Neurology & Neurosurgery

Attendees at Sidra Medicine's 4th annual Pediatric Neuroscience Conference (PNC 2024)

Insights and Innovations in Pediatric Neuroscience: Highlights from PNC 2024

Attendees at Sidra Medicine's 4th annual Pediatric Neuroscience Conference (PNC 2024)
Children’s National Hospital served as a strategic partner for Sidra Medicine’s 4th annual Pediatric Neuroscience Conference (PNC 2024) which took place in Doha, Qatar. This collaboration emphasizes a mutual commitment to advancing pediatric neurological care and research.

The conference offered a robust platform for presenting groundbreaking research and clinical advancements across diverse subfields such as pediatric neurology, neurosurgery, neuro-critical care, neurogenetics, neuroimmunology and neuroradiology. Esteemed medical professionals and researchers from around the world convened to share insights and innovations that are shaping the future of pediatric neurological health.

Various speakers from Children’s National led in-depth discussions on diagnostic and therapeutic innovations aimed at enhancing outcomes for children with chronic neurological and neurosurgical conditions. PNC 2024 provided an excellent platform for healthcare professionals, researchers and academics to update their knowledge and engage with leading specialists in the field.

Presenters and topics from Children’s National included:
Attendees at Sidra Medicine's 4th annual Pediatric Neuroscience Conference (PNC 2024)

The collaborative efforts with Sidra Medicine highlight a shared vision of pushing the boundaries of pediatric neurological research and treatment, ultimately aiming to improve outcomes for young patients worldwide.

Seven key takeaways from the early development and implementation of the NeuroCardiac Critical Care Program.

Sharing development of the NeuroCardiac Critical Care Program through Congenital Heart Academy

Seven key takeaways from the early development and implementation of the NeuroCardiac Critical Care Program.

Jones presented seven key takeaways from the early development and implementation of the NeuroCardiac Critical Care Program.

“Neurodevelopmental care is not a decision on a day-to-day basis. It is a series of micro-decisions embedded in our practice every single day,” says Melissa Jones, MSN, APRN, CPNP-AC, director of the NeuroCardiac Critical Care Program at Children’s National Hospital.

Several years ago, Jones and colleagues in the Cardiac Intensive Care Unit (CICU) at Children’s National launched the program, which involved team education, implementation of evidence-based best practices, research and quality improvement efforts with the goal of optimizing brain neurodevelopment for patients in cardiac intensive care.

More than 1,000 people around the world registered for a recent virtual educational webinar hosted by the Congenital Heart Academy focused on the topics of neuroprotection for children with congenital heart disease. During the webinar the team outlined the process and lessons learned from developing this important, novel program.

What it is

The NeuroCardiac Critical Care Program at Children’s National is an integrated, multidisciplinary group of clinicians focused on eliminating secondary brain injury, optimizing brain development and promoting healthy family bonding in the CICU.

The team prioritizes several key areas, including:

  • Weekly neurodevelopmental rounds
  • Environmental changes
  • Pain and sedation management
  • Parent and caregiver engagement
  • Neuromonitoring guidelines

Jones presented the multidisciplinary team-oriented approach that led to the launch of the program, which continues to evolve and grow in the CICU today. She also offered a series of lessons learned, such as:

  • Even the patients who are the most fragile can receive developmentally supportive care.
  • Elevating and disseminating the expertise of the team is key.
  • Continuous staff educations and buy-in is essential.
  • Working with existing resources (people and material) is important.
  • Early and small wins can build traction for the team.
  • Taking care of parents is developmental care.
  • Culture change takes time, patience and persistence.

Children’s National leads the way

Children’s National is a national leader in the study of neurodevelopment across the lifespan of children born with congenital heart disease. This includes cutting edge work to understand the fetal brain, earlier diagnosis and intervention for heart disease and how congenital heart anomalies affect growth and development in utero, studies of neuroprotection strategies for use in the operating room, neurologically supportive approaches in cardiac critical care, and neuropsychological support systems as these children grow up and into adulthood.

Cardiac critical care and telehealth experts at Children’s National have been longstanding contributors to the knowledge sharing efforts of the Congenital Heart Academy from its beginning, including leading a precursor international, multi-disciplinary knowledge sharing telehealth series for critical care strategies started during the COVID-19 pandemic.

Watch the webinar: Development of the NeuroCardiac Critical Care Program

Illustration of low brain efficiency

Neighborhood disadvantage alters brain networks in unborn babies

Illustration of high and low brain efficiencyGrowing up in a disadvantaged neighborhood changes a child’s brain even before birth. These new findings, in the latest edition of The Journal of Pediatrics, underscore the need to support young families from a baby’s earliest days.

According to this new research from the Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital, exposure to neighborhood disadvantage lessens the functional integration of neural networks in the fetal brain, as seen on functional MRIs of healthy babies. The center compared the brains of 68 healthy babies on 79 scans. Researchers then mapped details about neural activity to a “social vulnerability index” from the Centers for Disease Control and Prevention, which indicates proximity to environmental stressors.

“We specifically looked at brain architecture to see how easily information flows between different regions,” said Kevin Cook, Ph.D., research faculty at the center and the manuscript’s first author. “To do this, we used graph theory, which borrows concepts from social network theory. It’s widely applied in computer science to understand how information flows within groups, and neuroscience has adapted it to study how information travels within the brain.”

What we found

Dr. Cook said researchers focused on the three metrics:

  • Path length, which measures how many stops information needs to make along its way through the brain.
  • Global efficiency, which measures the overall efficiency of the entire brain’s network.
  • Small-world propensity, which describes how the brain’s network is organized and indicates how well the brain is organized into smaller, efficient networks.

As social vulnerability increased, the research team found global efficiency decreased, meaning the brain’s neural network was less efficient. The path lengths were also longer in children with greater neighborhood disadvantage, reinforcing the global efficiency findings.

The fine print

The under-development and over-development of fetal brains may contribute to neurological disorders, such as autism, epilepsy and other conditions of interest to researchers. Yet science’s understanding of how the brain matures in utero is still limited.

In this study, researchers found a notable difference related to age. At the youngest gestational ages, path lengths are longer, and both global efficiency and small world propensity are lower. As the fetus gets closer to term, path length and global efficiency show rapid maturation and less advantaged fetuses catch up to their peers who have greater advantages.

Researchers saw the same findings for small-world propensity, but the maturation didn’t stop. These unborn babies overshot their peers and had greater small-world propensity, suggesting their brains are divided into a greater number of smaller networks than their advantaged peers.

“We believe that length and global efficiency are on a trend to overshoot,” Dr. Cook said. “These findings are notable because they agree with what we know about older children and adolescents. Greater disadvantage is associated with hyper- or over-maturation of the brain. Our findings suggest that this may be starting even before birth.”

What’s ahead

While still early, this research improves the understanding of how environmental complexities can impact an unborn baby. Catherine Limperopoulos, Ph.D., director of the research center, which opened in 2023, said this work will be foundational as they continue to study the impact of a child’s environment on development.

“These findings have important implications for understanding how status and disadvantage may have a cumulative effect on fetal brain development,” Dr. Limperopoulos said. “We must study and consider how to conceptualize the impact of socioenvironmental disadvantage in communities to better care for children and work to improve outcomes.”

Read the full study – “Greater Neighborhood Disadvantage Is Associated with Alterations in Fetal Functional Brain Network Structure” – in The Journal of Pediatrics.

conceptual illustration of brain and brainwaves

How targeted therapy can help overcome febrile infection-related epilepsy syndrome

conceptual illustration of brain and brainwaves

In their findings, experts achieved a promising functional and neurologic outcome.

Febrile infection-related epilepsy syndrome (FIRES) is a severe neurological condition characterized by the sudden onset of seizures following a febrile illness, often leading to significant morbidity. In a recent study led by Dana Harrar, M.D., Ph.D., director of Critical Care Neurology at Children’s National Hospital, a team of experts followed the case of a 5-year-old previously healthy male who presented with acute neurological decline. This culminated in super-refractory status epilepticus following a fever.

The study outlines a novel treatment regimen that involved early and aggressive cytokine-directed immunomodulatory therapy, alongside a comprehensive seizure management strategy that intentionally avoided barbiturate use. By tailoring treatment to the patient’s evolving cytokine profile and incorporating supportive measures such as the ketogenic diet and antiseizure medications, experts achieved a promising functional and neurologic outcome.

“Now, 2½ years later, he attends mainstream school, has attention-deficit hyperactivity disorder (ADHD), mild neurocognitive impairment and well-controlled epilepsy,” the experts write. “By using cytokine-directed immunotherapy and avoiding a barbiturate coma, we were able to successfully treat a pediatric patient with febrile infection-related epilepsy syndrome and achieve a good outcome.”

The study’s findings show that the understanding of FIRES is advancing as experts uncover the underlying mechanisms of this challenging neurological disorder. Physician-scientists in this field developed innovative treatment strategies targeting immune dysregulation and associated seizures. By following recent consensus recommendations and utilizing molecularly targeted immunotherapy along with serial cytokine profiling, Dr. Harrar and team successfully treated the 5-year-old patient, leading to a positive neurological outcome.

You can read the full study, Successful Management of Febrile Infection-Related Epilepsy Syndrome Using Cytokine-Directed Therapy, in the Journal of Child Neurology.

2024-25 US News Badges

Children’s National again ranked among the best in the nation by U.S. News & World Report

2024-25 US News BadgesChildren’s National Hospital in Washington, D.C., was ranked as a top hospital in the nation by the U.S. News & World Report 2024-25 Best Children’s Hospitals annual rankings. This marks the eighth straight year Children’s National has made the Honor Roll list. The Honor Roll is a distinction awarded to only 10 children’s hospitals nationwide.

This year, U.S. News ended ordinal rankings on its Honor Roll. Instead of assigning a numerical rank from 1 to 10, all hospitals on the Honor Roll will be recognized as having attained the highest standards of care in the nation.

In addition, Children’s National tied for #1 pediatric hospital in the Mid-Atlantic region, which includes New York, New Jersey, Delaware, Pennsylvania, the District of Columbia, West Virginia and Virginia. It’s also best in the Mid-Atlantic in Neonatology.

For the fourteenth straight year, Children’s National ranked in 10 specialty services. New this year, U.S. News included behavioral health as a service line in the rankings. Since it’s the first year, there are no ordinal rankings for behavioral health, but the Children’s National program was named one of the top 50 programs in the country.

“In my first year here, I witnessed what makes Children’s National so special — our commitment to collaboration, empowering one another, and charting a bold path forward for pediatric care,” said Michelle Riley-Brown, MHA, FACHE, president and chief executive officer of Children’s National. “I’m proud U.S. News again recognized Children’s National as one of the top in the nation and the highest-ranked pediatric hospital in D.C., Maryland and Virginia. Together, we’ll continue to push the boundaries of care, research and innovation to make a difference for those who matter most — the kids.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals and recognizes the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News.

“For nearly two decades, U.S. News has published Best Children’s Hospitals to empower the parents and caregivers of children with complex medical needs,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Children’s hospitals appearing on the U.S. News Honor Roll have a track record of delivering unparalleled specialized care.”

The bulk of the score for each specialty service is based on quality and outcomes data. The process includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with the most complex conditions.

The Children’s National specialty services that U.S. News ranked in the top 10 nationally are:

The other four specialties ranked among the top 50 are Behavioral Health, Cardiology and Heart Surgery, Pulmonology and Lung Surgery, and Urology.

Magnetic resonance imaging of a brain with seizures

Making pediatric epilepsy surgery more accessible

Magnetic resonance imaging of a brain with seizures

Pediatricians play a vital role in boosting the use of epilepsy surgery for better patient outcomes.

Despite proven safety, efficacy and cost effectiveness, surgery for pharmacoresistant epilepsy remains one of the most underutilized, evidence-based treatments in modern medicine. Pediatricians are key stakeholders for increasing epilepsy surgical utilization.

A recent commentary from Children’s National Hospital experts — led by Nathan Cohen, M.D., neurologist; Chima Oluigbo, M.D., neurosurgeon; and William D. Gaillard, M.D., chief of Neurology — offers a new perspective that highlights the issue and offers practical solutions to increase primary care physician comfort in the referral of patients for epilepsy surgery.

“Pediatricians should feel empowered to open discussions with epilepsy patients and their families about the possibility of surgery,” the authors write. “Through partnership with managing neurologists and early referral to epilepsy surgery centers, together we can begin to dismantle the significant barriers to access. Only by overcoming our own fears of initiating these discussions can we offer hope to those patients and families who need it the most.”

You can read the full commentary, “Breaking Barriers to Pediatric Epilepsy Surgery Utilization,” in The Journal of Pediatrics.

Douglas Postels and other clinicians in Malawi

Think tank improves care for Malawian children with seizures, epilepsy

  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
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Global health leaders from Children’s National Hospital joined Malawian clinical experts in a two-day think tank to improve the care of children with seizures in the southern African nation. Details were published recently in a perspective in the American Journal of Tropical Medicine and Hygiene.

“The meeting was eye-opening,” said Douglas Postels, M.D., a pediatric neurologist and faculty member with the Center for Translational Research at Children’s National. “Outcomes are changed when we collaborate directly and work through the best solutions for clinical care in low-resource settings. You must weigh what clinics need, what they can implement, and the impact on public health.”

The big picture

Dr. Postels does research and provides clinical care at Queen Elizabeth Central Hospital (QECH), the largest public hospital in Malawi. “Queen’s” is the only medical setting in a country of over 20 million people where an electroencephalogram (EEG) and specialty pediatric neurology services are available. QECH is an essential — and often overtaxed — component of neurological care in Malawi. Cerebral malaria, epilepsy and seizures from birth asphyxia drive the need for neurological evaluations.

EEG is available at QECH for all hospitalized children and is principally used to evaluate for subclinical seizures in those who are comatose. One of three trained technicians acquires the EEG, which is electronically sent to the United States for interpretation. Under best-case scenarios, the turnaround time between EEG collection and interpretation from a neurologist in the U.S. is about two hours. However, many obstacles can slow results, including misaligned schedules across time zones, internet outages and other complications.

With the support of the Center for Translational Research, Dr. Postels brought five U.S.-based colleagues to Africa to meet with Malawian clinicians to better understand local clinical care needs.  A think tank of U.S. and Malawian clinicians worked together to consider research directions and priorities for neurology care and EEG services.  Their conversation focused extensively on integrating technology in the places where it will be most clinically useful.

The fine print

Dr. Postels’ research group aims to aid in the clinical care of Malawian children with epilepsy and to make EEG accessible outside of Queen’s hospital. They hope to eventually create a point-of-care EEG system, linking a method of EEG acquisition that requires little training to apply with a software-based interpretation system.

Expanding the use of EEG in Malawi and throughout Africa has numerous clinical applications. Malawian clinicians hoped that EEG could be made available at their local hospitals and folded into a more comprehensive care algorithm for children at risk of seizures — “one aspect of a ‘packet of care’ that included better overall understanding of seizures, criteria for administration of antiseizure medications, and transfer to the central hospital,” according to the journal article.

Malawian clinicians were also interested in using EEG to detect increased intracranial pressure after head trauma, as neuroimaging services are nearly as rare as EEG in Malawi.  One pediatrician also wanted to use EEG to guide the treatment of neonatal seizures, which are common in children with birth asphyxia, and to use EEG as a prognostic biomarker.

“We hope to empower Malawian clinicians with clinical research skills and experiences so that they can lead and direct future discussions with patients, parents and stakeholders to advance neurologic care and research in their country,” Dr. Postels and his team wrote.

To dive deeper, read the complete perspective — “Establishing Priorities for Epilepsy Care and Electroencephalogram Use in Low-Resource Settings” — in the American Journal of Tropical Medicine and Hygiene.

Audience members at the NIAID Conference

Breaking free of illness: Experts explore solutions for chronic pediatric disorders

  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
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When new patients with chronic illnesses come to Roberta DeBiasi, M.D., M.S., division chief of Infectious Diseases, they are often drained of energy, far behind in school and fatigued by the lack of coordinated care among multiple specialists. She envisions a better way to care for these children: a data-driven, multidisciplinary clinic that can help diagnose and treat disorders facing chronically ill children.

In a keynote address, Dr. DeBiasi laid out her vision for improving care during the 7th annual symposium hosted by Children’s National Hospital and the National Institute of Allergy and Infectious Diseases (NIAID): “A new paradigm: Infection-associated chronic illnesses (IACI) affecting children.” Experts from across the country came together to discuss these IACIs, the importance of finding biomarkers to diagnose and monitor them, data-driven therapeutics to treat them, and the urgent need for protocols to guide physicians.

The patient benefit

A range of IACIs – including long COVID, Lyme disease, postural orthostatic tachycardia syndrome (POTS), dysautonomia and more – are stealing normal, active lifestyles from children. Dr. DeBiasi said patients need researchers who understand disease pathogenesis and have standardized disease classification to diagnose and treat these disorders. Because of the complexity of these cases, care delivery and coordination also need to change. Patients, she said, would best be served by beginning with a three-hour appointment in a multidisciplinary clinic with experts from behavioral health, rehabilitative medicine and other specialties.

“When one of these patients comes to us, we feel helpless as physicians,” Dr. DeBiasi said. “There’s no diagnostic, and we don’t know how to treat them…. It is, to me, very energizing that we’re going to be able to have a coordinated way to help these families. But to do that, we have to have standardized disease classification.”

Moving the field forward

In 2017, NIAID and Children’s National launched a clinical research partnership devoted to advancing the health of children with allergic, immunologic, auto-inflammatory and infectious diseases through collaborative research and education. The partnership — co-led by H. Clifford Lane, M.D., NIAID’s deputy director for Clinical Research and Special Projects, and Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National — promotes the prevention, diagnosis, treatment and cure of these childhood diseases. Every year, the partnership organizes and funds a symposium to explore cutting-edge research and pressing issues in pediatric care.

At this year’s gathering, experts examined the specifics of various disorders, including Alexandra Yonts, M.D. She provided new data from the Children’s National Post-Covid Program and explained that patients “are clamoring” for help with symptom management.

“There have been virtually no randomized, controlled trials and very little evidence on any sort of aggressive treatments, or pharmaceutical management options, especially in the pediatric population,” Dr. Yonts said. “Upon recent attendance at some long COVID meetings, there is an expressed concern, especially among pediatric providers, about trying medications off-label in these long COVID patients.”

Miss the symposium? You can learn more about the science they explored in the symposium’s recording on topics including:

  • Potential overarching mechanisms of disease, including pathogen- and host-mediated factors.
  • Identifying potential biomarkers for chronic illnesses
  • Treatments and multidisciplinary approaches for patients with IACIs
  • Patient advocacy
A clean room at CNRI.

CellBuilder: A ready-made solution for cell & gene therapy manufacturing

A clean room at CNRI.

With CellBuilder and our global partnerships, Children’s National hopes to expand access to groundbreaking cell and gene therapy treatments as they take off in the next five years.

With cell and gene therapies poised to reshape cancer and rare disease treatments, researchers at Children’s National Hospital are pioneering ready-to-use solutions that will bring these cutting-edge therapies directly to hospitals and other treatment centers, shrinking the distance between doctors and patients.

“The next five years are going to be a period of tremendous growth for cell and gene therapy,” said Patrick Hanley, Ph.D., chief and director of the Cellular Therapy Program at Children’s National. “Currently, there’s no shortage of interest from the medical community, but there’s a shortage of people who can manufacture and administer this care. We’re looking for ways to get these treatments to the patients by providing other institutions the tools they need to launch these programs cost-effectively, safely and efficiently.”

Called CellBuilder, the starter kits for cell and gene therapy programs could transform the landscape for pediatric patients.

The big picture

Dr. Hanley and many members of the Children’s National team have been working in cell and gene therapy for more than a decade, gaining extensive experience in the technical and regulatory hurdles inherent in creating treatments that target diseases at their source.

In cell therapy, a specific cell type is modified and transferred to a patient with a payload that can target a disease or disorder. For example, T cells may be modified and delivered to patients to teach their immune systems to fight cancer.  In gene therapy, a patient’s genetic code is modified to treat or prevent diseases, such as sickle cell disease, cancers and other genetic disorders. This can be done by introducing a healthy copy of a gene, repairing a faulty gene or altering a gene’s function.

Children’s National has become a leader in manufacturing virus-specific T cells, one method of delivering cell therapies, and the Cellular Therapy Program has conducted consortium-led, multi-center trials. Many other healthcare sites across the country want to start programs offering this care at their facilities.

The holdup in the field

Starting a cell and gene therapy program from scratch can take years of effort, training and money. That’s why Jay Tanna, M.S., R.A.C., quality assurance manager of the Cellular Therapy Program at Children’s National, said the team is creating CellBuilder starter kits, which include the manufacturing protocol and the resources necessary to launch a cell therapy program almost instantly.

“With a suitable knowledge base, institutions can start their own cellular therapy program at the point of care, using our manufacturing protocols, vetted reagents and other key elements of the process,” Tanna said. “Of course, interested institutions would have to meet regulatory requirements and establish a clean room to manufacture these therapies. If they want to use CellBuilder to run a clinical trial, they can do that. If they want to take it to become a licensed product, they can do that, too.”

Children’s National has worked with more than five institutions to build their virus-specific T-cell program and is now using the kits to accelerate and commercialize the process to increase patient access. The lab has also entered into a memorandum of understanding with the Tokyo-based Hitachi Global Life Solutions, Inc., an innovative modular clean room manufacturer, with the goal of offering a bundled solution.

Why we’re excited

Dr. Hanley and his colleagues say that the partnerships Children’s National is forging as they consult with other experts in this field will expand access to cell and gene therapy across the country—and hopefully around the world.

“It used to be that, to get a CAR T cell, you would collect the cells at Children’s National, ship them to a company like Novartis, have the therapy manufactured there and then shipped back,” said Michael Keller, M.D., who co-led a first-of-its kind immunotherapy trial as the Translational Research Laboratory director at the Children’s National Cell Enhancement and Technologies for Immunotherapy Program. “It was expensive, time-consuming and limited patient access. Now, there’s growing interest in manufacturing at each site, just like you would with a bone marrow transplant.”

With CellBuilder and our global partnerships, Children’s National hopes to expand access to groundbreaking cell and gene therapy treatments as they take off in the next five years, extending lifetimes and improving the quality of life for children suffering from rare disorders.

“We’re trying to capture the momentum underway in the field by providing this kit so that institutions don’t have to know how to do everything,” Dr. Hanley said. “We provide all the knowledge, a reagent list and everything else they need—and they provide the care.”

Cheng-Chia “Fred” Wu, M.D., Ph.D.

Expanding team innovates to take on pediatric brain tumors

Cheng-Chia “Fred” Wu, M.D., Ph.D.

Cheng-Chia “Fred” Wu, M.D., Ph.D., joins the team at the campus as an assistant professor at Virginia Tech’s Fralin Biomedical Research Institute. Image credit: Fralin Biomedical Research Institute.

Experience, talent and technology are coming together at the Children’s National Research & Innovation Campus to solve the complex challenges of treating pediatric brain tumors through a growing partnership between Children’s National Hospital and Virginia Tech.

Cheng-Chia “Fred” Wu, M.D., Ph.D., joins the team at the campus as an assistant professor at Virginia Tech’s Fralin Biomedical Research Institute. He hopes to improve treatment for pediatric brain tumors and other cancers by leveraging technological advances in focused ultrasound and studying how this modality can be combined with other novel therapies. Children’s National physicians and scientists are collaborating with Virginia Tech scientists to develop these new approaches.

Why we’re excited

Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research and interim chief academic officer at Children’s National, said collaborations — including the partnership between Children’s National and Virginia Tech — are essential to solving formidable scientific challenges.

“We are thrilled to be working with Dr. Wu to develop cutting-edge therapeutics for pediatric brain tumors,” Dr. Bollard said. “With Dr. Wu’s experience and the talents of our team at Children’s National, I have great hope that we will be able to combine our novel technologies and therapies to provide tremendous breakthroughs for treating pediatric patients with brain tumors.”

Before joining Virginia Tech, Dr. Wu was an assistant professor of radiation oncology at Columbia University Irving Medical Center in New York, where he treated pediatric cancers and central nervous system malignancies.

“Partnering with Children’s National connects us to a world-class clinical trial institute that has been a pioneer in treating brain tumors with focused ultrasound technology, and this presents a unique opportunity to help children and families struggling with cancer,” Dr. Wu said. “I can’t wait to see where this takes us.”

Dr. Wu played a key role in the Initiative for Drug Delivery Innovation for Childhood Brain Tumors at Columbia, developing a bench-to-bedside platform to facilitate the translation of promising technologies for targeted drug delivery in children with brain tumors.

Within three years, the team demonstrated the safety and feasibility of using focused ultrasound in the brainstem and adding radiation in preclinical models. They then opened two clinical trials for kids with relapsed diffuse midline glioma, an aggressive and difficult-to-treat brain tumor that occurs in the brain stem, thalamus and spinal cord.

What’s ahead

Dr. Wu will be involved in a wide range of research touching both organizations, including veterinary medicine and biomedical engineering at Virginia Tech.

Michael Friedlander, Ph.D., executive director of the Fralin Biomedical Research Institute at Virginia Tech Carilion and vice president for health sciences and technology at Virginia Tech, welcomed Dr. Wu’s experience as a pediatric radiation oncologist, translational physician-scientist and clinical trialist who has led an innovative drug delivery program.

“He is armed with a unique set of skills to identify promising new technology and implement it in areas of great need for treatment of pediatric cancers,” Dr. Friedlander said. “We are absolutely delighted to have Dr. Wu as part of the team.  He represents a powerful new part of the strong partnership between Virginia Tech and Children’s National Hospital for addressing pediatric brain cancer.”

Dr. Craig Sable in Uganda

Around the world

Our Global Health Initiative launched in 2016 with the goal of eliminating pediatric health disparities around the world. We aim to address the most pressing pediatric health issues through better care for medically underserved populations. This leadership helps us achieve our mission of caring for all children. A broad range of education and research projects improves health outcomes. They also offer enriching opportunities for experienced faculty and emerging leaders to advance clinical excellence.

Healing hearts in Uganda

Dr. Craig Sable in Uganda

Dr. Craig Sable and team train partners in Uganda.

Craig Sable, M.D., interim chief of Cardiology, improves care for young people with rheumatic heart disease (RHD) in Uganda. Donors, including the Karp Family Foundation, Huron Philanthropies, Zachary Blumenfeld Fund and the Wood family, make this possible. RHD affects 50 million people, mostly children, worldwide. It claims 400,000 lives each year.

Dr. Sable and Ugandan partners completed important research showing that early RHD detection, coupled with monthly penicillin treatment, can protect the heart. They are working on practical solutions, such as a new portable device with artificial intelligence (AI) that can easily screen for RHD.

In 2023, Dr. Sable led two missions in Uganda where he and his team did surgeries and special tests for 18 children with RHD. They also taught local doctors new skills to help more kids on their own.

Plastic surgery and reconstructive care in Kenya and Nepal

Each year our Craniofacial & Pediatric Plastic Surgery team, under the leadership of Johnston Family Professor of Pediatric Plastic Surgery and Chief of Pediatric Plastic Surgery Gary Rogers, M.D., J.D., LL.M., M.B.A., M.P.H., provides opportunities for fellows to participate in surgical missions.

In 2024, Perry Bradford, M.D., traveled to the Moi Teaching Hospital in Eldoret, Kenya where she provided patients with burn, pressure wound and cleft reconstruction. She built community connections with the local plastic surgeons and educated registrars and medical students. “This gave me firsthand experience working in a community with limited resources and forced me to be more creative,” Dr. Bradford says. “The experience inspired me to examine what it means to have consistent access to advanced tools and equipment.”

In 2022, a group traveled to Nepal to provide care. Some patients arrived after days of travel by yak or buffalo. One child with a burn injury recovered use of her hand. The team educated local providers to deliver life-changing treatments unavailable in Nepal.

Dr. Tesfaye Zelleke in Ethiopia

Dr. Tesfaye Zelleke, left, and team in Ethopia.

Elevating epilepsy care in Ethiopia

Neurologist Tesfaye Zelleke, M.D., and partners in Ethiopia are seeking to improve the lives of children with epilepsy. The BAND Foundation provides support. Ethiopia has a population of about 120 million yet only a handful of pediatric neurologists.

Dr. Zelleke’s team trained nonspecialist providers to diagnose and treat children in the primary care setting. They also launched a mobile epilepsy clinic to provide community care and build the capacity of local clinicians. In collaboration with advocacy groups, the team educates the public about epilepsy with a goal of reducing stigma.

New hope in Norway

In 2023, our Division of Colorectal & Pelvic Reconstruction shared its expertise with clinicians at Oslo University Hospital, Rikshospitalet, in Norway. This effort was a key first step in Oslo becoming the first dedicated colorectal center in Scandinavia.

Marc Levitt, M.D., and team members performed complex surgeries otherwise unavailable for waiting patients. They led an academic conference. They held clinics to educate nurses, reviewed patient records and made care recommendations. Specialized care enabled a young patient with significant bowel difficulties to recover function and lead a normal life.

The team will travel to South Africa, the Czech Republic and Spain in 2024. Donors, including The Dune Road Foundation and Deanna and Howard Bayless, make this work possible.

Improving outcomes for babies in the Congo

AI can be a valuable tool for diagnosing genetic conditions. It detects unique facial patterns that clinicians without genetics training can miss. However, existing facial analysis software struggles in nonwhite populations.

A team led by Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor of Research and Innovation and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation, is working to improve the newborn diagnosis rate worldwide. They are testing smartphone software in the Democratic Republic of Congo. Diverse newborn data improves AI’s ability to detect a variety of genetic conditions in more children. Early detection, diagnosis and informed care lead to better health outcomes.

Nephrology care for kids in Jamaica

Dr. Moxey-Mims and team in Jamaica

Jennifer Carver and Dr. Marva Moxey-Mims, center, with staff at Bustamante Children’s Hospital.

Marva Moxey-Mims, M.D.,, chief of Nephrology, is bringing care to children with kidney disease in Jamaica, with a goal of improving health equity. An International Pediatric Nephrology Association grant helped make it possible.

On a recent trip, Dr. Moxey-Mims and a small team — including Jennifer Carver, RN, CNN, lead peritoneal dialysis nurse at Children’s National, and three pediatric nephrologists from Jamaica — trained nearly 30 nurses from Jamaican hospitals. Nurses received hands-on dialysis education to improve their clinical skills. The team also worked to educate the community in disease awareness and prevention.

Read more stories like this one in the latest issue of Believe magazine.

stressed pregnant woman

Pandemic stress in pregnant mothers may affect anxiety regions of babies’ brains

stressed pregnant woman

The research from Children’s National Hospital provides mounting evidence that children of the pandemic, even those far too young to understand it, need ongoing assessments of developmental or mental health support later in life.

A critical part of the brain linked to risks for anxiety later in life – the left amygdala – was significantly smaller by volume in babies of mothers who reported stress during the COVID-19 pandemic, according to a new manuscript published in JAMA Network Open.

The right hippocampus, which governs spatial, visual and verbal memories, and the white matter were also reduced in children whose mothers reported stress.

The research from Children’s National Hospital provides mounting evidence that children of the pandemic, even those far too young to understand it, need ongoing assessments of developmental or mental health support later in life.

“Looking ahead, we want to use this information – and studies with similar findings – to empower pregnant mothers to request support to mitigate their stress, especially in the event of another global health crisis,” said Nickie Andescavage, M.D., a neonatologist and principal investigator at the Center for Prenatal, Neonatal & Maternal Health Research. “We also want to make sure babies born during COVID-19 get the services that they need in life if they develop anxiety or other mental health disorders.”

The fine print

Researchers at the center used magnetic resonance imaging (MRI) to compare the brains of 103 babies born between 2014 and 2019 prior to the pandemic to 59 born between 2020 and 2022. Mothers who had COVID-19 or other complications in their pregnancies were excluded. The babies underwent MRI imaging while in utero and again soon after delivery.

The mothers were evaluated for stress and anxiety, using the Spielberger State-Train Anxiety Inventory and other evidence-based scoring measures. Pre-pandemic, 21% of mothers reported elevated symptoms of anxiety; in the pandemic cohort, that number jumped to nearly 62%.

Their babies’ brains were also changed, as regions widely understood to control emotion and anxiety displayed smaller volumes on MRI imaging. Given the global impact of the pandemic and universal reports of mental distress worldwide, the potential impact of these findings may impact an entire generation of children born during the pandemic. The team is just beginning to unravel the medical significance.

What’s next

Catherine Limperopoulos, Ph.D., director of the Center for Prenatal, Neonatal & Maternal Health Research, said understanding the impact of stress is vital in supporting the healthy development of young children. Current studies are underway at her center to tease apart the role of stress in prenatal development and examine its long-term impact on development, including cognition, behavior and mental health.

“We all know that being pregnant can be quite stressful, and there are certain times of collective stress that can provide us windows to understand how the body and mind manage it,” Dr. Limperopoulos said. “At our center, we care deeply about the health of mothers and babies, and our researchers plan to continue investigating the role of stress in development to continue building data to show that mental health must be a greater priority.”

This study – “Prenatal maternal psychological distress during the COVID-19 pandemic and newborn brain development” – was supported by the National Institutes of Health, the Intellectual and Developmental Disabilities Research Center, and the A. James & Alice B. Clark Foundation. You can read the full study in JAMA Network Open.

illustration of neural network

Tailored care for children with autism improves EEG experience

illustration of neural network

Most children with a developmental disability can complete EEG with sufficient support.

Electroencephalographic (EEG) monitoring is an important tool for diagnosing seizures, epilepsy and other neurologic conditions. However, many children with developmental disabilities – including autism – have difficulty undergoing hospital tests like EEG. Furthermore, sometimes these tests are not prescribed or turn out to be unsuccessful.

In a new study published in Journal of Child Neurology, findings from a Children’s National Hospital team indicate that most children with a developmental disability can complete EEG with sufficient support. Special planning can identify children who would benefit from accommodations to help them complete the study successfully.

What this means

Children with developmental disabilities have an increased risk of epilepsy and need for overnight video EEG monitoring. However, video EEGs have historically been considered difficult to complete for this population. Experts at Children’s National implemented a coordinated team approach to help children with developmental disability tolerate overnight video EEGs. The project involved a caregiver preprocedure questionnaire shared with the team to create personalized care plans.

These findings suggested that most children with developmental disability can complete video EEG with sufficient support. Preprocedure planning can identify children who would benefit from additional accommodations.

Moving the field forward

The findings show new knowledge through vigorous research about the patient experience and the value of a team dedicated to helping children with development disabilities undergo hospital tests.

“We were excited to find that with special planning more children with developmental disabilities could complete an EEG,” said Elizabeth Wells, M.D., senior vice president at the Center for Neuroscience and Behavioral Medicine at Children’s National, and one of the study’s authors. “We thought it was important to study and discover that children from minority or underrepresented groups were less likely to access support services, which means more work and research is necessary to ensure that services are accessible to these children.”

The authors also found that parent-reported difficulties with communication and cooperation were associated with video EEG success, which means doctors and hospital teams should always ask about and listen to parent input in planning the tests for children.

How Children’s National is leading the way

Healthcare workers often express lack of confidence in their ability to provide care for patients with autism and associated disabilities and express an interest in gaining access to resources and training to better care.

By developing a program to improve care and then studying it through rigorous research, the authors aspire to spread a message of hope. Programs like these can ensure all kids get the tests and care they need. Children’s National is also a leader in showing the importance of parent input in medical care planning, which too often is not a routine part of care at hospitals.

Further research is necessary to clarify which supports are most helpful.

Three prior students who trained at Children’s National are lead authors, Kalyn Nix, Jessica Smith and Atara Siegel, Ph.D. Additional authors from Children’s National include Kathleen Atmore, Psy.D., and Elizabeth Wells, M.D.

You can read the full study, Individualized Care Delivery for Children With Autism and Related Disabilities Undergoing Overnight Video Electroencephalography (EEG): One Hospital’s Experience With a Coordinated Team Approach, in the Journal of Child Neurology.

illustration of a neural network

Adjusting key protein could improve brain function in children with fetal alcohol syndrome

illustration of a neural network

Researchers at Children’s National are testing whether controlling the level of apolipoprotein E could serve as an effective treatment for the poor neurobehavioral outcomes tied to fetal alcohol spectrum disorders.

Reduced levels of a protein – called apolipoprotein E – are responsible for the lifelong cognitive and neurological symptoms in fetal alcohol spectrum disorders (FASD), according to a new study published in the journal Molecular Psychiatry. For the first time, researchers at Children’s National Hospital are testing whether controlling the level of this protein could serve as an effective treatment for the poor neurobehavioral outcomes tied to FASD, which is believed to affect roughly 5% of school-aged children.

Apolipoprotein E is controlled by a gene called APOE, a well-known risk gene for Alzheimer’s disease. It contains the instructions that guide how the connections between neurons in the brain strengthen or weaken over time. This process, known as synaptic plasticity, is crucial for learning and memory formation. In this study, plasma was collected from participants at two sites in Western Ukraine, along with information about drinking behavior reported by their mothers.

What this means 

“In addition to Alzheimer’s risk, we found this gene is also a crucial contributor to cognitive problems in children with FASD,” said Kazue Hashimoto-Torii, Ph.D., a principal investigator at the Center for Neuroscience Research at Children’s National. “The new data shows that we understand the mechanism by which prenatal alcohol exposure causes a decrease of the APOE level in the brain. We will continue this work to help improve our understanding of FASD, hoping to replenish this important protein and improve outcomes for children with these disorders.”

FASD is an umbrella diagnosis for the physical and developmental challenges that face children who are exposed to alcohol in utero, including intellectual disability, delay in motor and language development and other neurological diagnoses. While alcohol alone is problematic to many aspects of development, evidence also suggests that genetic factors play a role. Only 4.3% of children with heavy alcohol exposure develop FASD, and twin studies have revealed that fraternal twins have different FASD outcomes compared to identical twins.

What’s next 

The research team at Children’s National wanted to pinpoint places where genes could play a role and consider therapies. For the first time, researchers found that plasma levels of APOE were reduced in children with FASD, which strongly supports a potential target for therapy. In addition, their genomics study found a variant of APOE increases the risk of cognitive problems in subjects who were prenatally exposed to alcohol, especially those of African ancestry.

“We found that providing a drug that activates the APOE receptor rescued learning deficits and anxiety in pre-clinical models,” said Masaaki Torii, Ph.D., principal investigator at the Center for Neuroscience Research. “The implications offer an exciting glimpse into possible therapies for some of the neurological harms associated with prenatal alcohol exposure and FASD.”

Read the full manuscript – Reduction of APOE accounts for neurobehavioral deficits in fetal alcohol spectrum disorders – in Molecular Psychiatry.

Dr. Anitha John addresses symposium attendees

Addressing long-term brain effects of congenital heart disease

Dr. Anitha John addresses symposium attendees

Dr. Anitha John, medical director of the Washington Adult Congenital Heart Program at Children’s National Hospital, presenting on the lifelong effects of congenital heart disease on brain health at a recent symposium.

About 81% of the 40,000 babies born in the United States with congenital heart disease (CHD) are expected to survive to at least age 35, according to the Centers for Disease Control and Prevention. As survival rates have increased in recent decades, clinicians treating CHD patients are seeking to improve outcomes by understanding the long-term health effects and complications that arise for them.

Anitha John, M.D., Ph.D., medical director of the Washington Adult Congenital Heart Program at Children’s National Hospital, presented an overview of what researchers currently know about the lifelong effects of CHD on brain health at a symposium focused on the heart-brain continuum presented by Children’s National Innovation Ventures, CobiCure and JLABS @ Washington, D.C. She also discussed critically needed advancements in monitoring technology to help clinicians better understand and address how CHD affects the brain.

Why it matters

Based on data collected from adults and children with the condition, Dr. John shared that people with CHD face many potential lifelong challenges and risks, which vary based on disease severity:

  • About one-third report a mood disorder, either anxiety or depression
  • 25% higher risk of substandard academic outcomes
  • 50% more likely to require special education services
  • Higher incidence of motor skills impairment
  • Higher lifetime prevalence of ADHD
  • Generally lower educational attainment at adulthood
  • Higher risk of autism spectrum disorders
  • Higher rate of dementia before the age of 65

Why do some people with CHD experience profound, lifelong brain effects? Dr. John notes that clinicians and researchers are seeking those answers, recognizing that they likely involve various factors and accumulating issues that occur over the entire lifespan, from fetal life onward.

Because the heart supplies the brain with oxygen through circulated blood, the diagnostic tool clinicians most want for patients of all ages is a technology that enables noninvasive monitoring of central venous pressure, an indicator of the volume of blood returning to the heart and the pressure within the heart. Currently, the most reliable way to measure this pressure is by an invasive procedure in which a catheter is inserted into the patient’s subclavian or internal jugular vein or by placing a device into the patient’s pulmonary artery. These procedures have limitations and cannot be used for routine surveillance.

What’s next

Dr. John says noninvasive central venous pressure monitoring is important to understanding and addressing what is causing brain injury in CHD patients. She says the challenges in developing this monitoring solution include the need for an individualized approach, a design that accommodates multidisciplinary use, sizing for patients from infants to adulthood, usability for all age groups and avoiding stigma for wearers.

To address this need, the Alliance for Pediatric Device Development – a consortium funded by the Food and Drug Administration and led by Children’s National – is partnering with CobiCure to issue a request for proposals for direct device funding. The goal is to provide funding to innovators who offer solutions to the dire unmet need for pediatric devices that provide noninvasive monitoring of the circulatory system and heart performance. Details will be announced in June 2024.

 

Maureen Lyon, Ph.D.

Pilot program improves well-being of families during advanced care planning


Children with life-limiting rare diseases and their caregivers face tremendous stress and anxiety about the heart-breaking decisions before them. A new intervention – designed at Children’s National Hospital to support the palliative needs of these families – improved their spiritual and emotional well-being, according to new research published in the journal, Pediatrics.

Called FACE Rare (FAmily CEntered Pediatric Advance Care Planning Intervention for Rare Diseases), the counseling tools were found to be safe, effective and increased feelings of peace among families in this underserved population.

“Seventy-four percent of the families in that intervention group reported feelings of sadness, yet 100% reported our pilot intervention was a worthwhile experience,” said Maureen Lyon, Ph.D., a clinical psychologist and principal investigator at the Center for Translational Research at Children’s National. “If you’re talking about the possibility that the worst thing in the world would happen to you – that your child might die, and what you would want for them – the families found that our intervention helped. They had a place to process their feelings and consider what would be important to their child.”

The big picture

A rare disease is defined as any condition affecting fewer than 200,000 people in the United States. In pediatrics, these diseases often require constant caregiving and require families to face the cruel reality that the diagnosis may be life-limiting. In such cases, clinical teams often decide that conversations about advanced care planning (ACP) are needed.

The pilot-phase, randomized trial enrolled patients from Children’s National between 2021 and 2023. Research nurses underwent two days of training to be certified in the new intervention. Families assigned to the new approach received three, weekly 60-minute sessions and were evaluated using evidence-based assessment tools, including the Carer Support Needs Assessment Tool/Action Plan and the Next Steps: Respecting Choices Pediatric ACP Conversation. Measures of anxiety and spiritualization were tracked, and families returned after three months for follow-up.

Patients had a range of diagnoses that put tremendous strain on the caregivers, including complex digestive disorders, white-matter diseases and rare forms of epilepsy. Yet the caregivers expressed similar challenges: having time for themselves during the day, knowing what to expect in the future for their child’s care, and managing financial, legal and work issues.

What we found

Reinforcing the need to improve engagement and support for these families, the investigative team found that those who received the FACE Rare intervention reported higher levels of spiritual meaning and peace than those who received “treatment as usual.” All families living below the poverty line reported greater anxiety, and noteworthy to the team, black caregivers were less likely to report caregiver distress than non-black caregivers.

Dr. Lyon said future research needs to understand better how families respond to the challenges of rare diseases and unique social determinants of health that can change the approach to care.

“In some cultures, and families, having strong feelings is discouraged,” Dr. Lyon said. “We want to give families and caregivers space to have these emotions and think about what would be important for their child, if the worst were to happen. They appreciated that they participated in the program and had the space to consider these difficult questions.”

Read the full study, “Advance Care Planning for Children with Rare Diseases: A Pilot RCT,” in Pediatrics.

2024 Pediatric Academic Societies meeting logo

Children’s National Hospital at the 2024 Pediatric Academic Societies meeting

Children’s National Hospital-affiliated participants will present at this year’s Pediatric Academic Societies meeting. The meeting will take place in Toronto, from May 2-6, 2024. For information on the presentations, please refer to the chart below.

Day Time Presenter(s) Title
5/3/2024 9:00 AM Stacey Stokes, M.D., M.P.H. APA QI: Informatics for Improvers: Leveraging Clinical Decision Support to Propel Data-Driven and Reliable Continuous Improvement
5/3/2024 12:00 PM Rana F. Hamdy, M.D., M.P.H., MSCE A Career in Antimicrobial Stewardship… so Much More to Explore
5/3/2024 12:00 PM Ashima Gulati, M.D., Ph.D., FASN Case Studies in Pediatric Kidney Diseases: Who, When and How to Order Genetic Testing?
5/3/2024 3:45 PM Priti Bhansali, M.D., ME.d. iSPOT an Improvement: Taking Peer Observation and Feedback to the Next Level
5/3/2024 3:45 PM Josepheen De Asis-Cruz, M.D., Ph.D. Maternal psychopathology and SSRI use during pregnancy are associated with altered fetal hippocampal connectivity in utero
5/4/2024 8:00 AM Andrea J. Boudreaux, Psy.D., M.P.H., M.H.A., F.A.C.H.E. A Doctor in the School Nurse’s Office? Bringing a Virtual School Based Program into Practice
5/4/2024 8:00 AM Jessica Hippolyte, M.D., M.P.H. A Practical Approach to a Thorny Issue: Evaluating the Role of Race, Ethnicity, and Ancestry in Clinical Decision-Making
5/4/2024 8:00 AM Ashraf S. Harahsheh, M.D., F.A.A.P., F.A.C.C. Cardiology 1
5/4/2024 8:00 AM Dewesh Agrawal, M.D. Emergency Medicine 1: Quality and Safety
5/4/2024 8:00 AM Lenore Jarvis, M.D., ME.d.
Amanda Stewart, M.D., M.P.H.		 From Bedside to State House: Daily Advocacy
5/4/2024 8:00 AM Aisha Barber, M.D., ME.d. Unionization in Pediatrics: A Pro-Con Debate
5/4/2024 8:45 AM Jillian E. Nickerson, M.D., M.S. Implementation of tele-psychiatry in an urban pediatric satellite emergency department
5/4/2024 9:00 AM Jessica Weisz, M.D. “TEACH”ing: Evaluation of a 3-Year Multimodal Child Poverty Curriculum
5/4/2024 9:00 AM Tameka T. Watson, M.D. Timing of Growth Failure in Very Premature Infants and Implications for Brain Development
5/4/2024 10:00 AM Neha H. Shah, M.D., M.P.H. Subspecialty Networking Breakout: Pediatric Hospital Medicine
5/4/2024 11:00 AM Lee S. Beers, M.D. AAP Presidential Plenary: Emerging Research on the Intersections of Mental Health, Impact of the Pandemic, and Equity, Diversity, and Inclusion
5/4/2024 11:00 AM Denver Brown, M.D. Implications of SDOH on glomerular disease, hypertension and CKD
5/4/2024 11:15 AM Josepheen De Asis-Cruz, M.D., Ph.D. Associations Between Antenatal Opioid Exposure and Newborn Functional Brain Connectivity
5/4/2024 2:00 PM Ian Chua, M.D., M.H.P.E. Addressing Misinformation & Building Competency for Gender Diverse Patient Care
5/5/2024 8:00 AM Tamara Gayle, M.D., ME.d., M.P.H. APA SIG: Pediatric Hospital Medicine – Creating an Inclusive and Sustainable Workplace
5/5/2024 8:00 AM Suma B. Hoffman, M.D., M.S.
Simranjeet S. Sran, M.E., ME.d., C.H.S.E., F.A.A.P.		 Hands on Workshop: Complex Resuscitations in Neonates and Infants – Managing High Acuity, Low Occurrence (HALO) Events
5/5/2024 8:15 AM Jaytoya Manget, D.N.P., M.S.P.H., F.N.P. Connecting the Dots to Improve Health and Education Equity: Results of A Pilot Program Integrating School Attendance Data into a Pediatric Primary Care
5/5/2024 8:55 AM Aisha Barber, M.D., ME.d. APA Pediatric Hospital Medicine SIG – Creating an Inclusive and Sustainable Workplace
5/5/2024 11:00 AM Nicola Brodie, M.D.
Julie Heier, Ph.D.
Courtney Horton, M.D.
Darcel Jackson, C.P.X.P., L.S.S.G.B.
Emma Whitmyre, Ph.D.		 Challenge Accepted: Integrating Behavioral Health in Primary Care for Children with Medical Complexity and Their Families
5/5/2024 11:00 AM Ian Chua, M.D., M.H.P.E.
Margarita Ramos, M.D., M.S.
Neha H. Shah, M.D., M.P.H.		 Embracing Failure: The Key To Success In Academic Medicine
5/5/2024 11:00 AM Caleb E. Ward, M.B., B.Chir., M.P.H. Emergency Medicine 4
5/5/2024 11:00 AM Dewesh Agrawal, M.D.
Terry Kind, M.D., M.P.H.		 Launching and Landing a Career in Medical Education: From Passion to Profession
5/5/2024 11:00 AM Nathaniel S. Beers, M.D., M.P.A.
Andrea J. Boudreaux, Psy.D., M.P.H., M.H.A., F.A.C.H.E.
Bianca Johnson, M.S.W.
Jaytoya Manget, D.N.P., M.S.P.H., F.N.P.
Jessica Weisz, M.D.		 School Attendance as a Vital Sign: Integrating school attendance into practice to advance health and educational equity
5/5/2024 11:05 AM Sudeepta Basu, M.D. SPR 2023 Bridging to Success Award: GABA-editing spectroscopy for understanding the developing brain in preterm infants.
5/5/2024 2:00 PM Allison M. Jackson, M.D., M.P.H. Child Protective Services Referrals in the Context of Intimate Partner Violence: Clinical Practice, Research, & Advocacy
5/5/2024 2:00 PM Josepheen De Asis-Cruz, M.D., Ph.D. In utero SSRI exposure alters fetal cerebral cortical development and structural brain connectivity
5/5/2024 2:00 PM Jeremy Kern, M.D.
Lydia Lissanu, M.D.
Elana Neshkes, M.D.
Laura A. Nicholson, M.S.N., R.N., C.P.N., C.H.S.E.
Grace Quinn, M.D.
Ariella M. Weinstock, M.D., M.S. Ed		 STRIVE for a restorative de-escalation: Strategies for a TRauma-Informed approach using Verbal and Environmental Skills
5/5/2024 2:45 PM Kristen Sgambat, Ph.D., RD Arterial stiffness, body composition, and perception of racism in pediatric kidney transplant recipients
5/5/2024 2:45 PM Sudeepta Basu, M.D. Cerebellar GABA and Glutamate Concentrations at Term-equivalent age Predicts 18-month Cognitive Deficits in Preterm Infants
5/6/2024 8:00 AM Ian Chua, M.D., M.H.P.E.
Gabrina Dixon, M.D., ME.d.
Tamara Gayle, M.D., ME.d., M.P.H.
Margarita Ramos, M.D., M.S.		 Amplify Your Voice: Media Strategies Beyond Conventional Academic Dissemination
5/6/2024 8:00 AM Stacey Stokes, M.D., M.P.H.
Padma Swamy, M.D., M.P.H.		 APA SIG: Health Informatics and Serving the Underserved Combined – Moving the Needle on Social Needs: From Screening to Data Management and Response
5/6/2024 8:00 AM Deena Berkowitz, M.D., M.P.H. APA Urgent Care SIG: You’re Not Too Busy To Think About Promotion: Leveraging Your Current Scholarly Activities For Academic Advancement
5/6/2024 8:30 AM Padma Swamy, M.D., M.P.H. Screening, data sharing, and resource allocation considerations when developing social needs interventions
5/6/2024 1:00 PM Christina Lindgren, M.D. APA Simulation-based Medical Education (SBME) SIG
5/6/2024 1:00 PM Gabrina Dixon, M.D., ME.d. Creating and Optimizing a Visiting Elective at your Institution for Underrepresented in Medicine (URiM) Students
5/6/2024 1:00 PM Junghoon Kim, Ph.D. Improved prediction of fetal neurobiological features by censoring high-motion frames in fetal functional MRI
5/6/2024 1:05 PM Christina Lindgren, M.D. Introduction to Conceptual Frameworks for Simulation Based Medical Education
5/6/2024 1:10 PM Suma B. Hoffman, M.D., M.S. Small Group Activity: Name That Conceptual Framework
5/6/2024 2:15 PM Margaret Rush, M.D., M.S.H.S. Racial disparities in hospital length of stay for bacterial tracheostomy associated infections

 

Marius George Linguraru giving a lecture on AI

Artificial – and accelerated – intelligence: endless applications to expand health equity

In the complex world of pediatric diseases, researchers need access to data to develop clinical trials and the participation of vulnerable patients to develop new devices and therapies. Both are in short supply, given that most children are born healthy, and most severe pediatric diseases are rare.

That creates a dilemma: how do researchers build a foundation to advance new treatments? Enter artificial intelligence (AI).

“AI is the equalizer: accelerated intelligence for sick kids. No other advance on the horizon holds more promise for improving equity and access to pediatric healthcare when diseases are rare and resources are limited,” says Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor in Research and Innovation and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI). “AI will shrink the distance between patient and provider, allowing our physicians and scientists to provide targeted healthcare for children more efficiently. The possibilities are endless.”

Why we’re excited

By pioneering AI innovation programs at Children’s National Hospital, Dr. Linguraru and the AI experts he leads are ensuring patients and families benefit from a coming wave of technological advances. The team is teaching AI to interpret complex data that could otherwise overwhelm clinicians. Their work will create systems to identify at-risk patients, forecast disease and treatment patterns, and support complex clinical decisions to optimize patient care and hospital resources. Already, the AI team at SZI has developed data-driven tools touching nearly every corner of the hospital:

  • AI for rheumatic heart disease (RHD): In partnership with Children’s National cardiology leaders, including Craig Sable, M.D., the Uganda Heart Institute and Cincinnati Children’s Hospital, the AI team has developed an algorithm that can use low-cost, portable ultrasound imaging to detect RHD in children and young adults, a disease that takes nearly 400,000 lives annually in limited-resource countries. Early testing shows the AI platform has the same accuracy as a cardiologist in detecting RHD, paving the way for earlier treatment with life-saving antibiotics. This year, Children’s National physicians will be in Uganda, screening 200,000 children with local cardiology experts and AI technology.
  • Newborn screening for genetic conditions with mGene: Working with Rare Disease Institute clinicians and Chief of Genetics and Metabolism Debra Regier, M.D., the AI team has built technology to detect rare genetic disorders, using an algorithm and a smartphone camera to identify subtle changes in facial features. Tested on patients from over 30 countries and published in The Lancet Digital Health, the application helps screen children for advanced care when a geneticist may not be within reach. With funding from the National Institutes of Health, Children’s National and its research partners are piloting a newborn screening program in the Democratic Republic of the Congo.
  • Pediatric brain tumors: To improve and personalize the treatment decisions for children with brain tumors, Dr. Linguraru’s team is working with Brain Tumor Institute Director Roger Packer, M.D., the Gilbert Family Distinguished Professor of Neurofibromatosis, on algorithms that can characterize and measure brain tumors with unprecedented precision. The team recently won the International Pediatric Brain Tumor Segmentation Challenge, distinguishing the Children’s National algorithm as among the best in the world.
  • Ultra-low field magnetic resonance imaging (MRI): With a grant from the Bill & Melinda Gates Foundation, the AI team is working alongside Children’s Hospital Los Angeles, King’s College London and the UNITY Consortium to expand global brain imaging capacity. The consortium is helping clinicians in limited-resource countries improve the treatment of neonatal neurological conditions, using AI to boost the quality of ultra-low field MRI and expand access to this portable and more affordable imaging option.
  • Federated learning: Children’s National has collaborated with NVIDIA and other industry leaders to accelerate AI advances through federated learning. Under this approach, institutions share AI models rather than data, allowing them to collaborate without exposing patient information or being constrained by essential data-sharing restrictions. The SZI team was the only pediatric partner invited to join the largest federated learning project of its kind, studying the lungs of COVID-19 patients. Details were published in Nature Medicine.

Children’s National leads the way

Looking ahead, the Children’s National AI team is pursuing a wide range of advances in clinical care. To support patients treated at multiple clinics, they are developing systems to harmonize images from different scanners and protocols, such as MRI machines made by different manufacturers. Similar work is underway to analyze pathology samples from different institutions consistently.

Automation is also making care more efficient. For example, using data from 1 million chest X-rays, the team is collaborating with NVIDIA to develop a conversational digital assistant that will allow physicians to think through 14 possible diagnoses.

Dr. Linguraru says he and his colleagues are galvanized by the jarring statistic that one in three children with a rare disease dies before age 5. While well-implemented AI initiatives can change outcomes, he says the work must be done thoughtfully.

“In the future, patients will be evaluated by human clinicians and machines with extraordinary powers to diagnose illness and determine treatments,” Dr. Linguraru said. “Our team at Children’s National is leading conversations about the future of pediatric healthcare with a focus on safety, resource allocation and basic equity.”

Learn more about our AI initiatives

Innovation leaders at Children’s National Hospital are building a community of AI caregivers through educational and community-building events. At the inaugural Symposium on Artificial Intelligence in 2023 at the Children’s National Research & Innovation Campus, experts from Virginia Tech, JLABS, Food and Drug Administration, Pfizer, Oracle Health, NVIDIA, AWS Health and elsewhere laid out a vision for using data to advance pediatric medicine. The symposium will return on Sept. 6.

Dr. Linguraru is the program chair of MICCAI 2024, the top international meeting on medical image computing and computer-assisted intervention and the preeminent forum for disseminating AI developments in healthcare. The conference is an educational platform for scientists and clinicians dedicated to AI in medical imaging, with a focus on global health equity. It will take place for the first time in Africa on Oct. 6-10.

 

 

Newborn baby in a crib

Pioneering research center aims to revolutionize prenatal and neonatal health

  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
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Catherine Limperopoulos, Ph.D., was drawn to understanding the developing brain, examining how early adverse environments for a mother can impact the baby at birth and extend throughout its entire lifetime. She has widened her lens – and expanded her team – to create the new Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital.

“Despite the obvious connection between mothers and babies, we know that conventional medicine often addresses the two beings separately. We want to change that,” said Dr. Limperopoulos, who also directs the Developing Brain Institute. “Given the current trajectory of medicine toward precision care and advanced imaging, we thought this was the right moment to channel our talent and resources into understanding this delicate and highly dynamic relationship.”

Moving the field forward

Since its establishment in July 2023, the new research center has gained recognition through high-impact scientific publications, featuring noteworthy studies exploring the early phases of human development.

Dr. Limperopoulos has been at the forefront of groundbreaking research, directing attention to the consequences of maternal stress on the unborn baby and the placenta. In addition, under the guidance of Kevin Cook, Ph.D., investigators published a pivotal study on the correlation between pain experienced by premature infants in the Neonatal Intensive Care Unit and the associated risks of autism and developmental delays.

Another area of research has focused on understanding the impact of congenital heart disease (CHD) on prenatal brain development, given the altered blood flow to the brain caused by these conditions during this period of rapid development. Led by Josepheen De Asis-Cruz, M.D., Ph.D., a research team uncovered variations in the functional connectivity of the brains of infants with CHD. In parallel, Nickie Andescavage, M.D., and her team employed advanced imaging techniques to identify potential biomarkers in infants with CHD, holding promise for guiding improved diagnostics and postnatal care. Separately, she is investigating the impact of COVID-19 on fetal brain development.

In the months ahead, the team plans to concentrate its efforts on these areas and several others, including the impact of infectious disease, social determinants of health and protecting developing brains from the negative impacts of maternal stress, pre-eclampsia and other conditions prevalent among expectant mothers.

Assembling a team

Given its robust research plan and opportunities for collaboration, the center pulled together expertise from across the hospital’s faculty and has attracted new talent from across the country, including several prominent faculty members:

  • Katherine L. Wisner, M.S., M.D., has accumulated extensive knowledge on the impact of maternal stress on babies throughout her career, and her deep background in psychiatry made her a natural addition to the center. While Dr. Wisner conducts research into the urgent need to prioritize maternal mental health, she will also be treating mothers as part of the DC Mother-Baby Wellness Initiative — a novel program based at Children’s National that allows mothers to more seamlessly get care for themselves and participate in mother-infant play groups timed to align with their clinical appointments.
  • Catherine J. Stoodley, B.S., M.S., D.Phil., brings extensive research into the role of the cerebellum in cognitive development. Dr. Stoodley uses clinical studies, neuroimaging, neuromodulation and behavioral testing to investigate the functional anatomy of the part of the brain responsible for cognition.
  • Katherine M. Ottolini, M.D., attending neonatologist, is developing NICU THRIVE – a research program studying the effects of tailored nutrition on the developing newborn brain, including the impact of fortifying human milk with protein, fat and carbohydrates. With a grant from the Gerber Foundation, Dr. Ottolini is working to understand how personalized fortification for high-risk babies could help them grow.

Early accolades

The new center brings together award-winning talent. This includes Yao Wu, Ph.D., who recently earned the American Heart Association’s Outstanding Research in Pediatric Cardiology award for her groundbreaking work in CHD, particularly for her research on the role of altered placental function and neurodevelopmental outcomes in toddlers with CHD. Dr. Wu became the third Children’s National faculty member to earn the distinction, joining an honor roll that includes Dr. Limperopoulos and David Wessel, M.D., executive vice president and chief medical officer.

Interim Chief Academic Officer Catherine Bollard, M.D., M.B.Ch.B., said the cross-disciplinary collaboration now underway at the new center has the potential to make a dramatic impact on the field of neonatology and early child development. “This group epitomizes the Team Science approach that we work tirelessly to foster at Children’s National,” Dr. Bollard said. “Given their energetic start, we know these scientists and physicians are poised to tackle some of the toughest questions in maternal-fetal medicine and beyond, which will improve outcomes for our most fragile patients.”

Before and after pictures of the patient's improved gait

Next-generation genomics testing holds key to undiagnosed rare disease

Before and after pictures of the patient's improved gaitSeth Berger, M.D., Ph.D., felt the pull to dig deeper when he started reading the chart. An 11-year-old boy had an abnormal gait and couldn’t even walk in a straight line down the sidewalk to go trick-or-treating. Yet workups with neurology, orthopedics and an exome analysis of the patient’s genetic code did not provide a diagnosis. He had been getting worse for roughly three years.

With one of the largest clinical genetics departments in the country, Children’s National Hospital receives more than 10,000 visits a year from patients like this middle schooler. Often, they are children and caregivers who are searching for answers and follow-up support for diagnoses of genetic disorders, which impact so few people that only highly trained geneticists and genetic counselors can get to the root of the disorder.

“In genetics, we are finding layers of understanding. A negative clinical test is not always the final answer because the significance of variants can often be missed or misunderstood,” said Dr. Berger, a medical geneticist and principal investigator in the Center for Genetics Medicine Research at Children’s National. “It can take extensive research and a deep knowledge of the limits of certain tests to reach a diagnosis.”

The fine print

On page 4 of the patient’s genetics report, Dr. Berger found a reference to a pair of variants with no known clinical impact. Dr. Berger recognized that the genes referenced could affect proteins that drive potentially treatable neurological outcomes.

Dr. Berger ordered further testing, including biochemical testing of the patient’s blood and a phenylalanine loading challenge, a test that measures how the body metabolizes certain amino acids. With the results, he confirmed a recessive GCH1 deficiency in the patient was causing a condition called DOPA-responsive dystonia, a disorder that causes involuntary muscle contractions, tremors and uncontrolled movements. Laura Schiffman Tochen, M.D., director of the Movement Disorders Program at Children’s National, started the patient on levodopa-carbidopa — a drug combination used to treat Parkinson’s disease and other neurological disorders — and within two hours the boy showed improvement. His gait was almost normal.

Why we’re excited

Dr. Berger presents at conferences on this case and several other medical mysteries that he’s recently solved in his clinical practice and his role at the Pediatric Mendelian Genomics Research Center, a Children’s National program immersed in a federally funded research study to better understand how differences in genetic material can affect human health. As part of his work, he’s joined the GREGoR project (Genomic Research to Elucidate the Genetics of Rare Disease), which hopes to increase the number of genetic disorders where a cause can be identified. The elite genetics consortium includes nationally recognized research centers – the University of California at Irvine, Broad Institute, University of Washington, Baylor University, Stanford University, Invitae and Children’s National – which are working together to harness cutting-edge genomics sequencing capabilities. They hope to enroll thousands in their research, funded by the National Institutes of Health.

“It’s truly stunning what genetic sequencing can find. The outcomes can be life-changing,” said Dr. Berger. “These cases with life-altering diagnoses don’t come along every day, but when they do, they make the hunt to find answers all the more worthwhile.”