Public Health News Archives

Children’s National in the News: 2023

January 18, 2024

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.

1. COVID during pregnancy dramatically increases the risk of complications and maternal death, large new study finds

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)

2. Rest isn’t necessarily best for concussion recovery in children, study says

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)

3. Pediatric hospital beds are in high demand for ailing children. Here’s why

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)

4. Anisha Abraham details impact of social media use on children: ‘True mental health crisis’

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)

5. Saving Antonio: Can a renowned hospital keep a boy from being shot again?

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)

6. Formerly conjoined twins reunite with doctors who separated them

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)

7. Asthma mortality rates differ by location, race/ethnicity, age

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)

8. How one Afghan family made the perilous journey across the U.S.-Mexico border

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)

9. When a child is shot, doctors must heal more than just bullet holes

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)

10. This 6-year-old is a pioneer in the quest to treat a deadly brain tumor

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)

11. F.D.A. approves sickle cell treatments, including one that uses CRISPR

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)

12. 6-year-old fulfils wish to dance in the Nutcracker

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)

The best of 2023 from Innovation District

December 27, 2023

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.

1. Advanced MRI hopes to improve outcomes for Fontan cardiac patients

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)

2. Children’s National gives first commercial dose of new FDA-approved gene therapy for Duchenne muscular dystrophy

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)

3. New model to treat Becker Muscular Dystrophy

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)

4. First infants in the U.S. with specially modified pacemakers show excellent early outcomes

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)

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

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)

6. AAP names Children’s National gun violence study one of the most influential articles ever published

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.
(2 min. read)

7. Why a colorectal transition program matters

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.
(3 min. read)

8. First-of-its-kind holistic program for managing pain in sickle cell disease

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.
(3 min read)

9. Recommendations for management of positive monosomy X on cell-free DNA screening

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.
(2 min. read)

10. Innovation in clubfoot management using 3D anatomical mapping

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.
(1 min. read)

11. Gender Self-Report seeks to capture the gender spectrum for broad research applications

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.
(2 min. read)

12. Cardiovascular and bone diseases in chronic kidney disease

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.
(1 min. read)

screenshot from Congenital Heart Initiative (CHI) Registry Participant Timeline

Congenital Heart Initiative beat recruitment goals, kicked off patient-engaged studies in year three

December 6, 2023

The Congenital Heart Initiative (CHI) is celebrating its third year as the first global patient-powered registry for adults with congenital heart disease (CHD). In 2023, the registry surpassed recruitment targets and launched a data intake process to allow researchers from around the world to submit proposals for patient-centered research and programs around the critical questions for adults with CHD who had their hearts repaired in childhood.

What it means

By recruiting over 4,600 participants in all 50 states and 37 countries, the CHI is now the largest patient-powered registry for adults with congenital heart disease. This is the first time researchers and clinicians have been able to access this type of robust data set to help them better understand and address the needs of people with CHD as they continue to age. Even better, the registry’s mechanisms allow for routine feedback and input about priorities directly from the growing patient population.

“Patient-centered research organizations, not providers or universities, have the greatest ability to lead this charge and lay the foundation for future breakthroughs. The inspiring efforts of all participants to date gives me hope that the next generation of advances is within reach,” says Matthew Lewis, M.D., an adult congenital heart specialist and CHI-RON site PI from Columbia University Medical Center.

An ongoing sub-study of CHI uses PCORnet®, the National Patient-Centered Clinical Research Network, to better understand how gaps in care impact the adult patient experience with CHD. The CHI-RON study (PCORI RD-2020C2-20347) fills in these gaps by exploring three distinct data types: patient-reported outcomes, health insurance claims and electronic health records. The effort is led by Children’s National Hospital and Louisiana Public Health Institute.

This year, CHI also launched some of its first studies, focused on pregnancy, health disparities and long-term health care follow-ups. The first academic manuscripts about these studies are expected to publish in the next year.

Why it matters

Although nearly 2 million adults in the United States are living with a congenital heart defect — more adults than children in fact — it’s been historically difficult to gather data on these conditions and to identify patient needs.

As children born with CHD become adults, they have a lot of worry and uncertainty about their limitations and abilities to achieve what might be considered common adult milestones. The research made possible by this registry and the mechanisms to communicate findings to both the participants and the larger clinical community will make a big difference in quality of life and hope to provide more answers to these important questions.

The CHI related meetings have allowed a space where patients and researchers can come together to discuss research priorities.

“Once you go and look at things from the patient’s point of view, there is no going back. It is going to be something that will redefine you as a researcher and a provider,” says Rohan D’Souza, M.D., a maternal-fetal medicine specialist who is an active participant in a PCORI-funded maternal health consortium focused on reducing maternal morbidity and mortality in CHD patients (PCORI EACB-23293).

The patient benefit

Additionally, because people with CHD live all over the United States and the world, it can be hard for them to connect with each other to share common questions and experiences with clinicians and each other. The patient-driven registry engages participants and hopes to help make greater connections between people who live with CHD. A key registry partner, the Adult Congenital Heart Association (ACHA), helps create opportunities, such as virtual Coffee Hours, for people in the registry to weigh in on research priorities and share feedback about CHI’s work.

“The ACHA Cafe was born out of a need for connection,” says Aliza Marlin, who founded and organized the café project on behalf of the ACHA. “A virtual social hour, coffee optional, gave the ACHD community a safe space to come together. Using it as a conduit to the Congenital Heart Initiative gave us an empowered voice in our own futures. It’s the perfect example of social connection leading to transformative possibilities.”

What’s next

Anitha John, M.D., medical director of the Washington Adult Congenital Heart Program and an adult congenital cardiologist at Children’s National Hospital who leads CHI, says that the future looks bright for the registry and the vital information it can provide.

The new data intake process launched this year, she adds, gives anyone with an interest the ability to submit ideas for new grants, projects and studies. The team will also continue to engage with registry participants, researchers and the centers who provide care for people with CHD, all with the goal of finding more answers to the key questions about how to accomplish specific goals, such as improving mental health, the health care transition and overall quality of life for adults with CHD. For more information on how to get involved, please email ACHDresearch@childrensnational.org.

Local context, health system integrations key to sustainable interventions after RHD diagnosis

November 2, 2023

Although entirely preventable, RHD, a disease of poverty and social disadvantage resulting in high morbidity and mortality, remains an ever-present burden in low- and middle-income countries, as well as rural, remote, marginalized and disenfranchised populations within high-income countries.

A rheumatic heart disease (RHD) work group convened by the National Heart, Lung, and Blood Institute (NHLBI) concludes that any priority intervention strategies to slow or stop late complications of RHD need to consider local contexts and should be integrated into health systems to meet the affected community’s needs in a sustainable way.

The group outlined priorities based on current available evidence to support the development and implementation of accessible, affordable and sustainable interventions in low-resource settings to manage RHD and its related complications.

Craig Sable, M.D., associate chief of Cardiology at Children’s National Hospital, served as a senior author on the recommendations, based on the work group findings.

Why it matters

Although entirely preventable, RHD, a disease of poverty and social disadvantage resulting in high morbidity and mortality, remains an ever-present burden in low- and middle-income countries, as well as rural, remote, marginalized and disenfranchised populations within high-income countries.

The NHLBI workshop sought to support RHD eradication efforts worldwide by:

Analyzing the current state of science
Identifying basic science and clinical research priorities

Each work group was assigned to review existing guidelines and research for different stages of the disease’s progression, which is now being published together as a set of five companion articles to raise the prioritization of RHD research and funding.

Moving the field forward

Due to the high prevalence of RHD in low- and middle-income countries, Dr. Sable’s work group focused on gaining a better understanding of the needs in the field from the five perspectives: people living with RHD, the community, healthcare providers, health systems and policymakers.

They identified several priorities and strategies, and they stressed that any interventional strategy, now or in the future, must be culturally safe and community-driven to ensure the creation of a locally and culturally relevant, sustainable continuum of care for people from historically marginalized populations.

What’s next

The authors emphasize that that over 300,000 deaths per year are the result of inadequate, underfunded and poorly integrated care. “Global vision and leadership to enact and implement available policies are needed to close large research gaps in all aspects at patient, health system and policy levels. Robust research and development are urgently needed to improve comprehensive tertiary care and ensure implementation of evidence-based interventions, while developing new innovations, technologies and interventions.”

You can read all the working group manuscripts, including this one: Tertiary Prevention and Treatment of Rheumatic Heart Disease: A National Heart, Lung, and Blood Institute Working Group Summary, in BMJ Global Health.

Learn more about the challenges of rheumatic heart disease in sub-Saharan Africa and other developing parts of the world through the Rheumatic Heart Disease microdocumentary series:

Earlier detection of cardiometabolic risk factors for kids may be possible through next generation biomarkers

October 31, 2023

The next generation of cardiometabolic biomarkers should pave the way for earlier detection of risk factors for conditions such as obesity, diabetes and heart disease in children.

American Heart Association statement finds potential future measures, reiterates importance of heart-healthy lifestyle from birth through adulthood.

The next generation of cardiometabolic biomarkers should pave the way for earlier detection of risk factors for conditions such as obesity, diabetes and heart disease in children, according to a new scientific statement from the American Heart Association published in the journal Circulation.

“The rising number of children with major risk factors for cardiometabolic conditions represents a potential tsunami of preventable disease for our healthcare system,” says the statement’s lead author Michele Mietus-Snyder, M.D., a preventive cardiologist and clinical research scientist at Children’s National Hospital. “But by the time a child is identified based on today’s clinical biomarkers, it’s often too late to reverse the disease trajectory.”

The big picture

The scientific statement included biomarkers that met three criteria:

Early and precise clinical detection of metabolic abnormalities before a child begins to show the current clinical signs such as high body mass index (BMI), blood pressure or cholesterol.
Mechanistic intervention targets providing immediate risk measures and giving clinicians new targets to personalize and optimize interventions.
Modifiable biomarkers that are capable of tracking progression toward or away from cardiometabolic health.

The statement’s identified biomarkers included measures of:

Epigenetic, or environmental, factors
Gut microbiome health
Small particle metabolites in the body
Different types of lipids and their impacts on cell membranes
Inflammation and inflammatory mediators

The authors proposed these biomarkers with the goal of “expanding awareness to include a whole new realm of biomarkers that precede the traditional risk factors we currently rely upon, such as BMI, blood pressure, cholesterol and blood sugar,” says Mietus-Snyder. “Ideally, these new biomarkers will be added to the array of measures used in clinical research to better assess their value for earlier identification and prevention of global patterns of cardiometabolic health and risk.”

Why it matters

The next generation cardiometabolic biomarkers outlined by the authors are all currently used in research studies and would need to be validated for clinical use. However, Mietus-Snyder notes that the data already collected from these biomarkers in research can make a difference in clinical practice by enhancing our understanding of the deep metabolic roots for children at risk.

Evidence reviewed in the statement shows the risk factors children are exposed to, even before birth, can set the stage for cardiovascular and metabolic health across the lifespan.

Interestingly, all the different factors reviewed have been found to alter the functioning of the mitochondria — the complex organelles responsible for producing the energy for the body that every cell and organ system in turn needs to function. Every class of biomarkers reviewed is also favorably influenced by heart-healthy nutrition, a simple but powerful tool known to improve mitochondrial function.

What’s next

Even as the new so-called ‘omic’ biomarkers reviewed in this statement are developed for clinical applications, there are things clinicians can do to optimize them and improve mitochondrial function, according to Mietus-Snyder.

Most important is to strengthen the collective dedication of care providers to removing the barriers that prevent people, especially expecting mothers and children, from living heart-healthy lifestyles.

We have long known lifestyle factors influence health. Even as complicated metabolic reasons for this are worked out, families can reset their metabolism by decreasing sedentary time and increasing activity, getting better and screen-free sleep, and eating more real foods, especially vegetables, fruits and whole grains, rich in fiber and nutrients, with fewer added sugars, chemicals, preservatives and trans fats. Clinicians can work with their patients to set goals in these areas.

“We know diet and lifestyle are effective to some degree for everyone but terribly underutilized. As clinicians, we have compelling reasons to re-dedicate ourselves to advocating for healthy lifestyle interventions with the families we serve and finding ways to help them implement them as early as possible. The evidence shows the sooner we can intervene for cardiometabolic health, the better.”

Using a multisystem approach to improve access to autism care in Washington, D.C.

October 5, 2023

Children with autism face significant barriers to accessing evaluations and intervention services.

An article in the journal Pediatrics reviews the outcomes from a collective, targeted advocacy approach to improving access to autism supports and resources for children and their families in Washington, D.C. The effort was led by Children’s National Hospital and engaged a multidisciplinary team from within the hospital and across a wide range of community sectors.

What this means

Children’s National and DC Autism Parents worked collaboratively with a coalition of organizations from the broader District of Columbia community to address some of the biggest challenges and barriers that prevent autistic children and their families from receiving the resources and support they need in the nation’s capital.

Why it matters

Children with autism face significant barriers to accessing evaluations and intervention services often because of confusing referral processes, lack of centralized coordination across organizations serving children with autism, insurance coverage gaps, multiyear waitlists for diagnostic services and limited provider knowledge about autism. Racism and systemic inequities also persist in autism care across the United States.

Long and growing wait times in autism diagnostic clinics and lack of centralized care coordination for autistic children are prevalent across the District of Columbia, and as a result, many children and families in the region continue to lack access to the support they need.

What’s unique

The study describes multiyear efforts (2017–2022) to improve autism care throughout the District of Columbia using a collective impact framework to unite organizations from different sectors. This approach features the creation of a common agenda (including defining goals and priorities), shared measurement, mutually reinforcing activities, continuous communication and infrastructure support.

Together, the group members sought to address barriers and overcome challenges at multiple levels of the healthcare system at the same time by focusing advocacy in three specific areas:

Infrastructure-building initiatives/system-level approaches.
Population- and community-level services to build capacity and connect providers and families to needed resources.
Direct services that provide innovative, gap-filling supports to children and families as a stopgap until the necessary supports can be more sustainably provided across the board.

Bottom line

While more work is needed to continue expanding the availability of needed services, the findings from this initial effort can inform the next steps in Washington, D.C., and serve as a model for a collective framework approach for autism services in other parts of the United States.

You can read the full study “A Multisystem Approach to Improving Autism Care” in the journal Pediatrics.

More information and resources about these autism initiatives can be found at:

New School-Friendly Health Systems Framework supports children’s health and learning

September 6, 2023

With school back in session and students, educators and health care workers still recovering from the COVID-19 pandemic, it is critical for the health and education sectors to align efforts and drive innovative programs and policy change efforts to address the needs of children.

With input from expert stakeholders, including pediatricians, educators, school administrators, school nurses, parents and community health experts, Children’s National led the development of a School-Friendly Health System (SFHS) framework that is based on lessons learned from nationally recognized baby-friendly hospitals and age-friendly health systems initiatives. Becoming school-friendly means that hospitals and health systems have made a commitment to actively help children achieve optimal health and reach their full academic potential.

The big picture

Given the fundamental interrelation of health and education, hospitals and health care organizations need a holistic framework to guide their school partnerships and other efforts to make a positive impact on patient and family engagement, health care costs, health outcomes and, ideally, patients’ learning experience. For hospitals and health systems, partnering with schools in their local service area will not only improve service delivery to the students receiving care from the health system but will help strengthen health outcomes and overall student performance.

Children’s National Hospital leads the way

Eight health care organizations and partners, led by Desiree de la Torre, M.P.H., M.B.A., Julia DeAngelo, M.P.H., and Danielle Dooley, M.D., M.Phil., of the Child Health Advocacy Institute (CHAI) through executive support of Tonya Kinlow, M.P.A., and Nathaniel Beers, M.D., M.P.A., at Children’s National and with support from RESOLVE (a Washington, D.C., -based nonprofit based specializing in collaborative process design), have formed to update the SFHS framework that was first released in 2021. The Collaborative includes interdisciplinary health care professionals who have been leading and coordinating early childhood and K-12 school health initiatives across the nation.

The SFHS framework includes five principles that reflect a broad range of competencies, practices, and policy positions that health care and school health experts and stakeholders view as emblematic of a SFHS – some already practiced, some aspirational:

A few significant updates to the framework include embedding the “All Touchpoints Approach” principle across the framework and adding an “Alignment” principle. Other changes included modifying the wording of the Accessibility and Accountability principles and adding a few new practices.

What’s next

The Collaborative is now focusing on developing tools to support implementation. Through securing additional funding, the Collaborative will evaluate how the adoption of the SFHS framework across diverse settings has strengthened the healthcare sector’s relationships with schools, students and families and ability to improve health and academic outcomes.

Read the full study at childrensnational.org/school-friendly For more information on the SFHS Learning Collaborative, contact School Partnerships@childrensnational.org.

Children’s National joins team to use AI to expand health knowledge in Kenya

August 16, 2023

Children’s National Hospital is joining a team of global health researchers to use large language models (LLMs) like ChatGPT to help Kenyan youth learn about their health and adopt lifestyles that may prevent cancer, diabetes and other non-communicable diseases.

The work, which is one of nearly 50 Grand Challenges Catalyzing Equitable Artificial Intelligence (AI) Use grants announced by the Bill & Melinda Gates Foundation, will harness the emerging power of AI to empower young people with information that they can carry through adulthood to reduce rates of unhealthy behaviors including physical inactivity, unhealthy diet and use of tobacco and alcohol.

“We are thrilled to be part of this effort to bring our AI expertise closer to young patients who would benefit dramatically from technology and health information,” said Marius George Linguraru, D.Phil., M.A., M.Sc., a co-principal investigator for the project, the Connor Family Professor in Research and Innovation at Children’s National and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation. “Using generative AI, we will build an application to enhance the knowledge, attitudes and healthy habits of Kenyan youth and use this as a foundation to improve health inequities around the globe.”

Why it matters

A lower middle-income country located on the east coast of Sub-Saharan Africa, Kenya is home to 50 million people and one of the continent’s fastest-growing economies. English is one of Kenya’s official languages, and the country has been recognized as a technology leader in Africa, with 82% of Kenyans having phone connectivity. Taken together, these factors make the country an ideal location to deploy an LLM-based platform designed to improve health information and attitudes.

The Gates Foundation selected this project from more than 1,300 grant applications. The nearly 50 funded projects are aimed at supporting low- and middle-income countries to harness the power of AI for good and help countries participate in the AI development process. The project’s findings will contribute to building an evidence base for testing LLMs that can fill wide gaps in access and equitable use of these tools. Each of the grants provides an opportunity to mitigate challenges experienced by communities, researchers and governments.

What’s next

The project development will be led by the National Cancer Institute of Kenya, with Linguraru and other global experts advising the effort from Kenyan institutions and Stanford University. Researchers plan to enroll youth from universities, shopping malls, markets, sporting events and other high-traffic locations. The study will look at participants’ risk factors and how their attitudes toward healthier lifestyles changed after engaging with the new LLM platform.

“The team is thrilled to be selected as one of the nearly 50 most promising AI proposals in the Gates Foundation Grand Challenge competition, and we look forward to seeing how our work can benefit the health of Kenyan youth,” said Dr. Martin Mwangi, principal investigator for the project and head of the Cancer Prevention and Control Directorate at the National Cancer Institute of Kenya. “If successful, we hope to share this model and the expertise we gain to expand health equity and knowledge to other regions.”

Pediatric hospitals underutilize systems to get at social challenges impacting health

July 31, 2023

Physicians treating hospitalized children rarely use a coding system established in 2015 for flagging social challenges and stressors that may be impacting patient health, according to new research from Children’s National Hospital.

Physicians treating hospitalized children rarely use a coding system established in 2015 for flagging social challenges and stressors that may be impacting patient health, according to new research from Children’s National Hospital published in Pediatrics. Known as social determinants of health (SDOH), these factors include food insecurity, homelessness and adverse childhood events like substance abuse at home, and they can greatly affect a child’s well-being.

“We only get so many touchpoints with our patients,” said Kaitlyn McQuistion, M.D., pediatric hospital medicine fellow at Children’s National and co-lead author of the paper. “Our research shows the screening itself provides valuable insight into our patients, making identification an important part of inpatient and post-discharge care. With this information, doctors can help families tap into social workers, community supports and other resources aimed at providing a more holistic approach to child health.”

The big picture

The American Academy of Pediatrics advises screening for unmet social needs and using the codes laid out in the International Classification of Disease, 10^th Revision (ICD-10), to flag risk factors for some of a hospital’s most vulnerable patients. In 2018, the American Hospital Association (AHA) clarified that the coding can be added by any healthcare professional accessing the chart, including nurses, social workers, case managers and physicians. The study looked at data from 4,000 hospitals in 48 states and the District of Columbia and found that use of the codes has remained low, even with the AHA’s clarification.

In practice, physicians know that screening and documentation are the essential first steps to help families find resources. Yet less than 2% of pediatric inpatients were coded as needing support. Most commonly, “problems related to upbringing” – a broad category indicating social needs and adverse childhood events – was used.

What’s ahead

Some providers are using these SDOH codes, or Z Codes, more often to address and improve health disparities. “Our mental health colleagues and those working with the Native American population, in particular, are using these tools more often to capture and disseminate critical information related to their patients’ social needs,” said Stacey Stokes, M.D., a hospitalist at Children’s National and co-lead author on the paper. “Their innovative approaches to address and improve health disparities may provide learning opportunities for institutions.”

The researchers said that more work needs to be done to take these successes to other populations, find ways to incentivize this work in billing and ensure that providers have community resources to address the needs that they uncover.

“The ultimate goal of this work is to identify patients with social needs affecting their health and connect them with resources,” said Kavita Parikh, M.D., director for the Research Division of Hospital Medicine. “There are many avenues to explore to find ways to better utilize this tool, including language learning models, improved training and stronger community resources.”

Survey: Teen parents with support report greater well-being

July 28, 2023

Teen parents in Washington, D.C., reported better physical and mental health when they had more social support, a community and people who understood their problems.

Teen parents in Washington, D.C., reported better physical and mental health when they had more social support, a community and people who understood their problems, according to a survey conducted as part of a novel city-wide program to bring together resources for young families.

“This should come as no surprise. Young parents say they do better – and their children do better – when they have the material and social supports they need,” said Yael Smiley, M.D., a pediatrician at Children’s National Hospital and lead author of research in BMC Public Health. “This work is the start of a roadmap to help young parents thrive.”

The big picture

Each year in D.C., 300 babies are born to teens, and the rate of births among teens ages 15 to 17 is higher than the national average. Dr. Smiley is a leader in a federally funded coalition run by D.C. Primary Care Association (DCPCA) to help support young parents called the D.C. Network for Expectant and Parenting Teens (DC NEXT!). This city-wide network brings together community partners to support teen mothers, fathers and their children.

A key feature of the program is its context team, a group of teen parents who provide insights into their experiences as young parents. They advise about research and support for their peers, and they also build a community and develop a platform to speak up about their issues. The context team recruited other parents to complete a well-being survey in 2022, which will guide programming and resources for families. An added benefit for the parents who participate in the context team: The survey data showed that a key facet of well-being for the young parents was feeling “like you were part of a group of friends or community” and having “someone who understood your problems.”

“The context team provides the type of community that the research shows is essential to the well-being of teen parents,” said Dr. Smiley. “Not only are the context team members helping us understand what teen parents need, but they are also helping each other thrive as young parents.”

The fine print

Among the findings of the survey of 107 teen parents in D.C.:

The most used resources were supplemental food programs – with 35% receiving Supplemental Nutrition Assistance Program (SNAP) benefits, 24% receiving support from the Special Supplemental Nutrition Program for Women, Infants and Children (WIC) and 18% accessing food pantries.
Also popular were Temporary Assistance for Needy Families (TANF), housing assistance and childcare.
Surprisingly, the largest group of respondents, 37%, reported receiving no resources.

Having higher social support was correlated with higher ratings for physical health, mental health and well-being. “With this research, we have even more evidence underscoring the need to connect young parents with programs and resources that will improve their well-being,” Smiley said. “I can’t wait to see what the leaders on the context team and in the DC NEXT! program do in the next phases of our work.”

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

June 21, 2023

Children’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:

Neonatology (No. 2), led by Division Chief Billie Lou Short, M.D.
Neurology and Neurosurgery (No. 3), led by Division Chiefs William D. Gaillard, M.D., and Robert F. Keating, M.D.
Cancer (No. 4), led by Division Chief Jeffrey S. Dome, M.D., Ph.D.
Nephrology (No. 6), led by Division Chief Marva Moxey-Mims, M.D.
Orthopedics (No. 7), led by Division Chief Matthew Oetgen, M.D., M.B.A.
Diabetes and Endocrinology (No. 7), led by Division Chief Andrew Dauber, M.D., MMSc.
Gastroenterology and GI surgery (No. 8), led by Division Chief Ian Leibowitz, M.D.
Pulmonology and Lung Surgery (tied for No. 9), led by Division Chief Anastassios Koumbourlis, M.D., M.P.H.

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

Using telehealth to study babies born to mothers infected with SARS-CoV-2

April 27, 2023

Continued advancements in telehealth methods to follow child neurodevelopment will help ensure robust child follow-up and inclusion of diverse cohorts.

Multiple studies have shown that SARS-CoV-2 infection can impact pregnant mothers and their fetuses but more research is needed to understand the long-term impact on the neurodevelopment of these children as they get older. Child neurodevelopmental evaluations are typically performed in-person. Since the COVID-19 pandemic began, the transition to telehealth methods was needed.

Continued advancements in telehealth methods to follow child neurodevelopment will help ensure robust child follow-up and inclusion of diverse cohorts, says a commentary in JAMA Network Open.

Why it matters

Commentary author Sarah Mulkey, M.D., prenatal-neonatal neurologist at Children’s National Hospital, highlights a new study that used a novel telehealth method to look for neurodevelopmental differences in infants ages 6-12 months born to mothers with SARS-CoV-2 infection compared to nonexposed infants of the same age and found no differences in neurodevelopment among the two cohorts. The study adapted a standardized assessment to a telehealth method.

“The results of this study provide needed reassurance to the many mothers who have experienced SARS-CoV-2 infection during pregnancy,” says Dr. Mulkey.

What’s been the hold up in the field?

“Developmental assessments that rely on observation of infants’ developmental skills can naturally make the transition to a telehealth platform,” says Sarah Mulkey, M.D., prenatal-neonatal neurologist at Children’s National Hospital and commentary author. “General movement assessment is an observation-based assessment of infants that can be captured by a parent or caregiver on video, and it has been used in neurodevelopmental outcomes studies of children after antenatal SARS-CoV-2 exposure.”

Moving the field forward

Child outcomes research can have improved enrollment and continuity of participant follow-up due to the availability of remote assessments. Neurodevelopmental tools are being developed that can be used on a telehealth platform or by parent recorded videos.

Researchers from the Children’s National Congenital Zika Virus Program have also developed telehealth-based methods for child outcome research that has been utilized in international Zika outcome studies funded by the Thrasher Research Fund and the NIH.

Read the full commentary, Use of Telehealth Methods to Track Infant Neurodevelopment After In Utero SARS-CoV-2 Exposure, in JAMA Network Open.

Most children diagnosed with Lyme disease recover fully after treatment

April 21, 2023

Lyme disease is caused by the bacterium Borrelia burgdorferi.

Experts found most children diagnosed with Lyme disease recovered within six months of completing antibiotic treatment, according to a new joint study published in Pediatric Research.

The study, which was supported through a partnership between Children’s National Research Institute (CNRI) at Children’s National Hospital and the National Institute of Allergy and Infectious Diseases (NIAID), also revealed that a notably small percentage of children took longer than six months to recover and experienced a significant impact on their daily functioning.

The big picture

This research studied the long-term outcomes of children with Lyme disease through a cross-sectional evaluation using validated surveys. The study collected survey responses from the parents of 102 children ages 5 to 18 years who had been diagnosed with Lyme disease between six months and 10 years before enrollment. Adolescents ages 10 to 18 years were also invited to complete adolescent-specific questionnaires.

According to these parent survey responses, the vast majority (87%) of fully recovered patients did so

within the initial 6-month period after completing antibiotic treatment, with approximately one third recovering within the first month post-treatment and an additional third recovering 1–3 months post-treatment. However, 13% of children who ultimately made a full recovery took longer to do so. At the time of study completion, 6% of children still experienced symptoms attributed to Lyme disease but only 1% experienced symptoms significant enough to impair daily functioning.

Why we’re excited

According to the authors, this study supports previous data showing an excellent overall prognosis for children with Lyme disease, which should help alleviate understandable parental stress associated with lingering non-specific symptoms among infected children.

“These findings can help clinicians manage families’ expectations about the varying post-treatment recovery times of pediatric Lyme disease patients,” says Roberta DeBiasi, M.D., chief of Infectious Diseases at Children’s National Hospital and one of the study’s authors. “It may also give parents relief in knowing that their child has an excellent chance of full recovery after treatment.”

What’s next

Common symptoms of Lyme disease include fever, headache, fatigue and a distinct skin rash called erythema migrans. Without treatment, the infection can spread to joints, the heart and the nervous system.

Antibiotic treatment resulting in full recovery is successful in most Lyme cases. For some, however, symptoms of pain, fatigue or difficulty thinking persist or return after antibiotic treatment. Symptoms that substantially reduce levels of activity and impact quality of life for more than six months after treatment are classified as post-treatment Lyme disease (PTLD) syndrome.

PTLD syndrome remains poorly understood in children and adults, and more research is needed to better understand these prolonged symptoms and identify treatment targets, according to the authors.

You can read the full study, Pediatric Lyme disease: systematic assessment of post-treatment symptoms and quality of life, in Pediatric Research.

Addressing COVID-19 vaccine hesitancy for parents and caregivers

April 21, 2023

A one-hour webinar delivered as a digital intervention can reduce parental COVID-19 vaccine hesitancy and parental uncertainty regarding vaccine information.

While vaccines have been proven to be highly effective in preventing COVID-19 infections and severe illness, the goal of achieving herd immunity remains unattainable without vaccinating children. Due to the prevalence of misinformation on the internet, Lauren Arrigoni, DNP, and Claire Boogaard, M.D., M.P.H., at Children’s National Hospital led a study, published in the Journal of Pediatric Health Care, that addresses parental hesitancy about giving their children COVID-19 vaccines. They found that a one-hour webinar delivered as a digital intervention can reduce parental COVID-19 vaccine hesitancy and parental uncertainty regarding vaccine information.

The big picture

The study used a pretest-posttest design with an adapted version of the Parental Attitudes about Childhood Vaccine (PACV) survey for COVID-19 vaccines. The webinar was streamed live and later posted to YouTube with survey QR codes and pre-survey and post-survey links in the video description. PACV data from the recorded webinar were collected over 4 weeks after the original webinar date. According to Dr. Arrigoni, the webinar itself was a collaborative work of a nurse and physicians to help provide scientific-based evidence for the general population of parents and caregivers. This project’s results have important implications for practice, as the researchers suggest that educational strategies have the potential to improve vaccination rates by increasing parental confidence in the COVID-19 vaccine. Using the webinar as a digital education strategy, the researchers also designed a dedicated section to empower parents to have conversations with their peers who may be hesitant about vaccines, even if they lack a medical background.

The patient benefit

While it was not possible to directly measure vaccination rates during this project due to time constraints, the researchers did find that parents who participated in the webinar felt more empowered and informed to make the decision to vaccinate their child. By addressing common parental concerns and providing accurate information, the webinar, as well as other types of digital interventions, could play a role in improving vaccination rates and ultimately contributing to truly ending the COVID-19 pandemic.

Why we’re excited

Overall, this study indicates that a webinar can effectively engage a large audience, educate them about vaccines and encourage conversations around vaccination.

“Vaccine hesitancy outreach can even begin before the vaccine is available,” says Dr. Arrigoni. “Applying these learned lessons can lead to more vaccinated individuals and would be an important step in battling parental vaccine hesitancy now and for future pandemics and viruses.”

You can read the full study, A Webinar to Improve Parental COVID-19 Vaccine Hesitancy, in the Journal of Pediatric Health Care.

Surgeon says treatment silos in childhood obesity need to end

March 8, 2023

An editorial in one of the top-ranked pediatric journals — Lancet Child & Adolescent Health — asserts that researchers studying obesity in children and adolescents should stop comparing medication versus bariatric surgery to see which is more effective.

Evan Nadler, M.D., director of the Child and Adolescent Weight-loss Surgery Program at Children’s National Hospital, writes that patients don’t want or need the answer to that question. Instead, the two treatment approaches should work together. He cites the field of oncology, where medicine and surgery work hand-in-hand as a comprehensive treatment plan that is individualized for each person’s unique condition.

Why it matters

“The obesity field has long been evaluating isolated interventions. There is a battle between those who favor medical therapy and those who favor surgical therapy,” Nadler writes. “Would it not make more sense for patients if we consider all available options until the desired weight loss is achieved rather than be a staunch advocate of the therapy that we personally like the best?”

According to Nadler, the American Academy of Pediatrics’ 15-year-process to release their first guidelines for treatment of childhood obesity is a consequence of these same silos.

“If medical and surgical oncologists figured out how to work together decades ago, why is it taking so long for practitioners who treat obesity to join forces and use combination therapy for their patients?”

Read the editorial in the Lancet Child & Adolescent Health.

Gender Self-Report seeks to capture the gender spectrum for broad research applications

January 31, 2023

A new validated self-report tool called the Gender Self-Report provides researchers 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, is outlined in a peer-reviewed article in the American Psychologist, a journal of the American Psychological Association.

The big picture

“This is the first broadly validated tool that allows us to measure inner gender experience across a large group of people in a rigorous way that doesn’t require a person to understand specialized gender-related terms,” says John Strang, Psy.D., who directs the Gender and Autism Program at Children’s National Hospital. He co-authored the journal article about the measure and co-led the initiative to develop it. “Typical gender assessments are fixed check boxes, which is problematic for capturing gender in people who are not familiar with many of the self-descriptors, which vary in their use and meaning.”

Strang notes that even open-field gender assessments can be problematic for people who experience gender diversity but are not aware of nuanced gender-related language.

Why it matters

This new gender characterization and inclusion method will allow researchers from a broad array of fields (e.g., social sciences, medicine, education) to model their participants’ inner gender experience more equitably in research. The resulting studies will be able to provide deeper understanding of how a person’s gender can play a role in study outcomes.

Senior author and statistical lead for the project, Ji Seung Yang, Ph.D., from University of Maryland Department of Human Development and Quantitative Methodology, foresees this tool as an important addition to the research toolkit for people studying neuroimaging, genetics and any other research that requires a more accurate and detailed picture of an individual’s gender experience.

What’s unique

The Gender Self-Report tool is the first of its kind to be developed and validated by researchers together with the gender diverse and neurodiverse communities directly. These efforts align with work in many fields of clinical research to ensure that study findings reflect the insights and experiences of the people who are being studied, rather than simply capturing the researcher’s external perspective of those participants.

The tool is appropriate for use by youth (as young as 10 years of age) and adults, gender diverse and cisgender individuals, and non-autistic and autistic people. The focus on inclusivity for autistic people is in keeping with the common intersection of autism and gender diversity (i.e., 11% of gender-diverse people are estimated to be autistic).

Gregory Wallace, Ph.D., co-author of the measure and associate professor in the Department of Speech, Language & Hearing Sciences at The George Washington University, calls the tool a “game changer for any research that needs to understand specifics about how gender experience can impact health-related or developmental differences.”

The Gender Self-Report: A Multidimensional Gender Characterization Tool for Gender-Diverse and Cisgender Youth and Adults, appears in the American Psychologist.

The role of pediatric cardiologists in addressing maternal health disparities

January 13, 2023

Black women are two to three times more likely to die from pregnancy-related complications. Most of the risk factors for these complications are cardiac in origin and preventable.

Pediatric cardiologists can and should work alongside other specialties to address the epidemic of maternal mortality that disproportionately affects Black women in the United States, says Annette Ansong, M.D., medical director of outpatient cardiology at Children’s National Hospital.

As co-chair of the Women and Children Committee of the Association of Black Cardiologists Inc., (ABC) Ansong says that cardiologists, especially pediatric cardiologists, have a role to play because “before they are Black women, they are Black girls.”

She talked about the impact of these health disparities and how cardiologists can play a role in addressing them at the American Heart Association Scientific Sessions in November 2022.

Why it matters

Dr. Ansong says that Black women are two to three times more likely to die from pregnancy-related complications. Most of the risk factors for these complications are cardiac in origin and preventable. Furthermore, many of these cardiovascular risk factors, such as obesity, start in youth and some even before birth. For example, children of pre-eclamptic moms have a higher risk of cardiovascular disease in the future, too.

How cardiologists can help

Pediatric cardiologists can be more proactive at helping Black girls grow up into strong, healthy Black women by making sure they are heart-healthy from a young age. That includes advocating for exercise, eating well and exploring innovative ways to encourage those habits.

Dr. Ansong says she makes a point to closely follow children based on the heart health history of their mothers—for example, “if mom had pre-eclampsia, I need to be keeping a closer eye on that child’s blood pressure” —to allow for early intervention and potentially prevent some devastating negative outcomes later in life.

Pediatricians and other specialists can also work with maternal-fetal medicine and other specialties to advocate for better tools to monitor women with pre-existing heart-related risk factors. This might include supporting efforts to enhance technology that makes self-monitoring easier, so women can keep closer eyes on their own blood pressure and share it with doctors in between appointments.

Most important, clinicians of all stripes should try to connect with patients to understand who they are, where they come from and how their stories impact their risk factors for health conditions.

The Women and Children’s Committee of the ABC launched the “We Are the Faces of Black Maternal Health” campaign in February 2022. The first-of-its-kind effort featured the stories of ABC members who had direct or indirect experiences with the impacts of maternal health on themselves, their children or someone they knew.

What’s next

The ABC “We Are the Faces of Black Maternal Health” re-launches this February to continue raising awareness but will also emphasize the need for investments in research about the causes of these disparities and possible prevention strategies to protect Black women.

Abstract Happy 2022 New Year greeting card with light bulb

The best of 2022 from Innovation District

January 6, 2023

A 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)

Healthcare leaders join to advance pediatric innovation

December 21, 2022

Children’s National Hospital and the National Capital Consortium for Pediatric Device Innovation (NCC-PDI) have opened a request for proposal to solicit companies interested in obtaining pediatric labeling for medical devices that may address an unmet need in the pediatric population and that already have clearance or approval for adult use by the U.S. Food & Drug Administration (FDA). The objective of this program is to generate the real-world evidence (RWE) needed to facilitate the pediatric regulatory pathway for U.S. market clearance. The deadline to apply is 5 p.m. EST on Feb. 9. To learn more and apply, visit http://www.innovate4kids.org.

Instead of assessing medical devices based on data derived from clinical trials, this pioneering initiative is focused on leveraging real-world data (RWD) that can be translated into RWE to gain FDA clearance or approval for use with children.

Convening a coalition of healthcare leaders

The new partnership aims to address the significant gap that exists between devices labeled for adults and children. Additional coalition partners include:

CobiCure
MedStar Health Research Institute
Center for Technology Innovation in Pediatrics (CTIP)
UCSF-Stanford Pediatric Device Consortium
Pennsylvania Pediatric Device Consortium
Southwest National Pediatric Device Consortium

Funded by the FDA and facilitated through NCC-PDI and the Office of Innovation Ventures at Children’s National, this program will provide winning companies with technical expertise, including but not limited to regulatory, study design and data science services.

“We are delighted to partner with this coalition of trusted healthcare leaders that share our vision for advancing pediatric health. We know all too well that pediatric device development presents several unique challenges and that children have medical device needs that are considerably different from adults,” says Kolaleh Eskandanian, Ph.D., M.B.A, P.M.P, vice president and chief innovation officer at Children’s National and principal investigator of NCC-PDI. “There are already a number of medical devices on the market that have been FDA cleared or approved and proven viable, and this partnership will help provide important evidence generation and other wraparound services to guide device creators through the regulatory path for pediatric labeling.”

Using RWE to facilitate the regulatory pathway

While Randomized Clinical Trials (RCT) have traditionally been the gold standard when investigating a medical product’s efficacy and safety, many important populations, including children, are excluded from RCTs for ethical reasons. This means that pediatric researchers must make safety and efficacy decisions in the absence of data from such trials. RWE, including data from electronic health records (EHRs), healthcare claims data, disease registries and data gathered through other health applications, can close this gap in pediatric studies. She said that MedStar Health’s capabilities in applying RWE will be a formidable asset to the chosen applicants.

Proposals for companies seeking pediatric labeling for their medical device will be reviewed by an esteemed panel of judges specializing in data science, medical device development, evidence generation, post-market surveillance and the FDA’s regulatory pathway. Children’s National and members of the coalition will provide selected companies with technical expertise in support of their effort to achieve pediatric labeling. This will include:

Access to mentors
A design study protocol implementing RWE generation best practices
Facilitation of IRB submission and study implementation
Data science support
Regulatory, reimbursement and supply chain consultation

About NCC-PDI

NCC-PDI is one of five consortia in the FDA’s Pediatric Device Consortia Grant Program created to support the development and commercialization of medical devices for children. NCC-PDI is led by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National and the A. James Clark School of Engineering at the University of Maryland, with support from partners MedTech Innovator and design firm Archimedic.

Congenital Heart Initiative celebrates global reach in first two years

December 7, 2022

The first patient-powered registry for adults living with congenital heart disease (CHD) — the Congential Heart Initiative (CHI) — celebrates a major milestone this month. In the two years since it was launched in December 2020, the registry has enrolled more than 3,227 adults with CHD from all 50 states and 28 countries.

Why it matters

Although nearly 2 million adults in the United States are living with a congenital heart defect — more adults than children in fact — it’s been historically difficult to gather data on these conditions and to identify patient needs.

“We developed this registry together with numerous patients and providers so it could become a platform for increasing our knowledge and improving care,” says Anitha John, M.D., medical director of the Washington Adult Congenital Heart Program and an adult congenital cardiologist at Children’s National Hospital, who helped drive the registry’s creation. “We want it to become a resource for patients and researchers, a place where they can learn more about what it means for adults who had their hearts repaired in childhood to live a long, healthy life.”

The patient benefit

As the treatments for children with CHDs have improved, people born with them are living longer and longer. That means that as they become adults, they have a lot of worry and uncertainty about their limitations and abilities to achieve what might be considered common adult milestones.

“Over the past two years, more than 3,000 adult congenital heart disease (ACHD) patients from around the world, including myself, have worked to support ACHD research by participating in the Congenital Heart Initiative (CHI),” says Scott Leezer, a single-ventricle heart defect patient and co-leader of research and advocacy for the Initiative. “This selfless and simple effort has produced an investment in improving our collective understanding of adult patients living with CHD. We hope these investments will pay dividends in helping guide new research strategies for future generations of CHD patients.”

Participants in the registry receive regular newsletters, highlighting findings and providing access to patient specific resources including content from the Adult Congenital Heart Association (ACHA), a key partner. The ACHA collaboration has also given registry participants the opportunity to interact with other people – a key request identified as important based on input from people who have already registered.

What’s unique

“There is no ’one-size-fits-all’ answer for adults with CHD,” Dr. John adds.

“While other CHD registries exist, they are provider-based and not patient-powered,” said John. “The CHI registry is driven by patients, supporting research but also providing information based on what is important according to the people with CHD themselves.”

What’s next

A sub-study of the CHI will use PCORnet®, the National Patient-Centered Clinical Research Network, to better understand how gaps in care impact the adult patient experience with CHD. The CHI-RON study, which stands for Congenital Heart Initiative: Redefining Outcomes and Navigation to Adult-Centered Care, will be the first of its kind to fill in these knowledge gaps by exploring three distinct types of data at once: patient reported outcomes, health insurance claims and electronic health records (EHR). The effort is led by Children’s National and Louisiana Public Health Institute.

“PCORnet is a fantastic resource to help us fully understand the impact of gaps in CHD care,” said Tom Carton, dual-principal investigator of CHI-RON and chief data officer at the Louisiana Public Health Institute. “It is essentially turbo-charging our patient-reported data with two additional layers of insights from claims and EHRs, unlocking answers that would be impossible to achieve in isolation.”

In terms of research, the CHI now has enough participants to allow researchers to complete some important studies about pregnancy, mental health and long-term health care follow ups, all of which will kick off within the next year. The research teams hope to engage centers across the United States to better determine the needs of individual ACHD centers and to understand how the CHI can help with these needs.