Gastroenterology & GI Surgery

2024 with a lightbulb instead of a zero

The best of 2024 from Innovation District

2024 with a lightbulb instead of a zero2024 marked another groundbreaking year for Children’s National Hospital, showcasing remarkable advances across the spectrum of pediatric medicine, research and healthcare innovation. From pioneering surgical procedures to breakthrough artificial intelligence applications, the institution continued to push the boundaries of what’s possible in children’s healthcare. Read on for our list of the most popular articles we published on Innovation District in 2024.

1. Prenatal COVID exposure associated with changes in newborn brain

A study led by researchers at Children’s National Hospital showed that babies born during the COVID-19 pandemic have differences in the size of certain structures in the brain, compared to infants born before the pandemic. The findings suggest that exposure to the coronavirus and being pregnant during the pandemic could play a role in shaping infant brain development.
(3 min. read)

2. Children’s National Hospital again ranked among the best in the nation by U.S. News & World Report

Children’s National Hospital was ranked as a top hospital in the nation by the U.S. News & World Report 2024-25 Best Children’s Hospitals annual rankings. This marks the eighth 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.
(2 min. read)

3. Children’s National performs first ever HIFU procedure on patient with cerebral palsy

In January 2023, a team of multidisciplinary doctors performed the first case in the world of using bilateral high intensity focused ultrasound (HIFU) pallidotomy on Jesus, a 22-year-old patient with dyskinetic cerebral palsy. The procedure is part of a clinical trial led by Chima Oluigbo, M.D., pediatric neurosurgeon at Children’s National Hospital.
(3 min. read)

4. Novel ultrasound device gets FDA breakthrough designation with Children’s National support

A novel ultrasound device developed by Bloom Standard received the Food and Drug Administration’s valued breakthrough device designation with the help of Children’s National Hospital. The device that enables autonomous, hands-free ultrasound scans to be performed anywhere, by any user.
(2 min. read)

5. First-of-its-kind pilot study on the impacts of Lyme disease in pregnancy and infant development

Understanding the effects of Lyme disease on the developing fetal brain is essential to ensure timely prenatal and postnatal treatments to protect the fetus and newborn. In response to this need, Children’s National Hospital is leading a pilot study to establish the groundwork needed for a larger study to determine the effect of in utero exposure to Lyme disease on pregnancy and early childhood neurodevelopmental outcomes.
(3 min. read)

6. Earliest hybrid HLHS heart surgery kids thrive 5 years later

Five years ago, Cayden was born 6 weeks early weighing less than four pounds and at risk of dying from her critical congenital heart disease. Today, she’s a happy five-year-old. Early diagnosis of her hypoplastic right ventricle, double inlet left ventricle and critical coarctation of the aorta allowed for the team at Children’s National Hospital to create a careful plan for safe delivery and to offer an innovative hybrid HLHS surgical approach at the hospital within 24 hours after she was born.
(1 min. read)

7. Wayne J. Franklin, M.D., F.A.C.C., named senior vice president of the Children’s National Heart Center

Children’s National Hospital appointed Wayne J. Franklin, M.D., F.A.C.C., as the new senior vice president (SVP) of the Children’s National Heart Center. In this role, Dr. Franklin oversees the full spectrum of heart care services including cardiac imaging and diagnostics, interventional cardiology, electrophysiology, cardiac anesthesia, cardiac surgery and cardiac intensive care.
(2 min. read)

8. Artificial – and accelerated – intelligence: endless applications to expand health equity

By pioneering artificial intelligence (AI) innovation programs at Children’s National Hospital, Marius George Linguraru, D.Phil., M.A., M.Sc., and the AI experts he leads are ensuring patients and families benefit from a coming wave of technological advances. The team is teaching AI to interpret complex data that could otherwise overwhelm clinicians.
(4 min. read)

9. Evidence review: Maternal mental conditions drive climbing death rate in U.S.

Painting a sobering picture, a research team led by Children’s National Hospital culled years of data demonstrating that maternal mental illness is an under-recognized contributor to the death of new mothers. They called for urgent action to address this public health crisis.
(3 min. read)

10. Nathan Kuppermann, M.D., M.P.H., named chief academic officer and chair of Pediatrics

Children’s National Hospital appointed Nathan Kuppermann, M.D., M.P.H., as its new executive vice president, chief academic officer and chair of Pediatrics. In this role, Dr. Kuppermann oversees research, education and innovation for the Children’s National Research Institute as well as academic and administrative leadership in the Department of Pediatrics at George Washington University School of Medicine & Health Services.
(2 min. read)

11. First global clinical trial achieves promising results for hypochondroplasia

Researchers from Children’s National Hospital presented findings from the first clinical trial of the medication vosoritide for children with hypochondroplasia – a rare genetic growth disorder. During the phase 2 trial, researchers found vosoritide increased the growth rate in children with hypochondroplasia, allowing them to grow on average an extra 1.8 cm per year.
(2 min. read)

12. Pioneering research center aims to revolutionize prenatal and neonatal health

Since its establishment in July 2023, the Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital has gained recognition through high-impact scientific publications, featuring noteworthy studies exploring the early phases of human development.
(3 min. read)

2024-25 US News Badges

Children’s National again ranked among the best in the nation by U.S. News & World Report

2024-25 US News BadgesChildren’s National Hospital in Washington, D.C., was ranked as a top hospital in the nation by the U.S. News & World Report 2024-25 Best Children’s Hospitals annual rankings. This marks the eighth 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.

This year, U.S. News ended ordinal rankings on its Honor Roll. Instead of assigning a numerical rank from 1 to 10, all hospitals on the Honor Roll will be recognized as having attained the highest standards of care in the nation.

In addition, Children’s National tied for #1 pediatric hospital in the Mid-Atlantic region, which includes New York, New Jersey, Delaware, Pennsylvania, the District of Columbia, West Virginia and Virginia. It’s also best in the Mid-Atlantic in Neonatology.

For the fourteenth straight year, Children’s National ranked in 10 specialty services. New this year, U.S. News included behavioral health as a service line in the rankings. Since it’s the first year, there are no ordinal rankings for behavioral health, but the Children’s National program was named one of the top 50 programs in the country.

“In my first year here, I witnessed what makes Children’s National so special — our commitment to collaboration, empowering one another, and charting a bold path forward for pediatric care,” said Michelle Riley-Brown, MHA, FACHE, president and chief executive officer of Children’s National. “I’m proud U.S. News again recognized Children’s National as one of the top in the nation and the highest-ranked pediatric hospital in D.C., Maryland and Virginia. Together, we’ll continue to push the boundaries of care, research and innovation to make a difference for those who matter most — the kids.”

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 nearly two decades, U.S. News has published Best Children’s Hospitals to empower the parents and caregivers of children with complex medical needs,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Children’s hospitals appearing on the U.S. News Honor Roll have a track record of delivering unparalleled 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 Children’s National specialty services that U.S. News ranked in the top 10 nationally are:

The other four specialties ranked among the top 50 are Behavioral Health, Cardiology and Heart Surgery, Pulmonology and Lung Surgery, and Urology.

colorectal 5 year infographic

Division of Colorectal & Pelvic Reconstruction: 5 year highlights

colorectal 5 year infographic

Dr. Marc Levitt with a young patient

Marc Levitt, M.D., reflects on the Colorectal & Pelvic Reconstruction Program

Dr. Marc Levitt with a young patient

Dr. Levitt shares insights into the program’s journey, key successes and future goals.

This September, the Colorectal and Pelvic Reconstruction Program at Children’s National proudly marks five years of commitment to patient-centered care for children and their families. Under the guidance of Division Chief Marc Levitt, M.D., the program has achieved remarkable advancements in clinical care, improving patient outcomes and enhancing family experiences.

Dr. Levitt shares insights into the program’s journey, key successes and future goals, highlighting the importance of collaboration among specialists and the continuous pursuit of innovative treatments.

Q: What was your vision for the program when it first launched? Has the vision changed?

A: When the Colorectal Program first launched, we aimed to establish a comprehensive program for colorectal patient care. This included integrating specialists across multiple disciplines, such as pediatric general surgery, urology, gynecology, gastroenterology, nursing, neurosurgery, orthopedics, pathology, radiology, anesthesia, psychology and pelvic floor therapy. Our efforts have exceeded expectations; the team has developed a cohesive and collaborative dynamic where each specialist contributes effectively to patient care. This integrated approach enhances outcomes, as patients and their families are at the center of our model, surrounded by a network of dedicated caregivers and coordinators, all focused on improving the patients’ quality of life.

Q: Can you highlight some key successes or achievements of the program?

A: The creation of our integrated colorectal care program at Children’s National has been a significant success, bolstered by strong institutional support. While this model requires considerable effort, it ultimately attracts patients and leads to outstanding outcomes. We’ve received patient inquiries from 48 states (AK,AL, AR, AZ, CA, CO, CT, D.C., DE, FL, GA, HI, IA, ID, IL, IN, KS, KY, LA, MA, MD, MI, MN, MO, MS, MT, NC, ND, NE, NH, NJ, NM, NV, NY, OH, OK, OR, PA, RI, SC, TN, TX, UT, VA, VT, WA, WI, WV) and 68 countries (Australia, Bahamas, Bangladesh, Bulgaria, Canada, Chile, Croatia, Cyprus, Dominican Republic, Ecuador, Egypt, England, Ethiopia, France, Germany, Greece, Guatemala, Haiti, Hong Kong, India, Ireland, Israel, Jamaica, Jordan, Kazakhstan, Kenya, Kuwait, Lithuania, Maldives, Mauritania, Mexico, Monaco, Morocco, Myanmar, The Netherlands, New Zealand, Nepal, Nigeria, Northern Ireland, Norway, Oman, Palestine, Pakistan, Paraguay, Papua New Guinea, Peru, Philippines, Portugal, Romania, Russia, Saint Lucia, Saudi Arabia, Scotland, Serbia, Singapore, Slovakia, Slovenia, Spain, Sri Lanka, South Korea, St. Kitts & Nevis, Sudan, Sweden, Ukraine, Uzbekistan, United Arab Emirates, Venezuela, Wales).

Some key successes include the development of innovative surgical procedures for conditions such as imperforate anus and anorectal malformations, advanced repair techniques for cloacal malformations, integrated care strategies for colonic dysmotility, and novel approaches for the newborn care of patients with cloacal exstrophy. All these new approaches were developed at Children’s National over the past five years.

In addition to clinical innovations, we have significantly enhanced our academic environment. Over the last five years, our team has authored more than one hundred articles and three well-regarded books, helping health care professionals around the world improve the care they provide to their patients. We have also trained surgical fellows and nurses in colorectal care and welcomed clinicians from over 30 countries for specialized training. This outreach is especially fulfilling, as it enables us to influence the care of children around the globe whom we may never meet, by sharing valuable skills and knowledge with these practitioners. 

Q: Can you share examples of particularly challenging cases or high-profile cases that have influenced the program’s clinical approach?

A: The way we care for a patient with a cloaca is unique – the entire team joins together in the operating room to assess the anatomy, and every member then gives their thoughts on how to approach the reconstruction. The very next day we perform that surgery, with the understanding of the anatomy in mind, and what plays out in that operating room is quite magical. This collaborative approach frequently leads to exceptional outcomes, as our diverse ideas come together to form a cohesive plan. Through this teamwork, we have developed creative ways to solve complex anatomical problems that no one individual surgeon would have thought of on their own.

Q: How have patient outcomes improved over time?

A: We have optimized the collaborative experience so that patients only need a single visit to see all the specialists they require. Our outcomes for complex colorectal surgeries have significantly improved, particularly for conditions like cloaca, Hirschsprung disease and anorectal malformations. Advances in surgical techniques and enhancements in nursing care have led to dramatically better results. We have reduced, and in some cases eliminated, complications from these surgeries, while also seeing improvements in bowel continence and kidney health. Our nursing teams — outpatient, operating room and inpatient — play a crucial role, offering unique insights into pre- and post-operative care that are vital for achieving successful outcomes.

Q: What have been the biggest challenges or barriers faced by the program and how have you addressed these challenges?

A: One of the biggest challenges we face is ensuring that patients can get to us. Many patients encounter obstacles with insurance companies that restrict travel outside their network, or they deal with the difficulties of traveling from far away for their surgery. We have worked diligently to address these issues by improving our insurance approval process and making families comfortable during their stay in Washington, D.C., including assistance with accommodations, necessary medical supplies, etc. If we can remove these barriers, we can care for more patients and make the experience easier for them and their families.

Q: How does the colorectal program collaborate with other departments or services? How has the program integrated from various specialties (e.g., urology, gynecology, GI) to enhance patient care?

A: We have specialists who are fully integrated in their roles on the colorectal and pelvic reconstruction team. General pediatric surgeons, urologists, gynecologists and gastroenterologists work both in the colorectal program and in their home program in a uniquely integrated fashion. For example, our urologists each spend half of their time in general urology and the other half in colorectal, handling the urologic aspects of colorectal patients’ care. In this way collaboration is enhanced and encouraged.

Q: What are the future goals or plans for the colorectal program?

A: Our future goals for the colorectal program are focused on expanding our impact and enhancing patient care. First and foremost, we aim to help as many patients as possible. We plan to collaborate with other colorectal centers worldwide to share knowledge and best practices. Training surgeons and nurses remains a priority, as we want to equip as many healthcare professionals as possible with the skills needed in this field. Additionally, we are committed to traveling to the developing world to provide care for children who cannot access services at Children’s National. Finally, we intend to enhance our research efforts, leveraging basic science to investigate and address conditions related to the colon, ultimately seeking to reduce or eliminate associated illnesses.

Audience members at the NIAID Conference

Breaking free of illness: Experts explore solutions for chronic pediatric disorders

  • NIAID Conference Attendees
  • Audience members at the NIAID Conference
  • NIAID Conference Attendees
  • A presentation at the NIAID Conference
  • A presentation at the NIAID Conference
  • A presentation at the NIAID Conference
  • Audience members at the NIAID Conference
  • A presentation at the NIAID Conference
  • Audience members at the NIAID Conference
  • A presentation at the NIAID Conference
  • Audience members at the NIAID Conference
  • A presentation at the NIAID Conference
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When new patients with chronic illnesses come to Roberta DeBiasi, M.D., M.S., division chief of Infectious Diseases, they are often drained of energy, far behind in school and fatigued by the lack of coordinated care among multiple specialists. She envisions a better way to care for these children: a data-driven, multidisciplinary clinic that can help diagnose and treat disorders facing chronically ill children.

In a keynote address, Dr. DeBiasi laid out her vision for improving care during the 7th annual symposium hosted by Children’s National Hospital and the National Institute of Allergy and Infectious Diseases (NIAID): “A new paradigm: Infection-associated chronic illnesses (IACI) affecting children.” Experts from across the country came together to discuss these IACIs, the importance of finding biomarkers to diagnose and monitor them, data-driven therapeutics to treat them, and the urgent need for protocols to guide physicians.

The patient benefit

A range of IACIs – including long COVID, Lyme disease, postural orthostatic tachycardia syndrome (POTS), dysautonomia and more – are stealing normal, active lifestyles from children. Dr. DeBiasi said patients need researchers who understand disease pathogenesis and have standardized disease classification to diagnose and treat these disorders. Because of the complexity of these cases, care delivery and coordination also need to change. Patients, she said, would best be served by beginning with a three-hour appointment in a multidisciplinary clinic with experts from behavioral health, rehabilitative medicine and other specialties.

“When one of these patients comes to us, we feel helpless as physicians,” Dr. DeBiasi said. “There’s no diagnostic, and we don’t know how to treat them…. It is, to me, very energizing that we’re going to be able to have a coordinated way to help these families. But to do that, we have to have standardized disease classification.”

Moving the field forward

In 2017, NIAID and Children’s National launched a clinical research partnership devoted to advancing the health of children with allergic, immunologic, auto-inflammatory and infectious diseases through collaborative research and education. The partnership — co-led by H. Clifford Lane, M.D., NIAID’s deputy director for Clinical Research and Special Projects, and Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National — promotes the prevention, diagnosis, treatment and cure of these childhood diseases. Every year, the partnership organizes and funds a symposium to explore cutting-edge research and pressing issues in pediatric care.

At this year’s gathering, experts examined the specifics of various disorders, including Alexandra Yonts, M.D. She provided new data from the Children’s National Post-Covid Program and explained that patients “are clamoring” for help with symptom management.

“There have been virtually no randomized, controlled trials and very little evidence on any sort of aggressive treatments, or pharmaceutical management options, especially in the pediatric population,” Dr. Yonts said. “Upon recent attendance at some long COVID meetings, there is an expressed concern, especially among pediatric providers, about trying medications off-label in these long COVID patients.”

Miss the symposium? You can learn more about the science they explored in the symposium’s recording on topics including:

  • Potential overarching mechanisms of disease, including pathogen- and host-mediated factors.
  • Identifying potential biomarkers for chronic illnesses
  • Treatments and multidisciplinary approaches for patients with IACIs
  • Patient advocacy
pediatric colorectal surgeons at Children’s National Hospital

Multi-disciplinary collaboration key in any approach to refractory functional constipation

pediatric colorectal surgeons at Children’s National HospitalFor the last several years, the physicians, advance practice providers, and nurses within the Division of Colorectal and Pelvic Reconstruction at Children’s National Hospital have sought to establish standardized protocols for diagnosing and treating children with severe constipation unresponsive to medical management. Their work seeks to create a collaborative approach that brings gastroenterologists with motility expertise and colorectal surgeons together from the start to streamline testing, analysis, and to avoid unnecessary surgical procedures.

To that end, the team has published several studies that make the case for such a protocol based on their experiences managing this unique population.

What it means

In a 2022 study, the team conducted a retrospective review of children referred for retractable functional constipation to the Division of Colorectal and Pelvic Reconstruction at Children’s National. A unique algorithm for assessment and treatment was created based on the data analysis.

The resulting proposed protocol for medically refractory constipation provided a collaborative framework to standardize the work-up and treatment. This protocol led to the use of antegrade flushes via appendicostomy to empty the colon and was able to in most cases avoid a colonic resection, something done much more commonly in the past

A 2023 study further reiterated the importance of these collaborative approaches, stating, “It is vitally important to recognize that constipation exists on a spectrum of severity with multiple potential etiologic factors and therefore treatment must be customized based on the individual’s symptom, anatomic and manometric findings.”

The 2023 study also introduced an enhanced standardized algorithm to evaluate and treat refractory constipation, with the central idea being a consolidation of evaluation and initiation of treatment into a single combined procedure. This means collaboration between gastroenterology and colorectal surgery in the operating room by including anorectal manometry at the start to rule out anatomic anomalies as the cause of the constipation and to test motility.

Why it matters

In the 2023 study, the authors wrote, “While algorithms for initial evaluation and treatment of functional constipation in children have been described, most articles in the pediatric literature lack details about the work-up and management of those refractory symptoms and the approaches described for such patients vary widely among clinicians.”

That means that children with functional constipation for whom medical treatment has failed face a drastically lower quality of life. Functional constipation and encopresis, or stool soiling, correlates with a higher prevalence of low self-esteem, social withdrawal, and depression. It is critical to correctly diagnose and efficiently treat these conditions and work together to do it quickly.

Children’s National leads the way

As one of the nation’s leaders in pediatric colorectal and pelvic reconstructive surgery, Children’s National, led by Division Chief Marc Levitt, M.D., receives more referrals asking for a surgical solution for severe pediatric constipation than any other center in the world.

Caring for such a large population positions the team to analyze evidence and establish protocols based on statistically relevant numbers of patients. The studies outlined above are the result of the innovative design of the Division of Colorectal and Pelvic Reconstruction, in which multiple divisions involved in the care of these patients, from Gastroenterology to Urology and beyond, experience an unprecedented ability to work together, including in the operating room. It is a fully integrated program with experts in colorectal surgery, GI motility, and colorectal nursing embedded into the same team.

While most children with functional constipation will respond to medical treatment, for those who need further help that includes surgical assessments, the evidence-based algorithms developed in these studies can truly be life-changing for these children and their families.

Read the studies in the Journal of Pediatric Surgery:

Marc Levitt, M.D., in the operating room.

Better standardized care for total colonic Hirschsprung disease

Marc Levitt, M.D., in the operating room.

Clinicians can better manage care for children with total colonic Hirschsprung disease by drawing on the collective expertise from the people who have seen and treated patients with this condition, says Marc Levitt, M.D.

Clinicians can better manage care for children with total colonic Hirschsprung disease, a rare form of the condition, by drawing on the collective expertise from the people who have seen and treated patients with this condition, says Marc Levitt, M.D., chief of Colorectal and Pelvic Reconstruction at Children’s National Hospital.

What is it?

Dr. Levitt and fellow experts within the APSA Hirschsprung Disease Interest group are working together to develop recommendations based on their collective experiences managing the condition. Their recommendations will include specifics for the diagnosis and management of total colonic Hirschsprung disease (a rare type of Hirschsprung disease that involves the entire colon) before and after reconstruction, such as diagnostic criteria, surgical approaches, bowel management, diet, antibiotic prophylaxis, colonic irrigations and post-surgical considerations.

Specifically, the group’s consensus findings and recommendations include:

  • Specific information about using a contrast enema for this diagnosis — namely that there is no obvious transition zone identified.
  • The importance of a surgical approach of colonic mapping to obtain definitive pathology and ileostomy at the time of diagnosis, which is key to getting a child with this condition feeding and growing.
  • Monitoring that growth with nutritional assessment and oral sodium supplementation are vital to their care.
  • High output stomas and loose stool after pull-through can be more readily treated now with anti-motility agents, and better care of the parastomal and perineal skin.

Why does it matter?

Children with total colonic Hirschsprung disease are unique among children with Hirschsprung disease. They present specific pre- and post-operative management challenges. Creating and sharing consensus guidelines will create a rational, expert-based approach to diagnosing and managing the condition based on direct clinical experience, that others can draw from. Because the condition is relatively rare, care decisions historically have been made based mainly on literature about it. However, that literature often shows a lack of clear consensus on how to approach the condition.

Children’s National leads the way

Dr. Levitt and his colleagues collectively have seen more cases of total colonic Hirschsprung disease than any other clinicians in the world and have helped to author better guidelines specifically to try to standardize the pre- and post-operative management of this patient group. The goal is to improve the likelihood of a better outcome for the child no matter where a family goes for care.

illustration of a gallbladder

Innovating improved outcomes with robotic gallbladder removal

Patients want to hear they are “in good hands” when choosing a surgeon. A Children’s National Hospital team is investigating whether those hands could be replaced with an autonomous robotic arm during pediatric cholecystectomy procedures.

“The role of autonomous surgery is at a pivot point,” said Anthony Sandler, M.D., senior vice president and surgeon-in-chief at Children’s National and director of the Sheikh Zayed Institute of Pediatric Surgical Innovation (SZI). “Just as it is with autonomously driving cars, we are testing whether a gallbladder removal can be controlled and managed by a robotic arm, rather than a laparoscopic surgeon. In preclinical models, we are evaluating whether we can take the next step forward. We believe we can, and this research will be proof of concept for autonomous surgery.”

The big picture

The rate of cholecystectomy has been on the rise among pediatric patients for the last two decades. It becomes necessary when the gallbladder becomes full of painful gallstones, often caused by obesity and inherited blood disorders like sickle cell disease. Across the country, 99% of gallbladder removals happen without complications.

Yet Children’s National hopes to develop technologies to boost that number even higher. Working with partners, the hospital has embarked on two contracts with the Advanced Research Projects Agency for Health (ARPA-H): a three-year and a two-year contract, worth $3.5 million each (75N91023C00048 & 75N91023C00053, respectively). The Children’s National team is collaborating on solutions with Optosurgical Chief Executive Officer Yoseph Kim, M.S.E., and Axel Krieger, Ph.D., associate professor of mechanical engineering at Johns Hopkins University.

They are tapping into two areas of expertise: robotic surgery and the development of a novel fluorescent dye that – when paired with advanced imaging – can aid surgeons in seeing bleeds during gallbladder removals.

The project also combines the talents of Dr. Sandler and Children’s National optical engineer Richard Cha, Ph.D., principal investigator at SZI, to explore how to integrate these technologies in the operating room.

“Autonomous gallbladder removal involves the identification of the target tissue – the bile duct, the cystic artery and the gallbladder. Our team’s new 3D imaging techniques will help visualize and work through the surgical steps, by locating each target,” Dr. Cha said. “When this technology and related programming come together, it could mark a significant step forward in pediatric surgery.”

The fine print

Given the gallbladder’s small size and accessible location just beneath the liver, cholecystectomy is most often done laparoscopically, using small cameras and incisions. There are three main steps: ligating – or closing off – the cystic artery, ligating the cystic duct while protecting the common bile duct, and removing the gallbladder. Drs. Sandler and Cha believe outcomes will improve if the expertise and delicacy of a robot are incorporated into the procedure.

“If you’re that one patient out of 100 who has significant bleeding post-operatively or, even worse, you are among the 0.5% of patients who have an injury to the bile duct, the impact on your wellbeing is significant after that surgery,” Dr. Sandler said. “Having that extra security of technology and guidance will be an incredible value-add for any patient undergoing this procedure.”

These projects have been funded in whole with federal funds from ARPA-H, National Institutes of Health, Department of Health and Human Services, under Contract No. 75N91023C00053 and Contract No. 75N91023C00048.

Dr. Craig Sable in Uganda

Around the world

Our Global Health Initiative launched in 2016 with the goal of eliminating pediatric health disparities around the world. We aim to address the most pressing pediatric health issues through better care for medically underserved populations. This leadership helps us achieve our mission of caring for all children. A broad range of education and research projects improves health outcomes. They also offer enriching opportunities for experienced faculty and emerging leaders to advance clinical excellence.

Healing hearts in Uganda

Dr. Craig Sable in Uganda

Dr. Craig Sable and team train partners in Uganda.

Craig Sable, M.D., interim chief of Cardiology, improves care for young people with rheumatic heart disease (RHD) in Uganda. Donors, including the Karp Family Foundation, Huron Philanthropies, Zachary Blumenfeld Fund and the Wood family, make this possible. RHD affects 50 million people, mostly children, worldwide. It claims 400,000 lives each year.

Dr. Sable and Ugandan partners completed important research showing that early RHD detection, coupled with monthly penicillin treatment, can protect the heart. They are working on practical solutions, such as a new portable device with artificial intelligence (AI) that can easily screen for RHD.

In 2023, Dr. Sable led two missions in Uganda where he and his team did surgeries and special tests for 18 children with RHD. They also taught local doctors new skills to help more kids on their own.

Plastic surgery and reconstructive care in Kenya and Nepal

Each year our Craniofacial & Pediatric Plastic Surgery team, under the leadership of Johnston Family Professor of Pediatric Plastic Surgery and Chief of Pediatric Plastic Surgery Gary Rogers, M.D., J.D., LL.M., M.B.A., M.P.H., provides opportunities for fellows to participate in surgical missions.

In 2024, Perry Bradford, M.D., traveled to the Moi Teaching Hospital in Eldoret, Kenya where she provided patients with burn, pressure wound and cleft reconstruction. She built community connections with the local plastic surgeons and educated registrars and medical students. “This gave me firsthand experience working in a community with limited resources and forced me to be more creative,” Dr. Bradford says. “The experience inspired me to examine what it means to have consistent access to advanced tools and equipment.”

In 2022, a group traveled to Nepal to provide care. Some patients arrived after days of travel by yak or buffalo. One child with a burn injury recovered use of her hand. The team educated local providers to deliver life-changing treatments unavailable in Nepal.

Dr. Tesfaye Zelleke in Ethiopia

Dr. Tesfaye Zelleke, left, and team in Ethopia.

Elevating epilepsy care in Ethiopia

Neurologist Tesfaye Zelleke, M.D., and partners in Ethiopia are seeking to improve the lives of children with epilepsy. The BAND Foundation provides support. Ethiopia has a population of about 120 million yet only a handful of pediatric neurologists.

Dr. Zelleke’s team trained nonspecialist providers to diagnose and treat children in the primary care setting. They also launched a mobile epilepsy clinic to provide community care and build the capacity of local clinicians. In collaboration with advocacy groups, the team educates the public about epilepsy with a goal of reducing stigma.

New hope in Norway

In 2023, our Division of Colorectal & Pelvic Reconstruction shared its expertise with clinicians at Oslo University Hospital, Rikshospitalet, in Norway. This effort was a key first step in Oslo becoming the first dedicated colorectal center in Scandinavia.

Marc Levitt, M.D., and team members performed complex surgeries otherwise unavailable for waiting patients. They led an academic conference. They held clinics to educate nurses, reviewed patient records and made care recommendations. Specialized care enabled a young patient with significant bowel difficulties to recover function and lead a normal life.

The team will travel to South Africa, the Czech Republic and Spain in 2024. Donors, including The Dune Road Foundation and Deanna and Howard Bayless, make this work possible.

Improving outcomes for babies in the Congo

AI can be a valuable tool for diagnosing genetic conditions. It detects unique facial patterns that clinicians without genetics training can miss. However, existing facial analysis software struggles in nonwhite populations.

A team led by Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor of Research and Innovation and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation, is working to improve the newborn diagnosis rate worldwide. They are testing smartphone software in the Democratic Republic of Congo. Diverse newborn data improves AI’s ability to detect a variety of genetic conditions in more children. Early detection, diagnosis and informed care lead to better health outcomes.

Nephrology care for kids in Jamaica

Dr. Moxey-Mims and team in Jamaica

Jennifer Carver and Dr. Marva Moxey-Mims, center, with staff at Bustamante Children’s Hospital.

Marva Moxey-Mims, M.D., chief of Nephrology, is bringing care to children with kidney disease in Jamaica, with a goal of improving health equity. An International Pediatric Nephrology Association grant helped make it possible.

On a recent trip, Dr. Moxey-Mims and a small team — including Jennifer Carver, RN, CNN, lead peritoneal dialysis nurse at Children’s National, and three pediatric nephrologists from Jamaica — trained nearly 30 nurses from Jamaican hospitals. Nurses received hands-on dialysis education to improve their clinical skills. The team also worked to educate the community in disease awareness and prevention.

Read more stories like this one in the latest issue of Believe magazine.

Maureen Lyon, Ph.D.

Pilot program improves well-being of families during advanced care planning


Children with life-limiting rare diseases and their caregivers face tremendous stress and anxiety about the heart-breaking decisions before them. A new intervention – designed at Children’s National Hospital to support the palliative needs of these families – improved their spiritual and emotional well-being, according to new research published in the journal, Pediatrics.

Called FACE Rare (FAmily CEntered Pediatric Advance Care Planning Intervention for Rare Diseases), the counseling tools were found to be safe, effective and increased feelings of peace among families in this underserved population.

“Seventy-four percent of the families in that intervention group reported feelings of sadness, yet 100% reported our pilot intervention was a worthwhile experience,” said Maureen Lyon, Ph.D., a clinical psychologist and principal investigator at the Center for Translational Research at Children’s National. “If you’re talking about the possibility that the worst thing in the world would happen to you – that your child might die, and what you would want for them – the families found that our intervention helped. They had a place to process their feelings and consider what would be important to their child.”

The big picture

A rare disease is defined as any condition affecting fewer than 200,000 people in the United States. In pediatrics, these diseases often require constant caregiving and require families to face the cruel reality that the diagnosis may be life-limiting. In such cases, clinical teams often decide that conversations about advanced care planning (ACP) are needed.

The pilot-phase, randomized trial enrolled patients from Children’s National between 2021 and 2023. Research nurses underwent two days of training to be certified in the new intervention. Families assigned to the new approach received three, weekly 60-minute sessions and were evaluated using evidence-based assessment tools, including the Carer Support Needs Assessment Tool/Action Plan and the Next Steps: Respecting Choices Pediatric ACP Conversation. Measures of anxiety and spiritualization were tracked, and families returned after three months for follow-up.

Patients had a range of diagnoses that put tremendous strain on the caregivers, including complex digestive disorders, white-matter diseases and rare forms of epilepsy. Yet the caregivers expressed similar challenges: having time for themselves during the day, knowing what to expect in the future for their child’s care, and managing financial, legal and work issues.

What we found

Reinforcing the need to improve engagement and support for these families, the investigative team found that those who received the FACE Rare intervention reported higher levels of spiritual meaning and peace than those who received “treatment as usual.” All families living below the poverty line reported greater anxiety, and noteworthy to the team, black caregivers were less likely to report caregiver distress than non-black caregivers.

Dr. Lyon said future research needs to understand better how families respond to the challenges of rare diseases and unique social determinants of health that can change the approach to care.

“In some cultures, and families, having strong feelings is discouraged,” Dr. Lyon said. “We want to give families and caregivers space to have these emotions and think about what would be important for their child, if the worst were to happen. They appreciated that they participated in the program and had the space to consider these difficult questions.”

Read the full study, “Advance Care Planning for Children with Rare Diseases: A Pilot RCT,” in Pediatrics.

Cloacal Malformations: Case Studies cover

First textbook for cloacal malformations offers deep dive into care and treatment

Cloacal Malformations: Case Studies cover“Within the field of pediatric colorectal and pelvic reconstruction, the most complex anatomic problem a pediatric surgeon can face is that of a cloacal malformation,” writes Marc A. Levitt, M.D., chief of Colorectal and Pelvic Reconstruction at Children’s National Hospital. A new, first-of-its kind textbook, Cloacal Malformations: Case Studies, seeks to shed light on the multi-disciplinary care required to care for people with this rare condition — when the urinary, gynecologic and colorectal system all exit the body via a common channel which requires reconstruction. The book will be available for pre-order on April 9, 2024.

What is it

Dr. Levitt’s fifth textbook, Cloacal Malformations: Case Studies, is the first comprehensive account of all the care elements needed to successfully treat and manage care for someone with a cloacal malformation. It includes preoperative evaluation and surgical planning, the anorectal malformation (ARM) index, surgical reconstruction, urological evaluation and long-term care. Also covered are the gynecologic evaluation and needed interventions, neurological implications, bowel management and the long-term management of patients with cloacal malformations.

The book’s associate editors hail from some of the subspecialties that are critical to caring for these conditions, including:

  • Tamador Al-Shamaileh, M.D., pediatric colorectal and general
  • Andrea T. Badillo, M.D., pediatric colorectal and pelvic reconstruction
  • Allison C Mayhew, M.D., pediatric gynecology
  • Teresa L. Russell, MS, colorectal and pelvic reconstruction research
  • Briony K Varda, M.D., pediatric urology
  • Richard J Wood, M.D., pediatric colorectal and pelvic reconstruction

Why it matters

This textbook is a compelling resource for all clinicians caring for patients with cloacal malformations. It includes case studies that provide insights into a variety of different clinical scenarios, the first publication to categorize the different types of cloaca, as well as full brand new colored illustrations to enable the reader to understand and gain experience from detailed descriptions.

It is the first of its kind to address the full spectrum needs of someone with a cloacal malformation and will help improve and standardize care for this complex patient population no matter where that patient lives in the world.

Children’s National leads the way

Dr. Levitt is the most experienced pediatric colorectal surgeon in the world. Having performed more than 15,000 procedures during his career, he has cared for more children with cloacal malformations (1,000 cases) and other complex congenital colorectal conditions than anyone else. The information he shares in this textbook, and his previous books, is intended to enhance the care of all children with colorectal and pelvic reconstructive needs, “whether they come into our clinic or are seen by a colleague anywhere in the world,” he notes.

The range of expertise involved in the creation of this textbook reflects Dr. Levitt’s unique approach to pediatric colorectal and pelvic reconstruction, which includes a strongly integrated team of pediatric colorectal surgeons, urologists, gynecologists, gastroenterologists and nurses.

Pre-order a copy of the textbook.

illustration of the gastrointestinal tract

Children’s National to host Pediatric Inflammatory Bowel Disease Research Days Meeting

illustration of the gastrointestinal tractThe division of Gastroenterology, Hepatology and Nutrition at Children’s National Hospital is proud to host the Pediatric Inflammatory Bowel Disease Research Days (PIBDRD) Meeting from April 4-5, 2024.

This two-day event will review recent advances in pediatric Inflammatory Bowel Disease (IBD) research, identify gaps in knowledge in pediatric IBD and identify target areas for future pediatric IBD research. Underlying themes will include sex differences in presentation, disease course and response to treatment; growth; nutrition; and other pediatric chronic inflammatory conditions.

This two-day event offers 13 CME credits and features a robust lineup of experts in the field of Inflammatory Bowel Disease and other chronic inflammatory conditions.

To review the this year’s agenda and register to attend, visit ChildrensNational.org/PIBDRD.

Ian Leibowitz

In the News: Advancing innovations in pediatric gastroenterology and hepatology

“The future is in AI and machine learning and how it allows large data sets to be utilized to a level of understanding that we currently don’t have…We have very rare monogenetic disorders where single gene is the cause of certain inflammatory valve diseases in young children and we’re starting to learn about what’s the right therapy by that gene and personalizing medicine… Not just precision medicine (which is better for a population) but really personalizing medicine.”

Learn more about what Ian Leibowitz, M.D., division chief of Gastroenterology, Hepatology and Nutrition Services, says as he discusses advances in clinical care algorithms that facilitate the timely diagnosis of critical conditions, efforts to increase access to medical and surgical treatment, and broaden awareness among primary care physicians to help ensure care is available and provided as early as possible to all patients.

collage of news outlet logos

Children’s National in the News: 2023

collage of news outlet logos
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)

ARPA-H logo

Children’s National selected as member of ARPA-H Investor Catalyst Hub spoke network

ARPA-H logoThe hospital will advocate for the unique needs of children as part of nationwide network working to accelerate transformative health solutions.

Children’s National Hospital was selected as a spoke for the Investor Catalyst Hub, a regional hub of ARPANET-H, a nationwide health innovation network launched by the Advanced Research Projects Agency for Health (ARPA-H).

The Investor Catalyst Hub seeks to accelerate the commercialization of groundbreaking and accessible biomedical solutions. It uses an innovative hub-and-spoke model designed to reach a wide range of nonprofit organizations and Minority-Serving Institutions, with the aim of delivering scalable healthcare outcomes for all Americans.

“The needs of children often differ significantly from those of adults. This partnership reflects our commitment to advancing pediatric healthcare through innovation and making sure we’re addressing those needs effectively,” said Kolaleh Eskandanian, Ph.D., M.B.A., vice president and chief innovation officer at Children’s National. “Leveraging the strength of this hub-and-spoke model, we anticipate delivering transformative solutions to enhance the health and well-being of the patients and families we serve.”

Children’s National joins a dynamic nationwide network of organizations aligned to ARPA-H’s overarching mission to improve health outcomes through the following research focus areas: health science futures, proactive health, scalable solutions and resilient systems. Investor Catalyst Hub spokes represent a broad spectrum of expertise, geographic diversity and community perspectives.

“Our spoke network embodies a rich and representative range of perspectives and expertise,” said Mark Marino, vice president of Growth Strategy and Development for VentureWell and project director for the Investor Catalyst Hub. “Our spokes comprise a richly diverse network that will be instrumental in ensuring that equitable health solutions reach communities across every state and tribal nation.”

As an Investor Catalyst Hub spoke, Children’s National gains access to potential funding and flexible contracting for faster award execution compared to traditional government contracts. Spoke membership also offers opportunities to provide input on ARPA-H challenge areas and priorities, along with access to valuable networking opportunities and a robust resource library.

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.”

AAP conference logo

Children’s National Hospital at the 2023 American Academy of Pediatrics meeting

There will be over 20 Children’s National Hospital-affiliated participants at this year’s American Academy of Pediatrics National Conference and Exhibition. The meeting will take place in Washington, D.C., from October 20 – October 24. We have compiled their sessions into a mini schedule below.

 

Date Time Presenter Title Division
10/20/2023 8:30 AM Vanessa Madrigal, M.D., M.S.C.E. Section on Cardiology & Cardiac Surgery Program: Day 1 Critical Care
10/20/2023 2:30 PM Kibileri Williams, M.B.B.S Appy Hour: a Current Update on Pediatric Appendicitis Surgery
10/20/2023 3:30 PM Roopa Kanakatti Shankar, M.D., M.S. Precocious Puberty: Puberty Suppression or Not? Endocrinology
10/21/2023 7:30 AM Allison Markowsky, M.D. What is Trending in the Newborn Nursery: Controversies and Evidence Hospital Medicine
10/21/2023 8:00 AM Jessica Herstek, M.D. Joint Program: Council on Clinical Information Technology and Council on Quality Improvement and Patient Safety Medical Informatics
10/21/2023 8:00 AM Nazrat Mirza, M.D., Sc.D. Section on Obesity Program IDEAL Clinic (Obesity Program)
10/21/2023 8:00 AM Hans Pohl, M.D. Section on Urology Program: Day 2 Urology
10/21/2023 9:00 AM Anil Darbari, M.D., M.B.B.S., M.B.A. Constipation: Getting it to Work Out in the End Gastroenterology, Hepatology and Nutrition
10/21/2023 9:00 AM Kibileri Williams, M.B.B.S Appy Hour: a Current Update on Pediatric Appendicitis Surgery
10/21/2023 1:30 PM Olanrewaju (Lanre) Falusi, M.D. Educational Program and Annual Assembly for Medical Students, Residents, and Fellowship Trainees Pediatrician
10/21/2023 2:00 PM Brian Reilly, M.D. Noise 201 – More than Headphones! Otolaryngology
10/21/2023 2:00 PM Erin Teeple, M.D. Hernias, Hydroceles, and Undescended Testicles: When to Wait and When to Operate Surgeon
10/21/2023 3:30 PM Amanda Stewart, M.D. Section on Emergency Medicine Program: Day 2 Emergency Medicine
10/21/2023 3:30 PM Shideh Majidi, M.D., M.S.C.S. Healthcare Disparities in Management of Type 1 Diabetes and Diabetes Technology Endocrinology
10/21/2023 3:30 PM Natasha Shur, M.D. Genetic Testing Boot Camp Geneticist (RDI)
10/21/2023 5:00 PM Danielle Dooley, M.D., M.Phil Connecting School Systems and Health Systems: Successes and Opportunities Pediatrician
10/22/2023 8:00 AM Jaytoya Manget, DNP, FNP Pediatricians and School Attendance: Innovative Approaches to Prevent Chronic Absenteeism
10/22/2023 8:00 AM Simone Lawson, M.D. Section on Emergency Medicine Program: Day 3 Emergency Medicine
10/22/2023 8:00 AM Hans Pohl, M.D. Section on Urology Program: Day 3 Urology
10/22/2023 1:00 PM Lenore Jarvis, M.D., M.Ed. Section on Early Career Physicians Program
10/22/2023 5:00 PM Brian Reilly, M.D. Pediatric Hearing Loss: What’s New in Diagnostics, Prevention and Treatments Otolaryngology
10/23/2023 8:00 AM Rosemary Thomas-Mohtat, M.D. Point-of-Care Ultrasound Fundamentals Course Emergency Medicine
10/23/2023 9:00 AM Matthew Oetgen, M.D., M.B.A. Section on Radiology Program: Imaging Diagnosis and Management of Osteoarticular Infections Orthopaedic Surgery and Sports Medicine
10/23/2023 9:00 AM Christina Feng, M.D. Masses for the Masses: Abdominal Masses in Children Surgeon
10/23/2023 9:00 AM Narendra Shet, M.D. Section on Radiology Program: Imaging Diagnosis and Management of Osteoarticular Infections Radiology
10/23/2023 9:00 AM Shireen Atabaki, M.D., M.P.H. Section on Advances in Therapeutics and Technology Program Telemedicine
10/23/2023 1:00 PM Brian Reilly, M.D. Pediatric Otolaryngology: Back to Basics Otolaryngology
10/23/2023 1:00 PM Sonali Basu, M.D. Point-of-Care Ultrasound Critical Competency Course CCM
10/23/2023 1:00 PM Vanessa Madrigal, M.D. Joint Program: Section on Bioethics, Section on LGBT Health and Wellness and Section on Minority Health, Equity, and Inclusion Critical Care
10/23/2023 2:00 PM Rebecca Persky, M.D. Menstrual Disorders: Primary or Secondary Amenorrhea Endocrinology
10/23/2023 5:00 PM Christina Feng, M.D. Masses for the Masses: Abdominal Masses in Children Surgeon
10/24/2023 9:00 AM Vanessa Madrigal, M.D. Section Showcase: Applying Ethics Principles and Tools To Advocate for Vulnerable Populations Critical Care

 

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.

Erin Teeple

Why a colorectal transition program matters

Erin Teeple

Erin Teeple, M.D.

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.

What is the Colorectal Transition Program?

The Colorectal Transition Program helps young adults with congenital or acquired colorectal conditions transition their care from pediatric care providers to adult care providers. It is critical that they receive guided transitional care because they often have complex medical and surgical histories from the time they are born affecting more than one organ system. A transitional care team which knows the patient on the pediatric side and can help deliver them to the right doctors and care providers in an adult setting will smooth the transition. Collaborative care across specialties including colorectal surgery, gynecology, urology and gastroenterology helps ensure continued care of all organ systems affected. Similar transition programs already exist for other conditions such as congenital heart disease and cystic fibrosis.

What’s new about this program?

I am both a pediatric surgeon and an adult colorectal surgeon, which means I can care for these children even prenatally and continue that care throughout adulthood. There is no other program in the United States led by a practicing pediatric surgeon who is also board-certified in adult colorectal surgery.

In addition, the collaboration between Children’s National Hospital and Medstar means we can bring unprecedented partnerships with urology, gynecology and gastroenterology on the adult side to offer collaborative care akin to the kind of care we offer our pediatric patients in the Division of Colorectal and Pelvic Reconstruction.

Who will benefit from this program?

Teenagers and adults with congenital and acquired colorectal disease, such as cloaca and other anorectal malformations, Hirschsprung disease, inflammatory bowel disease (IBD), pelvic floor dysfunction, familial adenomatous polyposis (FAP), those who have cecostomy or have had in their past a complex reconstruction will benefit the most from this program.

We have started to transition our existing young adults into this program. We also have seen many adults who have struggled to find care since leaving a pediatric care setting decades ago. These people have come from the local area as well as nationally and internationally to find experienced and collaborative care they have desperately sought.

How is Children’s National Hospital leading the way?

By recruiting the only U.S. surgeon boarded in both pediatric surgery and colorectal surgery, Children’s National will offer unique expertise to both initial surgical reconstruction and care and add a wealth of experience to the care of these children as they age into adulthood.

Having a devoted clinician with a foot in both the pediatric and adult worlds will foster long term relationships and build the depth of the team providing clinical care to all our patients and their families.

Why do you think it is so important to involve a surgeon with your training in the care of these kids?

A pediatric colorectal surgeon is critical to the initial care of an infant with congenital colorectal disease. My knowledge as an adult colorectal surgeon adds a different perspective to the treatment plan for our young patients but also will bring key insight to the process of transitioning that care to providers who specialize in adults later down the road. I also bring the knowledge and experience of a pediatric surgeon to the adult side, which gives me the ability to know the complex congenital anatomy that needed to be reconstructed when the patient was a child and to bring together a team optimized for the care of often complex conditions. Adults with these conditions also have unique needs that are different from the typical adult colorectal patient, and my specific expertise gives me the right skills to help them.