Public Health

kids running to school

New School-Friendly Health Systems Framework supports children’s health and learning

kids running to schoolWith school back in session and students, educators and health care workers still recovering from the COVID-19 pandemic, it is critical for the health and education sectors to align efforts and drive innovative programs and policy change efforts to address the needs of children.

With input from expert stakeholders, including pediatricians, educators, school administrators, school nurses, parents and community health experts, Children’s National led the development of a School-Friendly Health System (SFHS) framework that is based on lessons learned from nationally recognized baby-friendly hospitals and age-friendly health systems initiatives. Becoming school-friendly means that hospitals and health systems have made a commitment to actively help children achieve optimal health and reach their full academic potential.

The big picture

Given the fundamental interrelation of health and education, hospitals and health care organizations need a holistic framework to guide their school partnerships and other efforts to make a positive impact on patient and family engagement, health care costs, health outcomes and, ideally, patients’ learning experience. For hospitals and health systems, partnering with schools in their local service area will not only improve service delivery to the students receiving care from the health system but will help strengthen health outcomes and overall student performance.

Children’s National Hospital leads the way

Eight health care organizations and partners, led by Desiree de la Torre, M.P.H., M.B.A., Julia DeAngelo, M.P.H., and Danielle Dooley, M.D., M.Phil., of the Child Health Advocacy Institute (CHAI) through executive support of Tonya Kinlow, M.P.A., and Nathaniel Beers, M.D., M.P.A., at Children’s National and with support from RESOLVE (a Washington, D.C., -based nonprofit based specializing in collaborative process design), have formed to update the SFHS framework that was first released in 2021. The Collaborative includes interdisciplinary health care professionals who have been leading and coordinating early childhood and K-12 school health initiatives across the nation.

The SFHS framework includes five principles that reflect a broad range of competencies, practices, and policy positions that health care and school health experts and stakeholders view as emblematic of a SFHS – some already practiced, some aspirational:

School friendly health sysems infographic

A few significant updates to the framework include embedding the “All Touchpoints Approach” principle across the framework and adding an “Alignment” principle. Other changes included modifying the wording of the Accessibility and Accountability principles and adding a few new practices.

What’s next

The Collaborative is now focusing on developing tools to support implementation. Through securing additional funding, the Collaborative will evaluate how the adoption of the SFHS framework across diverse settings has strengthened the healthcare sector’s relationships with schools, students and families and ability to improve health and academic outcomes.

Read the full study at childrensnational.org/school-friendly For more information on the SFHS Learning Collaborative, contact School Partnerships@childrensnational.org.

Marius Linguraru, D.Phil., M.A., M.Sc., a co-principal investigator for the project, presents

Children’s National joins team to use AI to expand health knowledge in Kenya

Marius Linguraru, D.Phil., M.A., M.Sc., a co-principal investigator for the project, presentsChildren’s National Hospital is joining a team of global health researchers to use large language models (LLMs) like ChatGPT to help Kenyan youth learn about their health and adopt lifestyles that may prevent cancer, diabetes and other non-communicable diseases.

The work, which is one of nearly 50 Grand Challenges Catalyzing Equitable Artificial Intelligence (AI) Use grants announced by the Bill & Melinda Gates Foundation, will harness the emerging power of AI to empower young people with information that they can carry through adulthood to reduce rates of unhealthy behaviors including physical inactivity, unhealthy diet and use of tobacco and alcohol.

“We are thrilled to be part of this effort to bring our AI expertise closer to young patients who would benefit dramatically from technology and health information,” said Marius George Linguraru, D.Phil., M.A., M.Sc., a co-principal investigator for the project, the Connor Family Professor in Research and Innovation at Children’s National and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation. “Using generative AI, we will build an application to enhance the knowledge, attitudes and healthy habits of Kenyan youth and use this as a foundation to improve health inequities around the globe.”

Why it matters

A lower middle-income country located on the east coast of Sub-Saharan Africa, Kenya is home to 50 million people and one of the continent’s fastest-growing economies. English is one of Kenya’s official languages, and the country has been recognized as a technology leader in Africa, with 82% of Kenyans having phone connectivity. Taken together, these factors make the country an ideal location to deploy an LLM-based platform designed to improve health information and attitudes.

The Gates Foundation selected this project from more than 1,300 grant applications. The nearly 50 funded projects are aimed at supporting low- and middle-income countries to harness the power of AI for good and help countries participate in the AI development process. The project’s findings will contribute to building an evidence base for testing LLMs that can fill wide gaps in access and equitable use of these tools. Each of the grants provides an opportunity to mitigate challenges experienced by communities, researchers and governments.

What’s next

The project development will be led by the National Cancer Institute of Kenya, with Linguraru and other global experts advising the effort from Kenyan institutions and Stanford University. Researchers plan to enroll youth from universities, shopping malls, markets, sporting events and other high-traffic locations. The study will look at participants’ risk factors and how their attitudes toward healthier lifestyles changed after engaging with the new LLM platform.

“The team is thrilled to be selected as one of the nearly 50 most promising AI proposals in the Gates Foundation Grand Challenge competition, and we look forward to seeing how our work can benefit the health of Kenyan youth,” said Dr. Martin Mwangi, principal investigator for the project and head of the Cancer Prevention and Control Directorate at the National Cancer Institute of Kenya. “If successful, we hope to share this model and the expertise we gain to expand health equity and knowledge to other regions.”

girl being examined by doctor

Pediatric hospitals underutilize systems to get at social challenges impacting health

 

girl being examined by doctor

Physicians treating hospitalized children rarely use a coding system established in 2015 for flagging social challenges and stressors that may be impacting patient health, according to new research from Children’s National Hospital.

 

Physicians treating hospitalized children rarely use a coding system established in 2015 for flagging social challenges and stressors that may be impacting patient health, according to new research from Children’s National Hospital published in Pediatrics. Known as social determinants of health (SDOH), these factors include food insecurity, homelessness and adverse childhood events like substance abuse at home, and they can greatly affect a child’s well-being.

“We only get so many touchpoints with our patients,” said Kaitlyn McQuistion, M.D., pediatric hospital medicine fellow at Children’s National and co-lead author of the paper. “Our research shows the screening itself provides valuable insight into our patients, making identification an important part of inpatient and post-discharge care. With this information, doctors can help families tap into social workers, community supports and other resources aimed at providing a more holistic approach to child health.”

The big picture

The American Academy of Pediatrics advises screening for unmet social needs and using the codes laid out in the International Classification of Disease, 10th Revision (ICD-10), to flag risk factors for some of a hospital’s most vulnerable patients. In 2018, the American Hospital Association (AHA) clarified that the coding can be added by any healthcare professional accessing the chart, including nurses, social workers, case managers and physicians. The study looked at data from 4,000 hospitals in 48 states and the District of Columbia and found that use of the codes has remained low, even with the AHA’s clarification.

In practice, physicians know that screening and documentation are the essential first steps to help families find resources. Yet less than 2% of pediatric inpatients were coded as needing support. Most commonly, “problems related to upbringing” – a broad category indicating social needs and adverse childhood events – was used.

What’s ahead

Some providers are using these SDOH codes, or Z Codes, more often to address and improve health disparities. “Our mental health colleagues and those working with the Native American population, in particular, are using these tools more often to capture and disseminate critical information related to their patients’ social needs,” said Stacey Stokes, M.D., a hospitalist at Children’s National and co-lead author on the paper.  “Their innovative approaches to address and improve health disparities may provide learning opportunities for institutions.”

The researchers said that more work needs to be done to take these successes to other populations, find ways to incentivize this work in billing and ensure that providers have community resources to address the needs that they uncover.

“The ultimate goal of this work is to identify patients with social needs affecting their health and connect them with resources,” said Kavita Parikh, M.D., director for the Research Division of Hospital Medicine. “There are many avenues to explore to find ways to better utilize this tool, including language learning models, improved training and stronger community resources.”

smiling young family

Survey: Teen parents with support report greater well-being

smiling young family

Teen parents in Washington, D.C., reported better physical and mental health when they had more social support, a community and people who understood their problems.

Teen parents in Washington, D.C., reported better physical and mental health when they had more social support, a community and people who understood their problems, according to a survey conducted as part of a novel city-wide program to bring together resources for young families.

“This should come as no surprise. Young parents say they do better – and their children do better – when they have the material and social supports they need,” said Yael Smiley, M.D., a pediatrician at Children’s National Hospital and lead author of research in BMC Public Health. “This work is the start of a roadmap to help young parents thrive.”

The big picture

Each year in D.C., 300 babies are born to teens, and the rate of births among teens ages 15 to 17 is higher than the national average. Dr. Smiley is a leader in a federally funded coalition run by D.C. Primary Care Association (DCPCA) to help support young parents called the D.C. Network for Expectant and Parenting Teens (DC NEXT!). This city-wide network brings together community partners to support teen mothers, fathers and their children.

A key feature of the program is its context team, a group of teen parents who provide insights into their experiences as young parents. They advise about research and support for their peers, and they also build a community and develop a platform to speak up about their issues. The context team recruited other parents to complete a well-being survey in 2022, which will guide programming and resources for families. An added benefit for the parents who participate in the context team: The survey data showed that a key facet of well-being for the young parents was feeling “like you were part of a group of friends or community” and having “someone who understood your problems.”

“The context team provides the type of community that the research shows is essential to the well-being of teen parents,” said Dr. Smiley. “Not only are the context team members helping us understand what teen parents need, but they are also helping each other thrive as young parents.”

The fine print

Among the findings of the survey of 107 teen parents in D.C.:

  • The most used resources were supplemental food programs – with 35% receiving Supplemental Nutrition Assistance Program (SNAP) benefits, 24% receiving support from the Special Supplemental Nutrition Program for Women, Infants and Children (WIC) and 18% accessing food pantries.
  • Also popular were Temporary Assistance for Needy Families (TANF), housing assistance and childcare.
  • Surprisingly, the largest group of respondents, 37%, reported receiving no resources.

Having higher social support was correlated with higher ratings for physical health, mental health and well-being. “With this research, we have even more evidence underscoring the need to connect young parents with programs and resources that will improve their well-being,” Smiley said. “I can’t wait to see what the leaders on the context team and in the DC NEXT! program do in the next phases of our work.”

U.S. News Badges

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

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

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

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

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

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

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

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

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

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

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

mother and baby doing a telehealth call

Using telehealth to study babies born to mothers infected with SARS-CoV-2

mother and baby doing a telehealth call

Continued advancements in telehealth methods to follow child neurodevelopment will help ensure robust child follow-up and inclusion of diverse cohorts.

Multiple studies have shown that SARS-CoV-2 infection can impact pregnant mothers and their fetuses but more research is needed to understand the long-term impact on the neurodevelopment of these children as they get older. Child neurodevelopmental evaluations are typically performed in-person. Since the COVID-19 pandemic began, the transition to telehealth methods was needed.

Continued advancements in telehealth methods to follow child neurodevelopment will help ensure robust child follow-up and inclusion of diverse cohorts, says a commentary in JAMA Network Open.

Why it matters

Commentary author Sarah Mulkey, M.D., prenatal-neonatal neurologist at Children’s National Hospital, highlights a new study that used a novel telehealth method to look for neurodevelopmental differences in infants ages 6-12 months born to mothers with SARS-CoV-2 infection compared to nonexposed infants of the same age and found no differences in neurodevelopment among the two cohorts. The study adapted a standardized assessment to a telehealth method.

“The results of this study provide needed reassurance to the many mothers who have experienced SARS-CoV-2 infection during pregnancy,” says Dr. Mulkey.

What’s been the hold up in the field?

“Developmental assessments that rely on observation of infants’ developmental skills can naturally make the transition to a telehealth platform,” says Sarah Mulkey, M.D., prenatal-neonatal neurologist at Children’s National Hospital and commentary author. “General movement assessment is an observation-based assessment of infants that can be captured by a parent or caregiver on video, and it has been used in neurodevelopmental outcomes studies of children after antenatal SARS-CoV-2 exposure.”

Moving the field forward

Child outcomes research can have improved enrollment and continuity of participant follow-up due to the availability of remote assessments. Neurodevelopmental tools are being developed that can be used on a telehealth platform or by parent recorded videos.

Researchers from the Children’s National Congenital Zika Virus Program have also developed telehealth-based methods for child outcome research that has been utilized in international Zika outcome studies funded by the Thrasher Research Fund and the NIH.

Read the full commentary, Use of Telehealth Methods to Track Infant Neurodevelopment After In Utero SARS-CoV-2 Exposure, in JAMA Network Open.

Borrelia burgdorferi bacteria

Most children diagnosed with Lyme disease recover fully after treatment

Borrelia burgdorferi bacteria

Lyme disease is caused by the bacterium Borrelia burgdorferi.

Experts found most children diagnosed with Lyme disease recovered within six months of completing antibiotic treatment, according to a new joint study published in Pediatric Research.

The study, which was supported through a partnership between Children’s National Research Institute (CNRI) at Children’s National Hospital and the National Institute of Allergy and Infectious Diseases (NIAID), also revealed that a notably small percentage of children took longer than six months to recover and experienced a significant impact on their daily functioning.

The big picture

This research studied the long-term outcomes of children with Lyme disease through a cross-sectional evaluation using validated surveys. The study collected survey responses from the parents of 102 children ages 5 to 18 years who had been diagnosed with Lyme disease between six months and 10 years before enrollment. Adolescents ages 10 to 18 years were also invited to complete adolescent-specific questionnaires.

According to these parent survey responses, the vast majority (87%) of fully recovered patients did so

within the initial 6-month period after completing antibiotic treatment, with approximately one third recovering within the first month post-treatment and an additional third recovering 1–3 months post-treatment. However, 13% of children who ultimately made a full recovery took longer to do so. At the time of study completion, 6% of children still experienced symptoms attributed to Lyme disease but only 1% experienced symptoms significant enough to impair daily functioning.

Why we’re excited

According to the authors, this study supports previous data showing an excellent overall prognosis for children with Lyme disease, which should help alleviate understandable parental stress associated with lingering non-specific symptoms among infected children.

“These findings can help clinicians manage families’ expectations about the varying post-treatment recovery times of pediatric Lyme disease patients,” says Roberta DeBiasi, M.D., chief of Infectious Diseases at Children’s National Hospital and one of the study’s authors. “It may also give parents relief in knowing that their child has an excellent chance of full recovery after treatment.”

What’s next

Common symptoms of Lyme disease include fever, headache, fatigue and a distinct skin rash called erythema migrans. Without treatment, the infection can spread to joints, the heart and the nervous system.

Antibiotic treatment resulting in full recovery is successful in most Lyme cases. For some, however, symptoms of pain, fatigue or difficulty thinking persist or return after antibiotic treatment. Symptoms that substantially reduce levels of activity and impact quality of life for more than six months after treatment are classified as post-treatment Lyme disease (PTLD) syndrome.

PTLD syndrome remains poorly understood in children and adults, and more research is needed to better understand these prolonged symptoms and identify treatment targets, according to the authors.

You can read the full study, Pediatric Lyme disease: systematic assessment of post-treatment symptoms and quality of life, in Pediatric Research.

baby getting vaccinated

Addressing COVID-19 vaccine hesitancy for parents and caregivers

 

baby getting vaccinated

A one-hour webinar delivered as a digital intervention can reduce parental COVID-19 vaccine hesitancy and parental uncertainty regarding vaccine information.

While vaccines have been proven to be highly effective in preventing COVID-19 infections and severe illness, the goal of achieving herd immunity remains unattainable without vaccinating children. Due to the prevalence of misinformation on the internet, Lauren Arrigoni, DNP, and Claire Boogaard, M.D., M.P.H., at Children’s National Hospital led a study, published in the Journal of Pediatric Health Care, that addresses parental hesitancy about giving their children COVID-19 vaccines. They found that a one-hour webinar delivered as a digital intervention can reduce parental COVID-19 vaccine hesitancy and parental uncertainty regarding vaccine information.

The big picture

The study used a pretest-posttest design with an adapted version of the Parental Attitudes about Childhood Vaccine (PACV) survey for COVID-19 vaccines. The webinar was streamed live and later posted to YouTube with survey QR codes and pre-survey and post-survey links in the video description. PACV data from the recorded webinar were collected over 4 weeks after the original webinar date. According to Dr. Arrigoni, the webinar itself was a collaborative work of a nurse and physicians to help provide scientific-based evidence for the general population of parents and caregivers. This project’s results have important implications for practice, as the researchers suggest that educational strategies have the potential to improve vaccination rates by increasing parental confidence in the COVID-19 vaccine. Using the webinar as a digital education strategy, the researchers also designed a dedicated section to empower parents to have conversations with their peers who may be hesitant about vaccines, even if they lack a medical background.

The patient benefit

While it was not possible to directly measure vaccination rates during this project due to time constraints, the researchers did find that parents who participated in the webinar felt more empowered and informed to make the decision to vaccinate their child. By addressing common parental concerns and providing accurate information, the webinar, as well as other types of digital interventions, could play a role in improving vaccination rates and ultimately contributing to truly ending the COVID-19 pandemic.

Why we’re excited

Overall, this study indicates that a webinar can effectively engage a large audience, educate them about vaccines and encourage conversations around vaccination.

“Vaccine hesitancy outreach can even begin before the vaccine is available,” says Dr. Arrigoni. “Applying these learned lessons can lead to more vaccinated individuals and would be an important step in battling parental vaccine hesitancy now and for future pandemics and viruses.”

You can read the full study, A Webinar to Improve Parental COVID-19 Vaccine Hesitancy, in the Journal of Pediatric Health Care.

clinician measuring obese child's waist

Surgeon says treatment silos in childhood obesity need to end

clinician measuring obese child's waist

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

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

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

Why it matters

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

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

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

Read the editorial in the Lancet Child & Adolescent Health.

Gender Self-Report seeks to capture the gender spectrum for broad research applications

form with check boxes for genderA new validated self-report tool called the Gender Self-Report provides researchers a way to characterize the gender of research participants beyond their binary designated sex at birth.

The multi-dimensional Gender Self-Report, developed using a community-driven approach and then scientifically validated, is outlined in a peer-reviewed article in the American Psychologist, a journal of the American Psychological Association.

The big picture

“This is the first broadly validated tool that allows us to measure inner gender experience across a large group of people in a rigorous way that doesn’t require a person to understand specialized gender-related terms,” says John Strang, Psy.D., who directs the Gender and Autism Program at Children’s National Hospital. He co-authored the journal article about the measure and co-led the initiative to develop it. “Typical gender assessments are fixed check boxes, which is problematic for capturing gender in people who are not familiar with many of the self-descriptors, which vary in their use and meaning.”

Strang notes that even open-field gender assessments can be problematic for people who experience gender diversity but are not aware of nuanced gender-related language.

Why it matters

This new gender characterization and inclusion method will allow researchers from a broad array of fields (e.g., social sciences, medicine, education) to model their participants’ inner gender experience more equitably in research. The resulting studies will be able to provide deeper understanding of how a person’s gender can play a role in study outcomes.

Senior author and statistical lead for the project, Ji Seung Yang, Ph.D., from University of Maryland Department of Human Development and Quantitative Methodology, foresees this tool as an important addition to the research toolkit for people studying neuroimaging, genetics and any other research that requires a more accurate and detailed picture of an individual’s gender experience.

What’s unique

The Gender Self-Report tool is the first of its kind to be developed and validated by researchers together with the gender diverse and neurodiverse communities directly. These efforts align with work in many fields of clinical research to ensure that study findings reflect the insights and experiences of the people who are being studied, rather than simply capturing the researcher’s external perspective of those participants.

The tool is appropriate for use by youth (as young as 10 years of age) and adults, gender diverse and cisgender individuals, and non-autistic and autistic people. The focus on inclusivity for autistic people is in keeping with the common intersection of autism and gender diversity (i.e., 11% of gender-diverse people are estimated to be autistic).

Gregory Wallace, Ph.D., co-author of the measure and associate professor in the Department of Speech, Language & Hearing Sciences at The George Washington University, calls the tool a “game changer for any research that needs to understand specifics about how gender experience can impact health-related or developmental differences.”

The Gender Self-Report: A Multidimensional Gender Characterization Tool for Gender-Diverse and Cisgender Youth and Adults, appears in the American Psychologist.

pregnant woman on couch

The role of pediatric cardiologists in addressing maternal health disparities

pregnant woman on couch

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

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

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

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

Why it matters

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

How cardiologists can help

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

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

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

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

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

What’s next

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

Abstract Happy 2022 New Year greeting card with light bulb

The best of 2022 from Innovation District

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

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

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

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

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

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

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

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

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

5. New telehealth command center redefines hospital care

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

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

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

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

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

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

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

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

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

10. 2022: Pediatric colorectal and pelvic reconstructive surgery today

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

RFP collage of logos

Healthcare leaders join to advance pediatric innovation

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

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

Convening a coalition of healthcare leaders

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

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

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

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

Using RWE to facilitate the regulatory pathway

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

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

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

About NCC-PDI

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

Map showing CHI Registry sign-ups through December 2022

Congenital Heart Initiative celebrates global reach in first two years

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

Why it matters

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

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

The patient benefit

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

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

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

What’s unique

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

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

What’s next

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

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

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

pregnant woman

Early SARS-CoV-2 exposure may impact infant development

pregnant woman

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.

Sarah Mulkey

Exposure to Zika in utero may produce neurodevelopmental differences

Sarah Mulkey

“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.

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)

Innovations for health equity: Food pharmacy app wins Hackathon

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.

iLet Bionic Pancreas

Multicenter trial finds bionic pancreas improves Type 1 diabetes management

iLet Bionic Pancreas

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.

Paper cutouts of silhouette

Successful autism and ADHD tools go digital

Paper cutouts of silhouette

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.