The Garbose Family Special Delivery Unit, the world’s first obstetrical care unit for healthy mothers carrying fetuses with known birth defects, marked its 10th anniversary this year. Children’s Hospital of Philadelphia celebrated the milestone with a special ceremony honoring the unit, which is the delivery arm of CHOP’s internationally recognized Center for Fetal Diagnosis and Treatment (CFDT).

Launched in 2008, the 13-bed unit fulfills an important need in maternal-fetal medicine by allowing mother and baby to be simultaneously cared for in one medical center by a highly specialized, multidisciplinary team. Over the past decade, it has seen more than 3,600 deliveries — an average of 450 to 500 deliveries a year — for patients from all 50 states and dozens of countries.

Lynne and Bill Garbose made the unit possible with their lead philanthropic gift, motivated by the loss of their own newborn infant 30 years ago due to a fatal heart defect. Their loss came at a time when parents were unable to deliver their critically ill babies at a pediatric hospital anywhere in the United States.

Today, the unit houses two operating rooms for cesarean deliveries and fetal surgery. Its multidisciplinary team includes experts in maternal-fetal medicine, reproductive genetics, obstetrics, surgery, anesthesiology, neonatology, fetal cardiology, genetic counseling, midwifery, fetal imaging, social work, palliative care, lactation consulting, and respiratory therapy. In addition, fetal therapy coordinators guide patients through their experience.

What will the next 10 years bring? The Garboses decided to fund a new Garbose Family Endowed Research Fellowship in Pediatric and Fetal Surgery in a specialized laboratory program within the CFDT. The fellowship will support fetal researchers working to generate new breakthroughs and establish a pipeline of physician-researchers to help lead the next generation of fetal medicine experts.

The Food and Drug Administration approved the first disease-modifying drug for children and adolescents with relapsing forms of multiple sclerosis (MS). Brenda Banwell, MD, Chief of the Division of Neurology at Children's Hospital of Philadelphia, served as co-principal investigator of the study that supported the pediatric approval.

The drug, fingolimod, has been proven to significantly reduce relapses and brain lesions in children as young as 10 years old. It was previously indicated only for adult patients 18 or older to help control MS relapses, brain lesions, and disability progression. The expanded approval is expected to bring new hope for younger MS patients.

“Repeated relapses are more common in young people with MS than in adults,” Dr. Banwell noted. “This is heartening news for patients and their families.”

The FDA named fingolimod, which is marketed in the United States and Europe by Novartis, a breakthrough therapy in late 2017 for children with relapsing MS. Early study results found that fingolimod could reduce the frequency of MS flare-ups in pediatric patients by nearly 82 percent compared to other drug treatments. Later study findings indicated fingolimod reduced pediatric patients’ relapse rate and development of new brain lesions, and it slowed disability progression.

The World Allergy Organization (WAO) named the Division of Allergy and Immunology at Children’s Hospital of Philadelphia a Center of Excellence. The mission of the WAO’s Centers of Excellence is to intensify and accelerate multidisciplinary scientific and clinical innovation, education, and advocacy worldwide in allergy, asthma, and clinical immunology. The WAO also designated the Section of Allergy and Immunology at the Hospital of the University of Pennsylvania a Center of Excellence.

“We are honored to be named as a WAO Center of Excellence,” said Jonathan Spergel, MD, PhD, Section Chief of Allergy in the Division of Allergy and Immunology at CHOP. “This distinction recognizes not only the groundbreaking research being done at CHOP and Penn, but also the outstanding clinical care we deliver.”

CHOP’s Division of Allergy and Immunology is one of the largest pediatric allergy groups in the region and cares for patients with all aspects of allergic and immunologic issues. Its faculty has helped formulate many of the national guidelines used by physicians across the country and around the world. The division treats patients with difficult-to-control allergic disorders, including asthma and anaphylaxis. It also provides evaluation and drug desensitization for patients who have a history of anaphylactic reactions to necessary, life-saving medications.

Ongoing research efforts in collaboration with colleagues at CHOP and other institutions around the world are focused on eosinophilic esophagitis, allergen-specific immunoglobulin E (IgE)-mediated food allergies, food desensitization, asthma, and atopic dermatitis.

PolicyLab celebrated 10 years as a Center of Emphasis at Children’s Hospital of Philadelphia Research Institute. To plan for the next 10 years, PolicyLab announced it is refining its focus and reorganizing its resources to benefit families, colleagues, and policymakers.

Founded in 2008, PolicyLab researches, develops and implements evidence-based solutions in response to community needs and child health policy priorities. Researchers collaborate with communications and policy professionals to translate their work for program and policy audiences.

PolicyLab faculty and staff identified four areas, or portfolios, of child and family health to target in the next decade: Healthcare Coverage, Access and Quality; Intergenerational Family Services; Health Equity; and Adolescent Health and Well-being. Comprehensive goals have been developed by PolicyLab for each of the four areas.

In the Healthcare Coverage, Access and Quality portfolio, interdisciplinary teams will focus on aligning health insurance coverage, clinical services, and community service organizations with children’s unique needs to improve children’s health and well-being.

The Intergenerational Family Services portfolio will address the increasing barriers that pediatric patients’ parents and caregivers face in maintaining their own health and well-being, which is essential to keeping families healthy, by building support for sustainable family-centered programs in pediatric settings.

In the Health Equity portfolio, researchers will evaluate programs and policies to ensure that they do not inadvertently create avoidable or remediable differences in outcomes for marginalized children and adolescents. Challenges faced by racial/ethnic minorities, immigrants and refugees, LGBTQ youth, youth with complex medical needs, and those in the child welfare and juvenile justice systems, will be addressed.

The Adolescent Health and Well-being portfolio will focus on helping parents understand the challenges that adolescents with unique needs face in transitioning to healthy, productive adults. PolicyLab also aims to influence programs and policies addressing teens’ reproductive and sexual health, mental health, and special healthcare needs.

As a spectrum disorder, autism spectrum disorder (ASD) can look very different from one person to the next, with hundreds of possible genetic mutations that seem to play a role in how ASD is expressed in each individual. Gathering data and funding studies of the size and scope necessary to account for autism’s myriad expressions has been out of reach — until now. Established in October 2008, the Center for Autism Research (CAR) is uniquely poised to lead autism research into a new era.

Over the last decade, CAR has sought to understand the causes of ASD in order to develop effective treatments through research; to serve the needs of individuals with ASD and their families through education and guidance throughout the lifespan; and to train the next generation of master clinicians and scientists in state-of-the-science best practices for autism screening, diagnosis, and treatment.

“CHOP offers a unique set of circumstances that makes it possible to surmount these persistent challenges in autism research,” said Robert T. Schultz, PhD, CAR’s scientific director. “CHOP’s vast patient base of 10,000 patients per year with ASD combined with unparalleled clinical and research expertise in multiple specialties affords us an extremely rare opportunity to conduct research that is rigorous, reproducible, and ultimately translatable: to accelerate the pace at which we’re able to improve care and long-term outcomes for patients with ASD within the CHOP Care Network and throughout the country.”

A collaborative effort between Children’s Hospital of Philadelphia and the University of Pennsylvania, CAR and its researchers have achieved numerous milestones over the past 10 years.

Time magazine named the discovery of autism gene variants by Hakon Hakonarson, MD, PhD, director of the Center for Applied Genomics at CHOP, a top 10 medical breakthrough for 2009. “[To that point], researchers knew little about the genetic component of ASD; and what they did know was related to syndromic, rare forms of ASD seen in other conditions, such as Fragile X syndrome, 22q11.2 Deletion and Duplication Syndromes, and Prader-Willi syndrome,” Dr. Hakonarson stated.

The following year, Timothy Roberts, PhD, vice-chair of research for the Department of Radiology at CHOP, discovered that children with ASD process sounds a fraction of a second slower than typically developing peers, leading to cascading delays.

The 2011 Infant Brain Imaging Study found the brains of infants who develop ASD follow different developmental trajectory from 6 months of age, affecting brain connections later in life. In 2017, a significant follow-up finding revealed differences in brain activity patterns at 6 months old can predict which children will develop autism at age 2.

A Center of Emphasis at CHOP, CAR established the Technology and Innovation Lab in 2015, which focuses on developing new technologies to revolutionize autism diagnosis and treatment. Two years later, the lab created a tool to help diagnose autism based on a three-minute video of social conversation.

In 2016, John Herrington, PhD, assistant professor in the Department of Child Psychiatry and Behavioral Science at the Perelman School of Medicine, found the amygdala to be smaller and more active in children who have ASD as well as an anxiety disorder. These findings suggest that symptoms of anxiety aren’t merely a facet of the core ASD symptoms but suggest anxiety can co-occur as a separate diagnosis in children with ASD.

And this year, CAR undertook the first longitudinal examination of the effectiveness of universal screening of autism using the Modified Checklist for Autism in Toddlers (M-CHAT). Looking to the future, CAR is building an integrated clinical/research program known as the “Autism Learning Health System” that is a testing ground for new treatments and models of care, with the support of patients and families, research collaborators, and partners like the Philadelphia Eagles.

Learn more about this groundbreaking development in CAR’s 10^th Anniversary Report.

Celebrating its 10-year anniversary, the Center for Pediatric Clinical Effectiveness (CPCE) continues to discover and share knowledge about best practices in pediatric care. The Center of Emphasis at Children’s Hospital of Philadelphia Research Institute achieves this goal by facilitating, organizing, and centralizing the performance of clinical effectiveness research. Put simply, it looks for “what works?” in healthcare.

Among its many highlights from the past decade, CPCE promotes antibiotic stewardship by providing achievable benchmarks and high-impact targets for improving antibiotic prescribing, then developing and implementing effective interventions. For example, CPCE’s Promoting Antibiotic Stewardship in Pediatric Outpatient Settings summarizes seven years of research to improve the effectiveness and acceptability of outpatient antimicrobial stewardship. The research brief notes that antimicrobial stewardship programs can improve the patient experience, reduce costs for payers, and lead to healthier populations through reduction of antibiotic overuse and resistance.

The puzzling growing incidence of pediatric kidney stones has become another area of emphasis for CPCE. With uncertainty of how to best evaluate children with suspected kidney stones and effectively decrease recurrence for patients whose first stone developed during childhood, CPCE seeks to address these knowledge gaps. Research includes studies on the role of temperature and antibiotics in kidney stone risk and mobile device hydration reminders for adolescents.

CPCE also strives to close the knowledge gap in pediatric HIV prevention and treatment research, as data for best practices center on adult cases. In particular, researchers are addressing challenges with treatment adherence unique among adolescents with HIV, as well as increasing the information needed to make pediatric formulations of newer HIV medications available and to understand which medicines work best in this population.

Understanding the pros and cons of physiologic monitoring — in both clinical settings and at home — has drawn CPCE’s attention as well. When healthcare workers experience alarm fatigue, they become overwhelmed, distracted by, or desensitized to the numbers of alarms that monitors activate, which can lead to delayed response times or missed alarms. A CHOP-led team used a video-based approach to gather data on staff response times and false alarm rates. In another study, the same CHOP researcher found at-home infant physiological monitors may cause undue alarm to parents, with no evidence of medical benefits, especially to healthy babies.