Features

Gene Therapy for the Masses?
Gene Therapy for the Masses? 1024 683 Katie Brind'Amour, PhD, MS, CHES

Long-lived financial and logistical hurdles make bringing new gene therapy products to market a major challenge. To help bring more of these medical miracles to fruition, experts across industry, regulatory review, science and medicine have begun to problem solve together.  With the approval of the gene therapy Kymriah® (tisagenlecleucel) in 2017, the Food and Drug…

Innovations in Pediatric Cancer and Transplants: Q&A With Sara K. Rasmussen, MD, PhD
Innovations in Pediatric Cancer and Transplants: Q&A With Sara K. Rasmussen, MD, PhD 150 150 Pam Georgiana

Sara K. Rasmussen, MD, PhD, is a transplant and pediatric surgeon in the Department of Abdominal Transplant and Hepatopancreatobiliary Surgery and an associate principal investigator in the Center for Clinical and Translational Research in the Abigail Wexner Research Institute at Nationwide Children’s Hospital. She earned both her medical and doctoral degrees from West Virginia University…

The Endocrinologists’ Role in Caring for TPIAT Patients
The Endocrinologists’ Role in Caring for TPIAT Patients 1024 500 Lauren Dembeck
illustrated cross section of islets from pancreas

Children with recurrent acute or chronic pancreatitis often suffer debilitating pain and may endure an impaired quality of life. They may be frequently admitted to the hospital, disrupting their daily routines and activities. Patients may undergo medical and endoscopic interventions for both diagnostic and therapeutic purposes and may be offered surgical interventions. Total pancreatectomy with…

Changing the Landscape of Blood Disorder Treatments: A Long Time Coming
Changing the Landscape of Blood Disorder Treatments: A Long Time Coming 1024 574 Alaina Doklovic

New therapies, from virus-mediated gene therapy to CRISPR-based treatments, are poised to change the clinical landscape of blood disorder treatments and outcomes.   For more than two decades experts have been searching for curative therapies for blood disorders. They have invested time, money and expertise to bring these therapies to fruition, something that the Division…

Meet Toni Pearson, MD: Pediatric Movement Disorders Expert
Meet Toni Pearson, MD: Pediatric Movement Disorders Expert 150 150 Pam Georgiana

Toni S. Pearson, MD, joined Nationwide Children’s in 2022 as an attending pediatric neurologist and professor of pediatrics at The Ohio State University College of Medicine. She specializes in childhood movement disorders, such as dystonia, chorea, parkinsonism, ataxia, myoclonus, tremors, tics, and spasticity. Dr. Pearson is also developing a fellowship training program for a new…

Innovative Gene Therapy Approach for Treating Patients with Tuberous Sclerosis Type 2
Innovative Gene Therapy Approach for Treating Patients with Tuberous Sclerosis Type 2 150 150 Lauren Dembeck

Tuberous sclerosis complex (TSC) is a devastating genetic disease that affects nearly 1 in every 5,500 newborns and approximately 2 million people worldwide. The disease is characterized by the formation of non-malignant tumors throughout multiple organs, including the kidney, lungs, eyes, and heart, but predominantly the brain. It is typically diagnosed in infants and young…

Accelerating Pediatric Medical Device Innovation 
Accelerating Pediatric Medical Device Innovation  150 150 Erin Gregory

The Midwest Pediatric Device Consortium (MPDC) is a unique collective led by Nationwide Children’s Hospital and The Ohio State University, designed to facilitate the development, production, and distribution of pediatric medical devices. Comprising a diverse group of leaders in universities, healthcare systems, community organizations, and industry partners across Ohio, the MPDC is committed to transforming…

Probiotics Delivered in Biofilm State Protect the Intestines and Brain in NEC Model
Probiotics Delivered in Biofilm State Protect the Intestines and Brain in NEC Model 898 504 Abbie Miller

Biofilm formulation of Limosilactobacillus reuteri protects against necrotizing enterocolitis in piglet model.  Necrotizing enterocolitis (NEC) is a debilitating and deadly condition affecting infants born preterm. In NEC cases, intestinal tissues become inflamed, and in severe cases, there is ischemia and death of the involved intestines. Treatment for NEC often involves surgery to remove the dying…

Building Hope, Recovery, and Life Beyond Substance Use Disorder
Building Hope, Recovery, and Life Beyond Substance Use Disorder 1024 702 Abbie Miller

Five years ago, Pediatrics Nationwide dedicated its fall issue to a comprehensive look at the effects of the opioid crisis on children and families. At that time, Sarah Parker was working hard to stay sober, recovering from the very crisis holding the nation’s attention. Parker grew up in Chillicothe, Ohio, halfway between Portsmouth, Ohio, the…

Whole Child, Whole Family Care
Whole Child, Whole Family Care 150 150 Abbie Miller and Jeb Phillips

Meeting the needs of children and caregivers with evidence-based programming and intentional support for access. Parenting a young child can be tough in the best circumstances. But when you overlay poverty, inequity, and concerns about employment and education on top of it, parenting a young child can feel overwhelming. Whitney Raglin Bignall, PhD, has dedicated…

Tissue Engineered Trachea: State of the Research
Tissue Engineered Trachea: State of the Research 150 150 Lauren Dembeck

The promise of tissue-engineered trachea grafts is moving closer to the clinic, as recent preclinical studies have shown successful implantation and neovascularization. Breathing is an essential biological function that provides our bodies with the oxygen necessary for survival. However, most of us rarely think about the biological structures that make it possible. The trachea, commonly…

Caring for Incarcerated Children
Caring for Incarcerated Children 1024 537 Jeb Phillips

Young people in juvenile detention centers need health care. In fact, decades of studies show they most often need it more urgently than their peers who are not involved in the justice system – nearly 70% of “confined youth” have an unmet health care need (2010 study). So it makes some sense that “urgent care”…

Preventing Accidental Ingestion of Marijuana
Preventing Accidental Ingestion of Marijuana 1024 683 Abbie Miller

If you are a child, and you find a bag of fruit snacks — you don’t eat just one piece. The THC/marijuana products on the market today have an average of 10-20 mg in one serving. That’s one square of a chocolate bar, one gummy (which looks like a fruit snack) or one chip. “THC…

Growing Clinical Research at Nationwide Children’s With Cynthia Gerhardt, PhD
Growing Clinical Research at Nationwide Children’s With Cynthia Gerhardt, PhD 1024 683 Cynthia Gerhardt, PhD and Natalie Wilson

Dr. Gerhardt was appointed Chief Clinical Research Officer at Nationwide Children’s Hospital in April 2023. With integrating clinical care and research at the heart of the hospital’s strategic plan, she has big plans for ensuring our clinical research infrastructure continues to grow and evolve to support the best bench-to-bedside-and-back science for best outcomes. Learn more…

Implementation Science to Expand Contraception Access for Adolescents Hospitalized with Mental Health Issues
Implementation Science to Expand Contraception Access for Adolescents Hospitalized with Mental Health Issues 1024 575 Pam Georgiana
Black and white image of teen girl posing outside

Research has documented that adolescents with mental health concerns have a higher need for contraceptive counseling and care because they are at a higher risk for unintended pregnancy.[i] [ii] Unintended pregnancies can make mental health symptoms worse, especially depressive symptoms. [iii] Counseling and use of contraceptives are simple evidence-based strategies to reduce unplanned pregnancies. However,…

Ask A Specialist: What Running Distances Are Safe for Children?
Ask A Specialist: What Running Distances Are Safe for Children? 1024 895 Gabriella Gonzales, MD and James MacDonald, MD, MPH

Sports Medicine experts offer advice for pediatricians about what age it is appropriate for children to run and train for races such as the 5K, 10K, half marathon and marathon. Running is a great activity! It requires very little equipment and can be done individually or with a team. Running can be beneficial for children…

Kidney Support for Babies: Building a Comprehensive and Integrated Neonatal Kidney Support Therapy Program
Kidney Support for Babies: Building a Comprehensive and Integrated Neonatal Kidney Support Therapy Program 1024 575 Lauren Dembeck
Nurse caring for infant in Neonatal Intensive Care Unit (NICU)

Kidney support therapy (KST), commonly referred to as dialysis, is a life-saving procedure used to manage complications associated with acute kidney injury and kidney failure, such as fluid overload and electrolyte imbalances, or to remove toxins, such as those in patients with inborn errors of metabolism. Most KST or dialysis machines are designed for adults…

1 Year In, the Molecular Characterization Initiative Has Analyzed Samples From Over 1,000 Patients
1 Year In, the Molecular Characterization Initiative Has Analyzed Samples From Over 1,000 Patients 1024 614 Abbie Miller
abstract art of magnifying glass over DNA strand

Data from the analysis helps clinicians confirm diagnoses and identify targeted treatments. The data also support new pediatric cancer research through the National Cancer Institute’s Childhood Cancer Data Initiative.   The CCDI Molecular Characterization Initiative (MCI) is a project that aims to collect, analyze and report clinical and molecular data and is currently supporting Children’s…

Tissue Engineering and Fetal Medicine: A New Frontier for Congenital Heart Disease
Tissue Engineering and Fetal Medicine: A New Frontier for Congenital Heart Disease 1024 575 Abbie Miller
Fetus in utero receiving valvuloplasty

Procedures that utilize cardiac catheterization to improve fetal heart development are often successful, but they are not without risk. And even if they can successfully prevent the development of single ventricle disease, there is always ongoing heart valve disease (HVD). HVD may not be as life-threatening as single ventricle disease, but it requires lifelong care.…

Investigating Caregiver Disclosure of Pediatric Urologic Surgery
Investigating Caregiver Disclosure of Pediatric Urologic Surgery 1024 575 Mary Bates, PhD
Toddler playing with toys

A survey reveals most caregivers plan to disclose urologic surgery to their child but would like more guidance from providers. Some pediatric urologic surgeries are performed early in childhood and with short-term follow-up. When children have surgery before the age of memory formation, it is up to their caregiver to disclose this surgery to them.…

More Than a Building: Why Our Expanded Research Facilities Matter for Kids Everywhere
More Than a Building: Why Our Expanded Research Facilities Matter for Kids Everywhere 1024 624 Dennis Durbin, MD, MSCE

At Nationwide Children’s Hospital, we proudly acknowledge the significant role research has in improving patient care and overall child heath. The integration of research and clinical care is at the heart of the hospital’s strategic plan — a $3.3 billion investment over the next several years in our commitment to transform health outcomes for all…

How Language-Processing Technology Could Transform Medical Practice, Research and Patient Participation
How Language-Processing Technology Could Transform Medical Practice, Research and Patient Participation 150 150 Katie Brind'Amour, PhD, MS, CHES

Recent revolutions in researchers’ ability to process natural language sources, such as clinic visit notes, transcripts or medical diaries, could dramatically expand opportunities to improve health care and prevention health outreach.   Natural Language Processing (NLP) is a subfield of computer science and artificial intelligence that deals with the interaction between computers and human language.…

Advancing Genomics-Driven Precision Medicine in the NICU
Advancing Genomics-Driven Precision Medicine in the NICU 1024 683 Natalie Wilson

According to the Children’s Hospitals Neonatal Consortium, as many as half of newborns hospitalized in level IV neonatal intensive care units (NICUs) due to critical illness have an underlying genetic condition. Most don’t get their diagnosis for months or even years. However, clinical assays, new testing modalities and clinical trials are improving their care. Advanced…

Beyond the Wow Factor: Artificial Intelligence in Pediatrics
Beyond the Wow Factor: Artificial Intelligence in Pediatrics 1024 576 Katie Brind'Amour, PhD, MS, CHES

What promise do AI and machine learning hold for pediatrics, and how can their potential flourish while still safeguarding children’s health and privacy? Machine learning (ML) and artificial intelligence (AI) have exploded across the worlds of marketing and commerce in recent years. Streaming services track what you watch and suggest other content you may enjoy.…

Going Viral: The AAV Approach to Curing Cancer
Going Viral: The AAV Approach to Curing Cancer 1024 256 Emily Siebenmorgen

According to Timothy Cripe, MD, PhD, chief of the Division of Hematology and Oncology at Nationwide Children’s Hospital, it’s an incredible time to be working on cancer treatment – and now, targeted cancer prevention. “There’s so much going on in the cancer world these days,” says Dr. Cripe, who is also a principal investigator in…

Placing Value on a Pediatric Surgeon’s Academic Work
Placing Value on a Pediatric Surgeon’s Academic Work 1024 683 Mary Bates, PhD

The addition of an academic RVU system to an existing work RVU-based incentivization plan boosted academic productivity in the Department of Pediatric Surgery at Nationwide Children’s Hospital. At many institutions, physicians and surgeons are compensated using a productivity formula based on work relative value units (wRVUs). wRVUs commonly quantify clinical work, practice expenses and professional…

FDA Approves Micro-dystrophin: A Small Gene With Big Promise
FDA Approves Micro-dystrophin: A Small Gene With Big Promise 1024 632 Abbie Miller

UPDATE: On June 22, 2023, the FDA announced accelerated approval for SRP-9001/ELEVIDYS for Duchenne muscular dystrophy (DMD) following decades of research in the Center for Gene Therapy at Nationwide Children’s Hospital. The FDA approved the treatment for pediatric patients 4-5 years old with DMD. Jerry Mendell, MD, principal investigator in the Center for Gene Therapy…

Nephrology Education Ripe With Opportunities to Improve Health Equity
Nephrology Education Ripe With Opportunities to Improve Health Equity 1024 575 Katie Brind'Amour, PhD, MS, CHES

The Health Care Justice Committee of the American Society of Nephrology has released recommendations for incorporating anti-racism and social justice concepts into education and mentoring with the goal of improving health equity.   In 2020, leaders in the American Society of Nephrology (ASN) reached out to Ray Bignall II, MD, pediatric nephrologist and assistant chief…

5 Things Neonatologists Should Know About Vitamin K Deficiency
5 Things Neonatologists Should Know About Vitamin K Deficiency 1024 683 Mary Bates, PhD

Vitamin K prophylaxis is safe and effective. Why are more parents refusing it, and what can be done? In a new perspective paper in the Journal of Perinatology, researchers from Nationwide Children’s say that vitamin K prophylaxis is an essential component of newborn care and yet, parental refusal of the intervention is on the rise.…

Using 3D Motion Capture Technology to Improve Outcomes
Using 3D Motion Capture Technology to Improve Outcomes 150 150 Mary Bates, PhD

  Kirsten Tulchin-Francis, PhD Kirsten Tulchin-Francis, PhD, joined Nationwide Children’s in March 2022 as director of orthopedic research and director of the Honda Center for Gait Analysis and Mobility Enhancement. In a Q&A, she discusses how she uses motion capture technology to improve outcomes for pediatric patients. What is your area of expertise? I have…

5 Things to Know About Prune Belly Syndrome
5 Things to Know About Prune Belly Syndrome 1024 683 Abbie Miller
Linda Baker, MD

Linda Baker, MD, urologist, principal investigator, and one of the world’s foremost experts on prune belly syndrome, recently joined Nationwide Children’s Hospital as the co-director of the Kidney and Urinary Tract Center. She shares some important things to know about this rare disease. 1. Prune belly is a rare, congenital malformation. Prune belly syndrome (PBS)…

New Guidelines Offer a Clear Path for Expandable Endoprosthesis Use in Post-Sarcoma Limb Reconstruction
New Guidelines Offer a Clear Path for Expandable Endoprosthesis Use in Post-Sarcoma Limb Reconstruction 150 150 Abbie Miller

Limb salvage operation is the standard of care for pediatric extremity bone sarcomas. These tumors often occur during the second decade of life, when children are growing rapidly. Reconstructive surgeries must account for anticipated remaining growth and the potential for resulting limb inequity. Osteosarcoma and Ewing sarcoma are the most common primary malignant bone tumors,…

Reducing Surgery and Its Associated Risks Through Advanced Interventional Endoscopy
Reducing Surgery and Its Associated Risks Through Advanced Interventional Endoscopy 150 150 Katie Brind'Amour, PhD, MS, CHES

Specially trained pediatric endoscopists can perform a range of diagnostic, interventional and therapeutic procedures for children, allowing them to avoid referral to adult endoscopists or even open surgery.   Muhammad Khan, MD, MPH, FASGE, the new director of interventional and diagnostic endoscopy at Nationwide Children’s Hospital, has big plans for the field of pediatric endoscopy.…

Celebrating More Than 100 Heart Transplants — And a Host of Other Heart Program Achievements
Celebrating More Than 100 Heart Transplants — And a Host of Other Heart Program Achievements 1024 746 Deipanjan Nandi, MD, MSc

Deipanjan Nandi, MD, MSc, shares an update about treating heart failure at Nationwide Children’s Hospital. From transplant to ventricular assist devices and pharmaceutical options, the patient-centered care offers innovative options for patients with heart failure.

The New Book on Youth Suicide Prevention
The New Book on Youth Suicide Prevention 150 150 John Ackerman, PhD and Lisa Horowitz, PhD, MPH

Our new open access Springer Series book helps close the gap between the latest research in youth suicide prevention and how to make an impact in our communities.  Youth suicide is a public health crisis. It is the second leading cause of death among youth aged 10-17 in the United States, according to data from…

InSight: Total Pancreatectomy With Islet Autotranplantation: The Basics
InSight: Total Pancreatectomy With Islet Autotranplantation: The Basics 1024 500 Katie Brind'Amour, PhD, MS, CHES
illustrated cross section of islets from pancreas

Download a PDF version of this image.       This feature was published in the Fall/Winter 2022 print issue. Download the full issue.  Image credit: Mandy Root-Thompson for Nationwide Children’s

New Initiative Aims to Bring Personalized Diagnosis and Treatment to Children With Solid Tumors
New Initiative Aims to Bring Personalized Diagnosis and Treatment to Children With Solid Tumors 1024 614 Lauren Dembeck
abstract art of magnifying glass over DNA strand

Collaboration among National Cancer Institute, Children’s Oncology Group and Nationwide Children’s Hospital will offer molecular characterization of childhood cancers. The National Institutes of Health’s National Cancer Institute (NCI), Children’s Oncology Group (COG), and Nationwide Children’s Hospital are poised to change the direction of pediatric cancer diagnosis and care through a new initiative that is the…

What’s the Relevance of Emotional Intelligence in Medicine?
What’s the Relevance of Emotional Intelligence in Medicine? 1024 386 Mike Patrick, MD and Erica Banta, MBA, LDSS

The practice of clinical medicine requires an intelligent mindset. Strong cognitive intelligence (IQ) is required to develop and maintain a substantial knowledge base and to call upon the critical thinking skills needed to synthesize a hundred points of data into a meaningful clinical picture, correct diagnosis and appropriate management plan. But another type of intelligence…

TPIAT: A Way Forward for Chronic Pancreatitis
TPIAT: A Way Forward for Chronic Pancreatitis 1024 500 Katie Brind'Amour, PhD, MS, CHES
illustrated cross section of islets from pancreas

Nationwide Children’s Hospital has assembled a veritable “dream team” of pancreatitis and pediatric transplant surgery experts to offer what they hope will become the world’s preeminent pediatric center for complex pancreatic care. Imagine your child suffering from a sudden, debilitating episode of abdominal pain. No obvious cause, no cure, just pain so severe it requires…

How One Family In Appalachia Changed the Medical Field’s Understanding of Pancreatitis
How One Family In Appalachia Changed the Medical Field’s Understanding of Pancreatitis 150 150 Katie Brind'Amour, PhD, MS, CHES

More than 30 years ago, a teenager from Kentucky named Kevin Slone had his first attack of acute pancreatitis. Doctors at the Cleveland Clinic urged his father, Bobby Slone, to try to document whether other people in the family had similar stomach problems. Bobby Slone took the homework to heart, documenting incidences of similar symptoms…

Integrating Behavioral Health and Primary Care Increases Access and Equity
Integrating Behavioral Health and Primary Care Increases Access and Equity 1024 683 Mary Bates, PhD

With mental health concerns on the rise, more primary care providers are integrating behavioral health services in their practices.  Despite increasing demand for behavioral health care, multiple barriers still exist that limit access for pediatric patients. In recent years, primary care settings across the country have responded by integrating mental health specialists into their practices.…

Salivary MicroRNAs: A Promising Biomarker for Persistent Post-concussive Symptoms in Children With Concussion
Salivary MicroRNAs: A Promising Biomarker for Persistent Post-concussive Symptoms in Children With Concussion 1024 768 Lauren Dembeck

The multidisciplinary study used children’s saliva and genomic analysis to identify promising biomarkers for persistent post-concussive symptoms. Predicting clinical recovery of children with concussion is challenging. While concussion symptoms typically resolve within one to three weeks, up to one-third of children develop persistent post-concussive symptoms (PPCS), including headache, nausea, dizziness, blurred vision, and sensitivity to…

Functional Neurological Disorders: What Pediatric Neurologists Should Know
Functional Neurological Disorders: What Pediatric Neurologists Should Know 150 150 Mary Bates, PhD

The diagnosis is common, yet this group of disorders has been subjected to confusion and stigma throughout history. Functional neurological disorders are a collection of disorders in which patients experience neurological symptoms, such as numbness, weakness, seizure-like events, or abnormal gait or movements, but without a visible underlying pathology. Underlying mechanisms for these disorders are…

When Every Week Matters: Advancing a Treatment to the Clinic
When Every Week Matters: Advancing a Treatment to the Clinic 1024 491 Natalie Wilson
Illustrations of clock faces at different times and angles

Researchers and regulatory experts bring a potential new therapy for a deadly neurodegenerative disease from IND application to clinical trial enrollment in a matter of weeks, setting a new standard in translating therapies from bench to bedside. In October 2021, the Office of Research Regulatory Affairs (ORRA) at Nationwide Children’s Hospital submitted an Investigational New…

Optimizing the Body’s Natural Cancer Killers
Optimizing the Body’s Natural Cancer Killers 1024 649 Katie Brind'Amour, PhD, MS, CHES

Recent advances in the expansion and production of natural killer cells offers pediatric patients new hope for remission after high-risk cancer diagnoses. Natural killer (NK) cells are the innate immune system’s first line of defense for viral infections. Although these white blood cells don’t have the antigen-specific “memory” that characterizes T cells or the antibody-producing…

Moving Emergency Medicine Research Forward
Moving Emergency Medicine Research Forward 1024 614 Abbie Miller

Utilizing exception from informed consent studies and a national network of emergency medicine experts, clinician-scientists are advancing research to uncover best practices and improve care. Exception from informed consent (EFIC) is a special rule set utilized by the U.S. Food and Drug Administration (FDA) to support research in emergency situations. The word exception is particularly…

Improving Racial Diversity and Equity in Clinical Trials
Improving Racial Diversity and Equity in Clinical Trials 1024 511 Jeb Phillips

There is now broad consensus across medicine that clinical trials must be more representative of minority populations. How can that be achieved? Last year, a group of Nationwide Children’s Hospital neonatologists published an unusually pointed critique of racial and ethnic representation in neonatal clinical trials in the Journal of Perinatology. They wrote that the lack…

Exploring Dopamine Genotype as a Moderator of the Effect of Parental Behavior on Children’s Self-Control
Exploring Dopamine Genotype as a Moderator of the Effect of Parental Behavior on Children’s Self-Control 150 150 Daphne Vrantsidis, PhD

Did you know developmental psychologists have found the secret to life success? It’s not eating your vegetables or doing your homework or anything else your parents or teachers told you growing up. It’s how long you waited to eat a marshmallow when you were 4 years old. Delaying gratification — waiting 5 minutes to eat…

First In Human
First In Human 1024 752 Abbie Miller
Top-down view of heart valves

The Heart Center at Nationwide Children’s Hospital has a long history of helping to bring innovations from the lab to the patient. The latest, the Autus Valve, aims to improve care and outcomes for children with pulmonary valve disease. In December 2021, Mark Galantowicz, MD, cardiothoracic surgeon and director of The Heart Center at Nationwide…

Novel Digital Storybook Intervention Aims to Support Children With Hearing Loss
Novel Digital Storybook Intervention Aims to Support Children With Hearing Loss 1024 683 Abbie Miller

Hear Me Read, invented and developed at Nationwide Children’s Hospital, is now being validated in a prospective clinical trial. For children who are deaf or hard of hearing, access to sound via hearing aids and cochlear implants and intensive speech therapy with highly trained verbal therapists are important parts of developing speech and literacy. “Children…