Diagnostic Imaging & Radiology

pregnant woman looking at sonogram

Babies with congenital heart disease display disrupted brain function before birth

pregnant woman looking at sonogram

In their study, the team at Children’s National Hospital found that specific brain regions become especially vulnerable to injury around 24 weeks of pregnancy when developing babies begin to have high energy demands and rapid neurovascular changes.

For the first time, researchers have found that babies born with congenital heart disease (CHD) have alterations to the emerging functional connectivity of their brains in utero. The changes are related to the subtype of their CHD and their oxygen status before they have lifesaving surgery to treat their cardiac malformation, according to new findings published in the American Heart Association’s Circulation Research.

In their study, the team at Children’s National Hospital found that specific brain regions become especially vulnerable to injury around 24 weeks of pregnancy when developing babies begin to have high energy demands and rapid neurovascular changes. That leaves certain parts of the brain, including the brainstem, more susceptible to injury from cardiac complications and poor circulation.

“We used a special type of magnetic resonance imaging to safely study the brains of these unborn babies, and we found that they have weakened connectivity in the deep grey structures, which are responsible for sensation, movement, alertness and other core functions,” said Josepheen De Asis-Cruz, M.D., Ph.D., assistant professor at the Developing Brain Institute at Children’s National and an author of the study. “This offers an important clue in utero to the type of care the babies will eventually need when they are born.”

The big picture

In the past decade, the survival rate for fetuses with CHD has greatly improved. About 80% of cases – even some of the most high-risk heart defects – can be successfully treated or palliated with surgery and survive. Yet Dr. Cruz said researchers are finding that the rates of poor neurodevelopmental outcomes are about the same. That’s why she and her colleagues are looking at what precisely may be injuring the brains of these newborns prior to surgery, offering a possible roadmap to interventions.

The fine print

The research team studied 107 healthy, low-risk pregnancies and 75 pregnancies known to be complicated by CHD. They used functional connectivity magnetic resonance imaging (fcMRI) to examine the emerging connections of the brains of unborn babies, given fcMRI’s unique ability to query the brain in a resting state when a patient is unable to respond to tasks. They also studied the oxygen saturation levels of the babies after they were born and then mapped all of this information to the type of CHD that they were diagnosed with.

“Our findings indicate that the compromised connectivity in the brains of CHD patients before delivery is linked to hypoxia after birth,” Cruz said. “There were important differences in the low- and high-risk CHD groups. Babies born with transposition of the great arteries or hypoplastic left heart syndrome – two of the most high-risk diagnoses – have notable changes in their brain function, which could someday be used as biomarkers to guide their care.”

What’s ahead

Researchers at Children’s National are working together, using a variety of modalities, to move toward precision imaging in utero to help predict a child’s neurodevelopmental outcomes. The ultimate goal: better interventions sooner.

“This work is foundational. As we fine-tune more techniques to identify babies at risk, we can understand how environmental, genetic and epigenetic factors impact brain development and guide care decisions,” said Catherine Limperopoulos, Ph.D., director of the Center for Prenatal, Neonatal & Maternal Health Research and a senior author of the paper. “We can imagine a day where we can offer pregnant mothers highly detailed and personalized information about their unborn baby, and individualized interventions that lead to healthier lifetimes.”

data science illustration

Federated learning: A solution to AI’s data-sharing challenges

data science illustration

Federated learning can solve data-sharing challenges, allowing nimble collaboration across institutions to drive medical advances using artificial intelligence (AI).

Federated learning can solve data-sharing challenges, allowing nimble collaboration across institutions to drive medical advances using artificial intelligence (AI), according to a new manuscript from 10 thought leaders in AI and machine learning in medicine.

In Health Informatics Journal, these leading experts on how technology is shaping medicine shared a conversation that they had at the Radiology Society of North America’s conference. They weighed challenges facing AI, including barriers to data sharing because of privacy rules that prevent the distribution of information to different institutions. With federated learning, models are shared – rather than data – allowing institutions to aggregate information and collaborate with a master model.

“Federated learning offers tremendous promise,” said Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor of Research and Innovation, principal investigator at the Sheikh Zayed Institute of Pediatric Surgical Innovation and senior author on the manuscript. “As a community of experts, we have found that federated learning allows us to move away from the challenges of sharing data in central repositories. Instead, we share the models, which can be designed to protect privacy by limiting what’s shared outside of any given institution.”

A champion of pediatric health, Linguraru wants to ensure that children are represented in the development of models that advance science and medicine. “Sharing data is even more crucial when there are few patients, such as in rare diseases or pediatric populations,” he said. “In general, healthcare data suffers from inequitable representation in our public health systems and services.”

Learn more here about the challenges and potential solutions from experts at Rhino Health, Johns Hopkins University School of Medicine, NVIDIA, University of Cambridge, Ben-Gurion University Israel, MD Anderson Cancer Center, Dana-Farber Cancer Institute and Children’s National Hospital.

Winners of the International Conference on Medical Image Computing and Computer Assisted Intervention

AI team wins international competition to measure pediatric brain tumors

Winners of the International Conference on Medical Image Computing and Computer Assisted Intervention
Children’s National Hospital scientists won first place in a global competition to use artificial intelligence (AI) to analyze pediatric brain tumor volumes, demonstrating the team’s ground-breaking advances in imaging and machine learning.

During the International Conference on Medical Image Computing and Computer Assisted Intervention (MICCAI), the Children’s National team demonstrated the most accurate algorithm to study the volume of brain tumors – the most common solid tumors affecting children and adolescents and a leading cause of disease-related death at this young age. The technology could someday help oncologists understand the extent of a patient’s disease, quantify the efficacy of treatments and predict patient outcomes.

“The Brain Tumor Segmentation Challenge inspires leaders in medical imaging and deep learning to try to solve some of the most vexing problems facing radiologists, oncologists, computer engineers and data scientists,” said 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. “I am honored that our team won, and I’m even more thrilled for our clinicians and their patients, who need us to keep moving forward to find new ways to treat pediatric brain tumors.”

Why we’re excited

With roughly 4,000 children diagnosed yearly, pediatric brain tumors are consistently the most common type of pediatric solid tumor, second only to leukemia in pediatric malignancies. At the urging of Linguraru and one of his peers at the Children’s Hospital of Philadelphia, pediatric data was included in the international competition for the first time, helping to ensure that children are represented in medical and technological advances.

The contest required participants to use data from multiple institutions and consortia to test competing methods fairly. The Children’s National team created a method to tap into the power of two types of imaging and machine learning: 3D convolutional neural network and 3D Vision Transformer-based deep learning models. They identified regions of the brain affected by tumors, made shrewd data-processing decisions driven by the team’s experience in AI for pediatric healthcare and achieved state-of-the-art results.

The competition drew 18 teams who are leaders from across the AI and machine learning community. The runner-up teams were from NVIDIA and the University of Electronic Science and Technology of China.

The big picture

“Children’s National has an all-star lineup, and I am thrilled to see our scientists recognized on an international stage,” said interim Executive Vice President and Chief Academic Officer Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer for Immunology Research. “As we work to attack brain tumors from multiple angles, we continue to show our exceptional ability to create new and better tools for diagnosing, imaging and treating these devastating tumors.”

Marius George Linguraru

Marius George Linguraru, D.Phil., M.A., M.Sc., named as Connor Family Professor of Research and Innovation

Marius George Linguraru

“Artificial Intelligence may be the greatest tool we have for improving the quality of and access to medical care for children, especially those most vulnerable to health system inequities,” said Dr. Linguraru. “This professorship will help me extend our leadership in this vital field. The tools and care strategies we develop will benefit children worldwide.”

Children’s National Hospital named Marius George Linguraru, D.Phil., M.A., M.Sc., as the Connor Family Professor of Research and Innovation at Children’s National Hospital.

Dr. Linguraru is a principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National. He directs the award-winning Precision Medicine Imaging Group. He is also a professor of Radiology and Pediatrics and secondary professor of Biomedical Engineering at George Washington University.

About the award

Dr. Linguraru joins a distinguished group of 42 Children’s National physicians and scientists who hold an endowed chair. Professorships at Children’s National support groundbreaking work on behalf of children and their families and foster new discoveries and innovations in pediatric medicine. These appointments carry prestige and honor that reflect the recipient’s achievements and donor’s forethought to advance and sustain knowledge.

Dr. Linguraru is a global leader in harnessing the power of quantitative imaging and machine learning to rapidly and positively impact children’s health. Dr. Linguraru and his team use artificial intelligence (AI) and digital technology innovations to improve access to healthcare and the understanding of rare and newborn diseases. Their work enables clinicians to deliver care faster, evaluate responses to treatments and prevent health complications. They have positioned Children’s National as an international leader in the development of pediatric AI to ensure equitable care for all children.

“Artificial Intelligence may be the greatest tool we have for improving the quality of and access to medical care for children, especially those most vulnerable to health system inequities,” said Dr. Linguraru. “This professorship will help me extend our leadership in this vital field. The tools and care strategies we develop will benefit children worldwide.”

About the donors

The Connor family, through their vision and generosity, are ensuring that Dr. Linguraru and future holders of this professorship will launch bold, new initiatives to rapidly advance the field of pediatric research and innovation, elevate our leadership and improve the lifetimes of children.

“We strongly believe in the power of academic entrepreneurship to improve the health and wellbeing of children,” said Ed and Chris Connor, who are longtime donors and members of the Children’s National community. “This endowment is our way of supporting Children’s National’s work in research and innovation and recognizing Dr. Linguraru’s international leadership in using AI to benefit child health.”

Attendees at the inaugural symposium on AI in Pediatric Health and Rare Diseases

AI: The “single greatest tool” for improving access to pediatric healthcare

Attendees at the inaugural symposium on AI in Pediatric Health and Rare Diseases

The daylong event drew experts from the Food and Drug Administration, Pfizer, Oracle Health, NVIDIA, AWS Health and elsewhere to start building a community aimed at using data for the advancement of pediatric medicine.

The future of pediatric medicine holds the promise of artificial intelligence (AI) that can help diagnose rare diseases, provide roadmaps for safer surgeries, tap into predictive technologies to guide individual treatment plans and shrink the distance between patients in rural areas and specialty care providers.

These and dozens of other innovations were contemplated as scientists came together at the inaugural symposium on AI in Pediatric Health and Rare Diseases, hosted by Children’s National Hospital and the Fralin Biomedical Research Institute at Virginia Tech. The daylong event drew experts from the Food and Drug Administration, Pfizer, Oracle Health, NVIDIA, AWS Health and elsewhere to start building a community aimed at using data for the advancement of pediatric medicine.

“AI is the single greatest tool for improving equity and access to health care,” said symposium host Marius George Linguraru, D.Phil., M.A., M.Sc., principal investigator at the Sheikh Zayed Institute for Pediatric Surgical Innovation. “As a population, kids are vastly underrepresented in scientific research and resulting treatments, but pediatric specialties can use AI to provide medical care to kids more efficiently, more quickly and more effectively.”

What they’re saying

Scientists shared their progress in building digital twins to predict surgical outcomes, enhancing visualization to increase the precision of delicate interventions, establishing data command centers to anticipate risks for fragile patients and more. Over two dozen speakers shared their vision for the future of medicine, augmented by the power of AI:

  • Keynote speaker Subha Madhavan, Ph.D., vice president and head of AI and machine learning at Pfizer, discussed various use cases and the potential to bring drugs to market faster using real-world evidence and AI. She saw promise for pediatrics. “This is probably the most engaging mission: children’s health and rare diseases,” she said. “It’s hard to find another mission that’s as compelling.”
  • Brandon J. Nelson, Ph.D., staff fellow in the Division of Imaging, Diagnostics and Software Reliability at the Food and Drug Administration, shared ways AI will improve diagnostic imaging and reduce radiation exposure to patients, using more advanced image reconstruction and denoising techniques. “That is really our key take-home message,” he said. “We can get what … appear as higher dose images, but with less dose.”
  • Daniel Donoho, M.D., a neurosurgeon at Children’s National, introduced the audience to the potential of “Smart ORs”: operating rooms where systems can ingest surgery video and provide feedback and skill assessments. “We have to transform the art of surgery into a measurable and improvable scientific practice,” he said.
  • Debra Regier, M.D., chief of Genetics and Metabolism at Children’s National, discussed how AI could be used to diagnose and treat rare diseases by conducting deep dives into genetics and studying dysmorphisms in patients’ faces. Already, Children’s National has designed an app – mGene – that measures facial features and provides a risk score to help anyone in general practice determine if a child has a genetic condition. “The untrained eye can stay the untrained eye, and the family can continue to have faith in their provider,” she said.

What’s next

Linguraru and others stressed the need to design AI for kids, rather than borrow it from adults, to ensure medicine meets their unique needs. He noted that scientists will need to solve challenges, such as the lack of data inherent in rare pediatric disorders and the simple fact that children grow. “Children are not mini-adults,” Linguraru said. “There are big changes in a child’s life.”

The landscape will require thoughtfulness. Naren Ramakrishnan, Ph.D., director of the Sanghani Center for Artificial Intelligence & Analytics at Virginia Tech and symposium co-host, said that scientists are heading into an era with a new incarnation of public-private partnerships, but many questions remain about how data will be shared and organizations will connect. “It is not going to be business as usual, but what is this new business?” he asked.

U.S. News Badges

Children’s National Hospital ranked #5 in the nation on U.S. News & World Report’s Best Children’s Hospitals Honor Roll

U.S. News BadgesChildren’s National Hospital in Washington, D.C., was ranked #5 in the nation on the U.S. News & World Report 2023-24 Best Children’s Hospitals annual rankings. This marks the seventh 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.

For the thirteenth straight year, Children’s National also ranked in all 10 specialty services, with eight specialties ranked in the top 10 nationally. In addition, the hospital was ranked best in the Mid-Atlantic for neonatology, cancer, neurology and neurosurgery.

“Even from a team that is now a fixture on the list of the very best children’s hospitals in the nation, these results are phenomenal,” said Kurt Newman, M.D., president and chief executive officer of Children’s National. “It takes a ton of dedication and sacrifice to provide the best care anywhere and I could not be prouder of the team. Their commitment to excellence is in their DNA and will continue long after I retire as CEO later this month.”

“Congratulations to the entire Children’s National team on these truly incredible results. They leave me further humbled by the opportunity to lead this exceptional organization and contribute to its continued success,” said Michelle Riley-Brown, MHA, FACHE, who becomes the new president and CEO of Children’s National on July 1. “I am deeply committed to fostering a culture of collaboration, empowering our talented teams and charting a bold path forward to provide best in class pediatric care. Our focus will always remain on the kids.”

“I am incredibly proud of Kurt and the entire team. These rankings help families know that when they come to Children’s National, they’re receiving the best care available in the country,” said Horacio Rozanski, chair of the board of directors of Children’s National. “I’m confident that the organization’s next leader, Michelle Riley-Brown, will continue to ensure Children’s National is always a destination for excellent care.”

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 17 years, U.S. News has provided information to help parents of sick children and their doctors find the best children’s hospital to treat their illness or condition,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Children’s hospitals that are on the Honor Roll transcend in providing exceptional 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 eight Children’s National specialty services that U.S. News ranked in the top 10 nationally are:

The other two specialties ranked among the top 50 were cardiology and heart surgery, and urology.

stressed pregnant mom

Pandemic stress reshapes the placentas of expectant moms

stressed pregnant mom

Elevated maternal stress during the COVID-19 pandemic changed the structure, texture and other qualities of the placenta in pregnant mothers.

Elevated maternal stress during the COVID-19 pandemic changed the structure, texture and other qualities of the placenta in pregnant mothers – a critical connection between mothers and their unborn babies – according to new research from the Developing Brain Institute at Children’s National Hospital.

Published in Scientific Reports, the findings spotlight the underappreciated link between the mental health of pregnant mothers and the health of the placenta – a critical organ that develops during pregnancy to nourish and protect babies. The long-term neurodevelopmental impact on their children is under investigation.

“During the pandemic, mothers were exposed to a litany of negative stressors including social distancing, fear of dying, financial insecurity and more,” said Catherine Limperopoulos, Ph.D., chief and director of the Developing Brain Institute, which led the research. “We now know that this vital organ was changed for many mothers, and it’s essential that we continue to investigate the impact this may have had on children who were born during this global public health crisis.”

The big picture

Dr. Limperopoulos’s team compared magnetic resonance imaging (MRI) of 165 women who were pregnant before March 2020 to 63 women who became pregnant during the pandemic. Those pregnant during the pandemic were not knowingly exposed to COVID-19, and they collectively scored significantly higher on questionnaires measuring stress and depression. They were recruited at Children’s National as part of a clinical trial aimed at reducing pregnant women’s elevated stress levels during the pandemic.

The placenta is a temporary organ that grows during pregnancy to provide oxygen, nutrients and immunological protection to babies, and its health is vital to the well-being of the developing fetus. The data showed key changes in how the placenta grew and developed among women pregnant during the pandemic, especially when compared to placental growth and development among women who were pregnant before the pandemic. Changes in placental development also were associated with the infant’s birth weight at delivery. Importantly, these changes seem to be connected to maternal stress and depression symptoms.

Taken as a whole, the findings suggest that the disturbances measured on placental development in the womb may influence the placenta’s ability to support fetal health and wellness. “We are continuing to follow up on these mother-baby dyads to determine the long-term functional significance of these placental changes in utero,” Dr. Limperopoulos said.

Studies have shown that the placenta adapts to negative changes in the maternal environment and mental health status, and disruptions in placental function impact infant brain development and children’s neurobehavior and temperament.

The patient benefit

Dr. Limperopoulos’s research studying childbirth amid the pandemic builds on her extensive work investigating the impact of maternal stress on unborn children, including its adverse effect on brain structure and biochemistry. She’s also working on treatments and interventions to better support new families. Her program, DC Mother-Baby Wellness, brings together community partners to provide wrap-around care to expectant and new moms with elevated scores for stress, anxiety and depression.

“When identified early, maternal stress is a modifiable risk factor that can be treated with psychotherapy, social support and other personalized, evidence-based interventions,” Dr. Limperopoulos said. “We look forward to continued research in this area to better understand the mechanisms behind these biological changes and the needs of mothers and children who are born during pandemics, natural disasters and other significantly stressful events.”

illustration of a brain

Inducing strokes in newborns to treat hemimegalencephaly

“The number one thing people are perplexed by is how well these babies recover and how they can only live with half a brain,” said Tayyba Anwar, M.D., neonatal neurologist and co-director of the Hemimegalencephaly Program at Children’s National Hospital. “People think if a child has half a brain that’s damaged or dysplastic, how are they functioning? But babies are so resilient. It still amazes me.”

The big picture

Children’s National experts have pioneered a novel approach of inducing strokes to stop seizures and improve neurodevelopmental outcomes in newborns under three months old with hemimegalencephaly (HME).

The procedure, called an endovascular embolic hemispherectomy, can be safely used to provide definitive treatment of HME-related epilepsy in neonates and young infants, according to a study in the Journal of NeuroInterventional Surgery.

Prior to this approach, the standard treatment was an anatomic hemispherectomy — surgical removal of the affected half of the brain. But infants had to be at least three months old to undergo such a complex surgery. Delaying surgery meant the persistent seizures compromised the development of the healthy half of the brain.

What they’re saying

In this video, Dr. Anwar and Panagiotis Kratimenos, M.D., Ph.D., neonatologist and co-director of Research in Neonatology at Children’s National, discuss the critically important neonatal care provided to babies who undergo endovascular embolic hemispherectomy and how protocols have evolved with each case to make this less invasive approach a feasible early alternative to surgical hemispherectomy.

Drs. Anwar and Kratimenos are part of the multidisciplinary team of neonatal neurologists, neurointerventional radiologistsneonatologists and neurosurgeons performing endovascular hemispherectomies.

Abstract Happy 2022 New Year greeting card with light bulb

The best of 2022 from Innovation District

Abstract Happy 2022 New Year greeting card with light bulbA clinical trial testing a new drug to increase growth in children with short stature. The first ever high-intensity focused ultrasound procedure on a pediatric patient with neurofibromatosis. A low dose gene therapy vector that restores the ability of injured muscle fibers to repair. These were among the most popular articles we published on Innovation District in 2022. Read on for our full top 10 list.

1. Vosoritide shows promise for children with certain genetic growth disorders

Preliminary results from a phase II clinical trial at Children’s National Hospital showed that a new drug, vosoritide, can increase growth in children with certain growth disorders. This was the first clinical trial in the world testing vosoritide in children with certain genetic causes of short stature.
(2 min. read)

2. Children’s National uses HIFU to perform first ever non-invasive brain tumor procedure

Children’s National Hospital successfully performed the first ever high-intensity focused ultrasound (HIFU) non-invasive procedure on a pediatric patient with neurofibromatosis. This was the youngest patient to undergo HIFU treatment in the world.
(3 min. read)

3. Gene therapy offers potential long-term treatment for limb-girdle muscular dystrophy 2B

Using a single injection of a low dose gene therapy vector, researchers at Children’s National restored the ability of injured muscle fibers to repair in a way that reduced muscle degeneration and enhanced the functioning of the diseased muscle.
(3 min. read)

4. Catherine Bollard, M.D., M.B.Ch.B., selected to lead global Cancer Grand Challenges team

A world-class team of researchers co-led by Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National, was selected to receive a $25m Cancer Grand Challenges award to tackle solid tumors in children.
(4 min. read)

5. New telehealth command center redefines hospital care

Children’s National opened a new telehealth command center that uses cutting-edge technology to keep continuous watch over children with critical heart disease. The center offers improved collaborative communication to better help predict and prevent major events, like cardiac arrest.
(2 min. read)

6. Monika Goyal, M.D., recognized as the first endowed chair of Women in Science and Health

Children’s National named Monika Goyal, M.D., M.S.C.E., associate chief of Emergency Medicine, as the first endowed chair of Women in Science and Health (WISH) for her outstanding contributions in biomedical research.
(2 min. read)

7. Brain tumor team performs first ever LIFU procedure on pediatric DIPG patient

A team at Children’s National performed the first treatment with sonodynamic therapy utilizing low intensity focused ultrasound (LIFU) and 5-aminolevulinic acid (5-ALA) medication on a pediatric patient. The treatment was done noninvasively through an intact skull.
(3 min. read)

8. COVID-19’s impact on pregnant women and their babies

In an editorial, Roberta L. DeBiasi, M.D., M.S., provided a comprehensive review of what is known about the harmful effects of SARS-CoV-2 infection in pregnant women themselves, the effects on their newborns, the negative impact on the placenta and what still is unknown amid the rapidly evolving field.
(2 min. read)

9. Staged surgical hybrid strategy changes outcome for baby born with HLHS

Doctors at Children’s National used a staged, hybrid cardiac surgical strategy to care for a patient who was born with hypoplastic left heart syndrome (HLHS) at 28-weeks-old. Hybrid heart procedures blend traditional surgery and a minimally invasive interventional, or catheter-based, procedure.
(4 min. read)

10. 2022: Pediatric colorectal and pelvic reconstructive surgery today

In a review article in Seminars in Pediatric Surgery, Marc Levitt, M.D., chief of the Division of Colorectal and Pelvic Reconstruction at Children’s National, discussed the history of pediatric colorectal and pelvic reconstructive surgery and described the key advances that have improved patients’ lives.
(11 min. read)

Hyperfine Swoop System

$1.6m grant to boost MRI access globally for maternal, child health

Researchers at Children’s National Hospital are investigating ways to bring more portable and accessible low-field magnetic resonance imaging (MRI) to parts of the world that lack access to this critical diagnostic tool, thanks to a grant from the Bill & Melinda Gates Foundation.

The nearly $1.6 million in funding will enable clinicians to better treat pediatric neurological conditions including ischemic brain injury, hydrocephalus, micro- and macrocephaly and more, using analysis tools that are designed to handle the loss in image quality and related challenges inherent to low-field MRI. The research brings together teams at Children’s National and Children’s Hospital Los Angeles — two organizations with extensive experience in designing processing software tools for pediatric brain MRI analysis and data enhancement.

The patient benefit

“For 30 years, MRI has primarily helped patients in high-income countries. Our team is thrilled by the prospect of expanding this powerful tool to patients coming from a wide range of nations, geographies and socioeconomic backgrounds,” said Marius George Linguraru, D.Phil., M.A., M.Sc., principal investigator at the Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI). “Low-field MRI comes with great advantages including portability at the point of care of patients, lower clinical costs and the elimination of sedation for young children.”

Linguraru and his long-time collaborator, Natasha Lepore, Ph.D., principal investigator at The Saban Research Institute at Children’s Hospital Los Angeles, will analyze data from the brains of children from birth for the maternal and child health studies. The MRI data analyzed will form the basis for future studies of children’s brain anatomy in health and disease.

The big picture

Through the new grant, researchers will develop a suite of tools to help clinicians better analyze data and images from low-field MRI systems. These systems already have been integrated into interventional and observational studies to help characterize early neurodevelopmental patterns and identify drivers of abnormal development. They are also evaluating the efficacy of maternal and infant-focused interventions aimed at improving neurodevelopmental outcomes.

Why we’re excited

At Children’s National, SZI has installed a Hyperfine Swoop system, and Linguraru’s team is creating image enhancement tools tailored to the unique challenges of low-field MRI. Chief among them, conventional processing tools developed over the past several decades remain incompatible with the low-field data and require new software to take full advantage of the diagnostic power of imaging.

The work brings together a prestigious international consortium of scientists and clinicians from around the world to harness the power of computing and expand the reach of diagnostic imaging. Lepore said the team is eager to bring modern medical imaging to parts of the world that have missed its many benefits.

“Children’s brain development in underserved areas can be affected by so many factors, like malnutrition or anemia,” Lepore said. “The software we will design for the Hyperfine scanners will improve research into these factors, so the optimal interventions can be designed. We are excited to bring our expertise to this important and timely project.”

animation showing MRI cardiac imaging

Advanced MRI hopes to improve outcomes for Fontan cardiac patients

animation showing MRI cardiac imaging

Chief of Cardiac Surgery Yves d’Udekem, M.D., calls this “fourth-dimensional imaging” that identifies if blood flows swiftly, smoothly, or is subjected to swirls or turbulences that impede the effectiveness of the flow.

Cardiac imaging specialists and cardiac surgeons at Children’s National Hospital are applying advanced magnetic resonance imaging visualization techniques to understand the intricacies of blood flow within the heart chambers of children with single ventricle heart defects like hypoplastic left heart syndrome (HLHS).

The data allows surgeons to make critical corrections to the atrioventricular valve — the valve between the atrium and ventricle of the heart — before a child undergoes the single ventricle procedure known as the Fontan.

Yves d’Udekem, M.D., chief of Cardiac Surgery at Children’s National, says that eliminating leakage of the atrioventricular valve before a child undergoes the Fontan may improve a child’s quality of life after Fontan and reduce the likelihood of heart failure, transplant or death long term.

The big picture

Patients with only one functioning pumping chamber, or ventricle, have been on the same treatment trajectory for decades. However, critical international efforts to collect and analyze long-term outcomes for patients with Fontan circulations have led surgeons like d’Udekem to rethink what quality of life and a positive outcome means for these patients. This includes patients in the Australia and New Zealand Fontan Registry founded by d’Udekem while at Royal Children’s Hospital in Australia.

Research based on data in the patient registries shows that atrioventricular valve leakage plays a critical role in the outcomes for patients with single ventricle defects. For children with Fontan circulation, significant leakage of this valve leads to worse outcomes.

Moving the field forward

Treatment decisions for children with single ventricle heart defects are often made based on commonly used heart imaging to determine the effect of valve leakage based on two limited, key variables: the size and the squeeze of the heart. However, this is a late effect and may not reflect the true impact on children with single ventricle hearts.

The team at Children’s National — including d’Udekem and Yue-Hin Loke, M.D., cardiac MRI specialist and director of the 3D Cardiac Visualization Laboratory — use cardiac MRI to measure the flow between heart chambers. Special software can measure abnormal flow and energy losses inside the heart, drawing on principles of physics and engineering.

“Dr. Loke not only gathers three-dimensional imaging of the heart through every heartbeat, he also gathers brand new types of colored imaging of blood flow itself, showing how effectively it is propelled by the heart,” says d’Udekem. “This ‘fourth-dimensional imaging’ identifies whether the blood flows swiftly, smoothly or whether it is subjected to swirls or turbulences that impede the effectiveness of the flow.”

Children’s National leads the way

Harnessing the visualization technology and analysis for clinical care of patients with single ventricle defects is relatively new in the United States, but it has become a vital part of the routine, clinical pre-Fontan evaluations at Children’s National.

Few locations in the United States have the mechanisms and expertise to study abnormal flow patterns in children with single ventricle defects. Children’s National collaborates with engineers to help parse the information into clear-cut takeaways for the clinical teams to use in their treatment planning.

Also, while other centers have access to this technology, not many centers have cardiac surgeons like d’Udekem who have an active interest in applying the key learnings from this data as quickly as possible to improve outcomes for patients.

Loke describes the collaboration at Children’s National as a “unique crossroads of clinical need and clinical interest to help these kids in very bold ways.”

What’s next

d’Udekem and Loke are engaged in a comprehensive project that analyzes the impact of atrioventricular valve leakage to ensure that the flow inside the heart is optimized before a Fontan procedure.

The research will map the efficiency of blood flow between the atrium and ventricle before surgery and after a surgical correction is made. The goal is to test the hypothesis that better atrioventricular circulation before Fontan can make a big difference for patients’ long-term quality of life and overall health.

Illustration of brain and brainwaves

Effective treatment for children with hemimegalencephaly

Illustration of brain and brainwaves

Anatomic or functional hemispherectomy are established neurosurgical treatment options and are recommended for effective seizure control and improved neurodevelopmental outcome in patients with HME.

Endovascular hemispherectomy can be safely used to provide definitive treatment of hemimegalencephaly (HME) related epilepsy in neonates and young infants when intraprocedural events are managed effectively, a new study finds.

The authors of the study, which published in the Journal of NeuroInterventional Surgery, add that this less invasive novel approach should be considered a feasible early alternative to surgical hemispherectomy.

Why it matters

Anatomic or functional hemispherectomy are established neurosurgical treatment options and are recommended for effective seizure control and improved neurodevelopmental outcome in patients with HME. Hemispherectomy in the neonate, however, is associated with high surgical risks and most neurosurgeons defer surgical hemispherectomy until the patient is at least 8 weeks old. This delay comes at a significant neurocognitive cost as the uncontrolled seizures during this time of deferred surgery have a deleterious effect on future neurocognitive outcome.

Why we’re excited

“The procedure we have developed, endovascular hemispherectomy by transarterial embolization, acutely stops seizures and this cessation of seizures has been sustained in each of the treated patients,” says Monica Pearl, M.D., director of the Neurointerventional Radiology Program at Children’s National Hospital and the study’s lead author.

This treatment option – performed early in life – provides hope and a better quality of life for these patients post procedure.

What’s been the hold-up in the field?

Currently, the only effective treatment option is hemispherectomy. With the patient population of neonates and young infants, hemispherectomy has a very high mortality and complication rate resulting in most neurosurgeons deferring treatment until at least 8 weeks. This leaves neonates and young infants without effective treatment options and on multiple antiseizure medications in an effort to control the seizures

How does this work move the field forward?

“Embolization provides a highly effective treatment option that acutely stops seizures during a time period of critical neurodevelopment and one in which traditional open neurosurgical procedures are not viable options,” Dr. Pearl says. “Specifically, we can consider and perform embolization in children as young as one or two weeks of age rather than waiting until at least 8 weeks of age. The impact of earlier intervention – acutely stopping the seizures, reducing the dose and number of antiseizure medications and avoiding more invasive surgical procedures (hemispherectomy, shunt placement) – appears to be dramatic in our recent series. We are conducting long term studies to assess this effect on neurodevelopmental outcome.”

How is Children’s National leading in this space?

Dr. Pearl and the late Taeung Chang, M.D., neurologist at Children’s National, pioneered this concept and treatment pathway. The multidisciplinary team is led by Dr. Pearl, who has performed all the embolization procedures (transarterial embolization/endovascular hemispherectomy) and Tayyba Anwar, M.D., Co-Director, Hemimegalencephaly Program at Children’s National Hospital. Our epilepsy team, neonatology team and neurosurgery team work collaboratively managing the patients before and after each procedure.

MRI

Building “digital twins” to test complicated surgeries

 

MRI

Syed Anwar, Ph.D., is developing self-supervised algorithms for medical imaging.

Syed Anwar, Ph.D., joins the growing AI initiative in the Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI) at Children’s National Hospital with extensive research experience in machine learning and medical imaging from the University of Engineering and Technology in Taxila, Pakistan, the University of Sheffield, U.K., and the University of Central Florida through the Fulbright Scholars Program. At Children’s National, he’s grateful for the proximity between researchers and clinicians as he studies federated learning and works to build “digital twins” that allow medical teams to test complicated surgical and treatment plans on infants with disorders including Pierre Robin Sequence. This rare congenital birth defect is characterized by an underdeveloped jaw, backward displacement of the tongue and upper airway obstruction. Anwar works alongside Marius George Linguraru, D.Phil., M.A., M.Sc., principal investigator at SZI, and the Precision Medical Imaging Lab to increase AI capacity in all areas of pediatric care at the hospital.

Q: What is the focus of your research work?

A: The main theme is a digital twin. It’s an engineering innovation that people have been using for some time, especially in manufacturing and aviation. For example, you can create a digital simulation of an airplane with a flight simulator. Now, people are starting to use the power of data-driven digital twins for medical applications.

I’m working to create a digital twin for infants born with Pierre Robin Sequence, where they need to have surgical interventions for improving the structure of the bones in the jaws. It includes a lot of clinical approaches, including surgery and ways to address apnea and food intake.

There are multiple areas of clinical expertise involved. With a digital twin, we will have a digital representation of the patient, and the surgeon, the radiologist and other clinicians can experiment with a proposed intervention before actually touching the patient.

Syed Anwar

Syed Anwar, Ph.D., joins the growing AI initiative in the Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI) at Children’s National Hospital.

Q: How else are you using your engineering background in your research?

A: Another part of my work is federated learning, which is a type of machine learning. In artificial intelligence, we want big data as the starting point to train our deep learning models. When studying children, this is not always possible because we have smaller data sets.

Federated learning is a tool that helps in these situations. Data is kept at a local site. We train a model to learn from all that data at the different sites. One benefit is that we don’t need to share the data, which is very useful for preserving patient privacy. But you can still apply deep learning models and develop AI solutions using the distributed data for improved clinical outcomes.

Q: What do you see as the main hurdles you have to overcome?

A: For all medical data, and particularly for kids, the amount of data we see in a children’s hospital is small, particularly for rare diseases.

The second hurdle is good, quality labels. For example, if you are doing tumor segmentation, you still need to have some ground rules from a radiologist showing which part of the image is the tumor.

These challenges come together in another focus of my research – self-supervised learning, meaning we can train a machine to learn from the data itself, without the labels or ground rules. From a machine learning point of view, I am in the process of developing self-supervised algorithms for medical imaging and in general for medical data. It’s an amazing time to be in this research area and to enable the translation of AI driven solutions for clinical workflows.

Q: What excites you about being at Children’s National and working at SZI?

A: I come from an engineering background, and my research area has been medical imaging for some time, mainly magnetic resonance imaging. Before coming here, I was working at a university in Pakistan, teaching machine learning and conducting research related to medical imaging and biomedical signal processing. But I was missing strong connections with people caring for patients at the hospital.

lung ct scan

With COVID-19, artificial intelligence performs well to study diseased lungs

lung ct scan

New research shows that artificial intelligence can be rapidly designed to study the lung images of COVID-19 patients.

Artificial intelligence can be rapidly designed to study the lung images of COVID-19 patients, opening the door to the development of platforms that can provide more timely and patient-specific medical interventions during outbreaks, according to research published this month in Medical Image Analysis.

The findings come as part of a global test of AI’s power, called the COVID-19 Lung CT Lesion Segmentation Challenge 2020. More than 2,000 international teams came together to train the power of machine learning and imaging on COVID-19, led by researchers at Children’s National Hospital, AI tech giant NVIDIA and the National Institutes of Health (NIH).

The bottom line

Many of the competing AI platforms were successfully trained to analyze lung lesions in COVID-19 patients and measure acute issues including lung thickening, effusions and other clinical findings. Ten leaders were named in the competition, which ran between November and December 2020. The datasets included patients with a range of ages and disease severity.

Yet work remains before AI could be implemented in a clinical setting. The AI models performed comparably to radiologists when analyzing data similar to what the algorithms had already encountered. However, the AI was less valuable when trained on fresh data from other sources during the testing phase, indicating that systems may need to study larger and more diverse data sets to meet their full potential. This is a challenge with AI that has been noted by others too.

What they’re saying

“These are the first steps in learning how we can quickly and accurately train AI for clinical use,” said Marius George Linguraru, D.Phil., M.A., M.Sc., principal investigator at the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National, who led the Grand Challenge Initiative. “The global interest in COVID-19 gave us a groundbreaking opportunity to address a health crisis, and multidisciplinary teams can now focus that interest and energy on developing better tools and methods.”

Holger Roth, senior applied research scientist at NVIDIA, said the challenge gave researchers around the world a shared platform for developing and evaluating AI algorithms to quickly detect and quantify COVID lesions from lung CT images. “These models help researchers visualize and measure COVID-specific lesions of infected patients and can facilitate timelier and patient-specific medical interventions to better treat COVID,” he said.

Moving the field forward

The organizers see great potential for clinical use. In areas with limited resources, AI could help triage patients, guide the use of therapeutics or provide diagnoses when expensive testing is unavailable. AI-defined standardization in clinical trials could also uniformly measure the effects of the countermeasures used against the disease.

Linguraru and his colleagues recommend more challenges, like the lung segmentation challenge, to develop AI applications in biomedical spaces that can test the functionality of these platforms and harness their potential. Open-source AI algorithms and public curated data, such as those offered through the COVID-19 Lung CT Lesion Segmentation Challenge 2020, are valuable resources for the scientific and clinical communities to work together on advancing healthcare.

“The optimal treatment of COVID-19 and other diseases hinges on the ability of clinicians to understand disease throughout populations – in both adults and children,” Linguraru said. “We are making significant progress with AI, but we must walk before we can run.”

echocardiogram

AI may revolutionize rheumatic heart disease early diagnosis

echocardiogram

Researchers at Children’s National Hospital have created a new artificial intelligence (AI) algorithm that promises to be as successful at detecting early signs of rheumatic heart disease (RHD) in color Doppler echocardiography clips as expert clinicians.

Researchers at Children’s National Hospital have created a new artificial intelligence (AI) algorithm that promises to be as successful at detecting early signs of rheumatic heart disease (RHD) in color Doppler echocardiography clips as expert clinicians. Even better, this novel model diagnoses this deadly heart condition from echocardiography images of varying quality — including from low-resource settings — a huge challenge that has delayed efforts to automate RHD diagnosis for children in these areas.

Why it matters

Current estimates are that 40.5 million people worldwide live with rheumatic heart disease, and that it kills 306,000 people every year. Most of those affected are children, adolescents and young adults under age 25.

Though widely eradicated in nations such as the United States, rheumatic fever remains prevalent in developing countries, including those in sub-Saharan Africa. Recent studies have shown that, if detected soon enough, a regular dose of penicillin may slow the development and damage caused by RHD. But it has to be detected.

The hold-up in the field

Diagnosing RHD requires an ultrasound image of the heart, known as an echocardiogram. However, ultrasound in general is very variable as an imaging modality. It is full of texture and noise, making it one of the most challenging to interpret visually. Specialists undergo significant training to read them correctly. However, in areas where RHD is rampant, people who can successfully read these images are few and far between. Making matters worse, the devices used in these low resource settings have their own levels of varying quality, especially when compared to what is available in a well-resourced hospital elsewhere.

The research team hypothesized that a novel, automated deep learning-based method might detect successfully diagnose RHD, which would allow for more diagnoses in areas where specialists are limited. However, to date, machine learning has struggled the same way the human eye does with noisy ultrasound images.

Children’s National leads the way

Using approaches that led to successful objective digital biometric analysis software for non-invasive screening of genetic disease, researchers at the Sheikh Zayed Institute for Pediatric Surgical Innovation, including medical imaging scientist Pooneh Roshanitabrizi, Ph.D., and Marius Linguraru, D.Phil., M.A., M.Sc., principal investigator, partnered with clinicians from Children’s National Hospital, including Craig Sable, M.D., associate chief of Cardiology and director of Echocardiography, and cardiology fellow Kelsey Brown, M.D., who are heavily involved in efforts to research, improve treatments and ultimately eliminate the deadly impacts of RHD in children. The collaborators also included cardiac surgeons from the Uganda Heart Institute and cardiologists from Cincinnati Children’s Hospital Medical Center.

Dr. Linguraru’s team of AI and imaging scientists spent hours working with cardiologists, including Dr. Sable, to truly understand how they approach and assess RHD from echocardiograms. Building the tool based on that knowledge is why this tool stands apart from other efforts to use machine-learning for this purpose. Orienting the approach to the clinical steps of diagnosis is what led to the very first deep learning algorithm that diagnoses mild RHD with similar success to the specialists themselves. After the platform was built, 2,136 echocardiograms from 591 children treated at the Uganda Heart Institute fed the learning algorithm.

What’s next

The team will continue to collect data points based on clinical imaging data to refine and validate the tool. Ultimately, researchers will look for a way that the algorithm can work directly with ultrasound/echocardiogram machines. For example, the program might be run through an app that sits on top of an ultrasound device and works on the same platform to communicate directly with it, right in the clinic. By putting the two technologies together, care providers on the ground will be able to diagnose mild cases and prescribe prophylactic treatments like penicillin in one visit.

The first outcomes from the program were showcased in a presentation by Dr. Roshanitabrizi at one of the biggest and most prestigious medical imaging and AI computing meetings — the 25th International Conference on Medical Image Computing and Computer Assisted Intervention (MICCAI).

AI chip illustration

How radiologists and data scientists can collaborate to advance AI in clinical practice

AI chip illustration

The scientific community continues to debate AI’s possibility of outperforming humans in specific tasks. In the context of the machine’s performance versus the clinician, Linguraru et al. argue that the community must consider social, psychological and economic contexts in addition to the medical implications to answer this puzzling question.

In a special report published in Radiology: Artificial Intelligence, a Children’s National Hospital expert and other institutions discussed a shared multidisciplinary vision to develop radiologic and medical imaging techniques through advanced quantitative imaging biomarkers and artificial intelligence (AI).

“AI algorithms can construct, reconstruct and interpret radiologic images, but they also have the potential to guide the scanner and optimize its parameters,” said Marius George Linguraru, D.Phil., M.A., M.Sc., principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National. “The acquisition and analysis of radiologic images is personalized, and radiologists and technologists adapt their approach to every patient based on their experience. AI can simplify this process and make it faster.”

The scientific community continues to debate AI’s possibility of outperforming humans in specific tasks. In the context of the machine’s performance versus the clinician, Linguraru et al. argue that the community must consider social, psychological and economic contexts in addition to the medical implications to answer this puzzling question.

Still, they believe that developing a useful radiologic AI system designed with the participation of radiologists could complement and possibly surpass human’s interpretation of the visuals.

Given AI’s potential applications, the authors encouraged radiologists to access many freely available resources to learn about machine learning, and radiomics to familiarize with basic concepts. Coursera, for example, can teach radiologists about convolutional neural networks and other techniques used by AI researchers.

Conversely, AI experts must reach out to radiologists and participate in public speaking events about their work. According to the researchers, during those engagement opportunities, clinicians understood the labor-saving benefits of automatic complex measurements on millions of images—something that they have been doing manually for years.

There are also hurdles around this quest of automation, which Linguraru et al. hope both fields can sort out by working together. A critical challenge that the experts mentioned was earning the trust of clinicians that are skeptical about the “black box” functionality of AI models, which makes it hard to understand and explain the behavior of a model.

Some questions, too, need answers on how to best leverage both human intelligence and AI by using human-in-the-loop where people train, tune, and test a particular algorithm, or AI in-the-loop where this different framing generates AI input and reflection in human systems.

“The key is to have a good scientific premise to adequately train and validate the algorithms and make them clinically useful. At that point, we can trust the box,” said Linguraru. “In radiology, we should focus on AI systems with radiologists in-the-loop, but also on training radiologists with AI in-the-loop, particularly as AI systems are getting smarter and learning to work better with radiologists.”

The experts also provided possible solutions to sharing large datasets, how to build datasets that allows robust investigations and how to improve the quality of a model that might be compared against human’s gold standard.

This special report is the second in a series of panel discussions hosted by the Radiological Society of North America and the Medical Image Computing and Computer Assisted Intervention Society. The discussion builds upon the first in the series “Machine Learning for Radiology from Challenges to Clinical Applications” that touched on how to incentivize annotators to participate in projects, the promotion of “team science” to address research questions and challenges, among other topics.

Drs. Katie Donnelly, Panagiotis Kratimenos, Rana Hamdy, Shayna Coburn and Brynn Marks

Five Children’s National Hospital faculty named to Society for Pediatric Research

Drs. Katie Donnelly, Panagiotis Kratimenos, Rana Hamdy, Shayna Coburn and Brynn Marks

The Society for Pediatric Research (SPR) announced five new members from Children’s National Hospital: Drs. Rana Hamdy, Panagiotis Kratimenos, Brynn Marks, Shayna Coburn and Katie Donnelly.

The Society for Pediatric Research (SPR) announced five new members from Children’s National Hospital. Established in 1929, SPR’s mission is to create a multi-disciplinary network of diverse researchers to improve child health.

Membership in SPR is a recognized honor in academic pediatrics. It requires nomination by academic peers and leaders as well as recognition of one’s role as an independent, productive child health researcher.

“I am so proud of our faculty and all that they have accomplished. I am thrilled that they have been recognized for their achievements,” said Beth A. Tarini, M.D., M.S., SPR president and associate director for the Center for Translational Research at Children’s National Hospital.

SPR 2021 active new members from Children’s National are:

    • Katie Donnelly, M.D., M.P.H., attending physician in the Emergency Department at Children’s National Hospital. She is the medical director for Safe Kids DC, an organization dedicated to preventing accidental injuries in children in Washington DC. Her personal research interest is in preventing firearm injuries in children and she is a member of Safer through Advocacy, Firearm Education and Research (SAFER), a multidisciplinary team dedicated to firearm injury prevention at Children’s National. She is also the medical director of the newly founded hospital-based violence intervention program at Children’s National and an associate professor of pediatrics and emergency medicine at The George Washington University.“To be recognized by my peers as a researcher with a significant contribution to our field is very validating. It also opens a world of potential collaborations with excellent scientists, which is very exciting!” said Dr. Donnelly. “I am grateful for the immense support offered to me by the Division of Emergency Medicine to complete the research I am passionate about, especially my mentor Monika Goyal.”
    • Panagiotis Kratimenos, M.D., Ph.D., newborn intensivist and neuroscientist at Children’s National. He studies mechanisms of brain injury in the neonate, intending to prevent its sequelae later in life. Dr. Kratimenos’ interest lies in identifying therapies to prevent or improve neurodevelopmental disabilities of sick newborns caused by prematurity and perinatal insults.“Being a member of SPR is a deep honor for me. SPR has always been a ‘mentorship home’ for me since I was a trainee and a member of the SPR junior section,” said Dr. Kratimenos. “A membership in the SPR allows us to access a very diverse, outstanding team of pediatric academicians and researchers who support the development of physician-scientists, honors excellence through prestigious grants and awards, and advocates for children at any level either locally, nationally, or internationally.”
    • Rana Hamdy, M.D., M.P.H., M.S.C.E., pediatric infectious diseases physician at Children’s National and Director of the Antimicrobial Stewardship Program. She is an assistant professor of pediatrics at George Washington University School of Medicine and Health Sciences. Her area of expertise focuses on the prevention and treatment of antimicrobial resistant infections and the promotion of good antimicrobial stewardship in inpatient and outpatient settings.“It’s an honor to be joining the Society for Pediatric Research and becoming part of this distinguished multidisciplinary network of pediatric researchers,” said Dr. Hamdy. “I look forward to the opportunity to meet and work with SPR members, make connections for future collaborations, as well as encourage trainees to pursue pediatric research through the opportunities that SPR offers.”
    • Shayna Coburn, Ph.D., director of Psychosocial Services in the Celiac Disease Program at Children’s National. She is a licensed psychologist specializing in coping and interpersonal relationships in chronic illness treatment, particularly for conditions involving specialized diets. She holds an appointment as assistant professor of psychiatry and behavioral sciences at The George Washington University School of Medicine and Health Sciences. Her work has focused on promoting effective doctor-patient communication, reducing healthcare disparities and supporting successful adherence across the developmental span of childhood and adolescence. She currently has a Career Development Award from National Institute of Diabetes and Digestive and Kidney Diseases to refine and test a group intervention designed to improve self-management and quality of life in teens with celiac disease.
      “I hope that my background as a psychologist researcher will help diversify SPR. As an SPR member, I hope to encourage more opportunities for training, awards, and other programs that would be inclusive of clinician researchers who may not hold a traditional medical degree,” said Dr. Coburn.
    • Brynn Marks, M.D., M.S.-H.P.Ed., endocrinologist at Children’s National. As a clinical and translational scientist her goal is to use unique personal experiences and training to optimize both patient and provider knowledge of and behaviors surrounding diabetes technologies thereby realizing the potential of diabetes technologies improve the lives and clinical outcomes of all people living with diabetes. Her experiences as a person living with Type 1 diabetes have undoubtedly shaped her clinical and research interests in diabetes management and medical education.
      “It is an honor to be accepted for membership in the Society for Pediatric Research,” said Dr. Marks.  “Being nominated and recognized by peers in this interprofessional pediatric research community will allow me networking and growth opportunities as I continue to advance my research career.”
x-ray of child with dislocated hip

Hip surveillance helps identify dislocations in children with cerebral palsy

x-ray of child with dislocated hip

Hip surveillance is a process used to monitor the hips closely and frequently, identifying the problems earlier.

Children with cerebral palsy (CP) have an increased risk for hip displacement. Hip displacement in children with CP can happen slowly over time and can be painful, but a hip surveillance program can prevent this. Hip surveillance is a process used to monitor the hips closely and frequently, identifying the problems earlier. It is an ongoing process that continues for every child until skeletal maturity.

“Every child with cerebral palsy should be referred for hip surveillance regardless of determination by the Gross Motor Function Classification System,” said Sean Tabaie, M.D., orthopaedic surgeon at Children’s National Hospital.

Dr. Tabaie created a hip surveillance manual for primary care providers who care for this patient population. In most cases, these patients are monitored and followed closely by their primary care team. Education material regarding hip surveillance, including the background knowledge, is often not available to those practitioners in a concise format. To successfully initiate a hip surveillance program, it is important to promote education and provide the appropriate materials to that group of practitioners.

“Our goal is to improve the care of children with cerebral palsy by decreasing the overall presentation of dislocated hips in our clinic settings and promote the appropriate timing of referrals for evaluation of hip subluxation secondary to cerebral palsy or neuromuscular conditions,” said Tabaie.

Download the Surveillance Guidelines for Children with Cerebral Palsy here.

x-ray of a fracture

Genetic disorders, including osteogenesis imperfecta, rarely mimic child abuse

x-rays of fractures

In suspected child abuse cases, pediatric specialists are often called for clinical consultations or subpoenaed to courtrooms to discuss unexplained fractures and hemorrhage. During routine clinical practice, Children’s National Hospital (CNH) geneticists, child abuse pediatricians, orthopedists and radiologists use a multidisciplinary, stepwise approach that differentiates genetic disorders from cases of suspected child abuse. These teams provide a clear process for when clinical and radiological review suffices versus cases when judicious use of genetic testing and biochemical testing should be considered.

Every year, approximately 675,000 children suffer abuse or neglect, and at least 1,700 die from abuse — one of the leading causes of childhood morbidity and mortality nationwide.

Ill-intended arguments regarding genetic disorders often reach the court. Out of the 7,000 known rare diseases, rare bone diseases constitute about 5% of the cases. When there are true genetic disorders like osteogenesis imperfecta (OI), a group of genetic disorders that cause fractures, orthopedic specialists help tremendously with diagnosis and treatment.  The Children’s National Bone Health Program specializes in caring for healthy children, children with genetic bone conditions, and children whose bones have been damaged from illness or poor nutrition. Their team of experts enhances treatment to meet the needs of each child help us provide the best possible care for children with a broad range of bone health conditions  When a rare condition that causes bone fragility is suspected, these teams work together to provide proper diagnosis and management.

“OI is a diagnosis that can be made clinically with the help of geneticists, radiologists and orthopedists,” said Tanya Hinds, M.D., a child abuse pediatrician at Children’s National. “Outside of the newborn period, multiple unexplained fractures in infants with radiologically normal bones is suspicious for child physical abuse, not OI.”

When these regional cases reach the courtroom, Children’s National pediatricians often serve as clinician-educators and expert witnesses. According to Children’s National experts, clinicians must share the best available medical practices in both the hospital and courtroom.  Unfortunately, in some cases around the country, a handful of expert medical witnesses provide unique and unsubstantiated opinions, sometimes claiming the presence of a rare genetic disorder as a cause of fracture or hemorrhage, when this has not been diagnosed by mainstream genetics specialists.

“On the part of expert witnesses, scientifically sound explanations versus unfounded hypotheses can influence outcomes in civil proceedings, which determine a child’s placement and criminal proceedings, which determine judgment on the perpetrators,” said Natasha Shur, M.D., medical geneticist at Children’s National, and Nathaniel Robin, M.D., professor and clinical genetics director at the University of Alabama in an editorial published in Current Opinion in Pediatrics.

Dr. Hinds works on behalf of children to provide the best and most comprehensive work-up in cases of unexplained fractures or hemorrhage. As a board-certified child abuse pediatrician, she is responsible for implementing the evidence-based practice guidelines of the American Academy of Pediatrics and other similar societies.  Dr. Hinds mentioned that it is possible to use medical history, physical examination and diagnostic testing to differentiate traumatic causes of fractures and subdural hematomas from genetic causes, a belief she states is held by the vast majority of child serving clinicians.

“In cases of suspected child abuse, a multidisciplinary group of clinicians at Children’s National routinely provide comprehensive and top-rate care and consider alternative explanations for fractures,” said Eglal Shalaby-Rana, M.D., a radiologist at Children’s National who has partnered with the hospital’s Child and Adolescent Protection Center team on these challenging cases since 1991. “A multidisciplinary team is crucial to the evaluation and often includes additional specialists such as pediatric radiology, trauma surgery, hematology and in some cases genetics.”

Further, these clinician-educators and researchers at Children’s National call for increased publication and use of consensus guidelines such as the consensus statement on abusive head trauma published in Pediatric Radiology in 2018.  “Consensus guidelines synthesize the best available medical evidence and should be the basis for both clinical practice and education offered in the courtroom,” said Drs. Shur, Hinds and Shalaby-Rana.

 Distinguishing child abuse from genetic disorders

Drs. Hinds, Shalaby-Rana and Shur have served as expert witnesses and in turn wanted to come together to help develop frameworks that share scientifically sound information with peers who might encounter spurious arguments in courtrooms regarding genetic disorders as an explanation for physical abuse and inflicted fractures. Their 2021 literature review, published in Current Opinion in Pediatrics, addresses some of these issues.

To help distinguish child physical abuse from a genetic disorder, Drs. Shur, Hinds and Shalaby-Rana  worked as a multidisciplinary team to highlight best practices in six instances when genetic disorders were raised as explanations for inflicted fractures or hemorrhage, including Elhers-Danlos syndrome (EDS), osteogenesis imperfecta (OI), Menkes Disease and Glutaric Acidemia type I. In some cases, these explanations could be reasonable but should be diagnosed using routine clinical and radiological review, and when indicated, genetic and biochemical testing.

For instance, EDS is a diagnosis that is sometimes erroneously used to explain multiple fractures in cases of suspected physical abuse and can be misused in courtrooms. The most common EDS type is hypermobile EDS, often found in late adolescence or early adulthood. In some cases, babies erroneously receive hypermobility exams, or clinicians perform hypermobility assessments on parents of children with unexplained fractures — neither practice is indicated. Instead, the Children’s National team points out that children should receive a medical evaluation using the standard guidelines set by the American Academic of Pediatrics, American College of Radiology and other professional societies.

Dr. Shur also collaborated with radiologists at Boston Children’s Hospital in a related review published in Pediatric Radiology. There, George et al. addressed the clinical and molecular diagnosis criteria for EDS to help radiologists prevent misdiagnosis and support clinicians when seeing patients with multiple fractures.

“It is disturbing that the unsubstantiated EDS infant bone fragility hypothesis continues to be advanced in civil and criminal child abuse proceedings when fractures are not part of the diagnosis criteria for EDS,” said George et al.

The clinicians noted that the Beighton score, which helps diagnose hypermobile EDS, is not intended for children younger than 8 years old. Additionally, since the score provides insufficient data, other EDS features must be present, such as skin findings and connective tissue abnormalities.

OI, known as ‘‘brittle bone disease,’’ is a group of disorders that rarely present only with unexplained fractures. The researchers emphasize that infants and children with mild OI do not present exclusively with multiple fractures, which are specific to physical abuse such as multiple, bilateral rib fractures and classic metaphyseal lesions. Drs. Shur, Hinds and Shalaby-Rana share that pediatric specialists could overcome the diagnostic challenges between OI and child abuse through the inclusion of a genetic team in some cases, during the medical evaluation while also considering various criteria, such as family history, physical examination and laboratory findings. Molecular testing may be required in some instances, but it cannot substitute traditional clinical and radiology evaluations, according to these clinician-researchers.

Similarly, while Menkes disease can present with intracranial hemorrhage and fractures like child physical abuse, there are other distinguishing characteristics unique to Menkes disease, such as hair and facial dysmorphism. In a third related case-review published in Pediatric Radiology, Shur, Hinds and Shalaby-Rana et al. emphasize that diagnostic difficulties may arises when a multidisciplinary evaluation is omitted. They call upon all clinicians to provide ethical testimony in civil or criminal proceedings and to continue to utilize a multidisciplinary approach during daily clinical practice.

Irresponsible testimony and predatory journals

According to George et al., in collaboration with Dr. Shur, there are flawed publications on EDS associated with infant bone fragility that do not follow the gold standard of the scientific community. They believe this hypothesis must be rejected by experts in the field of pediatric imaging to safeguard the scientific integrity of the discipline. The lack of scientific design, peer review process and transparency causes negative consequences in the courtroom and threatens the proper adjudication of cases of suspected child physical abuse.

“Irresponsible testimony increasingly enters medico-legal proceedings dealing with allegations of child abuse, and so-called expert witnesses regularly cite these deeply flawed publications — in addition to misquoting the medical literature, loosely interpreting medical findings, presenting fictitious findings, and excluding salient and widely accepted facts from consideration,” said George et al.

In these pieces of literature, our Children’s National multidisciplinary team members reviewed the best available evidence and their collective decades of patient experience to highlight standard processes, which differentiate child physical abuse as a cause of fractures and hemorrhage from rare disorders.  Rare does not mean mysterious, and with education and a multidisciplinary approach, every child can receive the best possible medical work-up and care, according to Drs. Shur, Hinds and Shalaby-Rana. They urge all physicians to share only mainstream clinical medicine in the courtroom to help ensure the best possible social outcomes for children and their families.

red flags for irresponsible testimony

US News badges

For fifth year in a row, Children’s National Hospital nationally ranked a top 10 children’s hospital

US News badges

Children’s National Hospital in Washington, D.C., was ranked in the top 10 nationally in the U.S. News & World Report 2021-22 Best Children’s Hospitals annual rankings. This marks the fifth straight year Children’s National has made the Honor Roll list, which ranks the top 10 children’s hospitals nationwide. In addition, its neonatology program, which provides newborn intensive care, ranked No.1 among all children’s hospitals for the fifth year in a row.

For the eleventh straight year, Children’s National also ranked in all 10 specialty services, with seven specialties ranked in the top 10.

“It is always spectacular to be named one of the nation’s best children’s hospitals, but this year more than ever,” says Kurt Newman, M.D., president and CEO of Children’s National. “Every member of our organization helped us achieve this level of excellence, and they did it while sacrificing so much in order to help our country respond to and recover from the COVID-19 pandemic.”

“When choosing a hospital for a sick child, many parents want specialized expertise, convenience and caring medical professionals,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “The Best Children’s Hospitals rankings have always highlighted hospitals that excel in specialized care. As the pandemic continues to affect travel, finding high-quality care close to home has never been more important.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals. The rankings recognize the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News. The top 10 scorers are awarded a distinction called the Honor Roll.

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.

Below are links to the seven Children’s National specialty services that U.S. News ranked in the top 10 nationally:

The other three specialties ranked among the top 50 were cardiology and heart surgerygastroenterology and gastro-intestinal surgery, and urology.