New Treatment Option for Ph+ ALL, Video Game for Autism and Co-occurring ADHD, Science of Pediatric Palliative Care, Frontier Programs Named

In this edition of In the News, learn about digital medicine and what it means for children with autism spectrum disorder and co-occurring attention deficit/hyperactivity disorder (ADHD). Read about a variation in care study, a recent FDA approved drug treatment for a specific childhood cancer, and why two Children’s Hospital of Philadelphia researchers are calling for improved pediatric palliative care measures. And lastly, join us in celebrating the addition of six new initiatives to CHOP’s Frontier Programs.  

FDA Approves Dasatinib Tablets in Combination With Chemotherapy

Pediatric patients newly diagnosed with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) will have a new treatment available to them: Dasatinib tablets in combination with chemotherapy. Data gathered from the Phase 2 CA180-372 study by lead author Stephen Hunger, MD, chief of the Division of Oncology and director of the Center for Childhood Cancer Research at Children’s Hospital of Philadelphia led to the approval. In the study, dasatinib plus chemotherapy showed that dasatinib is an effective medication for physicians to consider for children and adolescents with Ph+ ALL.

“As treatments have advanced in recent years, we’ve seen improvements in outcomes for pediatric patients with Ph+ ALL overall, but there remains a need for additional options,” Dr. Hunger said. “The Phase 2 trial was particularly informative because it was designed to limit the use of cranial irradiation and stem cell transplant.”

ALL — the most common childhood cancer in the U.S. — represents 20 percent of all cancers diagnosed in persons under 20-years-old, or more than 3,000 new cases each year. Three percent of children who have ALL have the Ph+ subtype.

Learn more in the press release.

Study Team Evaluates Video Game as Investigational Therapy for Autistic Children

“Please play your video game,” said parents — never. But now that phrase may become an everyday utterance among parents and caregivers of children with autism spectrum disorder (ASD) and co-occurring ADHD.

A new digital treatment called Project: EVO — an action video game experience which requires the player to steer a hovercraft and perform other tasks — was developed by Akili Interactive, a prescription digital medicine company. Using this multi-tasking game as treatment may help these children improve cognitive control, according to a study conducted by CHOP researchers with results published recently in the Journal of Autism and Developmental Disorders.

As many as 50 percent of children with autism spectrum disorder have ADHD symptoms, but ADHD medications are less effective in children with both disorders. Also, children with autism and ADHD are at high risk for impaired cognitive function, making it difficult to stay focused and on task while ignoring distractions.

“Our study showed that children engaged with the Project: EVO treatment for the recommended amount of time, and that parents and children reported high rates of satisfaction with the treatment,” said Benjamin Yerys, PhD, psychologist in the Department of Child and Adolescent Psychiatry and Behavioral Sciences, faculty researcher at CHOP’s Center for Autism Research, and lead author of the study. “Based on the promising study results, we look forward to continuing to evaluate the potential for Project: EVO as a new treatment option for children with ASD and ADHD.”

Learn more in the CHOP press release.

CHOP Researchers Call for Improved Pediatric Palliative Care Measures

Outcome measurements for children with palliative care are not one dimensional, flat, or linear, just as stick figures are not true representation of living human beings, say the authors of commentary published this month in the journal Pediatrics.

“When the subjective aspects of the illness experience, such as symptom distress or quality of life, are incorporated into the illness model, the dimensionality of the model rises substantially … the simple model of illness is not an accurate rendition of the real multidimensional disorder,” opined CHOP doctors Allison Barz Leahy, MD, a fellow with the Cancer Immunotherapy Program and Chris Feudtner, MD, PhD, MPH, director of research for the Pediatric Advanced Care Team.

So how can researchers develop good outcome measures to advance the science of palliative care while remaining mindful of the “simplifications that separate science from clinical care?” Three measurements that take into account basic psychometrics are needed for a more comprehensive illness model: patient/parent preferences; perceptions, particularly patients’ self-perceptions; and time, according to Drs. Leahy and Feudtner.

“Let’s agree to forge ahead and improve pediatric palliative care measures to advance our scientifically based therapeutic knowledge base but at the same time stay respectfully aware of the current separation between these measures and the provision of high-quality care,” the authors summarized.

To learn more, read the full article in Pediatrics.

Study of Variation in Care Published in ‘Inflammatory Bowel Diseases’

It’s true that in medical practice, variation in care is common. It’s also true that reducing variation in care is shown to improve not only quality but also increase favorable outcomes in chronic diseases. In an article published this month in the journal Inflammatory Bowel Diseases, a study team took a close look at the variation in care in the management of children with Crohn’s disease (CD).

The researchers collected data from a 28-site multicenter inception CD cohort and analyzed for variations in diagnostic modalities, treatment, follow-up monitoring practices, and complicated disease outcomes over three years in 1,046 children. They determined no variation existed in the initial diagnostic approach among the sites. When medication exposure was analyzed, variations occurred within the first 90 days but were not significant over a three-year period. The same results occurred for anti-tumor necrosis factor exposure. Use of immunomodulators varied among centers both early on and over the course of the three-year study. As for follow-up care (small bowel imaging, colonoscopy surveillance), the researchers noted a significant variation at the geographic level after initial therapy.

The authors, including CHOP’s Robert Baldassano, MD, pediatric gastroenterologist and director of the Center for Pediatric Inflammatory Bowel Disease, concluded that due to “the widespread existence of variation in care and disease monitoring at geographic levels among pediatric CD patients, future implementation of various practice strategies may help reduce the variation of care.”

CHOP Adds Six New Frontier Programs

A trailblazing group of initiatives known as Frontier Programs at CHOP are pioneering new advances in children’s health at an astonishing pace. Within these programs visionary research is conducted and translated to cutting-edge clinical care. They offer answers often not available anywhere else in the world: a breakthrough therapy that teaches a child’s own cells to fight cancer, a first-of-its-kind surgery for chest deformities, a lifesaving treatment based on a new understanding of the body’s lymphatic system, and more. CHOP champions these programs and ensures that they receive critical support to accelerate their progress. Here’s a peek at six new Frontier Programs designated in 2019:

• Advancement of Hyperinsulinism Care and Research — Primary investigators Diva DeLeón-Crutchlow, MD, and N. Scott Adzick, MD, will be working to develop personalized medicine for patients with hyperinsulinism, a condition in which insulin regulation fails, resulting in dangerously low blood sugar levels that can damage the brain.
• Biomedical Optical Devices to Monitor Cerebral Health — With Daniel Licht, MD; Todd Kilbaugh, MD; and Wesley Baker, PhD, at the helm, this frontier program seeks to develop a first-of-its-kind noninvasive device that monitors brain oxygen levels.
• Complex Vascular Anomalies Program — Primary investigators Jean Belasco, MD, and Hakon Hakonarson, MD, PhD, will be leading this program as it seeks to build a national leading center to care for complex vascular anomalies.
• Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (CuRED): Top Docs Stefano Rivella, PhD; Janet Kwiatkowski, MD; Kim Smith-Whitley, MD; and Helge Hartung, MD, will be building on CHOP’s position as a global leader in cell and gene therapies. The program will develop and deliver novel gene therapies and stem cell transplants to patients with red blood cell disorders in a family-centered clinic.
• Congenital Diaphragmatic Hernia (CDH): Lead investigators Holly Hedrick, MD, and Emily Partridge, MD, PhD, will be leveraging CHOP’s standing as the largest CDH treatment center in the country. This program aims to become the international hub for the care of CDH and to eliminate morbidity and mortality through new research and the development of novel devices.
• Epilepsy NeuroGenetics Initiative (ENGIN): under the leadership of Ethan Goldberg, MD, PhD; Ingo Helbig, MD; Dennis Dlugos, MD; Eric Marsh, MD; Benjamin Kennedy, MD; and Sudha Kessler, MD, this program seeks to diagnose, treat, and cure epilepsy by expanding genetic testing to all children with epilepsy. Ultimately, the program aims to optimize medical and surgical care and drive development of precision therapies.

Discovery and innovation have been at the core of CHOP’s mission for more than 150 years. Today, our Frontier Programs bring the best minds to the most challenging conditions, once again redefining what’s possible.

ICYMI

Catch up on our headlines from our Dec. 28 In the News:

• CHOP Co-authored Study on Child Mortality Tops Most-Read 
• Investigators Find Relationship Between Crk Adaptor Proteins and the Integrin LFA-1
• Yael Mossé, MD, Named Patricia Brophy Endowed Chair in Pediatric Neuroblastoma Research
• CHOP and City of Philadelphia Team Up for ‘Healthier Together’
• Violence Intervention Study Examines Needs of Patients Following Assault 

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