Pulmonology and Sleep Medicine News

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Children’s National in the News: 2023

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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)

2023 with a lightbulb

The best of 2023 from Innovation District

2023 with a lightbulbAdvanced 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)

doctor talking to young girl with asthma

Caregiver language preference is associated with asthma outcomes

doctor talking to young girl with asthma

A team of researchers found that language barriers can contribute to increased asthma-related healthcare utilization including emergency department (ED) visits, hospitalizations and intensive care unit (ICU) visits.

Asthma is one of the most common chronic pediatric diseases, affecting more than 4 million children and accounting for approximately 24% of pediatric hospitalizations nationwide. Asthma disproportionally impacts historically marginalized racial and ethnic minorities in the United States, with these populations experiencing increased unscheduled asthma-related healthcare utilization and worse asthma outcomes. Families that speak languages other than English can experience increased patient safety events and worse outcomes in other disease processes, but limited data exists on the role of caregiver language preference on asthma morbidity.

The big picture

In a study published in Pediatrics, a team of researchers found that language barriers can contribute to increased asthma-related healthcare utilization including emergency department (ED) visits, hospitalizations and intensive care unit (ICU) visits.

“Families who speak languages other than English can experience significant barriers to quality healthcare from how they navigate a complex healthcare system to how they are able to communicate with their healthcare providers and receive timely care,” said Mickey Emmanuel, M.D., a pediatric emergency medicine fellow at Children’s National Hospital and lead author of the study. “Understanding how caregiver language preference contributes to pediatric asthma outcomes is crucial.”

What’s been the hold up in the field?

Prior research has characterized that caregivers with non-English language preference or NELP were less likely to receive education on asthma disease management and to be given asthma action plans in their preferred language or use asthma action plans. To date, limited data exists on the role of caregiver language preference on asthma utilization.

“Future studies utilizing qualitative and quality improvement methodology that puts the family’s needs at the center are key,” says Dr. Emmanuel. “Efforts to reduce asthma-related ED visits and hospitalizations for families who speak languages other than English must focus on understanding the unique barriers that caregivers face in caring for their children with asthma, and on delivering linguistically competent asthma care in the ambulatory and hospital settings.”

What’s next?

“This work has allowed us to identify that caregiver language preference is an important determinant for asthma outcomes and will hopefully fuel additional research focused on improving care for this population,” says Dr. Emmanuel.

From here, Children’s National will continue to work with notable mentors and leaders in the Language Equity space, with a clear commitment toward improving care for this population of patients.

Additional Children’s National researchers include: Rachel Margolis, Ph.D., Ranjodh Badh, B.A., Nikita Kachroo, A.E.-C., Stephen J. Teach, M.D., MPH,  and Kavita Parikh, M.D., MSHS.

Doctor helping child with asthma inhaler

Improving socioeconomic and educational factors may reduce asthma rates

Doctor helping child with asthma inhaler

Researchers believe that the characteristics of a child’s neighborhood may be driving inequities in rates of early childhood asthma.

Asthma is one of the most common, chronic pediatric diseases in the United States, complicated by persistent disparities in care and outcomes. While hospital systems are determined to tackle this issue, researchers believe that the characteristics of a child’s neighborhood may be driving inequities in rates of early childhood asthma.

In a study published in The Journal of Allergy and Clinical Immunology, researchers found that neighborhoods with improved socioeconomic and educational opportunity were more likely to have lower rates of asthma-related emergency department (ED) visits among children younger than five years old in Washington, D.C.

“The Child Opportunity Index (COI) incorporates 29 different measures of social determinants of health into one single measure. Our findings highlight that higher overall opportunity scores of a child’s neighborhood are associated with lower rates of emergency room visits for asthma-related illness,” says Jordan Tyris, M.D., a hospitalist and lead author of the study. “In particular, the socioeconomic and educational measures drove this association. This tells us that improving socioeconomic and educational factors in areas with higher asthma-related ED utilization might help reduce how often children are getting sick from their asthma.”

In this cross-sectional study, researchers examined 3,806 children who were under the age of 5 with physician-diagnosed asthma, who were included in the Washington, D.C., Pediatric Asthma Registry between January 2018 and December 2019. Children in this age group experience disproportionately high rates of asthma. Of the 3,806 children, 2,132 (56%) kids had a collective 5,852 ED encounters, and 821 (22%) of kids had a collective 1,418 hospitalizations.

According to Dr. Tyris, researchers had to work to define cohorts of children under 5 years of age with diagnosed asthma, due to the considerable overlap in early childhood between the clinical presentations of virally induced wheezing and asthma. Ultimately, this presents a barrier to conducting research that exclusively focuses on this young population.

“These findings highlight the importance of considering efforts to improve social, educational and economic-related characteristics of communities as another method to reduce asthma morbidity in early childhood,” said Dr. Tyris.

Patient and doctor demoing Rare-CAP technology

M.D. in your pocket: New platform allows rare disease patients to carry medical advice everywhere

When someone has a rare disease, a trip to the emergency room can be a daunting experience: Patients and their caregivers must share the particulars of their illness or injury, with the added burden of downloading a non-specialist on the details of a rare diagnosis that may change treatment decisions.

Innovators at Children’s National Hospital and Vanderbilt University Medical Center, supported by Takeda, are trying to simplify that experience using a new web-based platform called the Rare Disease Clinical Activity Protocols, or Rare-CAP. This revolutionary collection of medical information allows patients to carry the latest research-based guidance about their rare disorders in their phones, providing a simple QR code that can open a trove of considerations for any medical provider to evaluate as they work through treatment options for someone with an underlying rare disease.

“No one should worry about what happens when they need medical help, especially patients with rare diseases,” said Debra Regier, M.D., division chief of Genetics and Metabolism at Children’s National and Rare-CAP’s lead medical advisor. “We built this new tool because I have watched as my patient-families have wound up in an emergency room — after all, kids get sprains or fractures — but they don’t have the expertise of a rare disease specialist with them. My hope is that they’re going to pull out their phones and access Rare-CAP, which will explain their rare disease to a new provider who can provide more thoughtful and meaningful care.”

The big picture

A rare disease is defined as any disorder that affects less than 200,000 people in the United States. Some 30 million Americans are believed to be living with one of the 7,000 known rare disorders tracked by the National Organization of Rare Diseases (NORD). Led by Dr. Regier, the Rare Disease Institute at Children’s National is one of 40 NORD centers for excellence in the country that provide care, guidance and leadership for the wide array of disorders that make up the rare disease community.

While a key goal of Rare-CAP is to bolster patient self-advocacy, the platform will also allow medical providers to proactively search for protocols on rare diseases when they know they need specialized advice from experts at Children’s National, a network of tertiary care centers and patient organizations.

As a leading values-based, R&D-driven biopharmaceutical company, Takeda has committed $3.85 million to the project to help activate meaningful change and empower a brighter future for rare disease communities, providing a unique understanding of the struggle that patients and caregivers face when they need care.

“Our team, alongside the medical and rare disease community, saw the need for a single portal to collect standardized care protocols, and we are thrilled to see this innovative tool come to life,” said Tom Koutsavlis, M.D., head of U.S. Medical Affairs at Takeda. “People with rare diseases and their caregivers need faster access to authoritative medical information that providers anywhere can act on, this will lead to improving the standard of care, accelerating time to diagnosis and breaking down barriers to increase equitable access.”

The patient benefit

The creators of Rare-CAP imagined its use in a wide range of settings, including emergency rooms, surgical suites, dental offices, urgent care offices and school clinics. The platform will eventually profile thousands of rare diseases and lay out the implications for care, while also creating a dynamic conversation among users who can offer updates based on real-world experience and changes in medical guidance.

“Our patients are unique, and so is this tool,” Dr. Regier said. “As we roll out Rare-CAP, we believe it is just the beginning of the conversation to expand the platform and see its power for the patient and provider grow, with each entry and each new rare disease that’s added to the conversation.”

U.S. News Badges

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

U.S. News BadgesChildren’s National Hospital in Washington, D.C., was ranked #5 in the nation on the U.S. News & World Report 2023-24 Best Children’s Hospitals annual rankings. This marks the seventh straight year Children’s National has made the Honor Roll list. The Honor Roll is a distinction awarded to only 10 children’s hospitals nationwide.

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

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

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

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

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

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

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

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

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

William Sheehan

Allergist taps into biologics to treat asthma



6th Annual Children’s National Hospital – NIAID Symposium
Promoting Child Health: From Environmental Challenges to Pandemic
April 27 – 28, 2023 | Children’s National Research & Innovation Campus | 7144 13th Pl NW, Washington, DC 20012



 

William SheehanWilliam Sheehan, M.D., joined Children’s National Hospital in 2017 as a board-certified allergist and immunologist with a passion for helping patients with asthma, allergies, eczema and immunodeficiency. During his fellowship at Children’s Hospital of Boston, he investigated the impact of inner-city and school environments on children’s asthma, and he found reward in helping children breathe easier. Dr. Sheehan will be discussing the impact of the environment on childhood asthma at the upcoming CNH-NIAID Symposium on Promoting Child Health: From Environmental Challenges to Pandemic. He shared with Innovation District an overview of his research and his hope for using biologics to treat asthma.

Q: Could you talk about the research you have been involved in?

A: Since my fellowship, my work has been specifically targeting the interaction between environmental exposures in school and asthma. By intervening in the school environment, we could help dozens of children with asthma. In recent years and here at Children’s National, I have become interested in research regarding using biological medicines to treat inner-city children with asthma. These antibody medications, given by injection, target cells that cause inflammation and are used in children who have severe asthma that is not responding to conventional therapies.

Q: What is new about biologics for treating childhood asthma? How are researchers at Children’s National contributing to the field?

A: The first biologic medicine for treating asthma, called Omalizumab, was approved in 2003. In the last five years, four more biologics have been approved for treating childhood asthma, with more in the pipeline. At Children’s National, we have been trying to identify which biologic therapy is most effective for a particular child with asthma by looking at specific biomarkers. We also are examining new biomarkers that may be beneficial. Specifically, we are looking at the levels of regulatory B cells, an immune cell that acts as a brake to control allergic inflammation.  We have shown that these cells are lacking in children with severe asthma and may make these children better candidates for biologic therapies.

Q: How prevalent are young patients in the asthma population in D.C., and do you believe environmental factors associated with living in a city contribute to the higher incidence of asthma in this population?

A: D.C. is a challenging city for pediatric asthma. Not only is asthma prevalence higher here, but we also see higher rates of severe asthma requiring emergency visits, hospitalizations and even admissions to the Pediatric Intensive Care Unit.

While environmental factors such as pollution, second-hand smoke and exposure to mice and cockroaches can contribute to childhood asthma, I don’t think it’s the sole factor in D.C. There might be multiple factors at play, including social structures that make it difficult for families to access and maintain asthma care and medications. Additionally, there seems to be a genetic component as Black and Hispanic children tend to suffer from worse asthma than white children, regardless of the environment.

Q: What specific research areas or topics interest you for future study?

A: We have made significant progress in controlling asthma with biologics, but prevention is the next frontier. Current studies are focusing on treating vulnerable children at a very young age with the goal of preventing the development of asthma later in childhood. If we can’t prevent asthma, we should strive to achieve asthma “remission” through the use current medications and biologic therapies. The goal would be for individuals to go through their year without any symptoms or exacerbations, essentially living as though they don’t have asthma at all.

baby with with bronchopulmonary dysplasia

A team approach to complex bronchopulmonary dysplasia

“By the time a baby is diagnosed with bronchopulmonary dysplasia, families have already had a long journey with prematurity in the neonatal intensive care unit (NICU),” says Hallie Morris, M.D., neonatologist and lead of the Complex Bronchopulmonary Dysplasia (BPD) Program at Children’s National Hospital. “To be able to have a team that is focused on the holistic health of their child in the context of this diagnosis makes a world of difference to these families.”

The big picture

Some premature infants with BPD experience more severe respiratory disease with comorbidities associated with their underlying disease processes, but also factors related to their lengthened intensive care unit (ICU) stay. This includes delayed development with neurodevelopmental impairment, ICU delirium, pulmonary hypertension, airway disease, gastroesophageal reflux disease, feeding difficulties, retinopathy of prematurity and more.

The Complex BPD Program at Children’s National encompasses a group of specialists dedicated to improving the care of infants with BPD and other chronic lung disease of infancy. BPD places extreme demands on families. Education is a critical component for families and our team works to make sure they are well informed, have realistic expectations and understand their care plan.

What they’re saying

  • “Our program is unique in that it has the ability to follow the patient for several months in the NICU as well as after discharge,” says Maria Arroyo, M.D., pulmonologist and co-lead of the Complex BPD Program at Children’s National. “This includes a subacute facility where some of our NICU patients transfer to for continued respiratory weaning and rehabilitation with parent education and outpatient visits once families are home.”
  • “Since this program was created, we have improved patient care and outcomes with this interdisciplinary approach,” says John Berger, M., medical director for the Pulmonary Hypertension Program at Children’s National. “We expect that with our consistent and personalized care, patients will continue have better overall outcomes, less readmissions and improved neurodevelopmental outcomes.

Learn more about the Complex BPD Program.

person pouring pills from bottle to hand

Therapeutic antibiotics associated with reductions in microbial diversity in CF

person pouring pills from bottle to hand

Researchers found that both insufficient beta-lactam pharmacokinetics (PK) and broad-spectrum antibiotics were associated with a greater decrease in species richness at the end of antibiotic therapy compared to pulmonary exacerbations onset.

There are more than 70,000 children and adults living with cystic fibrosis (CF) worldwide. Those with this progressive disease frequently suffer from recurrent episodes of lung infection and inflammation called pulmonary exacerbations.

In a new observational study led by Andrea Hahn, M.D., infectious diseases specialist at Children’s National Hospital, researchers found that both insufficient beta-lactam pharmacokinetics (PK) and broad-spectrum antibiotics were associated with a greater decrease in species richness at the end of antibiotic therapy compared to pulmonary exacerbations onset.

In prior studies evaluating the association between beta-lactam PK, insufficient beta-lactam PK was associated with reduced short-term decreases in microbial diversity compared to sufficient beta-lactam dosing. In this study researchers found that insufficient beta-lactam PK was associated with a greater short-term decrease in microbial diversity.

Dr. Hahn’s team also found that an increased presence of beta-lactam antibiotic resistance genes was associated with lower microbial diversity and lower lung function.

These studies suggest that community-level antibiotic resistance, rather than the resistance patterns of the most prevalent bacteria identified in cultures, may serve as a useful predictor of lung function recovery in individuals with cystic fibrosis (CF). This finding may aid clinicians in selecting the most effective antibiotics to treat pulmonary exacerbations in CF patients, thus enhancing their clinical outcomes.

Read the full study in Nature’s Scientific Reports.

Authors on the study from Children’s National Hospital include Andrea Hahn, M.D., M.S., Aszia Burrell, Hollis Chaney, M.D.Iman Sami-Zakhari, M.D.Anastassios Koumbourlis, M.D., M.P.H., and Robert J. Freishtat, M.D., M.P.H.

boy using spirometer

Hydroxyurea initiation improves spirometry results in children with sickle cell anemia

boy using spirometer

Hydroxyurea therapy may help preserve lung function over time in children with sickle cell anemia.

Children’s National researchers participated in a recent study suggesting that the use of hydroxyurea (HU) therapy in children with sickle cell anemia (SCA) may help preserve lung function over time.

Sickle cell disease is associated with various pulmonary complications, including reactive airways, acute chest syndrome (ACS), pulmonary fibrosis and pulmonary arterial hypertension, which can lead to increased morbidity and mortality. Lung function tends to decline over time in individuals with SCA, and a more irreversible restrictive pathology develops with age and increasing respiratory complications.

Hydroxyurea is a disease-modifying therapy that has been shown to lower the rates of several complications of SCD, such as ACS and painful vaso-occlusive crises. It also might help lessen the severity of airway hyperreactivity in children.

This study provides evidence that hydroxyurea therapy may have a positive impact on pulmonary function in children with SCA, which could be of interest to doctors treating this patient population. By slowing the decline in lung function, hydroxyurea therapy may help reduce the risk of pulmonary complications and improve the overall health outcomes of children with SCA.

You can read the full study, Spirometric Changes After Initiation of Hydroxyurea in Children With Sickle Cell Anemia, in the Journal of Pediatric Hematology/Oncology.

Authors on the study from Children’s National Hospital include Dinesh Pillai, M.D., Deepika Darbari, M.D., and Anastassios Koumbourlis, M.D., M.P.H.

girl looking at medicine bottle

Effect of antibiotics on microorganisms and lung function in children with CF

girl looking at medicine bottle

The study suggests that the use of antibiotics to treat PEx in children with CF may not be as harmful to the airway microbiome as previously believed.

Cystic fibrosis (CF) is a disease that affects many people, especially children. Pulmonary exacerbations (PEx) are common in people with CF and can cause a decline in lung function. These PEx are often treated with antibiotics, but little is known about how antibiotics affect the airway microbiome (the collection of microorganisms in the lungs) of people with CF over time.

Experts from Children’s National Hospital took part in a recent study which looked at how the airway microbiome and lung function of children with CF changed over the course of a year following an initial PEx. The study found that the diversity of the airway microbiome increased over the year despite a decrease in lung function associated with repeated PEx events requiring antibiotic therapy. This suggests that repeated treatment with antibiotics may not have a negative impact on the overall diversity of microorganisms in the lungs.

It is important for pediatricians to understand how antibiotics affect the airway microbiome in children with CF because it can help them make more informed decisions about treatment options. The findings of this study suggest that the use of antibiotics to treat PEx in children with CF may not be as detrimental to the airway microbiome as previously thought. This information can help pediatricians provide better care for children with CF and ultimately improve their overall health outcomes.

You can read the full study, Impact of Antibiotics on the Lung Microbiome and Lung Function in Children With Cystic Fibrosis 1 Year After Hospitalization for an Initial Pulmonary Exacerbation, in Open Forum Infectious Diseases.

Authors on the study from Children’s National Hospital include Zaina Inam, M.D., Aszia Burrell, Hollis Chaney, M.D., Iman Sami-Zakhari, M.D., Anastassios Koumbourlis, M.D., M.P.H., Robert J. Freishtat, M.D., M.P.H., and Andrea Hahn, M.D., M.S.

asthma inhaler

New study links T cells and airway muscle in obese children with asthma

asthma inhaler

The study found that T cells from obese children with asthma adhere more to the airway muscle than T cells from lean children with asthma.

Obesity-related asthma is a distinct type of asthma that is increasing among children due to an increase in obesity. It is associated with high disease burden that is not responsive to currently available asthma therapies. Researchers from Children’s National are leading the way with the first study of its kind to show a link between T cells and airway muscle. This unique work represents an important step forward in understanding pediatric obesity-related asthma and has the potential to inform the development of new treatments.

What’s been the hold-up in the field?

Researchers do not understand the mechanisms that underlie obesity-related asthma. This lack of knowledge directly affects the ability to find medications that are effective against this disease.

How does this work move the field forward?

The study found that T cells from obese children with asthma adhere more to the airway muscle than T cells from lean children with asthma. This adhesion of T cells to airway muscle causes the muscle to be more contractile. This is the first study to link immune cells in obese children with asthma with airway-specific changes.

How will this work benefit patients?

The study has identified a mechanism where immune cells react with airway muscle. As part of this, few proteins will be studied further to see if they can be targets for medications specifically for obesity-related asthma.

How is Children’s National leading in this space?

Children’s National is leading in this space by initiating a first of its kind study to show the link between the T cells and airway muscle.

Authors on the study from Children’s National Hospital include: Deepa Rastogi, M.D., M.S., Changsuek Yon, Ph.D., and David A. Thompson.

You can read the full study, Crosstalk Between CD4+ T Cells and Airway Smooth Muscle in Pediatric Obesity-related Asthma, in the American Journal of Respiratory and Critical Care Medicine.

Abstract Happy 2022 New Year greeting card with light bulb

The best of 2022 from Innovation District

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

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

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

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

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

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

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

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

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

5. New telehealth command center redefines hospital care

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

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

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

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

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

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

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

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

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

10. 2022: Pediatric colorectal and pelvic reconstructive surgery today

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

Baby on ventilator

Autonomic markers of extubation readiness in premature infants

Baby on ventilator

Premature infants often require a breathing tube and mechanical ventilation as a mainstay in their therapy. It can be difficult to predict when these patients are ready to come off the ventilator (extubate).

Premature infants often require a breathing tube and mechanical ventilation as a mainstay in their therapy. It can be difficult to predict when these patients are ready to come off the ventilator (extubate). In a study from Pediatric Research, experts from Children’s National Hospital describe a model to predict the success of extubation using markers of autonomic tone (rest and digest versus fight or flight response). This study was led by Suma Hoffman, M.D., neonatologist and co-authored by Adré J. du Plessis, M.B.Ch.B., M.P.H., division chief of Prenatal Pediatrics Institute, Sarah Schlatterer, M.D., prenatal and neonatal neurologist, and Rathinaswamy B. Govindan, Ph.D.

The team deployed a study of 89 infants less than 28 weeks. Heart rate variability (HRV) metrics 24 hours prior to extubation were compared for those with and without extubation success within 72 hours. Receiver-operating curve analysis was conducted to determine the predictive ability of each metric and a predictive model was created.

Conclusions show that extubation success is associated with HRV. The authors demonstrate an autonomic imbalance with low sympathetic and elevated parasympathetic tone in those who failed. α1, a marker of sympathetic tone, was noted to be the best predictor of extubation success especially when incorporated into a clinical model.

Learn more about the study in Pediatric Research.

lung ct scan

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

lung ct scan

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

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

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

The bottom line

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

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

What they’re saying

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

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

Moving the field forward

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

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

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

Child using inhaler for asthma

Monoclonal antibody reduces asthma exacerbations in urban youth: Q&A with Stephen Teach, M.D.

Child using inhaler for asthmaNew research found that mepolizumab, a monoclonal antibody, decreased asthma flare-ups by 27% in Black and Hispanic children and adolescents who have a severe form of asthma, are prone to asthma attacks, and live in low-income urban neighborhoods.

Stephen Teach, M.D., principal investigator of the IMPACT DC asthma program and co-author of the NIAID-funded Inner City Asthma Consortium-conducted study, offers insight on the importance of this work and what it means for the future of pediatric asthma care at Children’s National Hospital.

What has been the hold-up in this field and how does this work move the field forward?

Urban, Black, and Hispanic children with asthma continue to suffer a disproportionate share of asthma-related morbidity. These findings show that innovative, phenotype-specific therapies are necessary to address their needs.

The study’s investigators were able to document a significant treatment effect in a particularly at-risk population and demonstrated specific gene pathways that were downregulated by treatment with mepolizumab.

What did you find that excites you about this work?

The exciting part of the Inner-City Asthma Consortium’s work has always been that it not only studies whether or not phenotype-driven interventions improve outcomes in high-risk kids, but how those interventions work from a fundamental mechanistic perspective. Those insights drive the science, and the potential clinical applications, forward in an iterative fashion.

How is Children’s National leading in this space?

Investigators from Children’s National continue to be national leaders in developing and testing innovative interventions to address the national disparities in pediatric asthma care and outcomes.

The study, published in the journal The Lancet was co-authored by William Sheehan, M.D., allergist.

The Division of Pulmonary Medicine at Children’s National has been ranked as one of the top ten programs in the nation by U.S. News & World Report.

boy using asthma inhaler

Social determinants of health and asthma morbidity in youth

boy using asthma inhaler

Researchers believe these findings can help develop localized interventions that can improve pediatric asthma in affected communities.

In a study published in the journal Pediatrics, researchers from Children’s National Hospital found that census-tract measures of decreased educational attainment and increased violent crime were associated with increased census-tract rates of pediatric asthma morbidity.

“Knowing these adverse measures of social determinants are associated with increased asthma-related emergency department and hospitalization at-risk rates, may be an opportunity to inform community-based interventions to reduce pediatric asthma morbidity,” says Jordan Tyris, a hospitalist and lead author of the study.

Researchers evaluated data from 15,492 children with asthma, ages 2-17, living in Washington, D.C., from January 2018 to December 2019. The team discovered that living in areas with greater violent crime and less educational achievement were associated with higher rates of hospitalizations and emergency department visits for asthma, in comparison to other social determinants.

The study authors suggest that there may be complex reasons behind this data, noting that violent crime can reflect toxic stress, less education can be associated with less knowledge about health and medicine and that children in these communities may be less likely to have primary care doctors. Researchers believe these findings can help develop localized interventions that can improve pediatric asthma in affected communities and that more research is needed on the drivers of asthma related sickness, including toxic stress, structural racism and access to medical care.

Other study authors include Anand Gourishankar, M.D., Nikita Kachroo AE-C, Stephen Teach, M.D., Kavita Parikh, M.D., all of Children’s National Hospital and Maranda C. Ward, Ed.D., MPH, of George Washington School of Medicine and Health Sciences.

US News Badges

Children’s National named to U.S. News & World Report’s Best Children’s Hospitals Honor Roll

US News BadgesChildren’s National Hospital in Washington, D.C., was ranked No. 5 nationally in the U.S. News & World Report 2022-23 Best Children’s Hospitals annual rankings. This marks the sixth straight year Children’s National has made the list, which ranks the top 10 children’s hospitals nationwide. In addition, its neonatology program, which provides newborn intensive care, ranked No.1 among all children’s hospitals for the sixth year in a row.

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

“In any year, it would take an incredible team to earn a number 5 in the nation ranking. This year, our team performed at the very highest levels, all while facing incredible challenges, including the ongoing pandemic, national workforce shortages and enormous stress,” said Kurt Newman, M.D., president and chief executive officer of Children’s National. “I could not be prouder of every member of our organization who maintained a commitment to our mission. Through their resilience, Children’s National continued to provide outstanding care families.”

“Choosing the right hospital for a sick child is a critical decision for many parents,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “The Best Children’s Hospitals rankings spotlight hospitals that excel in specialized 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.

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

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

emergency signs

Social risk interventions significantly reduce asthma-related ED visits

emergency signs

In a systematic review and meta-analysis published in JAMA Pediatrics, researchers at Children’s National Hospital found that interventions focused on health, environment and community were associated with the greatest reduction in asthma-related emergency department visits and hospitalizations among children.

In a systematic review and meta-analysis published in JAMA Pediatrics, researchers at Children’s National Hospital found that interventions focused on health, environment and community were associated with the greatest reduction in asthma-related emergency department visits and hospitalizations among children. The findings suggest that addressing social risks may be a crucial component of pediatric asthma care to improve health outcomes.

“There are persistent and striking disparities in asthma outcomes among children,” said Jordan Tyris, M.D., hospitalist fellow at Children’s National and lead author. “Understanding how to address these is of utmost importance.”

“Literature on the spectrum of social factors, including social needs, social risks and social determinants, has increased recently across many aspects of health care,” adds Dr. Tyris. “But much of this literature has focused on adults with chronic conditions, for example diabetes or high blood pressure.”

The study authors searched PubMed, Scopus, PsychINFO, SocINDEX and CINAHL from January 2008 to June 2021 for U.S.-based studies evaluating the associations of interventions addressing one or more social risks with asthma-related emergency department visits and hospitalizations among children. The systematic review included 38 of the original 641 identified articles (6%), and the meta-analysis included 19 articles (3%). Overall, participation in social risk–based interventions, particularly those that addressed health literacy, home environmental conditions and peer support were associated with significantly reduced risks for asthma-related emergency department visits and hospitalizations among children.

You can read the full study, “Social Risk Interventions and Health Care Utilization for Pediatric Asthma: A Systematic Review and Meta-analysis” in JAMA Pediatrics.

boy using a nebulizer

Algorithm for antibiotic use benefits patient care in cystic fibrosis

boy using a nebulizer

Despite national consensus guidelines for antibiotic treatment for pulmonary exacerbations in people with CF, prior research and systematic reviews have repeatedly demonstrated a longstanding lack of sufficient evidence for empiric antibiotic therapy recommendations, leading to a significant variation in how antibiotics are prescribed across CF care centers in the United States.

In a recent quality improvement project, researchers from Children’s National Hospital found that antimicrobial stewardship initiatives are beneficial in standardizing care and fostering positive working relationships between cystic fibrosis (CF) pulmonologists, infectious disease physicians and pharmacists.

Antimicrobial stewardship is a systematic effort to change prescribing attitudes that can provide benefit for people with CF. The objective of this effort was to decrease unwarranted use of broad-spectrum antibiotics for children and adolescents with CF. Through initiation of the empiric antibiotic algorithm, researchers found that the proportion of pulmonary exacerbations with antibiotic use consistent with the algorithm increased from 46.2% to 79.5%.

“This work will provide people with CF the expectation of a more consistent approach in their care, as well as the benefits of care from a multidisciplinary team of experts,” said Andrea Hahn, M.D., an infectious diseases specialist at Children’s National and co-author. “Implementing antimicrobial stewardship in the context of care to persons with CF provides benefit in this complex patient population.”

Despite national consensus guidelines for antibiotic treatment for pulmonary exacerbations in people with CF, prior research and systematic reviews have repeatedly demonstrated a longstanding lack of sufficient evidence for empiric antibiotic therapy recommendations, leading to a significant variation in how antibiotics are prescribed across CF care centers in the United States.

The researchers found that implementing antimicrobial stewardship when caring for people with CF provides benefit in this complex patient population.

“We would encourage other CF centers to explore their own trends in practice to determine whether a similar intervention may be both feasible and beneficial in the treatment of pulmonary exacerbations in persons with CF,” said Dr. Hahn.