Cardiology & Heart Surgery

Baby on ventilator

JAMA study shows no benefit to nitric oxide in cardiopulmonary bypass for young children

Baby on ventilator

An international clinical trial showed that nitric oxide doesn’t help kids recover faster from cardiac surgery with cardiopulmonary bypass.

A study published in JAMA finds that the practice of introducing nitric oxide into the gas flow of the cardiopulmonary bypass oxygenator does not improve recovery or reduce the amount of time a child under age 2 needs to be on a ventilator after cardiac surgery.

Children’s National Cardiac Surgery Chief Yves d’Udekem, M.D., Ph.D., co-authored the international study, which is already leading to changes in how hospitals around the world care for children with congenital heart disease (CHD).

The results are from a double-blind, randomized controlled trial with more than 1,200 participants across six centers in Australia, New Zealand and the Netherlands. The research team found that children under age 2 who had cardiac surgery with cardiopulmonary bypass spent about the same number of days on ventilators after surgery, whether nitric oxide was used during surgery or not.

“These findings do not support the use of nitric oxide delivered into the cardiopulmonary bypass oxygenator during heart surgery,” the authors conclude.

What this means

Previous smaller, single center studies had shown early indications that nitric oxide delivered during heart surgery could possibly improve recovery and shorten the need for respiratory support after surgery by reducing the occurrence of low cardiac output syndrome in children under age 2.

This large-scale international trial showed that this is not the case.

Why it matters

Based on these earlier studies, many hospitals in the United States and around the world who perform critical heart surgery on young children with congenital heart disease had already started to incorporate nitric oxide into cardiopulmonary bypass. This new, more robust data is helping hospitals reassess this practice. Many are stopping it altogether based on the findings.

This work is an important reminder of how valuable well-designed, large-scale, double-blind, randomized, controlled trials are to defining, improving and refining best practices in clinical care.

Also, trials of this size and significance in pediatrics generally, and CHD specifically, take a very long time to complete, if they are ever able to be completed at all. That’s because the number of children with these conditions is relatively small and spread out, even though CHD is the most common birth defect in the world. The authors say it is a major accomplishment to have completed a trial of this size and  in such a short time. Even better, the data gathered from this sample of patients from across international borders can be used to provide even more insights into how best to care for these children as they continue to grow and develop.

Ricardo Munoz

Ricardo Munoz, M.D., named MacCutcheon Family Professor in Cardiac Critical Care Medicine

Ricardo MunozChildren’s National Hospital named Ricardo Munoz, M.D., the MacCutcheon Family Professor in Cardiac Critical Care Medicine at Children’s National Hospital.

Dr. Munoz serves as chief of the Division of Cardiac Critical Care Medicine, executive director of Telehealth, and co-director of Children’s National Heart Institute at Children’s National Hospital. He is professor of pediatrics at the George Washington University School of Medicine and the main editor of several books in pediatric cardiac critical care medicine.

About the award

Dr. Munoz joins a distinguished group of 42 Children’s National physicians and scientists who hold an endowed chair. Professorships at Children’s National support groundbreaking work on behalf of children and their families and foster new discoveries and innovations in pediatric medicine. These appointments carry prestige and honor that reflect the recipient’s achievements and donor’s forethought to advance and sustain knowledge.

As chief of the Division of Cardiac Critical Care Medicine, Dr. Munoz leads a multidisciplinary team of specially trained physicians in providing intensive pediatric cardiac critical care. Dr. Munoz is credited as pioneering telemedicine for pediatric critical care, dedicated to increasing access and quality care for children with special hearts in the nation’s capital and across the world.

The MacCutcheon Family Foundation, through their vision and generosity, are ensuring that Dr. Munoz and future holders of this professorship will launch bold, new initiatives to rapidly advance the field of pediatric cardiac critical care, elevate our leadership and improve the lifetimes of children with special hearts.

About the donors

Jim MacCutcheon’s involvement with Children’s National dates back to 1983. He has served in many leadership positions, most notably on the Children’s National and Children’s National Hospital Foundation board of directors. Jim and his daughters, Megan MacCutcheon, Candice Kessler and Colleen Crowley have supported Children’s National Heart Institute by funding the development of the MacCutcheon Cardiovascular Operating Suite and the Halle MacCutcheon Playroom on the heart and kidney unit. They have also provided support for the Peter Holbrook Endowed Lecture in Critical Care Medicine, the mobile health clinic and various events and capital improvement projects.

The MacCutcheon family’s investment to establish the MacCutcheon Family Professorship in Cardiac Critical Care Medicine allows Dr. Munoz and his team to provide innovative care utilizing telemedicine and artificial intelligence in support of our patients with special hearts.

Mary Donofrio

Mary Donofrio, M.D., FAAP, FACC, FASE, named as The Van Metre Companies Professor of Fetal Cardiology

Mary Donofrio

Children’s National Hospital named Mary Donofrio, M.D., FAAP, FACC, FASE, as The Van Metre Companies Professor of Fetal Cardiology at Children’s National Hospital.

Children’s National Hospital named Mary Donofrio, M.D., FAAP, FACC, FASE, as The Van Metre Companies Professor of Fetal Cardiology at Children’s National Hospital.

Dr. Donofrio serves as Medical Director of the Prenatal Cardiology Program and Critical Care Delivery Program, Director of the Advanced Cardiac Imaging Fellowship and Co-Director of the Cardiac Neurodevelopmental Outcome Program at Children’s National. She is a Professor of Pediatrics at George Washington University and is the founding and current President of the Fetal Heart Society, a non-profit organization created to advance the field of fetal cardiovascular care and science through collaborative research, education and mentorship.

About the award

Dr. Donofrio joins a distinguished group of 42 Children’s National physicians and scientists who hold an endowed chair. Professorships at Children’s National support groundbreaking work on behalf of children and their families and foster new discoveries and innovations in pediatric medicine. These appointments carry prestige and honor that reflect the recipient’s achievements and donor’s forethought to advance and sustain knowledge.

Dr. Donofrio is an international expert in fetal cardiology. She specializes in the fetal diagnosis and assessment of cardiovascular disease and the in-utero and delivery room management of newborns with complex congenital heart disease (CHD). Dr. Donofrio created an evidenced-based risk-assessment protocol for delivery room management which is now the standard of care for newborns with CHD. Dr. Donofrio has been a co-investigator on several NIH sponsored studies assessing in utero factors that influence neurodevelopmental outcome in children with CHD and more recently a study designed to minimize brain injury during heart surgery using cardiopulmonary bypass. She has published more than 130 papers, including the American Heart Association Scientific Statement on the Diagnosis and Treatment of Fetal Cardiac Disease.

The Van Metre Companies, through their vision and generosity, are ensuring that Dr. Donofrio and future holders of this professorship will launch bold, new initiatives to rapidly advance the field of fetal cardiology, elevate our leadership and improve the lifetimes of children with special hearts.

About the donors

For the past 65 years, Van Metre Companies has remained one of the Greater Washington D.C. area’s most successful, private, multi-faceted real estate developers. Albert G. Van Metre, the founder of Van Metre Companies, established a tradition of philanthropy focused on local charities. As a homegrown business, perpetuating that legacy of local giving is both a responsibility and a source of pride. The Van Metre Companies Professor of Fetal Cardiology honors Albert G. Van Metre’s memory by continuing this tradition of commitment to the community they call home.

The Van Metre Companies hosts the Annual Van Metre 5K Run in support of Children’s National, a longstanding tradition that has raised nearly $3 million in the past 30 years. In 2010, Children’s National dedicated the Van Metre Companies Cardiovascular Surgery Operating Room, a state-of-the-art cardiovascular surgery suite which was funded through the Annual Van Metre 5K Run. They also established The Van Metre Companies Professorship in Cardiology held by Charles Berul, M.D., Chief of Cardiology and Co-director of Children’s National Heart Institute.

 

US News Badges

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

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

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

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

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

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

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

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

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

NCC-PDI Finalists

Pediatric medical device competition names finalists

Five finalists have been named in the prestigious annual “Make Your Medical Device Pitch for Kids!” competition presented by the National Capital Consortium for Pediatric Device Innovation (NCC-PDI). Representing innovations in pediatric technologies that aim to address unmet medical needs for children, these five finalists now have access to a pediatric accelerator program led by MedTech Innovator and will compete for a share of $150,000 in grant funding from the U.S. Food and Drug Administration (FDA) in the final virtual pitch event in October 2022. The pediatric pitch event is part of the 10th Annual Symposium on Pediatric Device Innovation, co-located with the MedTech Conference, powered by AdvaMed.

“Addressing unmet needs across pediatric populations is critical to advancing children’s health and we are delighted to once again work with pioneering companies that seek to bridge this care gap,” says Kolaleh Eskandanian, Ph.D., M.B.A, P.M.P, vice president and chief innovation officer at Children’s National Hospital and principal investigator of NCC-PDI. “As an FDA-funded consortium, NCC-PDI serves as a critical device development resource, bringing together individuals and institutions that support viable pediatric innovations and create faster pathways to commercialization. We congratulate this year’s finalists and look forward to seeing the progress made in the coming months as they navigate the accelerator program.”

The following are the five pediatric device innovations that judges selected for the final competition:

  • CorInnova – Houston, TX – Minimally invasive biventricular non-blood contacting cardiac assist device to treat heart failure.
  • Innovation Lab – La Palma, CA – Mechanical elbow brace stabilizes tremors for pediatric ataxic cerebral palsy to improve the performance of Activities of Daily Living (ADLs).
  • Prapela – Biddeford, ME – Prapela’s incubator pad is the first innovation to improve the treatment of apnea of prematurity in over twenty years.
  • Tympanogen – Richmond, VA – Perf-Fix replaces surgical eardrum repair with a nonsurgical clinic procedure
  • Xpan – Concord, Ont. – Xpan’s universal trocar enables safest and most dynamic access and effortless upsizing in conventional/mini/robotic procedures.

Beginning in June 2022, the five finalists will participate in a pediatric-focused track of the MedTech Innovator accelerator, the world’s largest accelerator of medical devices.

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, which lags significantly behind the progress of adult medical devices. 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, BioHealth Innovation and design firm Archimedic.

To date, NCC-PDI has mentored nearly 200 medical device sponsors to help advance their pediatric innovations, with 16 devices having received either their FDA market clearance or CE marking.

The accelerator program is the consortium’s latest addition to a network of resources and experts that it provides in support of pediatric innovators.

Eskandanian adds that supporting the progress of pediatric innovators is a key focus of the new Children’s National Research & Innovation Campus, a one-of-its-kind ecosystem that drives discoveries that save and improve the lives of children. On a nearly 12-acre portion of the former, historic Walter Reed Army Medical Center in Northwest Washington, D.C., Children’s National has combined its strengths with those of public and private partners, including industry, universities, federal agencies, start-up companies and academic medical centers. The campus provides a rich environment of public and private partners which, like the NCC-PDI network, will help bolster pediatric innovation and commercialization.

NCC-PDI Finalists social card

Yves D'Udekem

Yves d’Udekem, M.D., Ph.D., named as The Baier Family Distinguished Professor of Cardiac Surgery

Yves d'UdekemChildren’s National Hospital named Yves d’Udekem, M.D., Ph.D., as The Baier Family Distinguished Professor of Cardiac Surgery at Children’s National Hospital.

Dr. d’Udekem serves as chief of cardiac surgery at Children’s National Hospital, co-director of Children’s National Heart Institute, and professor of surgery and pediatrics, The George Washington University School of Medicine and Health Sciences.

About the award

Dr. d’Udekem joins a distinguished group of 42 Children’s National physicians and scientists who hold an endowed chair. Professorships at Children’s National support groundbreaking work on behalf of children and their families and foster new discoveries and innovations in pediatric medicine. These appointments carry prestige and honor that reflect the recipient’s achievements and donor’s forethought to advance and sustain knowledge.

Amy and Bret Baier, through their vision and generosity, are ensuring that Dr. d’Udekem and future holders of this professorship will launch bold, new initiatives to rapidly advance the field of pediatric cardiac surgery, elevate our leadership and improve the lifetimes of children with special hearts.

About the donors

The Baiers’ oldest son Paul was born with five congenital heart defects and needed open heart surgery when he was just 12 days old. He has since had three more open heart surgeries and countless procedures and visits to Children’s National. Dr. d’Udekem serves as Paul’s current heart surgeon.

Amy serves as Board Chair of Children’s National Hospital Foundation. Amy and Bret have served numerous times as co-chairs of the Children’s Ball, and Bret has emceed since 2008. They also are co-chairs for follow the leader, the hospital’s comprehensive campaign to fund bold initiatives in pediatric medicine. Children’s National is grateful for the Baiers’ generosity, leadership and commitment.

“I am forever grateful for the Baiers’ generosity and dedication to Children’s National,” said Kurt Newman, M.D., president and CEO of Children’s National Hospital. “With this endowed professorship, Yves will advance the field of pediatric cardiac surgery for kids with special hearts. I am honored to call Amy and Bret friends and partners in pediatric care.”

caspase molecule

Caspases may link brain cell degeneration and cardiac surgery

caspase molecule

The review summarizes both the known physiological roles of caspases as well as some of the well-characterized neurotoxic effects of anesthetics in pre-clinical models.

A review article in the journal Cell Press: Trends in Neuroscience outlines the wide variety of cellular signaling roles for caspase proteins — a type of cellular enzyme best known for its documented role in the natural process of cell death (apoptosis). The authors, including Nemanja Saric, Ph.D., Kazue Hashimoto-Torii, Ph.D., and Nobuyuki Ishibashi, M.D., all from Children’s National Research Institute, pay particular attention to what the scientific literature shows about caspases’ non-apoptotic roles in the neurons specifically. They also highlight research showing how, when activated during a cardiac surgery with anesthesia and cardiopulmonary bypass, these enzymes may contribute to the degeneration of brain cells seen in young children who undergo heart surgery for critical congenital heart defects (CHDs).

Why it matters

The review summarizes both the known physiological roles of caspases as well as some of the well-characterized neurotoxic effects of anesthetics in pre-clinical models.

The authors propose that these non-apoptotic activities of caspases may be behind some of the adverse effects on the developing brain related to cardiac surgery and anesthesia. Those adverse effects are known to increase risk of behavioral impairments in children with congenital heart disease who underwent cardiac surgery with both anesthesia and cardiopulmonary bypass at a very young age.

This work is the first to propose a possible link between developmental anesthesia neurotoxicity and caspase-dependent cellular responses.

The patient benefit

Better understanding of the time and dose-dependent effects of general anesthetics on the developing brain, particularly in children who have genetic predispositions to conditions such as CHDs, will help researchers understand their role (if any) in behavioral problems often encountered by these patients after surgery.

If found to be a contributing factor, perhaps new therapies to mitigate this caspase activity might be explored to alleviate some of these adverse effects on the developing brain.

What’s next?

The authors hope to stimulate more in-depth research into caspase signaling events, particularly related to how these signaling events change when an anesthetic is introduced. Deeper understanding of how anesthetics impact caspase activation in the developing brain will allow for better assessments of the risk for children who need major surgery early in life.

Children’s National leads the way

Children’s National Hospital leads studies funded by the U.S. Department of Defense to better understand how these other roles of caspases, which until now have not been well-documented, may contribute to brain cell degeneration when activated by prolonged anesthesia and cardiopulmonary bypass during cardiac surgery for congenital heart disease.

human heart

Heart anatomy determines outcomes of valve repair for single ventricle hearts after Fontan procedure

human heart

The data shows that the valve repair surgery itself doesn’t increase the likelihood of heart transplant or death. Instead, it is only those with right ventricle dominant heart function who are significantly more likely to have such a negative outcome.

A new study in the Journal of the American College of Cardiology finds the anatomy of the heart is a key predictor of how efforts to repair atrioventricular valve regurgitation — or a leaky heart valve — will impact children with single ventricle heart defects who have undergone a Fontan surgical procedure.

The study uses retrospective data from the largest database of patients who have had the Fontan procedure, the Australia and New Zealand Fontan Registry. The data shows that the valve repair surgery itself doesn’t increase the likelihood of heart transplant or death. Instead, it is only those with right ventricle dominant heart function who are significantly more likely to have such a negative outcome.

It was conducted by cardiac surgeons at Royal Children’s Hospital, including Yves d’Udekem, M.D., Ph.D., who is now chief of cardiac surgery at Children’s National Hospital. Dr. d’Udekem presented the findings at the recent American College of Cardiology Scientific Sessions in Washington, D.C.

What this means

Until now, it was unclear why patients who had undergone a Fontan heart procedure were more likely to need a heart transplant or die after they also underwent surgery to repair atrioventricular valve regurgitation. This type of leaking valve is common in patients who have undergone a Fontan procedure, and it can also be dangerous if left untreated. But because existing data showed poor outcomes following atrioventricular valve repair, it was considered high risk to perform this repair on children with Fontan circulation.

However, this study drilled down into the outcomes of atrioventricular valve repair for these patients and found that it isn’t the surgery that leads to a poor outcome. Instead, it’s a specific anatomic feature — having a dominant right ventricle — that is predictive of the outcome.

Up to now, it was unclear whether surgery should be offered to all patients with a Fontan circulation who had leaky atrioventricular valves. This study shows that things are different for patients with dominant left or dominant right ventricle. For patients with dominant right ventricle, leaving this regurgitation not repaired is much more likely to lead to death and transplantation, and these patients should be operated at the earlier stages of the deficiency of their valves.

The hold-up in the field

One of the biggest challenges to identifying evidence-based best practices for children born with single ventricle heart defects, which are critical congenital heart defects, is the small number of patients at any one institution each year. The Australia and New Zealand Fontan Registry, founded by Dr. d’Udekem and the team at Royal Children’s Hospital, forms one of the world’s longest standing databases of patient information, including outcomes, for this population.

The patient benefit

This data can help doctors and families make the best care decisions possible for children with single ventricle defects by understanding how each child’s unique anatomy may impact how their heart will respond to treatment.

What’s next

Dr. d’Udekem hopes results from this study will improve how doctors strategize and recommend (or not) surgical repair of atrioventricular valve regurgitation. Additionally, the study shows the value of centralized patient registries and data for informing the standard of care. Similar registries across the world may promise to provide even greater insight into the long-term outcomes for patients born with these congenital heart conditions.

CICU telemed command center

New telehealth command center redefines hospital care

CICU telemed command center

The new CICU command center redefines hospital care for children with the riskiest heart conditions.

Children’s National Hospital has opened a new telehealth command center that uses cutting-edge technology to keep continuous watch over the most fragile children with critical heart disease. The new command center, located in the Cardiac Intensive Care Unit (CICU), builds off a care model deployed outside the CICU in 2019, but now offers improved collaborative communication to better help predict and prevent major events, like cardiac arrest.

“The new center acts like an air traffic control tower. It allows our telehealth team to remotely monitor each patient room and alert the bedside team to any abnormal trends,” says Ricardo Munoz, M.D., executive director of the Telemedicine Program and chief of the Division of Critical Care Medicine at Children’s National. “Better communication with the bedside team and easier access to all members of the medical and surgical teams means we can provide the best possible care to these fragile infants.”

In the early stages of recovery after heart surgery, vulnerable patients can regress quickly with few outward physical symptoms. The telehealth virtual surveillance system flags early warning signs that a critically ill infant may suffer a serious adverse event.

The system combines traditional remote monitoring, video surveillance and an artificial intelligence algorithm. New features include:

  • Neuromonitoring: Doctors are developing an adaptable neuromonitoring system that can help signal an impending brain injury before it happens.
  • Echocardiography: A cardiologist, embedded in the new command center, is available to expedite and improve communication within the multidisciplinary team.
  • Real-time audio and video: A telemedicine cart is placed in the room of high-risk patients to allow for rapid communication with a bedside nurse.
  • E-CICU team: A team of CICU nurses and physicians implement a second layer of safety over patient care by doing a systematic review of video feeds from patient rooms, patient monitors, artificial intelligence tools trained to detect dangerous trends, labs and imaging studies.

Since the center’s launch, the system’s virtual surveillance has tracked 8,697 virtual surveillance activities, resulting in:

  • 2,350 non-critical communications
  • 199 critical communications that prevented major adverse events

“Parents of our highest-risk patients are comforted knowing our team of doctors and nurses are watching their child at every moment,” says Dr. Munoz. “We are one of the few pediatric hospitals in the world integrating telemedicine into our CICU. Our goal is to expand our monitoring capabilities both nationally and internationally to improve pediatric heart care.”

Children’s National is a recipient of the Telehealth Accreditation from URAC and is the first pediatric hospital to receive this recognition for its ability to leverage technologies to advance high-quality care that promotes clinical best practices, consumer protections and care continuity between patients and providers.

The Board of Visitors Telehealth Command Center was funded by an Auxiliary Board of Children’s National.

doctors doing heart surgery

Innovative hypoplastic left heart syndrome treatment offers hope for highest risk children

doctors doing heart surgery

A recently published study in the Journal of Thoracic and Cardiovascular Surgery is a unique report of outcomes for infants treated using a staged surgical approach such as the “hybrid strategy.”

Adopting a staged surgical strategy as the standard of care for medically fragile children with hypoplastic left heart syndrome (HLHS), a critical congenital heart defect, shows promise as an alternative care path for those who may not be ideal candidates for open heart surgery immediately after birth.

A recently published study in the Journal of Thoracic and Cardiovascular Surgery is a unique report of outcomes for infants treated using a staged surgical approach such as the “hybrid strategy.” This initially less-invasive technique involves the placement of small bands on both lung vessels, with or without the placement of a stent on the ductus arteriosus, as the first stage in surgical palliation.

The study reports results from applying this hybrid approach as a bridge to either a delayed Norwood operation or a comprehensive stage II operation. Over 3.5 years, 30 patients with HLHS were considered very high risk for surgery based on their preoperative risk factors such as low birth weight and/or gestational age, shock, and other medical conditions. During that time, the overall survival rate for this group was 70 percent. In the past, using traditional approaches, the survival chance for infants with HLHS and these high-risk factors was extremely limited.

Why it matters

This new surgical strategy gives the baby extra time to grow and allows doctors to collect detailed analysis of potentially treatable accompanying conditions. Furthermore, high-risk babies recover from birth trauma and have the chance to continue developing crucial organs before undergoing more traditional procedures for HLHS that require open-heart surgery with cardiopulmonary bypass. It also allows surgeons to make an individualized risk assessment for which surgical step should be taken next, replacing the historical “one size fits all” operative pathway for HLHS. The traditional operative pathway for HLHS is a series of three open-heart surgical procedures: the traditional Norwood operation, the bidirectional Glenn and the Fontan.

Advancing the standard of care for HLHS patients beyond the current best practice approach to improve outcomes for more newborns has been slow for several reasons, the authors note. One main reason is that performing a hybrid procedure on these particularly fragile infants requires advanced devices and additional technical expertise. As one example, the authors note that until recently, there was no stent available in the appropriate sizes and with the right material properties to work within such a tiny ductus arteriosus. However, case-by-case expanded access approval by the FDA has brought a new stent designed specifically for this use from Europe to the U.S. for the first time.

What they’re saying

“The Norwood operation revolutionized the care of children with HLHS in the 1980s and gave them a chance for survival,” says Can Yerebakan, M.D., senior author of the study and cardiac surgeon at Children’s National who oversees the hybrid program alongside Joshua Kanter, M.D., director of Interventional Cardiology. “This staged decision-making strategy may give the same kind of hope and offers an alternative pathway of care for high-risk patients who would otherwise have a dismal prognosis and extremely low chance   of survival in the newborn period. The success in these cases is, however, based on a multidisciplinary team approach.”

What’s next?

“This strategy not only shows promise for improved short-term survival in high-risk patients, but also boasts the potential to convert some patients to two-chamber circulation instead of one, which our team has done with 100% survival,” says Nicolle Ceneri, M.D., first author of the study and pediatric resident at Children’s National. “As time goes on, we are eager to discover how the use of this approach during such a tenuous period impacts the long-term outcomes for these children and their quality of life.”

Read about the smallest baby born with HLHS to survive to 18 months, who was treated using this hybrid surgical approach at Children’s National Hospital.

Anitha John

Youth with heart defects need a smooth transition to age-appropriate heart care, says AHA

Anitha John

Anitha John, M.D., Ph.D., director of the Washington Adult Congenital Heart Program (WACH) at Children’s National Hospital and incoming chair of the AHA Young Hearts council, served as lead author on the statement, which provides the latest evidence-based best practices for a successful transition from pediatric care to adult care.

The American Heart Association (AHA) issued a scientific statement capturing the best practices for helping children with congenital heart disease successfully transition to adulthood and receive health care tailored to their needs as they continue to age. Ensuring a smooth and supported transition and establishing relationships with these young patients as they grow into adults is key to maintaining their engagement and connection to health care decisions that will improve their long-term health and well-being.

Anitha John, M.D., Ph.D., director of the Washington Adult Congenital Heart Program (WACH) at Children’s National Hospital and incoming chair of the AHA Young Hearts council, served as lead author on the statement, which provides the latest evidence-based best practices for a successful transition from pediatric care to adult care. This work is critical given that today, thanks to the tremendous advances in care and treatment of congenital heart defects in the last two decades, there are more adults living with congenital heart conditions than children.

What this means

The AHA describes a scientific statement as an “expert analysis of current research” that “can inform future care guidelines.” This scientific statement demonstrates the scientific evidence supporting what adult congenital heart specialists have advised for years—that making sure children with congenital heart defects continue to stay engaged in their care and actively seek out health care specialized for them as they grow through adolescence and into adulthood is critical. Keeping that connection plays a pivotal role in their overall quality of life as they age.

Why it matters

Treatments and care for children with congenital heart defects has improved so greatly that, according to the AHA, “most people born with heart defects today, including those with complex heart conditions, survive past childhood and become adults.” But the same care they received as children is not enough to address their needs as adults. Adult-oriented congenital heart care can be the difference between a long and healthy life or continued health challenges and dangerous side effects. Adults with congenital heart conditions should seek out care that serves them best, and it should be accessible to everyone who needs it.

By issuing this updated scientific statement, the AHA is broadcasting the important take home message that adults with congenital heart disease and their care providers need (and should seek) access to an adult-focused program with expertise in caring for the unique challenges they face. Establishing that connection at the transition point from adolescence to adulthood can set the stage for long term engagement and health.

Children’s National Hospital leads the way

As director of the WACH program at Children’s National, Dr. John is one of the nation’s experts in care for adults living with congenital heart disease. She also leads significant patient-centered research efforts focused on understanding barriers to care and other challenges faced by these adult heart patients, including serving as co-principal investigator on one of the largest patient-centered studies of adults living with congenital heart disease, supported by the Patient-Centered Outcomes Research Institute (PCORI). The findings from these studies will help fuel further recommendations and guidelines that will improve the standards of care for these patients.

Read the AHA News overview of the Scientific Statement.

Read the Scientific Statement from the Journal of the American Heart Association.

Ashley Vela Mercedes

Staged surgical hybrid strategy changes outcome for baby born at 28 weeks with HLHS

Ashley Vela MercedesA staged, hybrid cardiac surgical strategy can give fragile infants with hypoplastic left heart syndrome (HLHS) critical time to grow and get stronger. It also gives doctors more time to understand and care for any complicating conditions before necessary open-heart surgery. Doctors at Children’s National Hospital used such a staged approach, called the “hybrid strategy,” to care for Ashley Vela Mercedes, who was born at only 28-weeks-old weighing 1.1 kilos.

Ashley, who is now a smiling and happy 18-month-old, is believed to be the smallest infant born at 28-weeks with HLHS to survive to this age. Though she will need ongoing care and future procedures, her family is grateful for the knowledge and technical expertise of her care team at Children’s National.

The hybrid program at Children’s National is led by Can Yerebakan, M.D., cardiac surgeon, and Joshua Kanter, M.D., director of Interventional Cardiology.

A hypoplastic left heart syndrome miracle

Ashley Vela Mercedes is a happy, smiling 18-month-old. She’s also a true miracle — the smallest baby in the world with HLHS to survive to this age.

Born when her mom was only 28 weeks pregnant, she was 1.1 kilos/2.4 pounds when she arrived — the size of a beanie baby stuffed animal — and her heart was about the size of a grape.

Between her premature birth and her medical issues including her critical HLHS heart condition, her parents, Ana Mercedes and Axel Vela were told that Ashley was unlikely to survive.

The Velas were frustrated, sad and scared. It started to sound like there wasn’t much hope for their tiny baby. They had always dreamed of having a family of their own. Nothing had prepared them for this.

Hope in the hybrid procedure

Their cardiologist, Jennifer Lindsey, M.D., wasn’t ready to give up either. She reached out to the team at Children’s National Hospital to see if Ashley might be considered for a new type of surgery — called a “hybrid procedure.” She hoped that this hybrid procedure might stabilize Ashley’s heart until she could grow strong enough for the open-heart surgeries she would need later.

Hybrid heart procedures blend traditional surgery and a minimally invasive interventional, or catheter-based, procedure. A stent is implanted in the newborn’s ductus arteriosus to hold it open and keep the baby’s blood flowing more efficiently. Usually, the ductus serves as a normal fetal blood vessel that increases blood flow to the mother’s placenta while in utero and closes after the baby is born, when it is not needed.

The hybrid procedure to hold open the ductus isn’t a permanent fix — it just buys the baby extra time to grow and develop before open-heart surgery to start rebuilding the heart in a more sustainable way. They are only considered an option for infants at extremely high risk for open-heart surgery. That’s also why very few congenital heart centers in the world offer them.

The hybrid team at Children’s National is led by Drs. Yerebakan and Kanter have worked together on more hybrid procedures for tiny and fragile infants than most other places. They’ve also worked with the U.S. Food and Drug Administration to safely bring a smaller and more flexible, toothpick-sized stent to the U.S. from Europe that’s made especially for use in the ductus arteriosus of special cases like Ashley.

Record-setting smallest hybrid surgery

Ashley Vela MercedesAccording to Gil Wernovsky, M.D., a cardiac intensive care doctor at Children’s National and Ashley’s Children’s National cardiologist, her birth weight was too low — she weighed 1.1 kilos or 2.4 pounds — for surgery. Her lungs also were not developed enough. She was so small, she lived in the hospital for several more weeks under the care of Dr. Lindsey. Dr. Wernovsky credits the team at Inova Children’s Hospital for providing the support Ashley needed so she could grow and develop to a place where the hybrid procedure was possible.

When she reached 1.7 kilos or 3.7 pounds, she was transported to the Cardiac Intensive Care Unit at Children’s National Hospital for her first procedure, the hybrid. At the time, she was officially the smallest baby to have a hybrid procedure at Children’s National. After the surgery, the Children’s National CICU was Ashley’s home for almost her entire first year of life.

“Landing here at Children’s was a blessing because we thought she was going to die. What has happened here is a miracle,” said Ana Mercedes. “I would like to tell Dr. Yerebakan and Dr. d’Udekem (the chief of Cardiac Surgery at Children’s National) that what they have done with my daughter is incredible, and I will never be able to fully repay them for their efforts.”

Over the next 11 months in the hospital, Ashley had many surgeries and catheterizations, countless other medical procedures and tests, and many, many ups and downs, including some very scary times when she required extracorporeal membrane oxygenation (ECMO) for critical life support.

Celebrating and thriving at home

Ashley Vela MercedesIn July 2021, Ashley went home for the first time in her life. She hasn’t needed to be hospitalized since her discharge. She’s monitored jointly by Dr. Lindsey and the Children’s National single ventricle monitoring program. That program stays in touch with families virtually three times each week. Her care team keeps up with her growth and development through telehealth. Ana Mercedes shares videos and photos of Ashley as part of her routine updates to the clinical team.

She still needs some medical support at home and will likely need additional medical interventions down the road, but for the time being, she is a more independent, happier and thriving little girl.

And she’s a miracle — she is the smallest baby in the world born with HLHS at such a small size and weight to survive to this age.

Ana Mercedes dreams of taking Ashley to the Dominican Republic one day, so they can enjoy the famous beaches and Ashley can be introduced to her heritage since that’s where Ana Mercedes is from.

“We are overall doing well. We have received support from our family and employers,” said Ana Mercedes. “We are extremely grateful to everyone who has given us a hand during this challenging time.”

The last year has been a roller coaster, but Ashley has made incredible progress and exceeded everyone’s expectations. Dr. Wernovsky notes, “Last year at Christmas time, this little girl was on her fourth catheter procedure and had been in intensive care for her entire life. One year later, she’s at home with her family and celebrating — that’s quite the Christmas miracle.”

Handrawn illustration of human Kidneys

Children’s National Nephrology team presents virtually at IPTA’s 11th Congress

Handrawn illustration of human KidneysThe International Pediatric Transplant Association (IPTA) is hosting their 11th Congress meeting March 26-29, 2022, and many Children’s National Hospital providers will be presenting throughout the conference. We hope you will join us!

Diversity, inequity and inclusivity in the practice of pediatric transplantation in the U.S.

Presenter: Marva Moxey-Mims, M.D., division chief, Nephrology

Dr. Moxey-Mims will review the trends in pediatric kidney transplantation over the past decade, focusing on differences by race and ethnicity, whether the gaps have narrowed over that time and steps that can be taken to increase equity.

Prevalence of mycophenolate mofetil discontinuation and subsequent outcomes in pediatric kidney transplant recipients: A PNRC study

Presenter: Asha Moudgil, M.D., medical director, Kidney Transplant

Mycophenolate mofetil (MMF) is a common maintenance immunosuppressant in children receiving kidney transplants but is often discontinued for various reasons. In this multi-center Pediatric Nephrology Research Consortium study, researchers sought to determine the prevalence and reasons for MMF discontinuation and its association with patient and allograft outcomes. They will be discussing their methodology and results from this study.

Additional Children’s National staff involved in the study include:

Changes in diastolic function and cardiac geometry after pediatric kidney transplantation: A longitudinal study

Presenter: Kristen Sgambat, Ph.D., clinical assistant professor

Children with end stage kidney disease are at high risk for cardiovascular morbidities. Indicators of systolic function, such as ejection fraction and fractional shortening, are often preserved and may not reveal cardiac dysfunction until it is severe. Longitudinal changes in diastolic function and cardiac geometry have not been well studied.

Additional Children’s National staff involved in the study include:

  • Asha Moudgil, M.D., medical director, Kidney Transplant
  • Sarah Clauss, M.D., cardiologist

COVID19 in pediatric kidney transplant recipients: Incidence, outcomes, and response to vaccine

Presenter: Asha Moudgil, M.D., medical director, Kidney Transplant

At the start of the pandemic, no information was available on the outcomes of pediatric kidney transplant recipients with COVID-19. When the COVID-19 vaccine became available, response of immunosuppressed children to the vaccine was not known. While more information has become available in adult transplant recipients, information on pediatric transplant recipients remains limited.

The team will discuss their methodology of collecting information and their results and conclusions.

Additional Children’s National staff involved in the study include:

Poster presentation: Psychological functioning and adaptive behavior in pediatric patients awaiting renal transplantation.

Presenter: Kaushal Amatya, Ph.D., psychologist

Psychosocial functioning of children with chronic kidney disease (CKD) at pretransplant evaluation is associated with transplant readiness and post-transplant outcomes. Higher prevalence of emotional/behavioral issues is noted in children with CKD compared to healthy counterparts. Although issues with functional impairment is often reported, research on adaptive functioning using a validated measure is lacking. The study aimed to explore psychological and adaptive functioning in pediatric pre-transplant patients to identify areas in need of intervention.

Additional Children’s National staff involved in the study include:

  • Asha Moudgil, M.D., medical director, Kidney Transplant
  • Paige Johnson, Psychology resident

 

doctor listening to patients heart

Children’s National leads patient-centered study of adult congenital heart disease

doctor listening to patients heart

The team will work with the 14 research institutions across the United States to collect data and then examine rates of complications or associated illnesses, as well as how adult patients have accessed health care throughout their lives.

Investigators at Children’s National Hospital and the Louisiana Public Health Institute are leading a $4.9 million research effort to study how gaps in health care affect the health and well-being of adults with congenital heart disease (CHD), supported by the Patient-Centered Outcomes Research Institute (PCORI).

The research is led by Anitha John, M.D., Ph.D., medical director of the Washington Adult Congenital Heart Program at Children’s National Hospital and Thomas Carton, Ph.D., chief data officer at the Louisiana Public Health Institute, as well as two patient co-investigators, Ruth Phillippi and Scott Leezer. The study leads also include representatives from the Adult Congenital Heart Association and Anu Agarwal, M.D., who represents the University of California – San Francisco (UCSF).

“With the increasing number of adult patients with CHD, it is important for us to understand how current recommended practices influence patient outcomes,” says Dr. John. “This project will guide us on how to best care for our patients, not just through childhood, but across their entire lifespans. Most importantly, this project will involve direct outreach to patients, incorporating patient reported outcomes as a measure of long-term outcomes.”

Together, the team will work with the 14 research institutions across the United States to collect data and then examine rates of complications or associated illnesses, as well as how adult patients have accessed health care throughout their lives. The findings may help predict which patients are at greater risk of falling out of routine health care, and when these gaps in care are likely to occur across a patient’s lifespan. The study will also correlate findings with how patients are actually feeling in their everyday lives.

The 14 participating institutions are:

  • Ochsner Health
  • Children’s Hospital of Philadelphia
  • Icahn School of Medicine at Mount Sinai
  • University of California – San Francisco
  • Nationwide Children’s Hospital
  • Duke University Health System
  • NYU Grossman School of Medicine
  • Nicklaus Children’s Hospital
  • Children’s Hospital Colorado
  • University of Miami
  • Columbia University Irving Medical Center
  • Cincinnati Children’s Hospital Medical Center
  • Weill Cornell Medical College
  • University of Florida

“This unprecedented look at the health of adults living with congenital heart disease allows us to get a full spectrum view by combining clinical data with patient-reported health data,” says Dr. Carton.

The first patient-powered registry for adults with CHD — the Congenital Heart Initiative (CHI) is a key component of this research. Launched with seed funding from Children’s National and the Heart Research Alliance at UCSF, the CHI is led by Dr. John and her team at Children’s National in addition to a broad multi-stakeholder advisory board, including patients. The CHI was co-developed with input from patients, clinicians and researchers and continues to involve these voices in the advancement of the registry. Patients who are recruited for this research will participate via enrollment in the registry, which will allow researchers to ask patients directly about health, wellness and any specific barriers to care. Learn more about CHI’s progress in their first annual report.

“Patients, like myself, are charting a new course and we desire answers to significant questions, as do our providers, about impact of lifelong specialized care, along with improved understanding of the quality-of-life patients experience,” says Leezer. “This project represents a huge step forward towards obtaining answers for the adult CHD community.”

The study also draws on the vast health data resources of PCORnet®, the National Patient-Centered Clinical Research Institute, to conduct this study more efficiently. With health records for 66 million patients available for observational studies, the PCORI-funded PCORnet provides vast scale to power research on conditions affecting even small numbers of people.

“We are confident that this research will yield critical learnings that will empower the community, align resources and spur future innovation to better meet the specialized care needs of this emerging population,” says Mark Roeder, president and chief executive officer for the Adult Congenital Heart Association.

PCORI is an independent, nonprofit organization authorized by Congress in 2010 to fund research that will provide patients, their caregivers and clinicians and other health care decision makers with the evidence-based information needed to make better-informed health care choices.

NCC-PDI logo

Pediatric medical device competition takes aim at congenital heart disease

NCC-PDI logo

Consistent with its mission of addressing the most pressing pediatric device needs, this year’s competition focused on innovations in electrophysiology devices that monitor and treat congenital heart disease (CHD) and arrhythmias in pediatric patients.

The National Capital Consortium for Pediatric Device Innovation (NCC-PDI) announces five awardees chosen in its prestigious annual “Make Your Medical Device Pitch for Kids!” competition to share $150,000 in grant funding from the U.S. Food and Drug Administration (FDA) to support the advancement of pediatric medical devices. In an unprecedented decision, the competition judges determined that all five finalists were deserving of a grant award and recognition for the potential patient benefit and commercial viability of their innovations.

Consistent with its mission of addressing the most pressing pediatric device needs, this year’s competition, conducted by NCC-PDI partner MedTech Innovator, focused on innovations in electrophysiology devices that monitor and treat congenital heart disease (CHD) and arrhythmias in pediatric patients. The virtual pediatric pitch event was part of the 9th Annual Symposium on Pediatric Device Innovation.

This year’s pediatric device innovation awardees are:

  • PeriCor – The Children’s Hospital at Montefiore – New York, NY, and Children’s National Hospital – PeriTorq, a catheter grip tool for use during pediatric cardiac interventional procedures;
  • Inkspace Imaging – Pleasanton, CA – a pediatric cardiac and vascular MRI coil;
  • Karios Technologies – Charlottesville, VA – Tissue Shield, a technology to prevent scar tissue formation (adhesions) on the heart after surgery;
  • Sibel – Niles, IL – ANNE One, ICU-grade wireless sensors for cardiopulmonary monitoring in neonates with congenital heart defects;
  • Starlight Cardiovascular – San Diego, CA – Project Lifeline, a less-invasive way to maintain sufficient circulation in newborns with ductal-dependent circulation that increases safety, procedural success and ease of use.

Congenital heart disease (CHD) affects six out of 1,000 babies born in the U.S. each year and is often complicated by arrhythmias, a condition where the heart beats too rapidly, too slowly or irregularly due to a misfiring of the body’s electrical impulses. While the last decade brought great advances in technologies that improve the care of adult arrhythmias, pediatric patients have been left behind, with only five devices approved for use in children in the same period. As a result, pediatric specialists are often using off-label or improvised devices to treat pediatric arrhythmias, including in the smallest newborns.

“Recognizing this unmet need, NCC-PDI opened the challenge earlier this year to select companies to enter MedTech Innovator’s pediatric accelerator program, made possible by NCC-PDI. The five companies have immensely benefited from the accelerator program and are well-positioned to compete for funding. They have the potential to advance pediatric health and provide a greater standard of care for children living with CHD,” says Kolaleh Eskandanian, Ph.D., M.B.A, P.M.P, vice president and chief innovation officer at Children’s National Hospital and principal investigator of NCC-PDI. “For too long, the unique needs of children have been overlooked in pediatric device development. Thanks to the support of the FDA, we are able to build our challenge competitions around the direst unmet needs, which are determined through a thorough needs assessment and market analysis conducted to inform each request for proposal. The funding incentivizes pediatric innovation and helps more companies navigate the path to commercialization.”

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, which lags significantly behind the progress of adult medical devices. NCC-PDI is led by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Hospital and the A. James Clark School of Engineering at the University of Maryland, with support from partners MedTech Innovator,  BioHealth Innovation and design firm Archimedic.

A pediatric accelerator program, powered by MedTech Innovator, is the consortium’s latest addition to a network of resources and experts that NCC-PDI provides in support of pediatric innovators. All five of this year’s competition finalists had an opportunity to participate in the year-long accelerator program.

Eskandanian adds that supporting the progress of pediatric innovators is a key focus of the new Children’s National Research & Innovation Campus, a one-of-its-kind ecosystem that drives discoveries that save and improve the lives of children. On a nearly 12-acre portion of the former, historic Walter Reed Army Medical Center in Northwest Washington, D.C., Children’s National has combined its strengths with those of public and private partners, including industry, universities, federal agencies, start-up companies and academic medical centers, the campus provides a rich environment of public and private partners which, like the NCC-PDI network, will help bolster pediatric innovation and commercialization.

Charles Berul

Charles Berul, M.D., receives Meritorious Achievement Award

Charles Berul

Charles Berul, M.D., chief of Cardiology at Children’s National Hospital has earned a lifetime achievement award, the 2021 Council on Lifelong Congenital Heart Disease and Heart Health in the Young (Young Hearts) Meritorious Achievement Award.

The Meritorious Achievement Award recognizes a person whose achievements have made a significant impact in the field of congenital heart disease and heart health in the young and have helped to further the mission of the Young Hearts council. The council’s mission is to improve the health of children and adults with congenital heart disease and acquired heart disease during childhood through research, education, prevention and advocacy.

Dr. Berul received this award in recognition of his lifelong achievements in the field of pediatric electrophysiology. He is known for his development of innovative electrophysiologic studies for phenotypic evaluations of genetically manipulated pre-clinical models. Over the past two decades, his research focus and passion have been to develop novel minimally invasive approaches to the heart and improving methods for pediatric pacing and defibrillation.

He has also mentored dozens of trainees who have gone on to successful careers and particularly advocates for young investigators and clinician-scientists. He is known for his collaborative style and for supporting advancement of faculty physicians in academic medicine.

Dr. Berul has served on multiple society committees, task forces and writing groups, and is currently an associate editor for the Heart Rhythm Society’s journal. He is also actively involved in other key organizations such as Mended Little Hearts and the Pediatric and Congenital Electrophysiology Society (PACES). He has more than 300 publications and is an invited speaker nationally and internationally in the areas of pediatric cardiac electrophysiology and miniaturized device development.

Dr. Berul received the award on November 12 during a virtual presentation at the American Heart Association’s Scientific Sessions. He is the fourth Children’s National cardiologist to be recognized with this prestigious honor from the council in the last decade.

Dr. Sable performing an echocardiogram in Uganda

Penicillin slows impacts of rheumatic heart disease in Ugandan children

Dr. Sable performing an echocardiogram in Uganda

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

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

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

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

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

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

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

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

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

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

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

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

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

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

sick child in palliative care hospital bed

New study compares first and second wave of MIS-C

sick child in palliative care hospital bed

When comparing the first and second wave of patients diagnosed with multi-system inflammatory syndrome in children (MIS-C), the second wave patients had more severe illness, according to a new prospective cohort study at Children’s National Hospital in Washington, D.C.

When comparing the first and second wave of patients diagnosed with multi-system inflammatory syndrome in children (MIS-C), the second wave patients had more severe illness, according to a new prospective cohort study of 106 patients at Children’s National Hospital in Washington, D.C. The results, published in The Pediatric Infectious Disease Journal, show that despite increased severity in the second wave cohort, both cohorts had similarities in cardiac outcomes and length of stay. Researchers are still working to better understand the exact immunologic mechanisms that trigger MIS-C and the specific factors accounting for its rare occurrence.

“We’ve now seen three distinct waves of MIS-C since the beginning of the pandemic, each wave following national spikes in cases,” said Roberta DeBiasi, M.D., chief of the Division of Pediatric Infectious Diseases at Children’s National and co-author of the study. “Kids in the second wave cohort had potentially experienced intermittent and/or repeated exposures to the virus circulating in their communities. In turn, this may have served as repeated triggers for their immune system which created the more severe inflammatory response.”

In this new study, key demographic features Children’s National researchers previously identified held true across both waves – including the fact that Black and Latino children are significantly more affected than white children.  Of the 106 patients, 54% were Black and 39% were Hispanic. The authors also noted that 75% of the patients were otherwise healthy children with no underlying medical conditions.

“While we believe the most recent third wave associated with the delta variant surge is tapering off, the findings from the first two waves provide important baseline information and are highly relevant for clinicians across the country that are evaluating and treating kids with MIS-C,” said Dr. DeBiasi.

Children’s National has cared for more than 4,200 symptomatic patients with SAR-CoV-2 infection and more than 185 MIS-C patients since the pandemic began. The first wave of MIS-C patients were hospitalized between March 2020 and October 2020. Second wave patients were hospitalized between November 2020 and April 2021. Each wave came 4-6 weeks following periods of COVID-19 surges in the community.

In the study, researchers compared patient demographics, clinical features, laboratory results, radiographic images, therapies and outcomes. The second wave cohort had a higher proportion of children 15 years of age or older. Patients also presented more frequently with shortness of breath and required more advanced respiratory and inotropic support. Researchers also found that patients in the second wave were less likely to test positive for SARS-CoV-2 on a PCR test.

Dr. DeBiasi and her team hope to unlock even more insights as they now analyze data from the third wave associated with the delta variant, which currently appears to have affected less children than the previous two. Children’s National is also working in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID) to study the long-term effects of MIS-C and COVID-19 on the pediatric population after recovery. This is among the largest and longest studies being conducted, and researchers are hopeful the findings will help improve treatment of COVID-19 and MIS-C in the pediatric population both nationally and around the world.

“Our timely established multidisciplinary MIS-C task force here at Children’s National allowed us to reduce the learning curve,” said Ashraf S. Harahsheh, M.D., F.A.A.P., F.A.C.C., director of Quality Outcomes in Cardiology and co-first author of the study. “Experience from other centers showed that immunotherapy was utilized more frequently in recent MIS-C cohorts leading to reduction in percentage of cardiac complications. On the other hand, and despite having increased illness severity in the second cohort, our approach with prompt immunotherapy helped stabilize the rate of cardiac complications.”

brain network illustration

Cardiopulmonary bypass may cause significant changes to developing brain and nerve cells

brain network illustration

Cardiopulmonary bypass, more commonly known as heart-and-lung bypass, has some unique impacts on the creation and growth of brain cells in the area of a child’s brain called the subventricular zone (SVZ), according to a study in the Annals of Neurology. The SVZ is a critical area for the growth and migration of neurons and nerve cells called neuroblasts, both of which ultimately contribute to the proper development of key brain structures and functions during the early years of life.

The findings, from a study conducted in the Cardiac Surgery Research Laboratory at Children’s National Hospital, provide new insight into the cellular impacts of the cardiopulmonary bypass machine on brain growth and development for newborn infants with congenital heart disease. They will have an important role in the refinement of strategies to help protect the fragile brains of children who require lifesaving cardiac surgery with cardiopulmonary bypass immediately after birth.

Specifically, the research team found that during cardiopulmonary bypass:

  • Creation of neurons (neurogenesis) in the neonatal and infant subventricular zone is altered.
  • Migration of nerve cells, called neuroblasts, to the frontal lobe is potentially disrupted.
  • Changes to the growth and movement of neurons in the SVZ are prolonged.
  • Cortical development and expansion is impaired.
  • Specific types of neurons found only in the brain and spinal cord, called interneurons, are also affected.

The study uses an innovative pre-clinical model of the developing brain that is more anatomically and physiologically similar to human neonates and infants than those used in prior studies and in most neurological laboratory-based research.

Cardiopulmonary bypass is one of several key factors thought to cause children with congenital heart disease to sometimes demonstrate delays in the development of cognitive and motor skills. These disabilities often persist into adolescence and adulthood and can ultimately represent long-term neurocognitive disabilities. It is also believed that genetic factors, abnormal blood flow to the brain while in utero or low cardiac output after surgical procedures on the heart may contribute to these challenges.

“Unraveling cellular and molecular events during surgery using this preclinical model will allow us to design therapeutic approaches that can be restorative or reparative to the neurogenic potential of the neuronal stem precursor cells found in the subventricular zone of the neonatal or infant brain,” says Nobuyuki Ishibashi. M.D., Foglia-Hills Professor of Pediatric Cardiac Research, director of the Cardiac Surgery Research Laboratory at Children’s National and senior author on the study. “In particular, previous studies in our laboratory have shown improvement in the neurogenic activities of these precursor cells when they are treated with mesenchymal stromal cells (MSCs).”

The findings from this study further support the work already underway in the NIH-funded MeDCaP clinical trial for neonates and infants undergoing cardiac surgery using the cardiopulmonary bypass machine. That trial uses the heart and lung machine itself to deliver MSCs directly into the main arteries that carry blood to the brain.

Drs. Wernovsky and Martin

Cardiac care leaders recognized for mentorship and innovation at AAP

Two Children’s National Hospital cardiac care leaders received prestigious recognition awards from the American Academy of Pediatrics (AAP) during that organization’s virtual National Conference and Exhibition in October 2021.

  • Gil Wernovsky, M.D., cardiac critical care specialist at Children’s National Hospital, received the 2021 Maria Serratto Master Educator Award from AAP Section on Pediatric Cardiology and Cardiac Surgery, celebrating his 30-plus-years as a clinician, educator, mentor and leader in the field.
  • Gerard Martin, M.D., FAAP, FACC, FAHA, C. Richard Beyda Professor of Cardiology, Children’s National Hospital, received the AAP Section on Advances in Therapeutics and Technology (SOATT) Achievement Award, in recognition of his work to establish the use of pulse oximetry to screen newborn infants for critical congenital heart disease in the first 24 hours of life.

Dr. Wernovsky: 2021 Maria Serratto Master Educator Award, AAP Section on Pediatric Cardiology and Cardiac Surgery

Gil Wernovsky

Gil Wernovsky, M.D., received the 2021 Maria Serratto Master Educator Award from AAP Section on Pediatric Cardiology and Cardiac Surgery.

The Master Educator Award is presented each year to a pediatric cardiologist or cardiothoracic surgeon who exemplifies excellence as an educator, mentor and/or leader in the field.

A practicing cardiac critical care specialist with more than 30 years’ experience in pediatric cardiology, Dr. Wernovsky trained and mentored more than 300 fellows in pediatric cardiology, cardiac surgery, neonatology, critical care medicine and cardiac anesthesia, in addition to countless residents and fellows. He also organizes national and international symposia to share expertise around the world. During the COVID-19 public health emergency, for example, he co-founded the Congenital Heart Academy (CHA). The CHA provides content from an international faculty of cardiac care to more than 26,000 practitioners in 112 countries and includes a thriving YouTube channel.

Dr. Wernovsky is also a founding member of several international societies focused on bringing together clinicians, researchers and students across sub-specialties of pediatric cardiology and cardiac surgery for knowledge exchange and best practice sharing. These include: the Pediatric Cardiac Intensive Care Society, World Society for Pediatric and Congenital Heart Surgery, the International Society of Pediatric Mechanical Circulatory Support and the Cardiac Neurodevelopmental Outcome Collaborative.

Dr. Wernovsky received the award on October 10 at the virtual Scientific Sessions of the 2021 American Academy of Pediatrics National Conference and Exhibition.

Dr. Martin: AAP Section on Advances in Therapeutics and Technology (SOATT) Achievement Award

Gerard Martin

Gerard Martin, M.D., FAAP, FACC, FAHA, C. Richard Beyda Professor of Cardiology, Children’s National Hospital, received the AAP Section on Advances in Therapeutics and Technology (SOATT) Achievement Award.

The Section on Advances in Therapeutics and Technology (SOATT) educates physicians, stimulates research and development and consults on therapeutics and technology-related matters for the AAP. The Achievement Award recognizes someone who has shown leadership in applying innovative approaches to solve pressing problems.

Dr. Martin is the C. Richard Beyda Professor of Cardiology and has cared for children at Children’s National for more than 30 years. As an advocate for congenital heart disease efforts nationally and internationally, he played an integral role in the development of an innovative use of existing hospital technology—the pulse oximeter—to detect critical congenital heart disease in newborn babies.

Today, Dr. Martin and colleagues across the United States and around the world have worked to make this screening method a standard of care for newborns everywhere. It is a part of the Health Resources and Services Administration (HRSA) Recommended Uniform Screening Panel and has become law in every state. They continue to conduct research to refine the recommendations and hone-in on the most effective ways to harness these tools.

Dr. Martin was selected for this award in 2020. He accepted it and offered remarks during the 2021 virtual AAP National Conference and Exhibition on Monday, October 11, 2021.