From cancer drugs designed to match a child’s specific molecular abnormality, to a phone app that can offer a dermatologic diagnosis quicker than an office visit, the future of children’s healthcare is unfolding right before our eyes at the Children’s Hospital of Philadelphia Research Institute. Read on for more exciting headlines from this week, including highlights from our inaugural “Deciphering Beckwith-Wiedemann Syndrome” conference and a special congratulation to one CHOP psychologist.
Philadelphia Psychological Association Awards Katherine Dahlsgaard ‘Psychology in Media Award’
With digital media churning out news faster than ever, it’s important for today’s families to receive clear, balanced, and accurate information about the latest health research. Here at CHOP, Katherine Dahlsgaard, PhD, ABPP, clinical director of the Anxiety Behaviors Clinic, plays an active role in helping to ensure parents and children get exactly that.
As a regular writer for the Philadelphia Inquirer and a frequent guest on public television and radio, Dr. Dahlsgaard brings complex, evidence-based research to families, addressing a wealth of behavioral health subjects including ADHD in girls and the importance of sleep for adolescents. In recognition of her hard work, the Pennsylvania Psychological Association (PPA) recently honored Dr. Dahlsgaard with the 2017 Psychology in Media Award, given each year to a member of the medical community who effectively translates evidence-based science to the public and demonstrates the importance of research in psychological treatment.
“It’s wonderful to be recognized by my peers for the work that I do,” stated Dr. Dahlsgaard in a press release. “Mental health can be treated with such stigma, so it’s important to make sure we do all we can to educate people.”
You can read more about Dr. Dahlsgaard’s new award in the official PPA press release.
Enrollment Begins for Pediatric Match: A Precision Cancer Medicine Trial
Can cancer therapies targeted to specific molecular abnormalities help children whose solid tumors don’t respond to standard treatment? Researchers at CHOP and a network of nationwide institutions are one step closer to finding out.
Earlier this week, the National Cancer Institute (NCI) and Children’s Oncology Group (COG) announced open enrollment for their NCI-COG Pediatric Molecular Analysis for Therapy Choice (Pediatric MATCH) trials. Pediatric MATCH will investigate the effectiveness of more than eight study drugs contributed by pharmaceutical companies, with each drug targeted toward a specific genetic mutation. The study is enrolling children and adolescents from ages 1 to 21 who have solid tumors such as non-Hodgkin lymphomas, brain tumors, and histiocytoses that no longer respond to standard treatment.
In the NCI press release, Peter Adamson, MD, chair of the COG and oncologist at CHOP, made a statement: “Pediatric MATCH is a cutting-edge trial in many ways. It will bring molecular analysis, coupled to a portfolio of new targeted agents, to children and adolescents with relapsed cancer across the United States. Importantly, it will also help us learn more about relapsed cancer in pediatric patients, catalyzing research aimed at developing better treatments.”
We previously wrote about Pediatric MATCH, a joint collaboration between the NCI and the COG, in its developmental days on Cornerstone. You can learn more about the NCI’s enrollment announcement in their press release.
New Study Finds Mobile Health App for Dermatology Can Help Families
How can we reduce wait times for pediatric dermatology appointments while still giving families the information they need? There just may be an app for that.
In a new study published in Telemedicine journal and e-health, the official journal of the American Telemedicine Association, researchers from CHOP took a closer look at what families thought of a teladermatology app that would connect them with a pediatric dermatologist. Using the app, families can take photos of a child or adolescent’s skin condition, answer a few questions or provide a description, and then submit their case. A dermatologist would then review the case, diagnose the condition, and provide a prescription or treatment recommendation.
In the current study of 197 parents and one adolescent, the majority reported that they found the app usable, acceptable, and believed that it sped up the treatment process. The study was led by Alex Fiks, MD, pediatrician and holder of the Distinguished Chair in the department of Pediatrics at CHOP.
To learn more about the study, you can read the abstract online.
JAMA Ophthalmology Publishes New Findings on CHOP ROP Model
Premature babies born before 31 weeks have a high risk of retinopathy of prematurity (ROP): a disease of blood vessels in the eye that can lead to lifelong blindness. Currently, clinicians diagnose ROP by examining an infant’s birth weight and gestational age at birth in the neonatal intensive care unit. While effective, this method has a low specificity for identifying infants at risk for severe ROP, and it can also result in a high number of unnecessary examinations. (Previous research suggests that fewer than 10 percent of babies receiving exams actually require treatment.) On top of that, these exams can be stressful for babies as well as resource-intensive on the part of physicians.
In a new study published in JAMA Ophthalmology, CHOP researchers tested the effectiveness of the Children’s Hospital of Philadelphia Retinopathy of Prematurity model (CHOP ROP). CHOP ROP is designed to provide a more specific way of determining which infants require treatment using analysis that incorporates slow weight gain rate (as well as birth rate and gestational age) into ROP prediction.
An earlier study on the subject found that the model accurately predicted all infants who required treatment and also reduced the number of exams when compared with current approaches. In this new study of 7,483 premature infants, researchers found that the model was able to reduce the number of infants requiring exams by 34.4 percent (if only high-risk infants received exams) and also correctly predicted 452 out of 459 infants with type 1 ROP.
To learn more, you can find the study online.
Research Institute Hosts Deciphering Beckwith-Wiedemann Syndrome Conference
The Orphan Disease Center of the Perelman School of Medicine at the University of Pennsylvania is creating a new Program of Excellence to support Beckwith-Wiedemann syndrome (BWS) research within the CHOP and Penn communities. BWS is a rare overgrowth disorder involving genetic and epigenetic changes that occur approximately every one in 10,500 births.
“The Beckwith-Wiedemann Syndrome Program of Excellence brings together experts from across CHOP and Penn to improve the clinical care of BWS patients through translational and basic science research with an eye toward improved diagnostics and therapeutic development,” said Jennifer Kalish, MD, PhD, attending physician and geneticist at CHOP and an assistant professor of pediatrics at Penn, who has championed a team of specialists from the two institutions with unique expertise in BWS.
Several of those team members, including geneticists, endocrinologists, plastic surgeons, pediatric oncologists, dental specialists, researchers and others, gave talks at an inaugural Deciphering Beckwith-Wiedemann conference held July 21-23 at the Research Institute. Attendees learned about new tumor screening guidelines and diagnostic criteria for BWS. Just as important as hearing about the latest science, BWS families also had the valuable opportunity to make connections and friendships.
Read about the BWS conference, and learn more in Bench to Bedside and Cornerstone about a BWS Registry begun by CHOP three years ago that has become a vital resource to filling critical gaps in research data and clinical knowledge about BWS.
Recently on Cornerstone, we took a quick snapshot of research from the COG, as they develop a new standardized dosing method for anticancer drugs in infants. We also spent an exciting day in the life of Julie Uchitel, a student from the CHOP Research Institute Summer Scholars Program.
Catch up on our headlines from our July 13 edition of In the News:
- FDA Panel Votes “Yes” To Recommend CTL019 Approval
- Dr. Thomas Power Talks ADHD and Driving for Medscape
- JAMA Neurology Study Looks at Brain Health Years After High School Football
- Healthy Teen Years Make for Strong Bones
- Pediatric Nurses Thrive in Positive Work Environments
- CHOP Team Identifies New Genetic Syndrome
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