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Abbie Roth

To Test or Not to Test: The Inherited Thrombophilia Question
To Test or Not to Test: The Inherited Thrombophilia Question 150 150 Abbie Roth

When a result provides questionable or limited clinical utility, physicians and genetic counselors must educate patients and providers on the pros and cons of genetic testing. Between the increased ease of clinical genetic testing and the direct-to-consumer genetic testing kits now available, physicians and genetic counselors face education challenges that simply did not exist a…

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Building Heart Valves From Extracellular Matrix
Building Heart Valves From Extracellular Matrix 150 150 Abbie Roth

Physicians at Nationwide Children’s Hospital describe the use of an extracellular matrix cylindar valve in the mitral position. At The Heart Center at Nationwide Children’s, Patrick McConnell, MD, a pediatric cardiothoracic surgeon, is using an alternative to the traditional cadaveric, mechanical or biological replacement valve. In a recent case report published in Echocardiography, Dr. McConnell and his colleagues describe the…

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Two Classes of GGAA-Microsatellites in a Ewing Sarcoma Context
Two Classes of GGAA-Microsatellites in a Ewing Sarcoma Context 150 150 Abbie Roth

Characterization of GGAA-microsatellites provides insight in the role of noncoding DNA in cancer susceptibility and therapeutic development. In a study published in PLOS ONE, researchers describe two types of GGAA-microsatellites and their roles in EWS/FLI binding and gene regulation in Ewing sarcoma. Ewing sarcoma is the second most common pediatric bone malignancy. It is initiated by chromosomal…

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Phase 1 Study Shows Promise of Gene Replacement Therapy for Spinal Muscular Atrophy Type 1
Phase 1 Study Shows Promise of Gene Replacement Therapy for Spinal Muscular Atrophy Type 1 1024 575 Abbie Roth

The phase 1 clinical study shows that gene therapy extends survival of patients and supports achievement of milestones previously unseen in the natural course of the disease. A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1), according to a study published in…

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On the Road to Eliminating RSV
On the Road to Eliminating RSV 150 150 Abbie Roth

With the recent publication of two papers, researchers shed light on factors that influence disease severity and immune response to respiratory syncytial virus infection in infants and young children. “RSV is a very common infection in infants and young children – almost everyone will be infected by age 2. In the United States, 2-3 percent…

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A New Paradigm for Treating Transcription Factor-Driven Cancers
A New Paradigm for Treating Transcription Factor-Driven Cancers 150 150 Abbie Roth

Researchers describe how the association of the EWS/FLI transcription factor with GGAA-microsatellites drives Ewing sarcoma. In the current issue of Proceedings of the National Academy of Sciences, researchers from Nationwide Children’s Hospital describe a new paradigm for treating transcription factor-driven cancers. The study focuses on Ewing sarcoma and how the EWS/FLI transcription factor drives the malignancy…

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Taking Innovation to Heart: Next Gen Interventions in Heart Valve Disease
Taking Innovation to Heart: Next Gen Interventions in Heart Valve Disease 1024 575 Abbie Roth

From bioengineers to interventional cardiologists, molecular biologists to cardiothoracic surgeons, experts with diverse backgrounds are focusing on the problem of heart valve disease in children. Heart valve disease affects more than 5 million Americans. And while acquired disease in the adult population certainly accounts for much of this, children with heart valve disease face multiple…

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Creative Reality: Using a New Platform Technology to Improve Patient Experience
Creative Reality: Using a New Platform Technology to Improve Patient Experience 1024 575 Abbie Roth

Amy Dunn, MD, had a problem. Some of her patients in the hematology clinic at Nationwide Children’s Hospital receive hundreds of needle sticks each year. “Needle phobia is very real for these patients and their families,” says Dr. Dunn, director of Pediatric Hematology at Nationwide Children’s. “In some cases, ports need to be implanted so that these…

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Mechanism for Expulsion of DNA from NTHI Described
Mechanism for Expulsion of DNA from NTHI Described 150 150 Abbie Roth

Researchers at Nationwide Children’s publish breakthrough discovery revealing how DNA and DNABII proteins are released into the biofilm matrix. Nontypeable Haemophilus influenzae (NTHI) bacteria, a common culprit in otitis media, are known for their ability to create dense biofilms. As the subject of much biofilm and vaccine research, they are increasingly understood as complex and surprising organisms.…

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Immune Profiling Leads to Implications for Immunotherapy for NF1-Associated Tumors
Immune Profiling Leads to Implications for Immunotherapy for NF1-Associated Tumors 150 150 Abbie Roth

Profiling reveals histologic subtype distinctions and heterogeneity of neurofibromatosis type 1-associated tumors. Neurofibromatosis type 1 (NF1) — an autosomal dominant disorder affecting approximately one in every 3,500 people — results in dysfunctional neurofibromin, a protein expressed throughout the body and involved in the RAS signaling pathway. Virtually all patients with NF1 develop benign peripheral nerve…

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Neurosurgery May Provide Lasting Relief From Spasticity
Neurosurgery May Provide Lasting Relief From Spasticity 1024 575 Abbie Roth

Selective dorsal rhizotomy can provide life-changing results, but patients work hard in physical and occupational therapy to get there. When Sushma Manjunatha watches her daughter sleep, she sees something new. Instead of being tightly curled up, with legs pulled in, her daughter’s legs are stretched out, finally relaxed. This change is the result of a…

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The First Study of Oncolytic HSV-1 in Children and Young Adults With Cancer Indicates Safety and Tolerability
The First Study of Oncolytic HSV-1 in Children and Young Adults With Cancer Indicates Safety and Tolerability 1024 575 Abbie Roth

The phase 1 trial shows safety and tolerance of HSV1716 in the pediatric population; evidence of viral replication in the blood and acute inflammation on PET/CT scans suggest a phase 2 trial for dosage and efficacy is warranted. HSV1716 – an oncolytic herpes simplex virus-1 – has been studied in adults via injection into the…

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A Narrow Focus: Perfecting Tissue Engineered Vascular Grafts
A Narrow Focus: Perfecting Tissue Engineered Vascular Grafts 1024 575 Abbie Roth
image of heart with fontan conduit highlighted

A pair of surgeon-researchers is perfecting tissue engineered vascular grafts through bench, clinical and computational modeling studies.

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Discovery to Drug Development: Expanding the Role of Academic Centers
Discovery to Drug Development: Expanding the Role of Academic Centers 1024 575 Abbie Roth

As more researchers at academic centers become involved in drug development, institutions are responding with support and guidance. Researchers at academic institutions regularly make discoveries about disease processes and potential therapeutic agents. Translational medicine is focused on moving these discoveries out of the laboratory and into the clinic where they can potentially help patients. But…

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Human Trafficking: How many victims have you treated?
Human Trafficking: How many victims have you treated? 1024 575 Abbie Roth

Data shows that health care providers may be coming into contact with victims and those at risk with more frequency than expected. How many victims have you treated? The answer is probably higher than you think. According to a report published in the Annals of Health Law, 88 percent of sex trafficking survivors reported contact with…

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Two Investigational Antitumor Agents Work Better Together Against MPNST and Neuroblastoma
Two Investigational Antitumor Agents Work Better Together Against MPNST and Neuroblastoma 150 150 Abbie Roth

The synergistic effects of a kinase inhibitor and an oncolytic herpes virus show promise for difficult-to-treat neuroblastomas and malignant peripheral nerve sheath tumors. Two investigational agents, Aurora A kinase inhibitor (alisertib) and HSV1716, a virus derived from HSV-1 and attenuated by the deletion of RL1, have shown some antitumor efficacy in early clinical trials as…

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Hormonal Contraception Safer Than Expected For Women With Diabetes
Hormonal Contraception Safer Than Expected For Women With Diabetes 150 150 Abbie Roth

Women with diabetes often fall through the cracks when it comes to prescription contraception. A new study illuminates the issues and highlights safe options. The use of contraception to prevent unplanned pregnancies is an important aspect of women’s health. For women with chronic health conditions, family planning has important implications for the health of the…

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Subclinical Muscle Involvement May be Missed With Amyopathic Juvenile Dermatomyositis Diagnosis
Subclinical Muscle Involvement May be Missed With Amyopathic Juvenile Dermatomyositis Diagnosis 150 150 Abbie Roth

Study shows that some children diagnosed with amyopathic dermatomyositis have subclinical muscle involvement, highlighting the need for standardized workup and treatment protocols. Juvenile dermatomyositis (JDM) is an autoimmune disease that classically presents in preschool to school aged children with a rash and muscle weakness. In some cases, children may present with the rash, but no muscle…

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For AYA Females With Rhabdomyosarcoma, Consider Routine Examination or Imaging of Breasts
For AYA Females With Rhabdomyosarcoma, Consider Routine Examination or Imaging of Breasts 150 150 Abbie Roth

Rhabdomyosarcoma with breast involvement is found almost exclusively in females. A recent case series highlights the need for guidelines for follow-up imaging. Rhabdomyosarcoma (RMS) is a soft tissue sarcoma associated with metastasis and inferior outcomes in adolescent and young adult (AYA) patients (those with cancer diagnosis at age 15-39 years). Metastases to the breast have…

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What Do Space Rocks Have to Do With Preterm Birth?
What Do Space Rocks Have to Do With Preterm Birth? 150 150 Abbie Roth

A brief history of nanobacteria and their implications for human health. I remember when nanobacteria were a really big deal. Press-conference-by-POTUS-about-evidence-of-extraterrestrial-life-level big deal. I hadn’t thought much about them until recently, when they made a surprise appearance in a presentation on idiopathic preterm birth by Irina Buhimschi, MD, director of the Center for Perinatal Research at The…

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From Military Zones to Pediatric Trauma Centers, Implementing Massive Transfusion Protocols
From Military Zones to Pediatric Trauma Centers, Implementing Massive Transfusion Protocols 150 150 Abbie Roth

While military and adult research has shown massive transfusion protocols to be lifesaving, implementation and validation in pediatrics lags. When someone is critically injured with life-threatening bleeding, the primary objective of the care team is to stop the bleeding and replace the lost blood. Historically, children in this situation were administered red blood cells (RBCs)…

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Solving the Problem of Managing Big Genomic Data
Solving the Problem of Managing Big Genomic Data 150 150 Abbie Roth

Researchers at Nationwide Children’s Hospital complete a first-of-its-kind project to evaluate a large-scale genomic data management system on the scale of up to one million genomes. The influx of genomics data resulting from the increasing affordability of whole exome/genome sequencing and President Obama’s Precision Medicine Initiative requires a novel technological solution to data storage, communication…

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How to Integrate Genomics into Clinical Practice
How to Integrate Genomics into Clinical Practice 150 150 Abbie Roth

Recommendations from the Clinical Genetics Think Tank outline five key areas of focus for bringing genome and exome sequencing into the clinic. Clinical genome and exome sequencing (CGES) as a diagnostic tool is altering practice for clinical geneticists, genetic counselors and other clinical specialists. The Clinical Genetics Think Tank (CGTT) has identified five areas of…

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In Sight: Two Stage Surgery for Epilepsy
In Sight: Two Stage Surgery for Epilepsy 150 150 Abbie Roth

Surgery proves to be a viable option for patients with medically refractory epilepsy. Childhood onset epilepsy affects 1 percent of children worldwide. About 25 to 30 percent of these patients will have medically refractory epilepsy, continuing to have seizures despite using two or more antiseizure medications. Options for this group of patients include intercranial epilepsy surgery, Vagus…

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Harnessing the Immune System: Has the Cure for Cancer Been Within Us All Along?
Harnessing the Immune System: Has the Cure for Cancer Been Within Us All Along? 1024 575 Abbie Roth

By learning to manipulate the immune system to target cancer cells, clinician-scientists are ushering in a new era in cancer treatments. The advances in cancer immunotherapy have been headline-making, and some clinical studies have produced stories of near-miraculous recoveries. From the immunotherapy drug credited with curing former president Jimmy Carter’s cancer (pembrolizumab) to the promising…

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Researchers at Nationwide Children’s Hospital Describe New Type of Cancer Therapy
Researchers at Nationwide Children’s Hospital Describe New Type of Cancer Therapy 150 150 Abbie Roth

The new chemotherapy is effective against both pediatric and adult cancers, and it allows other chemotherapies to more readily reach their targets. A study conducted at Nationwide Children’s Hospital has found that a new chemotherapy is effective against both pediatric and adult cancers, and that it allows other chemotherapies to more readily reach their targets.…

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Considering Peptide Tuning of Natural Killer (NK) Cells
Considering Peptide Tuning of Natural Killer (NK) Cells 150 150 Abbie Roth

Researchers combine modeling and functional studies of interactions between MHC-I and NK cells to learn how they operate on a molecular level. Natural killer (NK) cells are cytotoxic lymphocytes — important immune cells of the innate immune system. NK cells are increasingly understood to be significant in the way the body responds to viruses, such…

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Specialty Pharmacy for Cystic Fibrosis Improves Access to Medication
Specialty Pharmacy for Cystic Fibrosis Improves Access to Medication 150 150 Abbie Roth

In a project started to increase the number of patients taking Orkambi®, access and adherence increases for all medications. The Nationwide Children’s Hospital Cystic Fibrosis (CF) Program cares for a large population of patients of all ages with cystic fibrosis.  The program has participated in many recent clinical trials, including one that led to the FDA approval…

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First Implantable Hemodynamic Monitoring Device in Single Ventricle Fontan Anatomy
First Implantable Hemodynamic Monitoring Device in Single Ventricle Fontan Anatomy 150 150 Abbie Roth

The first IHM in a patient with Fontan anatomy and physiology enables real-time monitoring of central venous and pulmonary artery pressures. While the Fontan procedure has improved the short- and mid-term outcomes for patients born with single ventricle anatomy, long-term complications of Fontan circulation include heart failure. These complications are thought to be secondary to…

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Achieving CLARITY
Achieving CLARITY 1024 575 Abbie Roth
conceptual art of DNA

Multidisciplinary team from Nationwide Children’s wins international genomics competition. Accurate, patient-centered, comprehensive. That’s how the judges and leaders of the CLARITY Undiagnosed Challenge described the work of a team from Nationwide Children’s Hospital. Those same words could be used to describe the highly motivated and diverse team who surprised themselves by winning the challenge. “When we entered the challenge, we knew we would learn…

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Simulating Surgery With High-Performance Computing
Simulating Surgery With High-Performance Computing 1024 575 Abbie Roth

By applying high-performance computing to the field of otolaryngology, a team of researchers is developing a simulation environment for teaching surgical techniques related to the temporal bone. The purpose of training — whether a fire drill or practicing a surgical technique — is to create successes and avoid failures. “The impact of training is safety…

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The Smallest Victims of the Opioid Crisis
The Smallest Victims of the Opioid Crisis 1024 575 Abbie Roth
Black and white photo of infant crying in hospital

Research, protocols and community connections lead to help infants with neonatal abstinence syndrome and their mothers. It’s a sound you’d never forget. The panicked, high-pitched cry of a newborn withdrawing from the drugs his mother took. All the sensations that drive an addict to use again and again just for the relief – shaking, vomiting,…

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Vaccine Fails to Reactivate Immunity to Hepatitis C Virus
Vaccine Fails to Reactivate Immunity to Hepatitis C Virus 150 150 Abbie Roth

T cells remain inactivated even after immunization in subjects with persistent, controlled infections. Two papers recently published in Hepatology uncovered evidence of permanent immune system damage after hepatitis C virus (HCV) infection. The studies used a vaccine currently in clinical trials to attempt to restore immunity against HCV in animal models and humans with chronic HCV infection. “In chronic HCV, CD8+…

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Taking Aim at the Opioid Problem
Taking Aim at the Opioid Problem 1024 683 Abbie Roth
open bottle of pills

Pediatric specialists offer practical advice for protecting vulnerable patients from a growing epidemic. The opioid epidemic in the United States is so widespread that even parents and teachers are now being issued opioid overdose kits complete with naloxone. It’s in rural communities, suburban neighborhoods and inner cities. It’s so far-reaching that physicians and non-experts alike are being called to work together…

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How to Increase Adherence to Screening Guidelines for Children With Down Syndrome
How to Increase Adherence to Screening Guidelines for Children With Down Syndrome 150 150 Abbie Roth

Recent study shows that while most families and physicians support the guidelines, patients are not receiving the recommended screenings. Children with Down syndrome have multiple medical conditions and cognitive impairments related to the additional genetic material from chromosome 21. Because of common comorbid conditions in the Down syndrome population such as heart defects, hearing loss,…

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New Approach to Gathering Protein Signatures
New Approach to Gathering Protein Signatures 150 150 Abbie Roth

In the first comprehensive study of its kind, researchers simultaneously analyzed both host and bacteria protein signatures from a single, tiny tissue sample. A novel study from researchers at Nationwide Children’s Hospital has shown that it is possible to obtain both host and bacteria protein signatures in a sample smaller than the average human biopsy.…

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Loss of MHCI in Motor Neurons Leads to ALS Astrocyte Toxicity
Loss of MHCI in Motor Neurons Leads to ALS Astrocyte Toxicity 150 150 Abbie Roth

New research shines a light on the role of MHCI in astrocyte-induced death of motor neurons in amyotrophic lateral sclerosis and pointing to a potential therapeutic target. Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a devastating progressive neurodegenerative disease that results in the death of motor neurons, the nerve cells that…

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Putting the Brakes on Myopia Progression
Putting the Brakes on Myopia Progression 150 150 Abbie Roth

Researchers are studying how well commercially available bifocal contact lenses limit the progression of myopia. Myopia, or nearsightedness, is a growing problem in the United States and around the world. Research suggests 30 to 40 percent of adults in Europe and the United States and up to 80 percent in Asia have myopia. It usually…

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Boosting Research With a Down Syndrome Biobank
Boosting Research With a Down Syndrome Biobank 150 150 Abbie Roth

A new biobank for Down syndrome blood samples will enable clinical and translational researchers everywhere to shed light on conditions related to Down syndrome, including Alzheimer’s disease. Certain conditions such as congenital heart disease, childhood leukemia and epilepsy are more common in patients with Down syndrome than in the general population. Other conditions, including solid…

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HIV in the Millennial World
HIV in the Millennial World 150 150 Abbie Roth

Adolescents and young adults ages 13 to 24 comprise an increasingly large proportion of new HIV infections in the United States. A new generation of youth is experiencing increasing rates of HIV transmission, and data from the Centers for Disease Control and Prevention (CDC) indicate half of those infected youth (ages 13-24) don’t even know…

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The Transfusion Evolution
The Transfusion Evolution 576 367 Abbie Roth

The first successful open heart surgery with cardiopulmonary bypass was performed in 1953, but it wasn’t until the 1970s that these surgeries began to have high success rates — due in large part to the availability of fresh whole blood transfusions. However, fresh whole blood is difficult to attain. In response, blood component transfusions became…

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Acetabular “Fleck” Sign Predictive of Labral Avulsion
Acetabular “Fleck” Sign Predictive of Labral Avulsion 150 150 Abbie Roth

A “fleck” sign on the postreduction CT scan calls for high suspicion of labral pathology, even in cases of congruent closed reductions. Traumatic posterior hip dislocations and subluxations are typically treated with a closed reduction in pediatric patients. For patients who have a congruent hip reduction, the course of treatment often ends here. However, a new study…

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Revealing the Secrets of Sepsis
Revealing the Secrets of Sepsis 969 533 Abbie Roth
Illustration of NK Cells, T Cells, other immune cells floating across white background

Charting new territory in the understanding of how the immune system responds to sepsis. Two children are admitted to the hospital with sepsis. Both receive antibiotics and fluid resuscitation within the critical first hour. Why does one get better after the initial crisis while the other goes on to develop additional infections and multiple organ…

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Sickle Cell Disease: Global Disparities in Prevalance and Outcomes
Sickle Cell Disease: Global Disparities in Prevalance and Outcomes 150 150 Abbie Roth

Unique challenges exist for people with sickle cell disease depending on where they live. An estimated 300,000 to 500,000 babies worldwide are born with sickle cell disease (SCD) each year. In Africa and India, where SCD is most prevalent, newborn testing is not performed, and many children with sickle cell disease die before they are…

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Methadone Weaning: The Role of the Pharmacist
Methadone Weaning: The Role of the Pharmacist 150 150 Abbie Roth

Pharmacist-led methadone tapers are improving outcomes and reducing practice variation in pediatric intensive care units. In neonatal and pediatric intensive care units, opioid use is necessary for controlling pain and as an adjunct to sedation during mechanical ventilation. However, after as few as five days of use, approximately 50 percent of patients will experience withdrawal…

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Visualizing Gene Therapy for SMA
Visualizing Gene Therapy for SMA 150 150 Abbie Roth

Spinal muscular atrophy (SMA), a degenerative neuromuscular disease, is the most common genetic cause of death for infants. Virtually all children affected with SMA type 1 die by 2 years of age. In 2014, Jerry Mendell, MD, director of the Center for Gene Therapy in the Research Institute at Nationwide Children’s Hospital, began a phase 1 gene transfer clinical…

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Planning, Teamwork and Technology Essential to Conjoined Twins’ Separation
Planning, Teamwork and Technology Essential to Conjoined Twins’ Separation 150 150 Abbie Roth

In a meticulously planned 16-hour operation, a 30-person team from four specialties successfully separated 11-month-old twins conjoined at the buttocks and lower spine. Specialists from General Pediatric Surgery, Plastic and Reconstructive Surgery, Neurosurgery and Colorectal Surgery worked together to plan and execute a successful separation of conjoined twin girls, Acen and Apio, earlier this month. “In surgery,…

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Stress Dose Hydrocortisone Effects on Neurodevelopment for Extremely Low Birthweight Infants
Stress Dose Hydrocortisone Effects on Neurodevelopment for Extremely Low Birthweight Infants 150 150 Abbie Roth

First placebo-controlled study on stress dose hydrocortisone and neurodevelopment shows that higher doses of hydrocortisone are not associated with brain injury or neurodevelopmental impairments, but may not be effective in reducing risk for bronchopulmonary dysplasia. Hydrocortisone is one of the 15 most frequently prescribed medications in extremely low birth weight (≤1000 g) infants in the…

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Treating Sickle Cell Disease in the United States
Treating Sickle Cell Disease in the United States 1024 575 Abbie Roth

While people with sickle cell disease have better outcomes in the United States and other Western countries, progress in treating the disease has been slow moving. Sickle cell disease is marked by painful sickle cell crises, in which sickle-shaped cells get distorted in the small vessels and cause problems including disrupting blood flow and a…

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Making Progress: Advances in Life-Long Care for ACHD
Making Progress: Advances in Life-Long Care for ACHD 150 150 Abbie Roth

With adults comprising two-thirds of the CHD population, the AHA has released a scientific statement outlining the needs of this growing demographic. The need for a life-long approach to cardiac care for patients with congenital heart disease is a crusade that has been long fought by pediatric cardiologists and adult CHD specialists for over 40…

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