Surgical Innovation

illustration of a brain

Inducing strokes in newborns to treat hemimegalencephaly

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

The big picture

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

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

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

What they’re saying

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

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

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.

Illustration of brain and brainwaves

Effective treatment for children with hemimegalencephaly

Illustration of brain and brainwaves

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

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

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

Why it matters

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

Why we’re excited

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

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

What’s been the hold-up in the field?

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

How does this work move the field forward?

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

How is Children’s National leading in this space?

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

girl playing basketball

Sphincter reconstruction offers new hope for children with Hirschsprung disease

girl playing basketball

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

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

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

Why it matters

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

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

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

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

Why we’re excited

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

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

The patient benefit

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

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).

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.

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

Everyone Poops Book Cover

2022: Pediatric colorectal and pelvic reconstructive surgery today

Everyone Poops book coverAdapted from Levitt MA. New and exciting advances in pediatric colorectal and pelvic reconstructive surgery – 2021 update. Semin Pediatr Surg. 2020 Dec;29(6):150992.

As demonstrated in the popular children’s book by Taro Gomi, Everyone Poops, the physiology of stooling is a focus of early childhood development and a subject of concern for both parents and children. References to pediatric colorectal problems go back many thousands of years. In fact, the Babylonian Talmud, written in the year 200 CE, recommends that “an infant whose anus is not visible should be rubbed with oil and stood in the sun… and where it shows transparent the area should be torn crosswise with a barley grain.” Surgical techniques to manage such patients have certainly evolved since that time, but the basic principles of care remain the same.

How we got here

The modern story of the care of patients with anorectal malformations (ARMs) began in the 1940s in Melbourne, Australia, when Henry Douglas Stephens worked to define the anatomy of children with ARMs by analyzing the anatomy of twelve deceased patients with these conditions. He continued to focus on this specialty for the rest of his career and published two books on the topic in 1963 and 1971. Prior to his groundbreaking work, the anatomy of such patients was only a concept that existed in surgeons’ minds – without anatomic precision – since no one had actually seen the anatomy. These concepts were depicted in the bible of pediatric surgery in North America, the Gross textbook, which in retrospect was both oversimplified and inaccurate.

During his autopsy dissections, Stephens came to the key anatomic conclusion that the puborectalis muscle (the sphincters) lay behind the urethra. He devised an operation based on this concept: first, the urethra was identified, then a space between it and the puborectalis was dissected, and then the rectosigmoid was pulled through that space. A small incision in the perineum for the pulled-through bowel was made, within which the new anus was created. The perineal dissection was a blind maneuver. During the same time period, William Kiesewetter in Pittsburgh proposed his version of the sacral abdominoperineal pull-through using similar anatomic principles.

Justin Kelly was one of Stephens’ trainees in Australia who learned how to do this operation. At Boston Children’s Hospital in the late 1960’s, he taught what he had learned from Stephens to the surgeons there, including another trainee, Alberto Pena. Pena and his fellow surgical residents benefitted from exposure to Kelly as well as master surgeon Hardy Hendren, who operated on patients across town at Massachusetts General Hospital. Hendren, the pioneer in the care of children with cloacal anomalies, passed away this year at the age of 96.

Pena completed his training in Boston and went to Mexico City in 1972 at the age of 34 to become the head of surgery at the National Pediatric Institute. He tells the story that when he asked his new pediatric surgery faculty to choose an area of specialization, no one chose colorectal, so he decided to take on that group of patients and thus embarked on his revolutionary colorectal career. Pena at first applied the technique he had learned from Kelly to repair anorectal malformations, but he became increasingly frustrated by the procedure. He felt that the maneuvers offered very poor exposure to the anatomy, and over time his incision grew longer and longer. In 1980, Pena’s collaboration with Pieter de Vries – who had come to Mexico City to work on these cases with Pena – culminated in the first posterior sagittal anorectoplasty [Figure 1]. That same year, Pena presented his findings at a meeting of the Pacific Association of Pediatric Surgeons.

imperforate anus repair

Figure 1: Original diagrams of the posterior sagittal approach to repair imperforate anus

This posterior sagittal approach opened a beneficial Pandora’s Box in surgery. It allowed for a true understanding of the pelvic anatomy and led to the care of many conditions that were previously – to use Pena’s words – “too difficult to reach from above (via laparotomy) and too difficult to reach from below (perineally).” This new approach influenced the repair of cloacal malformations, urogenital sinus, pelvic tumors, urethral problems, reoperations for imperforate anus and Hirschsprung disease (HD), a transpubic approach for complex genitourinary problems, and a comprehensive strategy for the management of cloacal exstrophy. In addition to his surgical contributions, Pena also conceived of the intervention that has arguably improved patients’ quality of life the most: a focused approach to the bowel management of fecal incontinence. Thanks to such bowel management programs, now available at many centers across the world, thousands of children are no longer wearing diapers and have said goodbye to their stomas. The impact of bowel management is perhaps comparable to that of intermittent catheterization for patients with urinary incontinence.

My personal journey in this field began in 1992, when I was an eager medical student and signed up for an elective in pediatric surgery with Alberto Pena. This experience changed my career trajectory in a very dramatic and positive way. Medicine was becoming increasingly complex, and fields such as neonatal care, transplantation, and cardiology were benefitting from a collaborative approach. It became clear that the colorectal field needed the same approach. As a comparative example, consider the project of building a bridge. How does such a project start? The cement layers do not show up one day and lay cement prior to the steel team deciding where to place the beams. The project must begin with all parties meeting together to develop a comprehensive plan. Amazingly, however, that type of collaborative planning does not often happen in the care of medically complex patients. It most assuredly needs to.

The value of multi-disciplinary care

Medical complexity requires integrated and collaborative care because all the anatomic structures that need to be managed are located right next to each other and because each has a unique and complex physiology that can affect the other systems. To achieve success, patients with anorectal malformations, Hirschsprung disease, fecal incontinence (related to a variety of conditions), and colonic motility disorders require care from a variety of specialists throughout their lives. These include providers in the fields of colorectal surgery, urology, gynecology, gastroenterology, motility, orthopedics, neurosurgery, anesthesia, pathology, radiology, psychology, social work, nutrition, and many others. Perhaps most important to the achievement of a good functional result, however, is a patient’s connection to superb nursing care. A complex colorectal operation takes about four hours to perform, but to get a good result, it takes an additional 96 hours of work, the vast majority of which involves nursing care. The value of good nursing partners to ensure successful surgery cannot be overemphasized. They have unique skills in identifying and solving problems, a willingness to get down in the weeds, and are always striving to fill the gaps in care.

What parents want and need

Having met many parents with newborns diagnosed with colorectal problems, I have made several observations. First, it seems that no parent has ever imagined that their child could have a problem with stooling – this is a physiologic ability that is taken for granted. When they are told about the problem with their baby, they are uniformly shocked that something like this could happen. Second, when discussing that their child will need surgery to correct their colorectal anatomy, parents don’t focus on the surgical technique and elegance of the reconstruction, as surgeons tend to do. Instead, parents dwell on whether the surgery will create a working reconstructed anatomy that will allow their child to stool without difficulty or embarrassing accidents. As surgeons we need to remember this. We always need to understand what it is that the family and patient wishes us to deliver, and we need to strive to achieve those goals. As proud of our surgical skills as we are, it is the functional outcome that matters most.

Where we are in 2022

In 2022, the advances in the field of pediatric colorectal and pelvic reconstruction are significant. They include new techniques and ideas that over time have made a dramatic and positive impact on the care and quality of life of children who suffer from colorectal problems. Here are a few such advances:

  1. Prenatal diagnosis of anorectal and cloacal malformations has been progressively improving. Perinatologists have learned to look for specific findings, such as a pelvic mass in a female with a single kidney, and consider that it could be a cloaca. Assessment of perineal anatomy, pubic bone integrity, sacral development, abnormalities of the radius bone, as well as cardiac, spinal, and renal anomalies may lead the clinician to consider that a fetus may have an anorectal malformation.
  2. Management of the newborn, particularly in the fields of newborn radiology and neonatal care, has dramatically improved as neonatal techniques have advanced. Specific to the colorectal patient have been advances in radiology such as assessments of hydronephrosis, 3D reconstruction of cloacal anomalies, and ultrasound-guided distal colostography. Further advances include improved techniques in the management of hydrocolpos and stoma care, to name a few.
  3. The treatment of associated urologic anomalies has diminished chronic renal disease, and proactive bladder management is reducing the need for bladder augmentations and renal transplantation.
  4. Understanding the gynecologic collaboration has helped clinicians define the Mullerian anatomy and better plan for menstruation and future obstetric potential.
  5. Prediction of continence, even in the newborn period, requires an understanding of the associated problems with the sacrum and spine. This knowledge has allowed clinicians to have more robust conversations with families about their child’s future.
  6. The decision of whether to do a newborn repair versus a colostomy must be guided by the surgeon’s experience and the clinical circumstances in which they find themselves.
  7. The defining of anatomy allows patients to be compared across medical centers, and for treatment options and outcomes to be uniformly analyzed. Keeping track of one’s outcomes and always striving to improve should be basic tenets of surgical practice.
  8. Recognizing the value of laparoscopy and knowing for which cases this approach should be applied. Morbidities associated with a laparoscopic approach for a rectum in an ARM patient well below the peritoneal reflection have been noted. In HD cases, laparoscopy can limit the stretching of the sphincters which occurs during the transanal rectal dissection.
  9. Development of a treatment algorithm for the management of cloacal malformations which considers the importance of their common channel and urethral lengths.
  10. Recognizing key complications after ARM and Hirschsprung surgery, knowing when and how to do a reoperation, determining the outcomes of such reoperations, and ultimately figuring out how to avoid complications altogether.
  11. Understanding the causes of fecal incontinence, the amount of incontinence that can be anticipated, and the surgical contributors to achieving continence.
  12. Development of bowel management programs in multiple centers and committing to following these patients in the long term.
  13. Learning the pathophysiology of motility disorders and developing treatment protocols, as a result of the vital collaboration between surgery and GI/motility. Medical treatments with laxatives, rectal enemas, botox injection of the anal canal, and surgical adjuncts such as antegrade colonic flush options and sometimes colon resections are key aspects of the treatment armamentarium.
  14. Developing a collaboration between colorectal surgery and urology allows the clinical team to know when the colon can be used for a bladder augment (which not only offers an augment option but also can improve antegrade flushes of the colon) or if the appendix can be shared between Malone and Mitrofanoff. The collaboration with urology to plan the management of both urinary and fecal continence during the same operation is a very valuable trend. This proactive planning has improved the lives of many patients and has reduced the numbers of operations they need as well as their hospital stays.
  15. Sacral Nerve Stimulation (SNS) has shown promise in the management of urinary incontinence and seems to have a role in improving fecal continence and promoting motility, as an adjunct to treatments for constipation.
  16. Development of dedicated colorectal centers that are integrated and collaborative allows a team to tabulate their results and strive for better outcomes. The value of a collaborative model for the care of such patients cannot be overstated, not only for patient convenience, but also for creating an integrated plan for their care. These exist now in many parts of the country and care is available in nearly every region, reducing a family’s need to travel great distances away from their home to access care.
  17. Establishing a transition to adult programs, as with congenital heart disease and cystic fibrosis. Colorectal surgeons are obligated to develop a transition plan for their patients as they enter adulthood.
  18. Using Basic Science to advance the field, including tissue engineering and genetics, will be revolutionary. We should be able to imagine the day when cloacal reconstruction could be based on a previously tissue-engineered segment of vagina, produced by the patient’s own stem cells. In addition, the genetics of anorectal malformations as well as Hirschsprung disease are being vigorously pursued which will impact parental counseling and potential therapies.
  19. Real time data used to follow outcomes is needed to keep track of complications as well. That information can be used to adjust protocols which will improve results.
  20. Development of international consortiums will help patients in a way that is not achievable by a single institution. Consortiums allow ideas to be spread rapidly which will dramatically affect how many patients can be helped and how quickly. The Pediatric Colorectal and Pelvic Learning Consortium, pcplc.org, is well on its way to achieving these goals.
  21. Bringing complex care to all corners of the world because there is a great deficiency in advanced colorectal care in the developing world. The care of colorectal patients in a resource-limited setting has unique challenges, but creative solutions by innovative surgeons in those areas have a led to dramatic improvements in care.
  22. Parent/patient organizations provide education, advocacy, and support for families at all stages of their child’s care. With internet access readily available, colorectal patients and their families can now access the welcoming environment of these organizations, and no longer feel as lost and alone as in previous years.

Why it matters

Given all these wonderful advances, we must continue to reaffirm the key principles stated by Sir Dennis Browne that “the aim of pediatric surgery is to set a standard, not to seek a monopoly.” With an integrated approach to the care of this complex group of patients, great things can be achieved. I am hopeful that the caregivers and parent/patient group organizations who commit to the care of children with colorectal problems and understand the daily struggle of improving a patient’s quality of life will learn the skills and tricks necessary to achieve good results. If they do, they will help many children.

Finally, I will share a humorous piece written by my daughter, Jess Levitt, regarding the value of bringing order to chaos. Its message is particularly relevant to the care of children with colorectal problems in 2022, as we build on our efforts to improve, streamline, and transform the formerly chaotic process through collaboration and education. As we endeavor to advance this field, we need to remember what Dr. Pena often said: “It is not the unanswered questions, but rather the unquestioned answers that one must pursue.”

“A” must come before “B,” which must come before “C,” everybody knows that. But what if the Millercamps of this world did not have to sit next to the Millerchips when it comes to seating arrangements? Can Pat Zawatsky be called before Jack Aaronson when the teacher is taking attendance? Do those 26 letters that make up all the dialogue, signs, thoughts, books, and titles in the English-speaking department of the world need their specific spots in line? Everyone can sing the well-known jingle from A to Z, but not many people can tell you why the alphabet is the way it is.

For almost as long as humans have had the English language, they have had the alphabet. The good ol’ ABCs. However, the alphabet represents the human need for order and stability. I believe that the same thinking that went into the construct of time and even government went into the alphabet. Justifiably, lack of order leads to chaos. Knife-throwing, gun-shooting chaos, in the case of lack of governmental order. Listen to me when I tell you that there is absolutely no reason that the alphabet is arranged the way that it is. Moreover, the alphabet is simply a product of human nature and how it leads people to establish order for things that do not require it.

Now I know this sounds crazy, but bear with me. Only if you really peel away the layers of the alphabet will you find the true weight it carries. People organized the letters of our speech into a specific order simply because there wasn’t already one. Questioning this order will enlighten you on the true meaning of it. Really dig deep into the meaning behind the social construct that is the alphabet. Short and sweet as it may be, the order of the ABCs is much less than meets the eye. There is no reason that “J” should fall before “K”! Understand this. Very important as order is, it is only a result of human nature.

What’s next? X-rays become independent of Xylophones in children’s books of ABCs?

You know what the best part is? Zero chance you even noticed that each sentence in this essay is in alphabetical order.

– Jess Levitt

 

2022 Hackathon logo

Addressing health equity issues through an app innovation competition

Children’s National Hospital launches The Health Equity in Research Hackathon — a team-based collaborative competition that empowers researchers to address health equity issues in the community through innovative apps. A panel of expert judges will select winning app ideas for full development, including finding grant opportunities, access to mentors and collaborators.

The big picture

“This Hackathon is a great chance for our research community to address larger issues related to advancing health equity within translational and clinical research,” said Patrick O’Keefe, administrative director for the Clinical and Translational Science Institute at Children’s National (CTSI-CN). “We are thrilled to see how people collaborate to create solutions for big problems that have traditionally slowed research and contributed to the vast inequities in health we see today.”

Additional details

The 2022 hackathon will be a two-part event. During Part I, slated for June 17, participants will gather in diverse teams to discuss and refine the selected app ideas. They will learn from technical and scientific experts and brainstorm app-based approaches to address health equity.

Each team will pitch their ideas to a panel of judges, and the winning app(s) will advance to Part II of the hackathon planned for Fall 2022, where app developers will build the selected apps.

“We encourage anyone at Children’s or George Washington University to submit an app idea – even if it is not fully formed — as long as they think it would help reduce health disparities through improving the research process,” said Lisa Guay-Woodford, M.D., director for Clinical and Translational Science Institute and Center for Translational Research at Children’s National. “We also hope researchers, staff and students who don’t have app ideas at this time will consider attending anyway to participate in the lively development process of the Hackathon.”

Anyone within the Children’s National and George Washington University research communities can submit an app idea for consideration. No app development experience is necessary to enter.

Why it matters

Health equity also means bringing the community into the research process. Thus, in part II, Children’s National will partner with high schools and universities in the area to incorporate voices who are often under-represented in the science and technology field.

“Our community is home to brilliant young minds at our local high schools and universities,” said Chaya Merrill, Dr.P.H., director for Child Health Data Lab at Children’s National. “We are excited to create an opportunity for them to work alongside experienced app builders – at the intersection of health equity and technology – by engaging in Part II of the Hackathon to build the winning apps!”

The hackathon will take place at Children’s Research and Innovation Campus in partnership with CTSI-CN and the Center for Translational Research.

The app idea submission deadline is on May 20, 2022, by 5pm. You may apply here. If you have questions about completing this submission, please email Patrick O’Keefe at pokeefe@childrensnational.org.

2022 Hackathon logo

parathyroid close-up

A new imaging device with AI may reduce complications during thyroid surgery

parathyroid close-upSurgeons perform approximately 150,000 thyroidectomies in the United States. Post-surgical complications from this procedure frequently occur due to the misidentification or accidental removal of healthy parathyroid glands. On average, 27% of these patients suffer from transient or permanent hypocalcemia, a condition in which the blood has too little calcium, leading to lifelong complications and socioeconomic burden.

To improve parathyroid detection during surgery, Children’s National Hospital experts developed a prototype equipped with a dual-sensor imaging device and a deep learning algorithm that accurately detects parathyroids, according to a new study published in the Journal of Biophotonics.

“What excited us in this study was that even deep-seated tissues were able to be imaged without light loss, and high resolution imaging was possible due to the unique optical design,” said Richard Jaepyeong Cha, Ph.D., council member of the International Society of Innovative Technologies for Endocrine Surgery and principal investigator for the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Hospital. “Moreover, in several cases, parathyroid autofluorescence was detected even before the surgeon dissected the parathyroid gland, and while it was covered by fat and/or fascia.”

What’s unique

This is the first study that uses color RGB/NIR paired imaging-based parathyroid detection by incorporating multi-modal (both RGB light and near-infrared autofluorescence, or NIRAF, ground truth imaging) data into parathyroid identification using a deep learning algorithm.

The patient benefit

“We envision that our technology will open a new door for the digital imaging paradigm of dye-free, temporally unlimited, and precise parathyroid detection and preservation,” said Richard. “Successful translation of this technology will potentially reduce the risk of hypoparathyroidism after common thyroid surgery and improve the clinical outcomes.”

The results support the effectiveness of their novel approach despite the small sample size, which can potentially improve specificity in the identification of parathyroid glands during parathyroid and thyroid surgeries.

The hold-up in the field

It is often difficult for surgeons with naked eyes to identify parathyroid glands from thyroid tissue because of the small size, the variable position, and similar appearance to the surrounding tissues.

Since 2011, surgeons have benefited from using NIRAF, a non-invasive optical method for intraoperative real-time localization of parathyroids.

While the NIRAF technology has gained traction among endocrine surgery community, false negatives can occur with current devices that use the NIRAF technology in secondary hyperparathyroidism cases. According to Kim et al., the technology is still suboptimal, and a significant percentage of parathyroid is being missed.

Children’s National Hospital leads the way

Engineers in Children’s National are leading this field through several innovations:

  • Non-dye injected, label-free use in real-time in comparison to temporally limited ICG angiography. This technology was featured as the cover article in the journal Lasers in Surgery and Medicine 54(3), 2022.).
  • Simultaneous perfusion assessment from four glands at any time during operation.
  • Arterial flow detection from pulsatile information in well-perfused PG vasculature.
  • Quantified parathyroid detection and classification with prediction values using deep learning technique.

You can read the full study “A co-axial excitation, dual-RGB/NIR paired imaging system toward computer-aided detection (CAD) of parathyroid glands in situ and ex vivo” in the Journal of Biophotonics.

overview of parathyroid surgery procedure

Drs. Kane and Petrosyan

POEM procedure is safe and effective for children with esophageal achalasia

Drs. Kane and Petrosyan

Drs. Petrosyan and Kane combined perform more POEM procedures for children than any other pediatric surgeons in the United States.

Peroral endoscopic myotomy (POEM) is a safe and effective procedure to treat pediatric achalasia according to a single-center outcomes study in the Journal of Pediatric Surgery.

Authors Timothy Kane, M.D., chief of General and Thoracic Surgery at Children’s National Hospital, and Mikael Petrosyan, M.D., MBA, associate chief of that division, together perform more of these procedures than any other pediatric surgeons in the United States.

Their experience with POEM shows that it is as safe and effective as the current standard of care for pediatric achalasia, which is a procedure called the laparoscopic Heller myotomy (LHM). Even better, previous research in adults and now in pediatric patients, has shown that those who undergo POEM as an alternative to LHM report less pain and often require shorter hospital stays after surgery.

Why it matters

POEM has been an option for adults with achalasia for many years, but not for children because it requires technical skill and expertise not readily available everywhere. More studies of young patients with successful outcomes following POEM procedures can help make the case for training more pediatric surgeons to learn this approach, and help this alternative method become an additional surgical option for children with achalasia.

The hold-up in the field

Achalasia is a rare condition in adults (1/100,000) and even less common in children, occurring in only 0.1 per 100,000 patients with an estimated prevalence of 10 per 100,000. The rarity of achalasia in children compared with adults makes collecting enough statistically significant evidence about how best to treat them difficult, more so than for other more common pediatric surgical conditions.

Children’s National Hospital leads the way

Children’s National Hospital is one of the only children’s hospitals in the country to offer the option of POEM for treatment of these conditions in children — and Drs. Kane and Petrosyan combined perform more of these procedures than any other pediatric surgeons in the United States.

The surgeons at Children’s National offer POEM as a primary intervention for children with esophageal achalasia and are also applying the same approach for pediatric gastroparesis as well.

Children’s National Hospital is one of only 12 children’s hospitals in the country, and the only hospital in the Washington, D.C., region, to be verified as a Level 1 Children’s Surgery Center by the American College of Surgeons (ACS) Children’s Surgery Verification Quality Improvement Program. This distinction recognizes surgery centers whose quality improvement programs have measurably improved pediatric surgical quality, prevented complications, reduced costs and saved lives.

Bottom line

Given their reported outcomes so far, the authors believe that in the long term this approach may replace the current pediatric standard of care, the LHM. More research is needed to make this case, however, including long-term follow-up studies of the patients who have undergone the procedure so far.

You can read the full study, “Per Oral Endoscopic Myotomy (POEM) for Pediatric Achalasia: Institutional Experience and Outcomes,” in the Journal of Pediatric Surgery.

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.”

Diego Preciado

Diego Preciado, M.D., Ph.D., named division chief of Otolaryngology

Diego Preciado

“It is the privilege of a lifetime for me to be able to continue my service to Children’s National in this leadership role,” said Dr. Preciado.

Children’s National Hospital has named Diego Preciado, M.D., Ph.D., as its new chief of the Division of Pediatric Otolaryngology. Dr. Preciado joined Children’s National in 2006 where he has practiced for the past 16 years.

“It is the privilege of a lifetime for me to be able to continue my service to Children’s National in this leadership role,” said Dr. Preciado. “Every otolaryngologist in the world knows of the extraordinary work and tradition of excellence established by my predecessor Dr. George Zalzal. I will work with the surgeon-in-chief to accelerate our mission by engaging our diverse and loyal faculty to become the global leader for complex pediatric otolaryngology care, research, education and advocacy.”

Dr. Preciado is a professor with tenure of Surgery and Pediatrics, Genomics and Precision Medicine at Children’s National and George Washington University. He holds the Joseph E. Robert Endowed Chair in Pediatric Otolaryngology, has served as the vice chief of the Division of Pediatric Otolaryngology for the past seven years, and the program director of the ACGME accredited Pediatric Otolaryngology fellowship since 2009. His clinical practice interests are primarily focused on pediatric airway reconstruction, childhood hearing loss/cochlear implants and velopharyngeal insufficiency.

Dr. Preciado’s translational research lab has been funded over the years through various awards including R01, U01 and R21 National Institutes of Health grants. He has authored over 130 peer-reviewed manuscripts, 20 book chapters, and has been an invited speaker regionally, nationally and internationally. In 2020, he received the Robert Ruben Scientific Achievement Award from the Society for Ear Nose and Throat Advances in Children.

He served as past president of the American Society of Pediatric Otolaryngology (2020-2021) and currently serves as president of the International Society for Otitis Media (2019-2023). He is a senior examiner for the American Board of Otolaryngology – Head and Neck Surgery where he also serves on the Complex Pediatric Otolaryngology Steering Committee.

“Given Dr. Preciado’s superb clinical, research and leadership background, we know the division is well-positioned for continued growth under his guidance,” said Dr. Anthony Sandler, M.D., senior vice president and surgeon-in-chief of the Joseph E. Robert Jr., Center for Surgical Care at Children’s National.

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.

scientist using the HoloUS platform

New platform uses augmented reality glasses for ultrasound-guided procedures

Children’s National experts developed a novel visualization platform, called “HoloUS,” for ultrasound-guided procedures using commercially available see-through augmented reality (AR) glasses, according to a pre-clinical study published in the International Journal of Computer Assisted Radiology and Surgery. The finding suggests that the HoloUS application enhanced the user experience and simplified hand-eye coordination.

“By eliminating head-turning and back-and-forth viewing of the patient and the ultrasound screen, our solution could improve the safety, accuracy, intuitiveness, and length of ultrasound-guided procedures, while making them easier to learn and perform,” said Raj Shekhar, M.S., Ph.D., principal investigator for Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National and senior author in the study.

Ultrasound-guided procedures demand a high degree of hand-eye coordination to advance a needle with one hand while holding the ultrasound probe with another as they visualize the live ultrasound image. With this application, the practitioner will wear the AR glasses that will allow them to visualize the ultrasound image rendered live in their field of view.

“We are excited about the possibility of improving the accuracy and efficiency from augmented reality visualization because these benefits may translate clinically in the upcoming years after conducting carefully designed clinical studies,” said Trong Nguyen, staff scientist for Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National and lead author.

Microsoft’s HoloLense headset, often used for hands-free work and video gaming, set the stage to develop the custom application that would display information from a portable ultrasound machine connected through a wireless network in the form of a hologram. The voice controls on the HoloLense allowed scaling and movement of the ultrasound imaging while displaying in real-time.

Largely driven by video gaming and consumer applications, AR and virtual reality technologies have been growing and are expected to grow at an even faster clip in the coming decade.

“We are a handful of researchers exploring the innovative application of the technology for ultrasound visualization,” said Shekhar. “We are trying to establish ourselves as the leader in the use of AR for pediatric ultrasound procedures.”

To further advance this technology, Shekhar’s team has an IRB protocol pending that will continue to shed light on the benefits through more bench testing and an NIH grant proposal that is also pending. In the meantime, they will adopt second-generation glasses to continue to improve the technology.

two doctors perform surgery

Can complex pediatric surgery interventions be standardized to facilitate telementoring?

two doctors perform surgery

The study’s authors write, “These discussions are particularly relevant to surgeons in small or rural practices who provide much-needed care to underserved populations and have decreased exposure to these index cases. Conversely, in some developing countries where prevalence of rare congenital surgical conditions is higher, there is a shortage of adequately trained pediatric surgeons. Each of these scenarios involves a mismatch in experience and exposure, which can result in poor patient outcomes and inadequate healthcare delivery.”

How does a surgeon-in-training get enough exposure to rare or complex cases to serve the patients who need them? How does a practicing surgeon perform enough cases each year to maintain proficiency at such index cases?

The authors of a study in the Journal of Pediatric Surgery, including Marc Levitt, M.D., chief of the Division of Colorectal and Pelvic Reconstruction at Children’s National Hospital, write that, “These discussions are particularly relevant to surgeons in small or rural practices who provide much-needed care to underserved populations and have decreased exposure to these index cases. Conversely, in some developing countries where prevalence of rare congenital surgical conditions is higher, there is a shortage of adequately trained pediatric surgeons. Each of these scenarios involves a mismatch in experience and exposure, which can result in poor patient outcomes and inadequate healthcare delivery.”

Telementoring is one strategy being explored by the American College of Surgeons’ Telementoring Task Force initiative. Pediatric anorectal malformations (ARM), pediatric colorectal surgical procedure, posterior sagittal anorectoplasty (PSARP) were the “index” areas for the pilot study. Once the expert established the areas of great need, they will test the feasibility of a curriculum and training program using telementoring in pediatric surgery. The ACS Task Force notes that these conditions are relatively rare and require a particular skill level to manage appropriately, making them good candidates for the study.

The Journal of Pediatric Surgery study presents a process for mapping out a standardized curriculum for these procedures. First, the authors sought expert consensus on three interoperative checklists that form a de facto curriculum for teaching, learning and performing ARM and PSARP procedures. Second, a multidisciplinary team of medical educators and pediatric surgery experts drafted the checklists. The authors then sought review and input from pediatric colorectal surgery experts at 10 institutions worldwide, who comprised the study’s colorectal pediatric surgery subject matter expert panel. To be considered “expert,” participants had to meet or exceed several strict inclusion criteria related to years in practice and experience with these case types.

Institutions of the colorectal pediatric surgery subject matter expert panel.

Institutions of the colorectal pediatric surgery subject matter expert panel.

The process led to a successful set of consensus documents. “To our knowledge, this is the first study to establish and standardize key intraoperative objectives using a modified-Delphi method in pediatric surgery,” the authors write. “Although this process can be quite time consuming, it provides an incredible opportunity to standardize intraoperative teaching and expectations of trainees. Future studies will expand these checklists into developing a competency assessment tool involving assessment for validity and reliability in a clinical setting to ultimately improve patient safety through standardization.”

Dr. Levitt says the overarching goal of this work is “to improve the surgical technique everywhere [to] thereby help as many kids as we can, even those we will never meet.”