Latest News

• share
• favorite_border

Children’s Hospital of Philadelphia (CHOP) has helped launch a new computational platform that will harmonize pediatric cancer data, allowing researchers, pharmaceutical companies, and advocacy groups to accelerate the pace of drug development for pediatric cancer. With funding from the National Cancer Institute (NCI) via a subcontract with Leidos Biomedical Research, current operator of the NCI’s Frederick National Laboratory for Cancer Research, CHOP researchers have created the Molecular Targets Platform to facilitate pediatric research in response to the Research to Accelerate Cures and Equity (RACE) for Children Act, which requires companies to test cancer drugs in children that are used in adults when there is a shared molecular target.

• share
• favorite_border

Attending daycare in the first three years of life is linked with an increase in lung complications in children who were born prematurely and diagnosed with a form of chronic lung disease, according to new research led by Children’s Hospital of Philadelphia (CHOP).

• share
• favorite_border

Young adolescents who are targets of cyberbullying are more likely to report suicidal thoughts and attempts, an association that goes above and beyond the link between suicidality and traditional offline bullying.

• share
• favorite_border

Researchers found that while many child neurology visits can be effectively conducted through telemedicine, younger children and those with neuromuscular disorders are more likely to require in-person evaluation.

• share
• favorite_border

In an honor reserved for only the most significant achievements in advancing children’s health, Children’s Hospital of Philadelphia (CHOP) has awarded its Gold Medal to Katherine High, MD, a gene therapy pioneer and one of the lead developers of the first in vivo gene therapy approved by the Food and Drug Administration (FDA). Bestowed only 12 times in CHOP’s 167-year history, the Gold Medal highlights Dr. High’s groundbreaking discoveries at CHOP, which led to a gene therapy treatment for a rare form of inherited blindness and advanced gene therapy for hemophilia to late-stage testing.

• share
• favorite_border

Ten years ago, Tom and Kari Whitehead came to Children’s Hospital of Philadelphia (CHOP) looking for a miracle. Their 6-year-old daughter, Emily, had relapsed in her battle with acute lymphoblastic leukemia (ALL), after many months of unsuccessful chemotherapy and a disease that had progressed so rapidly that she was ineligible for a bone marrow transplant to treat it. Her family came to CHOP in the hopes that Dr. Stephan Grupp, a pioneer in the field of cellular immunotherapy, could provide the miracle they were looking for.

• share
• favorite_border

Researchers at Children’s Hospital of Philadelphia (CHOP) have developed a new tool to help researchers interpret the clinical significance of somatic mutations in cancer. The tool, known as CancerVar, incorporates machine learning frameworks to go beyond merely identifying somatic cancer mutations and interpret the potential significance of those mutations in terms of cancer diagnosis, prognosis, and targetability. A paper describing CancerVar was published today in Science Advances.

• share
• favorite_border

Neighborhood characteristics, including poverty and crowding within homes, were associated with higher rates of SARS-CoV-2 in pregnancy during the prevaccination era of the pandemic, according to a new study led by researchers at Children’s Hospital of Philadelphia (CHOP). The findings, which were published today in the journal Obstetrics and Gynecology, may partially explain the high rates of COVID-19, the disease caused by SARS-CoV-2, among Black and Hispanic patients.

• share
• favorite_border

In a new study, researchers demonstrated that the benefits of endurance exercise can vary based on the type of mutation involved in mitochondrial disease, and while the benefits of exercise outweigh the risks, the mitochondrial genetic status of patients should be taken into consideration when recommending exercise as therapy.

• share
• favorite_border

Researchers found that new drivers licensed before age 18 who are subject to mandatory driver education, including behind-the-wheel training and Graduated Driver Licensing (GDL) restrictions, were less likely to crash than drivers licensed at age 18 who are exempt from these requirements.

• share
• favorite_border

The combined effects of systemic and interpersonal racism layered on top of negative experiences within the COVID-19 pandemic were associated with depression and anxiety among Black people in the postpartum period, according to a new study by researchers in The Intergenerational Exposome Program (IGNITE) of Children’s Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania. The findings were published today in JAMA Psychiatry.

• share
• favorite_border

Researchers at Children’s Hospital of Philadelphia (CHOP) have shown that a targeted treatment they developed is effective at controlling blood sugar in patients with hyperinsulinism (HI), a genetic disease in which the pancreas produces too much insulin. The findings, which were published today in Diabetes Care, provide further evidence that the treatment could prevent hypoglycemia in patients with HI and may preclude the need for a full removal of their pancreas, a current standard treatment for severe diffuse HI.

• share
• favorite_border

An international team including researchers from the Lifespan Brain Institute (LiBI) of Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania have created a new tool that benchmarks brain development over the human lifespan, based on magnetic resonance imaging (MRI) data from more than 100,000 individuals. The work was jointly led by LiBI researchers and colleagues at the University of Cambridge. Described today in Nature, the interactive open resource, known as BrainChart, harmonizes brain images in a way that will allow researchers to measure brain development against reference charts like those used for evaluating children’s height and weight.

• share
• favorite_border

Researchers found that opioid prescriptions for children who underwent one of eight common outpatient surgeries declined over a period of five years. These findings suggest that clinicians are using more discretion when considering which pediatric patients require an opioid prescription after their procedures.

• share
• favorite_border

Researchers have found that dysfunction in an important cell subtype in the brain’s neuronal network contribute to chronic symptoms in the neurodevelopmental disorder Dravet syndrome.

• share
• favorite_border

Adding the proteasome inhibitor bortezomib to chemotherapy significantly improved overall survival in children and young adults with newly diagnosed T-cell lymphoblastic lymphoma (T-LL), according to a Children’s Oncology Group (COG) study led by researchers at Children’s Hospital of Philadelphia (CHOP). This international phase 3 clinical trial also found that radiation could be eliminated in 90% of children with T-cell acute lymphoblastic leukemia (T-ALL) when the chemotherapy regimen was intensified.

• share
• favorite_border

Researchers have found that developmental delays associated with Leigh syndrome, the most common pediatric mitochondrial disorder, may occur earlier than previously recognized – even before metabolic stroke and regression – which could provide clinicians with an opportunity for earlier diagnosis and therapeutic interventions.

• share
• favorite_border

Fusion oncogenes, such as RET- and NTRK-gene fusions, are associated with more invasive pediatric thyroid cancers, correlating with the highest risk of metastases and a lower likelihood of achieving remission one year after initial therapy, according to a new study by researchers at Children’s Hospital of Philadelphia (CHOP). The findings, which were published in the Journal of Clinical Oncology, contrast those previously established in adults, for whom BRAF mutations, not fusion oncogenes, are associated with more invasive disease that is less response to therapy.

• share
• favorite_border

Researchers have developed a new method of measuring motor imitation, adding to a growing set of computational behavior analysis tools that can detect and characterize motor differences in children with autism.

• share
• favorite_border

Researchers have developed a comprehensive sequencing test specifically for mitochondrial DNA (mtDNA). This new clinical diagnostic test provides important information as to whether and to what level variations in the mtDNA are present in different tissues from a patient with suspected mitochondrial disease, leading to more precise diagnosis and more personalized treatment options.

• share
• favorite_border

Infants born via uncomplicated cesarean delivery, without labor or membrane rupture before delivery and no concern for infection, should not need antibiotics at birth, according to a study by researchers at Children’s Hospital of Philadelphia (CHOP). The findings, which were published today in Pediatrics, could help clinicians tailor the use of early antibiotics in newborns. Only those deemed at risk for infection should receive antibiotics, thereby reducing unnecessary use.

• share
• favorite_border

Rising temperatures due to climate change will lead to an increase in cases of kidney stones over the next seven decades, even if measures are put in place to reduce greenhouse gas emissions, according to a new study by researchers at Children’s Hospital of Philadelphia (CHOP). Based on data from South Carolina, the study found the increase will be steeper if no action is taken, but an uptick will occur even with mitigation actions, costing the state healthcare system approximately $57 million in the latter scenario and $99 million if nothing is done. The findings were published today in Scientific Reports.

• share
• favorite_border

Aberrant splicing of messenger RNAs encoding surface antigen CD22 leads to downregulation of this protein in pediatric B-lymphoblastic leukemia (B-ALL), rendering malignant cells resistant to the effects of CD22-directed immunotherapies, according to a recent study by researchers at Children’s Hospital of Philadelphia (CHOP). The findings could allow oncologists to screen new patients to see if their leukemic cells contain alternatively spliced CD22 mRNA variants, which could reveal which patients might not respond to anti-CD22 therapies and would need alternative treatment plans. The study was published in Blood Cancer Discovery, a journal of the American Association for Cancer Research.

• share
• favorite_border

Being the parent or sibling of a child with a life-threatening condition can take a mental and physical toll on other members of the family. A new study is one of the first to empirically measure the extent of this burden on families, with parents and siblings 50% to 70% more likely than their peers to receive health care for mental and physical health issues, accompanied by medication for these issues, than families of children without a life-threatening condition.

• share
• favorite_border

A new study found that only 8% of pediatric healthcare and behavioral service providers feel prepared to assess whether their autistic patients are ready to drive. These findings suggest a critical need to develop resources that prepare providers and others who work with autistic youth to effectively facilitate independence and mobility as their patients become adults.

• share
• favorite_border

A new study has implicated several genes involved in a variety of bodily functions associated with the hypothalamus, a notoriously difficult-to-study region of the brain. The findings could help clinicians identify potential causes of dysfunction for many important traits regulated by the hypothalamus, such as sleep, stress, and reproduction.

• share
• favorite_border

Patients with a rare immunodeficiency disorder who are treated with lifelong immunoglobulin replacement therapy (IRT) have a lower risk of premature death than patients treated with a hematopoietic stem cell transplant (HSCT), but they also have a reduced quality of life and must assume a substantial financial burden, according to a new study led by Children’s Hospital of Philadelphia (CHOP). Using a computational model to calculate the costs and benefits of IRT and HSCT for patients with agammaglobulinemia, the researchers concluded that the high cost of IRT in the U.S. undermines its cost-utility, particularly when compared to Canada and European countries, where the cost of IRT is nearly a third of what it is in the U.S.

• share
• favorite_border

In a breakthrough for the treatment of aggressive solid cancers, researchers at Children’s Hospital of Philadelphia (CHOP) have developed a novel cancer therapy that targets proteins inside cancer cells that are essential for tumor growth and survival but have been historically impossible to reach. Using the power of large data sets and advanced computational approaches, the researchers were able to identify peptides that are presented on the surface of tumor cells and can be targeted with “peptide-centric” chimeric antigen receptors (PC-CARs), a new class of engineered T cells, stimulating an immune response that eradicates tumors.

• share
• favorite_border

The Center for Data Driven Discovery at CHOP will procure molecular characterization for thousands of these brain tumor samples providing an unprecedented level of insight into devastating cancers and paving the way for future therapeutic interventions.

• share
• favorite_border

Healthy full-term infants born during the COVID-19 pandemic had substantially shorter hospital stays than those born before the pandemic, with no change in hospital readmissions after discharge, according to a new study led by researchers at Children’s Hospital of Philadelphia (CHOP), in collaboration with colleagues at the Yale School of Medicine, Nemours duPont, and Epic Systems Corporation. The findings, published today in Pediatrics, suggest shorter hospital stays for healthy term infants might be safe outside of a pandemic scenario, with implications for health care costs and best practices.

• share
• favorite_border

A research team led by Children’s Hospital of Philadelphia (CHOP) has analyzed the birth volume and geographic distribution of obstetric hospitals across the United States and found that a plurality of those hospitals delivers fewer than 500 infants per year. The researchers also found that nearly a fifth of low-volume hospitals are more than 30 miles from another obstetric hospital, and more than half are in rural communities.

• share
• favorite_border

Stringent lockdown measures imposed in the spring of 2020 led to a dramatic drop in vaccinations among both children and adults, according to a new study led by Children’s Hospital of Philadelphia (CHOP).

• share
• favorite_border

Researchers have received a $5.3 million grant to conduct the Eliminating Monitor Overuse (EMO) clinical trial, seeking to discover how best to reduce the overuse of unnecessary monitoring strategies for infants who have a common lung infection called bronchiolitis.

• share
• favorite_border

Researchers at Children’s Hospital of Philadelphia (CHOP) have demonstrated that an experimental device can improve blood sugar control in patients who developed diabetes after their pancreas was removed to treat their hyperinsulinism, a genetic disease in which the pancreas produces too much insulin. Using a combination of continuous glucose monitoring, two hormone pumps, and an algorithm, the device, known as the bihormonal bionic pancreas (BHBP) and developed by researchers at Boston University, helped HI patients with diabetes maintain stable glucose levels over the study period.

• share
• favorite_border

Researchers successfully developed and validated a new outcome measure to monitor disease severity and progression in patients with impaired skeletal muscle function caused by mitochondrial disease. This measure, named ‘Mitochondrial Myopathy-Composite Assessment Tool’ (‘MM-COAST’), is important to identify specific skeletal muscle and neurological problems in both children and adults with mitochondrial disease and may be used to measure response to treatment interventions in future clinical trials.