Public Health Archives

Healthcare leaders join to advance pediatric innovation

December 21, 2022

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

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

Convening a coalition of healthcare leaders

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

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

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

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

Using RWE to facilitate the regulatory pathway

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

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

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

About NCC-PDI

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

Congenital Heart Initiative celebrates global reach in first two years

December 7, 2022

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

Why it matters

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

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

The patient benefit

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

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

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

What’s unique

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

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

What’s next

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

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

In terms of research, the CHI now has enough participants to allow researchers to complete some important studies about pregnancy, mental health and long-term health care follow ups, all of which will kick off within the next year. The research teams hope to engage centers across the United States to better determine the needs of individual ACHD centers and to understand how the CHI can help with these needs.

Early SARS-CoV-2 exposure may impact infant development

November 30, 2022

The study found that some infants with in utero or early-life exposure to SARS-CoV-2 had borderline to low developmental screening scores.

Early SARS-CoV-2 exposure may impact neurodevelopment, especially among infants exposed in utero to symptomatic parents. This is according to a new study led by Sarah Mulkey, M.D., Ph.D., prenatal-neonatal neurologist in the Prenatal Pediatrics Institute at Children’s National Hospital. Dr. Mulkey and team conclude that vaccination and other precautions to reduce early-in-life infection may protect against neurodevelopmental delays. Children with early SARS-CoV-2 exposure should have additional long-term screening for neurodevelopmental delays.

Children’s National Hospital leads the way

The developing brain is vulnerable to both direct and indirect effects of infection during pregnancy and in the early neonatal period. To chart the impact of this exposure, the team created a clinical follow-up protocol in the Congenital Infection Program at Children’s National to chart the development of 34 infants exposed to SARS-CoV-2 in utero or in the neonatal period.

What we hoped to discover

“We conducted this study because we know that infants, when exposed to maternal COVID-19 infection in utero can be exposed to inflammation, fever and an abnormal intrauterine environment. SARS-CoV-2 can also affect the placenta, and in turn, the developing brain,” Dr. Mulkey shared with Healio.

This study aimed to determine if infants with early SARS-CoV-2 exposure developed abnormal neurodevelopment in infancy and the factors that may impact neurodevelopment differences. The study found that some infants with in utero or early-life exposure to SARS-CoV-2 had borderline to low developmental screening scores, most common among babies born to mothers with symptomatic COVID-19. Researchers followed the infants in their first months of life, gauging how the exposure affected their neurologic development. Results were demonstrated using a screening test called the Ages & Stages Questionnaires (ASQ), and those whose scores were borderline or low were most often born to mothers with symptomatic COVID-19.

Why it matters

In conducting this study, the team found that babies born during the pandemic, specifically under these conditions, do, in fact, require additional follow-up in the early stages of life. We may also see more differences in developmental outcomes as children get older.

“Any measure we can take to help prevent infections for mothers in their pregnancy can improve long-term developmental outcomes for children,” says Dr. Mulkey.

Other members of the Children’s National team that contributed to this work include Roberta L. DeBiasi, M.D., M.S.; Meagan E. Williams, M.S.P.H.; Nadia Jadeed, R.N.C.; Anqing Zhang, Ph.D.; and Smitha Israel, B.S.N.

Dr. Mulkey also published a recent article in the American Journal of Obstetrics & Gynecology that found the COVID-19 vaccine may protect pregnant women from SARS-CoV-2 placentitis and stillbirth. This work builds upon Dr. Mulkey’s longitudinal studies on Zika virus infection in pregnancy and long-term impacts on the child, funded by the Thrasher Research Fund and the National Institutes of Health.

Exposure to Zika in utero may produce neurodevelopmental differences

November 30, 2022

“There are still many unanswered questions about the long-term impacts of Zika on children exposed in utero,” says Sarah Mulkey, M.D., Ph.D., a prenatal-neonatal neurologist in the Prenatal Pediatrics Institute at Children’s National Hospital.

Children who are exposed to the Zika virus while in the womb, but who are not subsequently diagnosed with Zika-related birth defects and congenital Zika syndrome (CZS), may still display differences in some aspects of cognitive development, mood and mobility compared to unexposed children, reports a study published in Pediatric Research. These findings suggest that Zika-exposed children may need some additional support and monitoring as they get older.

“There are still many unanswered questions about the long-term impacts of Zika on children exposed in utero,” says Sarah Mulkey, M.D., Ph.D., a prenatal-neonatal neurologist in the Prenatal Pediatrics Institute at Children’s National Hospital and the study’s first author. “These findings are another piece of the puzzle that provides insight into the long-term neurodevelopment of children with prenatal Zika virus exposure. Further evaluation is needed as these children get older.”

It has not been clear how children who were exposed to the Zika virus in the womb during the 2015–2017 epidemic, but who did not develop CZS and serious neurological complications, will develop as they get older.

Dr. Mulkey and colleagues examined the neurodevelopment of 55 children aged 3-5 years who were exposed to Zika in the womb in Sabanalarga, Colombia, and compared them to 70 control children aged 4-5 years who had not been exposed to Zika. Assessments occurred between December 2020 and February 2021. Health professionals tested the children’s motor skills (such as manual dexterity, aiming and catching, and balance) and their readiness for school (including knowledge of colors, letters, numbers and shapes). Parents completed three questionnaires providing information about their child’s cognitive function (such as memory and emotional control), behavioral and physical conditions (such as responsibility and mobility), and their parenting experience (including whether they felt distress).

Parents of Zika-exposed children reported significantly lower levels of mobility and responsibility compared to control children, although differences in cognitive function scores were not significant. Additionally, parents of 6 (11%) Zika-exposed children reported mood problems compared to 1 (1%) of control children, and Zika-exposed parents were significantly more likely to report parental distress.

Professional testing revealed no significant differences in the Zika-exposed children’s manual dexterity, such as their ability to catch an object or post a coin through a slot, compared to the control children. Both Zika-exposed and control children also scored lowly on readiness for school.

The authors highlight that parental responses may have been influenced by the Zika-exposed children’s parents’ perceptions or increased worry about the development of their child. Some differences in results may also have been caused by the age – and therefore developmental – differences between the groups of children.

The authors conclude that while these Zika-exposed children are making progress as they develop, they may need additional support as they prepare to start school.

Dr. Mulkey is committed to studying the long-term neurodevelopmental impacts that viruses like Zika and SARS-CoV-2 have on infants born to mothers who were infected during pregnancy through research with the Congenital Infection Program at Children’s National and in collaboration with colleagues in Colombia.

Five winners selected in prestigious pediatric device competition

October 26, 2022

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 10^th 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.

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)

Innovations for health equity: Food pharmacy app wins Hackathon

September 29, 2022

When families come into the endocrinology clinic, 66% of prediabetes and Type 2 diabetes patients screen positive for food insecurity. One remedy: a smartphone app envisioned by Children’s National Hospital researchers to communicate with families between visits and provide resources to help stock pantries with nutritious foods.

The Children’s National Food Pharmacy app is on its way from idea to reality, thanks to the inaugural Health Equity in Research Hackathon event at the Children’s National Research & Innovation Campus. This team-based, “Shark Tank”-like competition involved roughly 50 experts designing creative healthcare solutions that could be delivered through ubiquitous smartphones.

“It takes a village to raise a child, and we want to show that at Children’s National we are part of that community,” said food pharmacy founder and diabetes educator Alexis Richardson, M.S., R.D., L.D.N., C.D.C.E.S.

Why it matters

The rate of new-onset Type 2 diabetes increased by a staggering 182% during the first nine months of the COVID-19 pandemic. Now, the Children’s National food pharmacy provides families that screen positive for food insecurity during quarterly clinic appointments with a 50-pound bag of medically-tailored groceries.

The new app, as envisioned, would follow them home to connect them with food bank information and other nutritional resources, eliminating paper forms and other hurdles that get in the way of care.

What’s ahead

Children’s National leaders are committed to making the proposal a reality. “We are going to support today’s winner through the next steps to prepare them to enter the app development pipeline at the Sheikh Zayed Institute,” said Lisa Guay-Woodford, M.D., director for the Clinical and Translational Science Institute at Children’s National (CTSI-CN) and one of the main judges of the competition.

The app development will happen in the months ahead. Kevin Cleary, Ph.D., technical director of the Sheikh Zayed Institute of Pediatric Surgical Innovation, said the Hackathon planted the seeds. “It really depends on the drive of the individual to see the idea to fruition,” Cleary told competitors.

Other app entries were encouraged to continue their work:

The Surgical Checklist, led by Brian K. Reilly, M.D., co-director of the Cochlear Implant Program: this app would help patients and providers successfully navigate the often-confusing pre-operative checklist, including required physical exams, lab work, imaging and pre-procedure fasting. Reilly said the hospital handles about 15,000 cases a year, and about 10% are rescheduled, often for reasons that could be avoided with digital organization and reminders for families.
More than Determined, led by Pediatrician Jessica Lazerov, M.D., M.B.A.: this app aims to give time-strapped providers a platform to better understand and address social determinants of health – such as access to safe housing, education and jobs – that can promote better preventative care outcomes.

The Health Equity in Research Hackathon was created by the new Health Equity in Research Unit, a joint initiative between the CTSI-CN and the Center for Translational Research within the Children’s National Research Institute.

Dr. Lisa Guay-Woodford and the winners of the Health Equity in Research Hackathon

Dr. Lisa Guay-Woodford, director for the Clinical and Translational Science Institute, joins the winners of the inaugural Health Equity in Research Hackathon: the Children’s National Food Pharmacy. The team’s proposed app will connect families facing food insecurity with resources and guidance for nutritious eating.

Multicenter trial finds bionic pancreas improves Type 1 diabetes management

September 29, 2022

Compared to other available artificial pancreas technologies, the bionic pancreas requires less user input and provides more automation because the device’s algorithms continually adjust insulin doses automatically.

A device known as a bionic pancreas, which uses next-generation technology to automatically deliver insulin, was more effective at maintaining blood glucose levels within normal range than standard-of-care management among people with Type 1 diabetes, a new multicenter clinical trial found.

The trial, conducted partly at Children’s National Hospital, was primarily funded by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), part of the National Institutes of Health, and published in the New England Journal of Medicine.

Automated insulin delivery systems – also called artificial pancreas or closed-loop control systems – track a person’s blood glucose levels using a continuous glucose monitor and automatically deliver the hormone insulin using a pump. These systems replace reliance on insulin delivery by multiple daily injections, pumps without automation and testing glucose levels using more labor-intensive systems, such as fingersticks.

“A diagnosis of Type 1 diabetes can be overwhelming for a child and their family,” said Fran Cogen, M.D., C.D.C.E.S., principal investigator at Children’s National and director of the Childhood and Adolescent Diabetes Program. “It is extremely important to monitor and manage glucose levels throughout the day to prevent serious complications like eye problems, kidney disease, heart and blood vessel disease. This new technology may give patients and families a sense of relief from some of the daily stressors that come with the diagnosis of this chronic disease.”

Compared to other available artificial pancreas technologies, the bionic pancreas requires less user input and provides more automation because the device’s algorithms continually adjust insulin doses automatically. Users of the bionic pancreas also do not have to count carbohydrates, nor initiate doses of insulin to correct for high blood glucose. In addition, healthcare providers do not need to make periodic adjustments to the device’s settings.

The 13-week trial, conducted at Children’s National and 15 other U.S. clinical sites, enrolled 326 participants ages 6 to 79 years who had Type 1 diabetes and had been using insulin for at least one year. Participants were randomly assigned to either a treatment group using the bionic pancreas device or a standard-of-care control group using their personal pre-study insulin delivery method.

The study found:

In participants using the bionic pancreas, glycated hemoglobin improved from 7.9% to 7.3%, yet remained unchanged among the standard-of-care control group.
The bionic pancreas group participants spent 11% more time within the targeted blood glucose range compared to the control group.
Results were similar in youth and adult participants.
Improvements in blood glucose control were greatest among participants who had higher blood glucose levels at the beginning of the study.

Hyperglycemia caused by equipment problems was the most frequently reported adverse event in the bionic pancreas group. The number of mild hypoglycemia events and frequency of severe hypoglycemia were not different in the two groups.

“The results of this study will bring hope to patients, families and providers that there are technologies being created to help ease the burden of diabetes management and keep glucose levels more stable,” said Kimberly Boucher, M.S.H.S., B.S.N., R.N., clinical research manager of Endocrinology at Children’s National.

The study is one of several pivotal trials funded by NIDDK to advance artificial pancreas technology and look at factors including safety, efficacy, user-friendliness, physical and emotional health of participants, and cost. To date, these trials have provided the important safety and efficacy data needed for regulatory review and licensure to make the technology commercially available. The Jaeb Center for Health Research in Tampa, Florida, served as coordinating center.

Funding for the study was provided by NIDDK grant 1UC4DK108612 to Boston University, by an Investigator-Initiated Study award from Novo Nordisk, and by Beta Bionics, Inc., which also provided the experimental bionic pancreas devices used in the study. Insulin and some supplies were donated by Novo Nordisk, Eli Lilly, Dexcom and Ascensia Diabetes Care. Partial support for the development of the experimental bionic pancreas device was provided by NIDDK SBIR grant 1R44DK120234 to Beta Bionics, Inc.

You can read the full study, Multicenter, Randomized Trial of a Bionic Pancreas in Type 1 Diabetes, in the New England Journal of Medicine.

Successful autism and ADHD tools go digital

September 25, 2022

A team is working to implement a successful, evidence-based online training and tele-support system for the Unstuck and On Target (UOT) program.

A team from Children’s National Hospital, Children’s Hospital Colorado and The Institute for Innovation and Implementation at the University of Maryland, Baltimore is working to implement a successful, evidence-based online training and tele-support system for the Unstuck and On Target (UOT) program. The program is now available for free to any parent or educator who needs it.

What is it?

Since 2020, this team has piloted UOT video training with 293 school-based staff across 230 elementary schools in Colorado and Virginia. The work follows a related PCORI-funded research project, Improving Classroom Behaviors Among Students with Symptoms of Autism Spectrum Disorder or Attention Deficit Hyperactivity Disorder, led by Children’s National and Children’s Colorado researchers. That project demonstrated the effectiveness of UOT at improving the executive functioning – or self-regulation skills including flexible thinking, planning and emotional-control – of school-aged children in Title 1 schools. The training focuses on the executive function of elementary school-aged children with autism spectrum disorder (ASD) or attention deficit hyperactivity disorder (ADHD).

In addition to creating more accessible training for educators, the team created short, free videos highlighting executive functioning tips and tricks that parents can employ at home. These videos, evaluated by 100 parents and revised based on their input, are now available to parents nationwide.

The availability of this training is possible due to a $2 million contract awarded to Children’s Hospital Colorado’s (Children’s Colorado) Pediatric Mental Health Institute and Children’s National by the Patient-Centered Outcomes Research Institute (PCORI) in 2020.

Why it matters

There are many children, including those in low-income or rural settings, that don’t have access to clinics that offer services to support executive functioning skills, such as planning and flexibility, that they need. But all children have access to a school. Now, UOT training is online and accessible so any school with internet access can offer UOT where school staff (including special educators, teachers, paraprofessionals and counselors) can actively teach students how to plan, set goals and be flexible. The team’s next goal is to create a comparable video training for the high school version of UOT.

“These free, accessible and effective tools for improving children’s social-emotional development are building skills that are more important today than ever,” said Lauren Kenworthy, Ph.D., director of the Center for Autism Spectrum Disorders at Children’s National. “The vast majority (96%) of caregivers and educators found these tools useful and relevant. That feedback is a testament to our team’s efforts to make sure these resources were created and validated as usable, approachable and actionable for everyone who needs them.”

More information

For educators – Find resources on Unstuck and On Target, including links to the free trainings, tips and tricks and FAQs. Teachers can also receive continuing education credits (CEUs) for this training.

For parents – Find resources on Unstuck and On Target’s parent training videos

For schools – Add free Unstuck and On Target parent videos to your school district’s relevant websites, landing pages and newsletters.

AI may revolutionize rheumatic heart disease early diagnosis

September 20, 2022

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 25^th International Conference on Medical Image Computing and Computer Assisted Intervention (MICCAI).

Children’s National Innovation Day aims to advance pioneering pediatric life science projects

August 23, 2022

Children’s National Research & Innovation Campus’ Innovation Day will feature life science projects focused on improving pediatric care.

Pioneering life science projects focused on improving pediatric care will be on display at the Children’s National Research & Innovation Campus when the hospital hosts its 2022 Innovation Day on Friday, August 26. Hosted by Children’s National Innovation Ventures, the program’s goal is to showcase life sciences and healthcare projects that are mature enough to look for a co-developer, strategic partner, investor or licensing vehicle.

“For us, a successful Innovation Day means we are able to match these entrepreneurs with the strategic partner they need at this stage of their device development journey,” says Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National Hospital and executive director of the Sheikh Zayed Institute for Pediatric Surgical Innovation.

“Data continues to show that the national Capital Region (NCR) remains one of the most robust life sciences and technology hubs in the country with no shortage of visionary leaders. There is no better place to conduct this important work as we seek more ways to advance pediatric medical technologies to improve health outcomes.”

A total of 17 projects will be showcased at the event, with each having up to 10-minute window to make their presentation. All attendees will be provided a survey, enabling valuable feedback for presenters as they look to take their next strategic step on the pathway to further development and commercialization.

In addition to presenting projects, the 2022 Innovation Day will also feature startup companies whose mission is aligned with Children’s National’s quest to bring novel pediatric medical products to patients and families. Throughout the day, attendees will have the opportunity to meet with the hospital’s researchers, academic entrepreneurs and prominent stakeholders in the life science and healthcare innovation ecosystem in the region. Interested investors and strategics can also request one-on-one meetings with startups and research teams.

Eskandanian, who also serves as the executive director of the FDA-funded National Capital Consortium for Pediatric Device Innovation (NCC-PDI), notes that accelerating this pathway to commercialization for pediatric products will bring more viable technologies to market, an important step in addressing the ongoing development disparity in innovations developed for children versus adults.

“For too long, children have been left behind in the development and commercialization of medical products and we remain committed to altering the trend,” Eskandanian says. “Children’s National’s Innovation Ventures team is working closely with the presenting innovators to provide any and all support that we can to get these products to the next stage.”

Registration for Children’s National’s 2022 Innovation Day is currently open. Those interested in attending the day-long showcase can register at https://conta.cc/3CrAfTl.

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, helps to bolster pediatric innovation and commercialization.

Second dose of Pfizer COVID-19 vaccine safe for children with allergic reaction to first dose

July 13, 2022

A new study published in The Journal of Allergy and Clinical Immunology: In Practice found that pediatric patients who experienced an adverse reaction to the first dose of the Pfizer-BioNTech COVID-19 vaccine or with suspected polyethylene glycol or polysorbate allergies can safely receive the second dose in a supervised setting. Until now, previous studies demonstrating second dose safety after a reaction to the first dose have only included adult patients.

“These results reaffirm similar studies performed in adults and provide additional assurance specific to the pediatric population,” says Joel Brooks, D.O., M.P.H., allergist and immunologist at Children’s National Hospital and corresponding author of the study. “We found that most of these initial reactions are not supportive of an IgE-mediated mechanism.”

The researchers evaluated 13 children referred to a specialized vaccine clinic for suspected immediate allergic reactions to the first dose of the Pfizer-BioNTech COVID-19 vaccine from May 2021 to February 2022. Nine of the 13 children were evaluated after experiencing an allergic reaction following the first dose. All nine successfully received the second dose with no or minimal symptoms.

The other four children were evaluated after clinical histories of PEG/polysorbate allergy. Three of the four received both Pfizer vaccine doses with no symptoms. The fourth patient elected to receive the Janssen COVID-19 vaccine.

“It is important that children 6 months and older receive two doses of the COVID-19 vaccine for full protection from severe illness and hospitalization due to COVID-19 infection,” adds Dr. Brooks. “Children with potential anaphylaxis should undergo careful evaluation to weigh the benefits and risks of the second dose.”

You can read the full study, “Safety outcomes of SARS-CoV-2 vaccination in pediatric patients with a first dose reaction history or allergy to polyethylene glycol or polysorbate,” here.

Pediatric medical device competition names finalists

June 3, 2022

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 10^th 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.

Pandemic-related stressors in pregnant women affect fetal brain development

May 26, 2022

Dr. Catherine Limperopoulos walking with a mom.

Prolonged levels of stress and depression during the COVID-19 pandemic contributed to altering key features of fetal brain development — even if the mother was not infected by the virus. This is what a study published in Communications Medicine suggests after following more than 200 pregnant women. The study, led by Children’s National Hospital experts, emphasized the need for more scientific inquiry to shed light on the long-term neurodevelopmental consequences of their findings and COVID-19 exposures on fetal brain development.

“Understanding how contemporary stressors may influence fetal brain development during pregnancy has major implications for basic science and informing public policy initiatives,” said Catherine Limperopoulos, Ph.D., chief and director of the Developing Brain Institute at Children’s National and senior author of the study. “With this work, we are able to show there’s a problem, it’s happening prenatally, and we can use this model to start exploring how we can reduce stress in moms and support unborn babies.”

To better understand the effects of environmental exposures on the fetus during pregnancy, further confirmation of the team’s latest findings is needed by ruling out other possibilities, such as maternal nutrition, financial security and genetic factors.

The psychosocial impact of COVID-19 on fetal brain development remains vastly understudied. The neurologic underpinnings of fetal development that turn into psycho-behavioral disorders later in life, including bipolar disorder, mood disorder or anxiety disorder, remain complex and difficult to explain.

Among the 202 participants from the Washington D.C. metropolitan area, 137 were part of the pre-pandemic cohort and 65 were part of the pandemic cohort.

Through advanced MRI imaging techniques and reconstruction of high-resolution 3D brain models, the researchers found a reduction of fetal white matter, hippocampal and cerebellar volumes and delayed brain gyrification in COVID-19 pandemic-era pregnancies. Validated maternal stress, anxiety and depression scales were also used to compare the scores between the two cohorts.

This study builds upon previous work from the Developing Brain Institute led by Limperopoulos, which discovered that anxiety in pregnant women appears to affect the brain development of their babies. Her team also found that maternal mental health, even in high socioeconomic status, alters the structure and biochemistry of the developing fetal brain, emphasizing the importance of mental health support for pregnant women.

“We’re looking at modifiable conditions,” said Limperopoulos. “What’s clear is the next frontier is intervening early to see how we can prevent or reduce stress in the mom’s current setting.”

Children’s National Hospital at the 2022 Pediatric Academic Societies Meeting

April 18, 2022

Are you attending the 2022 Pediatric Academic Societies meeting this week? There will be over 20 Children’s National Hospital-affiliated participants at this year’s meeting. We have compiled their sessions into a mini schedule below.

Name	Department	Role	Topic	Date	Time
Kristen Sgambat, Ph.D., R.D.	Center for Translational Research	Speaker	Fueling our patients for success: Optimizing nutritional support for kids with kidney disease	4/22/2022	2:30 PM
Priti Bhansali, M.D., M.Ed.	Child Health Advocacy Institute	Co-speaker	APA Division Directors/Faculty Development Combined SIG	4/23/2022	8:00 AM
Karen Smith, M.D., M.Ed. Neha Shah, M.D., M.P.H.		Workshop co-leaders	Don’t Struggle In Solitude: Recovery and Peer Support after Unanticipated Outcomes, Errors, and Difficult Conversations	4/23/2022	8:00 AM
Ian Chua, M.D., M.H.P.E. Gabrina Dixon, M.D., M.Ed. Margarita Ramos, M.D., M.S.		Workshop co-leaders	Finding the DEI in LGBTQIA: Incorporating LGBTQIA diversity in your environment	4/23/2022	8:00 AM
Kevin M. Cook, Ph.D.		Co-presenter	Early exposure to the extra-uterine environment in premature infants is associated with altered functional brain connectivity compared to in-utero age-matched fetuses	4/23/2022	8:15 AM
Gabrina Dixon, M.D., M.Ed. Terry Kind, M.D., M.P.H.		Workshop co-leaders	Changing the system: Best practices in supporting and advancing underrepresented in medicine (UIM) medical students	4/23/2022	10:00 AM
Yao Wu, Ph.D.		Oral abstract presenter	Impaired prenatal brain growth predicts adverse neurodevelopmental outcomes in infants with congenital heart disease	4/23/2022	10:00 AM
Lee S. Beers, M.D.	General and Community Pediatrics	Moderator	Scholarship in the Domain of Child Health Advocacy: Making It Work in the Academic Medical Center	4/23/2022	10:00 AM
Chaya Merrill, Dr.P.H.	Center for Translational Research	Speaker	Using data to advance advocacy in the academic medical center	4/23/2022	10:00 AM
Yuan-Chiao Lu, Ph.D.		Oral abstract presenter	Delayed Fetal Cortical Maturation Predicts 18-Month Neurodevelopment in Infants with Congenital Heart Disease	4/23/2022	10:15 AM
Olanrewaju O. Falusi, M.D., M.Ed.	Child Health Advocacy Institute	Speaker	Generating currency for advancement and professional development in the domain of advocacy	4/23/2022	10:15 AM
Subechhya Pradhan, Ph.D.		Oral abstract presenter	Abnormal in-vivo brain biochemistry in fetuses with complex congenital heart disease	4/23/2022	10:30 AM
Lenore R. Jarvis, M.D., M.Ed.	Emergency Medicine and Trauma Services	Speaker	Academic advocacy for the subspecialist	4/23/2022	10:30 AM
Jillian E. Nickerson, M.D., M.S.	Emergency Medicine and Trauma Services	Presenter	Utilizing an Online Module Platform to Teach Newborn Delivery and Resuscitation Skills to Pediatric Emergency Medicine Providers	4/23/2022	10:30 AM
Lee S. Beers, M.D.	General and Community Pediatrics	Presenting Author	Leadership in legislative advocacy at the national level	4/23/2022	11:00 AM
Kevin M. Cook, Ph.D.		Oral abstract presenter	Relative neighborhood disadvantage is associated with increased functional network segregation in fetal brains	4/23/2022	11:15 AM
Jung-Hoon Kim, Ph.D.		Presenting Author	Gestational age-related changes in the fetal functional connectome: in utero evidence for the global signal	4/23/2022	1:00 PM
Ioannis Koutroulis, M.D., Ph.D., M.B.A.	Emergency Medicine and Trauma Services	Oral abstract presenter	Immunometabolism in septic encephalopathy: a novel therapeutic target	4/23/2022	1:00 PM
Terry Kind, M.D., M.P.H.	General and Community Pediatrics	Workshop co-leaders	Making Meaning from the Data: Exploring Coding in Qualitative Research	4/23/2022	1:00 PM
Josepheen D. Cruz, M.D., Ph.D.		Oral abstract presenter	Cortical thickness changes in fetuses exposed to heightened maternal psychological distress	4/23/2022	1:30 PM
Monika Goyal, M.D., M.S.C.E.	Emergency Medicine and Trauma Services	Moderator	Adolescent Medicine II	4/24/2022	8:00 AM
Binny Chokshi, M.D., M.Ed. Yael Smiley, M.D.		Workshop co-leaders	Applying The Collective Impact Model to Pediatric Health Interventions	4/24/2022	8:00 AM
Aisha Barber, M.D. M.Ed.	Hospital Medicine	Workshop co-leaders	Demystifying DEI in Recruitment: Strategies for Creating a Diverse and Inclusive Residency and Fellow Training Environment	4/24/2022	8:00 AM
Panagiotis Kratimenos, M.D., Ph.D.	Neonatology	Oral abstract presenter	Maternal Immune Activation and Hypoxia Induces Cerebellar Injury	4/24/2022	8:45 AM
Monika Goyal, M.D., M.S.C.E.	Emergency Medicine and Trauma Services	Co-moderator	Emergency Medicine I	4/24/2022	10:00 AM
Priti Bhansali, M.D., M.Ed.	Hospital Medicine	Workshop co-leaders	Making the Most of Peer Mentors within a Diverse Developmental Network: Supporting Scholarship and Academic Advancement	4/24/2022	10:00 AM
Ian Chua, M.D., M.H.P.E. Gabrina Dixon, M.D., M.Ed. Karen Smith, M.D., M.Ed.	Hospital Medicine	Workshop co-leaders	The Art of Negotiation: Applying Negotiation Frameworks to Get More of What You Want in Your Academic Career	4/24/2022	10:00 AM
Matthew Magyar, M.D.	Hospital Medicine	Oral abstract presenter	The association between social needs and unscheduled healthcare utilization among a nationally representative sample of children with asthma	4/24/2022	10:00 AM
Lena A. Saleh, M.D., M.P.H.		Oral abstract presenter	Machine Learning to Predict the Need for Intensive Care for Pediatric Asthma Exacerbation	4/24/2022	10:30 AM
Christina R. Rojas, M.D.	Emergency Medicine and Trauma Services	Oral abstract presenter	Pediatric Emergency Department Undertriage for Patients with Limited English Proficiency	4/24/2022	10:30 AM
Ololade Okito, M.D.	Neonatology	Workshop co-leaders	Best Practices in DEI Recruitment: Holistic Review and Addressing Systemic Bias	4/25/2022	8:00 AM
Jennifer H. Klein, M.D.		Presenter	Geography of pediatric health: Using geospatial analysis tools in pediatric care	4/25/2022	8:00 AM
Anand Gourishankar, M.B.B.S., M.R.C.P., M.A.S.	Hospital Medicine	Presenter	Geospatial analysis in pediatric health: Principles, pitfalls, and practice	4/25/2022	8:00 AM
Sarah D. Schlatterer, M.D., Ph.D.	Neurology	Oral abstract presenter	Autonomic Dysfunction and Hemodynamic Instability Precedes Cardiac Arrest in Infants with Congenital Heart Disease	4/25/2022	8:15 AM
Chaya Merrill, Dr.P.H.		Presenter	Mapping neighborhood-level inequities using the Childhood Opportunity Index	4/25/2022	8:20 AM
Jennifer H. Klein, M.D.		Speaker	Geospatial distribution of congenital heart disease	4/25/2022	8:40 AM
Ioannis Koutroulis, M.D., Ph.D., M.B.A.	Emergency Medicine and Trauma Services	Panelist	APA Urgent Care SIG	4/25/2022	10:00 AM
Priti Bhansali, M.D., M.Ed.	Hospital Medicine	Workshop co-leaders	From Mediocre to Masterly: Using Cognitive Interviewing to Improve the Validity of Your Survey	4/25/2022	10:00 AM
Beth A. Tarini, M.D., M.S.	General and Community Pediatrics	Speaker	SPR Presidential Plenary: “Transforming the Culture of Pediatric Research: We Are the Problem and the Solution	4/25/2022	10:00 AM
Deena Berkowitz, M.D., M.P.H.	Emergency Medicine and Trauma Services	Speaker	2. UC fellowships and accreditation: the APA pipeline	4/25/2022	10:30 AM
John T. Kulesa, M.D.	Hospital Medicine	Oral abstract presenter	A Descriptive Model for Prioritization and Resource Allocation in Academic Global Health Partnerships	4/25/2022	10:30 AM
Ariella Slovin, M.D.	General and Community Pediatrics	Speaker	APA Well-being and Vitality SIG	4/25/2022	1:00 PM
Melissa Baiyewu, M.H.A., C.H.E.S. Lin Chun-Seeley, M.A. Desiree D. de la Torre, M.P.H., M.B.A. Olanrewaju O. Falusi, M.D., M.Ed. Chaya Merrill, Dr.P.H.	General and Community Pediatrics	Workshop co-leaders	Training Faculty Members to Model and Teach Health Equity: A New Faculty Development Curriculum	4/25/2022	1:00 PM
Ariella Slovin, M.D.	General and Community Pediatrics	Speaker	Wellness and Vitality SIG: Overview of endeavors to date and status report on well-being of APA Members	4/25/2022	1:30 PM
Beth A. Tarini, M.D., M.S.	General and Community Pediatrics	Speaker	Navigating Research Careers Through the Currents of Policy and Politics	4/25/2022	1:36 PM
Rebecca S. Lundberg, M.D.		Oral abstract presenter	Early parenteral nutrition support and preterm cerebellar metabolic maturation	4/25/2022	2:00 PM
Aisha Barber, M.D., M.Ed.	Hospital Medicine	Workshop co-leader	Moving with the Tide: Taking Steps Toward Anti-Racism and Equity	4/233/22	1:00 PM

Medical device pitch competition returns with $150K in FDA awards

April 12, 2022

“This pitch competition helps to recognize and support the advancement of innovations that can specifically address the needs of pediatric patients,” 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.

Recognizing the continued gap in the development and commercialization of medical devices for children versus adults, the National Capital Consortium for Pediatric Device Innovation (NCC-PDI), in collaboration with MedTech Innovator, is accepting applications through April 22, 2022, for its annual “Make Your Medical Device Pitch for Kids!” competition. Recognizing the wide range of unmet needs for diagnostic and therapeutic devices designed especially for children, this year’s competition is open to any innovation in medical technology that addresses a significant unmet need in pediatric medical care.

“As one of the five FDA Pediatric Device Consortia, NCC-PDI is focused on seeking out and addressing significant unmet needs in pediatric medical technology,” 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. “While great advances are made in adult medical devices, children are often left behind because the pediatric market is small and there are not incentives to develop for pediatrics. This pitch competition helps to recognize and support the advancement of innovations that can specifically address the needs of pediatric patients.”

Using a virtual format, semi-finalists chosen from all submissions will make their first pitch on May 20, 2022. Up to six finalists selected from this first round will earn participation in a special pediatric-focused track of the MedTech Innovator accelerator program, the largest medical device accelerator in the world, beginning in June 2022. These innovators will then participate in the competition finals in the fall 2022 where judges will award up to $150,000 in FDA-sponsored grants to the devices selected as most impactful and commercially viable.

Unlike devices for adults, the development and commercialization of pediatric medical devices lags behind by approximately five to 10 years. Programs like the NCC-PDI pitch competition and MedTech Innovator accelerator program offer innovators access to expert insight and consultation to help overcome regulatory hurdles and advance the product’s development path.

NCC-PDI is one of five members 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, BioHealth Innovation and design firm Archimedic.

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.

Applications for the “Make Your Medical Device Pitch for Kids!” competition are open now through April 22 for innovations that address unmet pediatric needs.

Grand Rounds analyzes the state of life sciences industry in Maryland

April 7, 2022

A recent Children’s National Grand Rounds panel analyzed the state of the life sciences industry in the region and the potential steps that can be taken to accelerate the manufacturing landscape.

The BioHealth Capital Region, consisting of Maryland, Virginia and Washington, D.C., has surged in recent years to become one of the nation’s strongest biopharma clusters. While the region does not lack opportunity to cultivate and grow innovation, manufacturing business related to scientific research and development in Maryland is lagging compared to other leading states.

That was the message from Martin Rosendale, CEO of the Maryland Tech Council, who was recently the featured speaker of the Children’s National Grand Rounds panel, which analyzed the state of the life sciences industry in the region and the potential steps that can be taken to accelerate the manufacturing landscape. The Maryland Tech Council is a not-for-profit collaborative community actively engaged in building strong life sciences and technology industries.

Moderated by Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National Hospital, the panel cited data recently compiled by the Milken Institute Center for Regional Economics that demonstrated the degree to which Maryland’s life sciences manufacturing industry needs to catch-up.

“On the manufacturing business front, we’ve actually been lagging compared to the rest of the country. Other metros have grown faster than we have over the last five years, but that presents an opportunity,” says Rosendale. “We have the opportunity to work more with academia and medical institutions like Children’s National to produce more companies, more intellectual property and more startups.”

According to the Milken data, when compared to other leading states including Massachusetts (2^nd), California (3^rd), New York (8^th), Florida (9^th) and Texas (12^th), Maryland ranked as the 20^th state for life sciences manufacturing growth over a five-year period. Of the growth that occurred over that time, the greatest came in the Washington-Arlington-Alexandria and Baltimore-Columbia-Towson metros, where industry wages tend to be higher.

Kolaleh Eskandanian, vice president and chief innovation officer at Children’s National Hospital, recently moderated the Children’s National Grand Rounds panel, which analyzed the state of the life sciences industry in the BioHealth Capital Region.

Highlighting the region’s status as a rich biopharma cluster, and the fact that the innovation sector is growing up in and around Washington, D.C., Rosendale identified the key to help bolster life sciences manufacturing in the region – sustaining life sciences business spending in Maryland. Evidence has demonstrated that incentives are a core component of supporting business research and development, and sustained spending has helped support high-tech business formation and high-wage job creation. It has also generated job creation in occupations that require fewer credentials, such as non-tech-intensive industries.

Growth in these areas can be bolstered by Maryland’s existing life sciences landscape, which ranks as one of the strongest and most robust in the nation. It contributes more than 54,000 high paying jobs in the state with over 2,700 life science firms and more than 500 biotech firms, according to a recent Milken Institute report.

“The District of Columbia has a robust pipeline of up-and-coming talent to further develop the life sciences industry in the region. The potential this expertise offers is a key focus of the new Children’s National Research & Innovation Campus, to drives discoveries that save and improve the lives of children,” says Eskandanian. “Children’s National has combined our strengths with those of public and private partners, including industry, universities, federal agencies, start-up companies and academic medical centers, to provide a rich environment to help bolster pediatric innovation and commercialization to further advance the life sciences industry.”

Data from Genetic Engineering & Biotechnology News (GEN) continues to show that Maryland and the greater BioHealth Capital Region remains one of the country’s premier hubs for innovation companies. The rankings are based on five criteria including NIH funding, VC funding, patents, lab space and jobs. In 2021, the BioHealth Capital Region held its top-four ranking and is expected to rise to a top-three cluster by 2023. The current top-three clusters ranked in the United States are Boston/Cambridge, MA (1^st), the San Francisco Bay Area (2^nd) and New York/New Jersey (3^rd).

Children’s National receives LGBTQ+ Healthcare Equality Leader designation

March 28, 2022

Children’s National Hospital is one of 496 health care providers nationwide earning coveted “LGBTQ+ Healthcare Equality Leader” designation.

Children’s National Hospital has received a perfect score of 100 and the designation of “LGBTQ+ Healthcare Equality Leader” in the Human Rights Campaign Foundation’s 15th anniversary edition of the Healthcare Equality Index (HEI). This is the nation’s foremost benchmarking survey of health care facilities on policies and practices dedicated to the equitable treatment and inclusion of their LGBTQ+ patients, visitors and employees.

“Our hospital has been on its own journey toward greater diversity, equity and inclusion (DEI) since the early 2000s,” says Denice Cora-Bramble, M.D., M.B.A., chief diversity officer at Children’s National. “Over the past two years we have expanded the scope of our DEI work, which included assessing and improving the care to LGBTQ+ patients as well as the work environment for LGBTQ+ staff. Our improvement efforts are being recognized by this designation.”

The HEI evaluates and scores health care facilities on detailed criteria falling under four central pillars:

Foundational Policies and Training in LGBTQ+ Patient-Centered Care
LGBTQ+ Patient Services and Support
Employee Benefits and Policies
Patient and Community Engagement

“Every person deserves to have access to quality health care, be respected and heard by their doctor and feel safe in the facility where they are receiving care. But LGBTQ+ people are often subject to discrimination in all spaces, including health care facilities, which leads to members of the community avoiding care and anticipating our voices will not be respected in an incredibly vulnerable environment,” said Tari Hanneman, director of Health & Aging at The Human Rights Campaign. “The HEI, at its core, strives to ensure LGBTQ+ people are protected and affirmed by their health care providers and feel safe seeking services. Our active participants are truly pioneering the health care industry by implementing robust, comprehensive LGBTQ+ inclusive policies that hopefully, because of their work, will become standard practice.”

In addition to active survey participants, the Human Rights Campaign Foundation proactively researched the key policies for LGBTQ+ inclusion at over 1,300 non-participating hospitals. Those hospitals were much less likely to have LGBTQ+ non-discrimination policies in place, which is a stark contrast to the near-perfect adoption by active participants. Among the researched hospitals, in which the Human Rights Campaign Foundation was able to find or obtain enumerated patient non-discrimination policies, only 70% have policies that include both sexual orientation and gender identity compared to 99% of HEI participants.

“We are designing effective, data-driven and responsive programs that will lead to a more diverse, equitable and inclusive workplace,” says George Francois, M.B.A., executive director of the Center for Cancer and Blood Disorders and chair of the Children’s National DEI Program’s LGBTQ+ sub-committee. Francois was a driving force behind the improvement efforts. “More importantly, we are determined to ensure the continued delivery of high-quality health care services to all patients.”

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

March 17, 2022

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

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

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

What this means

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

Why it matters

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

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

Children’s National Hospital leads the way

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

Read the AHA News overview of the Scientific Statement.

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

Researcher to decipher how viruses affect the developing brain with nearly $1M NIH award

March 15, 2022

Zika virus in blood with red blood cells, a virus which causes Zika fever found in Brazil and other tropical countries.

The National Institutes of Health (NIH) awarded Children’s National Hospital nearly $1M of research support toward uncovering the specific cellular response that happens inside a developing brain once it is infected with a virus, including how the neuron gets infected, and how it dies or survives. The research is expected to gather critical information that can inform prenatal neuro-precision therapies to prevent or attenuate the effects of endemic and pandemic viruses in the future.

“We need to use all of the information we have from ongoing and past pandemics to prevent tomorrow’s public health crisis,” said Youssef Kousa, MS, D.O., Ph.D., neonatal critical care neurologist and physician-scientist at Children’s National. “There is still here a whole lot to learn and discover. We could eventually — and this is the vision that’s inspiring us — prevent neurodevelopmental disorders before a baby is born by understanding more about the interaction between the virus, mother, fetus, and environment, among other factors.”

Different viruses, including HIV, CMV, Zika and rubella, injure the developing brain in very similar ways. This line of work was fostered first by the clinical research team led by Adre du Plessis, M.B.Ch.B., and Sarah Mulkey, M.D., supported by Catherine Limperopoulos, Ph.D., chief and director of the Developing Brain Institute at Children’s National.

The clinical research findings then led to the NIH support, which then inspired more basic science research. Fast forward to now, Kousa will study how Zika affects the human brain and extrapolate what is learned and discovered for a broader understanding of neurovirology.

The research program is supported by senior scientists and advisors, including Tarik Haydar, Ph.D., and Eric Vilain, M.D., Ph.D., both at Children’s National and Avindra Nath, M.D., at NIH, as well as other leading researchers at various U.S. centers.

“This is a team effort;” added Kousa, “I’m thankful to have a group of pioneering and seasoned researchers engaged with me throughout this process to provide invaluable guidance.”

Many viruses can harm the developing brain when they replicate in the absence of host defenses, including the gene regulatory networks responsible for the neuronal response. As a result, viral infections can lead to brain injury and neurodevelopmental delays and disorders such as intellectual disability, seizures that are difficult to treat, and vision or hearing loss.

The big picture

Youssef Kousa, MS, D.O., Ph.D., neonatal critical care neurologist and physician-scientist at Children’s National.

The translational research supported by NIH with this award synergistically complements nationally recognized clinical research programs and ongoing prospective cohort studies at Children’s National to identify the full spectrum of neurodevelopmental clinical outcomes after prenatal Zika and other viral infections led by Dr. Mulkey and Roberta DeBiasi, M.D., M.S..

The award also builds upon strengths at the Children’s National Research & Innovation Campus, which is in proximity to federal science agencies. Children’s National experts from the Center for Genetic Medicine Research, known for pediatric genomic and precision medicine, joined forces with the Center of Neuroscience Research and the NIH-NINDS intramural research program to focus on examining prenatal and childhood neurological disorders.

Kousa received this competitive career development award from the National Institute of Neurological Disorders and Stroke of the National Institutes of Health under Award Number K08NS119882. The research content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

The hold-up in the field

Many neurodevelopmental disorders are caused by endemic viruses, such as CMV, and by viral pandemics, including rubella as seen in the 1960s and Zika since 2015. By studying Zika and other prenatal viral infections, Kousa and team hope to gain deeper biological understanding of the viral effects toward developing therapies for anticipating, treating and preventing virally induced prenatal brain injury in the long-term future.

To date, little is known about how viruses affect developing neurons and, as a result, prenatal brain injury is not yet treatable. To bridge the gap towards prenatal neuro-precision therapies, the research explores how genes regulate neuronal developmental and viral clearance by innovatively integrating three systems:

Cerebral organoids, which illuminate how a neuron reacts to a viral infection
Pre-clinical models that link prenatal brain injury to postnatal neurodevelopmental outcomes
Populational genomics to identify human genetic risk or protective factors for prenatal brain injury

Given the scope and complexity of this issue, the international Zika Genetics Consortium, founded in 2015 by Kousa and a team of leading investigators across the world, provides critical samples and resources for the third arm of the research by performing comprehensive genomic analyses using sequencing data collected from diverse human populations throughout Central and South America, which are not as heavily sequenced as Western populations. Through partnerships with the Centers for Disease Control and Prevention and NIH, the consortium’s database and biorepository houses thousands of patient records and biospecimens for research studies to better understand how viruses affect the developing human brain.

“It is inspiring to imagine that, in the longer term, we could recognize early on the level of brain-injury risk faced by a developing fetus and have the tools to mitigate ensuing complications,” said Kousa. “What is driving this research is the vision that one day, brain injury could be prevented from happening before a baby is born.”

Social risk interventions significantly reduce asthma-related ED visits

March 4, 2022

In a systematic review and meta-analysis published in JAMA Pediatrics, researchers at Children’s National Hospital found that interventions focused on health, environment and community were associated with the greatest reduction in asthma-related emergency department visits and hospitalizations among children.

In a systematic review and meta-analysis published in JAMA Pediatrics, researchers at Children’s National Hospital found that interventions focused on health, environment and community were associated with the greatest reduction in asthma-related emergency department visits and hospitalizations among children. The findings suggest that addressing social risks may be a crucial component of pediatric asthma care to improve health outcomes.

“There are persistent and striking disparities in asthma outcomes among children,” said Jordan Tyris, M.D., hospitalist fellow at Children’s National and lead author. “Understanding how to address these is of utmost importance.”

“Literature on the spectrum of social factors, including social needs, social risks and social determinants, has increased recently across many aspects of health care,” adds Dr. Tyris. “But much of this literature has focused on adults with chronic conditions, for example diabetes or high blood pressure.”

The study authors searched PubMed, Scopus, PsychINFO, SocINDEX and CINAHL from January 2008 to June 2021 for U.S.-based studies evaluating the associations of interventions addressing one or more social risks with asthma-related emergency department visits and hospitalizations among children. The systematic review included 38 of the original 641 identified articles (6%), and the meta-analysis included 19 articles (3%). Overall, participation in social risk–based interventions, particularly those that addressed health literacy, home environmental conditions and peer support were associated with significantly reduced risks for asthma-related emergency department visits and hospitalizations among children.

You can read the full study, “Social Risk Interventions and Health Care Utilization for Pediatric Asthma: A Systematic Review and Meta-analysis” in JAMA Pediatrics.