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Hematology/Oncology/BMT

Treating Pediatric Melanoma: Ultrasound Surveillance vs. Completion Lymph Node Dissection for Sentinel Node Positive Patients
 Treating Pediatric Melanoma: Ultrasound Surveillance vs. Completion Lymph Node Dissection for Sentinel Node Positive Patients 1024 683 Pam Georgiana
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Treatments for pediatric melanoma are usually based on adult studies, but increasing recognition of the differences between pediatric and adult melanoma could change that. Pediatric melanoma is rare in the United States. The Skin Cancer Foundation reports that about 400 children under 20 years old are diagnosed with melanoma each year in the United States.…

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Combined Pediatric-Adult Anticoagulation Program Exceeds Goals
 Combined Pediatric-Adult Anticoagulation Program Exceeds Goals 1024 580 Mary Bates, PhD
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The program achieved excellent quality of anticoagulation therapy in children and adults. The goal of most anticoagulation programs is to improve the care of adult patients on warfarin therapy, specifically by treating and preventing thromboembolic events while minimizing bleeding risk. In a new study, researchers from Nationwide Children’s Hospital report excellent outcomes for a combined…

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Oncolytic Virus Immunotherapy Safe and Tolerable in Children With Relapsed or Refractory Solid Tumors
 Oncolytic Virus Immunotherapy Safe and Tolerable in Children With Relapsed or Refractory Solid Tumors 1024 683 Lauren Dembeck
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The overall survival rates for children with childhood cancers is now over 80%; however, for children with relapsed and refractory tumors, the survival rates remain low. “For example, children with relapsed or refractory neuroblastoma have a five-year survival of less than ten percent,” explains Keri A. Streby, MD, director of the Neuroblastoma Program in the…

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Fertility Concerns in Boys and Men With Sickle Cell Disease
 Fertility Concerns in Boys and Men With Sickle Cell Disease 1024 575 Mary Bates, PhD
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Color photo of Black father holding infant on shoulder in front of nursery background with clouds on the wall

Sickle cell disease and its treatments may impact fertility, but more research is needed to help counsel this population. Sickle cell disease (SCD) affects approximately 100,000 Americans, most of whom belong to underserved, minority populations. Advances in disease-modifying therapies, such as hydroxyurea, have led to better clinical outcomes. However, as more individuals with SCD survive…

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What Drives Nephrotic Syndrome Hypercoagulopathy?
 What Drives Nephrotic Syndrome Hypercoagulopathy? 150 150 Mary Bates, PhD
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Antithrombin deficiency does not play a major mechanistic role in the increased clotting risk of nephrotic syndrome, according to a new study. Patients with nephrotic syndrome are at risk for life-threatening venous thromboembolism complications. This risk is thought to be driven by an acquired hypercoagulopathy, or excessive blood clotting. Although the mechanisms underlying this hypercoagulopathy…

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Is Growth Hormone Replacement Safe Following Central Nervous System Tumors?
 Is Growth Hormone Replacement Safe Following Central Nervous System Tumors? 1024 683 Mary Bates, PhD
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Research suggests the benefits of growth hormone therapy outweigh the risks for pediatric cancer survivors. In a new review paper, researchers from Nationwide Children’s Hospital evaluated the risks and benefits associated with administering growth hormone to pediatric patients after diagnosis of a central nervous system tumor. The authors conclude that the therapy has many important…

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Comprehensive Care Model and Integrated Sickle Cell Disease-Pulmonary Clinic Improves Access and Outcomes
 Comprehensive Care Model and Integrated Sickle Cell Disease-Pulmonary Clinic Improves Access and Outcomes 1024 683 Jessica Nye, PhD
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A comprehensive care model which incorporates pulmonology services in the sickle cell disease (SCD) clinic aims to reduce access barriers and improve outcomes.   “Our sickle cell clinic at Nationwide Children’s Hospital used to see patients just for their comprehensive [sickle cell] care. One of the biggest morbidities and causes for mortality in patients with…

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Cancer-Causing Gene and Treatment Target for Ultra-Rare Rhabdomyosarcoma Confirmed Via Multiple Models
 Cancer-Causing Gene and Treatment Target for Ultra-Rare Rhabdomyosarcoma Confirmed Via Multiple Models 1024 764 Katie Brind'Amour, PhD, MS, CHES
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An international team has validated a cancer-causing gene fusion — and therapeutic targets — for an unusual presentation of muscle cancer in infants. In 2016, researchers first identified a novel gene mutation and fusion in rare cases of infants with rhabdomyosarcoma, a type of skeletal muscle-like cancer, normally only seen in school-age children and teenagers.…

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Virtual Support for Educating Parents of Newborns with Sickle Cell Trait
 Virtual Support for Educating Parents of Newborns with Sickle Cell Trait 1024 681 Mary Bates, PhD
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Black couple with newborn

A supplementary videoconferencing program engages parents and reduces worry about sickle cell trait status. Nearly 3 million people in the U.S. have sickle cell trait (SCT), in which they inherit one sickle cell gene and one normal gene. Individuals with SCT are usually asymptomatic; however, two parents with SCT have a 25% chance of having…

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Study Suggests Survivors of Pediatric Brain Tumors Have Altered Connectivity of Key Brain Regions
 Study Suggests Survivors of Pediatric Brain Tumors Have Altered Connectivity of Key Brain Regions 1024 575 Lauren Dembeck
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The findings contribute to a scant body of literature on the changes in functional connection within the brain and neurocognitive outcomes in survivors of pediatric brain tumors. Pediatric brain tumors are the most common solid tumor diagnosis among children in the United States. With advances in treatment, the 5-year survival rate among these children and…

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Ewing Sarcoma Transcription Factor Mediates Changes in Transcription by Reprogramming Chromatin Structure
 Ewing Sarcoma Transcription Factor Mediates Changes in Transcription by Reprogramming Chromatin Structure 1024 575 Abbie Miller
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yellow squiggly lines representing chromatin strands in the nucleus

By binding to DNA in the nucleus, the Ewing sarcoma-specific transcription factor EWS/FLI changes chromatin organization, leading to altered gene expression and the development of Ewing sarcoma.   A new report, published in Nucleic Acids Research, focuses on how the Ewing sarcoma-specific transcription factor EWS/FLI changes spatial organization of DNA in the nucleus of Ewing…

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Featured Researcher — Sarah O’Brien, MD
 Featured Researcher — Sarah O’Brien, MD 150 150 Katie Brind'Amour, PhD, MS, CHES
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The lab of Sarah O’Brien, MD, principal investigator in the Center for Child Health Equity and Outcomes Research at Nationwide Children’s Hospital, operates at the intersection of hematology and women’s health. Dr. O’Brien, who also leads the Young Women’s Hematology Clinic at Nationwide Children’s, applies her interests in big data and epidemiology to increase recognition…

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A Single-Dose Cancer Immunotherapy via Gene Transfer
 A Single-Dose Cancer Immunotherapy via Gene Transfer 150 150 Mary Bates, PhD
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Researchers at Nationwide Children’s Hospital report proof-of-principle results for a new gene therapy cancer treatment. The off-the-shelf, single-dose immunotherapy serves as an alternative to CAR-T therapy and can be engineered to be on-demand. T cells redirected to cancer cells either via a chimeric antigen receptor (CAR-T) or a bispecific molecule have been game-changers in treating…

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Investigators Develop Technique to Effectively Edit NK Cells to Target Specific Cancer Cells
 Investigators Develop Technique to Effectively Edit NK Cells to Target Specific Cancer Cells 1024 649 Katie Brind'Amour, PhD, MS, CHES
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The third of three major challenges in enabling the clinical potential of NK cells for cancer therapy has just been addressed, thanks to the combined talents of gene editing experts and NK cell specialists at Nationwide Children’s Hospital.   Originally, NK cells seemed promising for a natural therapy to fight cancer due to their innate…

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Hydroxyurea Dose and Adherence Affect Hematologic Outcomes in Children With Sickle Cell Disease
 Hydroxyurea Dose and Adherence Affect Hematologic Outcomes in Children With Sickle Cell Disease 1024 683 JoAnna Pendergrass, DVM
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Girl sitting on a bench with her phone smiling.

Optimal hydroxyurea dosing and medication adherence can improve hematologic outcomes for children with sickle cell anemia.   For pediatric patients with sickle cell anemia, hydroxyurea is highly effective at reducing disease complications. However, it can be challenging for these patients to take hydroxyurea for the long term. “There are many barriers to medication adherence in…

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

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Could a Sequence-Specific Splice Switching Oligonucleotide and Antibody Therapy Combination Successfully Treat Rhabdomyosarcoma?
 Could a Sequence-Specific Splice Switching Oligonucleotide and Antibody Therapy Combination Successfully Treat Rhabdomyosarcoma? 1024 683 Jessica Nye, PhD
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Blocking alternative splicing may be a viable therapeutic intervention strategy for reducing tumor growth in rhabdomyosarcoma (RMS).   RMS is the most common pediatric soft tissue sarcoma and is associated with poor prognosis, a high rate of metastasis and recurrence. RMS tumors overexpress insulin growth factor (IGF)-2, making the IGF pathway a potential interventional target.…

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Short- and Long-Term Kidney Issues in Children Treated for Leukemia
 Short- and Long-Term Kidney Issues in Children Treated for Leukemia 720 480 Mary Bates, PhD
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While outcomes for childhood acute lymphoblastic leukemia (ALL) have improved, researchers are finding long-term effects on kidney function that require monitoring. In a new study published recently in Pediatric Nephrology, researchers from Nationwide Children’s report the incidence of hypertension and kidney issues in pediatric patients diagnosed with acute lymphoblastic leukemia over a recent nine-year period.…

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Iron Deficiency and Fatigue Among Adolescents With Heavy Menstrual Bleeding
 Iron Deficiency and Fatigue Among Adolescents With Heavy Menstrual Bleeding 1024 673 Mary Bates, PhD
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Both iron deficiency and fatigue are common in adolescents with heavy menstrual bleeding and may not indicate presence of a bleeding disorder. In a new multicenter study of nearly 200 adolescents with heavy menstrual bleeding, researchers found a high prevalence of iron deficiency without concomitant anemia as well as a high prevalence of fatigue. The…

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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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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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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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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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Unraveling the Role of Pioneer Factors in Childhood Cancer
 Unraveling the Role of Pioneer Factors in Childhood Cancer 150 150 Abbie Miller
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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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Self-Administered Electronic Assessment Tool Helps Clinicians Screen for Pediatric Bleeding Disorders
 Self-Administered Electronic Assessment Tool Helps Clinicians Screen for Pediatric Bleeding Disorders 1024 684 JoAnna Pendergrass, DVM
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teen girl using cell phone

An electronic self-administered bleeding assessment tool is a time-saving measure to comprehensively evaluate a pediatric patient’s bleeding symptoms. Bleeding assessment tools (BATs), originally developed as research tools, are used clinically to comprehensively evaluate a patient’s bleeding symptoms. Along with clinical judgement, “BATs are used to discriminate between patients who do and do not have a…

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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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Featured Researcher — Susan Creary, MD, MSc
 Featured Researcher — Susan Creary, MD, MSc 150 150 Natalie Wilson
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Susan Creary, MD, MSc, is a principal investigator in the Center for Child Health Equity and Outcomes Research at the Abigail Wexner Research Institute at Nationwide Children’s Hospital, a physician in the Hematology, Oncology and Blood and Marrow Transplant (BMT) Clinic at Nationwide Children’s, and an associate professor of Pediatrics at The Ohio State University.…

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Indicators of Blood Clot Potential Directly Relate to Nephrotic Syndrome Severity
 Indicators of Blood Clot Potential Directly Relate to Nephrotic Syndrome Severity 150 150 Katie Brind'Amour, PhD, MS, CHES
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Researchers in the NEPTUNE network and collaborators at Nationwide Children’s Hospital have confirmed their preclinical studies demonstrating a direct correlation between the severity of disease and prothrombotic biomarkers in patients with nephrotic syndrome. Nephrotic syndrome (NS) dramatically increases the risk of a life-threatening blood clot, but until recently, clinicians have had no reasonable biomarker to…

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Targeting DIPG: The Most Puzzling of Pediatric Brain Tumors
 Targeting DIPG: The Most Puzzling of Pediatric Brain Tumors 772 447 Katie Brind'Amour, PhD, MS, CHES
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Survival has dramatically improved for numerous pediatric cancers over the last several decades, with a notable and very deadly exception: diffuse intrinsic pontine glioma (DIPG). Now, a community of researchers and clinician-scientists have set the stage for a renewed — and better-armed — assault against this beast of a brain tumor. The past 50 years…

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Modifying NK Cells With CRISPR/Cas9
 Modifying NK Cells With CRISPR/Cas9 969 533 Mary Bates, PhD
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Illustration of NK Cells, T Cells, other immune cells floating across white background

A proof-of-concept study shows success in modifying NK cells for multiple myeloma. In a new proof-of-concept study, researchers from Nationwide Children’s Hospital used CRISPR/Cas9 technology to genetically modify natural killer immune cells, which they then showed are able to address a recognized hurdle in immunotherapy of multiple myeloma. “It was an amazing feeling when we…

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Protecting an ‘Open Future:’ Improving Fertility Preservation Decision-Making for Young Males With Cancer
 Protecting an ‘Open Future:’ Improving Fertility Preservation Decision-Making for Young Males With Cancer 1024 575 Katie Brind'Amour, PhD, MS, CHES
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Color photo of Black father holding infant on shoulder in front of nursery background with clouds on the wall

Evidence from a new study highlights the importance of family-centered decision processes and clinician facilitation in improving fertility preservation uptake prior to cancer treatment. Numerous cancer treatments can reduce fertility or render some patients entirely infertile, but fertility preservation (FP) services have historically had low rates of uptake. When a young person is diagnosed with…

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Inverse Relationship Between Age and Dose of Blood Thinners for Children
 Inverse Relationship Between Age and Dose of Blood Thinners for Children 1024 495 Natalie Wilson
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blood cells

Infants and young children require higher doses of enoxaparin to treat blood clots than do older children. A blood clot, or thrombosis, isn’t a common problem in children, but it can be a dangerous one. Without proper treatment, a thrombosis can cause a host of long-term problems up to and including permanent damage to patients’…

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Novel Genetic Driver Discovered for Pediatric Meningiomas Using Molecular Profiling
 Novel Genetic Driver Discovered for Pediatric Meningiomas Using Molecular Profiling 1024 575 Katie Brind'Amour, PhD, MS, CHES
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Researchers have uncovered a rare subset of meningiomas with a genetic driver shared by another cancer type, opening the door to new therapeutic considerations. When an interesting or intractable cancer case arises at Nationwide Children’s Hospital, researchers and clinicians involved in the Brain Tumor Protocol through The Steve and Cindy Rasmussen Institute for Genomic Medicine…

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CLABSI in Hematology and Oncology: Progress Toward Zero
 CLABSI in Hematology and Oncology: Progress Toward Zero 1024 576 Katie Brind'Amour, PhD, MS, CHES
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Blood cells

A thorough review of five years of CLABSI data reveals key gaps in clinical knowledge that must be addressed to further reduce infection rates. For some time, any bloodstream infection (BSI) in a patient with a central venous catheter was considered a central line-associated bloodstream infection (CLABSI). In 2013, refined definitions enabled a distinction between…

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Boosting Natural Killer Cells for the Treatment of COVID-19
 Boosting Natural Killer Cells for the Treatment of COVID-19 150 150 Lauren Dembeck
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Natural killer cells — critical for immune surveillance and host defense — have an important role in cancer immunotherapy and antiviral responses. While driving to the laboratory one day, Brian Tullius, MA, MD, a fourth-year bone marrow transplant and cell therapies fellow at Nationwide Children’s in the Lee Lab, had an epiphany. “I thought, ‘wait…

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Can Smartphone Apps Keep AYA Oncology Patients Plugged in to Their Medication Routines?
 Can Smartphone Apps Keep AYA Oncology Patients Plugged in to Their Medication Routines? 1024 684 Natalie Wilson
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teen girl using cell phone

Studies show that suboptimal medication adherence may be a barrier to increased survival rates among adolescents and young adults with cancer. Anthony Audino, MD, explores the utility of medication adherence apps to meet the unique needs of these patients. The incidence of cancer among adolescents and young adults (AYA), or those 15-39 years old, has…

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Assessing the Impact of COVID-19 on Pediatric Cancer Research
 Assessing the Impact of COVID-19 on Pediatric Cancer Research 1024 575 Lauren Dembeck
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Times of uncertainty offer an opportunity to reflect and reevaluate priorities. “This is a time that warrants resiliency and different approaches for dealing with difficulties, and pediatric cancer research is a field that reflects the resilient spirit of the kids that we are striving to find cures for,” says Jeffery Auletta, MD, director of the Blood…

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Managing Lymphatic Flow Disorders: Expanding Care Through Collaboration
 Managing Lymphatic Flow Disorders: Expanding Care Through Collaboration 1024 683 Abbie Miller
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Alteration in the Lymphatic System Leading to Flow Disorders The lymphatic system plays a critical role in circulating a clear to yellow-colored fluid called lymph, which contains proteins, immune factors and cells throughout the body. It is also responsible for collecting fats and fat-soluble vitamins from the gastrointestinal tract in cloudy-appearing chylous fluid. This fluid…

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Novel Genetic Variant Identified in CARMIL2 Deficiency
 Novel Genetic Variant Identified in CARMIL2 Deficiency 1024 575 Lauren Dembeck
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International program facilitates the diagnosis of a rare immunodeficiency in two children caused by a novel genetic variant. A multidisciplinary team of physicians and scientists at Nationwide Children’s Hospital have identified a novel loss-of-function variant causing CARMIL2 deficiency, an inborn error of immunity, in two Saudi Arabian brothers born to consanguineous parents. The case was…

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Novel Use of Vedolizumab Resolves Steroid-Refractory Acute Graft-Versus-Host Disease in a Pediatric Transplant Recipient
 Novel Use of Vedolizumab Resolves Steroid-Refractory Acute Graft-Versus-Host Disease in a Pediatric Transplant Recipient 1024 575 Lauren Dembeck
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Vedolizumab is a monoclonal antibody that targets α4β7 integrin present on gut homing T cells and is indicated for adult inflammatory bowel disease Physicians at Nationwide Children’s Hospital have successfully treated steroid-refractory gastrointestinal acute graft-versus-host disease (SR GI aGVHD) with adjuvant vedolizumab therapy in a pediatric allogeneic hematopoietic cell transplant (HCT) recipient. The case was…

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Collaborative Study Seeks to Understand Gene Expression Changes During Acute Events in Patients With SCD
 Collaborative Study Seeks to Understand Gene Expression Changes During Acute Events in Patients With SCD 1024 575 Natalie Wilson
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Researchers explore gene responses among SCD patients hospitalized for acute complications to inform understandings of the under-studied disease. Sickle cell disease (SCD) is a chronic, genetic disorder characterized by structural changes in circulating red blood cells. According to the CDC, SCD affects approximately 100,000 individuals in the United States alone and accounts for 3,000 births…

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Preventing and Ameliorating Acute and Chronic Kidney Damage After Chemotherapy
 Preventing and Ameliorating Acute and Chronic Kidney Damage After Chemotherapy 720 480 Katie Brind'Amour, PhD, MS, CHES
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Research looking at mitochondrial targets in kidney health holds promise for cisplatin-induced kidney injury. Recent work exploring the mitochrondrial metabolism in the kidneys following exposure to cisplatin, a common chemotherapy, has revealed a key role of superoxide (O2•-, an indicator of oxidative stress in renal cells associated with cell damage and death) in both acute…

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Clinical Trials for Blood Cancers: Advancing Options for Children and Young Adults
 Clinical Trials for Blood Cancers: Advancing Options for Children and Young Adults 1024 683 Abbie Miller
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Most cancer treatments are first discovered, developed and approved for adult patients. But translating those therapies to pediatric and young adult patients with cancers can be more of a winding path than a superhighway. At Nationwide Children’s Hospital, the Division of Hematology, Oncology and Blood and Marrow Transplant is dedicated to participating in and supporting clinical…

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Hemophilia Gene Therapy Trials Aim to Reduce Patient Burden
 Hemophilia Gene Therapy Trials Aim to Reduce Patient Burden 1024 495 Katie Brind'Amour, PhD, MS, CHES
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blood cells

Clinical trials using gene therapy to boost the body’s production of clotting factor aim to remove the need for regular infusions — ideally giving patients years or even decades free from daily worry about their condition. Unlike most pediatric treatment centers, Nationwide Children’s Hospital is one of only a few dozen sites worldwide selected to…

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Epigenetics, Chromatin Architecture and a Judo Mechanism to Attack Cancer
 Epigenetics, Chromatin Architecture and a Judo Mechanism to Attack Cancer 1024 575 Abbie Miller
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yellow squiggly lines representing chromatin strands in the nucleus

Researchers broaden the understanding chromatin architecture in human disease. Epigenetics is the study of how genetic information is context-dependent: it is organized so it can be repressed, but also read, repaired and replicated. For example, transcription factors can “communicate” with each other through the chromatin-DNA interface, and work in combinations to regulate which genes are…

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Helping Children With Sickle Cell Disease Adhere to Their Medication
 Helping Children With Sickle Cell Disease Adhere to Their Medication 1024 575 Mary Bates, PhD
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Mobile intervention increases medication adherence in the children who engage with it. A study published in JMIR mHealth uHealth shows that an electronically delivered intervention can improve medication adherence in children with sickle cell disease. Hydroxyurea is a life-changing medication for children with sickle cell disease (SCD). However, many patients may not consistently receive their hydroxyurea. To…

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Understanding Drug Interactions in Diffuse Midline Gliomas
 Understanding Drug Interactions in Diffuse Midline Gliomas 1024 683 Lauren Dembeck
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In a recent publication in Science Translational Medicine, researchers studied combinations of approved and investigational drugs to identify promising pairs, such as panobinostat and marizomib, for the treatment of diffuse intrinsic pontine glioma (DIPG) and other diffuse midline gliomas (DMG). In recent years, studies have shown that diffuse intrinsic pontine glioma (DIPG) subtypes are driven…

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From Natural Killer Cells to Virus-Specific T Cells: What’s Next in Cellular Therapy
 From Natural Killer Cells to Virus-Specific T Cells: What’s Next in Cellular Therapy 969 533 Abbie Miller
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Illustration of NK Cells, T Cells, other immune cells floating across white background

Harnessing the power of the immune system to overcome cancer, improve outcomes of bone marrow transplants and fight viral infections in immunocompromised pediatric patients is at the heart of cell therapy research.   Natural Killers in Action Natural killer (NK) cells are a component of the innate immune system that mediates the death of cancer…

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Kidney Biopsy Analysis Reveals High Risk for Disease in Patients With Sickle Cell Disease
 Kidney Biopsy Analysis Reveals High Risk for Disease in Patients With Sickle Cell Disease 1024 732 Mary Bates, PhD
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Patients with SCD are at high risk for significant kidney disease. A retrospective study of biopsy findings from the Pediatric Nephrology Research Consortium shows just how high. Kidney damage is a progressive complication of sickle cell disease (SCD), sometimes eventually leading to kidney failure. As the life expectancy of individuals with SCD has improved, the…

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Targeting Alternative RNA Splicing in Pediatric Cancers
 Targeting Alternative RNA Splicing in Pediatric Cancers 150 150 Mary Bates, PhD
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Targeting alternative RNA splicing in tumor cells could lead to cancer therapies. RNA splicing, the enzymatic process of removing segments of premature RNA to produce mature RNA, is a key regulator of gene expression. Recent studies have identified variations in the transcriptomes of tumors due to alternative splicing changes, in addition to mutations in splicing…

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