Neonatology
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.
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.”
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:
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.”
The child-sized, lightweight, wearable band enables at-home monitoring of patient vital signs.
As their infant daughter recovered from a life-threatening virus, tech executive Jonathan Baut and his wife lived in a constant state of alert for any signs of a change in her condition. That experience prompted Baut to look for an at-home vital signs monitoring solution for parents facing medical challenges at home.
He located the technology in Belgium and found the clinical support to advance the innovation at Children’s National Hospital, tapping into its leadership of a pediatric device consortium funded by the Food and Drug Administration (FDA), the Alliance for Pediatric Device Innovation (APDI).
The technology platform, called Gabi SmartCare, features a tiny, lightweight, wearable band made for children that enables at-home monitoring of a patient’s vital signs including oxygen saturation, and heart rate movement. It also supports the collection of other data about health conditions through health assessment.
Wireless home-based monitoring could reduce hospital stays while improving the transitional care provided to patients at home. It could also aid in the home care of chronically ill patients.
Recently, Gabi SmartCare received its FDA clearance as a class II device for monitoring use. Baut says the multifaceted support he received from Children’s National and APDI helped him achieve this critical milestone, including the hospital’s role as one of two U.S. clinical trial sites for the device.
He explained that the APDI team also connected him with experts in device trials, regulatory processes and reimbursement, which helped him better understand the U.S. market and the nuances of the pediatric continuum of care.
Physicians can remotely monitor patient’s oxygen saturation and heart rate movement.
As he focuses on getting his product into hospitals, Baut already has additional monitoring features in development for Gabi SmartCare including respiratory rate, actigraphy, skin auto-calibration and sleep phases.
Reliable at-home monitoring tools can expand a pediatric hospital’s capabilities to support patients beyond the hospital setting. They can also help reduce trips to the emergency room and reduce the stress and anxiety parents experience when providing home care. Helping to advance devices like these can deliver those benefits to patient families at Children’s National and beyond.
“The pandemic underscored a great need for technologies that improve remote monitoring for children,” said Kolaleh Eskandanian, Ph.D. M.B.A., P.M.P., Children’s National vice president and chief innovation officer and program director of APDI. “At-home monitoring devices enable remote and continuous surveillance of pediatric patients, ensuring timely intervention and optimal care delivery even in remote or resource-limited settings. These technologies are needed now, and even more during public health emergencies.”
Lyme disease is caused by the bacterium Borrelia burgdorferi.
Understanding the effects of Lyme disease on the developing fetal brain is essential to ensure timely prenatal and postnatal treatments to protect the fetus and newborn. In response to this need, Children’s National Hospital is leading a pilot study to establish the groundwork needed for a larger study to determine the effect of in utero exposure to Lyme disease on pregnancy and early childhood neurodevelopmental outcomes.
“Insect-borne illnesses have more than doubled during the last twenty years as a result of multiple factors including environmental changes,” says Sarah Mulkey, M.D., Ph.D., principal investigator of the study and prenatal-neonatal neurologist at Children’s National Hospital. Dr. Mulkey is the Co-Director of the Congenital Infection Program at Children’s National Hospital and has led a long-term child outcome study of the mosquito-borne Zika virus infection in pregnancy and impacts on child neurodevelopment. She is now adding Lyme disease to her work on pregnancy and child outcomes. Lyme disease is transmitted to humans via an infected tick and can be acquired throughout much of the United States. “Very little is known about Lyme disease in pregnancy and if and how it may affect the fetus and impact child neurodevelopment,” said Dr. Mulkey.
This pilot study is funded by the Clinical Trials Network for Lyme and other Tickborne Diseases (CTN), supported by the Steven and Alexandra Cohen Foundation. Roberta DeBiasi, M.D., M.S., division chief of infectious diseases at Children’s National, is a CTN Node Principal Investigator. The study team at Children’s National works closely with members of the CTN as well as other Lyme disease-focused organizations and nonprofits to make sure that the pilot study addresses Lyme disease advocates’ research priorities in a scientifically rigorous way. “This is an important opportunity for us to engage with advocacy groups and others who have been stressing the need for this type of research for decades,” says Meagan Williams, M.S.P.H., C.C.R.C., the senior research coordinator for the study. “We know how important it is to maintain a patient-centered and trauma-sensitive lens in our work with families impacted by Lyme disease during pregnancy. We’ve been working hard to build this study in a way that centers our participants’ lived experiences and focuses on the topics they find important. We’re very grateful to have the support of Lyme disease focused organizations and advocates as we embark on this study because they know and have expressed exactly where the gaps are in the literature. It’s our job to do the work to fill those gaps.”
This pilot study aims to build upon existing research, case studies and advocacy to assess developmental and other family impacts of Lyme disease exposure during pregnancy.
Especially as tick season begins in Washington, D.C., and surrounding regions, the investigators are determined to make sure that all eligible people diagnosed with Lyme disease during their pregnancy are aware of the study and have the opportunity to participate. Pregnant volunteers can sign up for participation in the study here. Volunteers may be eligible to join if they are currently pregnant, live in the U.S. or Canada and were diagnosed with Lyme disease during pregnancy or have post-treatment Lyme disease syndrome (PTLDS).
Anyone interested in sharing information about the study with their clients or colleagues may email mewilliams@childrensnational.org for access to the team’s recruitment toolkit that includes flyers, FAQs and other resources. “We have resources available for clinicians, public health professionals, friends and family members and anyone else who may be able to help us identify participants and spread the word about this important study,” Williams says.
The study is registered on ClinicalTrials.gov (NCT06026969). If you have questions about the study, please reach out to Meagan at mewilliams@childrensnational.org or email cnhlymestudy@gmail.com.
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.”
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.
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:
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.”
Children’s National experts 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. Monica Pearl, M.D., neurointerventional radiologist, and Panagiotis Kratimenos, M.D., Ph.D., neonatologist, discuss why having a multidisciplinary team skilled at this procedure is the reason we’re the only center in the world capable of providing this treatment.
Traditionally, when a baby is diagnosed with hemimegalencephaly (HME), doctors turn to a hemispherectomy at 3 months of age, which involves surgically removing half of a baby’s brain. At Children’s National Hospital, our doctors pioneered the endovascular embolic hemispherectomy, an approach using induced controlled strokes to eliminate the affected part of the brain, halting seizures. Monica Pearl, M.D., neurointerventional radiologist, and Tammy Tsuchida, M.D., Ph.D., neonatal neurologist, talk about this life-changing procedure.
When a family from Texas received a shocking diagnosis for their newborn daughter, they knew there was one place they needed to go – Children’s National Hospital in Washington, D.C. At birth, Angelique was diagnosed with a rare and devastating condition known as hemimegalencephaly (HME) which causes uncontrollable and frequent seizures. Monica Pearl, M.D., neurointerventional radiologist, and the team at Children’s National have pioneered an approach to treat HME, where they induce controlled strokes to eliminate the affected part of the brain, halting seizures in their tracks. They’re the only team in the world doing this work. Angelique’s parents knew the clock was ticking — every day they waited meant irreversible damage to their daughter’s developing brain.
The team found differences in the brains of both infants whose mothers were infected with COVID while pregnant, as well as those born to mothers who did not test positive for the virus.
Babies born during the COVID-19 pandemic have differences in the size of certain structures in the brain, compared to infants born before the pandemic, according to a new study led by researchers at Children’s National Hospital.
The team found differences in the brains of both infants whose mothers were infected with COVID while pregnant, as well as those born to mothers who did not test positive for the virus, according to the study published in Cerebral Cortex.
The findings suggest that exposure to the coronavirus and being pregnant during the pandemic could play a role in shaping infant brain development, said Nickie Andescavage, M.D., the first author of the paper and associate chief for the Developing Brain Institute at Children’s National.
The study’s authors looked at three groups of infants: 108 born before the pandemic; 47 exposed to COVID before birth; and 55 unexposed infants. In all cases, researchers performed magnetic resonance imaging (MRI) scans of the newborns’ brains during the first few weeks of life. The MRI scans, which are non-invasive and do not expose patients to radiation, provided 3D images of the brain, allowing doctors to calculate the volume of different areas.
Researchers found several differences in the brains of babies exposed to COVID. They had larger volumes of the gray matter that makes up the brain’s outermost layer, compared to the two other groups. In contrast, an inner area of the brain, known as deep gray matter, was smaller than in unexposed babies. These are areas that contain large numbers of neurons that generate and process signals throughout the brain. “Their brains formed differently if they were exposed to COVID,” said Dr. Andescavage, adding that “those exposed to COVID had unique signatures” in the brain.
Doctors also measured the depths of the folds in the babies’ brains – a way to determine how the brain is maturing during early development. Babies born to mothers who had COVID in pregnancy had deeper grooves in the frontal lobe, while babies born during the pandemic – even without being exposed to COVID – had increased folds and grooves throughout the brain, compared to babies born before the pandemic. “There was something about being born during the pandemic that changed how the brain developed,” Dr. Andescavage said.
The study authors can’t fully explain what caused the differences in brain development in these babies, Dr. Andescavage said. But other studies have linked maternal stress and depression to changes in the newborn brain. In a future study, Dr. Andescavage and her colleagues will examine the relationship between infant brain development and how stress and anxiety during the pandemic may have played a role in early development.
Because the babies in the study were just a few weeks old, researchers don’t know if their altered brain development will affect how they learn or behave. Researchers plan to follow the children until age 6, allowing them to observe whether pandemic-era babies hit key developmental milestones on time, such as walking, talking, holding a crayon and learning the alphabet.
Researchers have been worried about the effect of COVID on the fetus since the beginning of the pandemic. Studies show that babies exposed to COVID in the womb may experience developmental impacts, and research is underway to better understand long-term outcomes.
Although the coronavirus rarely crosses the placenta to infect the fetus directly, there are other ways maternal infection can influence the developing baby. Dr. Andescavage said inflammation is one potential harm to a developing baby. In addition, if a pregnant woman becomes so sick that the levels of oxygen in her blood fall significantly, that can deprive the fetus of oxygen, she added.
In recent decades, studies of large populations have found that maternal infections with influenza and other viruses increased the risk of serious problems in children even years later, including autism, attention deficit hyperactivity disorder and schizophrenia, although the reasons behind the association are not well understood. Technology may allow doctors to answer a number of questions about COVID and the infant brain.
“With advanced imaging and MRI, we’re in a position now to be able to understand how the babies are developing in ways we never previously could,” Dr. Andescavage said. “That will better allow us to identify the exposures that may be harmful, and at what times babies may be especially vulnerable, to better position us to promote maternal wellness. This, in turn, helps infant wellness.”
More than 80% of maternal deaths in the United States are preventable, particularly the nearly 1 in 4 maternal fatalities that are attributable to mental health disorders.
Painting a sobering picture, a research team led by Children’s National Hospital culled years of data demonstrating that maternal mental illness is an under-recognized contributor to the death of new mothers. They are calling for urgent action to address this public health crisis in the latest edition of JAMA Psychiatry.
Backed by dozens of peer-reviewed studies and health policy sources, the journal’s special communication comes as maternal mortality soars in the United States to as much as three times the rate of other high-income countries.
“The contribution of mental health conditions to the maternal morbidity and mortality crisis that we have in America is not widely recognized,” said Katherine L. Wisner, M.D., associate chief of Perinatal Mental Health and member of the Center for Prenatal, Neonatal & Maternal Health Research at Children’s National. “We need to bring this to the attention of the public and policymakers to demand action to address the mental health crisis that is contributing to the demise of mothers in America.”
The evidence review laid out the risks facing new mothers: More than 80% of maternal deaths in the United States are preventable, particularly the nearly 1 in 4 maternal fatalities that are attributable to mental health disorders. Overdose and other maternal mental health conditions are taking the lives of more than twice as many women as postpartum hemorrhage, the second leading cause of maternal death. For non-Hispanic Black mothers, the mortality rate is a striking 2.6 times higher than non-Hispanic White mothers.
Yet the research team found that recent national efforts to combat maternal mortality have failed to address maternal mental health as “the public health crisis that it represents.” Even methodologies to measure maternal health statistics are inconsistent, which challenges efforts to shape health policy.
In examining 30 recent studies and another 15 historical references, the team – which included Caitlin Murphy, MPA, PNP, research scientist at the Milken School of Public Health at George Washington University, and Megan Thomas, M.D., FACOG, obstetrician at the University of Kansas School of Medicine – found ample data to support the need to elevate maternal mental health as a priority. Some examples:
Even with these sobering statistics, Dr. Wisner says that only 20 percent of women are screened for depression postpartum. “Given that this is a time that many mothers have contact with healthcare professionals, it’s critically important that all mothers are screened and offered treatment,” she said. “Mental health is fundamental to health — of the mother, the child and the entire family.”
Dr. Wisner is board-certified in general and child psychiatry. Throughout her research career, she has conducted research on maternal-infant interactions and family health. She recently joined the new Center for Prenatal, Neonatal & Maternal Health Research because of its vision to improve outcomes for the entire family by understanding the relationship between mothers and their babies.
“Throughout my career, I have fought hard against these silos that try to lock psychiatry into certain age categories,” Dr. Wisner said. “At Children’s National, we have a huge interest in reunifying the family. We want to ensure that we’re caring for unborn babies, infants and toddlers, while focusing on maternal health and the family in its broader context.”
The Children’s National Research Institute released its 2022-2023 Academic Annual Report. In the report, a summary of the past academic year highlights the accomplishments of each of the institute’s research centers, provides research funding figures and exalts some of the institute’s biggest milestones.
The stories in the report are a testament to the hard work and dedication of everyone at the Children’s National Research Institute.
We celebrated five decades of leadership and mentorship of Naomi Luban, M.D., and her incredible accomplishments in the W@TCH program, which have been instrumental in shaping the future of pediatric research.
We also celebrated innovation, highlighting our recent FDA award to lead a pediatric device consortium, which recognizes our commitment to developing innovative medical devices that improve the lives of children.
Breakthroughs at the Research & Innovation Campus continued as our researchers worked tirelessly to develop new treatments and therapies that will transform the lives of children and families around the world.
Taking a look at the breakthroughs happening in our now six research centers, we spotlighted the following stories:
A pediatric neurologist who specializes in children with congenital heart disease, Dr. Licht initially came to this area of research as he considered ways to ensure children’s brains have adequate oxygen delivery during heart care, preserving neurological health and improving long-term outcomes.
Daniel J. Licht, M.D., joins Children’s National Hospital with a vision: He believes non-invasive devices built using biomedical optics – or instruments using light – can give clinicians invaluable information about how the brain and other organs are functioning.
A pediatric neurologist who specializes in children with congenital heart disease, Dr. Licht initially came to this area of research as he considered ways to ensure children’s brains have adequate oxygen delivery during heart care, preserving neurological health and improving long-term outcomes. He sees countless applications for using the properties of light in pediatric medicine.
Dr. Licht, whose name coincidentally also means “light” in German, is planning to establish a program for biomedical optics at Children’s National, built on the pillars of education, innovation and commercialization. He wants to tap into the resources of the Sheik Zayed Institute of Pediatric Surgical Innovation and expertise across the hospital. He is launching this effort as part of the new Center for Prenatal, Neonatal & Maternal Health Research.
Q: How can light be used diagnostically?
A: I believe that light is truly the future of biomedical devices, especially in children. Light can penetrate human tissues deeply, whether it’s muscle, liver or kidney. For example, you can put a light at the end of an endoscope and someday do virtual biopsies. It’s all a matter of understanding the properties of light, and how to manipulate light to give you the answers that you need. The applications are truly infinite.
Q: What has your initial work in neurology shown?
A: One of the instruments that we have developed can measure cerebral blood flow and quantitatively show the oxygen use of the brain. That’s important because it’s easy to measure oxygen delivery, but it’s hard to balance supply-and-demand without knowing the patient’s unique demand. We now have preclinical data and information from about 500 patients.
In terms of what’s ahead, many therapies today aren’t targeted to the individual, so Johnny’s brain-oxygen demand may not be the same as Sarah’s brain-oxygen demand, even if they both have congenital heart disease. As a patient waits for surgery, we also have found that the brain-oxygen demand increases, but if the demand is not met, this can lead to pre-operative brain injury. This technology could change the whole conversation about the timing of surgery. In addition, we can measure the brain-oxygen demand intraoperatively. We are finding that we can actually define the right perfusion strategy for each patient, rather than making uniform decisions for all patients with a shared diagnosis.
Lastly, beyond the operating room, we can use this technology for countless conditions. It would help with the treatment of almost any disease in the critical care unit, when we are using tools like ECMO (extracorporeal membrane oxygenation, a salvage technique), and we need to monitor a patient’s status. We can also use it to measure intracranial pressure. In very simple terms, if a child with a shunt comes into the emergency room with a headache, we can noninvasively measure the pressure and see how it’s changed without a head CT. We can decide who needs to go to the operating room – and who doesn’t – without radiation.
Q: How did your career bring you to this point?
A: My interest has always been in brain injury and kids with congenital heart disease. Years ago, I started out using MRI because it was the technology that was bright and shiny at the time. I was part of a team that developed an MRI sequence for measuring cerebral blood flow. We made some discoveries that indicated the culprit for brain injury was not the surgeries. Instead, there was something with the babies.
Unfortunately, with MRI, it’s a big, expensive instrument, and you have to take the baby to the machine for a single point-in-time measurement. So I started working with a physicist at the University of Pennsylvania to develop a way to measure the motion of particles, specifically red blood cells, to study cerebral blood flow. We found ways to use light, and this is what I hope to build and commercialize at Children’s National. By the end of my career, I hope to be able to say that we got this into clinical care.
The research team found that patients who are Black and Medicaid-insured patients experienced the greatest disparities in postoperative sepsis, a rare complication in which patients suffer from infection that can cause multi-organ failure.
Evaluating more than 5 million pediatric hospital stays nationwide, researchers found children who are Black, Hispanic or insured with Medicaid face a greater risk of health events after surgeries than white patients, according to a new study published in the journal Pediatrics.
“We looked at the data, and we calculated the risks,” said Kavita Parikh, M.D., MSHS, medical director of Quality & Safety Research, research director of the Division of Hospital Medicine and first author on the multi-institute study. “Despite decades of focus on eliminating medical errors, we know that children continue to suffer substantial harms in hospital settings, and our study highlights where children who are Black, Hispanic or insured with Medicaid are at the greatest risk.”
The study analyzed data from more than 5.2 million hospitalizations collected by the 2019 Kids’ Inpatient Database, a national repository of data on hospital stays. It includes a 10% sample of newborns and an 80% sample of other pediatric discharges from 4,000 U.S. hospitals. More than 80% of patients were younger than 1 year of age.
The research team found that patients who are Black and Medicaid-insured patients experienced the greatest disparities in postoperative sepsis, a rare complication in which patients suffer from infection that can cause multi-organ failure. Patients who are Hispanic experienced the greatest disparity in postoperative respiratory failure, a complication that can limit breathing and ventilation.
Plausible factors cited include structural racism in the U.S. healthcare system, clinician bias, insufficient cultural responsiveness, communication barriers and limited access to high-quality healthcare.
The study – “Disparities in Racial, Ethnic, and Payor Groups for Pediatric Safety Events in U.S. Hospitals” – is foundational in understanding what is happening among pediatric patients. Dr. Parikh said that researchers now must conduct further studies into these alarming disparities and qualitative work to understand drivers, with the action-oriented goal of developing interventions to improve patient safety in the hospital for all children.
“We brought together leaders in pediatric medicine, health policy and public health to analyze this data, and we are committed to taking the next steps to improve outcomes for pediatric patients,” Dr. Parikh said. “It will take more patient-centered work and research, resources and multifaceted strategies to resolve these worrying disparities for our pediatric patients nationwide.”
Explore some of the notable medical advancements and stories of bravery that defined 2023, showcasing the steadfast commitment of healthcare professionals at Children’s National Hospital and the resilient spirit of the children they support. Delve into our 2023 news highlights for more.
According to a study published in British Medical Journal Global Health, women who get COVID during pregnancy are nearly eight times more likely to die and face a significantly elevated risk of ICU admission and pneumonia. Sarah Mulkey, M.D., prenatal-neonatologist neurologist, discussed findings based on her work with pregnant women and their babies.
(Fortune)
A study led by Christopher Vaughan, Psy.D., pediatric neuropsychologist, suggests that — despite what many people may presume — getting kids back to school quickly is the best way to boost their chance for a rapid recovery after a concussion.
(CNN)
David Wessel, M.D., executive vice president, chief medical officer and physician-in-chief, explained that one reason parents were still having trouble getting their children beds in a pediatric hospital or a pediatric unit after the fall 2022 respiratory surge is that pediatric hospitals are paid less by insurance.
(CNN)
Anisha Abraham, M.D., M.P.H., chief of the Division of Adolescent and Young Adult Medicine, joined America’s Newsroom to discuss the impact social media access has had on children’s mental health.
(FOX News)
After 13-year-old Antonio was nearly killed outside his mom’s apartment, Children’s National Hospital went beyond treating his bullet wounds. Read how our Youth Violence Intervention Program team supported him and his family during his recovery.
(The Washington Post)
Erin and Jade Buckles underwent a successful separation at Children’s National Hospital. Nearly 20 years later they returned to meet with some of the medical staff who helped make it happen.
(Good Morning America)
Shilpa Patel, M.D., M.P.H., medical director of the Children’s National IMPACT DC Asthma Clinic, weighed in on a letter published in Annals of Allergy, Asthma & Immunology, asserting that the disparities in mortality due to asthma in the United States vary based on whether they occurred in a hospital, ethnicity or race and age of the patient.
(Healio)
After one family embarked on a perilous journey from Afghanistan through Mexico to the U.S.-Mexico border, they eventually secured entry to the U.S. where Karen Smith, M.D., medical director of Global Services, aided the family’s transition and provided their daughter with necessary immediate medical treatment.
(NPR)
With the number of young people shot by guns on the rise in the U.S., providers and staff at Children’s National Hospital are trying to break the cycle of violence. But it’s not just the physical wounds though that need treating: young victims may also need help getting back on the right track — whether that means enrolling in school, finding a new group of friends or getting a job.
(BBC News)
Callie, a 6-year-old diagnosed with diffuse intrinsic pontine glioma, was treated with low-intensity focused ultrasound (LIFU) at Children’s National Hospital and is the second child in the world to receive this treatment for a brain tumor. LIFU is an emerging technology that experts like Hasan Syed, M.D., and Adrianna Fonseca, M.D., are trialing to treat this fatal childhood brain tumor.
(The Washington Post)
The FDA approved a new genetic therapy, giving people with sickle cell disease new opportunities to eliminate their symptoms. David Jacobsohn, M.B.A., M.D., confirmed that Children’s National Hospital is one of the authorized treatment centers and talked about giving priority to the sickest patients if they are on Vertex’s list.
(The New York Times)
After the potential need for open-heart surgery threatened Caroline’s Nutcracker performance, Manan Desai, M.D., a cardiac surgeon, figured out a less invasive procedure to help reduce her recovery time so she could perform in time for the holidays.
(Good Morning America)
Advanced MRI visualization techniques to follow blood flow in the hearts of cardiac patients. Gene therapy for pediatric patients with Duchenne muscular dystrophy. 3D-printed casts for treating clubfoot. These were among the most popular articles we published on Innovation District in 2023. Read on for our full list.
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. The data allows surgeons to make critical corrections to the atrioventricular valve before a child undergoes the single ventricle procedure known as the Fontan.
(3 min. read)
Children’s National Hospital became the first pediatric hospital to administer a commercial dose of Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of pediatric patients with Duchenne muscular dystrophy (DMD). Elevidys is a one-time intravenous gene therapy that aims to delay or halt the progression of DMD by delivering a modified, functional version of dystrophin to muscle cells.
(2 min. read)
Researchers at Children’s National Hospital developed a pre-clinical model to test drugs and therapies for Becker Muscular Dystrophy (BMD), a debilitating neuromuscular disease that is growing in numbers and lacks treatment options. The work provides scientists with a much-needed method to identify, develop and de-risk drugs for patients with BMD.
(2 min. read)
In 2022, five newborns with life-threatening congenital heart disease affecting their heart rhythms were the first in the United States to receive a novel modified pacemaker generator to stabilize their heart rhythms within days of birth. Two of the five cases were cared for at Children’s National Hospital. In a follow-up article, the team at Children’s National shared that “early post-operative performance of this device has been excellent.”
(2 min. read)
Experts from the Food and Drug Administration, Pfizer, Oracle Health, NVIDIA, AWS Health and elsewhere came together to discuss how pediatric specialties can use AI to provide medical care to kids more efficiently, more quickly and more effectively 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.
(3 min. read)
The American Academy of Pediatrics (AAP) named a 2019 study led by clinician-researchers at Children’s National Hospital one of the 12 most influential Pediatric Emergency Medicine articles ever published in the journal Pediatrics. The findings showed that states with stricter gun laws and laws requiring universal background checks for gun purchases had lower firearm-related pediatric mortality rates but that more investigation was needed to better understand the impact of firearm legislation on pediatric mortality.
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Children’s National Hospital recently welcomed pediatric and adult colorectal surgeon Erin Teeple, M.D., to the Division of Colorectal and Pelvic Reconstruction. Dr. Teeple is the only person in the United States who is board-certified as both a pediatric surgeon and adult colorectal surgeon, uniquely positioning her to care for people with both acquired and congenital colorectal disease and help them transition from pediatric care to adult caregivers.
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The sickle cell team at Children’s National Hospital received a grant from the Founders Auxiliary Board to launch a first-of-its-kind, personalized holistic transformative program for the management of pain in sickle cell disease. The clinic uses an inter-disciplinary approach of hematology, psychology, psychiatry, anesthesiology/pain medicine, acupuncture, mindfulness, relaxation and aromatherapy services.
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Non-invasive prenatal testing using cell-free DNA (cfDNA) is currently offered to all pregnant women regardless of the fetal risk. In a study published in the American Journal of Obstetrics and Gynecology, researchers from Children’s National Hospital provided context and expert recommendations for maternal and fetal evaluation and management when cfDNA screening is positive for monosomy X or Turner Syndrome.
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While clubfoot is relatively common and the treatment is highly successful, the weekly visits required for Ponseti casting can be a significant burden on families. Researchers at Children’s National Hospital are looking for a way to relieve that burden with a new study that could eliminate the weekly visits with a series of 3D-printed casts that families can switch out at home.
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A new validated self-report tool provides researchers with a way to characterize the gender of research participants beyond their binary designated sex at birth. The multi-dimensional Gender Self-Report, developed using a community-driven approach and then scientifically validated, was outlined in a peer-reviewed article in the American Psychologist, a journal of the American Psychological Association.
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In a study published by Advances in Chronic Kidney Disease, a team at Children’s National Hospital reviewed cardiovascular and bone diseases in chronic kidney disease and end-stage kidney disease patients with a focus on pediatric issues and concerns.
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Catherine Limperopoulos, Ph.D., Yao Wu, Ph.D., and David Wessel, M.D.
Children’s National Hospital is celebrating a remarkable milestone as three of its faculty members have been honored over 15 years with the American Heart Association’s Outstanding Research in Pediatric Cardiology Award. Yao Wu, Ph.D., became the latest researcher to earn the accolade for her groundbreaking work into congenital heart disease (CHD).
A research faculty member with the newly established Center for Prenatal, Neonatal & Maternal Health Research, Dr. Wu received the award specifically for her studies on the role of altered placental function, measured by advanced in utero imaging, and neurodevelopmental outcomes in toddlers with CHD.
Honored at the association’s annual meeting in Philadelphia, Dr. Wu returned to Children’s National to warm congratulations from her colleagues who had previously won the award: David Wessel, M.D., executive vice president and chief medical officer, and Catherine Limperopoulos, Ph.D., director of the new center.
“I am thrilled to pass the baton to one of our own,” Dr. Limperopoulos said. “Dr. Wu’s recognition speaks to the outstanding and innovative research happening at Children’s National among junior faculty who are focusing on advancing our understanding of congenital heart disease and its long-term neurodevelopmental outcomes.”
The prestigious award represents more than individual accomplishments; it symbolizes three generations of mentorship and collaboration at the hospital. In 2007, Dr. Wessel joined Children’s National to enhance the care of newborns across specialty services by expanding programs and research, with a focus on critically ill newborns with heart disease. He recruited and mentored Dr. Limperopoulos in 2010, who became one of his research partners and creator of the hospital’s Center for Prenatal, Neonatal & Maternal Health Research. Dr. Limperopoulos, in turn, recruited and mentored Dr. Wu, providing her with the tools to conduct advanced imaging on in-utero brains and placentas, as well as the development of children with CHD.
“Each one of us is in different phases of our careers, yet we are connected by our deep interest in advancing cardiac care for critically ill newborns,” Dr. Wessel said. “In this collaborative environment, we learn from each other to improve entire lifetimes for our patients.”
Dr. Wu said she believes in sharing scientific developments for the advancement of the entire medical community. “It was an honor to be chosen to join this esteemed club, which has a relentless focus on improving health outcomes,” she said.
The award winners shared five collaborations published in leading journals to contribute to the ongoing dialogue in the field and the innovative work happening at Children’s National:
Premature infants exposed to pain while in the Neonatal Intensive Care Unit (NICU) are at greater risk for motor delays, language deficits and autism, even in the absence of structural brain injuries, according to findings from the new Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital.
Premature infants exposed to pain while in the Neonatal Intensive Care Unit (NICU) are at greater risk for motor delays, language deficits and autism, even in the absence of structural brain injuries, according to findings from the new Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital.
The research sheds light on the potential outcomes of routine medical interventions – such as heel pricks, venipunctures and IV placements – and correlates these skin breaks to changes in neurological connectivity in the preterm infants’ brains. Published in BMC Medicine, the work provides valuable insights about the far-reaching impact of early medical care.
“We know that premature babies are often exposed to repeated medical interventions, light, sound and other stimuli that they would not experience in utero, and we wanted to better understand the long-term effect,” said Kevin Cook, Ph.D., research faculty at the new center and an expert in fetal and neonatal neurology. “Through this study, we can see that early and repeated exposure to pain appears to alter brain development and put children at risk for poor neurodevelopmental outcomes.”
Globally, nearly 1 in 10 babies is born preterm, and the Children’s National team was particularly interested in the experience of those born “very” and “extremely” preterm, which is considered any delivery earlier than 32 and 28 weeks of gestation, respectively. While rates of prematurity have been relatively stable, survival rates of these babies have increased remarkably in recent decades, thanks to improved interventions and therapies for preterm infants. Yet neurodevelopmental challenges among these children persist, with noteworthy risks of autism and other neurological deficits.
At Children’s National, researchers are working to understand the mechanism behind those challenges. Given that the late second trimester and the third trimester are critical periods for brain development, the team wanted to study the effects of exposing babies to the world outside the womb early.
Dr. Cook and his colleagues collected resting-state functional MRI (fMRI) scans from 148 infants born at least four weeks prematurely, along with 99 infants born full term. The fMRI scans, uniquely suited for studying the resting state of the brain in non-responsive infants, revealed significant hyperconnectivity within the cerebellum, which coordinates muscle activity, and the limbic and paralimbic regions, which govern emotions, motivation and cognitive functions.
Notably, the hyperconnectivity correlated with the number of skin break procedures, including heel pricks, venipunctures and IV placements. When the children returned for developmental evaluations at 18 months, the skin breaks were strongly associated with an increased risk of autism and lower motor and language scores. The toddlers identified at risk for autism had an average of 118 skin breaks, which is significantly more than the average of 65 skin breaks in those who were not at risk.
Catherine Limperopoulos, Ph.D., director of the Center for Prenatal, Neonatal & Maternal Health Research, said the findings have important implications for understanding how painful NICU procedures can impact long-term outcomes and how physicians conceptualize the risks of care given to preterm babies. She and her team at the center recommend further research into managing pain in premature babies, especially given the limits of current options and the known risk of opioids.
“With this foundational study, we should consider ways to improve pain management for preterm infants and methods to better weigh the interventions used on these incredibly vulnerable patients,” Dr. Limperopoulos said. “Saving their lives is certainly the priority, and the quality of that life should also be forefront of our minds.”
To date, four patients have benefited from modified aquapheresis at Children’s National in both the PICU and the CICU.
The Division of Nephrology at Children’s National Hospital now offers modified aquapheresis for the smallest patients with acute kidney injury or chronic kidney disease in intensive care units. Aadil Kakajiwala, M.D., MSCI, director of Pediatric Acute Kidney Support Therapies, has been the leader in establishing modified aquapheresis at Children’s National. He joined the faculty at Children’s National in 2021 after completing his pediatric nephrology fellowship at the Children’s Hospital of Philadelphia and pediatric critical care medicine fellowship at Children’s National.
To date, four patients have benefited from modified aquapheresis at Children’s National in both the PICU and the CICU. Dialysis equipment designed for adults has filter set volumes as high as 165ml. Since implementing modified aquapheresis, the new equipment’s filter set volume is just 35ml, making it a great option for dialyzing small patients as low as 1.8kg. This limits blood exposure to the patient and overcomes the limitation of obtaining larger vascular access by using a PICC line.
Dr. Kakajiwala looks to continue training nephrologists, intensive care unit providers and staff across the hospital on modified aquapheresis. “This new offering allows us to offer dialysis to our smallest patients. By utilizing modified aquapheresis, we ensure simultaneous removal of waste products along with fluid removal during the therapy,” says Dr. Kakajiwala. He hopes to work on standardizing all forms of renal replacement therapy offered at Children’s National across all care providers.
The hospital has been working for years on improving screenings and support for perinatal mood and anxiety disorders.
Physician researchers at Children’s National Hospital secured a $1.8 million grant from the Agency for Healthcare Research and Quality (AHRQ) that will fund a Perinatal Mood and Anxiety Patient Safety Lab. Neonatologists, pediatric emergency medicine physicians, psychologists, computer scientists and the Perinatal Mood and Anxiety Disorder team from Children’s National will partner with systems engineers at Virginia Tech and Human Factors experts at MedStar Patient Safety Institute to set up a learning lab. The lab will improve mental health screening, referral and treatment of parents and caregivers at the hospital.
“After multi-month admission to our NICU, 45% of parents screen positive for depression. I can’t think of any other disorder or disease that screens positive at 45%. This can’t be ignored,” says Lamia Soghier, M.D. M.Ed., M.B.A., neonatologist and medical director of the Neonatal Intensive Care Unit (NICU) at Children’s National. “Our goal is to provide safe, comprehensive, point-of-care access to mental health services for caregivers of infants treated at our hospital. I can’t think of a better team on the cutting edge that’s qualified to tackle this issue.”
The new grant will tackle three major aims:
Researchers from the Center for Prenatal, Neonatal & Maternal Health Research and population health experts from the Child Health Advocacy Institute at Children’s National will also support this work.
“Children’s National is truly an innovator in this space,” says Dr. Soghier. “There are very few pediatric hospitals working with families to screen for mental health in the NICU, and fewer tackling the problem in the ED. Our team is dedicated to paving this path.”
The hospital has been working for years on improving screenings and support for perinatal mood and anxiety disorders, which was originally made possible by an investment from the A. James & Alice B. Clark Foundation to Children’s National aimed at providing families with greater access to mental health care and community resources. This new AHRQ grant will support the trajectory and goals of this work.
“There is no better model to study preterm injury than the human brain. Our team, along with the expertise of the scientific advisory board of the Raynor Cerebellum Project, will approach this project in multiple ways to extract the most possible information from the extremely precious human tissues,” says Dr. Kratimenos.
Children’s National Hospital has received $1 million in funding as part of the Raynor Cerebellum Project, whose mission is to improve the lives of those with cerebellar disease in seven to ten years. Panagiotis Kratimenos, M.D., Ph.D., principal investigator and Co-Director of Research in the Division of Neonatology at Children’s National, says the goal of this work is to understand the mechanisms of injury due to prematurity in human cerebellum and identify opportunities for intervention.
This project is unique because it focuses on postmortem human cerebellum, addressing the effect of the immune dysregulation of the mother during preterm labor. “We have established a large cohort of human term and preterm subjects and we will leverage cutting edge techniques to understand how the immune system of the mother during preterm labor shapes the cerebellum in a way that becomes more vulnerable to subsequent insults,” says Dr. Kratimenos.
“There is no better model to study preterm injury than the human brain. Our team, along with the expertise of the scientific advisory board of the Raynor Cerebellum Project, will approach this project in multiple ways to extract the most possible information from the extremely precious human tissues. This will give us insight into the real mechanisms of preterm birth induced injury due to maternal immune dysregulation,” says Dr. Kratimenos.
