Cardiology & Heart Surgery

red and grey kidney illustration

Cardiovascular and bone diseases in chronic kidney disease

red and grey kidney illustration

A new study reviews cardiovascular and bone diseases in chronic kidney disease and end-stage kidney disease patients with a focus on pediatric issues and concerns.

In a study published by Advances in Chronic Kidney Disease and co-authored by Aadil Kakajiwala, M.D., MSCI, critical care specialist and nephrologist at Children’s National Hospital, a team reviewed cardiovascular and bone diseases in chronic kidney disease and end-stage kidney disease patients with a focus on pediatric issues and concerns.

Chronic kidney disease is common and causes significant morbidity including shortened lifespans and decrease in quality of life for patients. The major cause of mortality in chronic kidney disease is cardiovascular disease. Cardiovascular disease within the chronic kidney disease population is closely tied to disordered calcium and phosphorus metabolism. The metabolic bone disease of chronic kidney disease encompasses vascular calcification and the development of long-term cardiovascular disease.

Recent data suggest that aggressive management of metabolic bone disease can augment and improve cardiovascular disease status. Pediatric nephrologists need to manage the metabolic bone disease while keeping the ongoing linear growth and skeletal maturation in mind, which may be delayed in chronic kidney disease.

Read the full study in Advances in Chronic Kidney Disease.

Bear Institute PACK logo

Bear Institute Pediatric Accelerator Challenge for Kids winners announced

Bear Institute PACK logoIn December 2022, the Bear Institute, along with Children’s National Hospital and Oracle Health, hosted the second annual Bear Institute PACK (Pediatric Accelerator Challenge for Kids), a start-up competition aimed to foster pediatric digital health innovation.

Bear Institute PACK is inclusive of the entire pediatric health care community and addresses the large disparity in digital health innovation funding dedicated to children versus the rest of the population. “We have to do more for children, a population that can’t advocate for itself,” says Matt Macvey, M.B.A., MS, executive vice president and chief information officer at Children’s National Hospital. “Bear Institute PACK is an all-hands effort to provide increased support to those start-ups trying to bring new solutions to market for kids.”

Start-ups share their innovations and receive valuable feedback from expert judges while competing for a chance to win an on-site pilot and software development support. The competition features three rounds of judging: an initial review of applications from the Bear Institute PACK team, judging from participating pediatric healthcare providers and administrators and review from an expert panel of judges during finalist start-ups’ live pitches. This year’s start-up participants competed across four innovation tracks in the following areas of development: Early-Stage Innovation, Concept Validation, Early Commercialization and Growth Trajectory.

This 2022 winners, in four innovation tracks, are:

  • Early-Stage Innovation (“Even the biggest ideas start small”) Winner: PigPug Health
    Its solution uses neurofeedback, a non-invasive approach to treating brain-related conditions, and artificial intelligence to help children with ADHD and autism become more socialized.
  • Concept Validation (“Now it’s time to test it”) Winner: Global Continence, Inc.
    Its Soluu™, Bedwetting Mitigation Device, helps rapidly and permanently mitigate bedwetting with a neuromodulation process.
  • Early Commercialization (“Countdown to launch”) Winner: PyrAmes Inc.
    Its solution Boppli™ provides continuous, non-invasive blood pressure monitoring and streams data via Bluetooth to a mobile device.
  • Growth Trajectory (“The investment is growing”) Winner: maro
    Its full stack child development kit equips a child’s caretakers (at home, school and clinic) with easy access to tools and data needed to help them navigate tough conversations including mental health, diversity, empathy, and puberty and helps identify mental health at-risk students in schools.

“I was very impressed with this year’s start-up participants and their caliber of talent and passion for what they do. The finalist judges were tasked with selecting one winner in each innovation track, but the work each participant is doing for kids makes them all winners,” says Rebecca Laborde, Ph.D., chief scientist, vice president of Health Innovation and Scientific Advisory, Oracle Health. “Thank you to the entire pediatric healthcare community that comes together to help make this event a success. We believe that by bringing together like-minded individuals with the same goals, we can make a real difference in pediatric healthcare.”

More information on this year’s winners can be found on the Bear Institute PACK website.

President Joe Biden and First Lady Jill Biden tour the telehealth command center at Children's National Hospital

President Biden, First Lady tour cardiac telehealth command center

President Joe Biden and First Lady Jill Biden paid a recent visit to the Cardiac Intensive Care Unit (CICU) at Children’s National Hospital, where leaders of our cardiology services toured them through the state-of-the-art telehealth command center embedded on the unit.

The big picture

Children’s National is pioneering the integration of telemedicine into CICU care. It’s one of the few pediatric hospitals in the world to do this.

Experts liken the telehealth command center to an ‘air traffic control tower’ for the most vulnerable patients with critical heart disease. The President and First Lady saw how complex the environment is, with real-time monitoring of all 26 high-risk patients in the CICU. The system combines traditional remote monitoring, video surveillance and artificial intelligence tools.

What this means

“With this technology, we’re helping to predict and prevent major adverse events,” said Ricardo Munoz, M.D., executive director of the Telemedicine Program and chief of the Division of Critical Care Medicine at Children’s National. “For example, our neuromonitoring system can help signal an impending brain injury before it happens.”

Dr. Munoz says President Biden expressed interest in the prevention strategy of adverse events and this new approach to caring for children with critical cardiac illness.

What they’re saying

  • “It was important to share with the Biden’s that caring for these kids is a long-term endeavor, not simply a single surgery or procedure to fix their heart abnormality,” said Yves d’Udekem, M.D., Ph.D., chief of Cardiac Surgery at Children’s National. “That means making sure they have the earliest diagnoses, the best treatments from surgeons and others who truly understand their condition, and a technologically advanced, attentive place to recover and heal as safely as possible.”
  • “Many people don’t believe that ‘pediatrics’ and ‘innovation’ can co-exist,” said Annette Ansong, M.D., medical director of Outpatient Cardiology at Children’s National. “During the Biden’s visit, they were at the crux of a novel way to closely monitor some of our sickest children with the added ability to predict bad events before they happen.”

Dr. Ansong hopes bringing awareness of these cardiac capabilities to the President and First Lady is the first of many steps in seeking support for children with congenital and acquired heart disease.

pregnant woman on couch

The role of pediatric cardiologists in addressing maternal health disparities

pregnant woman on couch

Black women are two to three times more likely to die from pregnancy-related complications. Most of the risk factors for these complications are cardiac in origin and preventable.

Pediatric cardiologists can and should work alongside other specialties to address the epidemic of maternal mortality that disproportionately affects Black women in the United States, says Annette Ansong, M.D., medical director of outpatient cardiology at Children’s National Hospital.

As co-chair of the Women and Children Committee of the Association of Black Cardiologists Inc., (ABC) Ansong says that cardiologists, especially pediatric cardiologists, have a role to play because “before they are Black women, they are Black girls.”

She talked about the impact of these health disparities and how cardiologists can play a role in addressing them at the American Heart Association Scientific Sessions in November 2022.

Why it matters

Dr. Ansong says that Black women are two to three times more likely to die from pregnancy-related complications. Most of the risk factors for these complications are cardiac in origin and preventable. Furthermore, many of these cardiovascular risk factors, such as obesity, start in youth and some even before birth. For example, children of pre-eclamptic moms have a higher risk of cardiovascular disease in the future, too.

How cardiologists can help

Pediatric cardiologists can be more proactive at helping Black girls grow up into strong, healthy Black women by making sure they are heart-healthy from a young age. That includes advocating for exercise, eating well and exploring innovative ways to encourage those habits.

Dr. Ansong says she makes a point to closely follow children based on the heart health history of their mothers—for example, “if mom had pre-eclampsia, I need to be keeping a closer eye on that child’s blood pressure” —to allow for early intervention and potentially prevent some devastating negative outcomes later in life.

Pediatricians and other specialists can also work with maternal-fetal medicine and other specialties to advocate for better tools to monitor women with pre-existing heart-related risk factors. This might include supporting efforts to enhance technology that makes self-monitoring easier, so women can keep closer eyes on their own blood pressure and share it with doctors in between appointments.

Most important, clinicians of all stripes should try to connect with patients to understand who they are, where they come from and how their stories impact their risk factors for health conditions.

The Women and Children’s Committee of the ABC launched the “We Are the Faces of Black Maternal Health” campaign in February 2022. The first-of-its-kind effort featured the stories of ABC members who had direct or indirect experiences with the impacts of maternal health on themselves, their children or someone they knew.

What’s next

The ABC “We Are the Faces of Black Maternal Health” re-launches this February to continue raising awareness but will also emphasize the need for investments in research about the causes of these disparities and possible prevention strategies to protect Black women.

Abstract Happy 2022 New Year greeting card with light bulb

The best of 2022 from Innovation District

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

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

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

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

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

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

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

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

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

5. New telehealth command center redefines hospital care

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

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

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

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

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

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

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

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

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

10. 2022: Pediatric colorectal and pelvic reconstructive surgery today

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

animation showing MRI cardiac imaging

Advanced MRI hopes to improve outcomes for Fontan cardiac patients

animation showing MRI cardiac imaging

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.

RFP collage of logos

Healthcare leaders join to advance pediatric innovation

RFP collage of logosChildren’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.

Map showing CHI Registry sign-ups through December 2022

Congenital Heart Initiative celebrates global reach in first two years

The first patient-powered registry for adults living with congenital heart disease (CHD) — the Congential Heart Initiative (CHI) — celebrates a major milestone this month. In the two years since it was launched in December 2020, the registry has enrolled more than 3,227 adults with CHD from all 50 states and 28 countries.

Why it matters

Although nearly 2 million adults in the United States are living with a congenital heart defect — more adults than children in fact — it’s been historically difficult to gather data on these conditions and to identify patient needs.

“We developed this registry together with numerous patients and providers so it could become a platform for increasing our knowledge and improving care,” says Anitha John, M.D., medical director of the Washington Adult Congenital Heart Program and an adult congenital cardiologist at Children’s National Hospital, who helped drive the registry’s creation. “We want it to become a resource for patients and researchers, a place where they can learn more about what it means for adults who had their hearts repaired in childhood to live a long, healthy life.”

The patient benefit

As the treatments for children with CHDs have improved, people born with them are living longer and longer. That means that as they become adults, they have a lot of worry and uncertainty about their limitations and abilities to achieve what might be considered common adult milestones.

“Over the past two years, more than 3,000 adult congenital heart disease (ACHD) patients from around the world, including myself, have worked to support ACHD research by participating in the Congenital Heart Initiative (CHI),” says Scott Leezer, a single-ventricle heart defect patient and co-leader of research and advocacy for the Initiative. “This selfless and simple effort has produced an investment in improving our collective understanding of adult patients living with CHD. We hope these investments will pay dividends in helping guide new research strategies for future generations of CHD patients.”

Participants in the registry receive regular newsletters, highlighting findings and providing access to patient specific resources including content from the Adult Congenital Heart Association (ACHA), a key partner. The ACHA collaboration has also given registry participants the opportunity to interact with other people – a key request identified as important based on input from people who have already registered.

What’s unique

“There is no ’one-size-fits-all’ answer for adults with CHD,” Dr. John adds.

“While other CHD registries exist, they are provider-based and not patient-powered,” said John. “The CHI registry is driven by patients, supporting research but also providing information based on what is important according to the people with CHD themselves.”

What’s next

A sub-study of the CHI will use PCORnet®, the National Patient-Centered Clinical Research Network, to better understand how gaps in care impact the adult patient experience with CHD. The CHI-RON study, which stands for Congenital Heart Initiative: Redefining Outcomes and Navigation to Adult-Centered Care, will be the first of its kind to fill in these knowledge gaps by exploring three distinct types of data at once: patient reported outcomes, health insurance claims and electronic health records (EHR). The effort is led by Children’s National and Louisiana Public Health Institute.

“PCORnet is a fantastic resource to help us fully understand the impact of gaps in CHD care,” said Tom Carton, dual-principal investigator of CHI-RON and chief data officer at the Louisiana Public Health Institute. “It is essentially turbo-charging our patient-reported data with two additional layers of insights from claims and EHRs, unlocking answers that would be impossible to achieve in isolation.”

In terms of research, the CHI now has enough participants to allow researchers to complete some important studies about pregnancy, mental health and long-term health care follow ups, all of which will kick off within the next year. The research teams hope to engage centers across the United States to better determine the needs of individual ACHD centers and to understand how the CHI can help with these needs. Centers that care for people with ACHD can learn more about how to participate in the CHI at information sessions in 2023, including January 12 at 8 am or 6 pm and January 17 at 8 am or 6pm.

PeriPath surgery

NIH awards $1.8 million to trial pacemaker delivery system for children

PeriPath pacemaker

The PeriPath access port makes it possible for pacing and defibrillating leads to be placed in the smallest children through holes the size of a straw.

A $1.8 million Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH) is funding the first clinical trial of a novel device called PeriPath. The device makes it possible for pacing and defibrillating leads (or wires) to be placed in the smallest children through holes the size of a straw, eliminating thoracotomy or sternotomy procedures for children who are too small for transvenous implantation.

Even the tiniest pacemakers and defibrillators on the market today aren’t small enough for infants and young children with heart rhythm abnormalities. 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.

The research goal

Charles Berul, M.D., division chief of Cardiology and co-director of the Children’s National Heart Institute, partnered with engineers in the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Hospital to develop and test a first-of-its-kind minimally invasive pericardial access tool. The tool allows doctors to place pacing and defibrillation leads to the epicardial surface of the heart under direct visualization from an endoscope.

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.

Why it matters: Less pain, shorter and fewer surgeries

If successful, the device will eliminate the need for open chest surgery in patients who aren’t candidates for transvenous placement. The ability to place these leads percutaneously should:

  • Reduce pain and infection risk.
  • Decrease procedure times.
  • Minimize surgery complications that arise from open surgery.
  • Improve better visualization for pericardial punctures.
  • Allow other novel therapies such as epicardial ablation or, in the future, even drug/gene delivery into the pericardial space.

Any implanted pacemaker or defibrillator must be replaced every 5-10 years. A young child in critical need of such devices could face surgeries 10 or more times to replace the device and/or leads.

Pre-clinical testing shows early data that this percutaneous approach is as safe and effective as an open surgical technique, although it remains in early-stage evaluation.

What’s next

The NIH SBIR funding will allow the research team to assess long-term safety and efficacy and commercialize the PeriPath tool. Next steps are to:

  • Refine the design of PeriPath for production manufacturing, integrate testing protocols into a Quality Management System and conduct a pilot verification build. Success is defined as manufacturing production devices that pass 510(k) verification and validation testing.
  • Demonstrate substantial equivalence to predicate trocars through performance and handling validation testing using PeriPath to implant an epicardial lead in a pediatric simulator. If successful, the team will demonstrate equivalence and obtain investigational device exception (IDE).
  • In the latter part of the plan, to perform a first in human feasibility clinical study using PeriPath to implant a commercial pacemaker lead with institutional review board (IRB) approval in infants at Children’s National.

Bottom line

Dr. Berul says, “This research could have a transformative impact on current clinical practice by converting an open surgical approach to a minimally invasive percutaneous procedure.”

He also notes that while the study design focuses on the unique needs of infants and children with congenital heart disease – who are the primary focus of the device – the results of the trial may benefit thousands of adult patients who need pacemakers or defibrillators but who are not candidates for the transvenous placement.

NCC PDI 2022 pitch competition winners

Five winners selected in prestigious pediatric device competition

The National Capital Consortium for Pediatric Device Innovation (NCC-PDI) announced five awardees chosen in its prestigious “Make Your Medical Device Pitch for Kids!” competition. Each received a share of $150,000 in grant funding from the U.S. Food and Drug Administration (FDA), with awards ranging from $20,000 to $50,000 to support the advancement of pediatric medical devices.

Consistent with its mission of addressing the most pressing pediatric device needs, this year’s competition, moderated by MedTech Innovator, welcomed medical device technologies that address the broad unmet needs of children. The pediatric pitch event was part of the 10th Annual Symposium on Pediatric Device Innovation, co-located with the MedTech Conference, powered by AdvaMed.

This year’s pediatric device innovation awardees are:

  • 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.

“We are delighted to recognize these five innovations with critical NCC-PDI funding that will support their journey to commercialization. Improving pediatric healthcare is not possible without forward-thinking companies that seek to address the most dire unmet needs in children’s health,” 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. “We know all too well how challenging it is to bring pediatric medical devices to market, which is why we have created this rich ecosystem to identify promising medical device technologies and incentivize investment. We congratulate this year’s winning innovators and applaud their efforts to help bridge these important care gaps that are impacting children.”

Empowering Innovators

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 and design firm Archimedic.

A pediatric accelerator program, powered by MedTech Innovator, the largest medical device accelerator in the world, is a key part of the 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.

To date, NCC-PDI has mentored 250 medical device projects to help advance their pediatric innovations throughout all stages of the total product life cycle (TPLC).

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 2022 pitch competition winners

A total of $150K was awarded to five pediatric innovations during the medical device pitch competition at the 10th Annual Symposium on Pediatric Device Innovation, hosted by the National Capital Consortium for Pediatric Device Innovation (NCC-PDI). Award winners include (from left to right): Zaid Atto, founder and CEO at Xpan; John Konsin, CEO and co-founder of Prapela; Elaine Horn-Ranney, co-founder and CEO at Tympanogen; William Altman, CEO at CorInnova; and Sharief Taraman, pediatric neurologist at CHOC and University of California-Irvine partnering with Innovation Lab. (Photo credit: Children’s National Hospital)

echocardiogram

AI may revolutionize rheumatic heart disease early diagnosis

echocardiogram

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

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

Why it matters

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

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

The hold-up in the field

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

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

Children’s National leads the way

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

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

What’s next

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

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

Paper cutout of head with brainwaves

Lifesaving ICDs can cause anxiety, stress, PTSD for parents and kids

Paper cutout of head with brainwaves

Research shows that children with implantable cardioverter-defibrillators, and their parents, are at risk for anxiety, post-traumatic stress disorder and other psychological distress.

Recent advances in design and efficiency of implantable cardioverter-defibrillators (ICDs) have led to their increased use in younger patients, protecting more children with congenital heart disease from sudden cardiac arrest and sudden cardiac death, says a commentary in the journal Heart Rhythm. However, living longer with these devices and the day-to-day worry that they may have to deliver a lifesaving shock in the blink of an eye, may cause unusually high rates of anxiety, stress and other psychosocial distress for children with ICDs and their families.

Commentary authors Vicki Freedenberg, Ph.D., RN, electrophysiology nurse scientist, and Charles Berul, M.D., chief of cardiology, both from Children’s National Hospital, note that current available research shows both children with these ICDs and their parents are at risk for anxiety, post-traumatic stress disorder (PTSD) and other psychological distress. They highlight a new study published in the same journal that reports data related to prevalence and factors associated with PTSD in children with ICDs and their parents as a good start to better understanding these impacts.

Why it matters

Freedenberg and Berul say that the new study adds important information to an area without a lot of previous research. They also point out that understanding the long-term impacts of life with these devices is critical to ensuring the overall long-term health and wellbeing of both the children with these devices and their families.

What’s been the hold-up in the field?

The development of devices that work for younger children with congenital heart disease, including advances in ICDs and pacemakers, has increased in the last decade. In this time, studies of how these devices work for children have focused predominantly on clinical outcomes and questions related to clinical care.

As survival rates for children have increased, research needs to shift from the study of mortality and clinical outcomes toward understanding the full spectrum of how these devices impact daily life for these children and their families.

Moving the field forward

According to Freedenberg and Berul, the new study importantly includes both patient and parent perspectives, which is a first in this research area. They also offer recommendations for future studies, including the use of comparison groups to allow for generalization of findings. Researchers might also ask research questions to determine whether the device itself or the medical and non-medical factors that often occur simultaneously are more important to predicting mental health and wellbeing.

However, the commentary concludes with the most important takeaway: More research, with specific parameters focused on the impact of clinical interventions, is desperately needed to truly understand all the ways that children and their families are affected throughout life by the clinical care and support they receive.

Read the full commentary, Potential for shock leads to potential for stress, in the journal Heart Rhythm.

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 surgerygastroenterology 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.