Hematology/Oncology/BMT

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

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…

Understanding Drug Interactions in Diffuse Midline Gliomas
Understanding Drug Interactions in Diffuse Midline Gliomas 1024 683 Lauren Dembeck

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…

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
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…

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

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…

Targeting Alternative RNA Splicing in Pediatric Cancers
Targeting Alternative RNA Splicing in Pediatric Cancers 150 150 Mary Bates, PhD

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…

What Can We Learn From the Co-occurrence of a Genetic Disorder and Cancer?
What Can We Learn From the Co-occurrence of a Genetic Disorder and Cancer? 150 150 Mary Bates, PhD

Researchers investigate an unusual case of concurrent genetic and somatic diagnoses. In a new paper in the European Journal of Medical Genetics, researchers from Nationwide Children’s Hospital discuss the case of a 4-year-old with both a rare genetic disorder and medulloblastoma. Genetic analyses allowed them to evaluate the cause of the genetic disorder and provided support…

The Search to Identify Tumor Cells Evading Chemotherapy
The Search to Identify Tumor Cells Evading Chemotherapy 150 150 Sanjana Rajan

Graduate research associate Sanjana Rajan shares why her work to label and track cells before and after chemotherapy is the next step to preventing tumor relapse. For a long time, the cells within a tumor were thought to be similar to one another, like a bowl of chocolate chips. However, scientific studies have identified that…

Understanding the Role of LSD2 in the Molecular Genetics of Ewing Sarcoma
Understanding the Role of LSD2 in the Molecular Genetics of Ewing Sarcoma 150 150 Mary Bates, PhD

Study reveals novel potential drug target for Ewing sarcoma. Ewing sarcoma is a bone-associated tumor of mostly children and young adults. Despite aggressive multi-modal treatment strategies, five-year survival remains at 75% for patients with localized disease and 20% for patients with metastases. “Our laboratory is organized to try to understand how Ewing sarcoma develops at a…

Use of Cardiac MRI to Monitor Medical Treatment of Rhabdomyoma
Use of Cardiac MRI to Monitor Medical Treatment of Rhabdomyoma 150 150 Mary Bates, PhD

Case study shows success for medical therapy and MRI monitoring of a cardiac rhabdomyoma. In a new case study, researchers from Nationwide Children’s Hospital report treating cardiac rhabdomyoma in a newborn with an mTOR inhibitor called everolimus. They monitored the tumor’s regression using cardiac MRI. Cardiac rhabdomyoma is a common type of heart tumor in…

Looking to the Future: Optimizing Fertility Preservation Decisions in Pediatric Patients Newly Diagnosed with Cancer
Looking to the Future: Optimizing Fertility Preservation Decisions in Pediatric Patients Newly Diagnosed with Cancer 1024 575 Katie Brind'Amour, PhD, MS, CHES
Parent and child holding hands

Family-centered research aims to improve fertility preservation uptake and decision satisfaction among adolescents newly diagnosed with cancer.

An Infant. A Virus. An Emergency IND. A Life Saved.
An Infant. A Virus. An Emergency IND. A Life Saved. 150 150 Abbie Miller

Clinician scientists collaborate to use virus-specific T-cells from the mother to successfully treat a systemic adenovirus infection in a preterm infant. It’s not every day that researchers can say that they’ve written and submitted and emergency investigational new drug (EIND) application to the Food & Drug Administration (FDA) and saved a life. But that’s what…

Profile of a Cancer: Getting to Know Ewing Sarcoma
Profile of a Cancer: Getting to Know Ewing Sarcoma 150 150 Katie Brind'Amour, PhD, MS, CHES

Ewing sarcoma – a tumor type affecting the bone or soft tissue that primarily affects children and adolescents – has a 5-year survival rate of 70 percent among those with localized disease at diagnosis. Among children whose disease is metastatic, only 30 percent survive 5 years or longer. As a comparison, of all children diagnosed…

Generating the Genome: How Scientists Changed the Face of Cancer Research
Generating the Genome: How Scientists Changed the Face of Cancer Research 1024 683 Katie Brind'Amour, PhD, MS, CHES
Color photo; environmental portrait ofDrs. Elaine Mardis and Richard Wilson

Team science. Ongoing innovation. Brilliant minds. Here’s how The Cancer Genome Atlas spawned a revolution in cancer research and technology. The Cancer Genome Atlas is wrapping up. Its data now lives online in the Genomic Data Commons, freely available to the public. Reports of the primary findings for each studied tumor type have been published, and…

More Screening Needed for Young Females at Risk for von Willebrand Disease
More Screening Needed for Young Females at Risk for von Willebrand Disease 1024 575 Mary Bates, PhD
Black and white image of teen girl posing outside

A new study finds most girls with heavy menstrual bleeding are not screened for the bleeding disorder. Despite recommendations by the American College of Obstetricians and Gynecologists, approximately 90 percent of girls with heavy menstrual bleeding are not being screened for von Willebrand disease, according to a new study from Nationwide Children’s Hospital. “Von Willebrand disease is…

Is Caregiver Education About Sickle Cell Trait Effective?
Is Caregiver Education About Sickle Cell Trait Effective? 1024 575 Abbie Miller
Color photo of Black father holding infant on shoulder in front of nursery background with clouds on the wall

Researchers evaluate the standardized education in Ohio to determine if it achieves high caregiver knowledge. Despite universal newborn screening that detects the presence of sickle cell trait (SCT), only 16 percent of Americans with SCT know their status. To address this issue, in Ohio, in-person education is offered to caregivers of referred infants with SCT.…

How Can We Optimize Fertility Related Discussions With Male Survivors of Childhood Cancer?
How Can We Optimize Fertility Related Discussions With Male Survivors of Childhood Cancer? 150 150 Abbie Miller

A recent study suggests that fertility is a “later” issue for adolescents and young adults, rather than a “now” concern, leading to low rates of conversation with health care providers. Survival rates for pediatric cancers exceed 80 percent. But late effects can persist throughout the survivor’s lifetime. Among those late effects, infertility may have both…

Starting the Conversation on Sickle Cell Disease and Reproductive Health
Starting the Conversation on Sickle Cell Disease and Reproductive Health 1024 575 Katie Brind'Amour, PhD, MS, CHES

Adolescents and young adults with sickle cell disease — and their caregivers — care about future fertility. But what should doctors tell them? Until just a few decades ago, sickle cell disease (SCD) was often fatal in childhood. Now that more patients reach adulthood, clinicians and researchers need to ask — and answer — questions…

How to Identify and Treat Blood Clots in Pediatric Patients With Cancer
How to Identify and Treat Blood Clots in Pediatric Patients With Cancer 1024 575 Mary Bates, PhD
Black and white photo of a young patient with cancer wearing a mask and gown and holding an IV poll smiling and giving a thumbs up in the hospital

New guidelines will help doctors recognize and treat children at risk of venous thromboembolism. Prevention of blood clots is well described in adult cancer patients, but data on the problem in pediatric cancer patients is limited. In a new publication initiated at the request of the International Society of Thrombosis and Hemostasis, researchers provide guidance for…

What Happens When Cancer Remission Is in Question?
What Happens When Cancer Remission Is in Question? 1024 575 Mary Bates, PhD
Photo of leukemia cells under microscope

A new study looks at outcomes for children with leukemia when two tests of remission disagree. In both clinical practice and clinical trials, remission in leukemia is determined by morphological assessment — a person counting cancer cells in a bone marrow sample under a microscope. But more sophisticated technologies, such as flow cytometry or polymerase…

Large-Scale Genomics Study Identifies Children With High-Risk Cancer
Large-Scale Genomics Study Identifies Children With High-Risk Cancer 150 150 Mary Bates, PhD

Some subtypes of leukemia have a poor prognosis. Genomic studies are helping to identify these subtypes, leading to targeted therapies. Acute lymphoblastic leukemia (ALL), a malignancy of the white blood cells, is a common childhood cancer. Understanding the genomic changes underlying ALL has led to the development of new, targeted therapies, especially for patients with…

Conquering the Biological Politics of Cancer: Corruption, Coercion and Collusion
Conquering the Biological Politics of Cancer: Corruption, Coercion and Collusion 150 150 Timothy Cripe, MD, PhD

Understanding the “Three C’s” may provide the insights need to move the needle on the cancers with the bleakest prognoses. Broadly speaking, cancers fall into three categories: leukemias, brain tumors and other solid tumors. Since the dawn of chemotherapy in the 1940s, we’ve converted the most common type of leukemia, acute lymphoblastic leukemia, from incurable…

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

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…

Making Stem Cell Transplantation Work for Sickle Cell Disease
Making Stem Cell Transplantation Work for Sickle Cell Disease 150 150 Mary Bates, PhD

New regimen offers a promising approach for unrelated donor transplants. Sickle cell disease (SCD) is a blood disorder affecting 1 in every 500 African-American newborns. It is characterized by extreme pain and decreased longevity. Currently, the only cure for SCD lies in hematopoietic stem cell transplantation. A well-matched sibling donor is needed for the best…

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

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…

The Evolution of Precision Medicine for Children With Aggressive Brain Tumors
The Evolution of Precision Medicine for Children With Aggressive Brain Tumors 150 150 Jonathan Finlay, MB, ChB, FRCP

The Head Start 4 clinical trial introduces risk stratification and builds on the success of bone marrow transplantation for young children with malignant embryonal tumors. Cancers in the brain and spinal cord (the central nervous system, CNS) are now the most common cause of deaths due to disease in children and adolescents. Among these tumors,…

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

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…

“Impatient” Therapy: Physicians Too Aggressive in Treatment of ITP
“Impatient” Therapy: Physicians Too Aggressive in Treatment of ITP 150 150 Brianne Moore

Despite guidelines that advocate watching and waiting, physicians are still treating most patients with immune thrombocytopenia. When a child presents with easy bruising or bleeding, red skin spots and fatigue – symptoms similar to those for leukemia – families seek evaluation immediately. However, more often these cases are pediatric immune thrombocytopenia (ITP), an acquired disorder…

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 Miller

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…

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 Miller

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…

Combining Immunotherapies Effective Against Mouse Model of Cancer
Combining Immunotherapies Effective Against Mouse Model of Cancer 150 150 Mary Bates, PhD

Recent study results suggest that combining virotherapy and PD-1 blockades may be more effective than either approach alone. Rhabdomyosarcoma, a cancer made up of cells that normally develop into skeletal muscles, is the most common soft tissue cancer in children. If it is detected early and localized in certain areas, rhabdomyosarcoma is usually curable with…

Assessing Romantic Lives of Adult Survivors of Childhood Cancer
Assessing Romantic Lives of Adult Survivors of Childhood Cancer 150 150 Kevin Mayhood

In some ways, the group appears more resilient than survivors of adult-onset cancer or those with other childhood-onset diseases. Adult survivors of childhood cancer appear, on the whole, to have comparable romantic lives to others their age, new studies show. Surprisingly, the group doesn’t seem to carry the same burdens as men and women with…

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 Miller

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…

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 Miller

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…

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

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…

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 Miller

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…

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 Miller

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…

Changing the Game: Virtual Reality Distracts From Pain, Transforming the Patient Experience
Changing the Game: Virtual Reality Distracts From Pain, Transforming the Patient Experience 844 487 Gina Bericchia

A first-of-its-kind virtual reality experience from the hemophilia team and design experts at Nationwide Children’s Hospital and The Ohio State University distracts patients with an immersive environment of penguins, pirates and hermit crabs during infusions and other procedures. A pilot study is testing the feasibility of integrating the virtual reality technology into the clinic setting.…

Finding a Better Way to Diagnose and Treat Iron Deficiency in Young Women
Finding a Better Way to Diagnose and Treat Iron Deficiency in Young Women 150 150 Brianne Moore

Iron deficiency without anemia often goes undiagnosed in young women, and when caught, the standard treatment is often associated with poor compliance due to side effects. Dr. Sarah O’Brien’s research is focused on finding a solution. Even in developed countries, iron deficiency continues to be a prevalent nutritional disorder. It is common in women, especially…

Chromatin’s Role in Cancer Development
Chromatin’s Role in Cancer Development 150 150 Tiasha Letostak, PhD

A new study investigates the role of chromatin containing the histone H3 variant in the centromere region of chromosomes in the development of cancer. Although there are many types of cancer, all cancers have one thing in common: they develop when normal processes in the body break down. Unlike normal cells, cancer cells ignore signals…

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 Miller

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.…

Counseling Pediatric Patients on Fertility and Sexual Function
Counseling Pediatric Patients on Fertility and Sexual Function 150 150 Brianne Moore

How should we talk to patients and their families about these sensitive subjects? Since 2006, several medical societies have released guidelines for discussing fertility preservation prior to pediatric cancer treatment. According to the American Society for Reproductive Medicine, a “collaborative, multidisciplinary team approach” is vital for informing minors whose chemotherapy may affect their fertility status…

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

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…

Study Links Acute Infections With Later Functional Gastrointestinal Disorders
Study Links Acute Infections With Later Functional Gastrointestinal Disorders 150 150 Jeb Phillips

Link between acute infection in preschoolers and development of FGIDs in school-aged children could point toward interventions. Functional gastrointestinal disorders (FGIDs) are among the most common pediatric health conditions and often first appear in school-aged children. Acute diarrhea, also very common, often first manifests in preschoolers. A recently published prospective study shows that there may…

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

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…

Challenges of Adolescent and Young Adult Cancer
Challenges of Adolescent and Young Adult Cancer 150 150 Anthony Audino, MD

Among the unique challenges of AYA cancer, anxiety often increases after treatments are completed as the patient reintegrates back into “normal life.” The definition of the adolescent and young adult (AYA) cancer patient has evolved over time, but it is now formally defined as those diagnosed with cancer at ages 15 through 39. AYA patients are a…

What Do DALYs Mean for Pediatrics?
What Do DALYs Mean for Pediatrics? 150 150 Dave Ghose

An emerging, innovative metric could radically change childhood health policy. A new way of looking at illness is beginning to change how public health officials view life and death. The concept — called DALYs — offers a fuller view of disease that could have a big impact on pediatrics if embraced. Unlike death tolls —…

Adolescents, Young Adults and Cancer: What Are the Issues?
Adolescents, Young Adults and Cancer: What Are the Issues? 150 150 Abbie Miller

Adolescents and young adults with cancer have unique needs that may explain their plateau in survival rates, despite improved survival rates in other age groups. At an age when achieving independence and experiencing life milestones are most important, adolescents and young adults facing a cancer diagnosis must meet unique challenges. These challenges may contribute to…

A Watershed Moment for Cancer Virotherapy
A Watershed Moment for Cancer Virotherapy 150 150 Timothy Cripe, MD, PhD

A recent decision may define the future of virotherapy’s role in the clinical treatment of cancer. At 4:50 pm on Wednesday, April 29 2015, the votes were cast: 22 in favor, 1 against. With this overwhelming majority, an advisory committee sent a clear message to the FDA recommending the first marketing approval for a live virus cancer…