Optimizing Prenatal and Neonatal Care for Infants With Treatable Rare Diseases

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Unraveling the Genetic Mystery Behind a Rare Pediatric Disease

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New National “Vital Signs” to Measure Population-wide Pediatric Health

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How Does Thickened Formula Affect Reflux in Infants?
 How Does Thickened Formula Affect Reflux in Infants? 1024 683 Mary Bates, PhD
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Infants in the neonatal intensive care unit with suspected gastroesophageal reflux disease (GERD) are often fed formula with added rice cereal or other thickeners, despite limited evidence of its efficacy or long-term consequences. Additionally, the consequences of thickened feeds are not well-studied, says Sudarshan…

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Donor-Derived NK Cells Safe and Efficacious in Relapsed and Refractory AML
 Donor-Derived NK Cells Safe and Efficacious in Relapsed and Refractory AML 1024 649 Lauren Dembeck
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Researchers have used their approach for growing large numbers of NK cells to allow repeated dosing for a more effective and less toxic treatment.   Outcomes for patients with relapsed and refractory acute myeloid leukemia (R/R AML) have not significantly improved over the last…

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Telemedicine’s Past, Present and Future Role in Pediatric Inflammatory Bowel Disease Care
 Telemedicine’s Past, Present and Future Role in Pediatric Inflammatory Bowel Disease Care 1024 683 Erin Gregory
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A recent study sheds light on how telemedicine’s use during the pandemic has reshaped care delivery for children with IBD.   A recent study published in Journal of Pediatric Gastroenterology and Nutrition utilized electronic medical record data to compare the volume, duration and nature…

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Teen girl with backpack
Trauma and Resilience in Youth With Opioid Use and Eating Disorders
 Trauma and Resilience in Youth With Opioid Use and Eating Disorders 1024 575 Abbie Miller and Pam Georgiana
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Health care providers should expect patients to have hidden traumas and reduced resilience.   When treating adolescents and young adults in the Substance Use Treatment and Recovery program at Nationwide Children’s, medical director Erin R. McKnight, MD, MPH, noticed that while most of her…

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Nationwide Children’s Research Benefits Kids Everywhere
 Nationwide Children’s Research Benefits Kids Everywhere 1024 619 Nationwide Children's
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Pediatric research benefits children everywhere. Through the stories below, discover more about the breadth and depth of research at Nationwide Children’s that is improving child health. Optimizing Prenatal and Neonatal Care for Infants With Treatable Rare Diseases Not long ago, a diagnosis of molybdenum…

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Improving Medication Adherence and Clinical Outcomes After Hematopoietic Stem Cell Transplant
 Improving Medication Adherence and Clinical Outcomes After Hematopoietic Stem Cell Transplant 1024 683 JoAnna Pendergrass, DVM
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A clinical trial is evaluating the acceptability and efficacy of a mobile app in improving medication adherence, symptoms and clinical outcomes in pediatric hematopoietic stem cell patients. A research team led by Micah Skeens, PhD, APRN, FAAN, principal investigator in the Center for Biobehavioral…

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Optimizing Prenatal and Neonatal Care for Infants With Treatable Rare Diseases
 Optimizing Prenatal and Neonatal Care for Infants With Treatable Rare Diseases 1024 619 Katie Brind'Amour, PhD, MS, CHES
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As new treatments emerge and diagnostics improve, earlier interventions offer infants with rare metabolic and neurodegenerative conditions a future wildly different than ever before. Not long ago, a diagnosis of molybdenum cofactor deficiency (MoCD) type A meant death before kindergarten. Since the FDA approval…

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Turning Complications into Fuel for a New Approach to Better Outcomes
 Turning Complications into Fuel for a New Approach to Better Outcomes 1024 418 Alaina Doklovic
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A new web application enhances Morbidity and Mortality data storage and actively improves patient care. Morbidity and Mortality (M&M) Conferences are mandated meetings that occur regularly at all academic medical centers and are powerful opportunities for learning and reflection. These conferences have been around…

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Accelerating Advances in Epilepsy Surgery
 Accelerating Advances in Epilepsy Surgery 1024 683 Abbie Miller
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One program is bringing together clinical care, surgery and research to create a brighter future for children with intractable epilepsy. Childhood-onset epilepsy affects 1% of children worldwide. Up to a third of patients with epilepsy will have medically refractory epilepsy, continuing to have seizures…

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New Frontiers for Data Science in Pediatric Research
 New Frontiers for Data Science in Pediatric Research 1024 577 Peter White, PhD
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From discovery science to population health, researchers generate masses of data that hold immense potential to transform pediatric research, diagnostics, treatments and even guide disease prevention strategies. To make sense of this data deluge, we’ve been harnessing the power of cloud computing and cutting-edge…

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Unraveling the Genetic Mystery Behind a Rare Pediatric Disease
 Unraveling the Genetic Mystery Behind a Rare Pediatric Disease 980 429 Wendy Margolin
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Prune belly syndrome (PBS) is a rare, congenital urologic disease, affecting an estimated 1 out of 50,000 births. While most urologists will see fewer than three cases of PBS in their careers, Nationwide Children’s Hospital’s Linda Baker, MD, has treated more than 50 patients…

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The First 3 Years With PBS: Bryson’s Story
 The First 3 Years With PBS: Bryson’s Story 953 269 Wendy Margolin
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The first time Andrea Williams met her new baby was four days after his birth. Baby Bryson was born with the rare congenital disorder, prune belly syndrome (PBS), and was rushed to the Nationwide Children’s Hospital NICU immediately after birth. His enlarged belly meant…

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Connecting Well-being to Compassionate Care in Pediatric Residents
 Connecting Well-being to Compassionate Care in Pediatric Residents 1024 683 Madison Storm
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A multicenter study identified several well-being factors associated with pediatric residents’ confidence in providing calm, compassionate care to patients. Suzanne Reed, MD, has been interested in medical education since her training as a medical student. Captivated by the academic side of education, Dr. Reed…

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Proton Therapy – A New Era of Cancer Therapy for Children
 Proton Therapy – A New Era of Cancer Therapy for Children 1024 683 Abbie Miller
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Proton therapy offers benefits over traditional photon radiation therapy. Using proton therapy, doctors can give more specific, concentrated doses of radiation with less risk and damage to the surrounding tissue. Proton therapy is an advanced type of radiation treatment that uses protons instead of…

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Practical Advice for Investigator-Initiated Trials
 Practical Advice for Investigator-Initiated Trials 1024 363 Lauren Dembeck
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Investigator-initiated trials play a crucial role in advancing medical research and contributing to evidence-based medicine. These trials are initiated, designed and conducted by researchers, often physicians or scientists, rather than by pharmaceutical or biotechnology companies. They allow researchers to explore specific hypotheses, novel interventions…

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New National “Vital Signs” to Measure Population-wide Pediatric Health
 New National “Vital Signs” to Measure Population-wide Pediatric Health 1024 683 Jeb Phillips
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In 2017, an opinion piece in JAMA Pediatrics started this way: “We call for a national effort to develop a parsimonious set of quality and outcome measures for (all) children.” And now, for perhaps the first time in American pediatrics, that “parsimonious set: has been…

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Featured Researcher – Rajan Thakkar, MD
 Featured Researcher – Rajan Thakkar, MD 150 150 Alaina Doklovic
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Rajan K. Thakkar, MD, FACS, is a pediatric surgeon, the Trauma Medical Director, and the co-Director of the Burn Center at Nationwide Children’s Hospital. He is an associate professor of Surgery and Pediatrics at The Ohio State University College of Medicine and is a…

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Toddler playing with toys
Supporting Optimal Developmental Outcomes After Neonatal Opioid Withdrawal Syndrome
 Supporting Optimal Developmental Outcomes After Neonatal Opioid Withdrawal Syndrome 1024 575 Abbie Miller
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Children with prenatal opioid exposure have a higher risk for developmental and behavioral concerns. Prenatal opioid exposure is still far too common. Experts estimate that one infant who has been exposed to opioids is born every 15 minutes in the United States.  While standard…

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Scalable Delivery of Universal Suicide Prevention in Schools
 Scalable Delivery of Universal Suicide Prevention in Schools 1024 657 Pam Georgiana
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Implementing the Signs of Suicide (SOS) Prevention and Screening Program   Suicide is the second-leading cause of death among youth ages 10 to 19 years, and data indicate no sign of it decreasing as a major public health issue. While acute care and crisis…

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Infliximab Biosimilars Improve Growth Outcomes in Children With Inflammatory Bowel Disease
 Infliximab Biosimilars Improve Growth Outcomes in Children With Inflammatory Bowel Disease 1024 683 Mary Bates, PhD
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Treatment with either the infliximab originator or a biosimilar improves clinical and growth outcomes. In a new study, researchers at Nationwide Children’s Hospital report that children with inflammatory bowel disease showed similar growth and clinical outcomes whether they were treated with the infliximab originator…

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