Tag Archive for: global health

Boy in bed with oxygen mask

New tool helps doctors know when kids with pneumonia need hospital care

Boy in bed with oxygen mask

A new international study led by Children’s National Hospital and Lurie Children’s Hospital of Chicago introduces a validated tool to predict pneumonia severity in children, helping emergency clinicians make faster, evidence-based decisions about hospitalization and intensive care.

Pneumonia is one of the most common infections in children. In the U.S., it’s a leading reason why kids are admitted to the hospital. But for emergency doctors, it’s not always easy to know which cases are serious — and which children will get better at home.

Now, a new study led by doctors at Children’s National Hospital and Lurie Children’s Hospital of Chicago has created a tool to help. It’s based on research from over 2,200 children treated at emergency departments in 14 countries.

The tool is simple: it uses symptoms that doctors already look for — like how fast a child is breathing, whether they’re getting enough oxygen, and if they’re drinking fluids — to score how serious their pneumonia might be. The score helps doctors decide whether a child needs to stay in the hospital, go to intensive care or can safely recover at home.

What the study found

The research team looked at children ages 3 months to 13 years who came to emergency departments with community-acquired pneumonia — a kind of pneumonia picked up outside of a hospital. Most had mild cases. But about 1 in 20 developed severe symptoms, like needing breathing support or admission to intensive care.

The team found that certain symptoms (like fast breathing or heart rate, chest retractions (a sign of struggling to breathe), low oxygen levels, refusing to drink and already being on antibiotics before coming to the hospital) were linked to a higher risk of serious illness. On the other hand, children who had a runny nose or congestion were more likely to have mild illness.

Using this data, the team created a point-based score. For example, if a child had low oxygen levels, they’d get 3 to 6 points depending on how low it was. Chest retractions added 3 points. Having a runny nose subtracted a point. The higher the total score, the greater the risk of moderate or severe pneumonia.

How it helps

The model was tested and found to be highly accurate. It performed better than doctors’ judgment alone in earlier studies — especially in spotting the children most at risk. The scorecard gives emergency doctors a fast, evidence-based way to support the decisions they make under pressure.

“Emergency departments around the world see thousands of children with pneumonia every day, but until now, we haven’t had a reliable way to predict who’s truly at risk of getting sicker,” said co-PI and senior author Nathan Kuppermann, MD, MPH, executive vice president, chief academic officer and director of the Children’s National Research Institute. “This model gives clinicians a practical tool, rooted in data, to guide that decision and ultimately improve care and outcomes.”

What’s next

While the tool is ready to be used in hospitals now, the team plans to test it in more locations and study how it affects real-world decisions. They also hope to add biomarkers — lab tests that could improve the score’s accuracy even more.

For now, the study offers something simple and powerful: a better way to know when a child’s pneumonia might become serious — and when it won’t.

The study was published in The Lancet Child & Adolescent Health and is part of a larger effort by the Pediatric Emergency Research Network (PERN), which connects emergency departments in dozens of countries.

The Lead in Peds Episode 4

Podcast: Future ready: AI’s role in revolutionizing pediatric care

Read more
Doctors from Children's National Hospital in Uganda

Children’s National brings AI into the RHD early diagnosis equation

In December of 2024, a team that included experts from Children’s National Hospital traveled to Uganda to continue work on a pilot program applying artificial intelligence (AI) to the diagnosis of rheumatic heart disease (RHD). Ugandan health care providers have been trained and equipped to acquire echocardiograms for their patients but lack expertise in consistently being able to diagnose RHD by detecting leaky heart valves. The team created a tool that uses AI to predict RHD by identifying leaky heart valves on handheld ultrasound devices, then prompts a referral for a full echocardiogram.

The goal is to find ways to help people in Uganda diagnose RHD early, before a patient is in need of surgery, and initiate antibiotics so their heart can return to normal. The team of researchers, including fellow Kelsey Brown, MD, helped to implement additional steps toward this goal in December. According to Dr. Brown, the results were excellent. After four days of seeing patients, over 450 people were screened. The AI tool has an 86% accuracy rating. After returning from Uganda, the research team plans to work on the AI tool and further improve its accuracy rating. Eventually, the vision is that this tool can roll out on a larger scale for more places around the world to access it.

Craig Sable, MD, Marius Linguraru, DPhil, MA, MSc, and Pooneh Roshanitabrizi, PhD, from our Sheikh Zayed Institute, who developed the AI algorithms, worked in partnership with the Rheumatic Heart Disease Research Collaborative (RRCU) in Uganda. This trip was also made possible thanks to a grant funded through the Children’s National Global Health Initiative. Special thank you to our AI partner, US2.AI, who made the deployment of the AI models onto a tablet that provided real-time results, possible.

Children's National Hospital's winning team for the Brain Tumor Segmentation-Africa (BraTS-Africa) challenge

AI for good: Children’s National wins global competitions for measuring brain tumors

Children's National Hospital's winning team for the Brain Tumor Segmentation-Africa (BraTS-Africa) challenge

Meet the winners (left to right): Syed M. Anwar, Ph.D., M.S., principal investigator at Children’s National; Daniel Capellan Martin, M.Sc., Polytechnic University of Madrid; Abhijeet Parida, data scientist at Children’s National; and Austin Tapp, Ph.D., postdoctoral research fellow at Children’s National.

Using an award-winning artificial intelligence (AI) algorithm developed at Children’s National Hospital, researchers ranked first in the world in the Brain Tumor Segmentation-Africa (BraTS-Africa) challenge for their approach to identifying different parts of deadly gliomas. The details of their innovative method were recently published on arXiv, a curated research-sharing platform.

“Technology can bridge the gap in healthcare between high- and low-resource countries,” said Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor in Research and Innovation and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI). “By tailoring methods we created at our hospital to fit the needs of specific regions, such as sub-Saharan Africa, our research helps improve medical imaging and diagnosis in challenging environments.”

Dr. Linguraru was the program chair of the International Conference on Medical Image Computing and Computer Assisted Intervention (MICCAI) 2024 in Marrakesh, Morocco, the leading global meeting on AI in medical imaging.

Children’s National leads the way

Gliomas are a type of brain tumor with a high death rate and are especially difficult to diagnose in low- and middle-income countries. Given the increased need in Africa, researchers worldwide came together in Morocco to compete over the best way to accurately detect and measure tumors using MRI data and AI.

By applying advanced machine-learning techniques, the researchers adapted tools initially designed for well-resourced settings to work in countries with far fewer.

The study focused on transfer learning, a process in which an AI model is trained in advance on a large number of brain tumor images and then adjusted to work with smaller sets of new data. In this case, the models were adapted to work with local sub-Saharan African data using a strategy to combine different models’ strengths.

When tested, the approach achieved impressive accuracy scores. The Children’s National team, which included a colleague from the Polytechnic University of Madrid, ranked first in the BraTS-Africa 2024 challenge for identifying different parts of gliomas.

“To make the method widely available, the winning algorithm is shared online for others to use and improve upon,” Dr. Linguraru said. “My favorite part of these competitions is how they highlight the way innovation and collaboration can reduce global healthcare inequalities.”

The big picture

Children’s National researchers consistently lead global events using AI and advanced imaging to tackle complex healthcare challenges. In 2023, the team won a global contest to measure pediatric brain tumors at the MICCAI 2023 Conference. This year’s success in the BraTS-Africa challenge builds on this knowledge base and expands its use to adult gliomas.

At the Radiological Society of North America 2024 annual meeting, which drew 50,000 attendees, Zhifan Jiang, Ph.D., a staff scientist in the Precision Medical Imaging Lab at SZI, also won the Cum Laude Award for his scientific poster on applying AI to radiological images to predict severe outcomes for children with brain tumors caused by neurofibromatosis type 1.

“These achievements show how our science is leading the world in using AI for good,” Dr. Linguraru said. “Every day, we’re building on our knowledge of advanced imaging, brain tumors and AI to improve the diagnosis, measurement and treatment of deadly tumors — on a global scale.”

Attendees of the Brain Tumor Segmentation-Africa (BraTS-Africa) challenge

Douglas Postels and other clinicians in Malawi

Think tank improves care for Malawian children with seizures, epilepsy

  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
PreviousNext
123456

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

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

The big picture

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

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

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

The fine print

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

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

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

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

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

boy in hospital bed

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

boy in hospital bed

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:

echocardiogram

AI may revolutionize rheumatic heart disease early diagnosis

echocardiogram

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

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

Why it matters

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

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

The hold-up in the field

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

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

Children’s National leads the way

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

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

What’s next

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

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

Dr. Sable performing an echocardiogram in Uganda

Penicillin slows impacts of rheumatic heart disease in Ugandan children

Dr. Sable performing an echocardiogram in Uganda

“We know from previous studies that though it is not always well-documented, sub-Saharan Africa continues to have some of the highest numbers of people with rheumatic heart disease and the highest numbers of people dying from it,” said Craig Sable, M.D., associate chief of Cardiology at Children’s National Hospital and co-senior author of the study. “This study is the first large-scale clinical trial to show that early detection coupled with prophylactic treatment of penicillin is feasible and can prevent rheumatic heart disease from progressing and causing further damage to a child’s heart.”

Penicillin, a widely available and affordable antibiotic, may be one key to turning the tide on the deadly impacts of rheumatic heart disease (RHD) for children in developing nations. This according to the new findings of a large-scale, randomized controlled trial completed in Uganda and published in the New England Journal of Medicine.

The most devastating feature of RHD is severe heart valve damage that is caused by rheumatic fever — a condition that results from the body’s immune system trying to fight poorly treated, repeat infections from streptococcus bacteria, also known as strep throat. Though widely eradicated in nations such as the United States due to the swift detection and treatment of strep throat, rheumatic fever remains prevalent in developing countries including those in sub-Saharan Africa. Current estimates are that 40.5 million people worldwide live with rheumatic heart disease, and that it kills 306,000 people every year. Most of those affected are children, adolescents and young adults under age 25.

“We know from previous studies that though it is not always well-documented, sub-Saharan Africa continues to have some of the highest numbers of people with rheumatic heart disease and the highest numbers of people dying from it,” said Craig Sable, M.D., associate chief of Cardiology at Children’s National Hospital and co-senior author of the study. “This study is the first large-scale clinical trial to show that early detection coupled with prophylactic treatment of penicillin is feasible and can prevent rheumatic heart disease from progressing and causing further damage to a child’s heart.”

The study was led by an international panel of pediatric cardiac experts from institutions including Children’s National, Cincinnati Children’s Medical Center, the Uganda Heart Institute and Murdoch Children’s Research Institute in Melbourne, Australia.

“Our study found a cheap and easily available penicillin can prevent progression of latent rheumatic heart disease into more severe, irreversible valve damage that is commonly seen in our hospitals with little or no access to valve surgery,” said co-lead author Emmy Okello, M.D., chief of Cardiology at the Uganda Heart Institute.

To Andrea Beaton, M.D., associate professor of Cardiology at Cincinnati Children’s and co-lead author, this is the first contemporary randomized controlled trial in rheumatic heart disease. “The results are incredibly important on their own, but also demonstrate that high-quality clinical trials are feasible to address this neglected cardiovascular disease,” she said.

Beaton et al. named the trial Gwoko Adunu pa Lutino (GOAL), which means “protect the heart of a child.” The study enrolled 818 Ugandan children and adolescents ages 5 to 17 years old who were diagnosed with latent rheumatic heart disease to see if an injection of penicillin was effective at preventing their heart condition from worsening.

“There are many challenges with recruitment and retention of trial participants in areas like our study region in Uganda,” said Dr. Sable. “But it is critical to work together and overcome barriers, because we must study these treatments in the people most affected by the condition to understand how they, and others like them, may benefit from the findings.”

Of the 799 participants who completed the trial, the group receiving a prophylactic injection of penicillin (399 volunteers) had three participants show evidence of worsened rheumatic heart disease on repeat echocardiogram after two years. In contrast, 33 of the 400 volunteers in the control group, who received no treatment, showed similar progression on echocardiogram results.

Professor Andrew Steer, who is theme director of Infection and Immunity at Murdoch Children’s Research Institute in Melbourne and who served as senior author of the study, said screening for latent rheumatic heart disease was critical to stop progression because heart valve damage was largely untreatable. “Most patients are diagnosed when the disease is advanced and complications have already developed. If patients can be identified early, there is an opportunity for intervention and improved health outcomes.”

The results were shared in a special presentation at the American Heart Association’s Scientific Sessions on the same day that the findings were published in the New England Journal of Medicine.

The trial was supported by the Thrasher Pediatric Research Fund, Gift of Life International, Children’s National Hospital Foundation: Zachary Blumenfeld Fund, Children’s National Hospital Race for Every Child: Team Jocelyn, the Elias/Ginsburg Family, Wiley-Rein LLP, Phillips Foundation, AT&T Foundation, Heart Healers International, the Karp Family Foundation, Huron Philanthropies and the Cincinnati Children’s Hospital Heart Institute Research Core.

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:

Ugandan boy in hospital bed

Acute rheumatic fever often goes undiagnosed in sub-Saharan Africa

Ugandan boy in hospital bed

Despite low numbers of documented acute rheumatic fever cases in sub-Saharan Africa, the region continues to show some of the highest numbers of people with, and dying from, rheumatic heart disease, the serious heart damage caused by repeat instances of rheumatic fever.

Despite low numbers of documented acute rheumatic fever cases in sub-Saharan Africa, the region continues to show some of the highest numbers of people with, and dying from, rheumatic heart disease, the serious heart damage caused by repeat instances of rheumatic fever. A population-based study in the Lancet Global Health collected evidence of acute rheumatic fever in two areas of Uganda, providing the first quantifiable evidence in decades that the disease continues to take a deadly toll on the region’s people.

“These findings matter. Access to life-saving heart surgery is only available to a very small fraction of the hundreds of thousands of patients in Africa who have irreversible heart damage from rheumatic heart disease,” says Craig Sable, M.D., associate chief of Cardiology at Children’s National Hospital and one of the senior authors of the study. “It’s time to focus upstream on capturing these conditions sooner, even in low-resource settings, so we can implement life-sustaining and cost-saving preventive treatments that can prevent further heart damage.”

The authors, who hail from Uganda and several institutions around the United States, including Children’s National and Cincinnati Children’s Hospital Medical Center, note this is the first study to use an active case-finding strategy for diagnosing acute rheumatic fever. They also note that raising awareness in the community and among its healthcare workers while also finding new ways to overcome some of the diagnostic challenges in these low-resource settings greatly improved diagnosis and treatment of the condition.

The study also described clinical characteristics of children ages 5 to 14 presenting with both definitive and possible acute rheumatic fever, providing further clinical data points to help healthcare workers in these communities differentiate between this common infection and some of the other frequently diagnosed conditions in the region.

“With this study, we can now confidently dismiss the myth that acute rheumatic fever is rare in Africa,” the authors write. “It exists at elevated rates in low-resource settings such as Uganda, even though routine diagnosis remains uncommon. While these incidence data have likely underestimated the cases of acute rheumatic fever in two districts in Uganda, they show that opportunity exists to improve community sensitization and healthcare worker training to increase awareness of acute rheumatic fever. Ultimately this leads to diagnosing more children with the condition before they develop rheumatic heart disease, so that they can be offered secondary prophylaxis with penicillin.”

Children with suspected acute rheumatic fever participated in this population-based study. Data was collected over 12 months in Lira district (January 2018 to December 2018) and over nine months (June 2019 to February 2020) in Mbarara district.

Follow-up of children diagnosed in this study will provide more data on the outcomes of acute rheumatic fever, including a better understanding of the risk for a child to develop rheumatic heart disease.

This work was funded by the American Heart Association Children’s Strategically Focused Research Network Grant #17SFRN33670607 and by DEL‐15‐011 to THRiVE‐2 and General Electric.

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:


Crowded makeshift buildings of a shantytown

Calling greater attention to sub-Saharan Africa’s pressing challenges in pediatric cardiac care

Crowded makeshift buildings of a shantytown

Sub-Saharan Africa has only 0.19 pediatric cardiac surgeons per million children — nowhere near enough surgeons to care for all the pediatric congenital heart disease and acquired heart disease present in the people who live there.

A literature review in the journal Current Opinion in Cardiology draws further attention to the pressing needs for better pediatric cardiac care in regions of the world where the population continues to grow, but the development of specialty care for children continues to lag. The article focuses specifically on sub-Saharan Africa.

“If 40% of live births occur in Africa by 2050 as the projections suggest, congenital heart disease may well become the most important contributor to infant mortality rate in sub-Saharan Africa in the next three decades,” stated the authors, including Annette Ansong, M.D., who recently joined Children’s National Hospital as medical director of outpatient cardiology.

As highlighted previously by other authors within the Global Health Initiative at Children’s National and through the work of the American Heart Association, the region’s needs are already significant in  tackling the impacts of existing congenital heart disease and rheumatic heart disease. Rheumatic heart disease is a devastating long-term outcome of rheumatic fever caused by untreated streptococcus infections.

Annette Ansong

“If 40% of live births occur in Africa by 2050 as the projections suggest, congenital heart disease may well become the most important contributor to infant mortality rate in sub-Saharan Africa in the next three decades,” stated the authors, including Annette Ansong, M.D., who recently joined Children’s National Hospital as medical director of outpatient cardiology.

Dr. Ansong and colleagues reiterate the point that today, “whereas one cardiac center caters to approximately 120,000 people in North America, 33 million people in sub-Saharan Africa must depend on one center for care.” They also note that this region of Africa has only 0.19 pediatric cardiac surgeons per million children compared with more than 58 times as many in North America.

Changing the trajectory of pediatric cardiac care in sub-Saharan Africa will take motivation on several fronts, the authors write. Dedication to early detection and intervention (medical or surgical), an emphasis on building an in-country pipeline of human resources and skills’ sets are needed to tackle the increasing numbers of children requiring this specialty care. Political will and better financial resources can also support the training and development of centers that specialize in these capabilities.

Dr. Craig Sable

AHA doubles down on global support, prevention and research in rheumatic heart disease

Dr. Craig Sable

Dr. Craig Sable and pediatric cardiology colleagues led the creation of a scientific statement and advocacy statement focused on eradicating RHD.

A pair of articles appearing in the American Heart Association’s (AHA) journal Circulation lays out a call to action for advocacy and scientific priorities crucial to the global eradication of rheumatic heart disease (RHD).

Cardiologists from Children’s National Hospital, and others who completed their pediatric cardiology fellowships at Children’s National before moving on to careers at other institutions, have been active proponents and advocates for these efforts for many years and led key research and clinical care efforts related to RHD in other countries of the world.

These cardiologists, including the associate chief of cardiology at Children’s National, Craig Sable, M.D., who previously served as chair of the AHA Council on Lifelong Congenital Heart Disease and Heart Health in the Young, also helped lead the creation of these new published statements.

Contemporary diagnosis and management of rheumatic heart disease: Implications for closing the gap

This clinical and research statement “seeks to examine the current state of-the-art recommendations and to identify gaps in diagnosis and treatment globally that can inform strategies for reducing disease burden.”

Key recommendations and related challenges were mapped out, including:

  • The need for echocardiography screening based on World Heart Federation echocardiographic criteria for identifying patients earlier, when prophylaxis is more likely to be effective. However, the authors note that several important questions need to be answered before this can translate into public policy.
  • The creation of population-based registries to effectively enable optimal care and secondary penicillin prophylaxis within available resources, though the team acknowledges that challenges with penicillin procurement and concern with adverse reactions in patients with advanced disease remain important issues.
  • Heart failure management, prevention, early diagnosis and treatment of endocarditis, oral anticoagulation for atrial fibrillation and prosthetic valves used as vital therapeutic adjuncts.
  • Multidisciplinary team management of health of women with unoperated and operated rheumatic heart disease before, during and after pregnancy is the best approach, though it is a significant challenge.
  • Percutaneous balloon mitral valvuloplasty should be considered for patients with isolated mitral stenosis.
  • Timely heart valve surgery, especially valve repair for rheumatic mitral regurgitation, can mitigate the progression to heart failure, disability and death. However, some of these procedures are not available to the vast majority of patients in endemic regions.

The recommendations made in the scientific statement form the foundation for the advocacy companion document.

The AHA’s call to action for reducing the global burden of rheumatic heart disease: a policy statement from the AHA

The advocacy statement outlines five key areas of support:

  1. Professional healthcare worker education and training.
  2. Technical support for the implementation of evidence-based strategies for rheumatic fever/RHD prevention.
  3. Access to essential medications and technologies.
  4. Research.
  5. Advocacy to increase global awareness, resources and capacity for RHD control.

The authors write, “In bolstering the efforts of the American Heart Association to combat RHD, we hope to inspire others to collaborate, communicate and contribute.”

Speaking of the two statements as a whole, the authors of the scientific statement conclude that, “Ultimately, the combination of expanded treatment options, research and advocacy built on existing knowledge and science provides the best opportunity to address the burden of rheumatic heart disease.”

Read more about Children’s National Heart Institute’s research, education and clinical care in rheumatic heart disease.

Craig Sable, M.D., Associate Chief of the Division of Cardiology and Director of Echocardiography at Children’s National Health System, is working with hundreds of doctors to create a scalable solution to reduce the global burden of rheumatic heart disease (RHD). Dr. Sable received a lifetime achievement award — the 2018 Cardiovascular Disease in the Young (CVDY) Meritorious Achievement Award — from the American Heart Association for his work in Uganda.

Patients and staff at the Uganda Heart Institute

Lifesaving heart surgeries for RHD complications in Uganda go on despite COVID-19

Patients and staff at the Uganda Heart Institute

Patients and staff at the Uganda Heart Institute for RHD-related heart surgeries in Uganda, March 2020. These patients were originally scheduled as part of the cancelled medical mission, but UHI cardiovascular surgeon successfully managed these cases without the support of the mission doctors from the U.S.

In early March as countries around the globe began to wrestle with how best to tackle the spread of COVID-19, a group of doctors, nurses, researchers and other medical staff from Children’s National Hospital were wrestling with a distinct set of challenges: What to do about the 10 Ugandan children and adults who were currently scheduled for lifesaving heart surgery (and the countless others who would benefit from the continued training of the local heart surgery team) to correct complications of rheumatic heart disease (RHD) during an impending medical mission in the country.

Rheumatic heart disease impacts over 39 million people globally and causes nearly 300,000 deaths per year. RHD is the result of frequent, untreated streptococcal throat infections in childhood that ultimately cause the body’s immune system to repeatedly damage heart valves. It is completely preventable, yet the majority of the world’s children still live in impoverished and overcrowded conditions that predispose them to RHD. Most patients present with advanced valvular heart disease. For example, in Uganda, an RHD registry includes over 600 children with clinical RHD, of which nearly 40% die within four years and the median survival time from enrollment in the registry is only nine months. For these patients, heart surgery is the only viable solution for long-term survival and normal quality of life.

Patricia: 9-year-old from Gulu

Patricia: 9-year-old from Gulu (northern Uganda), had mitral valve replacement and was doing well on a recent follow-up visit at her home.

The scheduled trip from Washington was part of a nearly 20-year partnership** between doctors, nurses, researchers and other medical staff in the United States, including Craig Sable, M.D., associate chief of cardiology, and and Pranava Sinha, M.D.,pediatric cardiovascular surgeon, at Children’s National Hospital in Washington, D.C., and the Uganda Heart Institute in Kampala, Uganda. The partnership aims to tackle RHD head-on. It provides surgical skill transfer, allows for treatment of more complex patients, and increases sustainable surgical capacity for Uganda’s RHD patients over time. As a result, over the last 15 years more than 1,000 children have received lifesaving heart surgery in Uganda, with the Uganda Heart Institute (UHI) performing one to two heart valve surgeries every two weeks over the last few years.

Jackline: 12-year-old from Gulu

Jackline: 12-year-old from Gulu, had mitral valve repair and aortic valve replacement. Jackline and Patricia were diagnosed through one of our research programs and benefit from our novel telehealth program, which helps connect patients from remote parts of Uganda to specialists at UHI.

COVID-19 was changing the current plan, however. Travel between countries was limited, and the team from the U.S. wouldn’t have been permitted to leave the U.S. and return according to schedule. The trip, and the support teams who were scheduled to arrive to help with the surgeries, were cancelled. The U.S. team members who had already arrived in Uganda were sent home after helping their UHI colleagues set up and prepare for the surgeries as much as possible. Knowing that patients and families were counting on the surgery mission to go forward after waiting for months or years to have surgery for heart valve disease, UHI decided not to cancel the majority of the surgeries. Instead, for the first time, they planned and successfully completed five valve-related cases in a single week – several of them quite complex. The cardiologists and cardiac surgeons from Children’s National who were supposed to be in-country for these procedures were forced to limit their in person assistance to the set-up activities the week prior to surgery and telehealth consult during the procedures.

“It was hard not to be able to stay  and work with the UHI team to help these families,” says Dr. Sable. “But we are so proud of the UHI team for meeting this challenge on their own. We knew they had the skills to perform at this volume and complexity. It’s a proud moment to see the team accomplish this major milestone, and to see the patients they cared for thrive.”

The patients are the most important outcome: The five who had successful open-heart surgery are all doing well, either on their way to recovery or already discharged to their communities, where they will, for the first time in memory, be able to play, exercise and go to school or work.

Longer term, this success demonstrates the UHI medical team’s ability to manage greater surgical capacity even when surgical missions from the U.S. resume. The partnership’s goal is to complete at least 1,000 annual operations (both pediatric and adult), with the majority being performed by the local team. Having this capacity available will mean the difference between life and death for many children and adults who have RHD in Uganda and the surrounding countries.

**This work is supported by the Edwards Life Sciences/Thoracic Surgery Foundation, the Emirates Airline Foundation, Samaritan’s Purse Children’s Heart Project and Gift of Life International.

Pediatric angiography

Congenital heart disease more deadly in low-income countries

Pediatric angiography

Even though mortality from congenital heart disease (CHD) has declined over the last three decades as diagnosis and treatments have advanced, the chances for a child to survive a CHD diagnosis significantly differs based on the country where he or she is born.

This eye-opening finding is drawn from the first comprehensive study of congenital heart disease across 195 countries, prepared using data from the Global Burden of Diseases, Injuries and Risk Factors Study 2017 (GBD), and recently published in The Lancet.

“Previous congenital heart estimates came from few data sources, were geographically narrow and did not evaluate CHD throughout the life course,” write the authors, known collectively as the 2017 GBD Congenital Heart Disease Collaborators. Co-lead author Meghan D. Zimmerman, M.D., worked on the study while completing her pediatric cardiology and American Heart Association Global Health Fellowships at Children’s National Hospital, and two pediatric cardiologists from Children’s National, Cardiology Associate Chief Craig Sable, M.D., and Gerard Martin, M.D., medical director of Global Services, provided leadership and oversight of this paper. The remaining collaborators are from more than 45 institutions around the world, spanning cardiology, public health and schools of medicine on every continent.

This is the first time the GBD study data was used along with all available data sources and previous publications – making it the most comprehensive study on congenital heart disease burden to date. Key differences between this study and prior estimates include:

  • Anatomic groupings of CHD by type, rather than simply categorized as moderate, severe or critical.
  • Inclusion of new data sources, including data from screening programs, congenital registries, administrative data and data sources in mortality and survival.
  • A control mechanism to account for cases of CHD that remit on their own to reduce the risk of overestimating prevalence.
  • Inclusion of all cases of congenital heart disease, including those with chromosomal or genetic anomalies such as Trisomy 21 that often co-occur.

This more comprehensive data set led to findings that showed lower predicted long-term survival, higher remission, and lower prevalence than previous studies that extrapolated evidence from studies of high-income countries. However, it also means these new estimates are a more accurate representation of the current global state of affairs. Overall, the study found:

  • A 34.5% decline in deaths from congenital disease between 1990 to 2017.
  • Nearly 70% of deaths caused by CHD in 2017 (180,624) were in infants less than one year old.
  • Most CHD deaths occurred in countries within the low and low-middle socio-demographic index (SDI) quintiles.
  • Mortality rates get lower as a country’s SDI rises.
  • Birth prevalence of CHD was not related to a country’s socio-demographic status, but overall prevalence was much lower in the poorest countries of the world. This is because children in these countries do not have access to life saving surgical services.
  • Nearly 12 million people are currently living with CHD globally, 18.7% more than in 1990.
  • The burden of CHD is not fully realized by just looking at prevalence and mortality. The measure “Years of Life Lost” provides deeper insight into the staggering burden of CHD, taking into account both absolute mortality and age at death.

“In high income countries like the United States, we diagnose some heart conditions prenatally during the 20-week ultrasound,” says Gerard Martin, M.D., a pediatric cardiologist at Children’s National Hospital who contributed to the study. “We catch others right after birth with a pulse oximetry screening for critical congenital heart disease. We can operate to correct a critical issue within the first week of life. And now our CHD kids are growing and thriving through adulthood and having families of their own.”

“For children born in middle- and low-income countries, these data draw stark attention to what we as cardiologists already knew from our own work in these countries – the lack of diagnostic and treatment tools leads to lower survival rates for children born with CHD,” adds Craig Sable, M.D., associate chief of cardiology at Children’s National, another primary contributor. “This is one of the most significant publications I have been a part of as it highlights the substantial loss of life to CHD in infancy around the globe.”

The authors write, “The UN has prioritized reduction of premature deaths from heart disease, but to meet the target of ‘ending preventable deaths of newborns and children under 5 years of age,’ health policy makers will need to develop specific accountability measures that address barriers and improve access to care and treatment.”

The study also includes a 400-page appendix breaking down each area by type of congenital anomaly, world region and country.

Billie Lou Short and Kurt Newman at Research and Education Week

Research and Education Week honors innovative science

Billie Lou Short and Kurt Newman at Research and Education Week

Billie Lou Short, M.D., received the Ninth Annual Mentorship Award in Clinical Science.

People joke that Billie Lou Short, M.D., chief of Children’s Division of Neonatology, invented extracorporeal membrane oxygenation, known as ECMO for short. While Dr. Short did not invent ECMO, under her leadership Children’s National was the first pediatric hospital to use it. And over decades Children’s staff have perfected its use to save the lives of tiny, vulnerable newborns by temporarily taking over for their struggling hearts and lungs. For two consecutive years, Children’s neonatal intensive care unit has been named the nation’s No. 1 for newborns by U.S. News & World Report. “Despite all of these accomplishments, Dr. Short’s best legacy is what she has done as a mentor to countless trainees, nurses and faculty she’s touched during their careers. She touches every type of clinical staff member who has come through our neonatal intensive care unit,” says An Massaro, M.D., director of residency research.

For these achievements, Dr. Short received the Ninth Annual Mentorship Award in Clinical Science.

Anna Penn, M.D., Ph.D., has provided new insights into the central role that the placental hormone allopregnanolone plays in orderly fetal brain development, and her research team has created novel experimental models that mimic some of the brain injuries often seen in very preterm babies – an essential step that informs future neuroprotective strategies. Dr. Penn, a clinical neonatologist and developmental neuroscientist, “has been a primary adviser for 40 mentees throughout their careers and embodies Children’s core values of Compassion, Commitment and Connection,” says Claire-Marie Vacher, Ph.D.

For these achievements, Dr. Penn was selected to receive the Ninth Annual Mentorship Award in Basic and Translational Science.

The mentorship awards for Drs. Short and Penn were among dozens of honors given in conjunction with “Frontiers in Innovation,” the Ninth Annual Research and Education Week (REW) at Children’s National. In addition to seven keynote lectures, more than 350 posters were submitted from researchers – from high-school students to full-time faculty – about basic and translational science, clinical research, community-based research, education, training and quality improvement; five poster presenters were showcased via Facebook Live events hosted by Children’s Hospital Foundation.

Two faculty members won twice: Vicki Freedenberg, Ph.D., APRN, for research about mindfulness-based stress reduction and Adeline (Wei Li) Koay, MBBS, MSc, for research related to HIV. So many women at every stage of their research careers took to the stage to accept honors that Naomi L.C. Luban, M.D., Vice Chair of Academic Affairs, quipped that “this day is power to women.”

Here are the 2019 REW award winners:

2019 Elda Y. Arce Teaching Scholars Award
Barbara Jantausch, M.D.
Lowell Frank, M.D.

Suzanne Feetham, Ph.D., FAA, Nursing Research Support Award
Vicki Freedenberg, Ph.D., APRN, for “Psychosocial and biological effects of mindfulness-based stress reduction intervention in adolescents with CHD/CIEDs: a randomized control trial”
Renee’ Roberts Turner for “Peak and nadir experiences of mid-level nurse leaders”

2019-2020 Global Health Initiative Exploration in Global Health Awards
Nathalie Quion, M.D., for “Latino youth and families need assessment,” conducted in Washington
Sonia Voleti for “Handheld ultrasound machine task shifting,” conducted in Micronesia
Tania Ahluwalia, M.D., for “Simulation curriculum for emergency medicine,” conducted in India
Yvonne Yui for “Designated resuscitation teams in NICUs,” conducted in Ghana
Xiaoyan Song, Ph.D., MBBS, MSc, “Prevention of hospital-onset infections in PICUs,” conducted in China

Ninth Annual Research and Education Week Poster Session Awards

Basic and Translational Science
Faculty: Adeline (Wei Li) Koay, MBBS, MSc, for “Differences in the gut microbiome of HIV-infected versus HIV-exposed, uninfected infants”
Faculty: Hayk Barseghyan, Ph.D., for “Composite de novo Armenian human genome assembly and haplotyping via optical mapping and ultra-long read sequencing”
Staff: Damon K. McCullough, BS, for “Brain slicer: 3D-printed tissue processing tool for pediatric neuroscience research”
Staff: Antonio R. Porras, Ph.D., for “Integrated deep-learning method for genetic syndrome screening using facial photographs”
Post docs/fellows/residents: Lung Lau, M.D., for “A novel, sprayable and bio-absorbable sealant for wound dressings”
Post docs/fellows/residents: Kelsey F. Sugrue, Ph.D., for “HECTD1 is required for growth of the myocardium secondary to placental insufficiency”
Graduate students: Erin R. Bonner, BA, for “Comprehensive mutation profiling of pediatric diffuse midline gliomas using liquid biopsy”
High school/undergraduate students: Ali Sarhan for “Parental somato-gonadal mosaic genetic variants are a source of recurrent risk for de novo disorders and parental health concerns: a systematic review of the literature and meta-analysis”

Clinical Research
Faculty: Amy Hont, M.D., for “Ex vivo expanded multi-tumor antigen specific T-cells for the treatment of solid tumors”
Faculty: Lauren McLaughlin, M.D., for “EBV/LMP-specific T-cells maintain remissions of T- and B-cell EBV lymphomas after allogeneic bone marrow transplantation”

Staff: Iman A. Abdikarim, BA, for “Timing of allergenic food introduction among African American and Caucasian children with food allergy in the FORWARD study”
Staff: Gelina M. Sani, BS, for “Quantifying hematopoietic stem cells towards in utero gene therapy for treatment of sickle cell disease in fetal cord blood”
Post docs/fellows/residents: Amy H. Jones, M.D., for “To trach or not trach: exploration of parental conflict, regret and impacts on quality of life in tracheostomy decision-making”
Graduate students: Alyssa Dewyer, BS, for “Telemedicine support of cardiac care in Northern Uganda: leveraging hand-held echocardiography and task-shifting”
Graduate students: Natalie Pudalov, BA, “Cortical thickness asymmetries in MRI-abnormal pediatric epilepsy patients: a potential metric for surgery outcome”
High school/undergraduate students: Kia Yoshinaga for “Time to rhythm detection during pediatric cardiac arrest in a pediatric emergency department”

Community-Based Research
Faculty: Adeline (Wei Li) Koay, MBBS, MSc, for “Recent trends in the prevention of mother-to-child transmission (PMTCT) of HIV in the Washington, D.C., metropolitan area”
Staff: Gia M. Badolato, MPH, for “STI screening in an urban ED based on chief complaint”
Post docs/fellows/residents: Christina P. Ho, M.D., for “Pediatric urinary tract infection resistance patterns in the Washington, D.C., metropolitan area”
Graduate students: Noushine Sadeghi, BS, “Racial/ethnic disparities in receipt of sexual health services among adolescent females”

Education, Training and Program Development
Faculty: Cara Lichtenstein, M.D., MPH, for “Using a community bus trip to increase knowledge of health disparities”
Staff: Iana Y. Clarence, MPH, for “TEACHing residents to address child poverty: an innovative multimodal curriculum”
Post docs/fellows/residents: Johanna Kaufman, M.D., for “Inpatient consultation in pediatrics: a learning tool to improve communication”
High school/undergraduate students: Brett E. Pearson for “Analysis of unanticipated problems in CNMC human subjects research studies and implications for process improvement”

Quality and Performance Improvement
Faculty: Vicki Freedenberg, Ph.D., APRN, for “Implementing a mindfulness-based stress reduction curriculum in a congenital heart disease program”
Staff: Caleb Griffith, MPH, for “Assessing the sustainability of point-of-care HIV screening of adolescents in pediatric emergency departments”
Post docs/fellows/residents: Rebecca S. Zee, M.D., Ph.D., for “Implementation of the Accelerated Care of Torsion (ACT) pathway: a quality improvement initiative for testicular torsion”
Graduate students: Alysia Wiener, BS, for “Latency period in image-guided needle bone biopsy in children: a single center experience”

View images from the REW2019 award ceremony.