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Hematology/Oncology/BMT

A New Paradigm for Treating Transcription Factor-Driven Cancers
 A New Paradigm for Treating Transcription Factor-Driven Cancers 150 150 Abbie Miller
By: 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
By: 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
By: Abbie Miller
patient sitting in a medical chair wearing an orange dragon shaped VR headset

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
By: 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
By: 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
By: 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
By: Abbie Miller
Image of a microscopic colorful image of a herpes virus.

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
By: 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
By: 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
By: 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: Abbie Miller
Illustration depicting cancer immunotherapy: a person rides atop a large pink, textured cancer cell while holding loops of rope wrapped around the cell, suggesting control or targeting of cancer. Additional cancer-like cells float in the background against blue radial light beams, symbolizing efforts to harness the immune system to fight cancer.

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
By: Gina Bericchia
virtual reality landscape

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
By: 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
By: 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
By: 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
By: 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
By: 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
By: 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
By: Abbie Miller
A close-up medical illustration of blood vessels showing normal round red blood cells flowing through the vessel, with a cluster of rigid, crescent-shaped sickle cells causing a blockage. White blood cells are also visible against the dark background.

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
By: 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
By: 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
By: 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
By: 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…

Preserving Biopreservation
 Preserving Biopreservation 1024 575 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES
"Large stainless steel cryogenic storage tanks arranged in a row inside a laboratory or biorepository facility. Each tank is labeled 'MEDICAL NITROGEN' near the top and fitted with metal handrails and wheeled bases for mobility.

Funding challenges, operational complexity and poor visibility threaten the field of human tissue biobanking. How sustainable are biorepositories? The Children’s Oncology Group (COG) boasts the collaboration of more than 9,000 pediatric cancer experts. They treat patients and research disease at more than 200 hospitals around the world. Nine out of every 10 U.S. children with cancer receive…

Out of Drugs: Planning for the Unthinkable
 Out of Drugs: Planning for the Unthinkable 150 150 Dave Ghose
By: Dave Ghose

Pediatric cancer specialists have faced an enormous challenge in recent years. With critical chemotherapy drugs often in short supply, they’ve been forced to improvise alternative treatments or purchase medicine from questionable secondary sources. These practices raise safety and ethical concerns. Emerging evidence indicates that shortages are harming patients in some instances, exposing them to more…

Diagnosis Focus
 Diagnosis Focus 150 150 Katie Brind'Amour, PhD, MS, CHES
By: Katie Brind'Amour, PhD, MS, CHES

Up to one in 10 blood clots in women occur in teen contraceptive users. When a teenage girl presents with chest pain, most doctors likely think first of anxiety, muscle injury or heartburn. In most cases, this is entirely appropriate. But if the girl is taking birth control pills, the problem could be a pulmonary…