Surgical Innovation Archives

Evidence and expertise drive cardiac surgery innovation at Children’s National Hospital

December 21, 2023

“Our goal is to do the difficult and the impossible,” says Yves d’Udekem, M.D., Ph.D.

“Our goal is to do the difficult and the impossible,” says Yves d’Udekem, M.D., Ph.D., chief of Cardiac Surgery at Children’s National Hospital.

Dr. d’Udekem and the cardiac surgeons at Children’s National apply technical skill and expertise to offer renewed hope for the highest risk children with critical congenital heart disease, including those with single ventricle anomalies like hypoplastic left heart syndrome.

“When families have nowhere else to turn, they can turn to us,” he adds.

Why it matters

The cardiac surgery team has welcomed families from across the United States and around the world who seek experts in the care of these critical heart conditions. Their experience is building an important evidence base for better surgical approaches that will improve long-term outcomes for children with many different types of congenital heart disease, but especially for single ventricle conditions.

Innovation in cardiac surgery

Hybrid surgical strategy: Cardiac Surgeon Can Yerebakan, M.D., and Interventional Cardiology Director Joshua Kanter, M.D., are national leaders in the use of a hybrid surgical strategy for high-risk infants with single ventricle heart conditions. They can perform this procedure on babies as small as 1.1 kilograms. It allows critical time for the lungs and other organs to recover and get stronger after birth before the child undergoes more invasive procedures.
New uses for artificial hearts: d’Udekem showed proof-of-concept for the use of an artificial heart to give a child with a single ventricle the time for their own heart to recover rather than being transplanted. In this case, the child was supported by a left-ventricle assist device (LVAD) long term. As their own heart recovered, surgeons then performed successful procedures that seemed impossible to perform before.
Novel complex pulmonary artery reconstruction: Children’s National performs the most complex lobar and sub-lobar pulmonary artery reconstruction for children with complex pulmonary stenosis. Cardiac Surgeon Manan Desai, M.D., says the approach leverages interventional cardiac imaging and precision surgical techniques to correct stenosis in smaller lung arteries. This helps establish better right-sided pressure in the heart and likely reduces the chance of heart failure down the road.
Pediatric-focused advanced lung care and transplant: Children’s National is poised to become one of only a few locations in the United States to offer comprehensive care for children with complex lung conditions. In 2024, Cardiac Surgeon Aybala Tongut, M.D., will begin performing pediatric lung transplants as part of the hospital’s Advanced Lung Disease Program focused on the unique needs of children.

Children’s National leads the way

“It’s time to combine firsthand expertise and long-term outcomes from decades of congenital heart surgical procedures to refine our surgical techniques,” says Dr. d’Udekem. “We need to ensure patients with congenital heart disease, especially those with single ventricle heart defects, can thrive long term.”

Soon, the Children’s National team plans to re-examine the effectiveness of different techniques for the Fontan procedure. They’ll compare an extracardiac approach against the older lateral tunnel procedure to determine how best to reduce long-term pressure on the heart by creating larger conduits and improving blood flow.

More education is needed to ensure valve repairs for children with congenital heart disease, including single ventricle conditions, which have a high rate of failure and require reoperation, are as successful as can be. The goal is to avoid the need for reoperation or replacement procedures. This is why Children’s National recently hosted the inaugural Valve Repair Symposium. It featured practical cases illustrated with intraoperative video, echocardiography and MR images to bring critical knowledge about pediatric heart valve repair to more people in the field.

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

November 13, 2023

The 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.

Children’s National awarded nearly $7.5 million by FDA to lead pediatric device innovation consortium

November 2, 2023

Children’s National Hospital was awarded nearly $7.5 million in a five-year grant to continue its leadership of an FDA-funded pediatric device consortium. Building upon a decade of previous consortium leadership, the new consortium is Alliance for Pediatric Device Innovation (APDI) and features a new and expanded roster of partners that reflects its added focus on providing pediatric innovators with expert support on evidence generation, including the use of real-world evidence (RWE), for pediatric device development.

Collaborating for success

With the goal of helping more pediatric medical devices complete the journey to commercialization, APDI is led by Children’s National, with Kolaleh Eskandanian, Ph.D., M.B.A., vice president and chief innovation officer, serving as program director and principal investigator, and Julia Finkel, M.D., pediatric anesthesiologist and director of Pain Medicine Research and Development in the Sheikh Zayed Institute for Pediatric Surgical Innovation, serving as principal investigator.

Consortium members include Johns Hopkins University, CIMIT at Mass General Brigham, Tufts Medical Center, Medstar Health Research Institute and MedTech Color. Publicly traded OrthoPediatrics Corp., which exclusively focuses on advancing pediatric orthopedics, is serving as APDI’s strategic advisor and role model for device innovators whose primary focus is children.

Why we’re excited

Consortium initiatives got underway quickly with the announcement of a special MedTech Color edition of the “Make Your Medical Device Pitch for Kids!”^™competition that focuses on African American and Hispanic innovators. Interested innovators can find details and apply at MedTech Color Pitch Competition. The competition was announced at the recent MedTech Color networking breakfast on Oct. 10,2023 at The MedTech Conference powered by AdvaMed.

“We all benefit from greater equity and inclusion among pediatric MedTech founders, decision-makers, investigators and developers in more effectively addressing the needs of the entire pediatric population,” said Eskandanian. “We need the expertise and insights of innovators from diverse backgrounds, and we want to provide these talented individuals with more opportunities to present their work and share their perspectives on pediatric device development.”

Additional details

APDI is one of five FDA-funded consortia created to provide a platform of services, expertise and funding to help pediatric innovators bring medical devices to the market that specifically address the needs of children.

Novel camera + machine learning = hope for more precise neurosurgery

October 27, 2023

Researchers at Children’s National Hospital developed a compact imaging camera capable of seeing beyond the human visual spectrum to help segment healthy brain tissue from tumors during surgery. The groundbreaking technology will allow neurosurgeons to make more precise, real-time decisions in the operating room, rather than sending samples to pathology labs for biopsies.

In a manuscript published in Bioengineering, the team of engineers and neurosurgeons details how its snapshot hyperspectral imaging (sHSI) camera can be used to capture and process images of brain tissue, using the wide spectrum of light between visible and infrared wavelengths. That additional information — beyond the human eye — has the potential to allow for more accurate and complete tumor removal.

“In the hands of a neurosurgeon, this camera, when combined with machine learning, could dramatically improve outcomes for some of our most vulnerable brain tumor patients,” said Richard Jaepyeong Cha, Ph.D., an optical engineer and principal investigator at the Sheikh Zayed Institute of Pediatric Surgical Innovation. “We are able to attach the camera to a surgical microscope and process a significant amount of information from the patient while in the operating room. Not only could this lead to more complete tumor resection, it will also allow the surgeon to save as much healthy brain tissue as possible and reduce lifelong neurological complications.”

Why we’re excited

Brain tumors are the most common solid tumors in children, accounting for the highest number of pediatric cancer deaths globally each year. To develop a treatment plan, neurosurgeons need to understand the tumor’s features, including its type, grade of malignancy, location and its categorization as a primary or metastatic cancer. This information leads to decisions about how to remove or biopsy a tumor.

Under the current protocols, surgeons evaluate tumor margins in the operating room by examining the appearance of the brain tissue and sending out small samples to the pathology department for biopsies. This can lead to longer surgeries and difficult real-time surgical decisions. For instance, some low-grade tumors are visually indistinguishable from healthy brain tissue.

In four investigational cases approved by the hospital’s institutional research board, the sHSI camera was used in the operating room to help segment healthy pediatric brain tissue from tumors. Unlike the conventional red-green-blue (RGB) imaging cameras, which use only those three colors, HSI captures spectral data at each pixel of the image — a task too complex for the human eye — and sends it instantly for processing by an algorithm designed to assist in tumor segmentation.

What’s ahead

Despite the small dataset, the researchers were able to successfully segment healthy brain tissue from lesions with a high specificity during pediatric brain tumor resection procedures. Significant work remains to refine the technology and the machine learning behind it. Researchers also plan to integrate the sHSI camera into a laparoscope to visualize tumors that are not on the brain’s surface and collect data from more angles.

“As we develop these groundbreaking tools, we plan to continue to expand the dataset and refine the algorithm to make pediatric neurosurgery continually more precise,” said Naomi Kifle, M.S., research and development engineer at Children’s National and first author on the paper. “As our dataset grows, we hope to create a model that can distinguish healthy brain tissue, tumor and skull. This groundbreaking surgical tool shows significant promise.”

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

October 3, 2023

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

“Mask up!” Soon, AI may be prompting healthcare workers

September 6, 2023

Researchers at Children’s National Hospital are embarking on an effort to deploy computer vision and artificial intelligence (AI) to ensure medical professionals appropriately use personal protective equipment (PPE). This strikingly common problem touches almost every medical specialty and setting.

With nearly $2.2 million in grants from the National Institutes of Health, the team is combining their expertise with information scientists at Drexel University and engineers at Rutgers University to build a system that will alert doctors, nurses and other medical professionals of mistakes in how they are wearing their PPE. The goal is to better protect healthcare workers (HCWs) from dangerous viruses and bacteria that they may encounter — an issue laid bare with the COVID-19 pandemic and PPE shortages.

“If any kind of healthcare setting says they don’t have a problem with PPE non-adherence, it’s because they’re not monitoring it,” said Randall Burd, M.D., Ph.D., division chief of Trauma and Burn Surgery at Children’s National and the principal investigator on the project. “We need to solve this problem, so the medical community will be prepared for the next potential disaster that we might face.”

The big picture

The World Health Organization has estimated that between 80,000 and 180,000 HCWs died globally from COVID-19 between January 2020 and May 2021 — an irreplaceable loss of life that created significant gaps in the pandemic response. Research has shown that HCWs had an 11-fold greater infection risk than the workers in other professions, and those who were not wearing appropriate PPE had a 1/3 higher infection risk, compared to peers who followed best practices.

Burd said the Centers for Disease Control and Prevention has recommended that hospitals task observers to stand in the corner with a clipboard to watch clinicians work and confirm that they are being mindful of their PPE. However, “that’s just not scalable,” he said. “You can’t always have someone watching, especially when you may have 50 people in and out of an operating room on a challenging case. On top of that, the observers are generally trained clinicians who could be filling other roles.”

What’s ahead

Bringing together the engineering talents at Drexel and Rutgers with the clinical and machine-learning expertise at Children’s National, the researchers plan to build a computer-vision system that will watch whether HCWs are properly wearing PPE such as gloves, masks, eyewear, gowns and shoe covers.

The team is contemplating how the system will alert HCWs to any errors and is considering haptic watch alerts and other types of immediate feedback. The emerging power of AI brings tremendous advantages over the current, human-driven systems, said Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor in Research and Innovation at Children’s National and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation.

“Human observers only have one pair of eyes and may fatigue or get distracted,” Linguraru said. “Yet artificial intelligence, and computers in general, work without getting tired. We are excited to figure out how a computer can do this work – without ever blinking.”

Children’s National Hospital leads the way

Linguraru says that Children’s National and its partners make up the ideal team to tackle this vexing challenge because of their ability to assemble a multidisciplinary team of scientists and engineers who can work together with clinicians. “This is a dialogue,” he said. “A computer scientist, like myself, needs to understand the intricacies of complicated clinical realities, while a clinician needs to understand how AI can impact the practice of medicine. The team we are bringing together is intentional and poised to fix this problem.”

Cell therapy mitigates neurological impacts of cardiac surgery in pre-clinical model

September 6, 2023

Differences of cortical fractional anisotropy between cardiopulmonary bypass and control (left), cardiopulmonary bypass + mesenchymal stromal cells and cardiopulmonary bypass (center), and 3 groups (right).

A pre-clinical study in the journal JACC: Basic to Translational Science shows that infusing bone marrow-derived mesenchymal stromal cells (BM-MSCs) during cardiac surgery provides both cellular-level neuroprotection for the developing brain and improvements in behavior alterations after (or resulting from) surgery.

What this means

According to lead author Nobuyuki Ishibashi, M.D., Oxidative and inflammatory stresses that are thought to be related to cardiopulmonary bypass cause prolonged microglia activation and cortical dysmaturation in the neonatal and infant brain. These issues are a known contributor to neurodevelopmental impairments in children with congenital heart disease.

This study found that, in a pre-clinical model, the innovative use of cardiopulmonary bypass to deliver these mesenchymal stromal cells minimizes microglial activation and neuronal apoptosis (cell death), with subsequent improvement of cortical dysmaturation and behavioral alteration after neonatal cardiac surgery.

Additionally, the authors note that further transcriptomic analyses provided a possible mechanism for the success: Exosome-derived miRNAs such as miR-21-5p, which may be key drivers of the suppressed apoptosis and STAT3-mediated microglial activation observed following BM-MSC infusion.

Why it matters

Significant neurological delay is emerging as one of the most important current challenges for children with congenital heart disease, yet few treatment options are currently available.

Applications of BM-MSC treatment will provide a new therapeutic paradigm for potential MSC-based therapies as a form of neuroprotection in children with congenital heart disease.

Children’s National Hospital leads the way

The Ishibashi lab is the first research team to demonstrate the safety, efficacy and utility of using cardiopulmonary bypass to deliver BM-MSCs with the goal of improving neurological impairments in children undergoing surgery for congenital heart disease. In addition to this pre-clinical research, a phase 1 clinical trial, MeDCaP, is underway at Children’s National.

Recent additional funding from the NIH will allow the team to identify molecular signatures of BM-MSC treatment and mine specific BM-MSC exosomes for unique cardiopulmonary bypass pathology to further increase understanding of precisely how and why this cell-based treatment shows success.

Attendees at the inaugural symposium on AI in Pediatric Health and Rare Diseases

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

June 26, 2023

The daylong event drew experts from the Food and Drug Administration, Pfizer, Oracle Health, NVIDIA, AWS Health and elsewhere to start building a community aimed at using data for the advancement of pediatric medicine.

The future of pediatric medicine holds the promise of artificial intelligence (AI) that can help diagnose rare diseases, provide roadmaps for safer surgeries, tap into predictive technologies to guide individual treatment plans and shrink the distance between patients in rural areas and specialty care providers.

These and dozens of other innovations were contemplated as scientists came together 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. The daylong event drew experts from the Food and Drug Administration, Pfizer, Oracle Health, NVIDIA, AWS Health and elsewhere to start building a community aimed at using data for the advancement of pediatric medicine.

“AI is the single greatest tool for improving equity and access to health care,” said symposium host Marius George Linguraru, D.Phil., M.A., M.Sc., principal investigator at the Sheikh Zayed Institute for Pediatric Surgical Innovation. “As a population, kids are vastly underrepresented in scientific research and resulting treatments, but pediatric specialties can use AI to provide medical care to kids more efficiently, more quickly and more effectively.”

What they’re saying

Scientists shared their progress in building digital twins to predict surgical outcomes, enhancing visualization to increase the precision of delicate interventions, establishing data command centers to anticipate risks for fragile patients and more. Over two dozen speakers shared their vision for the future of medicine, augmented by the power of AI:

Keynote speaker Subha Madhavan, Ph.D., vice president and head of AI and machine learning at Pfizer, discussed various use cases and the potential to bring drugs to market faster using real-world evidence and AI. She saw promise for pediatrics. “This is probably the most engaging mission: children’s health and rare diseases,” she said. “It’s hard to find another mission that’s as compelling.”
Brandon J. Nelson, Ph.D., staff fellow in the Division of Imaging, Diagnostics and Software Reliability at the Food and Drug Administration, shared ways AI will improve diagnostic imaging and reduce radiation exposure to patients, using more advanced image reconstruction and denoising techniques. “That is really our key take-home message,” he said. “We can get what … appear as higher dose images, but with less dose.”
Daniel Donoho, M.D., a neurosurgeon at Children’s National, introduced the audience to the potential of “Smart ORs”: operating rooms where systems can ingest surgery video and provide feedback and skill assessments. “We have to transform the art of surgery into a measurable and improvable scientific practice,” he said.
Debra Regier, M.D., chief of Genetics and Metabolism at Children’s National, discussed how AI could be used to diagnose and treat rare diseases by conducting deep dives into genetics and studying dysmorphisms in patients’ faces. Already, Children’s National has designed an app – mGene – that measures facial features and provides a risk score to help anyone in general practice determine if a child has a genetic condition. “The untrained eye can stay the untrained eye, and the family can continue to have faith in their provider,” she said.

What’s next

Linguraru and others stressed the need to design AI for kids, rather than borrow it from adults, to ensure medicine meets their unique needs. He noted that scientists will need to solve challenges, such as the lack of data inherent in rare pediatric disorders and the simple fact that children grow. “Children are not mini-adults,” Linguraru said. “There are big changes in a child’s life.”

The landscape will require thoughtfulness. Naren Ramakrishnan, Ph.D., director of the Sanghani Center for Artificial Intelligence & Analytics at Virginia Tech and symposium co-host, said that scientists are heading into an era with a new incarnation of public-private partnerships, but many questions remain about how data will be shared and organizations will connect. “It is not going to be business as usual, but what is this new business?” he asked.

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

June 21, 2023

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

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

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

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

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

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

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

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

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

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

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

Why a colorectal transition program matters

June 12, 2023

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.

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

March 15, 2023

Chest/abdominal x-ray of neonate receiving a modified pediatric-sized implantable pulse generator, demonstrating epicardial suture-on bipolar lead and pulse generator in the upper abdominal pocket.

In 2022, five tiny, fragile 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.

An article in the journal Heart Rhythm assesses the outcomes to date for the infants who received pacemakers that were modified to work better in the smallest children who need them. The authors, including first author Charles Berul, M.D., chief of Cardiology at Children’s National Hospital, share that after following for between 6 and 9 months, “early post-operative performance of this device has been excellent.”

The big picture

Even the tiniest pacemakers and defibrillators on the market today aren’t small enough for infants and young children with heart rhythm abnormalities. So, for several years, Dr. Berul and colleagues at several other institutions have collaborated to adapt existing pacemakers, including the Medtronic Micra leadless pacing system, for use in tiny, critically ill newborns.

The specially modified pediatric-sized implantable pulse generator, called the Pediatric IPG, includes a Medtronic Micra sub-assembly that connects to an epicardial lead. While this makes the leadless pacemaker into one that uses leads, the resulting IPG is significantly smaller than any commercially available pacemaker previously on the market in the U.S.

The five infants in this case profile each received the modified Pediatric IPG at four separate institutions, and each surgery to implant the device was performed by a different cardiac surgeon. Two of the five cases were cared for at Children’s National. Cardiac surgeons Can Yerebakan, M.D., Ph.D., and Manan Desai, M.D. each performed one procedure.

The Pediatric IPG was authorized for use by emergency use exemptions from the federal Food and Drug Administration and with review and approval by each hospital’s Institutional Review Board, based on successful laboratory and pre-clinical models with favorable, though limited, results.

The patient benefit

All five infants were diagnosed with congenital complete heart block and required urgent pacing immediately after birth. The authors write:

“Permanent pacing in adults and older children is a routine, relatively simple implantation procedure. In the smallest of children, however, the generator is typically placed in the abdomen and can still present challenges in tiny babies under 2.5kg due to its bulk and dimensions, with risks of wound dehiscence, generator erosion and other complications.”

The authors note that the smaller profile of the Pediatric IPG reduces and has the potential to eliminate some of these challenges.

What’s next: Better delivery

Innovating smaller devices, including adapting current technology like the Medtronic Micra for pediatric use, is a good start but won’t be enough to eliminate some of the challenges for these patients. When a newborn or young child needs any pacemaker or defibrillator, they face open chest surgery. Their arteries and veins are just too small for even the smallest size transvenous pacemaker catheter.

That’s why Dr. Berul and engineers in the Sheikh Zayed Institute for Pediatric Surgical Innovation are working on a first-of-its-kind minimally invasive pericardial access tool. The team hypothesizes that this tool will allow for pacing and defibrillation therapy to be delivered through a single small port inserted through the skin that is about the size of a drinking straw.

You can read the full article Creative Concepts: Tiny Pacemakers for Tiny Babies in the journal Heart Rhythm.

4D flow explained: Advanced imaging measures critical blood flow characteristics of single ventricle hearts

March 13, 2023

Yue-Hin “Tom” Loke, M.D., pediatric cardiologist and director of the 3D Cardiac Visualization Laboratory at Children’s National Hospital, uses magnetic resonance imaging and software rendering to create novel 4D flow images of children with single ventricle congenital heart disease.

“My research measures the degree of vortex formation (and) the degree of energy loss in the atrium as potential measurements of heart health and uses these measurements as a potential gauge of the heart health of children born with single ventricle conditions including hypoplastic left heart syndrome,” he says. “This information can be used to guide the management of the care for children with congenital heart disease. This technology provides valuable insight into how well the heart is working, especially before the Fontan procedure.”

Learn more about the approach and how it impacts clinical care decisions in the Children’s National Heart Institute.

Surgeon says treatment silos in childhood obesity need to end

March 8, 2023

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

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

Why it matters

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

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

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

Read the editorial in the Lancet Child & Adolescent Health.

High-risk newborns with hypoplastic left heart syndrome benefit from hybrid approaches

March 8, 2023

“Hybrid treatment enables even patients who are extremely high risk for surgery to have a survival advantage.” — Dr. Yerebakan.

Can Yerebakan, M.D., Ph.D., associate chief of Cardiac Surgery, and Joshua Kanter, M.D., director of Interventional Cardiology, created a multi-disciplinary team at Children’s National Hospital to perform the staged surgical approach known as the “hybrid strategy” to support the smallest, most fragile babies born with hypoplastic left heart syndrome (HLHS).

Today, the team performs more of these procedures than almost any other heart center in the United States, and they’ve successful completed it for neonates as small as 1 kg.

The approach gives high-risk babies time to recover from birth trauma and continue developing crucial organs before undergoing more traditional, more-invasive HLHS procedures that require open-heart surgery with cardiopulmonary bypass. Surgeons also have more time to make complete individualized risk assessments for next steps on each case, replacing the historical “one size fits all” operative pathway for HLHS.

Read more about the hybrid surgical strategy for HLHS.

Inducing strokes in newborns to treat hemimegalencephaly

February 3, 2023

“The number one thing people are perplexed by is how well these babies recover and how they can only live with half a brain,” said Tayyba Anwar, M.D., neonatal neurologist and co-director of the Hemimegalencephaly Program at Children’s National Hospital. “People think if a child has half a brain that’s damaged or dysplastic, how are they functioning? But babies are so resilient. It still amazes me.”

The big picture

Children’s National experts have pioneered a novel approach of inducing strokes to stop seizures and improve neurodevelopmental outcomes in newborns under three months old with hemimegalencephaly (HME).

The procedure, called an endovascular embolic hemispherectomy, can be safely used to provide definitive treatment of HME-related epilepsy in neonates and young infants, according to a study in the Journal of NeuroInterventional Surgery.

Prior to this approach, the standard treatment was an anatomic hemispherectomy — surgical removal of the affected half of the brain. But infants had to be at least three months old to undergo such a complex surgery. Delaying surgery meant the persistent seizures compromised the development of the healthy half of the brain.

What they’re saying

In this video, Dr. Anwar and Panagiotis Kratimenos, M.D., Ph.D., neonatologist and co-director of Research in Neonatology at Children’s National, discuss the critically important neonatal care provided to babies who undergo endovascular embolic hemispherectomy and how protocols have evolved with each case to make this less invasive approach a feasible early alternative to surgical hemispherectomy.

Drs. Anwar and Kratimenos are part of the multidisciplinary team of neonatal neurologists, neurointerventional radiologists, neonatologists and neurosurgeons performing endovascular hemispherectomies.

Abstract Happy 2022 New Year greeting card with light bulb

The best of 2022 from Innovation District

January 6, 2023

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

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

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

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

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

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

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

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

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

5. New telehealth command center redefines hospital care

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

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

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

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

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

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

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

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

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

10. 2022: Pediatric colorectal and pelvic reconstructive surgery today

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

Advanced MRI hopes to improve outcomes for Fontan cardiac patients

January 5, 2023

Chief of Cardiac Surgery Yves d’Udekem, M.D., calls this “fourth-dimensional imaging” that identifies if blood flows swiftly, smoothly, or is subjected to swirls or turbulences that impede the effectiveness of the flow.

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 (HLHS).

The data allows surgeons to make critical corrections to the atrioventricular valve — the valve between the atrium and ventricle of the heart — before a child undergoes the single ventricle procedure known as the Fontan.

Yves d’Udekem, M.D., chief of Cardiac Surgery at Children’s National, says that eliminating leakage of the atrioventricular valve before a child undergoes the Fontan may improve a child’s quality of life after Fontan and reduce the likelihood of heart failure, transplant or death long term.

The big picture

Patients with only one functioning pumping chamber, or ventricle, have been on the same treatment trajectory for decades. However, critical international efforts to collect and analyze long-term outcomes for patients with Fontan circulations have led surgeons like d’Udekem to rethink what quality of life and a positive outcome means for these patients. This includes patients in the Australia and New Zealand Fontan Registry founded by d’Udekem while at Royal Children’s Hospital in Australia.

Research based on data in the patient registries shows that atrioventricular valve leakage plays a critical role in the outcomes for patients with single ventricle defects. For children with Fontan circulation, significant leakage of this valve leads to worse outcomes.

Moving the field forward

Treatment decisions for children with single ventricle heart defects are often made based on commonly used heart imaging to determine the effect of valve leakage based on two limited, key variables: the size and the squeeze of the heart. However, this is a late effect and may not reflect the true impact on children with single ventricle hearts.

The team at Children’s National — including d’Udekem and Yue-Hin Loke, M.D., cardiac MRI specialist and director of the 3D Cardiac Visualization Laboratory — use cardiac MRI to measure the flow between heart chambers. Special software can measure abnormal flow and energy losses inside the heart, drawing on principles of physics and engineering.

“Dr. Loke not only gathers three-dimensional imaging of the heart through every heartbeat, he also gathers brand new types of colored imaging of blood flow itself, showing how effectively it is propelled by the heart,” says d’Udekem. “This ‘fourth-dimensional imaging’ identifies whether the blood flows swiftly, smoothly or whether it is subjected to swirls or turbulences that impede the effectiveness of the flow.”

Children’s National leads the way

Harnessing the visualization technology and analysis for clinical care of patients with single ventricle defects is relatively new in the United States, but it has become a vital part of the routine, clinical pre-Fontan evaluations at Children’s National.

Few locations in the United States have the mechanisms and expertise to study abnormal flow patterns in children with single ventricle defects. Children’s National collaborates with engineers to help parse the information into clear-cut takeaways for the clinical teams to use in their treatment planning.

Also, while other centers have access to this technology, not many centers have cardiac surgeons like d’Udekem who have an active interest in applying the key learnings from this data as quickly as possible to improve outcomes for patients.

Loke describes the collaboration at Children’s National as a “unique crossroads of clinical need and clinical interest to help these kids in very bold ways.”

What’s next

d’Udekem and Loke are engaged in a comprehensive project that analyzes the impact of atrioventricular valve leakage to ensure that the flow inside the heart is optimized before a Fontan procedure.

The research will map the efficiency of blood flow between the atrium and ventricle before surgery and after a surgical correction is made. The goal is to test the hypothesis that better atrioventricular circulation before Fontan can make a big difference for patients’ long-term quality of life and overall health.

Healthcare leaders join to advance pediatric innovation

December 21, 2022

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

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

Convening a coalition of healthcare leaders

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

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

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

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

Using RWE to facilitate the regulatory pathway

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

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

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

About NCC-PDI

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

Effective treatment for children with hemimegalencephaly

December 1, 2022

Anatomic or functional hemispherectomy are established neurosurgical treatment options and are recommended for effective seizure control and improved neurodevelopmental outcome in patients with HME.

Endovascular hemispherectomy can be safely used to provide definitive treatment of hemimegalencephaly (HME) related epilepsy in neonates and young infants when intraprocedural events are managed effectively, a new study finds.

The authors of the study, which published in the Journal of NeuroInterventional Surgery, add that this less invasive novel approach should be considered a feasible early alternative to surgical hemispherectomy.

Why it matters

Anatomic or functional hemispherectomy are established neurosurgical treatment options and are recommended for effective seizure control and improved neurodevelopmental outcome in patients with HME. Hemispherectomy in the neonate, however, is associated with high surgical risks and most neurosurgeons defer surgical hemispherectomy until the patient is at least 8 weeks old. This delay comes at a significant neurocognitive cost as the uncontrolled seizures during this time of deferred surgery have a deleterious effect on future neurocognitive outcome.

Why we’re excited

“The procedure we have developed, endovascular hemispherectomy by transarterial embolization, acutely stops seizures and this cessation of seizures has been sustained in each of the treated patients,” says Monica Pearl, M.D., director of the Neurointerventional Radiology Program at Children’s National Hospital and the study’s lead author.

This treatment option – performed early in life – provides hope and a better quality of life for these patients post procedure.

What’s been the hold-up in the field?

Currently, the only effective treatment option is hemispherectomy. With the patient population of neonates and young infants, hemispherectomy has a very high mortality and complication rate resulting in most neurosurgeons deferring treatment until at least 8 weeks. This leaves neonates and young infants without effective treatment options and on multiple antiseizure medications in an effort to control the seizures

How does this work move the field forward?

“Embolization provides a highly effective treatment option that acutely stops seizures during a time period of critical neurodevelopment and one in which traditional open neurosurgical procedures are not viable options,” Dr. Pearl says. “Specifically, we can consider and perform embolization in children as young as one or two weeks of age rather than waiting until at least 8 weeks of age. The impact of earlier intervention – acutely stopping the seizures, reducing the dose and number of antiseizure medications and avoiding more invasive surgical procedures (hemispherectomy, shunt placement) – appears to be dramatic in our recent series. We are conducting long term studies to assess this effect on neurodevelopmental outcome.”

How is Children’s National leading in this space?

Dr. Pearl and the late Taeung Chang, M.D., neurologist at Children’s National, pioneered this concept and treatment pathway. The multidisciplinary team is led by Dr. Pearl, who has performed all the embolization procedures (transarterial embolization/endovascular hemispherectomy) and Tayyba Anwar, M.D., Co-Director, Hemimegalencephaly Program at Children’s National Hospital. Our epilepsy team, neonatology team and neurosurgery team work collaboratively managing the patients before and after each procedure.

Sphincter reconstruction offers new hope for children with Hirschsprung disease

November 17, 2022

After surgery, patients reported sleeping better, higher productivity, the ability to participate in sports and be away from home with confidence.

A new surgical procedure successfully addresses a common lingering challenge — incontinence – faced by some families with young children who have undergone surgery for Hirschsprung disease. This first-of-its kind procedure was pioneered by surgeons in the Division of Colorectal and Pelvic Reconstruction at Children’s National Hospital, led by Division Chief Marc Levitt, M.D.

The 12-month outcomes for early recipients of the procedure appear in the Journal of Pediatric Surgery.

Why it matters

“The goal of surgery for Hirschsprung disease is to give a child a chance to have normal bowel function, which plays a significant role in their quality of life,” says Dr. Levitt, who served as senior author on the study. “But sometimes after a child receives their pull-through operation — the first surgical step to treat Hirschsprung — they continue to be incontinent. This novel follow-up procedure is offering these kids and their families new hope for a more normal life.”

Hirschsprung disease is a congenital condition that affects one in 5,000 children each year. Children with this condition develop a host of health problems including intestinal blockage and severe constipation, caused by the inability of the colon, or large intestine, to work properly.

The pull-through procedure is the best treatment for children with Hirschsprung disease. The procedure removes the portion of the intestine that does not function so that the child can regain control.

However, after the procedure, some children continue to experience incontinence that requires routine enemas for their entire lives. Before now there was no solution to this problem.

Why we’re excited

The new pilot study shows long-term outcomes for seven patients between the ages of 2 and 18 who underwent the novel technical approach called sphincter reconstruction. Each of the seven were diagnosed with an anatomic or physical issue contributing to their incontinence. It was during a repair procedure for the first patient that Dr. Levitt and his team had the idea of a technique for sphincter reconstruction. After that, six more patients with similar anatomy were offered the procedure.

Four of the six now have normal bowel function. They reported sleeping better, higher productivity, the ability to participate in sports and be away from home with confidence. The remaining two have behavioral conditions that have delayed their progress but are on their way to similar success.

The patient benefit

“Parents and caregivers have told us repeatedly about how much enemas negatively impact their family quality of life,” says Dr. Levitt. “That’s what makes this exciting — this validated surgical approach offers new hope for families who previously thought their child would face a lifetime of issues that would limit their freedom. Now, we finally have something to offer.”