” The statement focused on rising suicide rates among Black youth in the United States.…

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“Left Behind Again:” Rural Children Enrolled in Medicaid Use Home Health Services Far Less Than Urban Children
 “Left Behind Again:” Rural Children Enrolled in Medicaid Use Home Health Services Far Less Than Urban Children 1024 575 Jeb Phillips
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Data from a pediatric accountable care organization is allowing physicians and researchers to explore the rural-urban health care gap. While it’s long been clear that children in rural areas have reduced access to some kinds of health care services compared to their urban peers, understanding the specifics of the care gap has proven difficult, because…

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Study Highlights Relationship Between SI Gene and Chronic, Idiopathic Loose Stool in Some Children
 Study Highlights Relationship Between SI Gene and Chronic, Idiopathic Loose Stool in Some Children 1024 680 Abbie Roth
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Close up color photo of little boy holding hands on his belly

Researchers conducted one of the largest prospective, multicenter trials in pediatrics looking at the connection between SI variants and common gastrointestinal symptoms. The SI gene codes for the sucrase-isomaltase enzyme, which is expressed in the intestinal brush border. This enzyme is important to the digestion of sucrose, and without it, maldigestion and poor absorption of…

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Leaving No Stone Unturned: Nationwide Children’s Joins Children’s Oncology Group Pediatric Early Phase Clinical Trial Network
 Leaving No Stone Unturned: Nationwide Children’s Joins Children’s Oncology Group Pediatric Early Phase Clinical Trial Network 1024 683 Natalie Wilson
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The Children’s Oncology Group (COG), the world’s largest and most recognized pediatric cancer research collaborative, recently selected Nationwide Children’s Hospital to join its Pediatric Early Phase Clinical Trial Network (PEP-CTN) through a peer review process. The COG PEP-CTN is a re-iteration of the COG Phase 1/Pilot Consortium and is comprised of 21 core member sites…

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Surgical Management of Recurrent Patellar Instability in the Pediatric Population
 Surgical Management of Recurrent Patellar Instability in the Pediatric Population 1024 575 Mary Bates, PhD
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Medial patellofemoral ligament reconstruction via the quadriceps turndown technique is safe and effective in young patients. In a new study, researchers from Nationwide Children’s Hospital demonstrate the safety and efficacy of the quadriceps turndown technique for medial patellofemoral ligament reconstruction in pediatric and adolescent patients. Patellar instability is a common knee condition in children and…

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Antibodies to Spinal Muscular Atrophy Gene Therapy Vector May Dissipate, Allowing More Children to Receive Treatment
 Antibodies to Spinal Muscular Atrophy Gene Therapy Vector May Dissipate, Allowing More Children to Receive Treatment 1024 681 Katie Brind'Amour, PhD, MS, CHES
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sleeping infant

A review of antibody titers for SMA patients revealed that levels may decrease with time, potentially enabling delayed dosing for children who otherwise might have been excluded from life-saving gene therapy. Adeno-associated viral (AAV) vectors are used to deliver gene therapies such as onasemnogene abeparvovec-xioi (ZOLGENSMA®), a recently approved gene therapy for spinal muscular atrophy…

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Keeping in Touch: Renin-Producing Cells Require Cell Adhesion Molecule to Survive
 Keeping in Touch: Renin-Producing Cells Require Cell Adhesion Molecule to Survive 1024 575 Lauren Dembeck
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Uncovering the molecular mechanisms underlying the renin-angiotensin-aldosterone system may provide insights into the long-term effects of hypertension and antihypertensive medications.  As the main source of renin in the human body, juxtaglomerular (JG) cells are crucial for blood pressure and fluid-electrolyte homeostasis. However, these cells cannot be cultured in vitro, making it challenging to study the…

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The Sound of Silence: The Impact of “Silent” Genetic Variation in Health and Disease
 The Sound of Silence: The Impact of “Silent” Genetic Variation in Health and Disease 1024 575 Lauren Dembeck
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Despite widespread scientific interest in messenger RNA (mRNA) structure, its role in human health and disease remains poorly understood. Researchers at Nationwide Children’s Hospital have developed novel software and metrics to identify “silent” genetic variants that affect mRNA folding and may underlie rare genetic disorders. Historically, synonymous or “silent” genetic variants — those in protein-coding…

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